Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy.

BACKGROUND

Nusinersen was the first approved disease-modifying therapy for all 5q-spinal muscular atrophy (SMA) patients regardless of age or disease severity. Its efficacy in adults has recently been demonstrated in a large cohort by motor outcome measures, which were only partially suitable to detect changes in very mildly or severely affected patients. Patient-reported outcome measures (PROs) have been suggested as a valuable addition. Here, we aimed to assess treatment satisfaction and investigate whether it may be a useful PRO to monitor SMA patients.

METHODS

We enrolled 91 mainly adult 5q-SMA patients treated with nusinersen in a national, multicenter, cross-sectional observational study. 21 patients underwent longitudinal follow up. Patients' satisfaction with treatment in four dimensions (global, effectiveness, convenience, side effects) was assessed by the Treatment Satisfaction Questionnaire for Medication German version 1.4 (TSQM-1.4

RESULTS

More than 90% of SMA patients were consistently satisfied over a median treatment duration of 10 months. Highest mean scores were observed in the dimensions 'side effects,' 'global satisfaction,' and 'effectiveness' (93.5 ± 14.8

CONCLUSION

Most patients were satisfied with nusinersen treatment effectiveness. Less severely affected patients indicated higher satisfaction. The TSQM-1.4

Informations de copyright

© The Author(s), 2021.

Déclaration de conflit d'intérêts

Conflict of interest statement: AO received honoraria as a speaker/consultant from the German Neuromuscular Society ‘Deutsche Gesellschaft fuer Muskelkranke’ (DGM e.V.), and Biogen GmbH. GR and MK received travel cost compensations from Biogen GmbH. CDW received honoraria and travel expenses from Biogen as an advisory board member and for lectures and as a consultant from Roche. BS received travel reimbursement and speaker honoraria from Biogen GmbH. IC received honoraria as speaker/consultant from Biogen GmbH. RG received honoraria as speaker/consultant from Biogen GmbH and Roche, and received research support from Biogen GmbH. MF reports non-financial support from Biogen outside the submitted work. LHM and CB report no disclosures. AH received personal fees and non-financial support from Biogen GmbH and from Destin, during the conduct of the study; grants from Helmholtz Foundation, BMBF, Innovationsausschuss des G-BA, DGM e.V., Schilling-Stiftung, outside the submitted work. MD received honoraria for advisory activities, talks and travel fees from Biogen GmbH, Roche and DGM e.V. PL received support for symposium organization from Biogen GmbH during the conduct of the study; and speaker honoraria from Desitin, BIAL, and AbbVie, and fees for advisory activities from Novartis, outside of the submitted work. ACL reports no disclosures. TH received honoraria as speaker/consultant from Biogen GmbH, Roche, Novartis, CSL Behring, Pfizer, Akcea, Sanofi-Aventis, and research support from Biogen GmbH and Sanofi-Aventis. OSK received honoraria as a speaker/consultant and/or funding for travel expenses from DGM e.V., Novartis, Biogen GmbH, the Jain Foundation and Biermann Verlag GmbH, and research support from the DGM e.V. SP received honoraria as speaker/consultant from Biogen GmbH, Roche, Novartis, Teva, Cytokinetics Inc., Desitin; and grants from DGM e.V, Federal Ministry of Education and Research, German Israeli Foundation for Scientific Research and Development, EU Joint Program for Neurodegenerative Disease Research.