TY - JOUR
T1 - Clinical course, treatment responses and outcomes in Chinese paediatric neuromyelitis optica spectrum disorder
AU - Zhou, Yifan
AU - Zhong, Xiaonan
AU - Shu, Yaqing
AU - Cui, Chunping
AU - Wang, Jingqi
AU - Wang, Yuge
AU - Li, Xiaojing
AU - Chen, Zhuanggui
AU - Peng, Lisheng
AU - Kermode, Allan
AU - Qiu, Wei
PY - 2019/2/1
Y1 - 2019/2/1
N2 - Background: Few data exists on paediatric neuromyelitis optica spectrum disorder (NMOSD). Here, we investigated the clinical presentation, treatment responses and long-term prognoses in a large cohort of patients with NMOSD and compared between children and adults with aquaporin-4 antibody (AQP4-IgG). Methods: A retrospective chart review of patients followed in multiple centres identified 127 patients with NMOSD (31 children; 96 adults). Data were collected through medical records and structured questionnaire. Results: In the paediatric cohort, median age at onset was 14 (range 7–17) years; 87% were female. AQP4 and myelin oligodendrocyte glycoprotein antibodies were detected in 82% and 16%, respectively. During a median disease duration of 48 months, 23% developed visual acuity of <6/60 Snellen, 3% were wheelchair-dependent. The frequency of brain/brainstem phenotype (18% vs 9%, p = 0.018) was more common in AQP4-IgG-positive children, while AQP4-IgG-positive adults were more likely to present transverse myelitis (TM) (44% vs 29%, p = 0.005) of all 452 episodes. Multivariable analyses showed that sustained disability was independently associated with the presence of TM (p = 0.030), brain/brainstem symptoms (p = 0.010), annualized relapse rate (p < 0.001) and possibly age of onset (p = 0.069). The reduction of ARR after azathioprine was more prominent in adults (79%) than in children (48%). Mycophenolate mofetil and rituximab decreased the relapse frequency of children, with a reduction of 94% and 100%, respectively. Conclusions: paediatric NMOSD is a severely disabling disorder characterized by repeated brain attacks and early disability accrual. Prompt therapy including mycophenolate mofetil and rituximab should be considered to improve paediatric care.
AB - Background: Few data exists on paediatric neuromyelitis optica spectrum disorder (NMOSD). Here, we investigated the clinical presentation, treatment responses and long-term prognoses in a large cohort of patients with NMOSD and compared between children and adults with aquaporin-4 antibody (AQP4-IgG). Methods: A retrospective chart review of patients followed in multiple centres identified 127 patients with NMOSD (31 children; 96 adults). Data were collected through medical records and structured questionnaire. Results: In the paediatric cohort, median age at onset was 14 (range 7–17) years; 87% were female. AQP4 and myelin oligodendrocyte glycoprotein antibodies were detected in 82% and 16%, respectively. During a median disease duration of 48 months, 23% developed visual acuity of <6/60 Snellen, 3% were wheelchair-dependent. The frequency of brain/brainstem phenotype (18% vs 9%, p = 0.018) was more common in AQP4-IgG-positive children, while AQP4-IgG-positive adults were more likely to present transverse myelitis (TM) (44% vs 29%, p = 0.005) of all 452 episodes. Multivariable analyses showed that sustained disability was independently associated with the presence of TM (p = 0.030), brain/brainstem symptoms (p = 0.010), annualized relapse rate (p < 0.001) and possibly age of onset (p = 0.069). The reduction of ARR after azathioprine was more prominent in adults (79%) than in children (48%). Mycophenolate mofetil and rituximab decreased the relapse frequency of children, with a reduction of 94% and 100%, respectively. Conclusions: paediatric NMOSD is a severely disabling disorder characterized by repeated brain attacks and early disability accrual. Prompt therapy including mycophenolate mofetil and rituximab should be considered to improve paediatric care.
KW - Age
KW - Disability
KW - Neuromyelitis optica spectrum disorder
KW - Predictor
KW - Treatment
UR - http://www.scopus.com/inward/record.url?scp=85059538200&partnerID=8YFLogxK
U2 - 10.1016/j.msard.2018.12.038
DO - 10.1016/j.msard.2018.12.038
M3 - Article
C2 - 30623860
AN - SCOPUS:85059538200
VL - 28
SP - 213
EP - 220
JO - Multiple Sclerosis and Related Disorders
JF - Multiple Sclerosis and Related Disorders
SN - 2211-0348
ER -