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Abstract

Vascular malformations are congenital anomalies of the vascular and/or lymphatic system that affect the head and neck region. The most common treatment options are sclerotherapy, laser therapy, surgery, and embolization. Because vascular malformations are variable in type, size, extent, and location, it is a challenge to select methods for evaluation of treatment outcome. Without standardized outcome reporting, it is difficult to compare and combine scientific evidence to support therapeutic decision making. Standardized collection and reporting of outcome data are the first steps toward a fair comparison between treatments. This article describes outcome measurements for vascular malformations and initiatives to improve outcome reporting.

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Chapter
In their very useful introduction to this textbook, C. Carrie Liu and Jennifer Shin describe evidence-based medicine as “the systematic search and evaluation of literature and then applying these findings to clinical decision-making.” This succinct description is intuitively appealing to any clinician hoping to improve his or her own clinical decision-making, and the authors expand it into a discussion that will prove a valuable reference for any reader of this book.
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Background: An important limitation in vascular malformation research is the heterogeneity in outcome measures used for the evaluation of treatment outcome. Objective: The Outcome measures for VAscular MAlformations (OVAMA) project aimed to reach international consensus on a core outcome set (COS) for clinical research on peripheral vascular malformations: lymphatic (LM), venous (VM) and arteriovenous malformations (AVM). In this consensus study, we determined what domains should constitute the COS. Methods: Thirty-six possibly relevant outcome domains were proposed to an international group of physicians, patients and the parents of patients. In a 3-round e-Delphi process using online surveys, participants repeatedly rated the importance of these domains on a 5-point Likert scale. Participants could also propose other relevant domains. This process was performed for LM, VM and AVM separately. Consensus was pre-defined as 80% agreement on the importance of a domain amongst both the physician group and the patient/parent group. Outcomes were then reevaluated in an online consensus meeting. Results: 167 physicians and 134 patients and parents of patients with LM (n=50), VM (n=71) and AVM (n=29) participated in the study. After three rounds and a consensus meeting, consensus was reached for all three types of vascular malformations on the core domains of radiological assessment, physician-reported location-specific signs, patient-reported severity of symptoms, pain, quality of life, satisfaction and adverse events. Vascular malformation type-specific signs and symptoms were included for LM, VM and AVM, separately. Conclusion: It is recommended to measure at least these core outcome domains in therapeutic-efficacy studies on peripheral vascular malformations. This article is protected by copyright. All rights reserved.
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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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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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This article is a report of the fourth meeting of the Harmonising Outcome Measures for Eczema (HOME) initiative held in Malmö, Sweden on 23-24 April 2015 (HOME IV). The aim of the meeting was to achieve consensus over the preferred outcome instruments for measuring patient-reported symptoms and quality of life for the HOME core outcome set for atopic eczema (AE). Following presentations, which included data from systematic reviews, consensus discussions were held in a mixture of whole group and small group discussions. Small groups were allocated a priori to ensure representation of different stakeholders and countries. Decisions were voted on using electronic keypads. For the patient-reported symptoms, the group agreed by vote that itch, sleep loss, dryness, redness/inflamed skin and irritated skin were all considered essential aspects of AE symptoms. Many instruments for capturing patient-reported symptoms were discussed [including the Patient-Oriented SCOring Atopic Dermatitis index, Patient-Oriented Eczema Measure (POEM), Self-Administered Eczema Area and Severity Index, Itch Severity Scale, Atopic Dermatitis Quickscore and the Nottingham Eczema Severity Score] and, by consensus, POEM was selected as the preferred instrument to measure patient-reported symptoms. Further work is needed to determine the reliability and measurement error of POEM. Further work is also required to establish the importance of pain/soreness and the importance of collecting information regarding the intensity of symptoms in addition to their frequency. Much of the discussion on quality of life concerned the Dermatology Life Quality Index and Quality of Life Index for Atopic Dermatitis; however, consensus on a preferred instrument for measuring this domain could not be reached. In summary, POEM is recommended as the HOME core outcome instrument for measuring AE symptoms.
