[Effectiveness and safety of rituximab for children with autoimmune diseases of the nervous system]

Objective: To assess the effectiveness and safety of rituximab in Chinese children with autoimmune diseases of the nervous system. Method: An ambispective cohort study enrolled patients with refractory and(or) relapse autoimmune diseases of nervous system from June 2010 to June 2016 in Peking University First Hospital.These patients failed to respond to steroids and(or)intravenous immunoglobulin (IVIG) were treated with rituximab and seen for follow-up visits once every 3 months.The effectiveness was assessed by modified Rankin scale (mRs) and the annualized relapse rate.B cell was repeatedly counted after the treatment.Side effects attributed to rituximab were recorded.Paired rank test and chi-square test were used to compare the mRs score and the recurrence rate (time/year) before and after the treatment. Result: A total of 38 patients (15 males and 23 females) with mean age of (6±4) years were treated with rituximab.Among those patients, 4 cases were in multiple sclerosis, 5 in neuromyelitis optica, 6 in opsoclonus myoclonus syndrome, 9 in myasthenia gravis, and 14 in autoimmune encephalitis and other nervous system autoimmune diseases.The course of the disease before rituximab treatment was from two months to 7.25 years, with the average of (21±19) months.The patients had been followed up for 2-52 months. The mRs score and recurrence rate of 38 patients before receiving rituximab was 3 points (3, 4) and 2.56 (1.80, 4.75) times per year, respectively, while patients after receiving rituximab were mRs score of 0 (0, 2) and had a recurrence rate of 0 (0, 0.17) per year.There was statistical difference before and after treatment (Z=-4.51 and -4.71, P<0.01). Rituximab had a definite benefit in 23 patients, probable benefit in 2 patients, possible benefit in 3 patients, no benefit in one patient, and the disease worsened in 2 patients.Therefore the total effective rate was 74%, except for 6 undetermined cases because of the short follow-up time, and one patient withdrew from the study due to allergic reaction.During the follow-up, only one patient with severe allergy gave up the rituximab treatment. And only one patient was found severe infection with Pneumocystis carinii pneumonia. Conclusion: Rituximab is an effective and safe treatment strategy for patients with refractory and relapse autoimmune diseases of CNS, especially in neuromyelitis optica and myasthenia gravis.The adverse events including infection and allergy during infusion are not common.