Responsiveness of quality‐of‐life measures in patients with peripheral vascular malformations: the OVAMA project

Abstract

Summary Background 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. Objectives To assess 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 the U.S.A., of whom 67 completed all baseline and follow‐up questionnaires. For all subscales of the SF‐36 and Skindex‐29, < 75% of the hypotheses were confirmed and the AUC was < 0·7. Conclusions 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. What's already known about this topic? Quality of life is often impaired in patients with vascular malformations. Quality of life is considered a core outcome domain for evaluating treatment of vascular malformations. To measure the effect of treatment on quality of life, a patient‐reported outcome measure is required that is responsive to changes in quality of life. What does this study add? This is the first study assessing the responsiveness of quality‐of‐life measures in patients with vascular malformations. The results seem to indicate that the Medical Outcomes Study Short Form 36 (SF‐36) and Skindex‐29 are not responsive to changes in quality of life in patients with vascular malformations. What are the clinical implications of this work? Medical Outcomes Study Short Form 36 (SF‐36) and Skindex‐29 are not ideal to assess the effect on quality of life over time, of treatment strategies for peripheral vascular malformations.

Responsiveness of quality of life measures in children with peripheral vascular malformations: The OVAMA project

“…A study on responsiveness of two generic QoL measures in adult patients with vascular malformations also found insufficient responsiveness, plausibly caused by large measurement errors. 20 A systematic review on outcome measurement instruments in vascular malformations found no other studies on responsiveness in this patient group or similar groups. 21 …”

Surgical management of auricular arteriovenous malformations: A literature review

“…“Radiological characteristics” are also not included in CDS because imaging follow‐up is not mandatory, but it should be reported if the examination was performed. In addition, although the OVAMA Steering Group has conducted studies to explore how to measure these core domains, there is no final core outcome measurement set 82–85 …”

“…In addition, although the OVAMA Steering Group has conducted studies to explore how to measure these core domains, there is no final core outcome measurement set. [82][83][84][85] After evaluating treatment outcomes, follow-up is necessary for patients who no longer need special intervention. 7,8,11,24 When conditions permit, both clinical and imaging follow-up should be used because imaging can detect lesion changes more accurately and earlier than clinical follow-up.…”

Development of a condition‐specific patient‐reported outcome measure for measuring symptoms and appearance in vascular malformations: the OVAMA questionnaire

“…Non‐condition‐specific domains are advised to be measured by non‐condition‐specific outcome measurement instruments 11 . However, broadly used instruments such as the Short Form‐36 and Skindex‐29 seem to fall short for detecting changes in outcomes over time in this specific patient population 12 . Newer instruments such as the Patient‐Reported Outcomes Measurement Information System (PROMIS) 13 item banks may be used in this patient population as they are more likely to adequately capture small differences in the domains falling under quality of life 14,15 .…”

“… 11 However, broadly used instruments such as the Short Form‐36 and Skindex‐29 seem to fall short for detecting changes in outcomes over time in this specific patient population. 12 Newer instruments such as the Patient‐Reported Outcomes Measurement Information System (PROMIS) 13 item banks may be used in this patient population as they are more likely to adequately capture small differences in the domains falling under quality of life. 14 , 15 To fully capture those domains, the following PROMIS scales were identified: ‘pain interference’, ‘physical functioning’, ‘anxiety’, ‘depression’ and ‘social participation’.…”