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Background and objectives: Complicated vascular anomalies have limited therapeutic options and cause significant morbidity and mortality. This Phase II trial enrolled patients with complicated vascular anomalies to determine the efficacy and safety of treatment with sirolimus for 12 courses; each course was defined as 28 days. Methods: Treatment consisted of a continuous dosing schedule of oral sirolimus starting at 0.8 mg/m(2) per dose twice daily, with pharmacokinetic-guided target serum trough levels of 10 to 15 ng/mL. The primary outcomes were responsiveness to sirolimus by the end of course 6 (evaluated according to functional impairment score, quality of life, and radiologic assessment) and the incidence of toxicities and/or infection-related deaths. Results: Sixty-one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6, and 53 were evaluable at the end of course 12. No patient had a complete response at the end of course 6 or 12 as anticipated. At the end of course 6, a total of 47 patients had a partial response, 3 patients had stable disease, and 7 patients had progressive disease. Two patients were taken off of study medicine secondary to persistent adverse effects. Grade 3 and higher toxicities attributable to sirolimus included blood/bone marrow toxicity in 27% of patients, gastrointestinal toxicity in 3%, and metabolic/laboratory toxicity in 3%. No toxicity-related deaths occurred. Conclusions: Sirolimus was efficacious and well tolerated in these study patients with complicated vascular anomalies. Clinical activity was reported in the majority of the disorders.
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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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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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Inconsistent reporting of outcomes in clinical trials of patients with non-specific low back pain (NSLBP) hinders comparison of findings and the reliability of systematic reviews. A core outcome set (COS) can address this issue as it defines a minimum set of outcomes that should be reported in all clinical trials. In 1998, Deyo et al. recommended a standardized set of outcomes for LBP clinical research. The aim of this study was to update these recommendations by determining which outcome domains should be included in a COS for clinical trials in NSLBP. An International Steering Committee established the methodology to develop this COS. The OMERACT Filter 2.0 framework was used to draw a list of potential core domains that were presented in a Delphi study. Researchers, care providers and patients were invited to participate in three Delphi rounds and were asked to judge which domains were core. A priori criteria for consensus were established before each round and were analysed together with arguments provided by panellists on importance, overlap, aggregation and/or addition of potential core domains. The Steering Committee discussed the final results and made final decisions. A set of 280 experts was invited to participate in the Delphi; response rates in the three rounds were 52, 50 and 45 %. Of 41 potential core domains presented in the first round, 13 had sufficient support to be presented for rating in the third round. Overall consensus was reached for the inclusion of three domains in this COS: 'physical functioning', 'pain intensity' and 'health-related quality of life'. Consensus on 'physical functioning' and 'pain intensity' was consistent across all stakeholders, 'health-related quality of life' was not supported by the patients, and all the other domains were not supported by two or more groups of stakeholders. Weighting all possible argumentations, the Steering Committee decided to include in the COS the three domains that reached overall consensus and the domain 'number of deaths'. The following outcome domains were included in this updated COS: 'physical functioning', 'pain intensity', 'health-related quality of life' and 'number of deaths'. The next step for the development of this COS will be to determine which measurement instruments best measure these domains.
