儿童原发性局灶节段肾小球硬化症62例临床治疗及预后分析

Clinical treatment and prognosis in children with focal segmental glomerular sclerosis in 62 cases

目的总结分析儿童原发性局灶节段肾小球硬化症(FSGS)临床资料,进一步分析原发性FSGS治疗及预后。方法对2007年6月至2012年6月首都医科大学附属北京儿童医院确诊的62例原发性FSGS患儿临床特点、病理类型、治疗方案与预后进行回顾性分析。结果 62例首发症状以浮肿伴蛋白尿多见,达49例(79.0%)。合并疾病中,上呼吸道感染35例(56.5%)。肾活检病理结果示非特殊型45例(72.6%),治疗后完全缓解13例(28.9%)、部分缓解15例(33.3%)、无效17例(37.8%);顶端型9例(14.5%),治疗后完全缓解5例(55.6%)、部分缓解3例(33.3%)、无效1例(11.1%);细胞型8例(12.9%),治疗后部分缓解2例(25.0%),无效6例(75%)。顶端型较非特殊型缓解率高(P<0.05);非特殊型缓解率较细胞型高(P<0.05)。治疗:泼尼松+环孢素组(P+Cs A组)完全缓解率36.0%(9/25),有效率76.0%(19/25);泼尼松+他克莫司组(P+TAC组)完全缓解率50.0%(6/12),有效率83.3%(10/12);泼尼松+环磷酰胺组(P+CTX组)完全缓解率12.0%(3/25),有效率36.0%(9/25)。P+Cs A组、P+TAC组缓解率明显好于P+CTX组(P<0.05);而P+Cs A组及P+TAC组缓解率差异无统计学意义(P>0.05)。病理类型及治疗方案选择与缓解率相关(r=0.142、0.327,P<0.05)。经Logistic回归发现血压升高与持续大量蛋白尿为疾病预后不良危险因素(P<0.05)。结论 FSGS缓解率与病理类型及治疗方案有关。积极应用多种免疫抑制剂联合治疗对缓解具有一定作用,其中联合TAC或Cs A缓解效果好。持续大量蛋白尿及血压升高为预后不良两个危险因素。

Objective To investigate the clinical and pathological characteristics of focal segmental glomerular sclero- sis （FSGS） in children, and to evaluate the associated factors of clinical outcome and the efficacy of treatment. Methods The clinical and pathological characteristics, the associated factors of clinical outcome and the efficacy of treatment were analyzed retrospectively in 62 children with FSGS from Jun. 2007 to Jun. 2012. Results Based on clini- cal and pathological classification： 49 patients with edema and proteinuria（79.0%）, 25 patients had hematuria（40.3%）, 5 patients had oliguria（8.1%）, 15 patients had hypertension（24.2%）, and 7 patients had acute renal injury（ 11.3%）.Re- nal pathology revealed 45 patients were of not-otherwise-specified type （NOS）（72.6%）, 9 patients with glomerular tip lesion type （Tips） （14.5%）, and 8 patients of cellular type （Cellular）（12.9%）. The complete remission rate of NOS was 28.9% and the partial remission rate was 33.3%, and no remission rate was 37.8%. The complete remission rate of Tips was 45.6% and the partial remission rate was 33.3%, and no remission rate was 11.1%. The complete remission rate of Cellular was 25% and no remission rate was 75%. The prognosis was correlated with the pathological characteristics and the therapy plan （r = 0.142,0.327, respectively, P 〈 0.05 ）.Of these FSGS patients, 12% patient experienced remission af-ter prednisone and cyclophosphamide pulse therapy （P ＋ CTX） and the total efficacy was 40%, which was statisti- cally lower than that of children who received prednisone and cyclosporin A （P ＋ CsA）or prednisone and tacrolimus （P ＋ TAC） （36.0% and 76.0% or 50.0%and 83.3%, respectively）.No signficant statistical difference in prognosis was found in patients with P ＋ CsA or P ＋ TAC group （P 〉 0.05 ）.High blood pressure and persistent large proteinuria were harmful to the prognosis （P 〈 0.05 ）, rather than age, gender, pathological type or treatment plan. Conclusion The prog- nosis is correlated with pathological type and treatment plan.Choosing the optimal treatment plan will be helpful for prog- nosis ; P ＋ CsA or P ＋ TAC treatment is better than P ＋ CTX pulse therapy. High blood pressure and persistent large pro- teinuria are harmful to the prognosis.