Abstract：Objective To evaluate the efficacy and safety of cyclophosphamide as a second-line drug in the treatment of children with anti-N-methyl-D-aspartate receptor (NMDAR) encephalitis. Methods Six children with anti-NMDAR encephalitis, who showed poor response to steroids and intravenous immunoglobulin, were given cyclophosphamide as a second-line immunotherapy. Follow-up was performed to evaluate the efficacy and safety of cyclophosphamide. Results After first-line immunotherapy for 1-4 weeks, the six patients had reduced psychiatric symptoms, seizures, and involuntary movements; three patients had an improved level of consciousness and were able to make simple conversations. However, all the patients still showed slow response, as well as cortical dysfunction symptoms such as aphasia, alexia, agraphia, acalculia, apraxia, and movement disorders. The six patients continued to receive cyclophosphamide as a sequential therapy. They were able to answer simple questions 7 days after treatment. Three school-aged patients were able to make simple calculation, had greatly improved reading and writing ability, and almost recovered self-care ability 2-3 weeks later. The cognitive function of the six patients was almost restored to the level before the onset of disease, and their living ability returned to normal 2-3 months later. During the treatment period, there were no adverse reactions or abnormal results of routine blood test and liver and kidney function tests. Conclusions Children with anti-NMDAR encephalitis should be given appropriate immunotherapy as soon as possible. Cyclophosphamide as a sequential therapy has good efficacy and safety.
ZHU Wei-Wen,LIAO Wei-Ping,YI Yong-Hong et al. Efficacy and safety of cyclophosphamide as a sequential immunotherapy drug for anti-N-methyl-D-aspartate receptor encephalitis in children[J]. CJCP, 2017, 19(6): 668-671.
Dalmau J, Gleichman AJ, Hughes EG, et al. Anti-NMDA-receptor encephalitis:case series and analysis of the effects of antibodies[J]. Lancet Neurol, 2008, 7(12):1091-1098.
[2]
Gable MS, Sheriff H, Dalmau J, et al. The frequency of autoimmune N-methyl-D-aspartate receptor encephalitis surpasses that of individual viral etiologies in young individuals enrolled in the California Encephalitis Project[J]. Clin Infect Dis, 2012, 54(7):899-904.
[3]
Dalmau J, Lancaster E, Martinez-Hernandez E, et al. Clinical experience and laboratory investigations in patients with anti-NMDAR encephalitis[J]. Lancet Neurol, 2011, 10(1):63-74.
[4]
Armangue T, Petit-Pedrol M, Dalmau J, et al. Autoimmune encephalitis in children[J]. J Child Neurol, 2012, 27(11):1460-1469.
[5]
Dalmau J, Tüzün EE, Wu HY, et al. Paraneoplastic anti-N-methyl-D-aspartate receptor encephalitis associated with ovarian teratoma[J]. Ann Neurol, 2007, 61(1):25-36.
[6]
Hughes EG, Peng X, Gleichman AJ, et al. Cellular and synaptic mechanisms of anti-NMDA receptor encephalitis[J]. J Neurosci, 2010, 30(17):5866-5875.
[7]
Irani SR, Bera K, Waters P, et al. N-methyl-D-aspartate antibody encephalitis:temporal progression of clinical and paraclinical observations in a predominantly non-paraneoplastic disorder of both sexes[J]. Brain, 2010, 133(Pt 6):1655-1667.
[8]
Zuliani L, Graus F, Giometto B, et al. Central nervous system neuronal surface antibody associated syndromes:review and guidelines for recognition[J]. J Neurosurg Psychiatry, 2012, 83(3):638-645.
[9]
Titulaer MJ, McCracken L, Gabilondo I, et al. Treatment and prognostic factors for long-term outcome in patients with anti-NMDA receptor encephalitis:an observational cohort study[J]. Lancet Neurol, 2013, 12(2):157-165.
