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Abstract

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.
Research letter
Finalizing the international core domain set for
peripheral vascular malformations: the OVAMA
project
DOI: 10.1111/bjd.18043
DEAR EDITOR, There is a variety of outcome reporting in the
clinical research on peripheral vascular malformations,
13
including capillary, venous, lymphatic, arteriovenous and
combined malformations. Without harmonization of outcome
measures, treatments cannot be properly compared. This ham-
pers the development of evidence-based treatment guidelines,
urgently needed for these challenging congenital conditions.
The mission of the Outcome measures for VAscular MAlfor-
mations (OVAMA) project is to uniform outcome reporting in
clinical research.
To evaluate treatment efficacy, the first step is deciding on
what to measure. 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’, ‘radio-
logical imaging’ and ‘lymphatic fluid leakage’.
4
A face-to-face
consensus meeting was organized to establish the final CDS.
As an addendum to the previous study, this letter describes
the conclusions of this face-to-face meeting and reports the
final CDS for peripheral vascular malformations.
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, otolaryngol-
ogy, paediatrics, paediatric haematology/oncology, radiology
and dermatology) and 15% were patient organization repre-
sentatives.
An overview of the e-Delphi and online consensus meeting
results was sent to all participants beforehand, and printed
summaries were provided. The undecided domains were then
separately discussed by the whole group and a final consensus
reached. In order to reach different results than in the online
consensus meeting, group unanimity on including/dropping/
changing each outcome domain was required before proceed-
ing to the next. The final CDS is presented in Figure 1.
The provisionally included domain ‘recurrence’ was
excluded from the final CDS, as participants agreed that it was
a reflection of other domains rather than a distinct domain.
The domain ‘appearance’, defined as the visible anatomical
characteristics of the vascular malformation such as size, col-
our and texture, was excluded from the e-Delphi study; how-
ever, it was considered essential during the online consensus
meeting. Participants noted during the e-Delphi study that
‘appearance’ may be confused with ‘body image’. As ‘appear-
ance’ often initiates treatment, participants suggested that it
should be incorporated in the final CDS. As it was considered
relevant from both the patient’s and the clinician’s perspective,
‘appearance’ was included as a patient-reported and clinician-
reported core domain.
‘Radiological imaging’ was found to be the instrument by
which the radiological characteristics are evaluated, so this
domain was changed to ‘radiological characteristics’. Because
follow-up radiological imaging is not routinely performed in
all cases, the domain ‘radiological characteristics’ was not con-
sidered obligatory, and was hence excluded from the final
CDS. However, if radiological imaging is performed before
and after treatment, it should be reported.
The group concluded that diagnosing ‘lymphatic fluid leak-
age’ requires medical knowledge that cannot be expected of
patients. Consequently, it was moved from the patient-
reported ‘symptoms’ to the ‘clinician-reported ‘signs’ in the
final CDS.
The general opinion of the group was that the domain cate-
gories ‘patient satisfaction’ and ‘adverse events’ should be
included in the final CDS, but were only relevant after treat-
ment has started, and therefore should only be measured at
follow-up.
No other domains or discussion points were left unresolved.
With this face-to-face consensus meeting, we successfully
finalized the CDS for clinical research in peripheral vascular
malformations (Fig. 1).
As measurement of these domains does not require invasive
or costly techniques, measurement of the relatively high num-
ber of domains is still considered feasible. By including many
international experts in the field and patients, this process
ensured a diversity of perspectives. The face-to-face set-up and
information provided beforehand enabled in-depth discussion
and enhanced participant engagement. An unavoidable limita-
tion was that only stakeholders present at the ISSVA
conference were able to participate.
©2019 The Authors. British Journal of Dermatology
published by John Wiley & Sons Ltd on behalf of British Association of Dermatologists
British Journal of Dermatology (2019) 1
This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use,
distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
This project represents a significant step towards mean-
ingful assessment and comparison of treatments for periph-
eral vascular malformations. The next step towards uniform
outcome reporting is determining how to measure these core
domains, i.e. developing a core outcome measurement
set. This project, involving an appraisal of available instru-
ments and development of a new disease-specific instru-
ment, is currently being carried out by the OVAMA
Steering Group.
