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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2024年11月13日

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  • Priya S. Kishnani,
  • Barry J. Byrne,
  • Kristl G. Claeys,
  • Jordi Díaz-Manera,
  • Mazen M. Dimachkie,
  • Hani Kushlaf,
  • Tahseen Mozaffar,
  • Mark Roberts,
  • Benedikt Schoser,
  • Noemi Hummel,
  • Agnieszka Kopiec,
  • Fred Holdbrook,
  • Simon Shohet,
  • Antonio Toscano,
  • Agnes Sebok,
  • Alan Pestronk,
  • Aleksandra Dominovic-Kovacevic,
  • Aneal Khan,
  • Blaž Koritnik,
  • Celine Tard,
  • Christopher Lindberg,
  • Colin Quinn,
  • Crystal Eldridge,
  • Cynthia Bodkin,
  • David Reyes-Leiva,
  • Derralynn Hughes,
  • Ela Stefanescu,
  • Emmanuelle Salort-Campana,
  • Ernest Butler,
  • Francoise Bouhour,
  • Gee Kim,
  • George Konstantinos Papadimas,
  • Giancarlo Parenti,
  • Halina Bartosik-Psujek,
  • Hashiguchi Akihiro,
  • Heather Lau,
  • Helio Pedro,
  • Henning Andersen,
  • Hernan Amartino,
  • Hideaki Shiraishi,
  • Hiroshi Kobayashi,
  • Ivaylo Tarnev,
  • Jaime Vengoechea,
  • Jennifer Avelar,
  • Jin-Hong Shin,
  • Jonathan Cauci,
  • Jorge Alonso-Pérez,
  • Jozsef Janszky,
  • Julie Berthy,
  • Cornelia Kornblum,
  • Kristina Gutschmidt,
  • Maria Judit Molnar,
  • Marie Wencel,
  • Mark Tarnopolsky,
  • Michel Tchan,
  • Miriam Freimer,
  • Nicola Longo,
  • Nuria Vidal-Fernandez,
  • Olimpia Musumeci,
  • Ozlem Goker-Alpan,
  • Patrick Deegan,
  • Paula R Clemens,
  • Richard Roxburgh,
  • Robert Henderson,
  • Robert Hopkin,
  • Sabrina Sacconi,
  • Simona Fecarotta,
  • Shahram Attarian,
  • Stephan Wenninger,
  • Stephanie Dearmey,
  • Tarekegn Hiwot,
  • Thomas Burrow,
  • Tobias Ruck,
  • Tomo Sawada,
  • Vescei Laszlo,
  • Wolfgang Löscher,
  • Yin-Hsiu Chien

摘要

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