BACKGROUND: Cladribine, a selective immune reconstitution therapy, is approved for the treatment of adult patients with highly active multiple sclerosis (MS). OBJECTIVES: Provide experience with cladribine therapy in a real-world setting. METHODS: This is a registry-based retrospective observational cohort study. First, using data from the Czech nationwide registry ReMuS, we analysed patients who initiated cladribine from September 1, 2018 to December 31, 2021. Second, we analysed a subgroup of patients who initiated cladribine between September 1, 2018 to June 30, 2020, thus possessing a follow-up period of at least 2 years. We evaluated demographic and MS characteristics including disease-modifying therapies (DMTs) before and after cladribine administration, relapses, Expanded Disability Status Scale (EDSS), and adherence. RESULTS: In total, 617 patients (335 with follow-up of at least 2 years) started cladribine therapy in the study period (mean age 37.0, mean disease duration 8.4 years, 74.1% females). In most cases, cladribine was administered as a second-line drug, a total of 80.7% had been escalated from a platform DMT. During 2 years before cladribine initiation, the average annualised relapse rate (ARR) was .67. Following cladribine initiation, the ARR decreased to .28 in the first year and .22 in the second year. Overall, across the entire two-year treatment period, 69.0% of patients were relapse-free and the average ARR was .25. As for EDSS development, the median baseline EDSS was 2.5 and remained stable even after 24 months. The adherence to treatment ranged of around 90%. CONCLUSION: This nationwide study confirms the efficacy of cladribine in real-world settings, especially in patients who are not treatment-naïve. In addition, the study shows an exceptionally high adherence rate, a finding that underscores the invaluable role of cladribine, but also the value of registry-based studies in capturing real-world clinical practice.

1st Department of Neurology University Hospital U Svate Anny and Masaryk University Brno Brno Czech Republic

Department of Economic Statistics Prague University of Economics and Business Prague Czech Republic

Department of Neurology 2nd Faculty of Medicine and Motol University Hospital Charles University Prague Czech Republic

Department of Neurology 3rd Faculty of Medicine Charles University Prague and Hospital Kralovske Vinohrady Prague Czech Republic

Department of Neurology and Centre of Clinical Neuroscience 1st Faculty of Medicine Charles University Prague and General University Hospital Prague Czech Republic

Department of Neurology Faculty of Medicine and University Hospital Hradec Kralove Charles University Prague Czech Republic

Department of Neurology Faculty of Medicine and University Hospital in Pilsen Charles University Plzen Czech Republic

Department of Neurology Faculty of Medicine Palacky University and University Hospital Olomouc Olomouc Czech Republic

Department of Neurology Hospital Ceske Budejovice Ceske Budejovice Czech Republic

Department of Neurology Hospital of Jihlava Jihlava Czech Republic

Department of Neurology Hospital Pardubice Pardubice Czech Republic

Department of Neurology KZ a s Hospital Teplice Teplice Czech Republic

Department of Neurology Thomayer Hospital Prague Czech Republic

Department of Neurology Tomas Bata Hospital Zlin Czech Republic

Department of Neurology University Hospital and Masaryk University Brno Brno Czech Republic

Department of Neurology University Hospital Ostrava and Medical Faculty Ostrava University Ostrava Czech Republic

Endowment Fund IMPULS Prague Czech Republic

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