Contributions
Abstract: P1254
Type: Poster Sessions
Abstract Category: Therapy - Risk management for disease modifying treatments
Introduction: Delayed-release dimethyl fumarate (DMF) demonstrated strong efficacy and a favourable benefit-risk profile in clinical studies of patients with relapsing-remitting multiple sclerosis (RRMS). ESTEEM is an ongoing, 5-year multinational prospective, non-interventional study (NCT02047097).
Objectives: To report interim safety and efficacy results in patients with RRMS treated with DMF under routine care in ESTEEM.
Methods: Patients newly prescribed DMF under routine clinical care were recruited from ~380 sites. The primary objective was to determine the incidence, type, and pattern of serious adverse events (SAEs) and AEs leading to treatment discontinuation. Secondary objectives included measures of RRMS disease activity (annualized release rate, ARR) and the following patient-reported outcomes (PROs): the Multiple Sclerosis Impact Score, 5-dimension QOL, visual analog scale of overall health, Modified Fatigue Impact Scale-5, and Work Productivity and Activity Impairment-MS.
Results: As of 6 April, 2017, 3075 patients had ≥1 dose of DMF and qualified for the analysis. Mean (SD) age was 40.8 (±11.4) years; 74.5% were female. One hundred and fifteen patients (3.7%) experienced SAEs; infections (n=26, 0.8%) and gastrointestinal disorders (n=14, 0.5%) were the most common. Of the 830 (27%) patients who discontinued treatment, 103 (12.4%) discontinued due to lack of efficacy and 440 (53.0%) discontinued primarily due adverse events. In total, 502 (16.3%) patients discontinued treatment due to treatment-emergent AEs; of these, 218 patients (7.1%) discontinued due to gastrointestinal AEs and 55 (1.8%) discontinued due to decreases in lymphocyte count/ occurrence of lymphopenia. Of 2664 patients with ≥1 lymphocyte count recorded, 28 (1.1%) had severe, prolonged lymphopenia (< 0.5x109/L for ≥6 months). Unadjusted ARR at year 2 (0.148, 95% CI 0.136, 0.161) was significantly lower than in the year prior to baseline (0.804, 95% CI 0.775, 0.834) representing 81.6% (P< 0.0001) reduction. Patients generally remained stable on PROs from baseline to 2 years.
Conclusions: Interim safety and efficacy results from the ESTEEM study suggest that the overall benefit-risk of DMF in RRMS patients remains favourable in routine clinical practice.
Supported by: Biogen.
Disclosure: Nicholas Everage: Employee of and holds stock/stock options in Biogen.
Cynthia C. Jones: Employee of and holds stock/stock options in Biogen.
Rajiv Das: Contract employee of Biogen.
Jerome Hanna: Employee of and holds stock/stock options in Biogen.
Shifang Liu: Employee of and holds stock/stock options in Biogen.
Konstantin Balashov: Speaker fees for Teva, consulting fees from Genzyme, Genentech, and Celgene. Grant/research support from Biogen.
Richard Macdonell: Honoraria from Biogen, Roche, Merck, Genzyme, and Novartis.
Jörg Windsheimer: Honoraria from Teva, Roche, Merck, Genzyme, and Novartis
Kathryn Giles: Consulting fees from EMD Serono, honoraria from Genzyme, and speaker fees and honoraria from Biogen.
Abstract: P1254
Type: Poster Sessions
Abstract Category: Therapy - Risk management for disease modifying treatments
Introduction: Delayed-release dimethyl fumarate (DMF) demonstrated strong efficacy and a favourable benefit-risk profile in clinical studies of patients with relapsing-remitting multiple sclerosis (RRMS). ESTEEM is an ongoing, 5-year multinational prospective, non-interventional study (NCT02047097).
Objectives: To report interim safety and efficacy results in patients with RRMS treated with DMF under routine care in ESTEEM.
Methods: Patients newly prescribed DMF under routine clinical care were recruited from ~380 sites. The primary objective was to determine the incidence, type, and pattern of serious adverse events (SAEs) and AEs leading to treatment discontinuation. Secondary objectives included measures of RRMS disease activity (annualized release rate, ARR) and the following patient-reported outcomes (PROs): the Multiple Sclerosis Impact Score, 5-dimension QOL, visual analog scale of overall health, Modified Fatigue Impact Scale-5, and Work Productivity and Activity Impairment-MS.
Results: As of 6 April, 2017, 3075 patients had ≥1 dose of DMF and qualified for the analysis. Mean (SD) age was 40.8 (±11.4) years; 74.5% were female. One hundred and fifteen patients (3.7%) experienced SAEs; infections (n=26, 0.8%) and gastrointestinal disorders (n=14, 0.5%) were the most common. Of the 830 (27%) patients who discontinued treatment, 103 (12.4%) discontinued due to lack of efficacy and 440 (53.0%) discontinued primarily due adverse events. In total, 502 (16.3%) patients discontinued treatment due to treatment-emergent AEs; of these, 218 patients (7.1%) discontinued due to gastrointestinal AEs and 55 (1.8%) discontinued due to decreases in lymphocyte count/ occurrence of lymphopenia. Of 2664 patients with ≥1 lymphocyte count recorded, 28 (1.1%) had severe, prolonged lymphopenia (< 0.5x109/L for ≥6 months). Unadjusted ARR at year 2 (0.148, 95% CI 0.136, 0.161) was significantly lower than in the year prior to baseline (0.804, 95% CI 0.775, 0.834) representing 81.6% (P< 0.0001) reduction. Patients generally remained stable on PROs from baseline to 2 years.
Conclusions: Interim safety and efficacy results from the ESTEEM study suggest that the overall benefit-risk of DMF in RRMS patients remains favourable in routine clinical practice.
Supported by: Biogen.
Disclosure: Nicholas Everage: Employee of and holds stock/stock options in Biogen.
Cynthia C. Jones: Employee of and holds stock/stock options in Biogen.
Rajiv Das: Contract employee of Biogen.
Jerome Hanna: Employee of and holds stock/stock options in Biogen.
Shifang Liu: Employee of and holds stock/stock options in Biogen.
Konstantin Balashov: Speaker fees for Teva, consulting fees from Genzyme, Genentech, and Celgene. Grant/research support from Biogen.
Richard Macdonell: Honoraria from Biogen, Roche, Merck, Genzyme, and Novartis.
Jörg Windsheimer: Honoraria from Teva, Roche, Merck, Genzyme, and Novartis
Kathryn Giles: Consulting fees from EMD Serono, honoraria from Genzyme, and speaker fees and honoraria from Biogen.