Determining Effectiveness of “Off-Label Therapies” for Multiple Sclerosis in a Real-World Setting

Abstract

Objective: To determine the factors, if any, that are associated with the efficacy of “off-label therapies” (OLTs) for multiple sclerosis (MS). Methods: Consecutive patients (N = 174) with relapsing–remitting MS (RRMS) or secondary progressive MS (SPMS) with relapses, on OLTs with a generic formulation of azathioprine, mycophenolate mofetil, or rituximab biosimilar for ≥2 years were included. Annualized relapse rate (ARR) and expanded disability status score (EDSS) 1 year before and ≥2 years after starting OLTs were recorded. Optical coherence tomography (OCT) was done at baseline and at the end of the study. Results: During a median period of 4.1 years (2.4–24), ARR reduced in all (P < 0.0001) and EDSS improved in RRMS (P < 0.0001) patients but not in SPMS (P < 0.31) patients. Good responders were those who had RRMS (P = 0.001, odds ratio [OR] 0.04, 95% confidence interval [CI] 0.01–0.15), female gender (P 0.008, OR 6.67, 95% CI 1.7–26.8), and had early access to OLT (P = 0.006, OR 1.2, 95% CI 1.05–1.40). Baseline peripapillary retinal nerve fiber layer thickness identified the risk of conversion to SPMS (P < 0.01, OR 1.03; 95% CI 1.01–1.06). Conclusions: This limited prospective study suggests that early identification of patients who could potentially respond to unconventional but accessible therapies may be valuable in the treatment of MS, particularly in resource-poor regions.

Keywords

• azathioprine
• multiple sclerosis
• mycophenolate mofetil
• off-label therapy
• rituximab