Effectiveness of first-line treatment for relapsing-remitting multiple sclerosis in Brazil: A 16-year non-concurrent cohort study

Abstract

Background Relapsing-remitting multiple sclerosis (RRMM) is a chronic, progressive, inflammatory and immune-mediated disease that affects the central nervous system and is characterized by episodes of neurological dysfunction followed by a period of remission. The pharmacological strategy aims to delay the progression of the disease and prevent relapse. Interferon beta and glatiramer are commonly used in the Brazilian public health system and are available to patients who meet the guideline criteria. The scenario of multiple treatments available and in development brings the need for discussion and evaluation of the technologies already available before the incorporation of new drugs. This study analyses the effectiveness of first-line treatment of RRMS measured by real-world evidence data, from the Brazilian National Health System (SUS). Methods and findings We conducted a non-concurrent national cohort between 2000 and 2015. The study population consisted of 22,722 patients with RRMS using one of the following first-line drugs of interest: glatiramer or one of three presentations of interferon beta. Kaplan-Meier analysis was used to estimate the time to treatment failure. A univariate and multivariate Cox proportional hazard model was used to evaluate factors associated with treatment failure. In addition, patients were propensity score-matched (1:1) in six groups of comparative first-line treatments to evaluate the effectiveness among them. The analysis indicated a higher risk of treatment failure in female patients (HR = 1.

Serum levels of IgM to phosphatidylcholine predict the response of multiple sclerosis patients to natalizumab or IFN-β

“…Treatments delay the disease progression, but have significant side effects 9 . In addition, the response to treatment is very variable, and 40% of the cases do not respond to treatment and a change to another therapy is required 6 . Thus, numerous studies aimed to characterize biomarkers of response to treatment.…”

“…Treatments delay MS progression, but do not cure it. In addition, 40% patients do not respond to first-line treatments 6 , 7 , and most available treatments show limited efficacy in the progressive phase of disease 8 . Moreover, side effects, such as cardiomyopathy, leukemia, progressive multifocal leukoencephalopathy, bradyarrhythmia, macular edema, herpes zoster virus infections, autoimmune thyroiditis, thrombocytopenia and glomerulonephritis are associated to immunosuppressive therapies 9 .…”

Autologous Hematopoietic Stem Cell Transplantation for the Treatment of Multiple Sclerosis

“…19,21,23 Approximately 25% of people on first-line DMTs experience initial treatment failure, in which case they are most often transitioned to another first-line DMT. 14 There is limited evidence, beyond consensus statements, describing when a person receiving first-line DMTs should be transitioned to second-line treatments such as alemtuzumab, fingolimod, and natalizumab. 37 In practice, people are typically transitioned when they experience 2 or more relapses over a period of 12 months, or have fewer relapses but show evidence of new brain lesions.…”

“…13 Moreover, first-and second-line DMTs may fail to improve outcomes for some people or may have limited effects on disease control, while still causing people to experience adverse effects. [14][15][16][17] Autologous hematopoietic stem cell transplantation (AHSCT), also referred to autologous hematopoietic cell transplantation, is a well-established immune reconstitution therapy for many blood cancers and is an emerging therapy for treating people with MS and other autoimmune disorders. 18,19 The procedure has the potential to provide a therapeutic option for people with aggressive or highly active RRMS for whom drug-based DMTs have had limited effect in disease control.…”

Autologous Hematopoietic Stem Cell Transplantation for the Treatment of Multiple Sclerosis

“…17,18 Moreover, first-and second-line DMTs may fail to improve outcomes for some people, or may have limited effects on disease control, although people will still experience the adverse effects associated with DMTs. [19][20][21][22] Autologous hematopoietic stem cell transplantation (AHSCT) is a well-established therapy for many blood cancers. It is also an emerging therapy for treating people with MS and other autoimmune disorders.…”