| Title: | Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease |
|---|
| Authors: | ID Kishnani, Priya S. (Author)
ID Byrne, Barry J. (Author)
ID Claeys, Kristl G. (Author)
ID Diaz-Manera, Jordi (Author)
ID Dimachkie, Mazen M. (Author)
ID Kushlaf, Hani (Author)
ID Mozaffar, Tahseen (Author)
ID Roberts, Mark (Author)
ID Schoser, Benedikt (Author)
ID Hummel, Noemi (Author)
ID Koritnik, Blaž (Research coworker), et al. |
|---|
| Files: |  PDF - Presentation file, download (2,21 MB)
MD5: A7CD749FC840E0C2F6C6A01732A633E3
PDF - Presentation file, download (2,21 MB)
MD5: A7CD749FC840E0C2F6C6A01732A633E3
|
|---|
| Language: | English |
|---|
| Typology: | 1.01 - Original Scientific Article |
|---|
| Organization: |  UKC LJ - Ljubljana University Medical Centre
|
|---|
| Abstract: | 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, Raschbuilt 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. |
|---|
| Keywords: | Pompe disease, patient-reported outcomes, health-related quality of life, SARS-Cov-2 |
|---|
| Publication status: | Published |
|---|
| Publication version: | Version of Record |
|---|
| Year of publishing: | 2024 |
|---|
| Number of pages: | str. 1-9 |
|---|
| Numbering: | Vol. 8, ǂarticle no. ǂ132 |
|---|
| PID: | 20.500.12556/DiRROS-27881  |
|---|
| UDC: | 616-097 |
|---|
| ISSN on article: | 2509-8020 |
|---|
| DOI: | 10.1186/s41687-024-00805-w |
|---|
| COBISS.SI-ID: | 230729987 |
|---|
| Note: |
|
|---|
| Publication date in DiRROS: | 26.02.2026 |
|---|
| Views: | 150 |
|---|
| Downloads: | 59 |
|---|
| Metadata: |    |
|---|
|
:
|
Copy citation |
|---|
| | | | Share: |  |
|---|
Hover the mouse pointer over a document title to show the abstract or click
on the title to get all document metadata. |
