［Abstract］　Objective　To analyze the clinical features and treatment outcomes of anti-melanoma differentiation-associated gene 5(MDA5) antibody-positive juvenile dermatomyositis(JDM). Methods　The clinical data of 487 pediatric patients with JDM who were admitted to the Department of Rheumatology of Beijing Children′s Hospital, Capital Medical University from June 2015 to February 2022 were collected, and among the pediatric patients, 41 cases(8.42%) were positive for anti-MDA5 antibody. The clinical characteristics, disease evaluation, therapeutic drugs and disease outcome of the pediatric patients were summarized and analyzed. Results　In the 41 cases of JDM with positive anti-MDA5 antibody, the most common clinical manifestation was abnormal skin and mucous membrane, followed by muscle weakness, and fever and arthritis were also common, and some pediatric patients had respiratory symptoms and skin ulcers. The majority of Cutaneous Assessment Tool(CAT) scores were 3 to 5 points. The scores of Childhood Myositis Assessment Scale(CMAS) were (33.88±9.57)points. The incidence rate of interstitial lung disease(ILD) was 87.80%, and 12.20% of the pediatric patients developed rapid progressive ILD(RP-ILD). Subcutaneous calcification occurred in 5 cases(12.20%) during the course of the disease. There were 90.24% of the anti-MDA5 antibody-positive JDM patients had high-risk physical signs. Intravenous methylprednisolone(IVMP)(63.41%), intravenous immunogloblin(IVIG)(68.29%) and oral cyclosporine A(65.85%) were the main treatment methods. Nine pediatric patients(21.95%) were orally administered tofacitinib. There were 75.61% of the pediatric patients presenting with a single course of the disease, and 19.51% with recurrent courses, and 4.88% with persistent active courses. The complete clinical response rates at 1, 2, 3 and 4 years were 65.79%(25/38), 65.38%(17/26), 63.64%(14/22) and 70.59%(12/17), respectively. The five-year clinical complete remission rate was 14.29%(2/14), and the two-year disease recurrence rate was 19.23%(5/26). During the 2-year follow-up, 95.00%(19/20) of the pediatric patients′ lung lesions were completely absorbed. At 3, 6, 9 and 12 months of follow-up, the median dose of prednisone was 1.40 mg/kg, 1.00 mg/kg, 0.71 mg/kg and 0.60 mg/kg, respectively, and the median duration of glucocorticoid discontinuation was 69 months. Until February 2022, glucocorticoids were discontinued in 15 of 41 cases(36.59%) and all drugs were discontinued in 9 cases(21.95%). Conclusion　Anti-MDA5 antibody-positive JDM is a special type of JDM, which mainly presents with characteristic rash, muscle weakness, fever and arthritis, and ILD is the main hazard. The complete clinical remission rate of the disease is still low, and the recurrence rate is relatively high.]]> 2024/9/29 12:29:45 ZHANG Junmei, XUE Yuan, KUANG Weiying, LI Chao, DENG Jianghong, TAN Xiaohua, LI Shipeng, LI Caifeng 6true ［Abstract］　Objective　To analyze the efficacy of infliximab in treatment of intestinal Behçet′s disease in children. Methods　The clinical data of 10 pediatric patients with intestinal Behçet′s disease who were admitted to the Department of Rheumatology of Beijing Children′s Hospital, Capital Medical University from January 2017 to December 2020 were retrospectively analyzed. All the pediatric patients were treated with infliximab more than 3 times. The involvement of digestive tract and other systems in the pediatric patients with intestinal Behçet′s disease, the treatment of hormones and immunosuppressants, the application of infliximab and follow-up results were explored. Results　Among the 10 pediatric patients, 7 cases were male and 3 cases were female. The age of the pediatric patients with onset was (8.18±3.66)years, and the median duration of disease was 3(1-72)months. All the 10 pediatric patients had digestive symptoms, including diarrhea in 6 cases, abdominal pain in 5 cases, bloody stool in 3 cases, vomiting in 1 case, abdominal distension in 1 case, and intestinal perforation in 3 cases. Digestive tract ultrasound and endoscopy indicated that ileocecal ulcer was the most common. There were 3 cases with concomitant vascular involvement. The dose of infliximab was 3-5 mg/(kg·per time). The duration of infliximab treatment was (20.9±9.5)months.The median follow-up time of the 10 pediatric patients was 