儿童难治性肾病的药物治疗研究

目的研究难治性肾病(RNS)单一强地松(PRED)用药,和与环磷酰胺(CTX)联合治疗及再加藻酸双酯钠(PSS)的三联用药的不同疗效、复发率和药物副反应。分析对比何种方案为优。方法将110例RNS患儿随机分成A、B、C三组。A组为PRED口服。B组为PRED与CTX联合治疗。C组为PRED、CTX、PSS三药联合治疗。总疗程均为12个月。结果1.A、B、C三组有效率分别为:20.7%、65.7%、91.3%。B、C组有效率高于A组40%以上,差异分别为高度显著性(P<0.01)和极显著性(0.01)。C组有效率高于B组且有显著性差异(P<0.05)。2.对勤复发类型:A、B、C三组有效率分别为25%、71.4%、95.2%。B、C组高于A组40%以上,差异有显著性(P<0.05)或极显著性(C、A间P<0.01)差异。C组有效率高于B组20%以上并有显著性差异(P<0.05)。3.对激素依赖类型,A、B、C三组有效率分别为22.2%、66.7%、92.9%。B、C组均高于A组40%以上。其中C、A间差异有极显著性(P<0.01),而B、A间差异有显著性(P<0.05)。4.对耐药病例:A、B、C三组有效率为12.5%、55.6%、81.8%。C、B组均高于A组40%以上。但A、C间差异有高度显著性(P<0.01),而B、A间差异无显著性(P>0.05)。5.耐药病例中不同病理类型三组疗效比较中,对非微小病变型(NMCNS),A组治疗6例均无效。B、C组有效率分别为50%和80%。但C、A组间差异有极显著性(P<0.01),而B、A组间无显著性差异(P=0.06)。6.A、B、C三组副反应发生率分别为62.1%、31.4%、13%。B、C组低于A组均有显著性差异(P<0.05)。C组副反应发生率低于B组,差异有显著性(P<0.05)。对完全缓解的RNS患儿停药一年,A、B、C三组复发率分别为50%(2/4)、20%(3/15)、11.5%(3/26)。结论对RNS患儿,用PRED和CTX联合用药比单用PRED的疗效有明显提高,特别是再加PSS的三联用药更具有提高疗效,降低复发率,减少副反应的优点。

Study of Drug Therapy of Children Refractory Nephrotic Syndrome

Objective To study and compare the therapeutic effects with three different therapeutic methods on children with refractory nephrotic syndrome (RNS), and to analyze which method is the best in treating children RNS. Methods Altogether 110 cases were randomly divided into three groups of A, B and C. The patients in group A received prednisone alone, group B received prednisone combined with CTX, and group C received combined prednisone-CTX and additional PSS. The total therapeutic course of the three groups was 12 months. Results 1. The effective rates of group A, B and C were 20.7%, 65.7% and 91.3% respectively. The effective rates of both group B and group C were 40% higher than that of group A respectively (P<0.01, P<0.01). The effective rate of group C was higher than that of group B (P<0.05). 2. The effective rates of frequent relapse cases in group A, B and C were 25.0%, 71.4% and 95.2% respectively; the effective rates of these cases in both group B and C were 40% higher than that of group A respectively (P<0.05, P<0.01); and the effective rate of group C was 20% higher than that of group B, and also showed significant difference. 3. The effective rates of the hormone-dependent cases in group A, B and C were 22.2%, 66.7% and 92.9% respectively, in which the effective rates of both group B and group C were 40% higher than that of group A respectively (P<0.05, P<0.01). 4. The effective rates of the patients which were drug-resistant in group A, B and C were 12.5%, 55.6% and 81.8% respectively. Furthermore, the effective rates of this type of patients in both group B and group C were 40% higher than that of group A respectively. 5. As to the different pathological type of the drug-resistant patients in these three groups, there were 6 NMCNS patients in group A who all had poor therapy effect; but the effective rates of the NMCNS patients in group B and group C were 50% and 80%, respectively. There was significant difference between group C and group A (P<0.01), but there was no difference between group B and group A (P>0.05). 6. The incidence rates of side effects in group A, B and C were 62.1% , 31.4% and 13.0% respectively; the incidence rates of side effects in both group B and group C were lower than that of group A respectively (P<0.05, P<0.05); and the incidence rate of side effects in group C was lower than that of group B (P<0.05). As to the complete remission patients after stopping therapy for one year, the recurrence rates of group A, B and C were 50%(2/4), 20%(3/15) and 11.5%(3/26) respectively. Conclusions Prednisone combined with CTX has the better therapeutic effect than prednisone alone on children with RNS. Furthermore, using three kind of drugs involving prednisone, CTX and PSS simultaneously can significantly improve the outcome, including the therapeutic effect, the relapse rate and side effects.