Key Results from PREFERMS: Real-World Patient Retention and Outcomes on Fingolimod Versus Platform Injectable Disease-Modifying Therapies in Early Relapsing–Remitting MS

Thursday, June 2, 2016
Exhibit Hall
Mark Cascione, MD , Tampa Neurology Associates, South Tampa Multiple Sclerosis Center, South Tampa, FL
Bruce AC Cree, MD, PhD , University of California San Francisco, San Francisco, CA
Daniel Wynn, MD , Consultants in Neurology MS Center, Northbrook, IL
Xiangyi Meng, PhD , Novartis Pharmaceuticals Corporation, East Hanover, NJ
Lesley Schofield, PhD , Novartis Pharmaceuticals Corporation, East Hanover, NJ
Nadia Tenenbaum, MD , Novartis Pharmaceuticals Corporation, East Hanover, NJ
Jeremy Bright, PhD , Oxford Pharmagenesis, Tubney, United Kingdom
Terence Smith, PhD , Oxford Pharmagenesis, Tubney, United Kingdom

Background: Suboptimal adherence to injectable disease-modifying therapy (iDMT) classes is well established. PREFERMS is the first large study of treatment retention with disease-modifying therapies and other outcomes in patients with relapsing–remitting multiple sclerosis (RRMS).

Objectives: To examine therapeutic retention on fingolimod 0.5mg versus iDMTs in PREFERMS, a randomized, prospective real-world study of patients with early RRMS.

Methods: PREFERMS was a 12-month, phase 4, open-label, active-controlled, randomized, multicenter study. At enrollment, patients were treatment-naïve, or had received only one iDMT class (interferon beta or glatiramer acetate). Investigators selected an iDMT for each patient, before subjects were randomized (1:1) to fingolimod 0.5mg or the selected iDMT. One treatment switch was allowed on study, after a minimum 3 months of treatment, unless required by an adverse event. The primary endpoint was percent retained on randomized treatment. Secondary endpoints included clinical, radiographical, patient-reported outcomes, and safety assessments. Sample-size and power calculations were based on retention proportions.

Results: 875 patients were randomized (fingolimod, n=436; iDMT, n=439). At baseline, mean time since diagnosis was 4.3 years and Expanded Disability Status Scale score was 2.4. In the full analysis set (n=861), 352 (81.3%) patients on fingolimod and 125 (29.2%) on iDMT completed randomized treatment (p<0.001). Despite the shorter duration of exposure to iDMTs, the annualized relapse rate was numerically lower with fingolimod (ratio, 0.70; p=0.084). There was less brain volume loss (change from baseline at month 6, 0.19% vs. 0.31%, p=0.011; month 12, 0.40% vs. 0.56%, p=0.076), less cortical grey matter loss (change from baseline at last assessment 0.09% vs. 0.29%; p=0.002), and treatment satisfaction was greater (Medication Satisfaction Questionnaire: p<0.001, all assessments). Safety outcomes aligned with respective labels.

Conclusions: Higher therapeutic retention, improved clinical and radiographical outcomes, greater treatment satisfaction and good tolerability support front-line use of fingolimod over platform iDMTs in patients with early RRMS.