M.M. LOKHORST
1
iD
S.E.R. HORBACH
1
iD
C.M.A.M. van der HORST
1
P.I. SPULS
2
On behalf of the
OVAMA Steering Group
1
Department of Plastic, Reconstructive and
Hand Surgery, Amsterdam University
Medical Centers, University of
Amsterdam, the Netherlands
2
Department of Dermatology, Amsterdam
University Medical Centers, University of
Amsterdam, the Netherlands
E-mail: m.m.lokhorst@amc.uva.nl
S.E.R. Horbach was OVAMA project coordinator during this study.
A full list of the OVAMA Steering Group collaborators is provided in
Appendix S1 (see Supporting Information).
References
1 Horbach SE, Lokhorst MM, Saeed P et al. Sclerotherapy for low-flow
vascular malformations of the head and neck: a systematic review
of sclerosing agents. J Plast Reconstr Aesthet Surg 2016; 69:295304.
2 Horbach SE, Rigter IM, Smitt JH et al. Intralesional bleomycin injec-
tions for vascular malformations: a systematic review and meta-ana-
lysis. Plast Reconstr Surg 2016; 137:24456.
3 Langbroek GB, Horbach SE, van der Vleuten CJ et al. Compression
therapy for congenital low-flow vascular malformations of the
extremities: a systematic review. Phlebology 2018; 33:513.
4 Horbach SER, van der Horst C, Blei F et al. Development of an inter-
national core outcome set for peripheral vascular malformations:
the OVAMA project. Br J Dermatol 2018; 178:47381.
5 Boers M, Kirwan JR, Wells G et al. Developing core outcome mea-
surement sets for clinical trials: OMERACT filter 20. J Clin Epidemiol
2014; 67:74553.
Funding sources: none.
Conflicts of interest: none to declare.
Supporting Information
Additional Supporting Information may be found in the online
version of this article at the publisher’s website:
Appendix S1 OVAMA SteeringGroup collaborators.
Fig. 1. Core domain set for peripheral vascular malformations. Domain categories and domains were based on the classification as reported in
Appendix S2 of the previously published core outcome set development study (Horbach SER, van der Horst C, Blei F et al. Development of an
international core outcome set for peripheral vascular malformations: the OVAMA project. Br J Dermatol 2018; 178: 47381).
a
Only relevant at
follow-up. AVM, only for arteriovenous malformations, LM, only for lymphatic malformations, VM, only for venous malformations.
©2019 The Authors. British Journal of Dermatology
published by John Wiley & Sons Ltd on behalf of British Association of Dermatologists
British Journal of Dermatology (2019)
2Research letter
... [5][6][7] The mechanism leading to pain is, however, often 1 dependent on the VM type. While venous stasis is predominantly responsible for the onset of 2 pain in venous malformations, arteriovenous shunting reduces capillary oxygen delivery causing 3 ischemia and ischemic pain in arteriovenous malformations, which is a more severe condition 4 and heralds the risk of ulceration, bleeding, and even congestive heart failure. 7 Therefore, the 5 symptom pain should be considered as a part of a more comprehensive issue. ...
... Therefore, the aim of this study was to investigate pain and risk factors for pain in patients with 21 peripheral VMs by using the novel condition-specific OVAMA (Outcome measures for 22 J o u r n a l P r e -p r o o f VAscular MAlformations) questionnaire. 4,11,12 Furthermore, we assessed how pain affects the 1 QoL in patients with VMs. maximal diameter) was also registered in the following categories: 0-5 cm, 5-10 cm, 10-30 cm, 1 and >30 cm. ...
... p<0.001). Quality of life 4 The T-scores of the PROMIS scales were calculated, median T-scores are shown in Table IV. 5 The mean rank of the T-scores were compared between patients who experienced pain and 6 patients who did not. Statistically significant different T-scores were found on the following 7 scales in adults: pain interference (p<0.001), ...