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Prostate cancer is a growing health problem worldwide. The management of localised prostate cancer is controversial. It is unclear which of several surgical, radiotherapeutic, ablative, and surveillance treatments is the most effective. All have cost, process and recovery, and morbidity implications which add to treatment decision-making complexity for patients and healthcare professionals. Evidence from randomised controlled trials (RCTs) is not optimal because of uncertainty as to what constitutes important outcomes. Another issue hampering evidence synthesis is heterogeneity of outcome definition, measurement, and reporting. This project aims to determine which outcomes are the most important to patients and healthcare professionals, and use these findings to recommend a standardised core outcome set for comparative effectiveness trials of treatments for localised prostate cancer, to optimise decision-making. The range of potentially important outcomes and measures will be identified through systematic reviews of the literature and semi-structured interviews with patients. A consultation exercise involving representatives from two key stakeholder groups (patients and healthcare professionals) will ratify the list of outcomes to be entered into a three round Delphi study. The Delphi process will refine and prioritise the list of identified outcomes. A methodological substudy (nested RCT design) will also be undertaken. Participants will be randomised after round one of the Delphi study to one of three feedback groups, based on different feedback strategies, in order to explore the potential impact of feedback strategies on participant responses. This may assist the design of a future core outcome set and Delphi studies. Following the Delphi study, a final consensus meeting attended by representatives from both stakeholder groups will determine the final recommended core outcome set. This study will inform clinical practice and future trials of interventions of localised prostate cancer by standardising a core outcome set which should be considered in comparative effectiveness studies for localised prostate cancer.
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Port-wine stains are a type of capillary malformation affecting 0.3% to 0.5% of the population. Port-wine stains present at birth as pink to erythematous patches on the skin and/or mucosa. Without treatment, the patches typically darken with age and may eventually develop nodular thickening or associated pyogenic granuloma. Laser and light treatments provide improvement through selective destruction of vasculature. A variety of vascular-selective lasers may be employed, with the pulsed dye laser being the most common and well studied. Early treatment produces more optimal results. Advances in imaging and laser treatment technologies demonstrate potential to further improve clinical outcomes.
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Background:: An ever-growing range of facial cosmetic products and treatments are available, but little clinical research is being done to understand treatment outcomes from the patients' perspective. The FACE-Q is a patient-reported outcome (PRO) instrument composed of more than 40 independently functioning scales and checklists. The aim of this paper is to describe the development and psychometric evaluation of five new FACE-Q scales. Methods:: FACE-Q scales were developed according to international guidelines for PRO instrument development. The following FACE-Q scales and a single symptom checklist (Recovery Early Symptoms) were evaluated in this study: Psychological Wellbeing; Social Function; Satisfaction with Decision to have Treatment; Satisfaction with Outcome of Treatment; and Early Life Impact of Treatment. Modern and traditional psychometric methods were used to examine reliability, validity and responsiveness. Results:: The sample included 702 participants from three studies. The FACE-Q scales were found to be reliable, valid, and responsive to clinical change. These findings were supported by Rasch Measurement Theory (e.g., overall chi-square values of p ≥ 0.06; Person Separation Index ≥ 0.81), traditional psychometric (e.g., Cronbach's alpha values ≥0.90) and responsiveness (i.e., significant improvement following facelift and lip treatment) analysis. Conclusion:: The FACE-Q measures concepts and symptoms important to facial aesthetic patients. The 5 scales and single symptom checklist described here can be used to measure what patients think about cosmetic treatments in a scientifically sound manner. As the cosmetics industry continues to expand, the patient perspective of treatment outcomes should be measured and reported.Level of Evidence: Diagnostic, III. Paper: limit each article to 3000 words, not including references or abstract.
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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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Port wine stains (PWS) are the most common vascular malformation of the skin, occurring in 0.3% to 0.5% of the population. Noninvasive laser irradiation with flashlamp-pumped pulsed dye lasers (selective photothermolysis) currently comprises the gold standard treatment of PWS; however, the majority of PWS fail to clear completely after selective photothermolysis. In this review, the clinically used PWS treatment modalities (pulsed dye lasers, alexandrite lasers, neodymium:yttrium-aluminum-garnet lasers, and intense pulsed light) and techniques (combination approaches, multiple passes, and epidermal cooling) are discussed. Retrospective analysis of clinical studies published between 1990 and 2011 was performed to determine therapeutic efficacies for each clinically used modality/technique. In addition, factors that have resulted in the high degree of therapeutic recalcitrance are identified, and emerging experimental treatment strategies are addressed, including the use of photodynamic therapy, immunomodulators, angiogenesis inhibitors, hypobaric pressure, and site-specific pharmaco-laser therapy.
