两种方案治疗182例郎格汉斯细胞组织细胞增生症的历史对照研究

发布时间：2018-06-21 20:29

  本文选题：组织细胞增生症 + 郎格汉斯细胞　； 参考：《中国循证儿科杂志》2017年02期

【摘要】：目的比较复旦大学附属儿科医院(我院)CHFU-LCH 2006方案(简称2006方案)和CHFU-LCH 2012方案(简称2012方案)治疗郎格汉斯细胞组织细胞增生症(LCH)患儿的疗效和不良反应。方法 2006年1月1日至2012年11月31日在我院接受2006方案治疗的LCH初治患儿纳入2006组,2012年12月1日至2015年12月31日在我院接受2012方案治疗的LCH初治患儿纳入2012组。两组均经病理确诊LCH,排除治疗6周内自动终止治疗者。每组进一步分为单系统LCH(SS-LCH)和多系统LCH(MS-LCH)亚组。所有患儿随访至2017年3月31日。治疗有效为无活动性病变或活动性病变好转。以Kaplan-Meier法计算5年预计总生存率(OS)和无病生存率(EFS)。不良反应根据WHO急性和亚急性毒性反应分级标准分为0~4级。比较两组治疗6和12周有效率,恶化、复发和死亡情况,5年预计OS、EFS和不良反应发生情况。结果 96例患儿进入2006组,男64例,女32例,中位年龄3.4岁,中位随访时间6.9年;86例患儿进入2012组,男59例,女27例,中位年龄2.9岁,中位随访时间4.0年。两组性别、诊断年龄、临床分型和危险器官受累(RO+)情况差异无统计学意义。(1)2006组和2012组比较,SS-LCH、MS-LCH亚组治疗6、12周,有效率和复发率差异均无统计学意义。(2)2006组和2012组MS-LCH亚组分别有4例和5例退出方案,转入其他挽救方案,分别有5例和4例死亡。(3)两组MSLCH患儿共93例,其中2岁5年预计EFS和OS均明显低于≥2岁患儿[EFS:(41.9±8.1)%vs(62.6±7.5)%,OS:(80.8±6.2)%vs(98.0±2.0)%],P均0.05;RO+患儿5年预计EFS和OS低于RO-患儿[EFS:(37.4±8.0)%vs(66.0±7.3)%,OS:(80.4±6.3)%vs(98.0±2.0)%],P均0.05;RO-患儿2岁和≥2岁5年预计EFS和OS差异无统计学意义;6周治疗无效患儿5年预计EFS低于6周治疗有效患儿[(33.1±7.9)%vs(70.8±7.2)%],P0.05。(4)2006组和2012组SS-LCH亚组5年预计EFS分别为(84.8±5.3)%和(86.7±5.6)%,5年预计OS均为100%;MS-LCH亚组5年预计EFS分别为(50.0±7.1)%和(53.2±10.0)%,5年预计OS分别为(90.0±4.1)%和(90.6±4.5)%;差异均无统计学意义。(5)2006组MS-LCH亚组化疗相关3/4级不良反应发生率(50.0%,25/50)高于2012组(23.3%,10/43),P=0.008 0。结论 CHFULCH 2012方案与2006方案疗效未发现有差别,化疗相关严重不良反应较轻,MS-LCH的5年EFS仍不满意。RO+和治疗6周反应情况是MS-LCH的重要预后影响因素。
[Abstract]:Objective to compare the efficacy and side effects of CHFU-LCH 2006 (2006) and CHFU-LCH 2012 (2012) in the treatment of Langerhans cell histiocytosis in children with Langerhans cell histiocytosis. Methods from January 1, 2006 to November 31, 2012, 2006 children with initial treatment received 2006 regimen in our hospital were enrolled in the study, and 2012 patients received 2012 regimen in our hospital from December 1, 2012 to December 31, 2015. LCHs were confirmed by pathology in both groups, excluding those who were automatically terminated within 6 weeks of treatment. Each group was further divided into single system LCHS-LCHS-LCH and multi-system LCHS-LCH subgroups. All children were followed up until March 31, 2017. The effective treatment was inactive lesions or improved active lesions. The overall and disease-free survival rates were calculated by Kaplan-Meier method. Adverse reactions were classified as 0 or 4 according to WHO classification criteria for acute and subacute toxic reactions. The efficacy, exacerbation, recurrence and death were compared between the two groups at 6 and 12 weeks. The incidence of EFS and adverse reactions were predicted for 5 years. Results 96 children were enrolled in group 2006 (64 males, 32 females, median age 3.4 years). The median follow-up time was 6.9 years. 86 patients (59 males and 27 females) were enrolled in the 2012 group. The median age was 2.9 years. The median follow-up time was 4.0 years. There was no significant difference in sex, diagnostic age, clinical type and risk organ involvement (RO) between the two groups. There was no significant difference between the two groups in the treatment of SS-LCHMS-LCH subgroup for 6 to 12 weeks. There was no significant difference in effective rate and recurrence rate. There were 4 cases in MS-LCH subgroup in 2006 and 5 cases in MS-LCH subgroup in 2012 group, and they were transferred to other rescue schemes, 5 cases and 4 cases died respectively. 93 cases of MSLCH children in two groups were treated with MS-LCH subgroup, and 93 cases with MS-LCH subgroup were treated with MS-LCH subgroup 2012. Both EFS and OS were significantly lower in 2 years old and 5 years old than those in children 鈮，

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