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Delayed-release Dimethyl Fumarate demonstrates sustained efficacy over nine years in newly diagnosed patients with relapsing-remitting multiple Sclerosis
r e v u e n e u r o l o g i q u e 1 7 5 ( 2 0 1 9 ) S45–S102
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Real-world Strategies for Management of Gastrointestinal Events in Patients Treated with Delayed-release Dimethyl Fumarate: EFFECT Gastrointestinal Sub-study Results
Delayed-release Dimethyl Fumarate demonstrates sustained efficacy over nine years in newly diagnosed patients with relapsing-remitting multiple Sclerosis
Min 1 ,
Sloane 2 ,
Fang 3 ,
Taylor 1,∗ ,
Jinny Jacob Fang Catherine Theodore Phillips 4 1 Biogen, Cambridge, États-Unis 2 Neurology, Beth Israel Deaconess Medical Center, Boston, États-Unis 3 Cytel Inc., Cambridge, États-Unis 4 Multiple sclerosis program, Baylor Institute for Immunology Research, Dallas, États-Unis ∗ Corresponding author. Adresse e-mail : [email protected] (C. Taylor)
Introduction Gastrointestinal (GI) adverse events (AEs) associated with DMF can result in premature treatment discontinuation. Objectives To characterize effective real-world patient education and management strategies at sites initiating treatment with delayed-release dimethyl fumarate (DMF), as well as the impact of GI events on treatment persistence. Patients and methods EFFECT (NCT02776072) is a retrospective study of patients age ≥ 18 treated with DMF in the clinical practice setting. Data were captured via retrospective medical record review at a single time point. Sites completed a structured questionnaire regarding typical GI management practices at the center using a scale of 0%, > 0–25%, > 25%–75%, and > 75%–100%. Results Overall, 826 DMF-treated patients were enrolled at 65 sites; 809 were eligible for analysis. Over Year 1, incidence of GI events was 27% (216/809). Sites reporting that counseling was likely to occur by both the prescriber and an additional health care provider had lower discontinuation rates vs. sites that did not (2% [6/286] vs. 7% [33/495]). Discussion Sites with lower vs. higher discontinuation rates were also likely to provide patients specific details regarding GI events (5% [33/693] vs. 10% [11/116]), recommend taking DMF with food (5% [39/750] vs. 9% [5/59]), or recommend using symptomatic GI therapies (3% [15/469] vs. 9% [29/340]). Conclusion Overall, treatment persistence was high in this study, potentially due to most sites reporting a high likelihood of counseling and mitigation strategy recommendations for potential DMF-related GI events. Keywords Persistence; Dimethyl fumarate; Effets gastro-intestinaux Disclosure of interest The authors have not supplied their declaration of competing interest. https://doi.org/10.1016/j.neurol.2019.01.262
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Ralf Gold 1 , Gavin Giovannoni 2 , Theodore Phillips 3 , Amit Bar-Or 4 , Robert J. Fox 5 , Chongshu Chen 6,∗ , Catherine Miller 6 1 Neurology, St. Josef-Hospital, Ruhr University Bochum, Bochum, Allemagne 2 Neurologie, Queen Mary University of London, Londres, Royaume-Uni 3 Multiple sclerosis program, Baylor Institute for Immunology Research, Dallas, États-Unis 4 Center for neuroinflammation and experimental therapeutics and department of neurology, Université de Pennsylvanie, Philadelphie, États-Unis 5 Mellen center for multiple sclerosis treatment and research, Cleveland Clinic, Cleveland, États-Unis 6 Biogen, Cambridge, États-Unis ∗ Corresponding author. Adresse e-mail : [email protected] (C. Chen) Introduction DMF demonstrated strong efficacy and a favourable benefit-risk profile in RRMS patients in Phase 3 studies (DEFINE/CONFIRM), which is sustained in the extension study, ENDORSE. Objectives To evaluate efficacy in newly diagnosed patients with RRMS treated with DMF for ∼9 years. Patients and methods An integrated analysis of newly diagnosed patients who continued after 2 years onto the ENDORSE study assessed annualised relapse rate (ARR) and EDSS score. Results are reported for patients treated with DMF 240 mg BID: placebo (PBO)/DMF (PBO for Years 0–2/DMF for Years 3–9) or continuous (DMF/DMF) treatment. Results The observed proportion of patients with EDSS ≤ 3.5 was 129/139 (93%) and 65/72 (90%) at Year 2, and 50/54 (93%) and 26/28 (93%) at Year 9 for DMF/DMF and PBO/DMF, respectively. For PBO/DMF, adjusted ARR (95% CI) was 0.25 (0.18–0.37) for Years 0–2 (PBO) and 0,09 (0,06–0.14) for Years 3–9 (DMF). Discussion NA. Conclusion The majority of patients remaining on study maintained walking abilities (EDSS ≤ 3.5) over 9 years and ARR remained low. Keywords Efficacité à long terme; ENDORSE; Dimethyl fumarate (DMF) Disclosure of interest The authors have not supplied their declaration of competing interest. https://doi.org/10.1016/j.neurol.2019.01.263 W54
Real-world data from over 10 years in the TYSABRI® Observational Program: Long-term safety and effectiveness of natalizumab in relapsing-remitting multiple sclerosis patients
Ludwig Kappos 1 , Helmut Butzkueven 2 , Tim Spelman 3 , Maria Trojano 4 , Heinz Wiendl 5 , Xiaoyu Jiang 6 , Rachna Kasliwal 6 , Nolan Campbell 6 , Pei-Ran Ho 6 , Stephanie Licata 6,∗ 1 Neurologic clinic and policlinic, departments of medicine, University Hospital and University of Basel, Bâle, Suisse 2 Neurology, Box Hill Hospital, Monash University, Melbourne, Australie