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Port-wine stains can be treated with a flash-lamp-pumped pulsed-dye laser, but it is uncertain whether this treatment is more effective if administered early in life, when the skin is thinner and the lesion is smaller. We prospectively studied 100 patients with a previously untreated port-wine stain of the head or neck. They were treated with the flash-lamp-pumped pulsed-dye laser and divided into four age groups (0 to 5, 6 to 11, 12 to 17, and 18 to 31 years). The outcome measure was lightening of the port-wine stain (reduction in the difference in color between the skin with the stain and contralateral healthy skin) as measured with a colorimeter after an average of five treatments (range, three to seven) of the entire lesion. Of the 100 patients, 11 could not be included in the analysis because they had received fewer than three or more than seven treatments, had an erroneous base-line color measurement, or were lost to follow-up. The sizes, locations, and colors of the port-wine stains were similar among the groups. When all 89 patients were analyzed together, the average reduction in the difference in color between the skin with the port-wine stain and contralateral healthy skin was 40 percent. The differences between age groups in the average reduction in color differences were not significant (P= 0.26). By the end of the study, only 7 of 89 patients had completed laser therapy, and in no case was clearance complete. Treatment was discontinued in all seven because the last three treatments did not lead to further lightening. We found no evidence that treatment of port-wine stains with the flash-lamp-pumped pulsed-dye laser in early childhood is more effective than treatment at a later age.
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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.
Article
Objectives To assess the treatment-induced changes of health-related quality-of-life (HRQoL) in patients with venous malformations (VM) who underwent ethanol gel sclerotherapy. Methods The prospective study in children and adults was approved by the local ethics committee. 31 patients (mean age 23.42 years, range 6.6 – 46.5; 26 female, 5 male) with VM were included. Patients’ self-assessed HRQoL was measured before and after treatment using psychometrically validated questionnaires for adults and children. Differences were analysed with a paired t test. Results58 sclerotherapy sessions were performed. The Physical Component Summary (PCS) at baseline was 43.69 and increased significantly (p = 0.01122) to 48.95 after treatment. The bodily pain (BP) scale increased significantly from 37.94 to 48.56 (p = 0.00002), the general health (GH) scale increased significantly from 46.69 to 52.17 (p = 0.00609). Baseline Physical Summary Score (PHS) in children increased significantly after treatment (p < 0.00001) from 25.25 to 45.89. The baseline Psychosocial Summary Score (PSS) in children was 51.08 and increased significantly (p = 0.00031) after treatment to 58.84. Conclusion Paediatric and adult patients with VM suffer from bodily pain with overall reduced physical functioning. After sclerotherapy, these restrictions are successfully returning to normal levels with a positive effect on mental and psychosocial domains. Key points• Sclerotherapy in venous malformation patients has an unknown effect on health-related quality-of-life• Prospective study showed improvements in bodily pain and general health in adults• Children and adults improved from treatment with ethanol gel• Sclerotherapy is an appropriate therapy
Article
Purpose: To assess cost-effectiveness of sclerotherapy for venous malformations (VMs) to improve patient quality of life (QOL). Materials and methods: This prospective study enrolled 28 patients with symptomatic VMs who underwent sclerotherapy. EuroQol-5 Dimension (EQ-5D) and Short-Form 36 (SF-36) Health Survey were used to measure health-related QOL. Questionnaires were collected before and 1, 3, 6, and 12 months after sclerotherapy. Quality-adjusted life years (QALYs) were calculated using EQ-5D score as a measure of health utility. Medical costs obtained from the hospital accounting system and other costs of staff, drugs, materials, and angiographic equipment were calculated for each procedure. Cost-effectiveness was analyzed using incremental cost-effectiveness ratio (ICER) as the medical cost/gain of QALYs. Results: Median EQ-5D scores improved from 0.768 (range, 0.705-1) to 1 (range, 0.768-1) after 6 months (P = .023) and 1 (range, 0.768-1) after 12 months (P = .063). The gain of QALYs at 12 months was 0.043. The mean medical cost was ¥281,228 ($2,337). The pain group (baseline bodily pain scale of SF-36 score < 70) showed greater improvement in median EQ-5D score, from 0.705 (range, 0.661-0.768) to 0.768 (range, 0.705-1) after 6 months (P = .041) and 0.768 (range, 0.768-1) after 12 months (P = .049). ICER at 12 months was ¥6,600,483 ($54,840) in the overall group and decreased to ¥3,998,113 ($33,218) in the pain group, < ¥6,000,000 ($49,850), threshold for acceptance of a public health benefit in Japan, even accounting for 50% increase in costs. Conclusions: Sclerotherapy was cost-effective for improving QOL for symptomatic VMs, especially for patients with moderate to severe pain.