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Objective Vascular malformations can negatively impact the patient’s quality of life. Pain is a common problem in these patients. The aim of this study was to investigate risk factors associated with pain and to assess how pain affects quality of life. Methods This prospective cross-sectional study was conducted in a tertiary vascular anomaly expertise center. Between June and December 2020, all patients from our local database (334 adults and 189 children) with peripheral vascular malformations were invited to complete the Outcome Measures for VAscular MAlformations (OVAMA) questionnaire to evaluate the presence, frequency, and intensity of pain. Additionally, patients were asked to complete several Patient-Reported Outcome Measurement Information System (PROMIS) scales to evaluate their quality of life. Risk factors associated with pain were identified in bivariate analysis and multivariable logistic regression. Quality of life domains were compared between patients who experienced pain and patients who did not. Results A total of 164 patients completed the questionnaire about pain and 133 patients completed all quality of life questionnaires. Approximately half of the patients (52%) reported pain in the past four weeks and 57% of these patients reported pain daily or several times a week. Female gender (p=0.009), lesions located in the upper extremity (p<0.001) or lower extremity (p<0.001), and intramuscular/intraosseous lesions (p=0.004) were independently associated with the presence of pain. The following quality of life domains were diminished in patients who experienced pain in comparison to patients who did not: pain interference (p<0.001), physical functioning (p<0.001), and social participation (p<0.001) in adults, and pain interference (p=0.001), mobility (p=0.001), and anxiety (p=0.024) in children. Conclusion Pain is a frequently reported complaint in patients with vascular malformations and is present in approximately half of the patients. Patients with lesions located in the upper or lower extremity, intramuscular/intraosseous lesions, and female patients are more likely to experience pain. The presence of pain negatively impacted patients’ quality of life. Although VM are a benign condition and expectative management is frequently applied, our study shows that pain is a serious concern and needs to be actively assessed. Pain is a sign of various etiologies, which should be examined in order to properly treat the pain.
... 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.
... 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 No GRC scale for the SF-36 subscale 'vitality' was formulated, because it was not considered a core outcome domain for patients with vascular malformations. 3,8 The change was reported on a seven-point Likert scale, based on previously reported GRC scales and the subjective significance questionnaire of Osoba. 19,20 Response options included 'very much worse', 'worse', 'somewhat worse', 'no change', 'somewhat better', 'better' and 'very much better'. ...
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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.
... Recently, as part of the Outcome measures for VAscular Malformations (OVAMA) project, a core domain set for vascular malformations was developed, including disease-specific symptoms, appearance, and quality of life. 6 Our opinion is that instead of measuring "recurrence" or clinician-reported "success rates," it is better to assess improvement or worsening in these core outcome domains with validated patient-reported outcome measurements, and possibly an additional quantitative analysis of regrowth of the lesion with imaging. ...
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Background: Surgical treatment of peripheral vascular malformations is widely performed as primary and secondary treatments. Excellent results have been reported; however, it is thought that complications are likely to occur because of damage to adjacent structures. This systematic review aimed to elucidate the indications and outcomes of surgical treatment of vascular malformations. Methods: PubMed, EMBASE, and the Cochrane Central Register of Controlled Trials were searched for studies reporting outcomes of surgery in at least 15 patients with a single type of peripheral soft-tissue vascular malformation. The authors extracted data on patient and lesion characteristics, treatment characteristics, and outcomes (including complications). Meta-analysis was conducted on recurrence and complication rates. Results: A total of 3042 articles were found, of which 24 were included: nine studies on arteriovenous malformations, seven on venous malformations, and eight on lymphatic malformations, totaling 980 patients. Meta-analyses showed pooled proportions for recurrences of 11 percent in arteriovenous malformations, 5 percent in venous malformations, and 9 percent in lymphatic malformations. Pooled proportions of major complications were 9 percent for arteriovenous malformations, 3 percent for venous malformations, and 1 percent for lymphatic malformations. The authors found a 5 percent pooled recurrence proportion in total resections, compared with 28 percent in subtotal resections. The pooled odds ratio for recurrence in total and subtotal resections showed a significant lower recurrence rate after total resection (odds ratio, 0.14, p = 0.02). Conclusions: Surgical treatment of vascular malformations appears to be effective and safe in many cases. However, it seems that surgery is performed predominantly in small lesions, and subtotal resection has a higher risk of recurrence than total resection.
... For this reason alone, it is essential that teams use standardised outcome measures to gather some evidence of efficacy for individual patients, for specific interventions and for different disease entities. The OVAMA project is working towards uniform outcome reporting in clinical research on peripheral vascular malformations (Lokhorst et al. 2019). Tools such as quality of life (QoL) assessments also help children and families to give structured feedback and gain some personal objectivity in the treatment of their condition. ...
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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 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.
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.