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... In previous studies, the OVAMA collaborative developed a core domain set (CDS) for evaluating treatment in vascular malformations ( Figure 1). 8,9 A CDS is a set of outcome domains that should be measured at the minimum when evaluating treatment effect in a certain health condition. 10 The next step towards homogeneity in outcome use and reporting was determining how to measure these core domains. ...
... Concepts of interest were identified in previous studies. 8,9,16 At first, the literature was searched extensively to determine all outcome domains measured in research on peripheral vascular malformations. 16 Based on these outcome domains, via an international e-Delphi study and two consensus meetings, a CDS was developed wherein outcome domains were defined ( Figure 1). ...
... 16 Based on these outcome domains, via an international e-Delphi study and two consensus meetings, a CDS was developed wherein outcome domains were defined ( Figure 1). 8,9 In total, 167 physicians and 134 patients/parents of younger patients participated to ensure inclusion of the patient's perspective. ...
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Summary Background The symptoms and appearance of vascular malformations can severely harm a patient’s quality of life. The aim of treatment of vascular malformations generally is to improve condition‐specific symptoms and/or appearance. Therefore, it is highly important to start testing treatment effects in clinical studies from the patient’s perspective. Objectives The objective of this study was to develop a patient‐reported outcome measure (PROM) for measuring symptoms and appearance in patients with vascular malformations. Methods A first draft of the PROM was based on the previously internationally developed core outcome set. The qualitative part of this study involved interviews with 14 patients, which led to a second draft. The second draft was field‐tested cross‐sectionally, after which groups of items were evaluated for adequate internal consistency (Cronbach’s alpha >0.7) to form composite scores. Construct validity was evaluated by testing 13 predefined hypotheses on known‐group differences. Results The patient interviews ensured adequate content validity and resulted in a general symptom scale with 6 items, head/neck symptom scale with 8 items and an appearance scale with 9 items. Cronbach’s alpha was adequate for two composite scores: a general symptom score (0.88) and an appearance score (0.85). Ten out of 13 hypotheses on known‐group differences were confirmed, confirming adequate construct validity. Conclusions With the development of the OVAMA questionnaire, outcomes of patients with vascular malformations can now be evaluated from the patients’ perspective. This may help improve the development of evidence‐based treatments and the overall care for patients with vascular malformations.
... Vascular malformations are rare congenital vascular anomalies, which grow proportionally with age, and can be of venous, lymphatic, arteriovenous, capillary or combined origin. 1 Patients with peripheral vascular malformations commonly experience disfigurement, pain, bleeding, impaired mobility, growth disturbances and bleeding and thrombotic complications. [1][2][3][4][5] Patients have overall poorer quality of life (QoL) than the general population. 6 Some lesions can be life-threatening, but most patients seek treatment to improve aspects of their QoL. ...
... QoL and other patient-reported outcomes (PROs) were therefore recently included in the core domain set (CDS) for clinical research in vascular malformations (excluding capillary). 3 A CDS is a minimum set of outcome domains that should be measured when evaluating treatment outcomes in a certain health condition. 7 The CDS results came from an international consensus project, the Outcome Measures for Vascular Malformations (OVAMA) project, 3,8 which aims at uniform outcome reporting by determining what and how to measure. ...
... 3 A CDS is a minimum set of outcome domains that should be measured when evaluating treatment outcomes in a certain health condition. 7 The CDS results came from an international consensus project, the Outcome Measures for Vascular Malformations (OVAMA) project, 3,8 which aims at uniform outcome reporting by determining what and how to measure. ...
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Introduction: The OVAMA (Outcome measures for vascular malformations) project determined quality of life (QoL) as a core outcome domain for patients with vascular malformations. In order to measure how current therapeutic strategies alter QoL in these patients, a patient-reported outcome measurement (PROM) responsive to changes in QoL is required. We therefore assessed the responsiveness of two widely used generic QoL PROMs, the Medical Outcomes Study Short Form 36 (SF-36) and Skindex-29, in adult patients with vascular malformations. Methods: In an international multicentre prospective study, treated and untreated patients completed the SF-36 and Skindex-29 at baseline and after a follow-up period of 6-8 weeks. Global Rating of Change (GRC) scales assessing various QoL-related outcome domains were additionally completed. Per subscale, responsiveness was assessed using two methods: by testing hypotheses on expected correlation strength between change scores of the questionnaires and the GRC scales, and by calculating the area under the receiver operating characteristics curve (AUC). The questionnaires were considered responsive if ≥75% of the hypotheses were confirmed or if the AUC was ≥0.7. Results: Eighty-nine participants were recruited in three centres in the Netherlands and United States, of which 67 completed all baseline and follow-up questionnaires. For all subscales of the SF-36 and Skindex-29, less than 75% of the hypotheses were confirmed and the AUC was <0.7. Discussion: Our findings suggest that the SF-36 and Skindex-29 seemed unresponsive to change in QoL. This suggests that alternative PROMs are needed to measure - and ultimately improve - QoL in patients with vascular malformations.
... In a previous study, we developed a core domain set (CDS) for peripheral vascular malformations, excluding capillary malformations. 4 A CDS is a minimum set of outcome domains that should be measured when evaluating treatment outcomes in health conditions. 5 This international consensus project, involving 167 physician and 134 patient/ parent contributors, consisted of a three-round e-Delphi study and an online consensus meeting. ...
... For some domains consensus was not achieved, specifically 'recurrence', 'appearance', 'radiological imaging' and 'lymphatic fluid leakage'. 4 A face-to-face consensus meeting was organized to establish the final CDS. ...
... The meeting was chaired by the then coordinator of the OVAMA project, and was held at the International Society for the Study of Vascular Anomalies (ISSVA) conference (28 May to 1 June 2018) in Amsterdam, the Netherlands. All previous study participants (n = 301) 4 were invited to join. Participants included 26 experts of the OVAMA Consensus Group; 85% represented various medical specialities (surgery, otolaryngology, paediatrics, paediatric haematology/oncology, radiology and dermatology) and 15% were patient organization representatives. ...
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There is a variety of outcome reporting in the clinical research on peripheral vascular malformations including capillary, venous, lymphatic, arteriovenous and combined malformations. Without harmonization of outcome measures, treatments cannot be properly compared. This hampers the development of evidence‐based treatment guidelines, urgently needed for these challenging congenital conditions. The mission of the Outcome measures for VAscular MAlformations (OVAMA) project is to uniform outcome reporting in clinical research. This article is protected by copyright. All rights reserved.
... 23 Over the last few years, a rise in dermatological COSs has become evident, for example in peripheral vascular malformations, congenital melanocytic naevi, and vitiligo. [24][25][26] Moreover, a dermatology-specific framework was recently developed to support COS developers in this field. 27 The Core Outcome Set for Capillary Malformations (COS-CAM) project was initiated, as currently no COS exists for CMs. ...
... Yet, the number of patients during our consensus meeting is similar to that of other COS development projects. 25,39 We believe that, as long as no decision to include or exclude an outcome subdomain was overturned by the small patient cohort, it is inconsequential. Furthermore, a clear predominance of patients with SWS was evident during both the e-Delphi rounds and the consensus meeting, which could have biased the results. ...
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Background: There is limited evidence on the best available treatment options for capillary malformations (CMs), mainly due to the absence of uniform outcome measures in trials on therapies. A Core Outcome Set (COS) enables standard reporting of trial outcomes, which facilitates comparison of treatment results. Objectives: To develop a core outcome domain set (CDS), as part of a core outcome set (COS), for clinical research on CMs. Methods: Sixty-seven potentially relevant outcome subdomains were recognized based on the literature, focus group sessions, and input from the COSCAM working group. These outcome subdomains were presented in an online Delphi study to CM experts (medical specialists and authors of relevant literature) and (parents of) CM patients (international patient associations). During three e-Delphi study rounds, the participants repeatedly scored the importance of these outcome subdomains on a 7-point Likert scale. Participants could also propose other relevant outcome subdomains. Consensus was defined as ≥80% agreement as to the importance of an outcome subdomain amongst both stakeholder groups. The CDS was finalized during an online consensus meeting. Results: A total of 269 participants from 45 countries participated in the first e-Delphi study round. Of these, 106 were CM experts from 32 countries, counting predominantly dermatologists (59%) and plastic surgeons (18%). Moreover, 163 (parents of) CM patients from 28 countries participated, of whom 58 percent had Sturge-Weber syndrome (SWS). During the two subsequent e-Delphi study rounds, 189 and 148 participants participated, respectively. After the entire consensus process, consensus was reached on 11 outcome subdomains: colour/redness, thickness, noticeability, distortion of anatomical structures, glaucoma, overall health-related quality of life, emotional functioning, social functioning, tolerability of treatment, patient satisfaction with treatment results and recurrence. Conclusion: We recommend the CDS to be used as a minimum reporting standard in all future CM therapy trials. Our next step will be to select suitable outcome measurement instruments to score the core outcome subdomains.
