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. What's new? Kaposiform hemangioendothelioma (KHE) is an aggressive disease with high morbidity and mortality. Responses to standard therapies have been variable and unpredictable. In this study, the authors found that the drug sirolimus (rapamycin) provides a safe and effective treatment option for patients with progressive KHE. They caution, however, that the regimen should be tailored to individual patients. In cases complicated by Kasabach-Merritt phenomenon (KMP), a life-threatening coagulopathy, the short-term addition of prednisolone is also recommended.
[1]Sichuan Univ, Div Oncol, Dept Pediat Surg, West China Hosp, 37 Guo Xue Xiang, Chengdu 610041, Peoples R China.
[2]Sichuan Univ, Pediat Intens Care Unit, Dept Crit Care Med, West China Hosp, 37 Guo Xue Xiang, Chengdu 610041, Peoples R China.
[3]Fudan Univ, Childrens Hosp, Dept Pediat Surg, Div Oncol, Shanghai, Peoples R China.
[4]Sichuan Univ, West China Univ Hosp 2, Pediat Intens Care Unit, Chengdu, Peoples R China.
[5]Chengdu Women & Childrens Cent Hosp, Dept Vasc & Intervent Radiol, Chengdu, Peoples R China.
[6]Sichuan Univ, West China Hosp, Dept Radiol, Chengdu, Peoples R China.
[7]Chengdu Shangjin Nanhu Hosp, Dept Pediat Surg, Chengdu, Peoples R China.
[8]Shanghai Childrens Hosp, Dept Pediat Surg, Shanghai, Peoples R China.
[9]Harvard Med Sch, Boston Childrens Hosp, Vasc Biol Program, Boston, MA USA.
[10]Harvard Med Sch, Boston Childrens Hosp, Dept Surg, Boston, MA USA.
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