Long-term effectiveness and safety of ocrelizumab: a single-centre real-world study
- PMID: 40581721
- DOI: 10.1007/s00415-025-13224-8
Long-term effectiveness and safety of ocrelizumab: a single-centre real-world study
Abstract
Background: This retrospective single-centre study evaluated the effectiveness and safety of ocrelizumab (OCR) in relapse-onset multiple sclerosis (RMS) and primary progressive MS (PPMS) and identified predictors of treatment response.
Methods: We included 260 RMS and 73 PPMS patients treated with ocrelizumab for ≥ 1 year at our MS Centre until May 2024.
Results: Median follow-up was 3.90 years for RMS and 4.23 years for PPMS. Within 2 years from treatment initiation, annualized relapse rate (ARR) decreased from 0.412 to 0.014 and was maintained low throughout follow-up in RMS, with no relapses in PPMS. MRI activity significantly declined and was maintained in both groups (p < 0.0001). After 3 years, confirmed disability progression (CDP)-free survival was high in relapsing-remitting MS (> 97%) and lower in secondary progressive MS (48.9%) and PPMS (57.2%). Predictors of ocrelizumab inefficacy included higher baseline Expanded Disability Status Scale (EDSS), older age and longer disease duration in RMS; male sex, older age and prior lower-efficacy treatments in PPMS. Adverse events were in line with previous clinical studies, with hypogammaglobulinemia and recurrent infections being the most frequent.
Conclusions: In this study we confirm ocrelizumab sustained efficacy in controlling inflammatory disease activity, with greater impact in RMS, with a favourable safety profile. Early treatment initiation is crucial to prevent irreversible disability accumulation.
Keywords: Anti-CD20; Multiple sclerosis; NEDA; Ocrelizumab; Real-life; Safety.
© 2025. Springer-Verlag GmbH Germany, part of Springer Nature.
Conflict of interest statement
Declarations. Conflicts of interest: The authors declare that they have no competing interests in relation to this work. Potential conflicts of interest outside the submitted work are as follows: TZ has nothing to disclose. LM received compensations for speaking activities and/or for participating to advisory board from Merck, Celgene, Biogen, Sanofi, Novartis, Roche, Alexion, and Amgen. SG received compensation for speaking activities from Bristol Squibb Meyer, Novartis, Merck. AN has nothing to disclose. CZ received compensation for speaking activities, and/or consulting services, from Alexion, AstraZeneca, Biogen, Bristol Myers Squibb, Janssen, Merck, Novartis, Roche, Sanofi. IG has nothing to disclose. AG received compensation for speaking activities from Novartis. FE received consulting and speaking fees from Novartis and Sanofi Genzyme. MGS has nothing to disclose. PMVR has nothing to disclose. M.A. Rocca received consulting fees from Biogen, Bristol Myers Squibb, Roche; and speaker honoraria from Alexion, Biogen, Bristol Myers Squibb, Celgene, Horizon Therapeutics Italy, Merck Serono SpA, Mitsubishi-Tanabe Pharma, Neuraxpharm, Novartis, Roche, Sandoz, and Sanofi. She receives research support from the MS Society of Canada, the Italian Ministry of Health, the Italian Ministry of University and Research, and Fondazione Italiana Sclerosi Multipla. She is Associate Editor for Multiple Sclerosis and Related Disorders; and Associate Co-Editor for Europe and Africa for Multiple Sclerosis Journal. M. Filippi is Editor-in-Chief of the Journal of Neurology, Associate Editor of Human Brain Mapping, Neurological Sciences, and Radiology; received compensation for consulting services from Alexion, Almirall, Biogen, Merck, Novartis, Roche, Sanofi; speaking activities from Bayer, Biogen, Celgene, Chiesi Italia SpA, Eli Lilly, Genzyme, Janssen, Merck-Serono, Neopharmed Gentili, Novartis, Novo Nordisk, Roche, Sanofi, Takeda, and TEVA; participation in Advisory Boards for Alexion, Biogen, Bristol-Myers Squibb, Merck, Novartis, Roche, Sanofi, Sanofi-Aventis, Sanofi-Genzyme, Takeda; scientific direction of educational events for Biogen, Merck, Roche, Celgene, Bristol-Myers Squibb, Lilly, Novartis, Sanofi-Genzyme; he receives research support from Biogen Idec, Merck-Serono, Novartis, Roche, the Italian Ministry of Health, the Italian Ministry of University and Research, and Fondazione Italiana Sclerosi Multipla. Ethical standards: Approval was received from the institutional ethical standards committee on human experimentation of IRCCS Ospedale San Raffaele for any experiments using human subjects. Written informed consent was obtained from all subjects prior to study participation according to the Declaration of Helsinki.
References
-
- Hauser SL, Bar-Or A, Comi G et al (2017) Ocrelizumab versus interferon beta-1a in relapsing multiple sclerosis. N Engl J Med 376(3):221–234. https://doi.org/10.1056/NEJMoa1601277 - DOI - PubMed
-
- Montalban X, Hauser SL, Kappos L et al (2017) Ocrelizumab versus placebo in primary progressive multiple sclerosis. N Engl J Med 376(3):209–220. https://doi.org/10.1056/NEJMoa1606468 - DOI - PubMed
-
- Filippi M, Amato MP, Centonze D, Gallo P, Gasperini C, Inglese M, Patti F, Pozzilli C, Preziosa P, Trojano M (2022) Early use of high-efficacy disease-modifying therapies makes the difference in people with multiple sclerosis: an expert opinion. J Neurol 269(10):5382–5394. https://doi.org/10.1007/s00415-022-11193-w - DOI - PubMed - PMC
-
- Hauser SL, Kappos L, Arnold DL et al (2020) Five years of ocrelizumab in relapsing multiple sclerosis: OPERA studies open-label extension. Neurology 95(13):e1854–e1867. https://doi.org/10.1212/WNL.0000000000010376 - DOI - PubMed - PMC
-
- Wolinsky JS, Arnold DL, Brochet B et al (2020) Long-term follow-up from the ORATORIO trial of ocrelizumab for primary progressive multiple sclerosis: a post-hoc analysis from the ongoing open-label extension of the randomised, placebo-controlled, phase 3 trial. Lancet Neurol 19(12):998–1009. https://doi.org/10.1016/S1474-4422(20)30342-2 - DOI - PubMed
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