... Such exercises, with finetuning of the outcomes during the process of reaching consensus on the instruments, have been followed before. [19][20][21] We defined a 'domain' as an aspect of disease that should be measured, such as cognitive functioning, 22 whereas an 'outcome' describes a concept or construct that is a part of a domain, such as learning difficulties or memory lapse for the domain 'cognitive functioning'. 23 Specific objectives were: (i) to identify domains and outcomes that have been reported in the literature and are considered important by patient representatives (i.e. ...
... 35 Several COS studies on skin diseases set examples of outcome classification. 20,34,36,37 We chose to enrich the COMET domain classification by adding the classification from the World Health Organization ICF for skin anatomy and functions. Domains that were classified as 'functioning' in the COMET taxonomy were grouped into 'quality of life' during our consensus meeting. ...
Article
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Background: Congenital melanocytic naevi (CMN) can have a great impact on patients' lives due to perceived stigmatization and the risk of melanoma development and neurological complications. Development of a core outcome set for care and research of CMN will allow standard reporting of outcomes. This will enable comparison of outcomes, allowing professionals to offer advice about best management options. In previous research, stakeholders (patients, parents, and professionals) reached consensus on the core domains of the core outcome set. To select the appropriate measurement instruments, the domains should be specified by outcomes. Objectives: To reach consensus on the specific core outcomes describing the core domains pertaining to both CMN clinical care and research. Methods: A list of provisional outcomes, obtained earlier, was critically reviewed by the OCOMEN research team and by relevant stakeholders through an online questionnaire, to refine this list and provide clear definitions for every outcome. When needed, discussion with individual participants was undertaken over the telephone or by e-mail. During an online consensus meeting, stakeholders discussed the inclusion of potential outcomes. After the meeting, participants voted for inclusion of outcomes in two rounds. Results: Forty-four stakeholders from nineteen countries participated in the study. Nine core outcomes were included in the core outcome set relative to clinical care and ten core outcomes for research. Conclusion: These core outcomes will enable standard reporting in future care and research of CMN. This study facilitates the next step of core outcome set development: selecting the appropriate measurement instruments for every outcome.
... 10,11 As extra-cranial AVM is rare, large series are limited. 7,8,12 There are several Korean series, one with 29 patients with AVM of the foot, 13 one with 66 patients with AVM of the trunk and extremities, 14 and one with 31 patients with AVM of the hand. 15 In United States and European cohorts, 14 to 30 patients with extracranial AVM have been described. ...
Article
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Objectives: Arteriovenous malformations of the lower extremities (AVMLE) can present as simple or complex combined or syndromic forms (e.g. Parkes Weber Syndrome). We aimed to characterize the differences in clinical presentation and natural history of these potentially life and limb threatening congenital vascular malformations. Methods: We conducted a retrospective analysis of a consecutive series of patients with AVMLE, who presented to a tertiary referral center in Switzerland between 2008 and 2018. Clinical baseline characteristics, D-dimer level and course were summarized and differences between simple, non-syndromic and combined or syndromic AVMLE determined. Odds ratios (OR) and 95% confidence intervals (CI) were estimated using logistic regression models. Results: Overall, 506 patients were prospectively enrolled in the Bernese Congenital Vascular Malformation Registry, 31 (6%) with AVMLE. There were 16 women and 15 men with a mean age of 18 years at first diagnosis (1 month - 72 years). Simple AVMLE was present in 22 (71%), combined or syndromic AVMLE with limb overgrowth in 9 patients (29%), respectively. Common symptoms and signs were pain 25 (81%), swelling 21 (68%) and soft tissue hypertrophy 13 (42%). Among combined or syndromic patients, 3 patients died from wound infection with sepsis or disseminated intravascular coagulation with bleeding complications (intracranial hemorrhage and bleeding from extensive leg ulcers). Combined or syndromic patients presented more often with bleeding (67% vs. 5%; p<0.001), malformation related infection (44% vs. 5%; p=0,017) and leg length difference (56% vs. 14%; p=0.049). D-dimer levels were elevated (mean 17256 μg/L, range 1557 μg/L to 80000 μg/L) and angiographic appearance showed complex, mixed type of AVMs, including interstitial type IV, in all patients with combined or syndromic AVMLE. Conclusion: Patients with congenital simple AVMLE most often present with benign clinical features and rarely complications related to hemodynamic changes. Patients with combined or syndromic AVMLE often face serious outcomesdominated by complications other than direct high flow related heart failure.
... 6 Prior consensus for trials on vascular malformations had identified the following core domains: radiological assessment, physician-reported location-specific signs, patient-reported severity of symptoms, pain, quality of life, satisfaction and adverse events. 7 In the absence of a measure addressing the core domains of symptom severity and appearance, the authors undertook development of such a patient-reported outcome measure for interventional clinical trials in peripheral vascular malformations. 1 As we enter a new era of formal clinical trials of innovative treatments uncovered by careful iterative research, let us hope that the same care and attention will also be applied by trialists to selecting the outcome measures that matter most: to patients, clinicians and other relevant stakeholderscore outcome sets. ...
Article
Linked Article: Lokhorst et al. Br J Dermatol 2021; 185:797–803.
... As a result, patient-reported outcome assessments such as quality-of-life questionnaires and pain assessment by visual analog scale may be the best tools to measure efficacy, but these may be confounded effects due to many patients experiencing symptoms since birth [54]. There is an unmet need for validated patientreported outcome tools that are specifically designed to assess quality of life in these populations; quality of life has been identified as a core outcome domain by the ongoing Outcome Measures for VAscular MAlformations (OVAMA) project [60][61][62][63][64]. ...
Article
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Background PIK3CA -related disorders include vascular malformations and overgrowth of various tissues that are caused by postzygotic, somatic variants in the gene encoding phosphatidylinositol-3-kinase (PI3K) catalytic subunit alpha. These mutations result in activation of the PI3K/AKT/mTOR signaling pathway. The goals of this review are to provide education on the underlying mechanism of disease for this group of rare conditions and to summarize recent advancements in the understanding of, as well as current and emerging treatment options for PIK3CA -related disorders. Main body PIK3CA -related disorders include PIK3CA -related overgrowth spectrum (PROS), PIK3CA -related vascular malformations, and PIK3CA -related nonvascular lesions. Somatic activating mutations (predominantly in hotspots in the helical and kinase domains of PIK3CA, but also in other domains), lead to hyperactivation of the PI3K signaling pathway, which results in abnormal tissue growth. Diagnosis is complicated by the variability and overlap in phenotypes associated with PIK3CA -related disorders and should be performed by clinicians with the required expertise along with coordinated care from a multidisciplinary team. Although tissue mosaicism presents challenges for confirmation of PIK3CA mutations, next-generation sequencing and tissue selection have improved detection. Clinical improvement, radiological response, and patient-reported outcomes are typically used to assess treatment response in clinical studies of patients with PIK3CA -related disorders, but objective assessment of treatment response is difficult using imaging (due to the heterogeneous nature of these disorders, superimposed upon patient growth and development). Despite their limitations, patient-reported outcome tools may be best suited to gauge patient improvement. New therapeutic options are needed to provide an alternative or supplement to standard approaches such as surgery and sclerotherapy. Currently, there are no systemic agents that have regulatory approval for these disorders, but the mTOR inhibitor sirolimus has been used for several years in clinical trials and off label to address symptoms. There are also other agents under investigation for PIK3CA -related disorders that act as inhibitors to target different components of the PI3K signaling pathway including AKT (miransertib) and PI3K alpha (alpelisib). Conclusion Management of patients with PIK3CA -related disorders requires a multidisciplinary approach. Further results from ongoing clinical studies of agents targeting the PI3K pathway are highly anticipated.
... Although this evidence is informative and supportive for the scales chosen in the current study, ultimately the 9-point scale was chosen because it is recommended by the Grading of Recommendations Assessment, Development and Evaluation (GRADE) working group to assess the importance of evidence and is the scale used in DelphiManager, a web-based system designed by the COMET Initiative for facilitating and managing Delphi surveys [2,26,27]. The 5-point rating scale was arbitrarily chosen as the comparator scale because it had been used previously in COS development Delphi studies [28][29][30], and because 5-point rating scales are relatively common across research studies that include a rating scale. ...
