Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France

• Published in: Journal of comparative effectiveness research 2023-05, Vol.12 (5), p.e220178-e220178
• Main Authors: Wojciechowski, Piotr, Wdowiak, Marlena, Hakimi, Zalmai, Wilson, Koo, Fishman, Jesse, Nazir, Jameel, Toumi, Mondher
• Format: Article
• Language: English
• Subjects: Adult; Cross-Sectional Studies; eortc qlq-c30; eq-5d-5l; France; Hemoglobinuria, Paroxysmal; Humans; Methodology; paroxysmal nocturnal hemoglobinuria; Quality of Life; Surveys and Questionnaires; utility mapping
• ISSN: 2042-6305; 2042-6313
• DOI: 10.57264/cer-2022-0178

To map patient-level data collected on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC) QLQ-C30 to EQ-5D-5L data for estimating health-state utilities in patients with paroxysmal nocturnal hemoglobinuria (PNH). European cross-sectional PNH patient survey data populated regression models mapping EORTC QLQ-C30 domains (covariates: sex and baseline age) to utilities calculated with the EQ-5D-5L French value set. A genetic algorithm allowed selection of the best-fitting between a set of models with and without interaction terms. We validated the selected algorithm using EQ-5D-5L utilities converted from EORTC QLQ-C30 data collected in the PEGASUS phase III, randomized controlled trial of pegcetacoplan versus eculizumab in adults with PNH. Selected through the genetic algorithm, the ordinary least squares model without interactions provided highly stable results across study visits (mean [±SD] utilities 0.58 [±0.42] to 0.89 [±0.10]), and showed the best predictive validity. The new PNH EQ-5D-5L direct mapping developed using a genetic algorithm enabled calculation of reliable health-state utility data required for cost-utility analysis in health technology assessments supporting treatments of PNH.