Tang J J, Xu X J, Wang Y C, Bai S T, Wang L, Ni X L, Liu Y F
Department of Hematology and Oncology, Children's Hospital, First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, China.
Zhonghua Er Ke Za Zhi. 2021 Jan 2;59(1):37-41. doi: 10.3760/cma.j.cn112140-20200605-00584.
To analyze the clinical characteristics and long-term outcome of Langerhans cell histiocytosis with multisystem involvement (MS-LCH) in children, and to evaluate the efficacy of modified DAL-HX83/90 protocol. This retrospective study included 53 patients with MS-LCH admitted to the Department of Pediatric Hematology and Oncology, First Affiliated Hospital of Zhengzhou University from January 2011 to May 2019. Modified DAL-HX83/90 protocol was used in all patients as an initial treatment. The patients were divided into the group with (RO+) or without (RO-) risk organ involvement. The RO+group was further divided into two groups, as RO+Ⅰ group (lung involvement only) and RO+Ⅱ group (extra-pulmonary, with or without lung involvement). The clinical characteristics and the long-term outcome were summarized. Event-free survival (EFS) and overall survival (OS) curves were analyzed with Kaplan-Meier method. Univariate and multivariate analysis of prognostic factors including age, sex, risk organ involvement and response to 6-week induction were analyzed with Log-Rank test and Cox proportional hazards models. Among the 53 children with MS-LCH, 34 were male and 19 were female. The age of onset was 21 months (3 months-13 years). There 22 were in RO+group, with 12 in RO+Ⅰ group and 10 in RO+Ⅱ group, and 31 in RO-group. The follow-up period was 51 (12-144) months. The overall response rate of 6-week induction was 89% (47/53), and the recurrence rate was 30% (16/53). The 5-year EFS and OS were (67±6) % and (83±5) %, respectively. Univariate analysis showed that the 5-year EFS and OS of patients who responded well to 6-week induction chemotherapy were significantly higher than those who had no response ((76±6) % . 0, (88±4) % . (41±22) %, χ = 34.743, 10.608, both <0.05). The 5-year EFS and OS of RO-group were significantly higher than that of RO+group ((80±7) % . (49±10) %, (93±4) % . (70±10) %, χ=6.022, 4.793, both <0.05). And the 5-year EFS of RO+Ⅰ group was significantly higher than that of RO+Ⅱ group ((83±10) % . (10±9) %, χ=9.501, =0.002). While age and sex were not significantly associated with 5-year EFS and OS (all >0.05). Cox proportional hazard regression model showed that response to 6-week induction chemotherapy was the independent risk factor for EFS (=13.114, 95% 3.759-45.742, <0.01) and OS (=7.748, 95% 1.542-38.920, =0.013). Most of the children without risk organ involvement treated with modified DAL-HX83/90 protocol could achieve long-term survival. However, the children involved liver, spleen, or hematopoietic system had a high risk of disease progression and recurrence.
分析儿童多系统受累朗格汉斯细胞组织细胞增多症（MS-LCH）的临床特征及长期预后，并评估改良DAL-HX83/90方案的疗效。本回顾性研究纳入了2011年1月至2019年5月在郑州大学第一附属医院小儿血液肿瘤科住院的53例MS-LCH患者。所有患者均采用改良DAL-HX83/90方案作为初始治疗。将患者分为有（RO+）或无（RO-）危险器官受累组。RO+组进一步分为两组，即RO+Ⅰ组（仅肺部受累）和RO+Ⅱ组（肺外受累，有或无肺部受累）。总结临床特征及长期预后。采用Kaplan-Meier法分析无事件生存（EFS）和总生存（OS）曲线。采用Log-Rank检验和Cox比例风险模型对年龄、性别、危险器官受累及6周诱导缓解反应等预后因素进行单因素和多因素分析。53例MS-LCH患儿中，男34例，女19例。发病年龄为２１个月（3个月至13岁）。RO+组22例，其中RO+Ⅰ组12例，RO+Ⅱ组10例；RO-组31例。随访时间为51（12～144）个月。6周诱导缓解的总有效率为89%（47/53），复发率为30%（16/53）。5年EFS和OS分别为（67±6）%和（83±5）%。单因素分析显示，6周诱导化疗反应良好的患者5年EFS和OS显著高于无反应者（（76±6）%对（41±22）%，χ² = 34.743；（88±4）%对（70±10）%，χ² = 10.608，均P<0.05）。RO-组5年EFS和OS显著高于RO+组（（80±7）%对（49±10）%，χ² = 6.022；（93±4）%对（70±10）%，χ² = 4.793，均P<0.05）。RO+Ⅰ组5年EFS显著高于RO+Ⅱ组（（83±10）%对（10±9）%，χ² = 9.501，P = 0.002）。而年龄和性别与5年EFS和OS无显著相关性（均P>0.05）。Cox比例风险回归模型显示，6周诱导化疗反应是EFS（β = 13.114，95%CI 3.759～45.742，P<0.01）和OS（β = 7.748，95%CI 1.542～38.920，P = 0.013）的独立危险因素。多数无危险器官受累的儿童采用改良DAL-HX83/90方案治疗可获得长期生存。然而，累及肝脏、脾脏或造血系统的儿童疾病进展和复发风险高。
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