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- Title
Risdiplam therapy in adults with 5q-SMA: observational study on motor function and treatment satisfaction.
- Authors
Bjelica, Bogdan; Wohnrade, Camilla; Cespedes, Iraima; Osmanovic, Alma; Schreiber-Katz, Olivia; Petri, Susanne
- Abstract
Background: We aimed to describe the experience of a single neuromuscular center in Germany in treating adult spinal muscular atrophy (SMA) patients with risdiplam and to analyze motor function and treatment satisfaction during a follow-up period up to 20 months. Methods: Fourteen patients with type 2 or 3 SMA (seven with SMA type 2, six with SMA type 3; age range: 18–51) were included. The Revised Upper Limb Module (RULM) and the Hammersmith Functional Motor Scale Expanded (HFMSE) were recorded at baseline and at follow-up (month 4, 8, 12, 16, 20). Treatment adverse events were collected at every follow-up visit. Patients' treatment satisfaction was assessed by the Treatment Satisfaction Questionnaire for Medication (TSQM). Results: Half of the patients reached the 20-month follow-up. Based on the HFMSE score, no patients had clinically meaningful improvement. Twelve remained stable (92.3%), two showed transient clinically meaningful deterioration (15.4%) and one experienced lasting clinically meaningful deterioration (7.7%). Based on the RULM scores, seven patients were either stable or demonstrated clinically meaningful improvement (53.8%) and six showed clinically meaningful deterioration (46.2%). There was no treatment withdrawal during the follow-up. The most common adverse events were skin rash/increased skin sensitivity to sunlight (n = 3), diarrhea (n = 3), aphthous ulcer (n = 3) and abdominal pain (n = 2). Most patients stated to be at least "satisfied" with the medication. Conclusions: Risdiplam was well tolerated. Half of the patients remained stable or improved after risdiplam initiation. Larger and multicentric studies are needed to better understand the long-term effects of risdiplam in adult SMA.
- Subjects
GERMANY; PATIENT satisfaction; SPINAL muscular atrophy; TERMINATION of treatment; CANKER sores; SCIENTIFIC observation
- Publication
BMC Neurology, 2024, Vol 24, Issue 1, p1
- ISSN
1471-2377
- Publication type
Article
- DOI
10.1186/s12883-024-03562-x