000271333 001__ 271333
000271333 005__ 20240901004627.0
000271333 0247_ $$2doi$$a10.1002/mus.28182
000271333 0247_ $$2pmid$$apmid:39031772
000271333 0247_ $$2ISSN$$a0148-639X
000271333 0247_ $$2ISSN$$a1097-4598
000271333 0247_ $$2altmetric$$aaltmetric:165710145
000271333 037__ $$aDZNE-2024-01035
000271333 041__ $$aEnglish
000271333 082__ $$a610
000271333 1001_ $$00000-0002-2736-7350$$aMeyer, Thomas$$b0
000271333 245__ $$aClinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months.
000271333 260__ $$aNew York, NY [u.a.]$$bWiley$$c2024
000271333 3367_ $$2DRIVER$$aarticle
000271333 3367_ $$2DataCite$$aOutput Types/Journal article
000271333 3367_ $$0PUB:(DE-HGF)16$$2PUB:(DE-HGF)$$aJournal Article$$bjournal$$mjournal$$s1724751192_21470
000271333 3367_ $$2BibTeX$$aARTICLE
000271333 3367_ $$2ORCID$$aJOURNAL_ARTICLE
000271333 3367_ $$00$$2EndNote$$aJournal Article
000271333 520__ $$aIn 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.
000271333 536__ $$0G:(DE-HGF)POF4-353$$a353 - Clinical and Health Care Research (POF4-353)$$cPOF4-353$$fPOF IV$$x0
000271333 588__ $$aDataset connected to CrossRef, PubMed, , Journals: pub.dzne.de
000271333 650_7 $$2Other$$aamyotrophic lateral sclerosis (ALS)
000271333 650_7 $$2Other$$aclinical course
000271333 650_7 $$2Other$$aneurofilament light chain (NfL)
000271333 650_7 $$2Other$$apatient‐reported outcomes
000271333 650_7 $$2Other$$atofersen
000271333 650_7 $$0EC 1.15.1.1$$2NLM Chemicals$$aSuperoxide Dismutase-1
000271333 650_7 $$2NLM Chemicals$$aNeurofilament Proteins
000271333 650_7 $$2NLM Chemicals$$aSOD1 protein, human
000271333 650_7 $$2NLM Chemicals$$aneurofilament protein L
000271333 650_7 $$2NLM Chemicals$$aOligonucleotides
000271333 650_2 $$2MeSH$$aHumans
000271333 650_2 $$2MeSH$$aAmyotrophic Lateral Sclerosis: drug therapy
000271333 650_2 $$2MeSH$$aAmyotrophic Lateral Sclerosis: genetics
000271333 650_2 $$2MeSH$$aMale
000271333 650_2 $$2MeSH$$aFemale
000271333 650_2 $$2MeSH$$aPatient Reported Outcome Measures
000271333 650_2 $$2MeSH$$aMiddle Aged
000271333 650_2 $$2MeSH$$aAged
000271333 650_2 $$2MeSH$$aSuperoxide Dismutase-1: genetics
000271333 650_2 $$2MeSH$$aNeurofilament Proteins: blood
000271333 650_2 $$2MeSH$$aTreatment Outcome
000271333 650_2 $$2MeSH$$aDisease Progression
000271333 650_2 $$2MeSH$$aAdult
000271333 650_2 $$2MeSH$$aOligonucleotides: therapeutic use
000271333 7001_ $$aSchumann, Peggy$$b1
000271333 7001_ $$0P:(DE-2719)9001116$$aWeydt, Patrick$$b2$$udzne
000271333 7001_ $$aPetri, Susanne$$b3
000271333 7001_ $$aWeishaupt, Jochen H$$b4
000271333 7001_ $$aWeyen, Ute$$b5
000271333 7001_ $$aKoch, Jan C$$b6
000271333 7001_ $$0P:(DE-2719)2811849$$aGünther, René$$b7$$udzne
000271333 7001_ $$00000-0002-2172-7386$$aRegensburger, Martin$$b8
000271333 7001_ $$00000-0001-6133-1397$$aBoentert, Matthias$$b9
000271333 7001_ $$aWiesenfarth, Maximilian$$b10
000271333 7001_ $$aKoc, Yasemin$$b11
000271333 7001_ $$aKolzarek, Felix$$b12
000271333 7001_ $$aKettemann, Dagmar$$b13
000271333 7001_ $$aNorden, Jenny$$b14
000271333 7001_ $$aBernsen, Sarah$$b15
000271333 7001_ $$aElmas, Zeynep$$b16
000271333 7001_ $$aConrad, Julian$$b17
000271333 7001_ $$aValkadinov, Ivan$$b18
000271333 7001_ $$00000-0003-1453-1681$$aVidovic, Maximilian$$b19
000271333 7001_ $$0P:(DE-2719)9001951$$aDorst, Johannes$$b20$$udzne
000271333 7001_ $$0P:(DE-2719)2812633$$aLudolph, Albert C$$b21$$udzne
000271333 7001_ $$aHesebeck-Brinckmann, Jasper$$b22
000271333 7001_ $$aSpittel, Susanne$$b23
000271333 7001_ $$aMünch, Christoph$$b24
000271333 7001_ $$aMaier, André$$b25
000271333 7001_ $$0P:(DE-2719)2812030$$aKörtvelyessy, Peter$$b26$$udzne
000271333 773__ $$0PERI:(DE-600)1476641-3$$a10.1002/mus.28182$$gVol. 70, no. 3, p. 333 - 345$$n3$$p333 - 345$$tMuscle & nerve$$v70$$x0148-639X$$y2024