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Real-world Strategies for Management of Gastrointestinal Events in Patients Treated with Delayed-release Dimethyl Fumarate: EFFECT Gastrointestinal Sub-study Results
Delayed-release Dimethyl Fumarate demonstrates sustained efficacy over nine years in newly diagnosed patients with relapsing-remitting multiple Sclerosis
Min 1 ,
Sloane 2 ,
Fang 3 ,
Taylor 1,∗ ,
Jinny Jacob Fang Catherine Theodore Phillips 4 1 Biogen, Cambridge, États-Unis 2 Neurology, Beth Israel Deaconess Medical Center, Boston, États-Unis 3 Cytel Inc., Cambridge, États-Unis 4 Multiple sclerosis program, Baylor Institute for Immunology Research, Dallas, États-Unis ∗ Corresponding author. Adresse e-mail : [email protected] (C. Taylor)
Introduction Gastrointestinal (GI) adverse events (AEs) associated with DMF can result in premature treatment discontinuation. Objectives To characterize effective real-world patient education and management strategies at sites initiating treatment with delayed-release dimethyl fumarate (DMF), as well as the impact of GI events on treatment persistence. Patients and methods EFFECT (NCT02776072) is a retrospective study of patients age ≥ 18 treated with DMF in the clinical practice setting. Data were captured via retrospective medical record review at a single time point. Sites completed a structured questionnaire regarding typical GI management practices at the center using a scale of 0%, > 0–25%, > 25%–75%, and > 75%–100%. Results Overall, 826 DMF-treated patients were enrolled at 65 sites; 809 were eligible for analysis. Over Year 1, incidence of GI events was 27% (216/809). Sites reporting that counseling was likely to occur by both the prescriber and an additional health care provider had lower discontinuation rates vs. sites that did not (2% [6/286] vs. 7% [33/495]). Discussion Sites with lower vs. higher discontinuation rates were also likely to provide patients specific details regarding GI events (5% [33/693] vs. 10% [11/116]), recommend taking DMF with food (5% [39/750] vs. 9% [5/59]), or recommend using symptomatic GI therapies (3% [15/469] vs. 9% [29/340]). Conclusion Overall, treatment persistence was high in this study, potentially due to most sites reporting a high likelihood of counseling and mitigation strategy recommendations for potential DMF-related GI events. Keywords Persistence; Dimethyl fumarate; Effets gastro-intestinaux Disclosure of interest The authors have not supplied their declaration of competing interest. https://doi.org/10.1016/j.neurol.2019.01.262
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Ralf Gold 1 , Gavin Giovannoni 2 , Theodore Phillips 3 , Amit Bar-Or 4 , Robert J. Fox 5 , Chongshu Chen 6,∗ , Catherine Miller 6 1 Neurology, St. Josef-Hospital, Ruhr University Bochum, Bochum, Allemagne 2 Neurologie, Queen Mary University of London, Londres, Royaume-Uni 3 Multiple sclerosis program, Baylor Institute for Immunology Research, Dallas, États-Unis 4 Center for neuroinflammation and experimental therapeutics and department of neurology, Université de Pennsylvanie, Philadelphie, États-Unis 5 Mellen center for multiple sclerosis treatment and research, Cleveland Clinic, Cleveland, États-Unis 6 Biogen, Cambridge, États-Unis ∗ Corresponding author. Adresse e-mail : [email protected] (C. Chen) Introduction DMF demonstrated strong efficacy and a favourable benefit-risk profile in RRMS patients in Phase 3 studies (DEFINE/CONFIRM), which is sustained in the extension study, ENDORSE. Objectives To evaluate efficacy in newly diagnosed patients with RRMS treated with DMF for ∼9 years. Patients and methods An integrated analysis of newly diagnosed patients who continued after 2 years onto the ENDORSE study assessed annualised relapse rate (ARR) and EDSS score. Results are reported for patients treated with DMF 240 mg BID: placebo (PBO)/DMF (PBO for Years 0–2/DMF for Years 3–9) or continuous (DMF/DMF) treatment. Results The observed proportion of patients with EDSS ≤ 3.5 was 129/139 (93%) and 65/72 (90%) at Year 2, and 50/54 (93%) and 26/28 (93%) at Year 9 for DMF/DMF and PBO/DMF, respectively. For PBO/DMF, adjusted ARR (95% CI) was 0.25 (0.18–0.37) for Years 0–2 (PBO) and 0,09 (0,06–0.14) for Years 3–9 (DMF). Discussion NA. Conclusion The majority of patients remaining on study maintained walking abilities (EDSS ≤ 3.5) over 9 years and ARR remained low. Keywords Efficacité à long terme; ENDORSE; Dimethyl fumarate (DMF) Disclosure of interest The authors have not supplied their declaration of competing interest. https://doi.org/10.1016/j.neurol.2019.01.263 W54
Real-world data from over 10 years in the TYSABRI® Observational Program: Long-term safety and effectiveness of natalizumab in relapsing-remitting multiple sclerosis patients
Ludwig Kappos 1 , Helmut Butzkueven 2 , Tim Spelman 3 , Maria Trojano 4 , Heinz Wiendl 5 , Xiaoyu Jiang 6 , Rachna Kasliwal 6 , Nolan Campbell 6 , Pei-Ran Ho 6 , Stephanie Licata 6,∗ 1 Neurologic clinic and policlinic, departments of medicine, University Hospital and University of Basel, Bâle, Suisse 2 Neurology, Box Hill Hospital, Monash University, Melbourne, Australie