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

Author:

Pandit Lekha1,Mustafa Sharik2,Sudhir Akshatha1,Malapur Puneeth3,D’Cunha Anitha1

Affiliation:

1. Center for Advanced Neurological Research, Nitte University, Mangalore, Karnataka, India

2. Department of Neurology, Prime Healthcare Group, Dubai, United Arab Emirates

3. Department of Neurology, Badr Al Samaa Hospital, Muscat, Oman

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.

Publisher

Medknow

Reference16 articles.

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