Buchumschlag

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

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 o...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Hauptverfasser: Meyer, Thomas (VerfasserIn) , Schumann, Peggy (VerfasserIn) , Weydt, Patrick (VerfasserIn) , Petri, Susanne (VerfasserIn) , Weishaupt, Jochen H. (VerfasserIn) , Weyen, Ute (VerfasserIn) , Koch, Jan C. (VerfasserIn) , Günther, René (VerfasserIn) , Regensburger, Martin (VerfasserIn) , Boentert, Matthias (VerfasserIn) , Wiesenfarth, Maximilian (VerfasserIn) , Koc, Yasemin (VerfasserIn) , Kolzarek, Felix (VerfasserIn) , Kettemann, Dagmar (VerfasserIn) , Norden, Jenny (VerfasserIn) , Bernsen, Sarah (VerfasserIn) , Elmas, Zeynep (VerfasserIn) , Conrad, Julian (VerfasserIn) , Valkadinov, Ivan (VerfasserIn) , Vidovic, Maximilian (VerfasserIn) , Dorst, Johannes (VerfasserIn) , Ludolph, Albert C. (VerfasserIn) , Hesebeck-Brinckmann, Jasper (VerfasserIn) , Spittel, Susanne (VerfasserIn) , Münch, Christoph (VerfasserIn) , Maier, André (VerfasserIn) , Körtvélyessy, Péter (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: September 2024
In: Muscle & nerve
Year: 2024, Jahrgang: 70, Heft: 3, Pages: 333-345
ISSN:1097-4598
DOI:10.1002/mus.28182
Online-Zugang:Verlag, kostenfrei, Volltext: https://doi.org/10.1002/mus.28182
Verlag, kostenfrei, Volltext: http://onlinelibrary.wiley.com/doi/abs/10.1002/mus.28182
Volltext
Verfasserangaben:Thomas Meyer MD, Peggy Schumann MSc, Patrick Weydt MD, Susanne Petri MD, Jochen H. Weishaupt MD, Ute Weyen MD, Jan C. Koch MD, René Günther MD, Martin Regensburger MD, Matthias Boentert MD, Maximilian Wiesenfarth MD, Yasemin Koc RN, Felix Kolzarek MSc, Dagmar Kettemann MD, Jenny Norden MD, Sarah Bernsen MD, Zeynep Elmas MD, Julian Conrad MD, Ivan Valkadinov MD, Maximilian Vidovic MD, Johannes Dorst MD, Albert C. Ludolph MD, Jasper Hesebeck-Brinckmann MD, Susanne Spittel PhD, Christoph Münch MD, André Maier MD, Péter Körtvélyessy MD

