Clofarabine salvage therapy for refractory high-risk langerhans cell histiocytosis

Clofarabine salvage therapy for refractory high-risk langerhans cell histiocytosis
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DOI:
10.1002/pbc.24436
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发表时间:
2013-06-01
影响因子:
3.2
通讯作者:
Campbell, Patrick K.
Campbell, Patrick K.
中科院分区:
医学3区
文献类型:
--
作者:
Abraham, Allistair;Alsultan, Abdulrahman;Campbell, Patrick K.

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难治性多系统朗格汉斯细胞组织细胞增生症(LCH)的儿童患者尽管进行了积极的化疗,但预后仍很差。阿糖胞苷和克拉屈滨的挽救治疗已显示出作为有效治疗的前景,但与显著毒性相关。先前的报告描述了两名难治性LCH患者,他们对氯法拉滨单药治疗反应迅速,毒性最小。在这份报告中,我们描述了四名儿童难治性,风险器官阳性LCH谁与氯法拉滨治疗,并提供后续的两个先前报告的情况下。研究结果支持开展一项正式试验,评估氯法拉滨作为难治性LCH的一线挽救治疗。儿科血液癌症2013; 60:E19 E22。(c)2012 Wiley Periodicals,Inc.
Pediatric patients with refractory multisystem Langerhans cell histiocytosis (LCH) have a poor prognosis despite aggressive chemotherapy. Salvage therapy with cytarabine and cladribine has shown promise as an effective treatment but is associated with significant toxicity. A previous report described two patients with refractory LCH who had a rapid response to single-agent clofarabine with minimal toxicity. In this report, we describe four children with refractory, risk-organ-positive LCH who were treated with clofarabine and provide follow-up for the two previously reported cases. The results support development of a formal trial evaluating clofarabine as front-line salvage for refractory LCH. Pediatr Blood Cancer 2013; 60: E19E22. (c) 2012 Wiley Periodicals, Inc.