17.5(14-54)months. Nine pediatric patients received methylprednisolone pulse therapy. The initial dose of prednisone was 1.6-2.0 mg/(kg·d). Methotrexate was used in 8 cases, cyclosporine A in 5 cases, thalidomide in 5 cases, and mycophenolate mofetil in 1 case. After treatment, all the pediatric patients had no clinical symptoms, and their inflammatory indicators and gastrointestinal ultrasound were normal, and their condition was stable. Infliximab was discontinued in 6 cases. There were no allergic reactions or active infections during the infusion of infliximab. Conclusion　Infliximab is a well-tolerated and effective treatment for children with intestinal Behçet′s disease.]]> 2024/9/29 0:00:00 LI Chao, DENG Jianghong, WANG Jiang, KUANG Weiying, ZHANG Junmei, TAN Xiaohua, LI Shipeng, LI Caifeng 5true ［Abstract］　Objective　To evaluate the long-term prognosis of childhood-onset systemic lupus erythematosus(cSLE) after discontinuation of glucocorticoids(GC) therapy. Methods　The clinical data of 15 patients with cSLE who were diagnosed in Beijing Children′s Hospital, Capital Medical University from January 2006 to December 2020 were retrospectively analyzed. All the patients underwent long-term follow-up and their GC therapy was discontinued before the age of 18, and the patients were followed up for at least 1 year after discontinuation of GC therapy. Results　Of the 15 patients with cSLE, 4 cases were diagnosed with lupus nephritis and 3 with neuropsychiatric lupus. All the patients were treated with oral prednisone at a median dose of 1.7(1.1, 2.0)mg/(kg·d). The median time to reach lupus low disease activity state(LLDAS) was 30(18, 36)months, and the median time to reach clinical remission was 38(26, 46)months. All the patients maintained clinical remission and serologically normal［anti-double-stranded deoxyribonucleic acid(dsDNA) antibodies-negative and normal complement levels］ before discontinuation of GC therapy. The duration of GC therapy was 53(45, 82)months. After GC was discontinued, all the patients continued to take disease-modifying anti-rheumatic drugs(DMARDs). The median follow-up time after discontinuing GC therapy was 21(16, 44)months. One padiatric patient experienced a mild relapse at the 8th month after discontinuation of GC therapy. Conclusion　It is feasible for the cSLE patients to discontinue GC therapy after achieving long-term clinical remission. However, larger scale clinical studies are needed to further validate the feasibility of discontinuing GC therapy in the cSLE patients.]]> 2024/9/29 12:29:45 LI Shipeng, KUANG Weiying, DENG Jianghong, ZHANG Junmei, TAN Xiaohua, LI Chao, LI Caifeng 4true ［Abstract］　Objective　To study the treatment, follow-up and prognosis of pediatric patients with juvenile dermatomyositis(JDM), and to explore the factors affecting the recurrence of the disease. Methods　The clinical data of pediatric patients with JDM who were diagnosed and treated in the Department of Rheumatology of Beijing Children′s Hospital, Capital Medical University from June 2014 to June 2019 and were followed up for at least 6 months were retrospectively analyzed. Kaplan-Meier method was used to plot the survival curve, and Cox regression was used to analyze the factors affecting the recurrence of the disease. Results　Among the 192 pediatric patients with JDM, the ratio of males to females was 1∶1.46, and the median age of the patients with onset was 65.5(43.5, 98.8)months. The combination treatment of tofacitinib, thalidomide, and tocilizumab improved the disease in some refractory pediatric patients with JDM. Kaplan-Meier survival curve showed that the median time of the pediatric patients to achieve complete clinical response was 12 months. The results of multivariate Cox regression analysis showed that long time from disease onset to treatment［RR(95%CI): 1.042(1.001-1.084), P=0.044］ was a risk factor of disease recurrence, and positive heliotrope rash［RR(95%CI):0.208(0.049-0.894), P=0.035］ at the time of disease onset was a protective factor of avoiding disease recurrence. Conclusion　Most pediatric patients with JDM can achieve complete clinical response after treatment, and some of them may experience the disease recurrence. The application of thalidomide, tofacitinib and tocilizumab may improve the prognosis of the pediatric patients with refractory JDM. The long time from disease onset to treatment is a risk factor of the disease recurrence.]]