Article
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Background As the development of core outcome sets (COS) increases, guidance for developing and reporting high-quality COS continues to evolve; however, a number of methodological uncertainties still remain. The objectives of this study were: (1) to explore the impact of including patient interviews in developing a COS, (2) to examine the impact of using a 5-point versus a 9-point rating scale during Delphi consensus methods on outcome selection and (3) to inform and contribute to COS development methodology by advancing the evidence base on COS development techniques. Methods Semi-structured patient interviews and a nested randomised controlled parallel group trial as part of the Pelvic Girdle Pain Core Outcome Set project (PGP-COS). Patient interviews, as an adjunct to a systematic review of outcomes reported in previous studies, were undertaken to identify preliminary outcomes for including in a Delphi consensus survey. In the Delphi survey, participants were randomised (1:1) to a 5-point or 9-point rating scale for rating the importance of the list of preliminary outcomes. Results Four of the eight patient interview derived outcomes were included in the preliminary COS, however, none of these outcomes were included in the final PGP-COS. The 5-point rating scale resulted in twice as many outcomes reaching consensus after the 3-round Delphi survey compared to the 9-point scale. Consensus on all five outcomes included in the final PGP-COS was achieved by participants allocated the 5-point rating scale, whereas consensus on four of these was achieved by those using the 9-point scale. Conclusions Using patient interviews to identify preliminary outcomes as an adjunct to conducting a systematic review of outcomes measured in the literature did not appear to influence outcome selection in developing the COS in this study. The use of different rating scales in a Delphi survey, however, did appear to impact on outcome selection. The 5-point scale demonstrated greater congruency than the 9-point scale with the outcomes included in the final PGP-COS. Future research to substantiate our findings and to explore the impact of other rating scales on outcome selection during COS development, however, is warranted.
... Apart from the cosmetic problems, patients commonly experience pain, bleeding, functional impairment and thrombotic complications. [1][2][3][4][5] Symptoms and complications vary depending on the type, extent and localization of the lesion. ...
Article
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Background The OVAMA (Outcome Measures for VAscular MAlformations) project determined quality of life (QoL) as a core outcome domain for evaluating treatment effect in vascular malformations. To correctly evaluate treatment effect on QoL, patient-reported outcome measures (PROMs) are needed that are responsive to changes. In children with vascular malformations, we explored if two widely used PROMs were responsive to changes: the Pediatric Quality of Life Inventory (PedsQL) and the Children's Dermatology Life Quality Index (CDLQI). Methods In an international multicenter prospective study, conservatively and invasively treated children completed the PedsQL and CDLQI at baseline and after follow-up of 6–8 weeks. At follow-up, change in health was assessed by a global rating of change (GRC) scale. Responsiveness was assessed by testing hypotheses on expected correlation strength between change scores of the PROMs and the GRC scale, and by calculating the area under the receiver operating characteristics curve (AUC). The PROMs were considered responsive if ≥75% of the hypotheses were confirmed or if the AUC was ≥0.7. Results Twenty-nine children were recruited in three centers in the Netherlands and United States, of which 25 completed all baseline and follow-up measurements. For both the PedsQL and CDLQI, less than 75% of the hypotheses were confirmed and the AUC was <0.7. Discussion The results suggest that these PROMs are not sufficiently responsive for evaluating treatment effect in peripheral vascular malformations. Our study emphasizes the need for assessing responsiveness before using a PROM in evaluating treatment effect.
... The 9-point scale is recommended by the Grading of Recommendations Assessment, Development and Evaluation (GRADE) working group to assess the importance of evidence and is the scale used in DelphiManager, a web-based system designed by the COMET Initiative for facilitating and managing Delphi surveys [2,21,22]. The 5-point rating scale was arbitrarily chosen as the comparator scale because it had been used previously in COS development Delphi studies [23][24][25], and because 5-point rating scales are relatively common across research studies that include a rating scale. Participants used the same rating scale they were initially allocated for all three Delphi rounds. ...
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Background: As the development of core outcome sets (COS) increases, guidance for developing and reporting high-quality COS continues to evolve; however, a number of methodological uncertainties still remain. The objectives of this study were: (1) to explore the impact of including patient interviews in developing a COS, (2) to examine the impact of using a 5-point versus a 9-point rating scale during Delphi consensus methods on outcome selection and the COS and (3) to inform and contribute to COS development methodology by advancing the evidence base on COS development techniques. Methods: Semi-structured patient interviews and a nested randomised controlled parallel group trial as part of the Pelvic Girdle Pain Core Outcome Set project (PGP-COS). Patient interviews, as an adjunct to a systematic review of outcomes reported in previous studies, were undertaken to identify preliminary outcomes for including in a Delphi consensus survey. In the Delphi survey, participants were randomised (1:1) to a 5-point or 9-point scale for rating the importance of the list of preliminary outcomes. Results: None of the outcomes identified from the patient interviews were included in the final PGP-COS. The 5-point rating scale resulted in twice as many outcomes reaching consensus after the 3-round Delphi survey compared to the 9-point scale. Consensus on all five outcomes included in the final PGP-COS was achieved by participants allocated the 5-point rating scale, whereas consensus on four of these was achieved by those using the 9-point scale. Conclusions: Using patient interviews to identify preliminary outcomes as an adjunct to conducting a systematic review of outcomes measured in the literature does not appear to influence outcome selection in developing a COS. The use of different rating scales in a Delphi survey, however, does appear to impact on outcome selection. The 5-point scale demonstrated greater congruency than the 9-point scale with the outcomes included in the final PGP-COS. Use of a 5-point scale might be considered for Delphi surveys in COS development studies. Future research to substantiate our findings and to explore the impact of other rating scales on outcome selection during COS development, however, is still warranted.
... The Cochrane Skin-Core OUtcome Set INitiative (CS-COU-SIN) is a multi-professional international initiative that supports COS groups working to improve and standardize outcome assessment and reporting in dermatology clinical trials and reviews. 6,7 Currently, 17 COSs are in development within CS-COUSIN for a broad range of dermatological conditions such as atopic dermatitis, 8,9 acne, nail psoriasis, 10 vitiligo, 11 different types of skin cancer, 12 vascular malformations, 13 congenital melanocytic naevi, 14 hidradenitis suppurativa, 15 and incontinence associated dermatitis 16 (www.cs-cousin.org/). A major problem is that across different dermatological conditions, various expressions and definitions for identical or similar outcome domains are used (e.g. ...
Article
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Background Standardized outcome reporting is crucial for trial evidence synthesis and translation of findings into clinical decision making. The OMERACT 2.0 Filter and COMET outcome domain taxonomy propose frameworks for consistent reporting of outcomes. There is an absence of a uniform dermatology‐specific reporting strategy that uses precise and consistent outcome definitions. Objectives Our aim was to map efficacy/effectiveness outcomes assessed in dermatological trials to the OMERACT 2.0 Filter as a starting point for developing an outcome taxonomy in dermatology. Methods We critically appraised 10 Cochrane Skin Reviews randomly selected from all 69 Cochrane Skin Reviews published until 01/2015 and the 220 trials included covering a broad spectrum of dermatological conditions and interventions. Efficacy/effectiveness outcomes were mapped to core areas and domains according to the OMERACT 2.0 Filter. The extracted trial outcomes were used for critical appraisal of outcome reporting in dermatology trials and for the preliminary development of a dermatology‐specific outcome taxonomy. Results The allocation of 1,086 extracted efficacy/effectiveness outcomes to the OMERACT 2.0 Filter resulted in a hierarchically structured dermatology‐specific outcome classification. In 506 outcomes (47%) the outcome concept to be measured was insufficiently described, hindering meaningful evidence synthesis. Although the core areas assessed in different dermatology trials of the same condition overlap considerably, quantitative evidence synthesis usually failed due to imprecise outcome definitions, non‐comparable outcome measurement instruments, metrics and reporting. Conclusions We present an efficacy/effectiveness outcome classification as a starting point for a dermatology‐specific taxonomy to provide trialists and reviewers with the opportunity to better synthesize and compare evidence.
... Without a standardized method of assessing lesion appearance, outcome measures of lesion response to treatment remain subjective, and are prone to biases, thus limiting comparability between studies, and the overall quality of systematic reviews. This highlights the need to develop standardized tools to assess clinical appearance of IH lesions and other vascular anomalies [97]. ...
Article
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Introduction 15% of proliferating infantile haemangioma (IH) require intervention because of the threat to function or life, ulceration or tissue distortion. Propranolol is the mainstay treatment for problematic proliferating IH. Other β-blockers and angiotensin-converting enzyme (ACE) inhibitors have been explored as alternative treatments. Areas covered The demonstration of a haemogenic endothelium origin of IH with a neural crest phenotype and multi-lineage differentiation capacity, regulated by the renin-angiotensin system, underscores its programmed biologic behaviour and accelerated involution induced by propranolol, other β-blockers and ACE inhibitors. We review the indications, dosing regimens, duration of treatment, efficacy and adverse effects of propranolol and therapeutic alternatives including oral atenolol and acebutolol, nadolol, intralesional propranolol injections, topical propranolol and timolol, and oral captopril. Expert opinion Improved understanding of the biology of IH provides insights into the mechanism of action underscoring its accelerated involution induced by propranolol, other β-blockers and ACE inhibitors. More research is required to understand the optimal dosing and duration, efficacy and safety of these alternative therapies. Recent demonstration of propranolol’s actions mediated by non-β-adrenergic isomer R-propranolol on stem cells, offers an immense opportunity to harness the efficacy of β-blockers, to induce accelerated involution of IH, while mitigating their β-adrenergic receptor-mediated adverse effects.
