PMID- 37192586 OWN - NLM STAT- MEDLINE DCOM- 20230620 LR - 20230620 IS - 2211-0356 (Electronic) IS - 2211-0348 (Linking) VI - 75 DP - 2023 Jul TI - Safety and effectiveness of cladribine tablets for multiple sclerosis: Results from a single-center real-world cohort. PG - 104735 LID - S2211-0348(23)00239-0 [pii] LID - 10.1016/j.msard.2023.104735 [doi] AB - BACKGROUND: Cladribine tablets are a highly effective immune reconstitution therapy licensed for treating relapsing multiple sclerosis (RMS) in Europe since 2017. Currently, there is a high demand for real-world data from different clinical settings on the effectiveness and safety profile of cladribine in MS. METHODS: Within this report, we retrospectively evaluated the outcomes of RMS patients who received cladribine between August 2018 and November 2021 at our Belgian institute. Patients with data for three effectiveness endpoints, more specifically, relapses, MRI observations, and confirmed disability worsening were incorporated into the analysis of 'no evidence of disease activity' (NEDA-3) re-baselined at 3 months. Safety endpoints included lymphopenia, liver transaminases, and adverse events (AEs) during follow-up. Descriptive statistics and time-to-event analysis were performed, including subgroup analysis by pre-treatment. RESULTS: Of the 84 RMS patients included in this study (age 42 [33-50], 64.3% female, diagnosis duration 6 [2-11] years, baseline EDSS 2.5 [1.5-3.6]), 14 (16.7%) patients experienced relapses, while disability progression and brain MRI activity occurred in 8.5% (6/71) and 6.3% (5/79). This resulted in 72.6% (n = 69, standard error 6%) retaining NEDA-3 status at the mean follow-up time of 22.6 +/- 11.5 months. During the first year after cladribine initiation, disease activity prevailed more in patients with >/=2 prior DMTs and those switching from fingolimod, although both trends were not statistically significant. In terms of safety, 67.9% reported at least one AE during follow-up, the most frequent being fatigue (64.9%) and skin-related problems (38.6%). CONCLUSION: Overall, our research results confirm cladribine's safety and effectiveness among RMS patients in real-world conditions. After the re-baseline, we observed high rates of NEDA-3-retention, and no new safety signals were noted. CI - Copyright (c) 2023 Elsevier B.V. All rights reserved. FAU - Aerts, Sofie AU - Aerts S AD - Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Biomedical Research Institute (BIOMED), Agoralaan, Diepenbeek 3590, Belgium; Noorderhart, Revalidatie en MS, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Rehabilitation Research Center, Agoralaan, Diepenbeek 3590, Belgium. Electronic address: sofie.aerts@uhasselt.be. FAU - Khan, Hamza AU - Khan H AD - Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Biomedical Research Institute (BIOMED), Agoralaan, Diepenbeek 3590, Belgium; UHasselt, Data Science Institute, Agoralaan, Diepenbeek 3590, Belgium; The D-Lab, Department of Precision Medicine, GROW - School for Oncology, Maastricht University, Universiteitssingel 40, Maastricht 6229 ER, the Netherlands. FAU - Severijns, Deborah AU - Severijns D AD - Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; Noorderhart, Revalidatie en MS, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Rehabilitation Research Center, Agoralaan, Diepenbeek 3590, Belgium. FAU - Popescu, Veronica AU - Popescu V AD - Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Biomedical Research Institute (BIOMED), Agoralaan, Diepenbeek 3590, Belgium; Noorderhart, Revalidatie en MS, Boemerangstraat 2, Pelt 3900, Belgium. FAU - Peeters, Liesbet M AU - Peeters LM AD - Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Biomedical Research Institute (BIOMED), Agoralaan, Diepenbeek 3590, Belgium; UHasselt, Data Science Institute, Agoralaan, Diepenbeek 3590, Belgium. FAU - Van Wijmeersch, Bart AU - Van Wijmeersch B AD - Universitair MS Centrum (UMSC) Hasselt-Pelt, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Biomedical Research Institute (BIOMED), Agoralaan, Diepenbeek 3590, Belgium; Noorderhart, Revalidatie en MS, Boemerangstraat 2, Pelt 3900, Belgium; UHasselt, Rehabilitation Research Center, Agoralaan, Diepenbeek 3590, Belgium. LA - eng PT - Journal Article DEP - 20230425 PL - Netherlands TA - Mult Scler Relat Disord JT - Multiple sclerosis and related disorders JID - 101580247 RN - 47M74X9YT5 (Cladribine) RN - 0 (Immunosuppressive Agents) RN - 0 (Tablets) SB - IM MH - Adult MH - Female MH - Humans MH - Male MH - Cladribine/adverse effects MH - Immunosuppressive Agents/adverse effects MH - *Multiple Sclerosis/drug therapy MH - *Multiple Sclerosis, Relapsing-Remitting/diagnostic imaging/drug therapy/chemically induced MH - Recurrence MH - Retrospective Studies MH - Tablets MH - Middle Aged OTO - NOTNLM OT - Cladribine tablets OT - Multiple sclerosis OT - NEDA-3 OT - Real-world OT - Safety COIS- Declaration of Competing Interest Bart Van Wijmeersch has received honoraria, travel, and research grants from Almirall, BMS, Biogen, Imcyse, Janssen, Merck Serono, Novartis, Roche, Sanofi Genzyme, and Teva Pharmaceuticals. Veronica Popescu has received honoraria, travel, and research grants from Almirall, Biogen, Medtronic, Merck, Novartis, Roche, Sanofi Genzyme, and Teva Pharmaceuticals. The remaining authors have no disclosures related to this study. EDAT- 2023/05/16 19:10 MHDA- 2023/06/19 13:08 CRDT- 2023/05/16 18:02 PHST- 2023/03/03 00:00 [received] PHST- 2023/04/24 00:00 [accepted] PHST- 2023/06/19 13:08 [medline] PHST- 2023/05/16 19:10 [pubmed] PHST- 2023/05/16 18:02 [entrez] AID - S2211-0348(23)00239-0 [pii] AID - 10.1016/j.msard.2023.104735 [doi] PST - ppublish SO - Mult Scler Relat Disord. 2023 Jul;75:104735. doi: 10.1016/j.msard.2023.104735. Epub 2023 Apr 25.