检索历史 清除

目的:分析系统性红斑狼疮(SLE)合并血栓性微血管病(TMA)患儿的临床特征，并通过其治疗及随访情况明确患儿的临床结局及相关危险因素。方法:单中心回顾性病例对照研究。选取2017年1月至2023年1月首都医科大学附属北京儿童医院确诊为SLE合并TMA的患儿为TMA组，并选取SLE未合并TMA患儿作为对照组。根据TMA组患儿治疗后的预后情况进一步分为预后良好组及预后不良组。收集患儿年龄、性别、SLE疾病活动、诊断时及血栓时的临床表现、实验室检查、治疗策略和预后及随访结果。计数资料比较采用 χ2检验和 Z检验。计量配对资料比较采用 t检验。采用Fisher′s精确概率法比较2组分类变量间的差异。采用单因素Logistic回归分析对预后不良危险因素进行分析。 结果:TMA组29例，TMA发生率占SLE患儿的2.53%；对照组33例。诊断TMA年龄为13岁5个月(9岁1个月5 d至17岁4个月)，常见临床表现依次为肾脏受累(28例，96.55%)，血液系统受累(26例，89.66%)，浆膜腔积液(17例，58.62%)，皮疹(13例，44.82%)，神经系统受累(12例，41.38%)。TMA组发生胸膜炎或心包炎、肾脏受累及神经系统受累多于对照组(17例比3例、28例比10例、12例比3例)；TMA组的面部皮疹及关节炎表现均低于对照组(13例比25例、4例比17例)，差异均有统计学意义(均 P<0.05)。TMA组SLE疾病活动指数评分[(24.14±9.42)分]明显高于对照组[(10.18±9.42)分]，差异有统计学意义( t＝3.233， P<0.05)。TMA组患儿的血红蛋白水平、血小板、补体C3水平均明显低于对照组，双链DNA抗体、乳酸脱氢酶、D二聚体、尿素、肌酐、铁蛋白水平、尿蛋白定量均明显高于对照组，差异均有统计学意义(均 P<0.05)。TMA组中5例ADAMTS13活性降低，5例补体C5b9明显增高。TMA组中共15例(51.72%)患儿行肾活检，其中13例合并肾TMA。TMA组中28例(96.6%)患儿接受激素治疗，17例患儿接受血浆置换，12例联合免疫抑制剂及生物制剂治疗，19例(65.5%)好转，10例(34.5%)因病情恶化放弃治疗。预后良好组的尿素水平及外周血破碎红细胞百分率明显低于预后不良组[(13.18±4.39) mmol/L比(21.16±10.14) mmol/L， t＝2.975， P＝0.006；8/17(47.06%)比7/7(100%)， χ2＝5.929， P＝0.015]。单因素Logistic回归分析显示，破碎红细胞、ADAMTS13活性及尿素均为预后不良的独立危险因素(均 P<0.05)。 结论:对于中重度疾病活动的SLE患儿，尤其是以溶血性贫血、血小板减少、肾功能异常为突出表现者，需警惕合并TMA的风险，早期诊断、及早制定诊疗策略至关重要。

Objective:To analyze the clinical characteristics of children with systemic lupus erythematosus (SLE) combined with thrombotic microangiopathy (TMA), and clarify the clinical outcomes and related risk factors of pediatric patients through their treatment and follow-up.Methods:This was a single-center retrospective case-control study. Children diagnosed with SLE combined with TMA between January 2017 and January 2023 at Beijing Children′s Hospital, Capital Medical University, were selected as the TMA group, and SLE children without TMA were selected as the control group.According to the prognosis, children in the TMA group were further divided into the good prognosis group and the poor prognosis group.The data of the children were collected, including age, gender, SLE disease activity, clinical presentations at the time of diagnosis and at the time of thrombosis, laboratory examinations, treatment strategies, prognosis, and follow-up results.The chi-square test and Z-test were used for comparison of count data.The t-test was used for comparison of metrological pairing data.The Fisher′s exact test was used to compare the differences between the 2 groups in categorical variables.The univariate Logistic regression was used to analyze the risk factors of poor prognosis. Results:There were 29 cases in the TMA group, and the incidence of TMA accounted for 2.53% of SLE patients; 33 cases were in the control group.The age at diagnosis of TMA was 13 years and 5 months (ranging from 9 years, 1 month and 5 days to 17 years and 4 months).The common clinical manifestations in order of prevalence were renal involvement (28 cases, 96.55%), hematologic involvement (26 cases, 89.66%), serous effusion (17 cases, 58.62%), rash (13 cases, 44.82%), and neurologic involvement (12 cases, 41.38%).Pleurisy or pericarditis, renal involvement and neurological involvement occurred more often in the TMA group than in the control group (17 cases vs.3 cases, 28 cases vs.10 cases, 12 cases vs.3 cases), and the TMA group showed less facial rash and arthritis than the control group (13 cases vs.25 cases, 4 cases vs.17 cases), and the differences were statistically significant (all P<0.05).The Systemic Lupus Erythematosus Disease Activity Index score in the TMA group [(24.14±9.42) scores] was significantly higher than that in the control group [(10.18±9.42) scores], and the difference was statistically significant ( t=3.233, P<0.05).The hemoglobin level, platelet count, and complement C3 level of the children in the TMA group were significantly lower than those in the control group, whereas the double stranded DNA antibody, lactate dehydrogenase, D-dimer, urea, creatinine, ferritin level, and urine protein quantitation were significantly higher than those in the control group, and the differences were statistically significant (all P<0.05).In the TMA group, 5 cases had decreased ADAMTS13 activity, and 5 cases had significantly increased complement C5b9.A total of 15 cases (51.72%) in the TMA group underwent renal biopsy, and 13 of them had combined renal TMA.In the TMA group, 28 patients (96.6%) received hormone therapy, 17 patients received plasma exchange, and 12 patients were treated with immunosuppressants and biologics; 19 patients (65.5%) improved, and 10 patients (34.5%) gave up the treatment due to deterioration of the disease.The urea level and peripheral blood fragmented erythrocyte rate in the good prognosis group were significantly lower than those in the poor prognosis group [(13.18±4.39) mmol/L vs.(21.16±10.14) mmol/L, t=2.975, P=0.006; 8/17 (47.06%) vs.7/7 (100%), χ2=5.929, P=0.015].The univariate Logistic regression analysis showed that the fragmented erythrocyte, ADAMTS13 activity and urea were the independent risk factors for poor prognosis (all P<0.05). Conclusions:SLE patients with moderate-to-severe disease activity, especially children with hemolytic anemia, thrombocytopenia, and renal dysfunction as prominent manifestations, should be alert to the risk of TMA.Early diagnosis and treatment are crucial.