Clinical Efficacy Analysis of 38 Pediatric Cases of Recurrent and Refractory Langerhans Cell Histiocytosis
10.19746/j.cnki.issn1009-2137.2025.06.041
- VernacularTitle:38例儿童复发难治性朗格汉斯组织细胞增生症的临床疗效分析
- Author:
Jie WANG
1
;
Kai-Lan CHEN
1
;
Bin WU
1
;
Lan-Nan ZHANG
1
Author Information
1. 华中科技大学同济医学院附属武汉儿童医院血液肿瘤科,湖北武汉 430016
- Publication Type:Journal Article
- Keywords:
Langerhans cell histiocytosis;
relapsed-refractory;
second-line treatment;
clofarabine
- From:
Journal of Experimental Hematology
2025;33(6):1814-1819
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To explore the efficacy and drug-related toxicity of pediatric patients with recurrent and refractory Langerhans cell histiocytosis(LCH).Methods:The clinical data of 38 children with refractory and recurrent LCH diagnosed in the Department of Hematology and Oncology of Wuhan Children's Hospital from January 2016 to June 2023 were retrospectively analyzed.The patients received three treatment regimens:regimen A was Cytarabine and Dexamethasone combined with Vindesine,regimen B was Cladribine with Cytarabine,and regimen C was Clofarabine monotherapy.The efficacy and safety of three second-line regimens were evaluated.Results:Thirty-eight children with refractory and recurrent LCH included 22 males and 16 females,with an age at diagnosis of 2.4(0.4-11.5)years.The median follow-up time was 5.5(1.5-8.5)years.Twenty-one children without risk organ involvement were treated with regimen A,and the 5-year overall survival(OS)rate was 100％,and event-free survival(EFS)rate was(85.7±8.8)％.Among the 17 children with risk organ involvement,11 cases were treated with regimen B,after 4 courses of treatment,6 cases achieved no active disease(NAD)and 5 children achieved active disease better(ADB),and 5-year OS rate reached 100％,and 5-year EFS reached 81.8％±11.6％;6 cases were treated with regimen C,and after 6 courses of treatment,4 cases achieved ADB and 2 cases achieved NAD.Children in group A had hematologic adverse reactions(WHO grade Ⅰ-Ⅲ),all children in group B had hematologic adverse reactions(WHO grade Ⅳ),and three cases had hepatobiliary adverse reactions(WHO grade Ⅱ),and children in group C had hematologic adverse reactions(WHO grade Ⅱ).Conclusion:The second-line treatment regimens for children with recurrent and refractory LCH based on the involvement of organs at risk has significant efficacy,and further expansion of the sample size and optimization of the treatment regimens are still needed to reduce long-term recurrence rates and toxicity of the treatment.
