Real-World Experience with Diroximel Fumarate in Patients with Multiple Sclerosis: A Prospective Multicenter Study.

Autor: Aguirre C; Neurology Department, Hospital Universitario de La Princesa, Calle de Diego de León, 62, 28006, Madrid, Spain. claraguih@hotmail.com., Alonso-Torres A; Neurology Department, Hospital Regional de Málaga, Málaga, Spain., Agüera E; Maimonides Biomedical Research Institute of Cordoba (IMIBIC), Hospital Universitario Reina Sofía, University of Cordoba, Córdoba, Spain., García-Domínguez JM; Neurology Department, Hospital General Universitario Gregorio Marañón, Madrid, Spain., Montero-Escribano P; Neurology Department, Hospital Clínico San Carlos, IdISSC, Madrid, Spain., González-Quintanilla V; Neurology Department, Hospital Universitario Marqués de Valdecilla, Santander, Spain., Costa-Frossard L; Neurology Department, Hospital Universitario Ramón y Cajal, Madrid, Spain., Oreja-Guevara C; Neurology Department, Hospital Clínico San Carlos, IdISSC, Madrid, Spain., Reyes-Garrido V; Neurology Department, Hospital Regional de Málaga, Málaga, Spain., Caminero-Rodríguez AB; Neurology Department, Complejo Asistencial de Ávila, Ávila, Spain., Riancho J; Neurology Department, Hospital General de Sierrallana, IDIVAL, CIBERNED, Dpto Medicina y Psiquiatría Universidad de Cantabria, Torrelavega, Spain., Sánchez O; Neurology Department, Hospital General Universitario Nuestra Señora del Prado, Talavera de la Reina, Spain., Forero L; Neurology Department, Hospital Puerta del Mar, Cádiz, Spain., Pérez-Parra F; Neurology Department, Hospital del Henares, Madrid, Spain., Ares-Luque A; Neurology Department, Complejo Asistencial Universitario de León, León, Spain., Téllez N; Neurology Department, Hospital Clínico Universitario de Valladolid, Valladolid, Spain., Arzalluz-Luque J; Neurology Department, Hospital Universitario Virgen Macarena, Sevilla, Spain., Iglesias F; Neurology Department, Hospital Universitario de Burgos, Burgos, Spain., Casado-Ruiz V; Neurology Department, Hospital de Mataró, Mataró, Spain., Castellano-Vicente AJ; Neurology Department, Hospital General de Valdepeñas, Ciudad Real, Spain., Borrega L; Neurology Department, Hospital Universitario Fundación Alcorcón, Madrid, Spain., Galán V; Neurology Department, Hospital Universitario de Toledo, Toledo, Spain., de Antonio LAR; Neurology Department, Hospital Universitario de Fuenlabrada, Madrid, Spain., Romero C; Neurology Department, Hospital Universitario de Albacete, Albacete, Spain., García-Rodríguez R; Neurology Department, Centro Médico de Asturias, Oviedo, Spain., Cano-Orgaz AT; Neurology Department, Hospital de Mataró, Mataró, Spain., Sánchez-Menoyo JL; Neurology Department, Hospital Universitario de Galdakao Usansolo, Galdakao (Bizkaia), Spain., Pérez-Ruiz D; Neurology Department, Hospital Comarcal del Bierzo, Ponferrada, Spain., Gutiérrez-Martin F; Neurology Department, Hospital Río Carrión, Palencia, Spain., Hernández-Echevarría L; Neurology Department, Complejo Asistencial Universitario de León, León, Spain., Meca-Lallana V; Neurology Department, Hospital Universitario de La Princesa, Calle de Diego de León, 62, 28006, Madrid, Spain.
Jazyk: angličtina
Zdroj: Clinical drug investigation [Clin Drug Investig] 2024 Nov; Vol. 44 (11), pp. 829-838. Date of Electronic Publication: 2024 Oct 28.
DOI: 10.1007/s40261-024-01397-5
Abstrakt: Background: Current literature and a real-world study suggest that diroximel fumarate (DRF) is safer than dimethyl fumarate (DMF) in the treatment of multiple sclerosis (MS). However, no real-world study to date has significantly addressed the efficacy of this treatment.
Objectives: This study aims to elucidate the safety, tolerability, and efficacy of DRF in a real-world setting, utilizing data from a Spanish national registry of patients commencing DRF therapy post-market introduction.
Methods: In this multicenter, prospective observational study, data were collected from MS patients who initiated DRF treatment. The study monitored demographic and clinical characteristics, safety outcomes (including adverse events, reasons for discontinuation, and lymphocyte counts), and efficacy outcomes (radiological and clinical activity).
Results: A total of 195 MS patients across 26 neurological departments were included, predominantly female (79.5%), with a mean age of 42.17 years, and a mean duration of treatment with DRF of 6.3 months. Most patients (70.3%) reported no adverse events, while gastrointestinal issues and flushing were the most common adverse events observed. The majority of patients (84.6%) continued with DRF treatment, with tolerability issues being the primary reason for discontinuation. Efficacy analysis showed low relapse rates post-DRF initiation, with most patients exhibiting stable or improved Expanded Disability Status Scale scores and radiological assessments demonstrating minimal activity.