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
The Lymphatic Malformation Function (LMF) instrument is a preliminary parent-report assessment designed to measure outcomes in children with cervicofacial lymphatic malformation (LM). This study aimed to assess the measurement properties of the LMF, refine it, test criterion validity, and evaluate the test-retest reliability. Cross-sectional. Two pediatric tertiary referral centers. Parents of 60 children from 6 months to 15 years old with cervicofacial LM. Parents were recruited via mail and online. The LMF was administered on paper or online initially and again within 21 days. Response distributions and interitem correlations were examined for item reduction. Exploratory factor analysis was conducted on retained items. Cronbach's α, Spearman correlation, and intraclass correlation (ICC) coefficients were calculated to test internal consistency, criterion validity (compared to stage), and test-retest reliability, respectively. One item was removed due to a floor effect. The response scale was collapsed from a 5-point scale to a 3-point scale due to skewness. Six items were discarded due to redundancy (interitem correlations >0.7); 2 items were discarded due to factor loadings <0.4. Exploratory factor analysis revealed a 2-factor structure explaining 84% of variance, and the domains Signs and Impacts had good internal consistency (all Cronbach's α >0.80 and <0.90), significant association with stage (P < .05), and good overall test-retest reliability (ICC, 0.82). The LMF has been refined into a 12-item, 2-domain instrument measuring LM-specific signs and impacts with internal consistency, criterion validity, and test-retest reliability. © American Academy of Otolaryngology—Head and Neck Surgery Foundation 2015.
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.
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Lymphatic malformations can be challenging to treat. Mainstay interventions including surgery and sclerotherapy are invasive and can result in local recurrence and complications. We sought to assess the effect of 20 weeks of oral sildenafil on reducing lymphatic malformation volume and symptoms in children. Seven children (4 boys, 3 girls; ages 13-85 months) with lymphatic malformations were given oral sildenafil for 20 weeks in this open-label study. The volume of the lymphatic malformation was calculated blindly using magnetic resonance imaging performed before and after 20 weeks of sildenafil. Lymphatic malformations were assessed clinically on weeks 4, 12, 20, and 32. Both the physician and parents evaluated the lymphatic malformation in comparison with baseline. Four subjects had a lymphatic malformation volume decrease (1.0%-31.7%). In 2 subjects, despite a lymphatic malformation volume increase (1.1%-3.7%), clinical improvement was noted while on sildenafil. One subject had a 29.6% increase in lymphatic malformation volume and no therapeutic response. Lymphatic malformations of all 6 subjects who experienced a therapeutic response on sildenafil softened and became easily compressible. Adverse events were minimal. A randomized controlled trial will be necessary to verify the effects of sildenafil on lymphatic malformations. Sildenafil can reduce lymphatic malformation volume and symptoms in some children.
