Effectiveness of delayed-release dimethyl fumarate on patient-reported outcomes and clinical measures in patients with relapsing-remitting multiple sclerosis in a real-world clinical setting: PROTEC

T Berger, B Brochet, L Brambilla, P S Giacomini, X Montalbán, A Vasco Salgado, R Su, A Bretagne, T Berger, B Brochet, L Brambilla, P S Giacomini, X Montalbán, A Vasco Salgado, R Su, A Bretagne

Abstract

Background: Patient-reported outcomes (PRO) and clinical outcomes give a broad assessment of relapsing-remitting multiple sclerosis (RRMS) disease.

Objective: The aim is to evaluate the effectiveness of delayed-release dimethyl fumarate (DMF) on disease activity and PROs in patients with RRMS in the clinic.

Methods: PROTEC, a phase 4, open-label, 12-month observational study, assessed annualized relapse rate (ARR), proportion of patients relapsed, and changes in PROs. Newly diagnosed and early MS (≤3.5 EDSS and ≤1 relapse in the prior year) patient subgroups were evaluated.

Results: Unadjusted ARR at 12 months post-DMF versus 12 months before DMF initiation was 75% lower (0.161 vs. 0.643, p < 0.0001) overall (n = 1105) and 84%, 77%, and 71% lower in newly diagnosed, ≤3.5 EDSS, and ≤1 relapse subgroups, respectively. Overall, 88% of patients were relapse-free 12 months after DMF initiation (84%, newly diagnosed; 88%, ≤3.5 EDSS; 88%, ≤1 relapse). PRO measures for fatigue, treatment satisfaction, daily living, and work improved significantly over 12 months of DMF versus baseline.

Conclusion: At 12 months after versus 12 months before DMF initiation, ARR was significantly lower, the majority of patients were relapse-free, and multiple PRO measures showed improvement (overall and for subgroups), suggesting that DMF is effective based on clinical outcomes and from a patient perspective.Clinical trial: A Study Evaluating the Effectiveness of Tecfidera (Dimethyl Fumarate) on Multiple Sclerosis (MS) Disease Activity and Patient-Reported Outcomes (PROTEC), NCT01930708, https://ichgcp.net/clinical-trials-registry/NCT01930708.

Keywords: Dimethyl fumarate; activities of daily living; multiple sclerosis; patient-reported outcome measures; quality of life; relapsing–remitting; treatment outcomes.

© The Author(s) 2019.

Figures

Figure 1.
Figure 1.
Patient disposition for the PROTEC study is shown. AE: adverse events, DMF: delayed-release DMF.
Figure 2.
Figure 2.
ARR at 12 months before and after DMF initiation for (A) all patients and (B) early MS subgroups. aBased on empirical (robust) SE from a generalized estimating equation using an unadjusted Poisson regression model. bARR was calculated as the total number of relapses that occurred during that period of time for all patients, divided by the total number of patient-years followed in that period. Subgroups are defined as: EDSS = baseline EDSS score ≤3.5; Relapse = ≤1 relapse in the prior year. DMF: delayed-release DMF.

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