MARC

LEADER 00000caa a2200000 c 4500
001 1909504920
003 DE-627
005 20250709093509.0
007 cr uuu---uuuuu
008 241126s2024 xx |||||o 00| ||eng c
024 7 |a 10.1002/mus.28182  |2 doi 
035 |a (DE-627)1909504920 
035 |a (DE-599)KXP1909504920 
035 |a (OCoLC)1475648078 
040 |a DE-627  |b ger  |c DE-627  |e rda 
041 |a eng 
084 |a 33  |2 sdnb 
100 1 |a Meyer, Thomas  |d 1967-  |e VerfasserIn  |0 (DE-588)122622065  |0 (DE-627)705974596  |0 (DE-576)293352143  |4 aut 
245 1 0 |a Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS  |b a multicenter observational study over 18 months  |c Thomas Meyer MD, Peggy Schumann MSc, Patrick Weydt MD, Susanne Petri MD, Jochen H. Weishaupt MD, Ute Weyen MD, Jan C. Koch MD, René Günther MD, Martin Regensburger MD, Matthias Boentert MD, Maximilian Wiesenfarth MD, Yasemin Koc RN, Felix Kolzarek MSc, Dagmar Kettemann MD, Jenny Norden MD, Sarah Bernsen MD, Zeynep Elmas MD, Julian Conrad MD, Ivan Valkadinov MD, Maximilian Vidovic MD, Johannes Dorst MD, Albert C. Ludolph MD, Jasper Hesebeck-Brinckmann MD, Susanne Spittel PhD, Christoph Münch MD, André Maier MD, Péter Körtvélyessy MD 
264 1 |c September 2024 
300 |a 13 
336 |a Text  |b txt  |2 rdacontent 
337 |a Computermedien  |b c  |2 rdamedia 
338 |a Online-Ressource  |b cr  |2 rdacarrier 
500 |a Erstmals veröffentlicht: 20. Juni 2024 
500 |a Gesehen am 26.11.2024 
520 |a 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. 
650 4 |a amyotrophic lateral sclerosis (ALS) 
650 4 |a clinical course 
650 4 |a neurofilament light chain (NfL) 
650 4 |a patient-reported outcomes 
650 4 |a tofersen 
700 1 |a Schumann, Peggy  |e VerfasserIn  |4 aut 
700 1 |a Weydt, Patrick  |e VerfasserIn  |4 aut 
700 1 |a Petri, Susanne  |e VerfasserIn  |4 aut 
700 1 |a Weishaupt, Jochen H.  |d 1971-  |e VerfasserIn  |0 (DE-588)122148924  |0 (DE-627)705789039  |0 (DE-576)293117810  |4 aut 
700 1 |a Weyen, Ute  |e VerfasserIn  |4 aut 
700 1 |a Koch, Jan C.  |e VerfasserIn  |4 aut 
700 1 |a Günther, René  |e VerfasserIn  |4 aut 
700 1 |a Regensburger, Martin  |e VerfasserIn  |4 aut 
700 1 |a Boentert, Matthias  |e VerfasserIn  |4 aut 
700 1 |a Wiesenfarth, Maximilian  |e VerfasserIn  |4 aut 
700 1 |a Koc, Yasemin  |e VerfasserIn  |4 aut 
700 1 |a Kolzarek, Felix  |e VerfasserIn  |4 aut 
700 1 |a Kettemann, Dagmar  |e VerfasserIn  |0 (DE-588)1167602706  |0 (DE-627)1031197184  |0 (DE-576)511211643  |4 aut 
700 1 |a Norden, Jenny  |e VerfasserIn  |4 aut 
700 1 |a Bernsen, Sarah  |e VerfasserIn  |4 aut 
700 1 |a Elmas, Zeynep  |e VerfasserIn  |4 aut 
700 1 |a Conrad, Julian  |d 1984-  |e VerfasserIn  |0 (DE-588)1045448508  |0 (DE-627)774514965  |0 (DE-576)399216510  |4 aut 
700 1 |a Valkadinov, Ivan  |e VerfasserIn  |0 (DE-588)1275314384  |0 (DE-627)1826773231  |4 aut 
700 1 |a Vidovic, Maximilian  |e VerfasserIn  |4 aut 
700 1 |a Dorst, Johannes  |e VerfasserIn  |4 aut 
700 1 |a Ludolph, Albert C.  |d 1953-  |e VerfasserIn  |0 (DE-588)1081317531  |0 (DE-627)845970100  |0 (DE-576)454116195  |4 aut 
700 1 |a Hesebeck-Brinckmann, Jasper  |d 1995-  |e VerfasserIn  |0 (DE-588)1349350052  |0 (DE-627)1909505617  |4 aut 
700 1 |a Spittel, Susanne  |e VerfasserIn  |4 aut 
700 1 |a Münch, Christoph  |e VerfasserIn  |4 aut 
700 1 |a Maier, André  |e VerfasserIn  |4 aut 
700 1 |a Körtvélyessy, Péter  |d 1977-  |e VerfasserIn  |0 (DE-588)1042157146  |0 (DE-627)768240603  |0 (DE-576)393716511  |4 aut 
773 0 8 |i Enthalten in  |t Muscle & nerve  |d New York, NY [u.a.] : Wiley, 1978  |g 70(2024), 3 vom: Sept., Seite 333-345  |h Online-Ressource  |w (DE-627)270128891  |w (DE-600)1476641-3  |w (DE-576)079876161  |x 1097-4598  |7 nnas  |a Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS a multicenter observational study over 18 months 
773 1 8 |g volume:70  |g year:2024  |g number:3  |g month:09  |g pages:333-345  |g extent:13  |a Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS a multicenter observational study over 18 months 
856 4 0 |u https://doi.org/10.1002/mus.28182  |x Verlag  |x Resolving-System  |z kostenfrei  |3 Volltext 
856 4 0 |u http://onlinelibrary.wiley.com/doi/abs/10.1002/mus.28182  |x Verlag  |z kostenfrei  |3 Volltext 
951 |a AR 
992 |a 20241126 
993 |a Article 
994 |a 2024 
998 |g 1349350052  |a Hesebeck-Brinckmann, Jasper  |m 1349350052:Hesebeck-Brinckmann, Jasper  |d 60000  |e 60000PH1349350052  |k 0/60000/  |p 22 
998 |g 1275314384  |a Valkadinov, Ivan  |m 1275314384:Valkadinov, Ivan  |d 60000  |e 60000PV1275314384  |k 0/60000/  |p 19 
998 |g 1045448508  |a Conrad, Julian  |m 1045448508:Conrad, Julian  |d 60000  |d 62700  |d 60000  |e 60000PC1045448508  |e 62700PC1045448508  |e 60000PC1045448508  |k 0/60000/  |k 1/60000/62700/  |k 0/60000/  |p 18 
