Sirolimus for the Treatment of Complicated Vascular Anomalies in Children
Sirolimus for the Treatment of Complicated Vascular Anomalies in Children
复制标题
DOI：
10.1002/pbc.23124
复制
发表时间：
2011-12-01
影响因子：
3.2
通讯作者：
Adams, Denise M.
中科院分区：
文献类型：
--
作者：
Hammill, Adrienne M.;Wentzel, MarySue;Adams, Denise M.
背景资料。血管异常包括各种不同的诊断。虽然婴儿血管瘤很常见，但这些情况中的大多数都相当罕见，还没有得到广泛的研究。其中一些病变虽然是良性的，但可能会损害重要结构，使其变形，甚至会危及生命。血管肿瘤，如Kaposi型血管内皮瘤(KHE)和复杂的血管畸形已被证明特别难以治疗。程序。在这里，我们回顾评估了一系列患有复杂的、危及生命的血管异常的患者，这些患者在多个其他治疗失败后，在两个中心接受了mTOR抑制剂西罗莫司的同情治疗。结果。这些患者的临床状况有显著改善，副作用可耐受。结论。西罗莫司对患有危及生命的血管畸形的患者似乎是有效和安全的，并且是治疗这些疾病的重要工具。这些发现目前正在第二阶段的安全性和有效性试验中得到进一步评估。儿科血癌杂志2011；57：1018-1024。(C)2011年Wiley-Liss，Inc.
Background. Vascular anomalies comprise a diverse group of diagnoses. While infantile hemangiomas are common, the majority of these conditions are quite rare and have not been widely studied. Some of these lesions, though benign, can impair vital structures, be deforming, or even become life-threatening. Vascular tumors such as kaposiform hemangioendotheliomas (KHE) and complicated vascular malformations have proven particularly difficult to treat. Procedure. Here we retrospectively evaluate a series of six patients with complicated, life-threatening vascular anomalies who were treated with the mTOR inhibitor sirolimus for compassionate use at two centers after failing multiple other therapies. Results. These patients showed significant improvement in clinical status with tolerable side effects. Conclusions. Sirolimus appears to be effective and safe in patients with life-threatening vascular anomalies and represents an important tool in treating these diseases. These findings are currently being further evaluated in a Phase II safety and efficacy trial. Pediatr Blood Cancer 2011; 57: 1018-1024. (C) 2011 Wiley-Liss, Inc.