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Int J Cancer. 2017 Aug 15;141(4):848-855. doi: 10.1002/ijc.30775. Epub 2017 May 26.

Sirolimus for the treatment of progressive kaposiform hemangioendothelioma: A multicenter retrospective study.

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Division of Oncology, Department of Pediatric Surgery, West China Hospital of Sichuan University, Chengdu, China.
Pediatric Intensive Care Unit, Department of Critical Care Medicine, West China Hospital of Sichuan University, Chengdu, China.
Division of Oncology, Department of Pediatric Surgery, Children's Hospital of Fudan University, Shanghai, China.
Pediatric Intensive Care Unit, West China Second University Hospital, Sichuan University, Chengdu, China.
Department of Vascular & Interventional Radiology, Chengdu Women and Children's Central Hospital, Chengdu, China.
Department of Radiology, West China Hospital of Sichuan University, Chengdu, China.
Department of Pediatric Surgery, Chengdu Shangjin Nanhu Hospital, Chengdu, China.
Department of Pediatric Surgery, Shanghai Children's Hospital, Shanghai, China.
Vascular Biology Program and Department of Surgery, Boston Children's Hospital, Harvard Medical School, Boston, MA.


Kaposiform hemangioendothelioma (KHE) is an aggressive disease with high morbidity and mortality. The aim of this study was to retrospectively evaluate the efficacy and safety of sirolimus for the treatment of progressive KHE. A multicenter, retrospective cohort study was conducted in patients with progressive KHE treated with sirolimus. A total of 52 patients were analyzed. Thirty-seven (71%) patients exhibited Kasabach-Merritt phenomenon (KMP) and were significantly younger than the patients without KMP [95% confidence interval (CI), 14.39-41.61; p < 0.001]. Patients without KMP were all treated with sirolimus alone, whereas 21 KMP patients with severe symptoms received short-term combination therapy with prednisolone. Overall, 96% and 98% of patients showed improved relief of notable symptoms and/or improved complications at 6 and 12 months after treatment, respectively. After sirolimus treatment, significant decreases in mean severity scores occurred at 6 months (95% CI, 2.23-2.54, p < 0.001) and 12 months (95% CI, 1.53-1.90, p < 0.001). Compared to KMP patients, patients without KMP showed a response that was similar to but less pronounced during the 12 months of treatment (95% CI, 40.87-53.80; p < 0.001). For subgroup analysis of KMP patients, there were no significant differences in tumor shrinkage between those treated with combination therapy and those receiving sirolimus alone (95% CI, 18.11-25.02; p > 0.05). No patients permanently discontinued treatment due to toxicity-related events, and no drug-related deaths occurred. Sirolimus was effective and safe for the treatment of progressive KHE. Sirolimus may be considered as a first-line therapy or as part of a multidisciplinary approach for the treatment of KHE.


Kasabach-Merritt phenomenon; efficacy; kaposiform hemangioendothelioma; safety; sirolimus

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