Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease

Background: Late-onset Pompe disease (LOPD), a rare autosomal recessive multisystemic disorder, substantially impacts patients' day-to-day activities, outcomes, and health-related quality of life (HRQoL). The PROPEL trial compared cipaglucosidase alfa plus miglustat (cipa+mig) with alglucosidas...

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Autores: Kishnani, Priya S., Byrne, Barry J., Claeys, Kristl G., Diaz-Manera, Jordi|||0000-0003-2941-7988, Dimachkie, Mazen M., Kushlaf, Hani, Mozaffar, Tahseen, Roberts, Mark, Schoser, Benedikt|||0000-0002-2757-8131, Hummel, Noemi, Kopiec, Agnieszka, Holdbrook, Fred, Shohet, Simon, Toscano, Antonio, Chien, Yin-Hsiu, Löscher, Wolfgang, Laszlo, Vescei, Sawada, Tomo, Ruck, Tobias, Burrow, Thomas, Hiwot, Tarekegn, Dearmey, Stephanie, Wenninger, Stephan, Attarian, Shahram, Fecarotta, Simona, Sacconi, Sabrina, Hopkin, Robert, Henderson, Robert, Roxburgh, Richard, Clemens, Paula R., Deegan, Patrick, Goker-Alpan, Ozlem, Musumeci, Olimpia|||0000-0002-9208-1527, Vidal-Fernández, Núria|||0009-0008-3749-6448, Longo, Nicola, Freimer, Miriam, Tchan, Michel, Tarnopolsky, Mark, Wencel, Marie, Molnar, Maria Judit, Gutschmidt, Kristina, Kornblum, Cornelia, Berthy, Julie, Janszky, Jozsef, Alonso Pérez, Jorge|||0000-0001-8866-5186, Shin, Jin-Hong, Avelar, Jennifer, Vengoechea, Jaime, Tarnev, Ivaylo, Kobayashi, Hiroshi, Shiraishi, Hideaki, Amartino, Hernán, Andersen, Henning, Pedro, Helio, Lau, Heather, Akihiro, Hashiguchi, Bartosik-Psujek, Halina, Parenti, Giancarlo, Papadimas, George Konstantinos, Kim, Gee, Bouhour, Francoise, Butler, Ernest, Salort-Campana, Emmanuelle|||0000-0002-1846-3017, Stefanescu, Ela, Hughes, Derralynn A.|||0000-0003-4531-9173, Reyes-Leiva, David|||0000-0001-6983-7130, Bodkin, Cynthia, Eldridge, Crystal, Quinn, Colin|||0000-0003-0618-5854, Lindberg, Christopher, Tard, Celine, Koritnik, Blaž|||0000-0002-5083-8261, Khan, Aneal, Dominovic-Kovacevic, Aleksandra, Pestronk, Alan|||0000-0002-8991-5770, Sebok, Agnes
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:310159
Acceso en línea:https://ddd.uab.cat/record/310159
https://dx.doi.org/urn:doi:10.1186/s41687-024-00805-w
Access Level:acceso abierto
Palabra clave:Health-related quality of life
Patient-reported Outcome Measurement Information System
Patient-reported outcomes
Pompe disease
Descripción
Sumario:Background: Late-onset Pompe disease (LOPD), a rare autosomal recessive multisystemic disorder, substantially impacts patients' day-to-day activities, outcomes, and health-related quality of life (HRQoL). The PROPEL trial compared cipaglucosidase alfa plus miglustat (cipa+mig) with alglucosidase alfa plus placebo (alg+pbo) in adult patients with LOPD over 52 weeks and showed improved motor and respiratory function in patients switching treatment from standard-of-care enzyme replacement therapy (ERT) to cipa+mig at baseline. This study evaluated the impact of cipa+mig on patient-reported outcomes (PROs), including HRQoL in ERT-experienced patients, using data from PROPEL. Methods: PROs evaluated included the Subject's Global Impression of Change (SGIC), Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 20a, PROMIS Fatigue Short Form 8a, Rasch-built Pompe-specific Activity (R-PAct), and European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L). The proportions of responders in the cipa+mig arm and the alg+pbo arm were compared via chi-squared or Fisher's exact test (patient-level responder analysis), and least squares (LS) mean differences were calculated for change from baseline at Week 52 of the PRO measures (group-level analysis). Results: At Week 52, patient-level SGIC responder and group-level SGIC analyses favored cipa+mig compared with alg+pbo across all SGIC domains (e.g. 90 vs. 59% responders in the cipa+mig vs. the alg+pbo group for SGIC ability to move around; P = 0.0005; and LS mean difference 0.385; P = 0.02). Similarly, PROMIS Physical Function and Fatigue domains numerically favored cipa+mig in both analyses (e.g. 50 vs. 40% responders in the cipa+mig vs. alg+pbo arm for PROMIS Physical Function; P = 0.37; and LS mean difference 3.1; P = 0.11). R-PAct for both treatment groups was similar in the patient-level responder analysis, but numerically favored alg+pbo in the group-level analysis (35% responders in both arms; P = 0.95; and LS mean difference -0.8; P = 0.48). Self-care, usual activities, and depression/anxiety domains of EQ-5D-5L numerically favored cipa+mig in both analyses (e.g. 20 vs. 12% responders in the cipa+mig vs. alg+pbo arm for EQ-5D-5L self-care; P = 0.54; and LS mean difference -0.108; P = 0.52). Conclusions: Overall, switching treatment from alglucosidase alfa to cipa+mig positively impacted PRO measurements during the double-blind period of PROPEL. Trial registration: NCT03729362; Registration date: November 1, 2018; https://clinicaltrials.gov/study/NCT03729362.

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