Home > Publications Database > Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months. > print

001     271333
005     20240901004627.0
024 7 _ |a 10.1002/mus.28182
|2 doi
024 7 _ |a pmid:39031772
|2 pmid
024 7 _ |a 0148-639X
|2 ISSN
024 7 _ |a 1097-4598
|2 ISSN
024 7 _ |a altmetric:165710145
|2 altmetric
037 _ _ |a DZNE-2024-01035
041 _ _ |a English
082 _ _ |a 610
100 1 _ |a Meyer, Thomas
|0 0000-0002-2736-7350
|b 0
245 _ _ |a Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months.
260 _ _ |a New York, NY [u.a.]
|c 2024
|b Wiley
336 7 _ |a article
|2 DRIVER
336 7 _ |a Output Types/Journal article
|2 DataCite
336 7 _ |a Journal Article
|b journal
|m journal
|0 PUB:(DE-HGF)16
|s 1724751192_21470
|2 PUB:(DE-HGF)
336 7 _ |a ARTICLE
|2 BibTeX
336 7 _ |a JOURNAL_ARTICLE
|2 ORCID
336 7 _ |a Journal Article
|0 0
|2 EndNote
520 _ _ |a 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.
536 _ _ |a 353 - Clinical and Health Care Research (POF4-353)
|0 G:(DE-HGF)POF4-353
|c POF4-353
|f POF IV
|x 0
588 _ _ |a Dataset connected to CrossRef, PubMed, , Journals: pub.dzne.de
650 _ 7 |a amyotrophic lateral sclerosis (ALS)
|2 Other
650 _ 7 |a clinical course
|2 Other
650 _ 7 |a neurofilament light chain (NfL)
|2 Other
650 _ 7 |a patient‐reported outcomes
|2 Other
650 _ 7 |a tofersen
|2 Other
650 _ 7 |a Superoxide Dismutase-1
|0 EC 1.15.1.1
|2 NLM Chemicals
650 _ 7 |a Neurofilament Proteins
|2 NLM Chemicals
650 _ 7 |a SOD1 protein, human
|2 NLM Chemicals
650 _ 7 |a neurofilament protein L
|2 NLM Chemicals
650 _ 7 |a Oligonucleotides
|2 NLM Chemicals
650 _ 2 |a Humans
|2 MeSH
650 _ 2 |a Amyotrophic Lateral Sclerosis: drug therapy
|2 MeSH
650 _ 2 |a Amyotrophic Lateral Sclerosis: genetics
|2 MeSH
650 _ 2 |a Male
|2 MeSH
650 _ 2 |a Female
|2 MeSH
650 _ 2 |a Patient Reported Outcome Measures
|2 MeSH
650 _ 2 |a Middle Aged
|2 MeSH
650 _ 2 |a Aged
|2 MeSH
650 _ 2 |a Superoxide Dismutase-1: genetics
|2 MeSH
650 _ 2 |a Neurofilament Proteins: blood
|2 MeSH
650 _ 2 |a Treatment Outcome
|2 MeSH
650 _ 2 |a Disease Progression
|2 MeSH
650 _ 2 |a Adult
|2 MeSH
650 _ 2 |a Oligonucleotides: therapeutic use
|2 MeSH
700 1 _ |a Schumann, Peggy
|b 1
700 1 _ |a Weydt, Patrick
|0 P:(DE-2719)9001116
|b 2
|u dzne
700 1 _ |a Petri, Susanne
|b 3
700 1 _ |a Weishaupt, Jochen H
|b 4
700 1 _ |a Weyen, Ute
|b 5
700 1 _ |a Koch, Jan C
|b 6
700 1 _ |a Günther, René
|0 P:(DE-2719)2811849
|b 7
|u dzne
700 1 _ |a Regensburger, Martin
|0 0000-0002-2172-7386
|b 8
700 1 _ |a Boentert, Matthias
|0 0000-0001-6133-1397
|b 9
700 1 _ |a Wiesenfarth, Maximilian
|b 10
700 1 _ |a Koc, Yasemin
|b 11
700 1 _ |a Kolzarek, Felix
|b 12
700 1 _ |a Kettemann, Dagmar
|b 13
700 1 _ |a Norden, Jenny
|b 14
700 1 _ |a Bernsen, Sarah
|b 15
700 1 _ |a Elmas, Zeynep
|b 16
700 1 _ |a Conrad, Julian
|b 17
700 1 _ |a Valkadinov, Ivan
|b 18
700 1 _ |a Vidovic, Maximilian
|0 0000-0003-1453-1681
|b 19
700 1 _ |a Dorst, Johannes
|0 P:(DE-2719)9001951
|b 20
|u dzne
700 1 _ |a Ludolph, Albert C
|0 P:(DE-2719)2812633
|b 21
|u dzne
700 1 _ |a Hesebeck-Brinckmann, Jasper
|b 22
700 1 _ |a Spittel, Susanne
|b 23
700 1 _ |a Münch, Christoph
|b 24
700 1 _ |a Maier, André
|b 25
700 1 _ |a Körtvelyessy, Peter
|0 P:(DE-2719)2812030
|b 26
|u dzne
773 _ _ |a 10.1002/mus.28182
|g Vol. 70, no. 3, p. 333 - 345
|0 PERI:(DE-600)1476641-3
|n 3
|p 333 - 345
|t Muscle & nerve
|v 70
|y 2024
|x 0148-639X
