Contributions
Abstract: P922
Type: Poster Sessions
Abstract Category: Therapy - Long-term treatment monitoring
Background: Once-daily fingolimod (Gilenya®, Novartis Pharma AG) is a sphingosine 1-phosphate receptor modulator approved for the treatment of adults with relapsing-remitting multiple sclerosis (RRMS). Recently, fingolimod has been approved by the FDA for treatment of pediatric MS.
Objective: Here we present interim results on the effectiveness of fingolimod in two subgroups of young adult (< 20 and < 30 years of age) patients with RRMS treated up to 5 years in daily clinical practice.
Methods: PANGAEA is a non-interventional study, conducted in Germany, to investigate long-term safety, effectiveness and patient reported outcomes in daily clinical practice. Of the total enrolled 4229 patients, 81 (1.9%) were younger than 20 and 896 (21.1%) younger than 30 years of age.
Results: At baseline, patients < 20 (mean age 19.1 ±1.0 SD) and < 30 years (25.6 ±3.3 SD) had a mean annual relapse rate (ARR) of 1.9 (±0.25 95%CI) and 1.7 (±0.09 95%CI), respectively, compared to 1.5 (±0.04 95%CI) in the total study population (mean age 39.1 ±10.0 SD). EDSS score was 1.8 (±1.3 SD) and 2.2 (±1.5 SD) compared to 3.0 (±1.7 SD) in the total population. Time since diagnosis was 2.9 (±2.2 SD; < 20) and 4.4 (±3.1 SD; < 30) years compared to 8.2 (±6.3 SD) in the total population. The ARR improved in the first year by 67% (< 20) and 71% (< 30) to 0.63 (±0.20 95%CI) and 0.50 (±0.06 95%CI), respectively, and further improved to 0.33 (±0.41 95%CI; < 20) and 0.33 (±0.11 95%CI; < 30) in the fifth year of treatment. In each year of treatment approximately 90% of the patients in both subgroups had a stable EDSS or experienced a 6 months confirmed improvement. The proportion of patients free of relapses and 6 months confirmed disability progression increased from 45% (< 20) and 56% (< 30) in year 1 to 50% and 74% in year 4 of treatment. 43% (< 20) and 49% (< 30) of the patients neither had a relapse nor a 6 months confirmed disability progression over the observational period. Within 5 years of treatment the effectiveness was deemed “good” or “very good” by 80-100% (< 20) and 91-99% (< 30) of patients and physicians. 83% (< 20) and 70% (< 30) remained on therapy during the 5 year observational period. The nature of reported adverse events is consistent with previous findings from clinical trials.
Conclusion: The 5 year interim analysis provides real world evidence for a long-term treatment benefit in young adult patients across clinical and subjective measures of disease activity.
Disclosure: This study is funded by Novartis Pharma GmbH.
T. Ziemssen has received personal compensation for participating on advisory boards, trial steering committees and data and safety monitoring committees, as well as for scientific talks and project support from: Bayer HealthCare, Biogen, Elan, Genzyme, Merck Serono, Novartis, Roche, Sanofi-Aventis, Synthon and Teva.H. Albrecht has nothing to discloseL. Klotz received compensation for serving on scientific advisory boards for Genzyme and Novartis. She received speaker honoraria and travel support from Novartis, Merck Serono and CSL Behring. She receives research support from Novartis and Biogen.M. Lang has received research support from NovartisC. Lassek has received travel grants, speaker's honoraria, financial research support, consultancy fees from Teva, Merck Serono, Genzyme -Sanofi, Novartis, Bayer, Biogen.S. Schmidt has received speaking honoraria, travel compensations and has served on advisory boards for BayerVital, Biogen, MerckSerono, Novartis and Teva.B. Tackenberg has received travel reimbursements, speaker and consulting honoraria from Bayer Healthcare, Biogen, Genzyme, Merck Serono, Novartis, Sanofi-Aventis and Teva as well as research support from Bayer Healthcare, Biogen and Novartis.C. Cornelissen and B. Ettle are employees of the Novartis Pharma GmbH, Nuremberg, Germany.
