Safety, efficacy, and tolerability of alemtuzumab in pediatric patients with active relapsing-remitting multiple sclerosis: The LemKids study.

Autor:	Chitnis T; Department of Pediatric Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Arnold DL; McGill University, Montreal, QC, Canada; NeuroRx Research, Montreal, QC, Canada., Quartier P; Université Paris-Cité, Paris, France.; RAISE Rare Disease Reference Centre, Pediatric Immunology-Hematology and Rheumatology Unit, Hôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France., Chirieac M; Sanofi, Cambridge, MA, USA., Hu W; Sanofi, Beijing, China., Jurgensen S; Sanofi, Bridgewater, NJ, USA., Havrdova EK; Department of Neurology, First Faculty of Medicine, Charles University, Prague, Czech Republic.
Jazyk:	angličtina
Zdroj:	Multiple sclerosis (Houndmills, Basingstoke, England) [Mult Scler] 2024 Dec 01, pp. 13524585241295554. Date of Electronic Publication: 2024 Dec 01.
DOI:	10.1177/13524585241295554
Abstrakt:	Background: Limited licensed medications are available for multiple sclerosis (MS) in pediatric patients. Objective: To evaluate the efficacy, safety, and tolerability of alemtuzumab in pediatric patients with relapsing-remitting multiple sclerosis (RRMS) and disease activity on prior disease-modifying therapies (DMTs). Methods: LemKids was a multicenter, multinational, single-arm, open-label, switch (from ongoing DMT to alemtuzumab treatment) study in pediatric RRMS patients (aged 10-<18 years), with disease activity on DMT. The primary endpoint was a comparison of the number of new/enlarging T2 lesions on the magnetic resonance imaging of the brain between the prior-DMT period and alemtuzumab treatment. Results: This study was prematurely terminated due to low enrollment and an European Medicines Agency Article-20 pharmacovigilance review of alemtuzumab in adult RRMS. Of 46 screened patients, 16 were enrolled; 12 completed prior-DMT treatment period; 11 received alemtuzumab of whom 7 completed treatment. Patients on alemtuzumab developed fewer new/enlarging T2 lesions compared with prior-DMT (7 vs 178, relative risk (95% confidence interval): 0.04 (0.01-0.14)). No significant pharmacodynamic changes or safety concerns were noted in this limited dataset. Conclusion: Alemtuzumab treatment was associated with a low number of new/enlarging T2 lesions in pediatric patients with RRMS and was safe and well tolerated in seven patients during infusion and the initial 4 months. Competing Interests: Declaration of Conflicting InterestsThe author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: T.C. received consulting fees from Genentech-Roche and Novartis; is member of advisory boards of Novartis, Roche-Genentech, and Sanofi; received research support from Novartis, Sanofi, Serono, and Verily; and received speaking fees from Medscape. D.L.A. received personal compensation for serving as a consultant for Alexion, Biogen, Celgene, Eli Lilly, EMD Serono, Frequency Therapeutics, Genentech, Merck, Novartis, Roche, Sanofi, and Shionogi; and holds an equity interest in NeuroRx. P.Q. received consultancy or speaking fees from AbbVie, Amgen, Bristol-Myers Squibb, Chugai-Roche, Eli Lilly, Novartis, Novimmune, Pfizer, and Swedish Orphan Biovitrum; and participated in two data safety monitoring boards for Sanofi. M.C., W.H., and S.J. are employees of Sanofi; may hold shares and/or stock options in the company. E.K.H. received honoraria and grant support from Actelion, Biogen, Merck Serono, Janssen, Novartis, Receptos, Roche, Sanofi, and Teva; and support from the Ministry of Education of Czech Republic project (Cooperation LF1, research area Neuroscience) and the National Institute for Neurological Research (Programme EXCELES, ID Project No. LX22NPO5107—funded by the European Union-Next Generation EU).
Databáze:	MEDLINE
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