856 4 _ |u https://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.docx
856 4 _ |y OpenAccess
|u https://pub.dzne.de/record/271333/files/DZNE-2024-01035.pdf
856 4 _ |y OpenAccess
|x pdfa
|u https://pub.dzne.de/record/271333/files/DZNE-2024-01035.pdf?subformat=pdfa
856 4 _ |u https://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.doc
856 4 _ |u https://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.odt
856 4 _ |u https://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.pdf
909 C O |o oai:pub.dzne.de:271333
|p openaire
|p open_access
|p VDB
|p driver
|p dnbdelivery
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 2
|6 P:(DE-2719)9001116
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 7
|6 P:(DE-2719)2811849
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 20
|6 P:(DE-2719)9001951
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 21
|6 P:(DE-2719)2812633
910 1 _ |a External Institute
|0 I:(DE-HGF)0
|k Extern
|b 26
|6 P:(DE-2719)2812030
913 1 _ |a DE-HGF
|b Gesundheit
|l Neurodegenerative Diseases
|1 G:(DE-HGF)POF4-350
|0 G:(DE-HGF)POF4-353
|3 G:(DE-HGF)POF4
|2 G:(DE-HGF)POF4-300
|4 G:(DE-HGF)POF
|v Clinical and Health Care Research
|x 0
914 1 _ |y 2024
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0200
|2 StatID
|b SCOPUS
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0160
|2 StatID
|b Essential Science Indicators
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)1050
|2 StatID
|b BIOSIS Previews
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)1190
|2 StatID
|b Biological Abstracts
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0600
|2 StatID
|b Ebsco Academic Search
|d 2023-10-21
915 _ _ |a Creative Commons Attribution-NonCommercial-NoDerivs CC BY-NC-ND 4.0
|0 LIC:(DE-HGF)CCBYNCND4
|2 HGFVOC
915 _ _ |a JCR
|0 StatID:(DE-HGF)0100
|2 StatID
|b MUSCLE NERVE : 2022
|d 2023-10-21
915 _ _ |a DEAL Wiley
|0 StatID:(DE-HGF)3001
|2 StatID
|d 2023-10-21
|w ger
915 _ _ |a WoS
|0 StatID:(DE-HGF)0113
|2 StatID
|b Science Citation Index Expanded
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)1030
|2 StatID
|b Current Contents - Life Sciences
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0150
|2 StatID
|b Web of Science Core Collection
|d 2023-10-21
915 _ _ |a IF < 5
|0 StatID:(DE-HGF)9900
|2 StatID
|d 2023-10-21
915 _ _ |a OpenAccess
|0 StatID:(DE-HGF)0510
|2 StatID
915 _ _ |a Peer Review
|0 StatID:(DE-HGF)0030
|2 StatID
|b ASC
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0300
|2 StatID
|b Medline
|d 2023-10-21
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)1110
|2 StatID
|b Current Contents - Clinical Medicine
|d 2023-10-21
915 _ _ |a Nationallizenz
|0 StatID:(DE-HGF)0420
|2 StatID
|d 2023-10-21
|w ger
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0199
|2 StatID
|b Clarivate Analytics Master Journal List
|d 2023-10-21
920 1 _ |0 I:(DE-2719)1011001
|k Clinical Research (Bonn)
|l Clinical Research Coordination
|x 0
920 1 _ |0 I:(DE-2719)1710012
|k AG Falkenburger
|l Translational Parkinson Research
|x 1
920 1 _ |0 I:(DE-2719)5000077
|k Clinical Study Center (Ulm)
|l Clinical Study Center (Ulm)
|x 2
980 _ _ |a journal
980 _ _ |a VDB
980 _ _ |a UNRESTRICTED
980 _ _ |a I:(DE-2719)1011001
980 _ _ |a I:(DE-2719)1710012
980 _ _ |a I:(DE-2719)5000077
980 1 _ |a FullTexts

LibraryCollectionCLSMajorCLSMinorLanguageAuthor
Marc 21