Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis

Background: Muscle-specific tyrosine kinase (MuSK) autoantibody-positive (Ab+) generalised myasthenia gravis (gMG) is a rare and frequently severe subtype of gMG. Objectives: To assess the efficacy and safety of rozanolixizumab in the subgroup of patients with MuSK Ab+ gMG in the MycarinG study. Des...

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Autores: Habib, A.A., Sacconi, S., Cortés-Vicente, Elena|||0000-0002-1428-1072, Mahuwala, Z.K., Mantegazza, Renato|||0000-0002-9810-5737, Vissing, John|||0000-0001-6144-8544, Wiendl, Heinz|||0000-0003-4310-3432, Bril, V., Antonini, Giovanni, Grosskreutz, Julian|||0000-0001-9525-1424, Pascuzzi, Robert M., Utsugisawa, Kimiaki, Vu, Tuan H., Boehnlein, Marion, Greve, Bernhard, Woltering, Franz
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:324385
Acceso en línea:https://ddd.uab.cat/record/324385
https://dx.doi.org/urn:doi:10.1177/17562864241273036
Access Level:acceso abierto
Palabra clave:Muscle-specific tyrosine kinase
Muscle-specific tyrosine kinase autoantibody positive
Myasthenia gravis
Rozanolixizumab
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repository_id_str
spelling Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravisa subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG studyHabib, A.A.Sacconi, S.Cortés-Vicente, Elena|||0000-0002-1428-1072Mahuwala, Z.K.Mantegazza, Renato|||0000-0002-9810-5737Vissing, John|||0000-0001-6144-8544Wiendl, Heinz|||0000-0003-4310-3432Bril, V.Antonini, GiovanniGrosskreutz, Julian|||0000-0001-9525-1424Pascuzzi, Robert M.Utsugisawa, KimiakiVu, Tuan H.Boehnlein, MarionGreve, BernhardWoltering, FranzMuscle-specific tyrosine kinaseMuscle-specific tyrosine kinase autoantibody positiveMyasthenia gravisRozanolixizumabBackground: Muscle-specific tyrosine kinase (MuSK) autoantibody-positive (Ab+) generalised myasthenia gravis (gMG) is a rare and frequently severe subtype of gMG. Objectives: To assess the efficacy and safety of rozanolixizumab in the subgroup of patients with MuSK Ab+ gMG in the MycarinG study. Design: A randomised, double-blind, placebo-controlled phase III study. Methods: Patients with acetylcholine receptor (AChR) Ab+ or MuSK Ab+ gMG (aged ⩾18 years, Myasthenia Gravis Foundation of America Disease Class II-IVa, Myasthenia Gravis Activities of Daily Living [MG-‍ADL] score ⩾3.0 [non-ocular symptoms], Quantitative Myasthenia Gravis score ⩾11.0) were randomly assigned (1:1:1) to receive once-weekly subcutaneous infusions of rozanolixizumab 7 mg/kg, rozanolixizumab 10 mg/kg or placebo for 6 weeks, followed by an 8-week observation period. Randomisation was stratified by AChR and MuSK autoantibody status. The primary study endpoint was change from baseline to Day 43 in MG-ADL score. Treatment-emergent adverse events (TEAEs) were also assessed. Results: Overall, 200 patients were randomised, of whom 21 had MuSK Ab+ gMG and received rozanolixizumab 7 mg/kg (n = 5), 10 mg/kg (n = 8) or placebo (n = 8). In patients with MuSK Ab+ gMG, reductions from baseline to Day 43 in MG-ADL scores were observed: rozanolixizumab 7 mg/kg least squares mean (LSM) change (standard error), -7.28 (1.94); 10 mg/kg, -4.16 (1.78); and placebo, 2.28 (1.95). Rozanolixizumab 7 mg/kg LSM difference from placebo was -9.56 (97.5% confidence interval: -15.25, -3.87); 10 mg/kg, -6.45 (-11.03, -1.86). TEAEs were experienced by four (80.0%), five (62.5%) and three (37.5%) patients with MuSK Ab+ gMG receiving rozanolixizumab 7 mg/kg, 10 mg/kg and placebo, respectively. No patients experienced serious TEAEs. No deaths occurred. Conclusion: This subgroup analysis of adult patients with MuSK Ab+ gMG enrolled in the MycarinG study supports the use of rozanolixizumab as an effective treatment option for patients with gMG who have MuSK autoantibodies. Trial registration: ClinicalTrials.gov: NCT03971422 (https://clinicaltrials.gov/study/NCT03971422); EU Clinical Trials Register: EudraCT 2019-000968-18 (https://www.clinicaltrials‌register.eu/ctr-search/trial/2019-000968-18/GB).Universitat Autònoma de Barcelona. Departament de Medicina 22024-01-0120242024-01-01Articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://ddd.uab.cat/record/324385https://dx.doi.org/urn:doi:10.1177/17562864241273036reponame:Dipòsit Digital de Documents de la UABinstname:Universitat Autònoma de BarcelonaInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.https://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:ddd.uab.cat:3243852026-06-06T12:50:31Z
dc.title.none.fl_str_mv Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study
title Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
spellingShingle Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
Habib, A.A.
Muscle-specific tyrosine kinase
Muscle-specific tyrosine kinase autoantibody positive
Myasthenia gravis
Rozanolixizumab
title_short Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
title_full Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
title_fullStr Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
title_full_unstemmed Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
title_sort Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis
dc.creator.none.fl_str_mv Habib, A.A.
