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.

• Klíčová slova
• Cladribine, adherence, annualised relapse rate, expanded disability status scale, high-efficacy disease-modifying therapy, multiple sclerosis,
• Publikační typ
• časopisecké články MeSH

BACKGROUND: During long-term multiple sclerosis therapy, patient satisfaction with received treatment has considerable impact on treatment outcomes. Here we report the results of a non-interventional real-world study that mapped the treatment patterns of disease-modifying therapy (DMT) and assessed treatment satisfaction with DMT. PATIENTS AND METHODS: The SKARLET study was a non-interventional, cross-sectional study in Slovakia running from May 2016 to March 2017. Patients with relapsing-remitting multiple sclerosis on DMT for ≥3 months and ≤2 years (per local labelling) from 10 multiple sclerosis centers across Slovakia were included. The primary objective was to collect the Treatment Satisfaction Questionnaire for Medication version 9 (TSQM 9) score regarding perceived effectiveness, convenience and overall satisfaction with DMT. RESULTS: The following TSQM 9 scores (mean; 95% confidence interval) were reported from 415 patients: convenience (75.05; 73.49-76.61), effectiveness (68.15; 66.56-69.75) and global satisfaction scale (66.94; 65.26-68.62). All three parameters of the TSQM 9 were analyzed by the route of DMT administration, with infusions best rated for effectiveness and global satisfaction in comparison to oral dosage and injections. For convenience, however, oral dosage forms were appraised highly (82.66; 80.59-84.73) followed by infusions (74.40; 70.12-78.69), while injections were rated as the worst (66.92; 64.81-69.04). The difference of TSQM 9 scores according to the route of DMT administration is statistically significant for convenience (p < 0.001) and global satisfaction (p = 0.004), but not for effectiveness (p = 0.185). CONCLUSION: In the present study, it was confirmed that patients find oral DMTs as most convenient; however, the infusion form of treatment outweighs oral DMTs in global satisfaction and effectiveness. The differences of TSQM 9 scores among DMT dosage forms were significant for convenience and global satisfaction. In conclusion, the results of this detailed survey increase our understanding of RRMS patient population characteristics and patient satisfaction with DMT treatment.

• Klíčová slova
• disease-modifying therapy, multiple sclerosis, real-world, treatment pattern, treatment satisfaction,
• Publikační typ
• časopisecké články MeSH