儿童多脏器受累朗格汉斯细胞组织细胞增生症131例临床研究  

Clinical study of 131 children with multi-system Langerhans cell histiocytosis

作　　者：吴方方 高怡瑾[1] 潘慈[1] 陈静[1] 汤静燕[1] 

机构地区：[1]上海交通大学医学院附属上海儿童医学中心血液肿瘤科,200127

出　　处：《中华儿科杂志》2016年第5期349-353,共5页Chinese Journal of Pediatrics

摘　　要：目的分析多脏器受累的朗格汉斯细胞组织细胞增生症（MS—LCH）患儿的临床特点、治疗反应及预后相关因素。方法收集上海儿童医学中心血液肿瘤科2007年1月至2013年12月收治的131例MS—LCH患儿的病例资料。所有病例均采用上海儿童医学中心LCH方案（LCH-Ⅱ改良方案）化疗，采用Kaplan．Meier方法计算其总体生存率（0S）及无事件生存率（EFS），患儿组间EFS及0S差异行LogRank检验，多因素Cox回归分析其预后相关因素。结果131例患儿中男86例，女45例；中位发病年龄3岁（3个月-14岁）。6周诱导治疗后评估总有效率（完全缓解±好转）为79％（104／131），有危险器官受累者6周治疗后有效率为74％（48／65）。131例患儿3年EFS为（62±5）％，3年0s为（82±4）％。起病时年龄≤2岁、危险器官受累及6周治疗无效是影响LCH预后的重要因素，三组间3年os差异均有统计学意义（x^2=12．600、11．583、38．711，P=0．000、0．001、0．000）。对上述因素进一步采用多因素分析：危险器官受累及诱导治疗6周无效是影响MS—LCH患儿生存率的最主要因素（OR：12．352、14．356，P=0．001、0．000），起病时年龄≤2岁不是独立的危险因子（OR=1．013，P=0．207）。131例患儿复发或进展率28％（36／131），中位复发或进展时间为11（1—25）个月，其中有危险器官复发或进展者预后差，3年OS仅为（18±3）％。危险器官受累、6周诱导治疗无效者及较小年龄患儿更易发生病情进展或复发，差异均有统计学意义（x^2=15．747、7．289，Z=3．865；P=0．000、0．007、0．000）。结论危险器官受累及诱导治疗6周疗效不佳是影响MS-LCH患儿预后的最重要因素。再次诱导治疗可提高此部分患儿的生存率，有效的挽救方案是提高复发或进展患儿生存率的关键。Objective To analyze the clinical characteristics and treatment outcome of children with multi-system Langerhans cell histioeytosis （MS-LCH）. Method From January 2007 to December 2013, newly diagnosed patients with histopathologically-confirmed MS-LCH were enrolled in this retrospective study. All patients were treated on the Shanghai Children＇s Medical Center LCH protocol （ LCH- 11 modified protocol）. Survival was determined using the Kaplan-Meier method with differences between different groups compared using the Log-Rank test. Prognostic relevance of different parameters were analyzed by Cox proportional hazard model. Result Of the 131 patients （86 boys and 45 girls）, the median age was 3 years （range 3 months to 14 years）. Rapid response at week 6 was achieved in 79% （104/131） evaluable patients and 74% patients （48/65） with risk organ involvement. The 3-year event free survival （EFS） and 3-year overall survival （OS） for all cases were （62 ± 5 ） % and （ 82 ± 4 ） %. The 3-year OS was significantly different between age at diagnosis ≤ 2 years and 〉 2 years group. The 3-year OS was also significantly different between patients with and without risk organ involvement. The 3-year OS of patients who had rapid response at week 6 was significantly higher than that of those without rapid response （ X^2 = 12. 600,11. 583, 38. 711 ;P =0. 000,0. 001,0. 000）. Cox regression analysis showed that risk organ involvement and poor response at week 6 were the most important prognostic factors for patients with MS-LCH （ OR = 12. 352,14. 356;P = 0. 001,0. 000）. However, age was not the independent prognostic risk factor （ OR = 1. 013,P = 0. 207）. There were 36 patients （28% , 36/131 ） who experienced disease progression or relapse. The time to disease progression or relapse ranged from 1 to 25 months from the initial diagnosis （median 11 months）. Significantly lower OS （18 ± 3 ）% was observed in 20 patients with risk organ involvement at progression or relap

关 键 词：组织细胞增多症 郎格尔汉斯细胞 儿童 预后 

分 类 号：R725.9[医药卫生—儿科]