... It has become an increasingly important outcome measure from a clinical and epidemiological point of view (14). An international multicentre consensus study, the Outcome Measures for Vascular Malformations project, suggested core outcome sets for clinical research on peripheral vascular malformations, including lymphatic malformations, venous malformations and arteriovenous malformations (15). It agreed that HRQoL should be a core outcome measure for all types of vascular malformations. ...
Article
This is the peer reviewed version of the following article: Long-term health-related quality of life in children with lymphatic malformations treated with sclerotherapy generally matched age-appropriate standardised population norms, which has been published in final form at https://onlinelibrary.wiley.com/doi/10.1111/apa.14700. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions. ACCESS LINK: https://onlinelibrary.wiley.com/doi/10.1111/apa.14700 -------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------- ABSTRACT AIM: We assessed the long-term health-related quality of life (HRQoL) of children who received sclerotherapy for lymphatic malformations. This involved injecting drugs into the blood vessels to make them shrink. METHODS: Our cross-sectional study retrospectively reviewed patients who received OK-432 sclerotherapy injections at Karolinska University Hospital, Stockholm, Sweden, from 1998-2013. We studied 49 patients (63% female) aged 8-18 at least five years after their first injection. HRQoL was assessed with the KIDSCREEN-52 questionnaire and a study-specific questionnaire addressed disease consequences and patient satisfaction. We determined associations between HRQoL and disease and treatment and the patient’s sex. RESULTS: Overall HRQoL paralleled age-appropriate norms in the general population, but some subgroups had lower levels. Regression-based estimates showed that larger numbers of injections were negatively associated with HRQoL in the dimensions autonomy, parent relations and home life, financial resources and school environment (p = 0.01 to 0.03). Malformations in the head and neck area were negative predictors across dimensions and were strongest for psychological well being (p = 0.009), parent relations and home life (p = 0.017) and school environment (p = 0.006). CONCLUSION: Despite generally positive outcomes, multiple injections and malformations in the head and neck were associated with impaired HRQoL.
... All rights reserved. , the Outcome Measures for Vascular Malformations project, suggested core outcome sets for clinical research on peripheral vascular malformations, including lymphatic malformations, venous malformations and arterio venous malformations (15). It agreed that HRQoL should be a core outcome measure for all types of vascular malformations. ...
Article
Abstract AIM: We assessed the long-term health-related quality of life (HRQoL) of children who received sclerotherapy for lymphatic malformations. This involved injecting drugs into the blood vessels to make them shrink. METHODS: Our cross-sectional study retrospectively reviewed patients who received OK-432 sclerotherapy injections at Karolinska University Hospital, Stockholm, Sweden, from 1998-2013. We studied 49 patients (63% female) aged 8-18 at least five years after their first injection. HRQoL was assessed with the KIDSCREEN-52 questionnaire and a study-specific questionnaire addressed disease consequences and patient satisfaction. We determined associations between HRQoL and disease and treatment and the patient's sex. RESULTS: Overall HRQoL paralleled age-appropriate norms in the general population, but some subgroups had lower levels. Regression-based estimates showed that larger numbers of injections were negatively associated with HRQoL in the dimensions autonomy, parent relations and home life, financial resources and school environment (p = 0.01 to 0.03). Malformations in the head and neck area were negative predictors across dimensions and were strongest for psychological well being (p = 0.009), parent relations and home life (p = 0.017) and school environment (p = 0.006). CONCLUSION: Despite generally positive outcomes, multiple injections and malformations in the head and neck were associated with impaired HRQoL.
... 5 In vitiligo, similar variability in outcome reporting was found, 6 and this is also the case for other dermatological diseases such as acne, 7 hidradenitis suppurativa, 8 psoriasis 9 and vascular malformations. 10 Using incomparable outcomes is considered a waste of valuable healthcare resources and is unwanted. 11 Moreover, for the majority of dermatological diseases, we do not know which outcomes are considered most important to patients and their healthcare providers. ...
Article
Linked Articles: Horbach. Br J Dermatol 2018; 178:473–481. Thorlacius et al. Br J Dermatol 2018; 178:715–721.
Article
Introduction: Genetically-targeted drugs in vascular anomalies (VA) are used despite the absence of a validated severity score. The aim of this study was to evaluate the feasibility of grouping phenotypic VA clinical characteristics into a single severity score. Methods: A systematic literature review including children treated with sirolimus accompanied by a detailed description of phenotype and management was conducted. Demographic data and clinical features were extracted to define distinct categories of phenotypes. Results: Children with VA display two main phenotypes regardless of VA subtype, which may overlap. A systemic phenotype results from direct invasion and compression of vital structures generally leading to hospitalization and aggressive management in infancy. A functional phenotype is associated to chronic pain and disability manifesting mainly during early adolescence and managed in the outpatient setting. Conclusion: The two distinct phenotypes descibed could be the basis for developing a unified scoring system for VA severity assessment. This article is protected by copyright. All rights reserved.
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Auricular arteriovenous malformations (AVMs) can cause a variety of symptoms that seriously impact the patient's appearance, life, and mental well‐being. Surgery is the primary management method for auricular AVMs, but there is no consensus on how to surgically manage auricular AVMs. In this article, we document a comprehensive review of the characteristics, classification, and surgical interventions to treat auricular AVMs. Auricular arteriovenous malformations (AVMs) can cause a variety of symptoms that seriously impact the patient's appearance, life, and mental well‐being. Surgery is the primary management method for auricular AVMs, but there is no consensus on how to surgically manage auricular AVMs. In this article, we document a comprehensive review of the characteristics, classification, and surgical interventions to treat auricular AVMs.
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Life course studies are designed to “collect once, use multiple times” for observational and, increasingly, interventional research. Core Outcome Sets (COS) are minimum sets developed for clinical trials by multi-stakeholder consensus methodologies. We aimed to synthesize published COS that might guide outcomes selection for early life cohorts with an interventional focus. We searched PubMed, Medline, COMET, and CROWN for COS published before January 2021 relevant to four life stages (pregnancy, newborns, children <8 years, and parents (adults aged 18–50 years)). We synthesized core outcomes into overarching constructs. From 46 COS we synthesized 414 core outcomes into 118 constructs. “Quality of life”, “adverse events”, “medication use”, “hospitalization”, and “mortality” were consistent across all stages. For pregnancy, common constructs included “preterm birth”, “delivery mode”, “pre-eclampsia”, “gestational weight gain”, “gestational diabetes”, and “hemorrhage”; for newborns, “birthweight”, “small for gestational age”, “neurological damage”, and “morbidity” and “infection/sepsis”; for pediatrics, “pain”, “gastrointestinal morbidity”, “growth/weight”, “breastfeeding”, “feeding problems”, “hearing”, “neurodevelopmental morbidity”, and “social development”; and for adults, “disease burden”, “mental health”, “neurological function/stroke”, and “cardiovascular health/morbidity”. This COS synthesis generated outcome constructs that are of high value to stakeholders (participants, health providers, services), relevant to life course research, and could position cohorts for trial capabilities. We synthesized existing Core Outcome Sets as a transparent methodology that could prioritize outcomes for lifecourse cohorts with an interventional focus. “Quality of life”, “adverse events”, “medication use”, “hospitalization”, and “mortality” are important outcomes across pregnancy, newborns, childhood, and early-to-mid-adulthood (the age range relevant to parents). Other common outcomes (such as “birthweight”, “cognitive function/ability”, “psychological health”) are also highly relevant to lifecourse research. This synthesis could assist new early life cohorts to pre-select outcomes that are of high value to stakeholders (participants, health providers, services), are relevant to lifecourse research, and could position them for future trials and interventional capability.
