Assessment of cladribine therapy over time: effectiveness, safety and evaluation of treatment sequencing beyond year four
SYNOPSIS AND RESULTS
In the constantly evolving scenario of disease-modifying therapies (DMTs) in multiple sclerosis (MS), the advent of immune reconstitution therapy (IRT), administered in short-dosing periods to produce long-term immunological effects, offered the neurologist an additional tool against the irreversible disability accrual. (1-4) Cladribine is an oral IRT approved in Italy for the treatment of relapsing-remitting MS (RRMS) and prescribed in two treatment courses. (5) Growing evidence suggests that cladribine has a strong anti-inflammatory action, with a suppression of clinical and radiological disease activity for the first four years, but the approach to managing patients beyond the fifth year of treatment remains uncertain. (6-8) Real-world data from the Italian MS and Related Disorders Register (RISM) proved to be able to address unanswered research questions and face and resolve the multi-faceted criticisms emerging from daily clinical practice in MS. (9, 10) Therefore, the study aims to evaluate treatment strategies for DMT continuation after two years of cladribine. The purpose of the study is also to analyze the characteristics and predictors of presence of MS inflammatory activity between the 1st and 2nd cycle of treatment, evaluating also factors associated with the no evidence of disease activity (NEDA) loss over the years after cladribine stop. Finally, to investigate the immunological changes induced by cladribine and safety-related challenges (11), we will evaluate the lymphocyte repertoire of RRMS patients during and after treatment, assessing lymphopenia events. We will conduct a descriptive analysis of the therapeutic patterns in the years after the end of the therapy with cladribine, going to evaluate which therapies are chosen, when they were started and the presence of further possible therapeutic switches, characterizing patient groups in relation to the therapeutic choices made in the network of RISM centres. We will use predictive and prognostic statistical models to assess treatment success and the factors associated with treatment.
Fondazione Italiana Sclerosi Multipla – FISM – Ente del Terzo Settore/ETS e, in forma abbreviata, FISM ETS. Iscrizione al RUNTS Rep. N° 89695 - Fondazione con Riconoscimento di Personalità Giuridica - C.F. 95051730109
Assessment of cladribine therapy over time: effectiveness, safety and evaluation of treatment sequencing beyond year four
In the constantly evolving scenario of disease-modifying therapies (DMTs) in multiple sclerosis (MS), the advent of immune reconstitution therapy (IRT), administered in short-dosing periods to produce long-term immunological effects, offered the neurologist an additional tool against the irreversible disability accrual. (1-4) Cladribine is an oral IRT approved in Italy for the treatment of relapsing-remitting MS (RRMS) and prescribed in two treatment courses. (5) Growing evidence suggests that cladribine has a strong anti-inflammatory action, with a suppression of clinical and radiological disease activity for the first four years, but the approach to managing patients beyond the fifth year of treatment remains uncertain. (6-8) Real-world data from the Italian MS and Related Disorders Register (RISM) proved to be able to address unanswered research questions and face and resolve the multi-faceted criticisms emerging from daily clinical practice in MS. (9, 10) Therefore, the study aims to evaluate treatment strategies for DMT continuation after two years of cladribine. The purpose of the study is also to analyze the characteristics and predictors of presence of MS inflammatory activity between the 1st and 2nd cycle of treatment, evaluating also factors associated with the no evidence of disease activity (NEDA) loss over the years after cladribine stop. Finally, to investigate the immunological changes induced by cladribine and safety-related challenges (11), we will evaluate the lymphocyte repertoire of RRMS patients during and after treatment, assessing lymphopenia events. We will conduct a descriptive analysis of the therapeutic patterns in the years after the end of the therapy with cladribine, going to evaluate which therapies are chosen, when they were started and the presence of further possible therapeutic switches, characterizing patient groups in relation to the therapeutic choices made in the network of RISM centres. We will use predictive and prognostic statistical models to assess treatment success and the factors associated with treatment.
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