Contributions
Abstract: EP1485
Type: ePoster
Abstract Category: Therapy - disease modifying - Immunomodulation/Immunosuppression
Background: Delayed-release dimethyl fumarate (DMF) demonstrated strong efficacy and favourable benefit-risk profile in clinical studies of pts with relapsing-remitting multiple sclerosis (RRMS).
Objectives: To report results from the first interim analysis of ESTEEM (NCT02047097), an ongoing, multinational, 5-yr prospective, non-interventional study to evaluate the long-term safety and effectiveness of DMF in pts with RRMS in routine clinical practice.
Methods: Eligible pts treated with newly prescribed DMF under routine clinical care were recruited from ~380 sites globally. 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 the effectiveness of DMF on RRMS disease activity and pt-reported outcomes (PROs). PROs assessed were Multiple Sclerosis Impact Score (MSIS-29), quality of life data in 5 dimensions (EQ-5D-DL), pts" rating of their overall health using a visual analogue scale (EQ-VAS), the Modified Fatigue Impact Scale-5 (MFIS-5), and the Work Productivity and Activity Impairment-MS (WPAI-MS).
Results: As of 22 July 2015, 1000 pts had enrolled and accrued ≥6 mos follow-up time. Of the initial 1000 pts, 897 pts qualified for the interim analysis, with 781 (87%) remaining on study; 75.8% were female. Mean and median age at enrolment were 42 yrs. The majority of pts (73.4%) received ≥1 prior RRMS treatments (intramuscular interferon [IFN] β-1a, 35%; subcutaneous IFN β-1a, 31%; glatiramer acetate, 37%). SAEs were reported in 28 (3.1%) pts, of which infections (0.9%) and gastrointestinal (GI) disorders (0.6%) were the most common; no opportunistic infections were reported; 1 death (unrelated to DMF treatment) occurred. The most common AEs leading to treatment discontinuation were GI (8.9%) and vascular disorders (2.5%; ie, flushing/hot flushes). The annualised relapse rate (ARR) reported for the 12 mos prior to DMF initiation was 0.68 (95% confidence interval [CI]: 0.63, 0.73) and the ARR for 1 yr after DMF initiation was 0.18 (95% CI: 0.15, 0.21), representing a 74% lower ARR after DMF initiation
(P< 0.0001). Mean scores on MSIS-29, EQ-5D-DL, EQ-VAS, MFIS-5, and WPAI-MS improved or remained stable.
Conclusions: Results of this first interim analysis of ESTEEM suggest that the overall benefit-risk of DMF in RRMS pts remains favourable. DMF was associated with significantly lower ARR and PROs were stable or improved.
Disclosure:
Supported by: Biogen.
Nicholas J. Everage: employee of and holds stock/stock options in Biogen.
Claudia Prada: employee of and holds stock/stock options in Biogen.
Shifang Liu: employee of and holds stock/stock options in Biogen.
Konstantin Balashov: grant/research support from Biogen and Teva and speaker bureau member for Teva.
Richard Macdonell: grant/research support from Biogen.
Jörg Windsheimer: advisory boards for Teva, Roche, Merck, Genzyme, and Novartis.
Kathryn Giles: consulting fees from EMD Serono, advisory board for Genzyme, and speakers" bureau and advisory board for Biogen.
Abstract: EP1485
Type: ePoster
Abstract Category: Therapy - disease modifying - Immunomodulation/Immunosuppression
Background: Delayed-release dimethyl fumarate (DMF) demonstrated strong efficacy and favourable benefit-risk profile in clinical studies of pts with relapsing-remitting multiple sclerosis (RRMS).
Objectives: To report results from the first interim analysis of ESTEEM (NCT02047097), an ongoing, multinational, 5-yr prospective, non-interventional study to evaluate the long-term safety and effectiveness of DMF in pts with RRMS in routine clinical practice.
Methods: Eligible pts treated with newly prescribed DMF under routine clinical care were recruited from ~380 sites globally. 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 the effectiveness of DMF on RRMS disease activity and pt-reported outcomes (PROs). PROs assessed were Multiple Sclerosis Impact Score (MSIS-29), quality of life data in 5 dimensions (EQ-5D-DL), pts" rating of their overall health using a visual analogue scale (EQ-VAS), the Modified Fatigue Impact Scale-5 (MFIS-5), and the Work Productivity and Activity Impairment-MS (WPAI-MS).
Results: As of 22 July 2015, 1000 pts had enrolled and accrued ≥6 mos follow-up time. Of the initial 1000 pts, 897 pts qualified for the interim analysis, with 781 (87%) remaining on study; 75.8% were female. Mean and median age at enrolment were 42 yrs. The majority of pts (73.4%) received ≥1 prior RRMS treatments (intramuscular interferon [IFN] β-1a, 35%; subcutaneous IFN β-1a, 31%; glatiramer acetate, 37%). SAEs were reported in 28 (3.1%) pts, of which infections (0.9%) and gastrointestinal (GI) disorders (0.6%) were the most common; no opportunistic infections were reported; 1 death (unrelated to DMF treatment) occurred. The most common AEs leading to treatment discontinuation were GI (8.9%) and vascular disorders (2.5%; ie, flushing/hot flushes). The annualised relapse rate (ARR) reported for the 12 mos prior to DMF initiation was 0.68 (95% confidence interval [CI]: 0.63, 0.73) and the ARR for 1 yr after DMF initiation was 0.18 (95% CI: 0.15, 0.21), representing a 74% lower ARR after DMF initiation
(P< 0.0001). Mean scores on MSIS-29, EQ-5D-DL, EQ-VAS, MFIS-5, and WPAI-MS improved or remained stable.
Conclusions: Results of this first interim analysis of ESTEEM suggest that the overall benefit-risk of DMF in RRMS pts remains favourable. DMF was associated with significantly lower ARR and PROs were stable or improved.
Disclosure:
Supported by: Biogen.
Nicholas J. Everage: employee of and holds stock/stock options in Biogen.
Claudia Prada: employee of and holds stock/stock options in Biogen.
Shifang Liu: employee of and holds stock/stock options in Biogen.
Konstantin Balashov: grant/research support from Biogen and Teva and speaker bureau member for Teva.
Richard Macdonell: grant/research support from Biogen.
Jörg Windsheimer: advisory boards for Teva, Roche, Merck, Genzyme, and Novartis.
Kathryn Giles: consulting fees from EMD Serono, advisory board for Genzyme, and speakers" bureau and advisory board for Biogen.