Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients.

DOI: http://doi.org/10.1177/17562864211057661 Journal: Ther Adv Neurol Disord E-pub date: 1 Aug 2021 Authors: K Allen-Philbey, S De Trane, Z Mao, C Álvarez-González, J Mathews, A MacDougall, A Stennett, X Zhou, O Yildiz, A Adams, L Bianchi, C Blain, C Chapman, K Chung, CS Constantinescu, C Dalton, RA Farrell, L Fisniku, H Ford, B Gran, J Hobart, Z Khaleeli, M Mattoscio, S Pavitt, O Pearson, L Peruzzotti-Jametti, A Scalfari, B Sharrack, E Silber, EC Tallantyre, S Webb, BP Turner, M Marta, S Gnanapavan, G Juliusson, G Giovannoni, D Baker, K Schmierer

Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients.

Abstract:

OBJECTIVE: To report on safety and effectiveness of subcutaneous cladribine (Litak®) in multiple sclerosis (MS) patients. METHODS: Litak® was offered to MS-patients irrespective of disease course. Litak® 10 mg was administered for 3-4 days during week 1. Based on lymphocyte count at week 4, patients received another 0-3 doses at week 5. A second course was administered 11 months later. Follow-up included adverse events, relapses, expanded disability status scale (EDSS), 9-hole-peg and Timed-25-foot-walking tests, no-evidence-of-disease-activity (NEDA), no-evidence-of-progression-or-active-disease (NEPAD), MRI, cerebrospinal fluid (CSF) neurofilament light chain (NfL), and lymphocyte counts. RESULTS: In all, 208 patients received at least one course of treatment. Age at baseline was 44 (17-72) years and EDSS 0-8.5. Cladribine was generally well tolerated. One myocardial infarction, one breast cancer, and three severe skin reactions occurred without long-term sequelae. Two patients died (one pneumonia, one encephalitis). Lymphopenia grade 3 occurred in 5% and grade 4 in 0.5%. In 94 out of 116 pwMS with baseline and follow-up (BaFU) data after two treatment courses, EDSS remained stable or improved. At 18 months, 64% of patients with relapsing MS and BaFU data (n = 39) had NEDA. At 19 months, 62% of patients with progressive MS and BaFU data (n = 13) had NEPAD. Of n = 13 patients whose CSF-NfL at baseline was elevated, 77% were normalised within 12 months. CONCLUSIONS: Litak® was well tolerated. Effectiveness in relapsing MS appeared similar to cladribine tablets and was encouraging in progressive MS. Our data suggest cladribine may be safe and effective in MS-patients irrespective of their disease stage.