2′-Chlorodeoxyadenosine (2-CdA) as Salvage Therapy for Langerhans Cell Histiocytosis (LCH). Results of the LCH-S-98 Protocol of the Histiocyte Society
2′-Chlorodeoxyadenosine (2-CdA) as Salvage Therapy for Langerhans Cell Histiocytosis (LCH). Results of the LCH-S-98 Protocol of the Histiocyte Society
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DOI：
10.1002/pbc.22229
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发表时间：
2009-12-15
影响因子：
3.2
通讯作者：
Arceci, Robert J.
中科院分区：
文献类型：
--
作者：
Weitzman, Sheila;Braier, Jorge;Arceci, Robert J.
背景一项前瞻性11期组织细胞学会研究（LCH-S-98）评价了2-氯脱氧腺苷（2-CdA）单药治疗作为朗格汉斯细胞组织细胞增生症（LCH）挽救治疗的疗效。程序.对初始治疗无反应的低危和中危LCH患者以及低危慢性复发性LCH患者进行2-CdA治疗2 - 6个疗程后的反应和生存率评估。结果46例患者（55%）有危险器官受累;肺、肝、脾或造血系统（RO+），37例（45%）为RO-。22%的RO+患者反应良好，44%进展，62%的RO-患者反应，11%进展。RO+患者的2年预测生存率为48%，RO-患者为97%，RO+患者在非风险器官中再活化为100%，RO-患者在风险器官中再活化为67%。整个组的两年pSU为68%。73%对2-CdA反应不佳的患者死亡。65%的患者> 2岁，30%
Background. A prospective phase 11 Histiocyte Society Study, LCH-S-98, evaluated the efficacy of 2-chlorodeoxyadenosine (2-CdA) monotherapy as salvage therapy in Langerhans cell histiocytosis (LCH). Procedures. Patients with poor and intermediate risk LCH not responsive to initial therapy and patients with low-risk chronic recurrent LCH were evaluated for response and survival after treatment with 2-6 Courses of 2-CdA. Results. Forty-six patients (55%) had involvement of risk organs; lung, liver, spleen, or hematopoetic system (RO+), 37 (45%) were RO-. Twenty-two percent of RO+ patients had a good response while 44% progressed, 62% RO- patients responded, and 11% progressed. Two-year predicted Survival is 48% for RO+, 97% for RO- patients, 100% for RO+ patients reactivating in non-risk organs, 67% for RO- patients reactivating in risk organs. Two-year pSU for the entire group is 68%. Seventy-three percent of patients with a poor response to 2-CdA died. Sixty-five percent patients >2 years old and 30%