> 2024/9/29 12:29:45 WEN Xinran, ZHANG Junmei, KUANG Weiying, DENG Jianghong, TAN Xiaohua, LI Chao, LI Shipeng, LI Caifeng 3true ［Abstract］　Objective　To analyze the efficacy and safety of belimumab in treatment of lupus nephritis(LN) in children. Methods　The clinical data of 12 pediatric patients with LN who received the treatment of belimumab for 20 weeks in Department of Pediatrics, the People′s Hospital of Guangxi Zhuang Autonomous Region from July 1, 2021 to July 31, 2024 were retrospectively collected. The clinical manifestations, changes in immune and urinary system-related indicators, changes in disease activity, reduction of glucocorticoids and adverse drug reactions of the pediatric patients were analyzed before and after treatment. Results　Compared with those before belimumab treatment, the proportions of the pediatric patients with rash, fever, lupus nephritis and blood system damage decreased, and the dosage of glucocorticoids decreased, and the CD19⁺ cell count decreased, and the positive rate of anti-double stranded deoxyribonucleic acid(dsDNA) antibodies decreased, and 24-hour urinary protein quantification, urinary red blood cell count and urinary white blood cell count decreased after belimumab treatment, and the differences were statistically significant(P<0.05). There were statistically significant differences(P<0.01) in the Systemic Lupus Erythematosus(SLE) Disease Activity Index-2000(SLEDAI-2000) scores of the pediatric patients treated with belimumab at different time points(0, 2, 4, 8, 12, 16, 20 weeks), and the scores showed a downward trend with the extension of treatment time. Among the 12 pediatric patients with LN, 5 cases reached lupus low disease activity state(LLDAS) by the observation endpoint(20 weeks), and 3 cases reached clinical remission. During the study period, none of the pediatric patients had severe infection events and allergic reactions to belimumab. Conclusion　Belimumab helps to reduce SLEDAI-2000 scores and alleviate kidney injury in padiatric patients with LN, and helps to achieve LLDAS and clinical remission status, and helps to reduce the dosage of glucocorticoids to reduce adverse reactions.]]> 2024/9/29 12:29:45 1, DAI Yan¹, LI Xinye¹, LU Fuqing¹, ZHANG Diwen¹, WEN Xiaoling¹, WEN Zhihong¹, YOU Yanwu²]]> TANG Huihe¹, DAI Yan¹, LI Xinye¹, LU Fuqing¹, ZHANG Diwen¹, WEN Xiaoling¹, WEN Zhihong¹, YOU Yanwu² 2true ［Abstract］　Objective　To analyze the short-term efficacy of lymphoplasmapheresis(LPE) in treatment of 6 cases of severely active systemic lupus erythematosus(SLE) complicated with infections in children. Methods　The clinical data of 6 pediatric patients with severely active SLE complicated with infections who were treated in the People′s Hospital of Guangxi Zhuang Autonomous Region from January 2021 to January 2024 were analyzed. On the basis of traditional drug therapy, the 6 pediatric patients were treated with LPE, and their clinical therapeutic effect and the occurrence of adverse reactions were observed. Results　Compared with those before LPE treatment, peripheral blood leukocyte count and lymphocyte count did not change much, and platelet count, B cell count, natural killer(NK) cell count, anti-double-stranded deoxyribonucleic acid(DNA) antibodies level, anti-nuclear antibodies titer and immunoglobulin G(IgG) level, 24-hour urine protein quantitation and urinary red blood cell count all decreased to varying degrees, and the complement C3 increased significantly in the 6 pediatric patients after LPE treatment, and the content of hemoglobin decreased in 5 cases but increased in 1 case. No aggravation of vital organ involvement was observed in all the 6 pediatric patients, and all the affected organs gradually improved during the subsequent treatment. No adverse reactions occurred in all the 6 pediatric patients during LPE treatment. Conclusion　On the basis of traditional drug therapy, LPE treatment can obtain good clinical effect on severely active SLE complicated with infections in children in a short time.]]> 2024/9/29 0:00:00 1, SONG Kunling¹, MO Zhuning², WEI Jinshuang¹, LU Yuanfeng¹, WEN Zhihong¹, LI Hailan², DAI Yan¹]]> LU Fuqing¹, SONG Kunling¹, MO Zhuning², WEI Jinshuang¹, LU Yuanfeng¹, WEN Zhihong¹, LI Hailan², DAI Yan¹ 1true