Abstract: P922
Type: Poster Sessions
Abstract Category: Therapy - Long-term treatment monitoring
Background: Once-daily fingolimod (Gilenya®, Novartis Pharma AG) is a sphingosine 1-phosphate receptor modulator approved for the treatment of adults with relapsing-remitting multiple sclerosis (RRMS). Recently, fingolimod has been approved by the FDA for treatment of pediatric MS.
Objective: Here we present interim results on the effectiveness of fingolimod in two subgroups of young adult (< 20 and < 30 years of age) patients with RRMS treated up to 5 years in daily clinical practice.
Methods: PANGAEA is a non-interventional study, conducted in Germany, to investigate long-term safety, effectiveness and patient reported outcomes in daily clinical practice. Of the total enrolled 4229 patients, 81 (1.9%) were younger than 20 and 896 (21.1%) younger than 30 years of age.
Results: At baseline, patients < 20 (mean age 19.1 ±1.0 SD) and < 30 years (25.6 ±3.3 SD) had a mean annual relapse rate (ARR) of 1.9 (±0.25 95%CI) and 1.7 (±0.09 95%CI), respectively, compared to 1.5 (±0.04 95%CI) in the total study population (mean age 39.1 ±10.0 SD). EDSS score was 1.8 (±1.3 SD) and 2.2 (±1.5 SD) compared to 3.0 (±1.7 SD) in the total population. Time since diagnosis was 2.9 (±2.2 SD; < 20) and 4.4 (±3.1 SD; < 30) years compared to 8.2 (±6.3 SD) in the total population. The ARR improved in the first year by 67% (< 20) and 71% (< 30) to 0.63 (±0.20 95%CI) and 0.50 (±0.06 95%CI), respectively, and further improved to 0.33 (±0.41 95%CI; < 20) and 0.33 (±0.11 95%CI; < 30) in the fifth year of treatment. In each year of treatment approximately 90% of the patients in both subgroups had a stable EDSS or experienced a 6 months confirmed improvement. The proportion of patients free of relapses and 6 months confirmed disability progression increased from 45% (< 20) and 56% (< 30) in year 1 to 50% and 74% in year 4 of treatment. 43% (< 20) and 49% (< 30) of the patients neither had a relapse nor a 6 months confirmed disability progression over the observational period. Within 5 years of treatment the effectiveness was deemed “good” or “very good” by 80-100% (< 20) and 91-99% (< 30) of patients and physicians. 83% (< 20) and 70% (< 30) remained on therapy during the 5 year observational period. The nature of reported adverse events is consistent with previous findings from clinical trials.
Conclusion: The 5 year interim analysis provides real world evidence for a long-term treatment benefit in young adult patients across clinical and subjective measures of disease activity.
Disclosure: This study is funded by Novartis Pharma GmbH.
T. Ziemssen has received personal compensation for participating on advisory boards, trial steering committees and data and safety monitoring committees, as well as for scientific talks and project support from: Bayer HealthCare, Biogen, Elan, Genzyme, Merck Serono, Novartis, Roche, Sanofi-Aventis, Synthon and Teva.H. Albrecht has nothing to discloseL. Klotz received compensation for serving on scientific advisory boards for Genzyme and Novartis. She received speaker honoraria and travel support from Novartis, Merck Serono and CSL Behring. She receives research support from Novartis and Biogen.M. Lang has received research support from NovartisC. Lassek has received travel grants, speaker's honoraria, financial research support, consultancy fees from Teva, Merck Serono, Genzyme -Sanofi, Novartis, Bayer, Biogen.S. Schmidt has received speaking honoraria, travel compensations and has served on advisory boards for BayerVital, Biogen, MerckSerono, Novartis and Teva.B. Tackenberg has received travel reimbursements, speaker and consulting honoraria from Bayer Healthcare, Biogen, Genzyme, Merck Serono, Novartis, Sanofi-Aventis and Teva as well as research support from Bayer Healthcare, Biogen and Novartis.C. Cornelissen and B. Ettle are employees of the Novartis Pharma GmbH, Nuremberg, Germany.