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Background: As the development of core outcome sets (COS) increases, guidance for developing and reporting high-quality COS continues to evolve; however, a number of methodological uncertainties still remain. The objectives of this study were: (1) to explore the impact of including patient interviews in developing a COS, (2) to examine the impact of using a 5-point versus a 9-point rating scale during Delphi consensus methods on outcome selection and (3) to inform and contribute to COS development methodology by advancing the evidence base on COS development techniques. Methods: Semi-structured patient interviews and a nested randomised controlled parallel group trial as part of the Pelvic Girdle Pain Core Outcome Set project (PGP-COS). Patient interviews, as an adjunct to a systematic review of outcomes reported in previous studies, were undertaken to identify preliminary outcomes for including in a Delphi consensus survey. In the Delphi survey, participants were randomised (1:1) to a 5-point or 9-point rating scale for rating the importance of the list of preliminary outcomes. Results: Four of the eight patient interview derived outcomes were included in the preliminary COS, however, none of these outcomes were included in the final PGP-COS. The 5-point rating scale resulted in twice as many outcomes reaching consensus after the 3-round Delphi survey compared to the 9-point scale. Consensus on all five outcomes included in the final PGP-COS was achieved by participants allocated the 5-point rating scale, whereas consensus on four of these was achieved by those using the 9-point scale. Conclusions: Using patient interviews to identify preliminary outcomes as an adjunct to conducting a systematic review of outcomes measured in the literature did not appear to influence outcome selection in developing the COS in this study. The use of different rating scales in a Delphi survey, however, did appear to impact on outcome selection. The 5-point scale demonstrated greater congruency than the 9-point scale with the outcomes included in the final PGP-COS. Future research to substantiate our findings and to explore the impact of other rating scales on outcome selection during COS development, however, is warranted.
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Linked Article: Horbach et al. Br J Dermatol 2018; 178:473–481.
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Background: Despite increasing international interest, there is a lack of evidence about the most efficient, effective and acceptable ways to implement patient and public involvement (PPI) in clinical trials. Objective: To identify the priorities of UK PPI stakeholders for methodological research to help resolve uncertainties about PPI in clinical trials. Design: A modified Delphi process including a two round online survey and a stakeholder consensus meeting. Participants: In total, 237 people registered of whom 219 (92%) completed the first round. One hundred and eighty-seven of 219 (85%) completed the second; 25 stakeholders attended the consensus meeting. Results: Round 1 of the survey comprised 36 topics; 42 topics were considered in round 2 and at the consensus meeting. Approximately 96% of meeting participants rated the top three topics as equally important. These were as follows: developing strong and productive working relationships between researchers and PPI contributors; exploring PPI practices in selecting trial outcomes of importance to patients; and a systematic review of PPI activity to improve the accessibility and usefulness of trial information (eg participant information sheets) for participants. Conclusions: The prioritized methodological research topics indicate important areas of uncertainty about PPI in trials. Addressing these uncertainties will be critical to enhancing PPI. Our findings should be used in the planning and funding of PPI in clinical trials to help focus research efforts and minimize waste.
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Background: To be meaningful, a core outcome set (COS) should be relevant to all stakeholders including patients and carers. This review aimed to explore the methods by which patients and carers have been included as participants in COS development exercises and, in particular, the use and reporting of qualitative methods. Methods: In August 2015, a search of the Core Outcomes Measures in Effectiveness Trials (COMET) database was undertaken to identify papers involving patients and carers in COS development. Data were extracted to identify the data collection methods used in COS development, the number of health professionals, patients and carers participating in these, and the reported details of qualitative research undertaken. Results: Fifty-nine papers reporting patient and carer participation were included in the review, ten of which reported using qualitative methods. Although patients and carers participated in outcome elicitation for inclusion in COS processes, health professionals tended to dominate the prioritisation exercises. Of the ten qualitative papers, only three were reported as a clear pre-designed part of a COS process. Qualitative data were collected using interviews, focus groups or a combination of these. None of the qualitative papers reported an underpinning methodological framework and details regarding data saturation, reflexivity and resource use associated with data collection were often poorly reported. Five papers reported difficulty in achieving a diverse sample of participants and two reported that a large and varied range of outcomes were often identified by participants making subsequent rating and ranking difficult. Conclusions: Consideration of the best way to include patients and carers throughout the COS development process is needed. Additionally, further work is required to assess the potential role of qualitative methods in COS, to explore the knowledge produced by different qualitative data collection methods, and to evaluate the time and resources required to incorporate qualitative methods into COS development.
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Introduction Randomised controlled trials (RCTs) should have well-defined primary and secondary outcomes to answer questions generated by the main hypotheses. However, for the chronic, inflammatory skin disease hidradenitis suppurativa (HS), the reported outcome measures are numerous and diverse. A recent systematic review found a total of 30 outcome measure instruments in 12 RCTs. This use of a broad range of outcome measures can increase difficulties in interpretation and comparison of results and may potentially obstruct appropriate evidence synthesis by causing reporting bias. One strategy for dealing with these problems is to develop a core outcome set (COS). A COS is a list of outcomes that are meant as mandatory and should be measured and reported in all clinical trials. The aim of this study is to develop a COS for the management of HS. Method and analysis An international steering group of researchers, clinicians and a patient research partner will guide the COS development. 6 stakeholder groups are involved: patients, dermatologists, surgeons, nurses, industry representatives and drug regulatory authorities. A 1:1 ratio of patients:healthcare professionals is aimed for. The initial list of candidate items will be obtained by combining three data sets: (1) a systematic review of the literature, (2) US and Danish qualitative interview studies involving patients with HS and (3) an online healthcare professional (HCP) item generation survey. To reach consensus on the COS, 4 anonymous online Delphi rounds are then planned together with 2 face-to-face consensus meetings (1 in Europe and 1 in the USA) to ensure global representation. Ethics and dissemination The study will be performed according to the Helsinki declaration. All results from the study, including inconclusive or negative results, will be published in peer-reviewed indexed journals. The study will involve different stakeholder groups to ensure that the developed COS will be suitable and well accepted.
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Introduction Low-flow vascular malformations are congenital abnormalities of the veins, capillaries or lymphatic vessels or a combination of the previous. Compressive garments are frequently used as a first-line treatment option for low-flow vascular malformations of the extremities with the purpose of relieving symptoms. Yet, the benefits and harms of compression stockings remain unclear. Methods A systematic search was performed in MEDLINE, Embase, Cochrane Central Register of Controlled Trials including a hand search for studies measuring the benefits and harms of compression garments in treating low-flow vascular malformations. Two investigators performed study selection, quality assessment and data extraction independently. Results Of the 565 studies found, eventually five (totalling 101 patients with venous malformations or Klippel–Trenaunay syndrome receiving compression therapy) observational studies were included in the systematic review. Although the overall quality of these studies was poor, results suggest that compression garments might lessen intravascular coagulation, improve symptoms and appearance, diminish oedema, and protect against minor trauma. None of the studies quantified any harms of compression therapy. Conclusion Even though compression therapy is commonly used in the treatment of low-flow vascular malformations, available literature does not provide high-quality evidence to validate its use. We therefore advocate the need for prospective comparative trials with standardised outcome measures to study the benefits and harms of this treatment option.
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Background: Bariatric and metabolic surgery is used as a treatment for patients with severe and complex obesity. However, there is a need to improve outcome selection and reporting in bariatric surgery trials. A Core Outcome Set (COS), an agreed minimum set of outcomes reported in all studies of a specific condition, may achieve this. Here, we present the development of a COS for BARIAtric and metabolic surgery Clinical Trials-the BARIACT Study. Methods and findings: Outcomes identified from systematic reviews and patient interviews informed a questionnaire survey. Patients and health professionals were surveyed three times and asked to rate the importance of each item on a 1-9 scale. Delphi methods provided anonymised feedback to participants. Items not meeting predefined criteria were discarded between rounds. Remaining items were discussed at consensus meetings, held separately with patients and professionals, where the COS was agreed. Data sources identified 2,990 outcomes, which were used to develop a 130-item questionnaire. Round 1 response rates were moderate but subsequently improved to above 75% for other rounds. After rounds 2 and 3, 81 and 14 items were discarded, respectively, leaving 35 items for discussion at consensus meetings. The final COS included nine items: "weight," "diabetes status," "cardiovascular risk," "overall quality of life (QOL)," "mortality," "technical complications of the specific operation," "any re-operation/re-intervention," "dysphagia/regurgitation," and "micronutrient status." The main limitation of this study was that it was based in the United Kingdom only. Conclusions: The COS is recommended to be used as a minimum in all trials of bariatric and metabolic surgery. Adoption of the COS will improve data synthesis and the value of research data. Future work will establish methods for the measurement of the outcomes in the COS.
