Journal Article

DZNE-2024-01035

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png 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.DZNE* ; Petri, S. ; Weishaupt, J. H. ; Weyen, U. ; Koch, J. C. ; Günther, R.DZNE* ; 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.DZNE* ; Ludolph, A. C.DZNE* ; Hesebeck-Brinckmann, J. ; Spittel, S. ; Münch, C. ; Maier, A. ; Körtvelyessy, P.Extern*

2024
Wiley New York, NY [u.a.]

This record in other databases:    

Please use a persistent id in citations: doi:10.1002/mus.28182

Abstract: 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.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).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).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.

Keyword(s): Humans (MeSH) ; Amyotrophic Lateral Sclerosis: drug therapy (MeSH) ; Amyotrophic Lateral Sclerosis: genetics (MeSH) ; Male (MeSH) ; Female (MeSH) ; Patient Reported Outcome Measures (MeSH) ; Middle Aged (MeSH) ; Aged (MeSH) ; Superoxide Dismutase-1: genetics (MeSH) ; Neurofilament Proteins: blood (MeSH) ; Treatment Outcome (MeSH) ; Disease Progression (MeSH) ; Adult (MeSH) ; Oligonucleotides: therapeutic use (MeSH) ; amyotrophic lateral sclerosis (ALS) ; clinical course ; neurofilament light chain (NfL) ; patient‐reported outcomes ; tofersen ; Superoxide Dismutase-1 ; Neurofilament Proteins ; SOD1 protein, human ; neurofilament protein L ; Oligonucleotides

---

Contributing Institute(s):

1. Clinical Research Coordination (Clinical Research (Bonn))
2. Translational Parkinson Research (AG Falkenburger)
3. Clinical Study Center (Ulm) (Clinical Study Center (Ulm))

Research Program(s):

1. 353 - Clinical and Health Care Research (POF4-353) (POF4-353)

Appears in the scientific report 2024

---

Database coverage:
; ; ; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; Current Contents - Life Sciences ; DEAL Wiley ; Ebsco Academic Search ; Essential Science Indicators ; IF < 5 ; JCR ; Nationallizenz ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

---