Lilien, C.; Vrscaj, E.; Thapaliya, G.; Deconinck, N.; De Waele, L.; Duong, T.; Haberlová, J.; Kumhera, M.; Peirens, G.; Szabo, L.; Tahon, V.; Tang, W.J.; Benmhammed, N.; Médard, L.; Servais, L. Patients’ Perceptions of Nusinersen Effects According to Their Responder Status. J. Clin. Med.2024, 13, 3418.
Lilien, C.; Vrscaj, E.; Thapaliya, G.; Deconinck, N.; De Waele, L.; Duong, T.; Haberlová, J.; Kumhera, M.; Peirens, G.; Szabo, L.; Tahon, V.; Tang, W.J.; Benmhammed, N.; Médard, L.; Servais, L. Patients’ Perceptions of Nusinersen Effects According to Their Responder Status. J. Clin. Med. 2024, 13, 3418.
Lilien, C.; Vrscaj, E.; Thapaliya, G.; Deconinck, N.; De Waele, L.; Duong, T.; Haberlová, J.; Kumhera, M.; Peirens, G.; Szabo, L.; Tahon, V.; Tang, W.J.; Benmhammed, N.; Médard, L.; Servais, L. Patients’ Perceptions of Nusinersen Effects According to Their Responder Status. J. Clin. Med.2024, 13, 3418.
Lilien, C.; Vrscaj, E.; Thapaliya, G.; Deconinck, N.; De Waele, L.; Duong, T.; Haberlová, J.; Kumhera, M.; Peirens, G.; Szabo, L.; Tahon, V.; Tang, W.J.; Benmhammed, N.; Médard, L.; Servais, L. Patients’ Perceptions of Nusinersen Effects According to Their Responder Status. J. Clin. Med. 2024, 13, 3418.
Abstract
Background and Objective: Patients with spinal muscular atrophy (SMA) treated with a dis-ease-modifying therapy (DMT) are often classified as responders or non-responders based on at-tainment of a specific improvement threshold on validated functional scales. This categorization may significantly impact treatment reimbursement in some countries. The aim of this research is to evaluate the perception of treatment benefit by patients considered as responder or non-responder.
Methods: In this non-commercial multicenter study, 99 post-symptomatically treated SMA type I-III patients with a median age of 11.2 (0.39-57.4) years at treatment initiation were stratified into three groups based on their treatment outcomes, i.e. those exhibiting clinically significant improvement (N=41), those with non-clinically significant improvement (N=18), or those show-ing no improvement (N=40). Fifteen months after treatment initiation patients or patients’ care-givers were assessed using a patient-rated scoring system based on the Patient Global Impression of Change (PGIC) scale, comprising 22 questions targeting important aspects and tasks in the daily life of patients with SMA.
Results: We found no statistical difference in patient perception of treatment benefit in 17 out of 22 domains across patient groups.
Conclusion: Our results suggest that functional motor scales do not recapitulate patients and pa-tients’ caregivers experience about the effect of nusinersen treatment in SMA.
Copyright:
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
,
