Abstract Details

Title Effect of fingolimod in pediatric MS: further insights from sensitivity, supportive and post-hoc analyses from PARADIGMS.

Topic Multiple Sclerosis

Presentation(s) ES1 - (-)

Poster/Presentation
Number 005

Objective

Background In PARADIGMS, a double-blind phase III trial of 215 pediatric MS patients (age 10–<18 years), fingolimod administered up to 2 years significantly reduced both the annualized relapse rate (ARR) (primary endpoint) and the rate of new/newly enlarged T2 (n/neT2) lesions (key secondary endpoint) compared to IFN β-1a.

Design/Methods Predefined analyses of the endpoints were performed after excluding patients in the IFN β-1a arm who were IFN neutralizing antibody (Nabs)-positive at study end (sensitivity analysis) and in disease-modifying therapy (DMT)-naïve patients (supportive analysis). A post-hoc evaluation of 3-month confirmed disability progression (3M-CDP) was also conducted.

Results Excluding Nabs-positiveIFN β-1a patients (n=9) did not have any tangible impact on the primary and key secondary results (81.5% ARR reduction and 47.6% reduction in n/neT2 lesions, both p<0.001, versus 81.9% and 52.6% in the overall population, respectively). At baseline, 63.3% of patients were treatment naïve. The effect of fingolimod treatment in the DMT-naïve subpopulation (85.8% ARR reduction and 53.4% n/neT2 lesion reduction versus IFN β-1a, both p<0.001) was also consistent with the overall patient population. The Kaplan–Meier estimate of the percentage of patients without 3M-CDP up to 2 years was higher in the fingolimod group (95.2%) compared with the IFN β-1a group (84.7%) (p=0.015), with a risk reduction in 3M-CDP of 77.2% (hazard ratio=0.23, p=0.007). 3M-CDP sustained until the last observation yielded similar results. Expanded disability status scale (EDSS) measurements at different time points will be presented.

Conclusions Fingolimod in pediatric MS was associated with consistent control of disease activity across sensitivity/supportive analyses, and benefits on disability progression were observed over the treatment duration of up to 2 years.

Authors/Disclosures
	No disclosure on file
Peter Huppke	No disclosure on file
Brenda L. Banwell, MD, FAAN (Childrens Hospital of Philadelphia)	Dr. Banwell has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Banwell has received personal compensation in the range of $0-$499 for serving as a Consultant for UCB. Dr. Banwell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Banwell has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Banwell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Banwell has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. The institution of Dr. Banwell has received research support from National MS Society. The institution of Dr. Banwell has received research support from NIH.
Tanuja Chitnis, MD, FAAN (Brigham and Women's Hospital)	Dr. Chitnis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Chitnis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche-Genentech. Dr. Chitnis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Siemens. Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octave Biosciences. Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. The institution of Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Chitnis has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Academic CME. The institution of Dr. Chitnis has received research support from Novartis. The institution of Dr. Chitnis has received research support from Sanofi. The institution of Dr. Chitnis has received research support from Octave. The institution of Dr. Chitnis has received research support from Genentech-Roche. The institution of Dr. Chitnis has received research support from Tiziana Life Sciences. The institution of Dr. Chitnis has received research support from Bristol-Myers Squibb. The institution of Dr. Chitnis has received research support from Wesley Clover.
Jutta Gaertner, MD	Dr. Gaertner has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Gaertner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Dr. Gaertner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Gaertner has received personal compensation in the range of $500-$4,999 for serving as a Speaker with Biogen.
Lauren B. Krupp, MD, FAAN (NYU Langone Medical Center)	Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bristol Myers Squibb. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Celgene. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medscape. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EBIX. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman LaRoche. Dr. krupp has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for MMMK. Dr. krupp has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Patrick, Dolan, and Kaufman. Dr. krupp has received intellectual property interests from a discovery or technology relating to health care.
Emmanuelle Waubant, MD, PhD, FAAN (USCF MS Center)	The institution of Dr. Waubant has received research support from NIH. The institution of Dr. Waubant has received research support from NMSS. The institution of Dr. Waubant has received research support from PCORI. The institution of Dr. Waubant has received research support from Race to Erase MS. The institution of Dr. Waubant has received research support from Roche. The institution of Dr. Waubant has received research support from Department of Defense. Dr. Waubant has received publishing royalties from a publication relating to health care.
Tracy E. Stites, PhD	Dr. Stites has received personal compensation for serving as an employee of Novartis Pharmaceuticals. Dr. Stites has received stock or an ownership interest from Novartis Pharmaceuticals.
	No disclosure on file
Martin Merschhemke	No disclosure on file

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