Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet–Biedl syndrome: phase 3 trial results
Bardet–Biedl syndrome is a rare genetic disease associated with hyperphagia and early-onset, severe obesity. There is limited evidence on how hyperphagia and obesity afect health-related quality of life in patients with Bardet–Biedl syndrome, and on how management of these symptoms may infuence dise...
| Autores: | , , , , , , , , , , , |
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| Tipo de recurso: | artículo |
| Fecha de publicación: | 2023 |
| País: | España |
| Institución: | Universidad Autónoma de Madrid |
| Repositorio: | Biblos-e Archivo. Repositorio Institucional de la UAM |
| Idioma: | inglés |
| OAI Identifier: | oai:repositorio.uam.es:10486/709300 |
| Acceso en línea: | http://hdl.handle.net/10486/709300 https://dx.doi.org/10.1186/s13023-022-02602-4 |
| Access Level: | acceso abierto |
| Palabra clave: | BBS genetic obesity IWQOL-Lite PedsQL quality of life setmelanotide Medicina |
| Sumario: | Bardet–Biedl syndrome is a rare genetic disease associated with hyperphagia and early-onset, severe obesity. There is limited evidence on how hyperphagia and obesity afect health-related quality of life in patients with Bardet–Biedl syndrome, and on how management of these symptoms may infuence disease burden. This analysis evaluated changes in health-related quality of life in adults and children with Bardet–Biedl syndrome in a Phase 3 trial following 1 year of setmelanotide treatment (ClinicalTrials.gov identifer: NCT03746522). Methods Patients with Bardet–Biedl syndrome and obesity received 52 weeks of treatment with setmelanotide and completed various self-reported health-related quality of life measures. Patients aged<18 years or their caregiver completed the Pediatric Quality of Life Inventory (PedsQL; meaningful improvement, 4.4-point change); adults aged≥18 years completed the Impact of Weight on Quality of Life Questionnaire-Lite (IWQOL-Lite; meaningful improvement range, 7.7–12-point change). Descriptive outcomes were reported in patients with data both at active treatment baseline and after 52 weeks of treatment. Results Twenty patients (<18 years, n=9;≥18 years, n=11) reported health-related quality of life at baseline and 52 weeks. For children and adolescents, PedsQL score mean change from baseline after 52 weeks was+11.2; all patients with PedsQL impairment at baseline (n=4) experienced clinically meaningful improvement. In adults, IWQOL-Lite score mean change from baseline was+12.0. Of adults with IWQOL-Lite impairment at baseline (n=8), 62.5% experienced clinically meaningful improvement. In adults, IWQOL-Lite score was signifcantly correlated with changes in percent body weight (P=0.0037) and body mass index (P=0.0098). Conclusions After 1 year of setmelanotide, patients reported clinically meaningful improvements across multiple health-related quality of life measures. This study highlights the need to address the impaired health-related quality of life in Bardet–Biedl syndrome, and supports utility of setmelanotide for reducing this burden |
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