Effect of fingolimod on health-related quality of life in paediatric patients with multiple sclerosis: results from the phase 3 PARADIG MS Study

ORCID
0000-0001-7003-807X
Affiliation
Pediatric MS Center ,NYU Langone Health ,New York ,New York ,USA
Krupp, Lauren;
Affiliation
The Children’s Hospital of Philadelphia, Perelman School of Medicine ,University of Pennsylvania ,Philadelphia ,Pennsylvania ,USA
Banwell, Brenda;
ORCID
0000-0002-9897-4422
Affiliation
Pediatric Multiple Sclerosis Center ,Massachusetts General Hospital ,Boston ,Massachusetts ,USA
Chitnis, Tanuja;
Affiliation
Department of Pediatric Neurology ,French National Reference Center for Rare inflammatory and Auto-Immune Brain and Spinal Diseases, University Hospitals Paris Saclay, Bicêtre Hospital, Le Kremlin Bicêtre ,Paris ,France
Deiva, Kumaran;
Affiliation
Department of Pediatrics and Adolescent Medicine ,German Centre for Multiple Sclerosis in Childhood and Adolescence University Medical Centre ,Göttingen ,Germany
Gaertner, Jutta;
Affiliation
Centro Studi Sclerosi Multipla ,Ospedale di Gallarate ,Gallarate ,Italy
Ghezzi, Angelo;
GND
1261365941
Affiliation
Department of Pediatrics and Adolescent Medicine ,German Centre for Multiple Sclerosis in Childhood and Adolescence University Medical Centre ,Göttingen ,Germany
Huppke, Peter;
Affiliation
Department of Neurology ,University of California ,San Francisco ,California ,USA
Waubant, Emmanuelle;
Affiliation
Global Medical Affairs ,Novartis Pharma AG ,Basel ,Switzerland
DeLasHeras, Virginia;
Affiliation
Biostatistics ,Novartis Pharma AG ,Basel ,Switzerland
Azmon, Amin;
Affiliation
Global Drug Delivery ,Novartis Pharma AG ,Basel ,Switzerland
Karan, Rajesh

Background In the PARADIG MS Study, fingolimod demonstrated superior efficacy versus interferon (IFN) β-1a and comparable overall incidence of adverse events but slightly higher rate of serious adverse events in patients with paediatric-onset multiple sclerosis (PoMS). Here, we report the health-related quality of life (HRQoL) outcomes from PARADIG MS . Methods Patients with PoMS (N=215; aged 10–<18 years) were randomised to once-daily oral fingolimod (N=107) or once-weekly intramuscular IFN β-1a (N=108). HRQoL outcomes were assessed using the 23-item Pediatric Quality of Life (PedsQL) scale that comprises Physical and Psychosocial Health Summary Scores (including Emotional, Social and School Functioning). A post hoc inferential analysis evaluated changes in self-reported or parent-reported PedsQL scores from baseline up to 2 years between treatment groups using an analysis of covariance model. Results Treatment with fingolimod showed improvements versus IFN β-1a on the PedsQL scale in both the self-reported and parent-reported Total Scale Scores (4.66 vs −1.16, p≤0.001 and 2.71 vs −1.02, p≤0.05, respectively). The proportion of patients achieving a clinically meaningful improvement in the PedsQL Total Scale Score was two times higher with fingolimod versus IFN β-1a per the self-reported scores (47.5% vs 24.2%, p=0.001), and fingolimod was favoured versus IFN β-1a per the parent-reported scores (37.8% vs 24.7%, p=non-significant). Group differences in self-reported Total Scale Scores in favour of fingolimod were most pronounced among patients who had ≥2 relapses in the year prior to study entry or who showed improving or stable Expanded Disability Status Scale scores during the study. Conclusion Fingolimod improved HRQoL compared with IFN β-1a in patients with PoMS as evidenced by the self-reported and parent-reported PedsQL scores.

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