Abstract

SummaryBackgroundThe 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).MethodsIn 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.ResultsTwenty-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.DiscussionThe 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.

Highlights

  • Vascular malformations are rare congenital vessel anomalies

  • All correlations were low or moderate, except between the PedsQL ‘emotional’ scale and the Children’s Dermatology Life Quality Index (CDLQI) total score, which was high. This exploratory study suggests that the PedsQL and CDLQI are insufficiently responsive for measuring vascular malformation-related health problems in children

  • Disease-specific domains, such as vascular malformation symptoms and appearance, might be targeted more directly by therapy, and might be more suitable for detecting changes over time. This exploratory study suggests that the PedsQL and CDLQI are not sufficiently responsive for evaluating treatment effect in children with peripheral vascular malformations

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Summary

Introduction

Vascular malformations are rare congenital vessel anomalies. The lesions are classified according flow velocity and the types of vessels involved. 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. ✩ All authors give consent for publication. Written informed consent was obtained from all participants

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