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ABSTRACT: 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.
SUBMITTER: Lokhorst MM
PROVIDER: S-EPMC7753079 | biostudies-literature | 2021 Mar
REPOSITORIES: biostudies-literature
Lokhorst M M MM Horbach S E R SER Waner M M O T M TM van der Vleuten C J M CJM Spuls P I PI van der Horst C M A M CMAM
JPRAS open 20201130
<h4>Background</h4>The OVAMA (<b>O</b>utcome <b>M</b>easures for <b>VA</b>scular <b>MA</b>lformations) 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 Inve ...[more]