Objective: To observe the efficacy and prognosis of cladribine (2-CdA) combined with cytarabine (Ara-C) regimen in the treatment of relapsed refractory Langerhans cell histiocytosis (LCH) in children.
Methods: Nine patients with relapsed refractory LCH treated with the 2-CdA combined with Ara-C regimen in the Department of Hematology and Oncology of Wuhan Children's Hospital from July 2014 to February 2020 were retrospectively analyzed, and the efficacy and disease status were evaluated according to the Histiocyte Society Evaluation and Treatment Guidelines (2009) and the Disease Activity Score (DAS), the drug toxicity were evaluated according to the World Health Organization(WHO) grading criteria for chemotherapy. All patients were followed up for survival status and disease-related sequelae.
Results: Before the treatment combining 2-CdA and Ara-C, 7 of 9 patients were evaluated as active disease worse (ADW), and 2 as active disease stable (ADS) with a median disease activity score of 8 (4-15). Of 9 patients, 6 cases achieved non active disease (NAD) and 3 achieved active disease better (ADB) with a median disease activity score of 0 (0 to 5) after 2-6 courses of therapy. All 9 patients experienced WHO grade IV hematologic toxicity and 3 patients had hepatobiliary adverse effects (WHO grade I~II) after treatment. The median follow-up time was 31(1 to 50) months with all 9 patients survived, 3 of the 9 patients experienced sequelae to the disease with 2 combined liver cirrhosis as well as cholestatic hepatitis and 1 with oral desmopressin acetate tablets for diabetes insipidus.
Conclusion: 2-CdA combined with Ara-C is an effective regimen for the treatment of recurrent refractory LCH in children, and the main adverse effect is hematologic toxicity, which is mostly tolerated in children. Early treatment with this regimen may be considered for patients with multisystem LCH with risky organ involvement who have failed first-line therapy and for patients with relapse.
题目: 克拉屈滨联合阿糖胞苷治疗儿童复发难治性朗格汉斯细胞组织细胞增生症.
目的: 观察克拉屈滨(Cladribine,2-CdA)联合阿糖胞苷(Ara-C)方案治疗儿童复发难治性朗格汉斯细胞组织细胞增生症(LCH)的疗效及预后.
方法: 回顾性分析2014年7月至2020年2月于武汉儿童医院血液肿瘤科接受2-CdA联合Ara-C方案治疗的复发难治性LCH患儿9例,根据国际组织细胞协会LCH评估指南以及疾病活动度评分(DAS)对疗效以及疾病状态进行评估,根据世界卫生组织(WHO)化疗药物不良反应分级标准进行药物不良反应评估,随访患者生存状态以及疾病相关后遗症.
结果: 在接受2-CdA联合Ara-C方案治疗前,9例患者中有7例评估为活动性疾病进展(ADW),2例评估为活动性疾病稳定(ADS),中位DAS为8分(4-15分)。经过(2-6)个该疗程治疗后,有6例患者达到无活动性疾病(NAD),3例达到活动性疾病好转(ADB),中位DAS为0分(0-5分)。9例患者在治疗后均出现血液系统不良反应(WHO IV级),3例患者出现肝胆系统不良反应(WHO I-II级)。中位随访时间31(1-50)个月,9例患者均存活,3例出现疾病相关后遗症,2例合并肝硬化以及胆汁淤积性肝炎、1例因中枢性尿崩症口服醋酸去氨加压素片.
结论: 2-CdA联合Ara-C是治疗儿童复发难治性LCH的有效方案,主要药物不良反应为血液学毒性,儿童多可耐受。对于一线治疗失败的多系统LCH伴危险器官受累患者以及复发患者可考虑尽早使用2-CdA联合Ara-C方案治疗.
Keywords: Langerhans cell histiocytosis; children; cladribine.