Background Multiple sclerosis (MS) is a chronic disabling disease that is associated with negative effects on health-related quality of life (HRQoL) due to reduced physical and psychosocial functioning. Cladribine tablets 10 mg (3.5 mg/kg cumulative dose over 2 years) have been approved for the treatment of adult patients with highly active relapsing multiple sclerosis (RMS). The ongoing CLARIFY-MS study (NCT03369665; EudraCT number: 2017-002632-17) aims to assess the effect of cladribine tablets 3.5 mg/kg on HRQoL of patients with highly active RMS. Objective To report on the design of the CLARIFY-MS study, baseline patient characteristics, and results of a pre-planned interim analysis focusing on treatment satisfaction, safety, and tolerability that includes all data reported till 6 months after start of treatment. Methods The CLARIFY-MS study is a 2-year, open-label, single-arm, prospective, multicenter, phase IV study. Eligible patients with highly active RMS were assigned to receive cladribine tablets 3.5 mg/kg over 2 years. Treatment satisfaction was assessed using the Treatment Satisfaction Questionnaire for Medication (TSQM, v1.4; scale range from 0 to 100, higher values indicating higher satisfaction). Safety assessments, including occurrence of treatment-emergent adverse events (TEAEs; any adverse event reported after drug administration), serious adverse events (SAEs), and lymphocyte counts, were summarized descriptively. Results A total of 482 patients from 85 sites in Europe were treated with cladribine tablets. Mean patient age was 37.4 years, 338 (70.1%) were women, median EDSS was 2.5, and 345 (71.6%) were prior users of disease-modifying therapy (DMT). During the first 6 months after the start of treatment, and before reaching the full dose of cladribine tablets, mean TSQM global satisfaction score for the overall population was 70.4 (standard deviation, ± 18.48). The side effects score was 91.9 (± 17.68), convenience scored 86.6 (± 13.57), and effectiveness was 65.8 (± 21.14). A total of 275 patients (57.1%) reported at least one TEAE and 9 patients (1.9%) had a SAE. The majority of observed lymphopenia cases were of grade 1 or 2; 33 (6.8%) of the total study cohort had grade 3 lymphopenia, and no grade 4 lymphopenia was reported. Conclusion Patients reported high treatment satisfaction (TSQM) with cladribine tablets in this pre-planned interim analysis at 6 months. Few serious, and no unexpected, adverse events were reported, and there were no instances of grade 4 lymphopenia over the first 6 months. These preliminary data indicate good tolerability and convenience of administration of cladribine tablets in patients with highly active RMS.

Center for Neurology Lodz Poland

Charles University 1st Medical Faculty Prague Czech Republic

Charles University and University Hospital Hradec Králové Czech Republic

Department of Clinical Neuroscience Karolinska Institutet Stockholm Sweden

Department of Medical and Surgical Sciences and Advanced Technologies GF Ingrassia University of Catania and Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco University of Catania Catania Italy

Department of Neurology Neuroimmunology Centre of Multiple Sclerosis of Catalonia University Hospital Vall d'Hebron Barcelona Spain

Department of Psychology Royal Holloway University of London Egham United Kingdom

INSERM U 1215 University of Bordeaux Bordeaux France

Medical University of Lublin Lublin Poland

Merck Healthcare KGaA Darmstadt Germany

Unit of Neuroepidemiology Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy

University of Newcastle Newcastle NSW Australia; Division of Neurology John Hunter Hospital Newcastle NSW Australia

Zuyderland Medisch Centrum Sittard Maastricht University Medical Center Maastricht the Netherlands

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Improvements in quality of life over 2 years with cladribine tablets in people with relapsing multiple sclerosis: The CLARIFY-MS study