Project

Outcome measures for vascular malformations (OVAMA) project

Goal: The mission of the Outcome measures for VAscular MAlformations (OVAMA) project is to uniform outcome reporting in clinical research on vascular malformations. At first, a set of core outcome domains was developed. The next step is selecting or developing appropriate outcome measurement instruments for measuring the core domains, i.e. developing a core outcome measurement set. Currently, we are almost finished developing a disease-specific questionnaire: the OVAMA questionnaire.

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Max M Lokhorst
added a research item
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.
Max M Lokhorst
added a research item
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.
Max M Lokhorst
added an update
Have a look at our website: www.ovama.org. Here we explain our current projects and future plans.
 
Max M Lokhorst
added 3 research items
Purpose The Outcome measures for vascular malformation (OVAMA) group reached consensus on the core outcome domains for the core outcome set (COS) for peripheral vascular malformations (venous, lymphatic and arteriovenous malformations). However, it is unclear which instruments should be used to measure these domains. Therefore, our aims were to identify all outcome measurement instruments available for vascular malformations, and to evaluate their measurement properties. Methods With the first literature search, we identified outcomes and instruments previously used in prospective studies on vascular malformations. A second search yielded studies on measurement properties of patient- and physician-reported instruments that were either developed for vascular malformations, or used in prospective studies. If the latter instruments were not specifically validated for vascular malformations, we performed a third search for studies on measurement properties in clinically similar diseases (vascular or lymphatic diseases and benign tumors). We assessed the methodological quality of these studies following the Consensus-based Standards for the selection of health Measurement Instruments methodology, and evaluated the quality of the measurement properties. Results The first search yielded 27 studies, none using disease-specific instruments. The second and third search included 22 development and/or validation studies, concerning six instruments. Only the Lymphatic Malformation Function Instrument was developed specifically for vascular malformations. Other instruments were generic QoL instruments developed and/or partly validated for clinically similar diseases. Conclusions Additional research on measurement properties is needed to assess which instruments may be included in the COS. This review informs the instrument selection and/or the development of new instruments. Systematic review registration PROSPERO, 42017056242.
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.
Max M Lokhorst
added a project goal
The mission of the Outcome measures for VAscular MAlformations (OVAMA) project is to uniform outcome reporting in clinical research on vascular malformations. At first, a set of core outcome domains was developed. The next step is selecting or developing appropriate outcome measurement instruments for measuring the core domains, i.e. developing a core outcome measurement set. Currently, we are almost finished developing a disease-specific questionnaire: the OVAMA questionnaire.
 
Max M Lokhorst
added 2 research items
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.
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.