Abstract Details

Title Safety and Effectiveness of Dimethyl Fumarate Maintained Over 4 Years in Multiple Sclerosis Patients Treated in Routine Medical Practice

Topic Multiple Sclerosis

Presentation(s) P6 - Poster Session 6 (12:00 PM-1:00 PM)

Poster/Presentation
Number 9-022

Objective Report 4-year safety and effectiveness in patients with multiple sclerosis (MS) treated with dimethyl fumarate (DMF) under routine care.

Background DMF demonstrated a favorable benefit–risk profile in clinical studies of MS. Real-world studies enable characterization of risks that may only emerge with long-term exposure in clinical practice. ESTEEM (NCT02047097) is an ongoing 5-year study characterizing long-term safety and effectiveness of routinely prescribed DMF in MS.

Design/Methods Patients newly prescribed DMF were recruited from ~380 sites. The primary objective was to determine incidence, type, and pattern of serious adverse events (SAEs), and AEs leading to discontinuation. Secondary objectives included assessment of DMF effectiveness on annualized relapse rate (ARR) and patient-reported outcomes (PROs).

Results At April 6, 2018, 4140 patients had ≥1 dose of DMF and qualified for analysis. Among these, 1148 had a new MS diagnosis. Mean (SD) age was 40.3 (11.3) years; 74% were female. One hundred and eighty-seven patients (4.5%) experienced SAEs: infections (n=42; 1.0%) and gastrointestinal disorders (n=24; <1%) were most common. There were 1255 (30.3%) permanent discontinuations: 752 (18.2%) due to AEs (gastrointestinal AEs [318, 7.7%], and occurrence of lymphopenia [83, 2.0%]), and 182 (4.4%) due to lack of efficacy. Overall ARR over 4 years (0.12; 95% CI: 0.11–0.13) was significantly lower than in the year prior to baseline (0.82, 95% CI: 0.79–0.84), an 85.3% risk reduction [95% CI: 84.1–86.4, P<0.0001]). Among newly diagnosed patients at Year 4 (n=198), 3 had one relapse and the remaining had none. PROs were generally stable from baseline to Year 4.

Conclusions

Results from the 4-year interim analysis reveal no new safety concerns emerging from real-world use of DMF. There was a maintenance of DMF effectiveness for most patients who remained on treatment.

Support: Biogen

Authors/Disclosures
Konstantin E. Balashov, MD, PhD, F�鶹��ýӳ�� (Department of Neurology, BMC and BUSM) PRESENTER	Dr. Balashov has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech.
Krupa S. Pandey, MD (Hackensack University Medical Center)	Dr. Pandey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BMS. Dr. Pandey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi Genzyme. Dr. Pandey has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech . Dr. Pandey has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for BMS. Dr. Pandey has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen . Dr. Pandey has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Genentech . Dr. Pandey has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Horizon Therapeutics . The institution of Dr. Pandey has received research support from NIH. The institution of Dr. Pandey has received research support from CMSC.
Kathryn Giles	Kathryn Giles has stock in Synderdisc. The institution of Kathryn Giles has received research support from Biogen.
Richard A. Macdonell, MD, F�鶹��ýӳ�� (Austin Health)	Dr. Macdonell has nothing to disclose.
Jorg Windsheimer	Jorg Windsheimer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck, Biogen,BMS,Janssen,Roche,Hexal. Jorg Windsheimer has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen,Teva,BMS.
	No disclosure on file
Becky J. Parks, MD (Blueprint Medicines)	Dr. Parks has received personal compensation for serving as an employee of Alexion Pharmaceuticals. An immediate family member of Dr. Parks has received personal compensation for serving as an employee of Edward P. Evans Foundation. Dr. Parks has stock in Biogen. An immediate family member of Dr. Parks has stock in Regeneron. Dr. Parks has stock in Blueprint Medicines. Dr. Parks has stock in Sanofi. An immediate family member of Dr. Parks has stock in Vertex. Dr. Parks has stock in AstraZeneca. The institution of an immediate family member of Dr. Parks has received research support from NIH/NCI. The institution of an immediate family member of Dr. Parks has received research support from Leukemia & Lymphoma Society. The institution of an immediate family member of Dr. Parks has received research support from AstraZeneca Scholar Award.
	No disclosure on file
Cynthia Jones, PhD	Dr. Jones has received personal compensation for serving as an employee of Biogen. Dr. Jones has received stock or an ownership interest from Biogen.

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