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This systematic review assessed the available published evidence on the safety and efficacy of percutaneous sclerotherapy for patients with congenital vascular malformations. A systematic search of various electronic bibliographic databases was conducted in May 2013. A grey literature search was also performed. Study selection, data extraction, and assessment of study quality were undertaken by one reviewer and checked by a second. One systematic review, one randomized controlled trial, one non-randomized controlled trial, and 24 case series studies were included. However, deficiencies in reporting, small sample sizes, and marked inter-study heterogeneity precluded a definitive synthesis of the data. Ethanol sclerotherapy appeared to be potentially effective for treating venous malformations, with the majority of patients achieving some lesion regression. However, it is associated with a 16% risk of major complications, including deep tissue injury, deep vein thrombosis, and nerve injury. While there was limited evidence that sclerotherapy with OK-432 was an effective treatment for lymphatic malformations, evidence for the use of sclerotherapy in patients with arteriovenous malformations was unclear. Very limited evidence from case series studies suggested that sclerotherapy with ethanol and OK-432, administered over multiple sessions, was effective in the treatment of venous and lymphatic malformations, respectively. However, the value of percutaneous sclerotherapy as a pre-treatment for or an alternative to surgery is not known. Further evidence is required to delineate which patients will benefit most from percutaneous sclerotherapy to ensure that the advantages of treatment will outweigh the risks.
Article
Because the best possible treatment for venous malformations is unclear, this study systematically reviews the available literature regarding the effectiveness of different treatment options for the patient group. Venous malformations result from incorrect development of the veins during embryogenesis and are present at birth. Venous malformations may exhibit symptoms, such as pain, swelling, and inflammation of the vessel. A systematic literature search in PubMed and Embase was performed. Data regarding the design, participants, intervention and, treatment outcome (success and complications) were extracted. The validity of the studies was assessed with the Cochrane Collaboration's risk of bias tool. Thirty-five studies were identified studying the effectiveness of eight treatments: sclerotherapy/embolization with ethanol, gelified ethanol, bleomycin, polidocanol, sodium tetradecyl sulfate (STS), Ethibloc, surgery, and laser therapy. All of the included studies have a high or unclear risk of bias. The average biased reported success rates for ethanol, gelified ethanol, bleomycin, polidocanol, STS, Ethibloc, surgery, and laser therapy were 74, 89, 88, 90, 86, 65, 90, and 94 %, respectively. Until more valid evidence is available, the choice for treatment remains a shared decision between the patient and a multidisciplinary treatment group. From a cost perspective, sclerotherapy with STS or polidocanol should be the treatment of choice.
Article
Objective: Lymphatic malformations cause significant symptoms and functional deficits. Patients seek care for functional and symptomatic effects of their disease, but current disease burden and treatment outcome measures focus primarily on anatomy and histopathology. The authors describe disease impacts reported by patients and parents as a step toward more comprehensive disease burden assessments. Study design: Cross-sectional. Setting: Children's hospital vascular anomaly clinic. Subjects and methods: Participants were recruited through a pediatric vascular anomaly clinic. A panel of senior pediatric otolaryngologists and an outcomes scientist developed interview questions based on clinical and research experience and available literature. The outcomes scientist conducted parent and adolescent interviews. The panel reviewed responses to define relevant items within functional domains. Participants rated impact on daily life for each domain. Results: Thirty-one participants represented all 5 de Serres stages (mean [SD] age, 9 [6] years; n = 11 adolescents and 20 parents). Adolescents reported frequent sickness as the domain with greatest impact. Sleep was more affected in adolescents with higher stage lesions. Parents of younger children reported greatest impact on breastfeeding. For adolescents, lesion stage predicted perceived social stigma (controlling for age), whereas increasing age was associated with greater impact from swelling (controlling for stage). For parents, stage predicted breastfeeding impact (controlling for stage). Conclusion: This is the first detailed assessment of patient- and parent-reported functional and symptomatic impacts of head and neck lymphatic malformations. Both adolescent patients and parents of younger children reported significant symptom and functional effects of this disease.
The COSMIN checklist for assessing the methodological quality of studies on measurement properties of health status measurement instruments: an international Delphi study
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