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Background: Core outcome sets (COS) can enhance the relevance of research by ensuring that outcomes of importance to health service users and other people making choices about health care in a particular topic area are measured routinely. Over 200 COS to date have been developed, but the clarity of these reports is suboptimal. COS studies will not achieve their goal if reports of COS are not complete and transparent. Methods and findings: In recognition of these issues, an international group that included experienced COS developers, methodologists, journal editors, potential users of COS (clinical trialists, systematic reviewers, and clinical guideline developers), and patient representatives developed the Core Outcome Set-STAndards for Reporting (COS-STAR) Statement as a reporting guideline for COS studies. The developmental process consisted of an initial reporting item generation stage and a two-round Delphi survey involving nearly 200 participants representing key stakeholder groups, followed by a consensus meeting. The COS-STAR Statement consists of a checklist of 18 items considered essential for transparent and complete reporting in all COS studies. The checklist items focus on the introduction, methods, results, and discussion section of a manuscript describing the development of a particular COS. A limitation of the COS-STAR Statement is that it was developed without representative views of low- and middle-income countries. COS have equal relevance to studies conducted in these areas, and, subsequently, this guideline may need to evolve over time to encompass any additional challenges from developing COS in these areas. Conclusions: With many ongoing COS studies underway, the COS-STAR Statement should be a helpful resource to improve the reporting of COS studies for the benefit of all COS users.
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Background In cooperation with the Core Outcome Measures in Effectiveness Trials (COMET) initiative, the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) initiative aimed to develop a guideline on how to select outcome measurement instruments for outcomes (i.e., constructs or domains) included in a “Core Outcome Set” (COS). A COS is an agreed minimum set of outcomes that should be measured and reported in all clinical trials of a specific disease or trial population. Methods Informed by a literature review to identify potentially relevant tasks on outcome measurement instrument selection, a Delphi study was performed among a panel of international experts, representing diverse stakeholders. In three consecutive rounds, panelists were asked to rate the importance of different tasks in the selection of outcome measurement instruments, to justify their choices, and to add other relevant tasks. Consensus was defined as being achieved when 70 % or more of the panelists agreed and when fewer than 15 % of the panelists disagreed. ResultsOf the 481 invited experts, 120 agreed to participate of whom 95 (79 %) completed the first Delphi questionnaire. We reached consensus on four main steps in the selection of outcome measurement instruments for COS: Step 1, conceptual considerations; Step 2, finding existing outcome measurement instruments, by means of a systematic review and/or a literature search; Step 3, quality assessment of outcome measurement instruments, by means of the evaluation of the measurement properties and feasibility aspects of outcome measurement instruments; and Step 4, generic recommendations on the selection of outcome measurement instruments for outcomes included in a COS (consensus ranged from 70 to 99 %). Conclusions This study resulted in a consensus-based guideline on the methods for selecting outcome measurement instruments for outcomes included in a COS. This guideline can be used by COS developers in defining how to measure core outcomes.
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Background: Colorectal cancer (CRC) is a major cause of worldwide morbidity and mortality. Surgical treatment is common, and there is a great need to improve the delivery of such care. The gold standard for evaluating surgery is within well-designed randomized controlled trials (RCTs); however, the impact of RCTs is diminished by a lack of coordinated outcome measurement and reporting. A solution to these issues is to develop an agreed standard "core" set of outcomes to be measured in all trials to facilitate cross-study comparisons, meta-analysis, and minimize outcome reporting bias. This study defines a core outcome set for CRC surgery. Methods and findings: The scope of this COS includes clinical effectiveness trials of surgical interventions for colorectal cancer. Excluded were nonsurgical oncological interventions. Potential outcomes of importance to patients and professionals were identified through systematic literature reviews and patient interviews. All outcomes were transcribed verbatim and categorized into domains by two independent researchers. This informed a questionnaire survey that asked stakeholders (patients and professionals) from United Kingdom CRC centers to rate the importance of each domain. Respondents were resurveyed following group feedback (Delphi methods). Outcomes rated as less important were discarded after each survey round according to predefined criteria, and remaining outcomes were considered at three consensus meetings; two involving international professionals and a separate one with patients. A modified nominal group technique was used to gain the final consensus. Data sources identified 1,216 outcomes of CRC surgery that informed a 91 domain questionnaire. First round questionnaires were returned from 63 out of 81 (78%) centers, including 90 professionals, and 97 out of 267 (35%) patients. Second round response rates were high for all stakeholders (>80%). Analysis of responses lead to 45 and 23 outcome domains being retained after the first and second surveys, respectively. Consensus meetings generated agreement on a 12 domain COS. This constituted five perioperative outcome domains (including anastomotic leak), four quality of life outcome domains (including fecal urgency and incontinence), and three oncological outcome domains (including long-term survival). Conclusion: This study used robust consensus methodology to develop a core outcome set for use in colorectal cancer surgical trials. It is now necessary to validate the use of this set in research practice.
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A major obstacle of evidence-based clinical decision making is the use of nonstandardized, partly untested outcome measurement instruments. Core Outcome Sets (COSs) are currently developed in different medical fields to standardize and improve the selection of outcomes and outcome measurement instruments in clinical trials, in order to pool results of trials or to allow indirect comparison between interventions. A COS is an agreed minimum set of outcomes that should be measured and reported in all clinical trials of a specific disease or trial population. The international, multidisciplinary Cochrane Skin Group Core Outcome Set Initiative (CSG-COUSIN) aims to develop and implement COSs in dermatology, thus making trial evidence comparable and, herewith, more useful for clinical decision making. The inaugural meeting of CSG-COUSIN was held on 17-18 March 2015 in Dresden, Germany, as the exclusive theme of the Annual Cochrane Skin Group Meeting. In total, 29 individuals representing a broad mix of different stakeholder groups, professions, skills and perspectives attended. This report provides a description of existing COS initiatives in dermatology, highlights current methodological challenges in COS development, and presents the concept, aims and structure of CSG-COUSIN.
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Background: Sclerotherapy has become the gold standard for the first-line therapy of most venous (VMs) and lymphatic malformations (LMs) of the head and neck. Numerous sclerosing agents are used to treat these low-flow vascular malformations; however, to date, it remains unclear which sclerosing agent is superior in terms of effectiveness and safety. Methods: In a systematic review of the literature (1995-present), we compare the effectiveness and complications of the sclerosing agents most commonly used for cervicocraniofacial VMs and LMs. Results: The literature search yielded 1155 articles, among which 36 (1552 patients) were included in the systematic review. The quality of evidence was low. Pingyangmycin, absolute ethanol, OK-432, ethanolamine oleate, bleomycin, polidocanol, doxycycline, and sodium tetradecyl sulfate (STS) were the most reported sclerosing agents. All agents seem effective, and the mean overall response varies from 71% to 100%. Complications occurred more frequently after ethanol sclerotherapy (18%), compared to other sclerosing agents (0-6%). Cellulitis and ulceration were encountered following sclerotherapy with most sclerosing agents, but skin necrosis was particularly observed after ethanol. Facial nerve paralysis occurred only after OK-432 (0.05%) and ethanol sclerotherapy (6%). Conclusions: This systematic review could not identify a significantly superior sclerosing agent in terms of effectiveness, due to the low quality of the available evidence. Until stronger evidence is available, the difference in complication rates is potentially the deciding factor in the choice between sclerosing agents. As a significantly higher complication rate and more severe local complications were encountered after using absolute ethanol, we cannot recommend this agent for sclerotherapy of cervicofacial vascular malformations.
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Background Appropriate outcome selection is essential if research is to guide decision-making and inform policy. Systematic reviews of the clinical, cosmetic and patient-reported outcomes of reconstructive breast surgery, however, have demonstrated marked heterogeneity, and results from individual studies cannot be compared or combined. Use of a core outcome set may improve the situation. The BRAVO study developed a core outcome set for reconstructive breast surgery. Methods A long list of outcomes identified from systematic reviews and stakeholder interviews was used to inform a questionnaire survey. Key stakeholders defined as individuals involved in decision-making for reconstructive breast surgery, including patients, breast and plastic surgeons, specialist nurses and psychologists, were sampled purposively and sent the questionnaire (round 1). This asked them to rate the importance of each outcome on a 9-point Likert scale from 1 (not important) to 9 (extremely important). The proportion of respondents rating each item as very important (score 7–9) was calculated. This was fed back to participants in a second questionnaire (round 2). Respondents were asked to reprioritize outcomes based on the feedback received. Items considered very important after round 2 were discussed at consensus meetings, where the core outcome set was agreed. Results A total of 148 items were combined into 34 domains within six categories. Some 303 participants (51·4 per cent) (215 (49·5 per cent) of 434 patients; 88 (56·4 per cent) of 156 professionals) completed and returned the round 1 questionnaire, and 259 (85·5 per cent) reprioritized outcomes in round 2. Fifteen items were excluded based on questionnaire scores and 19 were carried forward to the consensus meetings, where a core outcome set containing 11 key outcomes was agreed. Conclusion The BRAVO study has used robust consensus methodology to develop a core outcome set for reconstructive breast surgery. Widespread adoption by the reconstructive community will improve the quality of outcome assessment in effectiveness studies. Future work will evaluate how these key outcomes should best be measured.
