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Review
. 2023 Feb;81(2):201-211.
doi: 10.1055/s-0043-1761432. Epub 2023 Mar 22.

Neuromyelitis optica spectrum disorders: a review with a focus on children and adolescents

Renata Barbosa Paolilo  1 , José Albino da Paz  1 , Samira Luisa Apóstolos-Pereira  1 , Carolina de Medeiros Rimkus  1 , Dagoberto Callegaro  1 , Douglas Kazutoshi Sato  2
Affiliations
Review

Neuromyelitis optica spectrum disorders: a review with a focus on children and adolescents

Renata Barbosa Paolilo et al. Arq Neuropsiquiatr. 2023 Feb.

Abstract
in English, Portuguese

Neuromyelitis optica spectrum disorder (NMOSD) is a rare and severe inflammatory disorder of the central nervous system (CNS). It is strongly associated with anti-aquaporin 4 antibodies (AQP4-IgG), and it mainly affects young women from non-white ethnicities. However, ∼ 5 to 10% of all cases have onset during childhood. Children and adolescents share the same clinical, radiologic, and laboratory presentation as adults. Thus, the same NMOSD diagnostic criteria are also applied to pediatric-onset patients, but data on NMOSD in this population is still scarce. In seronegative pediatric patients, there is a high frequency of the antibody against myelin oligodendrocyte glycoprotein (MOG-IgG) indicating another disease group, but the clinical distinction between these two diseases may be challenging. Three drugs (eculizumab, satralizumab, and inebilizumab) have been recently approved for the treatment of adult patients with AQP4-IgG-positive NMOSD. Only satralizumab has recruited adolescents in one of the two pivotal clinical trials. Additional clinical trials in pediatric NMOSD are urgently required to evaluate the safety and efficacy of these drugs in this population.

O espectro da neuromielite óptica (ENMO) é uma rara e grave doença inflamatória do sistema nervoso central (SNC), fortemente associada ao anticorpo anti-aquaporina 4 (AQP4-IgG) e que afeta preferencialmente mulheres jovens de etnias não-caucasianas. No entanto, aproximadamente de 5 a 10% de todos os casos se iniciam na infância. Crianças e adolescentes compartilham as mesmas características clínicas, radiológicas e laboratoriais dos adultos. Além disso, o mesmo critério diagnóstico de ENMO é aplicado para pacientes com início na infância. No entanto, dados da população pediátrica são escassos. Em pacientes pediátricos soronegativos, existe uma alta frequência de positividade ao anticorpo contra a glicoproteína na mielina do oligodendrócito (MOG-IgG), indicando outra patologia; porém, a distinção clínica entre as duas doenças é desafiadora. Três medicações (eculizumabe, inebilizumabe e satralizumabe) foram recentemente aprovadas para pacientes adultos com AQP4-IgG. Apenas um dos ensaios pivotais do satralizumabe recrutou adolescentes. Novos ensaios clínicos em pacientes pediátricos com ENMO são necessários para avaliar a segurança e eficácia destas drogas nesta população.

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Conflict of interest statement

The authors declare that there are no conflicts of interest related to this study. RBP: reports grants from Roche and Merck and speaker honoraria from Novartis, outside the submitted work; JAP: nothing to disclose; SLAP: reports grants, preceptorships and research support from Genzyme, Roche, Novartis, Merck-Serono, and Biogen, outside the submitted work; CMR: reports speaker honoraria from Roche and the Brazilian Committee for Treatment and Research of Multiple Sclerosis, outside the submitted work; DC: reports grants, preceptorships, and research support from Genzyme, Roche, Novartis, Merck-Serono, Teva and Biogen, outside the submitted work; DKS: reports grants from Conselho Nacional de Desenvolvimento Científico e Tecnológico (CNPq) – Brazil (425331/2016–4), grants from Fundação e Amparo à Pesquisa do Estado do Rio Grande do Sul (FAPERGS) – Brazil, grants and personal fees from TEVA and Merck, personal fees from Biogen, Roche, and Viela Bio, outside the submitted work.

Figures

Figure 1
Figure 1
Eugène Devic (left) and his pupil Fernand Gault doctoral thesis (right) Adapted from Jarius et al., 2013.
Figure 2
Figure 2
Typical Neuroimaging findings in pediatric AQP4-IgG positive neuromyelitis optica Spectrum Disorder. ( A) Unilateral optic neuritis; ( B) Bilateral optic neuritis with optic chiasm involvement; ( C) Longitudinally extensive transverse myelitis; ( D) Longitudinally extensive atrophy; ( E) Bright Spotty lesion; (F) and ( G) Area postrema involvement; ( H) Diencephalic lesion; ( I) Corpus callosum lesion.
See this image and copyright information in PMC

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