000271333 8564_ $$uhttps://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.docx
000271333 8564_ $$uhttps://pub.dzne.de/record/271333/files/DZNE-2024-01035.pdf$$yOpenAccess
000271333 8564_ $$uhttps://pub.dzne.de/record/271333/files/DZNE-2024-01035.pdf?subformat=pdfa$$xpdfa$$yOpenAccess
000271333 8564_ $$uhttps://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.doc
000271333 8564_ $$uhttps://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.odt
000271333 8564_ $$uhttps://pub.dzne.de/record/271333/files/DZNE-2024-01035%20SUP.pdf
000271333 909CO $$ooai:pub.dzne.de:271333$$pdnbdelivery$$pdriver$$pVDB$$popen_access$$popenaire
000271333 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)9001116$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b2$$kDZNE
000271333 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)2811849$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b7$$kDZNE
000271333 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)9001951$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b20$$kDZNE
000271333 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)2812633$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b21$$kDZNE
000271333 9101_ $$0I:(DE-HGF)0$$6P:(DE-2719)2812030$$aExternal Institute$$b26$$kExtern
000271333 9131_ $$0G:(DE-HGF)POF4-353$$1G:(DE-HGF)POF4-350$$2G:(DE-HGF)POF4-300$$3G:(DE-HGF)POF4$$4G:(DE-HGF)POF$$aDE-HGF$$bGesundheit$$lNeurodegenerative Diseases$$vClinical and Health Care Research$$x0
000271333 9141_ $$y2024
000271333 915__ $$0StatID:(DE-HGF)0200$$2StatID$$aDBCoverage$$bSCOPUS$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)0160$$2StatID$$aDBCoverage$$bEssential Science Indicators$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)1050$$2StatID$$aDBCoverage$$bBIOSIS Previews$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)1190$$2StatID$$aDBCoverage$$bBiological Abstracts$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)0600$$2StatID$$aDBCoverage$$bEbsco Academic Search$$d2023-10-21
000271333 915__ $$0LIC:(DE-HGF)CCBYNCND4$$2HGFVOC$$aCreative Commons Attribution-NonCommercial-NoDerivs CC BY-NC-ND 4.0
000271333 915__ $$0StatID:(DE-HGF)0100$$2StatID$$aJCR$$bMUSCLE NERVE : 2022$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)3001$$2StatID$$aDEAL Wiley$$d2023-10-21$$wger
000271333 915__ $$0StatID:(DE-HGF)0113$$2StatID$$aWoS$$bScience Citation Index Expanded$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)1030$$2StatID$$aDBCoverage$$bCurrent Contents - Life Sciences$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)0150$$2StatID$$aDBCoverage$$bWeb of Science Core Collection$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)9900$$2StatID$$aIF < 5$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)0510$$2StatID$$aOpenAccess
000271333 915__ $$0StatID:(DE-HGF)0030$$2StatID$$aPeer Review$$bASC$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)0300$$2StatID$$aDBCoverage$$bMedline$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)1110$$2StatID$$aDBCoverage$$bCurrent Contents - Clinical Medicine$$d2023-10-21
000271333 915__ $$0StatID:(DE-HGF)0420$$2StatID$$aNationallizenz$$d2023-10-21$$wger
000271333 915__ $$0StatID:(DE-HGF)0199$$2StatID$$aDBCoverage$$bClarivate Analytics Master Journal List$$d2023-10-21
000271333 9201_ $$0I:(DE-2719)1011001$$kClinical Research (Bonn)$$lClinical Research Coordination$$x0
000271333 9201_ $$0I:(DE-2719)1710012$$kAG Falkenburger$$lTranslational Parkinson Research$$x1
000271333 9201_ $$0I:(DE-2719)5000077$$kClinical Study Center (Ulm)$$lClinical Study Center (Ulm)$$x2
000271333 980__ $$ajournal
000271333 980__ $$aVDB
000271333 980__ $$aUNRESTRICTED
000271333 980__ $$aI:(DE-2719)1011001
000271333 980__ $$aI:(DE-2719)1710012
000271333 980__ $$aI:(DE-2719)5000077
000271333 9801_ $$aFullTexts