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Vascular anomalies represent a spectrum of disorders from a simple "birthmark" to life- threatening entities. Incorrect nomenclature and misdiagnoses are commonly experienced by patients with these anomalies. Accurate diagnosis is crucial for appropriate evaluation and management, often requiring multidisciplinary specialists. Classification schemes provide a consistent terminology and serve as a guide for pathologists, clinicians, and researchers. One of the goals of the International Society for the Study of Vascular Anomalies (ISSVA) is to achieve a uniform classification. The last classification (1997) stratified vascular lesions into vascular malformations and proliferative vascular lesions (tumors). However, additional disease entities have since been identified that are complex and less easily classified by generic headings, such as capillary malformation, venous malformation, lymphatic malformation, etc. We hereby present the updated official ISSVA classification of vascular anomalies. The general biological scheme of the classification is retained. The section on tumors has been expanded and lists the main recognized vascular tumors, classified as benign, locally aggressive or borderline, and malignant. A list of well-defined diseases is included under each generic heading in the "Simple Vascular Malformations" section. A short definition is added for eponyms. Two new sections were created: one dealing with the malformations of individually named vessels (previously referred to as "truncular" malformations); the second groups lesions of uncertain or debated nature (tumor versus malformation). The known genetic defects underlying vascular anomalies are included in an appendix. This classification is meant to be a framework, acknowledging that it will require modification as new scientific information becomes available. Copyright © 2015 by the American Academy of Pediatrics.
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Despite the importance of shared decision making for delivering patient-centered care in rheumatology, there is no consensus on how to measure its process and outcomes. The aim of this Outcome Measures in Rheumatology (OMERACT) working group is to determine the core set of domains for measuring shared decision making in intervention studies in adults with osteoarthritis (OA), from the perspectives of patients, health professionals, and researchers. We followed the OMERACT Filter 2.0 method to develop a draft core domain set by (1) forming an OMERACT working group; (2) conducting a review of domains of shared decision making; and (3) obtaining opinions of all those involved using a modified nominal group process held at a session activity at the OMERACT 12 meeting. In all, 26 people from Europe, North America, and Australia, including 5 patient research partners, participated in the session activity. Participants identified the following domains for measuring shared decision making to be included as part of the draft core set: (1) identifying the decision, (2) exchanging information, (3) clarifying views, (4) deliberating, (5) making the decision, (6) putting the decision into practice, and (7) assessing the effect of the decision. Contextual factors were also suggested. We proposed a draft core set of shared decision-making domains for OA intervention research studies. Next steps include a workshop at OMERACT 13 to reach consensus on these proposed domains in the wider OMERACT group, as well as to detail subdomains and assess instruments to develop a core outcome measurement set.
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Core outcome sets (COSs) are consensus-derived minimum sets of outcomes to be assessed in a specific situation. COSs are being increasingly developed to limit outcome-reporting bias, allow comparisons across trials, and strengthen clinical decision making. Despite the increasing interest in outcomes research, methods to develop COSs have not yet been standardized. The aim of this paper is to present the Harmonizing Outcomes Measures for Eczema (HOME) roadmap for the development and implementation of COSs, which was developed on the basis of our experience in the standardization of outcome measurements for atopic eczema. Following the establishment of a panel representing all relevant stakeholders and a research team experienced in outcomes research, the scope and setting of the core set should be defined. The next steps are the definition of a core set of outcome domains such as symptoms or quality of life, followed by the identification or development and validation of appropriate outcome measurement instruments to measure these core domains. Finally, the consented COS needs to be disseminated, implemented, and reviewed. We believe that the HOME roadmap is a useful methodological framework to develop COSs in dermatology, with the ultimate goal of better decision making and promoting patient-centered health care.Journal of Investigative Dermatology advance online publication, 4 September 2014; (2014) 0, 000-000. doi:10.1038/jid.2014.320.
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Background Lack of standardization of outcome measures limits the usefulness of clinical trial evidence to inform health care decisions. This can be addressed by agreeing on a minimum core set of outcome measures per health condition, containing measures relevant to patients and decision makers. Since 1992, the Outcome Measures in Rheumatology (OMERACT) consensus initiative has successfully developed core sets for many rheumatologic conditions, actively involving patients since 2002. Its expanding scope required an explicit formulation of its underlying conceptual framework and process. Methods Literature searches and iterative consensus process (surveys and group meetings) of stakeholders including patients, health professionals, and methodologists within and outside rheumatology. Results To comprehensively sample patient-centered and intervention-specific outcomes, a framework emerged that comprises three core “Areas,” namely Death, Life Impact, and Pathophysiological Manifestations; and one strongly recommended Resource Use. Through literature review and consensus process, core set development for any specific health condition starts by identifying at least one core “Domain” within each of the Areas to formulate the “Core Domain Set.” Next, at least one applicable measurement instrument for each core Domain is identified to formulate a “Core Outcome Measurement Set.” Each instrument must prove to be truthful (valid), discriminative, and feasible. In 2012, 96% of the voting participants (n = 125) at the OMERACT 11 consensus conference endorsed this model and process. Conclusion The OMERACT Filter 2.0 explicitly describes a comprehensive conceptual framework and a recommended process to develop core outcome measurement sets for rheumatology likely to be useful as a template in other areas of health care.
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This study examined how using Likert-type scales with either 5-point, 7-point or 10-point format affects the resultant data in terms of mean scores, and measures of dispersion and shape. Three groups of respondents were administered a series of eight questions (group n's = 300, 250, 185). Respondents were randomly selected members of the general public. A different scale format was administered to each group. The 5-and 7-point scales were rescaled to a comparable mean score out of ten. The study found that the 5-and 7-point scales produced the same mean score as each other, once they were rescaled. However, the 10-point format tended to produce slightly lower relative means than either the 5-or 7-point scales (after the latter were rescaled). The overall mean score of the eight questions was 0.3 scale points lower for the 10-point format compared to the rescaled 5-and 7-point formats. This difference was statistically significant at p = 0.04. In terms of the other data characteristics, there was very little difference among the scale formats in terms of variation about the mean, skewness or kurtosis. This study is 'good news' for research departments or agencies who ponder whether changing scale format will destroy the comparability of historical data. 5-and 7-point scales can easily be rescaled with the resultant data being quite comparable. In the case of comparing 5-or 7-point data to 10-point data, a straightforward rescaling and arithmetic adjustment easily facilitates the comparison. The study suggests that indicators of customer sentiment – such as satisfaction surveys – may be partially dependent on the choice of scale format. A 5-or 7-point scale is likely to produce slightly higher mean scores relative to the highest possible attainable score, compared to that produced from a 10-point scale.
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The Delphi method has proven a popular tool in information systems research for identifying and prioritizing issues for managerial decision-making. However, many past studies have not adopted a systematic approach to conduct a Delphi study. This article provides rigorous guidelines for the process of selecting appropriate experts for the study and gives detailed principles for making design choices during the process that ensure a valid study. A detailed example of a study to identify key factors affecting the diffusion of e-commerce in Sub-Saharan Africa illustrates the design choices that may be involved. We conclude with suggestions for theoretical applications.
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Ian Sinha and colleagues advise that when using the Delphi process to develop core outcome sets for clinical trials, patients and clinicians be involved, researchers and facilitators avoid imposing their views on participants, and attrition of participants be minimized.
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There is wide variation in the use of outcome measures for eczema. We performed a three-stage web-based international Delphi exercise to develop consensus-based sets of core outcome domains for eczema for "controlled trials" and "clinical recordkeeping". A total of 57 individuals from four stakeholder groups (consumers, clinical experts, regulatory agency representatives, and journal editors) representing 13 countries were asked to rate the importance of 19 outcome domains for eczema and to choose which domains should be included in two core sets of outcomes. Forty-six individuals (81%) participated. Participants received standardized feedback, including the group median, interquartile range, and previous responses, and the assessment was repeated in two subsequent rounds. We defined consensus a priori if at least 60% of the members of at least three stakeholder groups, including consumers, recommended domain inclusion in the core set. Consensus was achieved for inclusion of symptoms, physician-assessed clinical signs, and a measurement for long-term control of flares in the core set of outcome domains for eczema trials. We recommend including these three core outcomes in future eczema trials in order to enhance clinical interpretability and to enable meta-analyses across different studies. For recordkeeping, consensus was reached to regularly monitor eczema symptoms in clinical practice. Future work is needed to select which existing or new scales should be used to measure the domains identified as relevant for the core set.
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The Delphi technique is an approach used to gain consensus among a panel of experts. This is normally achieved through a series of rounds where information is fed back to panel members using questionnaires. It has been used extensively within social science research and is being increasingly employed by nurse researchers. This popularity has meant that the technique has been adapted in various ways and there is the possibility that the rigour associated with the original format has been threatened. This signals the need for a critical review of the Delphi as a robust and systematic approach to data collection. While there is a great volume of literature surrounding the "Delphi", there is a dearth of papers critically analysing the technique. This paper aims to examine critically the Delphi technique from a range of perspectives. Discussion will focus on problems of definition and the advantages and disadvantages and the techniques' application in nursing. The critique will be structured through an analysis of the key aspects of the Delphi process. These key aspects include analysis of sampling, anonymity, use of experts, rounds and application. The critical analysis highlights the increasing popularity of the Delphi and the modifications to the process which may cause methodological problems. Ultimately, the Delphi has much to offer in terms of gaining consensus from a wide range of individuals on specific topics.