Sacconi, S.
Cortés-Vicente, Elena|||0000-0002-1428-1072
Mahuwala, Z.K.
Mantegazza, Renato|||0000-0002-9810-5737
Vissing, John|||0000-0001-6144-8544
Wiendl, Heinz|||0000-0003-4310-3432
Bril, V.
Antonini, Giovanni
Grosskreutz, Julian|||0000-0001-9525-1424
Pascuzzi, Robert M.
Utsugisawa, Kimiaki
Vu, Tuan H.
Boehnlein, Marion
Greve, Bernhard
Woltering, Franz
author Habib, A.A.
author_facet Habib, A.A.
Sacconi, S.
Cortés-Vicente, Elena|||0000-0002-1428-1072
Mahuwala, Z.K.
Mantegazza, Renato|||0000-0002-9810-5737
Vissing, John|||0000-0001-6144-8544
Wiendl, Heinz|||0000-0003-4310-3432
Bril, V.
Antonini, Giovanni
Grosskreutz, Julian|||0000-0001-9525-1424
Pascuzzi, Robert M.
Utsugisawa, Kimiaki
Vu, Tuan H.
Boehnlein, Marion
Greve, Bernhard
Woltering, Franz
author_role author
author2 Sacconi, S.
Cortés-Vicente, Elena|||0000-0002-1428-1072
Mahuwala, Z.K.
Mantegazza, Renato|||0000-0002-9810-5737
Vissing, John|||0000-0001-6144-8544
Wiendl, Heinz|||0000-0003-4310-3432
Bril, V.
Antonini, Giovanni
Grosskreutz, Julian|||0000-0001-9525-1424
Pascuzzi, Robert M.
Utsugisawa, Kimiaki
Vu, Tuan H.
Boehnlein, Marion
Greve, Bernhard
Woltering, Franz
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Universitat Autònoma de Barcelona. Departament de Medicina
dc.subject.none.fl_str_mv Muscle-specific tyrosine kinase
Muscle-specific tyrosine kinase autoantibody positive
Myasthenia gravis
Rozanolixizumab
topic Muscle-specific tyrosine kinase
Muscle-specific tyrosine kinase autoantibody positive
Myasthenia gravis
Rozanolixizumab
description Background: Muscle-specific tyrosine kinase (MuSK) autoantibody-positive (Ab+) generalised myasthenia gravis (gMG) is a rare and frequently severe subtype of gMG. Objectives: To assess the efficacy and safety of rozanolixizumab in the subgroup of patients with MuSK Ab+ gMG in the MycarinG study. Design: A randomised, double-blind, placebo-controlled phase III study. Methods: Patients with acetylcholine receptor (AChR) Ab+ or MuSK Ab+ gMG (aged ⩾18 years, Myasthenia Gravis Foundation of America Disease Class II-IVa, Myasthenia Gravis Activities of Daily Living [MG-‍ADL] score ⩾3.0 [non-ocular symptoms], Quantitative Myasthenia Gravis score ⩾11.0) were randomly assigned (1:1:1) to receive once-weekly subcutaneous infusions of rozanolixizumab 7 mg/kg, rozanolixizumab 10 mg/kg or placebo for 6 weeks, followed by an 8-week observation period. Randomisation was stratified by AChR and MuSK autoantibody status. The primary study endpoint was change from baseline to Day 43 in MG-ADL score. Treatment-emergent adverse events (TEAEs) were also assessed. Results: Overall, 200 patients were randomised, of whom 21 had MuSK Ab+ gMG and received rozanolixizumab 7 mg/kg (n = 5), 10 mg/kg (n = 8) or placebo (n = 8). In patients with MuSK Ab+ gMG, reductions from baseline to Day 43 in MG-ADL scores were observed: rozanolixizumab 7 mg/kg least squares mean (LSM) change (standard error), -7.28 (1.94); 10 mg/kg, -4.16 (1.78); and placebo, 2.28 (1.95). Rozanolixizumab 7 mg/kg LSM difference from placebo was -9.56 (97.5% confidence interval: -15.25, -3.87); 10 mg/kg, -6.45 (-11.03, -1.86). TEAEs were experienced by four (80.0%), five (62.5%) and three (37.5%) patients with MuSK Ab+ gMG receiving rozanolixizumab 7 mg/kg, 10 mg/kg and placebo, respectively. No patients experienced serious TEAEs. No deaths occurred. Conclusion: This subgroup analysis of adult patients with MuSK Ab+ gMG enrolled in the MycarinG study supports the use of rozanolixizumab as an effective treatment option for patients with gMG who have MuSK autoantibodies. Trial registration: ClinicalTrials.gov: NCT03971422 (https://clinicaltrials.gov/study/NCT03971422); EU Clinical Trials Register: EudraCT 2019-000968-18 (https://www.clinicaltrials‌register.eu/ctr-search/trial/2019-000968-18/GB).
publishDate 2024
dc.date.none.fl_str_mv 2
2024-01-01
2024
2024-01-01
dc.type.none.fl_str_mv Article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://ddd.uab.cat/record/324385
https://dx.doi.org/urn:doi:10.1177/17562864241273036
url https://ddd.uab.cat/record/324385
https://dx.doi.org/urn:doi:10.1177/17562864241273036
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Dipòsit Digital de Documents de la UAB
instname:Universitat Autònoma de Barcelona
instname_str Universitat Autònoma de Barcelona
reponame_str Dipòsit Digital de Documents de la UAB
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