Chinello Matteo, Di Carlo Daniela, Olivieri Francesca, Balter Rita, De Bortoli Massimiliano, Vitale Virginia, Zaccaron Ada, Bonetti Elisa, Parisi Alice, Cesaro Simone
Pediatric Hematology Oncology, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
Mother and Child Department, University of Verona.
Mediterr J Hematol Infect Dis. 2018 Jul 1;10(1):e2018043. doi: 10.4084/MJHID.2018.043. eCollection 2018.
Kaposiform Hemangioendothelioma (KHE) is a rare vascular tumour of the infancy and the first decade of life. It is locally aggressive and potentially life threatening when associated with consumptive coagulopathy, known as Kasabach-Merritt syndrome (KMS). No consensus or guideline for the therapy has been reached because of the lack of prospective trials, and the different standard care suggestions are based on retrospective case series.
We report the case of a 9-month-old male with KHE and KMS in which the initial response, obtained with prednisone and vincristine, was subsequently consolidated and strengthened by long-term treatment with sirolimus, a mTOR inhibitor. A summary of the published data is presented as well.
The inhibition of mTOR pathway represents the most important therapeutic innovation introduced in the last few years for KHE. Our case shows the effectiveness and good tolerance of long-term therapy with sirolimus.
卡波西样血管内皮瘤（KHE）是一种罕见的发生于婴儿期及儿童早期的血管肿瘤。它具有局部侵袭性，当与消耗性凝血病相关时，即卡萨巴赫-梅里特综合征（KMS），可能危及生命。由于缺乏前瞻性试验，目前尚未达成治疗的共识或指南，不同的标准治疗建议基于回顾性病例系列。
我们报告了一名9个月大患有KHE和KMS的男性病例，最初使用泼尼松和长春新碱获得了缓解，随后通过mTOR抑制剂西罗莫司的长期治疗得以巩固和加强。同时还呈现了已发表数据的总结。
mTOR通路的抑制是近年来KHE治疗中最重要的治疗创新。我们的病例显示了西罗莫司长期治疗的有效性和良好耐受性。