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To evaluate the midterm outcomes of percutaneous sclerotherapy of lymphatic malformations (LMs) as judged by patients. A 13-item survey questionnaire was sent to 74 patients who had undergone at least one sclerotherapy procedure in our hospital from January 1997 through January 2003. Information regarding the anatomic location, specific symptoms reported, history, treatment satisfaction, postprocedural complications, and number of treatment sessions was elicited. Four sclerosing agents (as single agents or in combination with other agents) were used: ethanol, sodium tetradecyl sulfate 3% (STS), OK-432, and doxycycline. Fifty-five patients or their caregivers completed the survey. The patients' ages ranged from 6 months to 48 years at the time of the first procedure (mean, 12 y; median, 4 y). A majority of LMs were located in the cervicofacial region. The size and location of the lesion, recurrent infection, and pain were the most frequent indications for treatment. Fifty-one percent of these patients received sclerotherapy alone or in conjunction with surgery as primary treatment. Ethanol was the most common sclerosing agent used, followed by doxycycline, STS, and OK-432. Response varied with the type of LM, with 100%, 86%, and 43% of the patients reporting good to complete response for macrocystic, microcystic, and combined-type LMs, respectively. Skin blistering and ulcers were the most common complications. Permanent complications were uncommon and were largely related to ethanol use. Percutaneous sclerotherapy provides effective midterm primary treatment for LMs. Treatment outcomes appear to vary according to the morphology of the malformation.
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This letter describes the substantial activity on the Core Outcome Measure in Effectiveness Trials (COMET) website in 2015, updating our earlier progress reports for the period from the launch of the COMET website and database in August 2011 to December 2014. As in previous years, 2015 saw further increases in the annual number of visits to the website, the number of pages viewed and the number of searches undertaken. The sustained growth in use of the website and database suggests that COMET is continuing to gain interest and prominence, and that the resources are useful to people interested in the development of core outcome sets.
Article
Objective: To develop a set of core outcomes for studies on pregnant women with epilepsy. Design: Delphi consensus study. Population: Healthcare professionals, and patient representatives with lived experience of epilepsy in the UK. Methods: We used a modified Delphi method and a consultation meeting to achieve consensus. Potential outcomes were identified by systematic review, and were scored using a Likert scale anchored between 1 (least important) and 5 (most important). We included outcomes that scored ≥4 by >70% of participants, and outcomes that scored ≤2 by <15% of participants. Main outcome measures: Outcomes in studies on epilepsy in pregnancy. Results: Seventy-five healthcare professionals completed the first round, 48 (64%) completed the second round, and 37 (49%) completed the third round of the survey. Twenty-four patient representatives participated. The final core outcome set included 31 outcomes in three domains: neurological, offspring, and obstetric. Outcomes in the neurological domain were seizure control in pregnancy and postpartum, status epilepticus, maternal mortality, drowning, sudden unexpected death in epilepsy, postnatal depression, and quality of life. Offspring domain included congenital abnormalities (major and minor), fetal anticonvulsant syndrome, neurodevelopment, autism disorder, neonatal clinical complications, admission to a neonatal intensive care unit, and anthropometric measurements. The obstetric domain included live birth, stillbirth, miscarriage, ectopic, termination of pregnancy, admission to a high dependency or intensive care unit, breastfeeding, mode of delivery, preterm birth, pre-eclampsia, and eclampsia. Outcomes specific for studies on anti-epileptic drugs (AEDs) included maternal AED toxicity, AED compliance, neonatal withdrawal symptoms, and neonatal haemorrhagic disease. Conclusion: Embedding this core set in future clinical trials will promote the standardisation of reporting to inform clinical practice. Tweetable abstract: A Delphi method identifying core outcomes for epilepsy in pregnancy. Final core set includes 31 outcomes.
Article
Background: Vascular malformations are congenital anomalies of the vascular system. Intralesional bleomycin injections are commonly used to treat vascular malformations. However, pulmonary fibrosis could potentially be a severe complication, known from systemic bleomycin therapy for malignancies. In this study, the authors investigate the effectiveness and safety of bleomycin (A2, B2, and A5) injections for vascular malformations, when possible relative to other sclerosants. Methods: The authors performed a PubMed, Embase, Cochrane Central Register of Controlled Trials, and gray literature search for studies (1995 to the present) reporting outcome of intralesional bleomycin injections in patients with vascular malformations (n ≥ 10). Predefined outcome measures of interest were size reduction, symptom relief, quality of life, adverse events (including pulmonary fibrosis), and patient satisfaction. Results: Twenty-seven studies enrolling 1325 patients were included. Quality of evidence was generally low. Good to excellent size reduction was reported in 84 percent of lymphatic and 87 percent of venous malformations. Pulmonary fibrosis was never encountered. Meta-analysis of four studies on venous malformations treated with bleomycin versus other sclerosants showed similar size reduction (OR, 0.67; 95 percent CI, 0.24 to 1.88) but a significantly lower adverse event rate (OR, 0.1; 95 percent CI, 0.03 to 0.39) and fewer severe complications after bleomycin. Symptom relief, quality of life, and patient satisfaction were reported inadequately. Conclusions: The authors' data suggest that bleomycin is effective in reducing the size of lymphatic and venous malformations, and leads to a lower adverse event rate and fewer severe complications than other sclerosants. The included literature does not provide evidence that pulmonary fibrosis is a complication of intralesional bleomycin injections. This study represents the "best available" evidence; however, only low- to moderate-quality studies were available. Clinical question/level of evidence: Therapeutic, IV.
Article
To develop general and site-specific treatment effect and outcome measures to standardize the reporting of head and neck lymphatic malformation (HNLM) treatments. Consensus statement/expert opinion. Multiple tertiary academic institutions. The modified Delphi method is an iterative process of collecting expert opinions, refining opinions through discussion and feedback, statistically aggregating opinions, and using these aggregates to generate consensus opinion in the absence of other data. The modified Delphi method was used by a multi-institutional group of otolaryngology and interventional radiology experts in the field of vascular anomalies to formulate a list of recommended reporting outcomes for the study and treatment of head and neck lymphatic malformations. Through 3 rounds of iteration, 10 expert panelists refined 98 proposed outcome measures and 9 outcome categories to a final consensus set of 50 recommended outcome measures in 3 global categories (general, demographics, and treatment complications) and 5 site-specific categories (orbit, oral cavity, pharynx, larynx, and neck). We propose the first consensus set of standardized reporting measures for clinical and treatment outcomes in studies of HNLMs. Consistent outcome measures across future studies will facilitate comparison of treatment options and allow systematic review. We hope that these guidelines facilitate the design and reporting of subsequent HNLM studies. © American Academy of Otolaryngology—Head and Neck Surgery Foundation 2015.
Article
Vitiligo is the most common depigmentating disorder for which the 2010 Cochrane systematic review of interventions for vitiligo concluded there was a need for unified outcome measures to harmonize trial designs and allow meta-analyses. In this context, we performed a three stage, web-based, international electronic Delphi consensus study involving 101 participants from 3 stakeholder groups from 24 countries (dermatologists and researchers interested in vitiligo, patients with vitiligo, representatives of regulatory agencies, and journal editors). Participants were identified through the International Federation of Pigment Cell Societies (Asian, Japanese, European and Pan-american). Consensus was pre-defined as being achieved if >75% of participants in two stakeholder groups agreed that a domain (outcome) should be included in the core outcomes set. Three domains were deemed essential, i.e. repigmentation, side effects and harms of treatment, and maintenance of gained repigmentation. An additional four domains were recommended (to be reported if relevant to the intervention and trial design). These were cosmetic acceptability of results, quality of life, cessation of spreading of vitiligo, and tolerability/burden of treatment. Core outcomes domains were identified with a high degree of consensus amongst different stakeholder groups internationally. Future research is needed to develop and validate scales to measure each identified domain.This article is protected by copyright. All rights reserved.
Article
Mulliken and Glowacki, in 1982 created a classification system of vascular anomalies which divided vascular anomalies into tumors and malformations which provided the framework for great advances in the management of these patients. This classification system was recently expanded at the 2014 ISSVA workshop in Melbourne. This revision again provides much greater detail including newly named anomalies and identified genes to account for recent advances in knowledge and clinical associations. (C) 2014 Published by Elsevier Inc.
Consensus measurement in Delphi studies
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