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Lookup NU author(s): Professor Jordi Diaz ManeraORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© The Author(s) 2024.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
Author(s): Kishnani PS, Byrne BJ, Claeys KG, Diaz-Manera J, Dimachkie MM, Kushlaf H, Mozaffar T, Roberts M, Schoser B, Hummel N, Kopiec A, Holdbrook F, Shohet S, Toscano A, Chien Y-H, Loscher W, Laszlo V, Sawada T, Ruck T, Burrow T, Hiwot T, Dearmey S, Wenninger S, Attarian S, Fecarotta S, Sacconi S, Hopkin R, Henderson R, Roxburgh R, Clemens PR, Deegan P, Goker-Alpan O, Musumeci O, Vidal-Fernandez N, Longo N, Freimer M, Tchan M, Tarnopolsky M, Wencel M, Molnar MJ, Gutschmidt K, Kornblum C, Berthy J, Janszky J, Alonso-Perez J, Cauci J, Shin J-H, Avelar J, Vengoechea J, Tarnev I, Kobayashi H, Shiraishi H, Amartino H, Andersen H, Pedro H, Lau H, Akihiro H, Bartosik-Psujek H, Parenti G, Papadimas GK, Kim G, Bouhour F, Butler E, Salort-Campana E, Stefanescu E, Hughes D, Reyes-Leiva D, Bodkin C, Eldridge C, Quinn C, Lindberg C, Tard C, Koritnik B, Khan A, Dominovic-Kovacevic A, Pestronk A, Sebok A
Publication type: Article
Publication status: Published
Journal: Journal of Patient-Reported Outcomes
Year: 2024
Volume: 8
Issue: 1
Online publication date: 13/11/2024
Acceptance date: 26/10/2024
Date deposited: 26/11/2024
ISSN (electronic): 2509-8020
Publisher: Springer Science and Business Media Deutschland GmbH
URL: https://doi.org/10.1186/s41687-024-00805-w
DOI: 10.1186/s41687-024-00805-w
Data Access Statement: Data sharing proposals and requests for data from the PROPEL study will be reviewed on a case-by-case basis. Requests for data should be addressed to Nick Rees at nrees@amicusrx.com. Requests will be reviewed by a medical steering committee
PubMed id: 39535661
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