Yifan Zhou1, Xiaonan Zhong1, Yaqing Shu1, Chunping Cui1, Jingqi Wang1, Yuge Wang1, Xiaojing Li2, Zhuanggui Chen3, Lisheng Peng1, Allan Kermode4, Wei Qiu5. 1. Department of Neurology, The Third Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China. 2. Department of Neurology, Guangzhou Women and Children's Medical Center, Guangzhou, China. 3. Department of Pediatrics, The Third Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China. 4. Department of Neurology, The Third Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China; Centre for Neuromuscular and Neurological Disorders, University of Western Australia, Department of Neurology, Sir Charles Gairdner Hospital, Queen Elizabeth II Medical Centre, Perth, Australia; Institute of Immunology and Infectious Diseases, Murdoch University, Perth, Australia. 5. Department of Neurology, The Third Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China. Electronic address: qiuwei120@vip.163.com.
Abstract
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.
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.