Retrospective, multicenter, and multinational study of patients with AQP4-Ab NMOSD aged <18 years at disease onset from a center in Brazil and 13 European centers.
A total of 67 children were identified. 57.8%) were found to have permanent disability. A more severe disease course was seen in the non-White ethnicity with both a shorter time to first relapse and a worse Expanded Disability Status Scale score at last follow-up. No patient treated with rituximab as first-line therapy relapsed. Optic neuritis at onset was associated with a poor visual outcome below 20/200, and a younger age at onset was associated with cognitive impairment.
International consensus on treatment protocols for children is required to reduce heterogeneity of treatment regimens used worldwide.

Paolilo RB, Hacohen Y, Yazbeck E, Armangue T, Bruijstens A, Lechner C, ApÓstolos-Pereira SL, Martynenko Y, Breu M, De Medeiros Rimkus C, Wassmer E, Baumann M, Papetti L, Capobianco M, Kornek B, Rostásy K, Da Paz JA, Ciccarelli O, Lim M, Saiz A, Neuteboom R, Marignier R, Hemingway C, Sato DK, Deiva K. Treatment and outcome of aquaporin-4 antibody-positive NMOSD: A multinational pediatric study. Neurol Neuroimmunol Neuroinflamm.

The objective of this French paediatric cohort is to describe cognitive abilities through the evaluation of academic difficulties in children, aged 18 years and younger, with acute demyelinating syndromes (ADS) and myelin oligodendrocyte glycoprotein (MOG) antibodies.
Seventy‐six children (36 females, 40 males) were included in the study with a mean follow‐up of 4 years 7 months. Median age at disease onset was 9 years 1 month. 36 children relapsed and 20 had academic difficulties at the last follow‐up. Academic difficulties, as well as deep grey matter and putaminal lesions, were significantly more prevalent in children aged 10 years and younger.
We found that age at disease onset of 10 years and younger, acute disseminated encephalomyelitis at disease onset, and deep grey matter lesions were associated with academic difficulties.

Deiva K, Cobo-Calvo A, Maurey H, De Chalus A, Yazbeck E, Husson B, Vukusic S, Serguerra C, Horellou P, Marignier R, Kidbiosep Cohort. Risk factors for academic difficulties in children with myelin oligodendrocyte glycoprotein antibody‐associated acute demyelinating syndromes. Developmental Medicine & Child Neurology.

This study compared the scale of disability, fatigue, depression and quality of life of 26 children with MS (Multiple Sclerosis) and 11 children with ADS (Acute Demyelinating Syndromes).
Although not significant, severe fatigue was less frequently reported by patients with multiple sclerosis than children with ADS. Depression was reported more often in the multiple sclerosis group compared to the ADS group. Concerning the QoL in patients with multiple sclerosis, both parents and children reported poor emotional and school functioning. Physical and social functioning were rated as being good in both groups, and was significantly higher in the children’s group.
This clinical trial highlights the importance of fatigue and depression in children with ADS and particularly in paediatric onset multiple sclerosis. School difficulties and emotional functioning were the main concerns of parents and MS patients, and must be considered when requesting care and treatment proposal.

Florea A, Maurey H, Le Sauter M, Bellesme C, Sevin C, Deiva K. Service de Neurologie pédiatrique, CHU Bicêtre, APHP, Le Kremlin Bicêtre. Fatigue, Depression, and Quality of Life in Children With Multiple Sclerosis: A Comparative Study With Other Demyelinating Diseases. Developmental Medicine & Child Neurology.

In PARADIGMS, a double-blind phase III trial in 215 paediatric patients with multiple sclerosis (MS) (10 to <18 years), fingolimod administered for up to 2 years significantly reduced the annualised relapse rate (ARR) and rate of new/newly enlarged T2 (n/neT2) lesions compared with interferon (IFN) β-1a.
In the treatment-naïve subpopulation, fingolimod reduced ARR and n/neT2 lesions by 85.8% and 53.4%, respectively versus INF β-1a, compared with 81.9% and 52.6% in the overall population. Model-based ARR reductions in younger patients (≤12 years) were 91.9%-94.6%. Twice as many IFN β-1a-treated than fingolimod-treated patients had worse EDSS scores at study end.
Fingolimod in paediatric MS was associated with consistent control of disease activity versus IFN β-1a and resulted in less disability progression for up to 2 years.

Deiva K, Huppke P, Banwell B, Chitnis T, Gärtner J, Krupp L, Waubant E, Stites T, Pearce GL, Merschhemke M. Consistent Control of Disease Activity With Fingolimod vs IFN β-1a in Pediatric-Onset Multiple Sclerosis. Journal of Neurology Neurosurgery & Psychiatry.

PARADIGMS is the first, phase III, randomized, double-blind, double-placebo, clinical trial in childhood multiple sclerosis compared oral Fingolimod at a dose of 0.5 mg per day (0.25 mg per day for patients with a body weight of 40 kg) to intramuscular interferon beta-1a (IFNβ1a) at a dose 30 g per week.
Fingolimod significantly reduced the adjusted annualized relapse rate by 82%, compared to IFNβ1a treatment. The annualized rate of new or newly enlarged lesions on MRI was significantly lower with Fingolimod (4.39) than with IFNβ1a (9.27).
Adverse events, excluding relapses of multiple sclerosis, have been observed in 88.8% of patients treated with Fingolimod and 95.3% of those who received IFNβ1a. Serious adverse events have been reported in 16.8% of patients in the Fingolimod group, and 6.5% of patients in the IFNβ1a group.

Chitnis T, Arnold DL, Banwell B, Brück W, Ghezzi A, Giovannoni G, Greenberg B, Krupp L, Rostásy K, Tardieu M, Waubant E, Wolinsky JS, Bar-Or A, Stites T, Chen Y, Putzki N, Merschhemke M, Gärtner J PARADIGMS Study Group. Trial fo Fingolimod Versus Interferon beta-1a in Pediatric Multiple Sclerosis. New England Journal of Medicine.