Conclusion: This comprehensive analysis provides valuable insights into the real-world application of DRF, confirming its safety and tolerability while offering preliminary evidence of its efficacy in managing MS.
Competing Interests: Declarations Funding Writing and editorial assistance was provided by Content Ed Net (Madrid, Spain) with funding from Biogen. Conflict of interest C.A. has received lecture honoraria, consultancy fees, and travel expenses from Biogen, Bristol Mayers Squibb, Merck, Novartis, Sanofi-Genzyme and Roche. A.A.T. has received fees as a speaker, consultant or travel support from Biogen, Almirall, Merck, Roche, Sanofi, Sandoz and Janssen. E.A. has no disclosures for financial affiliation, financial support nor grants moneys and has disclosure for speaker honorarium from Novartis, Merck and Biogen. JM.G.D. has received compensation as a consultant, researcher, or speaker from Biogen, Sanofi, Roche, Zenas Biopharma, Almirall, Novartis, Merck, Bristol-Myers-Squidd, and Johnson & Johnson. PM-E has received speaker and consultation fees from Allergan, Almirall, Biogen Idec, Merck, Merz, Novartis and Sanofi-Genzyme. V.G.Q has no conflict of interest related to this work. L.C.F. has received lecture honoraria, consultancy fees, clinical research funding, and travel expenses from Almirall, Amgen, AstraZeneca, Biogen, Bristol Mayers Squibb, Janssen, Merck, Neuraxpharma, Novartis, Sanofi-Genzyme, Roche. C.O.G. has received speaker and consultation fees from Alexion, Biogen Idec, BMS, Horizon, Janssen, Merck, Novartis, Roche, Sanofi-Genzyme and Teva. V.R.G. has received fees as a speaker, consultant or travel support from Almirall, Merck, Alexion, Roche and Sanofi. AB.C.R. has received honoraria as speaker/meeting moderator/courses/symposium organized by Almirall Prodesfarma S.A, Biogen Idec Inc, Bristol-Myers-Squibb, Janssen Pharmaceutical; Merck-Serono, Mylan, Novartis Pharmaceutical, Roche, Sanofi-Genzyme, Teva Pharmaceuticals; and for congress assistance from Biogen Idec Inc, Bial, Merck-Serono, Novartis Pharmaceutical, Roche, Sanofi-Genzyme, Teva Pharmaceuticals. J.R. has received speaking/consulting fees and/or travel funding from Merck, Sanofi-Genzyme, Roche, Biogen, Novartis, BMS, Jannsen and Teva. O.S. has received fees from Almirall, Biogen, Novartis, Merck, Sanofi, Alter, Bial, Teva, Neuraxfarma, Esteve, Pfizer, Roche and UCB. L.F. has no conflict of interest related to this work. F.P.P has no conflict of interest related to this work. A.A.R. has received fees as a speaker and advisor, and funding for attendance at congresses and other medical meetings, from Bayer, Biogen, Bristol, Janssen, Merck, Novartis, Roche, Sanofi and Teva. J.A.L. received consultant fees from Merck. F.I. has received speaker honoraria and travel reimbursement from Biogen, Merck, Novartis, Roche Spain, and Genzyme-Sanofi. V.C.R has no conflict of interest related to this work. AJ.C.V. has no conflict of interest related to this work. L.B. has no conflict of interest related to this work. V.G. has received speaker fees from Merck, Roche, Biogen, Novartis and Sanofi. L.A.R.A. declares to have received travel aid and financial compensation for talks from BMS and Novartis. R.G.R received payments from Biogen for participating in training conferences. AT.C.O. has received payments from Biogen for participating in training conferences for other professionals. JL.S.M. accepted travel compensation from Novartis, Merck and Biogen, speaking honoraria from Biogen, Novartis, Sanofi, Merck, Almirall, Bayer and Teva and has participated in clinical trials by Biogen, Sanofi, Merck and Roche. D.P.R. has received honoraria as a speaker, funding to attend courses and conferences, and consultancies and research grants from Merck, Biogen, Novartis, Sanofi, Teva, Roche and Almirall. F.G.M has received speaker honoraria and travel reimbursement from Biogen, Merck, Novartis, Roche Spain, and Genzyme-Sanofi. L.H.E. has received speaker and consultation fees from Biogen, Merck, Novartis, Roche and Sanofi. Ethics approval The research protocol received approval from the independent ethics committee at the Hospital Universitario de La Princesa (10 November 2022, acta CEIM 5723). Consent to participate This investigation adhered to the principles of the Helsinki Declaration and complied with the EU General Data Protection Regulation (GDPR). All personal identifiers were excluded from the findings. Consent for publication Not applicable. Data availability statement Data are available from the corresponding author upon reasonable request. Author contributions CAH and VML made substantial contributions to the conception and design of the work, interpretation of data and drafting the manuscript. All authors contributed to the acquisition of data and revised the manuscript and approved the version to be published. Code availability Not applicable.
(© 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)
Databáze: MEDLINE
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