998 |g 122148924  |a Weishaupt, Jochen H.  |m 122148924:Weishaupt, Jochen H.  |d 60000  |d 62700  |e 60000PW122148924  |e 62700PW122148924  |k 0/60000/  |k 1/60000/62700/  |p 5 
999 |a KXP-PPN1909504920  |e 4622292378 
BIB |a Y 
SER |a journal 
JSO |a {"note":["Erstmals veröffentlicht: 20. Juni 2024","Gesehen am 26.11.2024"],"relHost":[{"pubHistory":["1.1978 -"],"language":["eng"],"physDesc":[{"extent":"Online-Ressource"}],"type":{"bibl":"periodical","media":"Online-Ressource"},"note":["Gesehen am 28.02.08"],"titleAlt":[{"title":"Muscle and nerve"}],"title":[{"subtitle":"official journal of the American Association of Electrodiagnostic Medicine","title":"Muscle & nerve","title_sort":"Muscle & nerve"}],"disp":"Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS a multicenter observational study over 18 monthsMuscle & nerve","recId":"270128891","part":{"issue":"3","year":"2024","pages":"333-345","volume":"70","extent":"13","text":"70(2024), 3 vom: Sept., Seite 333-345"},"id":{"eki":["270128891"],"zdb":["1476641-3"],"issn":["1097-4598"],"doi":["10.1002/(ISSN)1097-4598"]},"origin":[{"publisherPlace":"New York, NY [u.a.]","publisher":"Wiley","dateIssuedKey":"1978","dateIssuedDisp":"1978-"}]}],"title":[{"subtitle":"a multicenter observational study over 18 months","title":"Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS","title_sort":"Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS"}],"person":[{"given":"Thomas","role":"aut","family":"Meyer","display":"Meyer, Thomas"},{"family":"Schumann","role":"aut","given":"Peggy","display":"Schumann, Peggy"},{"display":"Weydt, Patrick","family":"Weydt","given":"Patrick","role":"aut"},{"role":"aut","given":"Susanne","family":"Petri","display":"Petri, Susanne"},{"role":"aut","given":"Jochen H.","family":"Weishaupt","display":"Weishaupt, Jochen H."},{"display":"Weyen, Ute","role":"aut","given":"Ute","family":"Weyen"},{"display":"Koch, Jan C.","role":"aut","given":"Jan C.","family":"Koch"},{"family":"Günther","given":"René","role":"aut","display":"Günther, René"},{"display":"Regensburger, Martin","family":"Regensburger","role":"aut","given":"Martin"},{"given":"Matthias","role":"aut","family":"Boentert","display":"Boentert, Matthias"},{"family":"Wiesenfarth","role":"aut","given":"Maximilian","display":"Wiesenfarth, Maximilian"},{"display":"Koc, Yasemin","given":"Yasemin","role":"aut","family":"Koc"},{"given":"Felix","role":"aut","family":"Kolzarek","display":"Kolzarek, Felix"},{"role":"aut","given":"Dagmar","family":"Kettemann","display":"Kettemann, Dagmar"},{"display":"Norden, Jenny","given":"Jenny","role":"aut","family":"Norden"},{"role":"aut","given":"Sarah","family":"Bernsen","display":"Bernsen, Sarah"},{"display":"Elmas, Zeynep","family":"Elmas","role":"aut","given":"Zeynep"},{"display":"Conrad, Julian","family":"Conrad","role":"aut","given":"Julian"},{"family":"Valkadinov","role":"aut","given":"Ivan","display":"Valkadinov, Ivan"},{"given":"Maximilian","role":"aut","family":"Vidovic","display":"Vidovic, Maximilian"},{"family":"Dorst","given":"Johannes","role":"aut","display":"Dorst, Johannes"},{"display":"Ludolph, Albert C.","role":"aut","given":"Albert C.","family":"Ludolph"},{"display":"Hesebeck-Brinckmann, Jasper","family":"Hesebeck-Brinckmann","role":"aut","given":"Jasper"},{"display":"Spittel, Susanne","given":"Susanne","role":"aut","family":"Spittel"},{"family":"Münch","role":"aut","given":"Christoph","display":"Münch, Christoph"},{"role":"aut","given":"André","family":"Maier","display":"Maier, André"},{"display":"Körtvélyessy, Péter","role":"aut","given":"Péter","family":"Körtvélyessy"}],"physDesc":[{"extent":"13 S."}],"language":["eng"],"type":{"media":"Online-Ressource","bibl":"article-journal"},"origin":[{"dateIssuedDisp":"September 2024","dateIssuedKey":"2024"}],"id":{"eki":["1909504920"],"doi":["10.1002/mus.28182"]},"recId":"1909504920","name":{"displayForm":["Thomas Meyer MD, Peggy Schumann MSc, Patrick Weydt MD, Susanne Petri MD, Jochen H. Weishaupt MD, Ute Weyen MD, Jan C. Koch MD, René Günther MD, Martin Regensburger MD, Matthias Boentert MD, Maximilian Wiesenfarth MD, Yasemin Koc RN, Felix Kolzarek MSc, Dagmar Kettemann MD, Jenny Norden MD, Sarah Bernsen MD, Zeynep Elmas MD, Julian Conrad MD, Ivan Valkadinov MD, Maximilian Vidovic MD, Johannes Dorst MD, Albert C. Ludolph MD, Jasper Hesebeck-Brinckmann MD, Susanne Spittel PhD, Christoph Münch MD, André Maier MD, Péter Körtvélyessy MD"]}} 
SRT |a MEYERTHOMACLINICALAN2024 

Ähnliche Einträge