Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS—A multicenter observational study over 18 months

Meyer T, Schumann P, Weydt P, Petri S, Weishaupt JH, Weyen U, Koch JC, Günther R, Regensburger M, Boentert M, Wiesenfarth M, Koc Y, Kolzarek F, Kettemann D, Norden J, Bernsen S, Elmas Z, Conrad J, Valkadinov I, Vidovic M, Dorst J, Ludolph AC, Hesebeck-Brinckmann J, Spittel S, Münch C, Maier A, Körtvélyessy P (2024)

Publication Type: Journal article

Publication year: 2024

Journal

Muscle & Nerve Wiley-Blackwell

DOI: 10.1002/mus.28182

Abstract

Introduction/Aims: In amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations (SOD1-ALS), tofersen received accelerated approval in the United States and is available via expanded access programs (EAP) outside the United States. This multicenter study investigates clinical and patient-reported outcomes (PRO) and serum neurofilament light chain (sNfL) during tofersen treatment in an EAP in Germany. Methods: Sixteen SOD1-ALS patients receiving tofersen for at least 6 months were analyzed. The ALS progression rate (ALS-PR), as measured by the monthly change of the ALS functional rating scale—revised (ALSFRS-R), slow vital capacity (SVC), and sNfL were investigated. PRO included the Measure Yourself Medical Outcome Profile (MYMOP2), Treatment Satisfaction Questionnaire for Medication (TSQM-9), and Net Promoter Score (NPS). Results: Mean tofersen treatment was 11 months (6–18 months). ALS-PR showed a mean change of −0.2 (range 0 to −1.1) and relative reduction by 25%. Seven patients demonstrated increased ALSFRS-R. SVC was stable (mean 88%, range −15% to +28%). sNfL decreased in all patients except one heterozygous D91A-SOD1 mutation carrier (mean change of sNfL −58%, range −91 to +27%, p <.01). MYMOP2 indicated improved symptom severity (n = 10) or yet perception of partial response (n = 6). TSQM-9 showed high global treatment satisfaction (mean 83, SD 16) although the convenience of drug administration was modest (mean 50, SD 27). NPS revealed a very high recommendation rate for tofersen (NPS +80). Discussion: Data from this EAP supported the clinical and sNfL response to tofersen in SOD1-ALS. PRO suggested a favorable patient perception of tofersen treatment in clinical practice.

Authors with CRIS profile

Martin Regensburger Department of Neurology

Additional Organisation(s)

FAU Profilzentrum Licht.Materie.Quantentechnologien (FAU LMQ)

Involved external institutions

Rheinische Friedrich-Wilhelms-Universität Bonn DE Germany (DE) Medizinische Hochschule Hannover (MHH) / Hannover Medical School DE Germany (DE) Ruprecht-Karls-Universität Heidelberg DE Germany (DE) Berufsgenossenschaftliches Universitätsklinikum Bergmannsheil DE Germany (DE) Universitätsklinikum Göttingen DE Germany (DE) Universitätsklinikum Carl Gustav Carus Dresden DE Germany (DE) Universitätsklinikum Münster DE Germany (DE) Universitätsklinikum Ulm DE Germany (DE) Berliner Institut für Gesundheitsforschung (BIH) DE Germany (DE) Ambulanzpartner Soziotechnologie APST GmbH DE Germany (DE)

How to cite

APA:

Meyer, T., Schumann, P., Weydt, P., Petri, S., Weishaupt, J.H., Weyen, U.,... Körtvélyessy, P. (2024). Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS—A multicenter observational study over 18 months. Muscle & Nerve. https://doi.org/10.1002/mus.28182

MLA:

Meyer, Thomas, et al. "Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS—A multicenter observational study over 18 months." Muscle & Nerve (2024).

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