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Treatment with sirolimus, an inhibitor of the mammalian target of rapamycin pathway, has improved the prognosis of patients with kaposiform hemangioendothelioma (KHE). However, the efficacy, durability and tolerability of long‐term sirolimus treatment in patients with KHE have not been well elucidated. We performed efficacy and safety assessments based on more than 4.5 years of follow‐up in patients receiving sirolimus therapy for KHE. One hundred sixty‐seven patients were analyzed, including 102 (61.1%) patients with the Kasabach‐Merritt phenomenon (KMP). Follow‐up was conducted after a median of 56.0 months. A total of 154 (92.2%) patients had a durable response to sirolimus treatment. No difference in durable response was found between patients without KMP and patients with KMP (95.4% vs 90.2%; difference, 5.2%; 95% confidence interval [CI], −4.0% to 13.1%). Rebound growth occurred in 17.3% of patients upon sirolimus discontinuation. Early treatment discontinuation (odds ratio [OR]: 3.103; 95% CI: 1.529‐6.299; P =.002) and mixed lesion type (OR: 2.271; 95% CI: 0.901‐5.727; P =.047) were associated with tumor rebound growth. No KHE‐related deaths occurred in this cohort. At the last follow‐up, approximately 17.4% of patients had active disease and/or changes in body structures to a variable extent. Serious adverse events occurred most commonly during the first year of sirolimus therapy. Follow‐up of almost 4.5 years demonstrated that the efficacy of sirolimus persisted over time and that long‐term treatment with sirolimus was not associated with unacceptable cumulative toxicities. However, nonresponse, tumor relapse and long‐term sequelae remained challenges despite intensified and prolonged sirolimus therapy. [ABSTRACT FROM AUTHOR]

Kaposiform hemangioendothelioma (KHE) and tufted angioma (TA) are rare infiltrative vascular tumors. Currently, no standard treatment regimens exist for KHE/TA. The purpose of our study was to evaluate the efficacy and safety of topical application of tacrolimus for superficial KHE/TA. We examined six patients with superficial KHE/TA. All patients were treated with tacrolimus 0.1% ointment twice daily for at least 12 months. The response rate was 100%, including three nearly complete remissions. Only one patient experienced local pruritus during treatment. The data constituted an intriguing rationale for clinical trials of topical tacrolimus in the treatment of superficial KHE/TA. [ABSTRACT FROM AUTHOR]

Abstract: Kasabach–Merritt phenomenon (KMP) occurred uniquely in patients with kaposiform hemangioendothelioma (KHE) and tufted angioma (TA). We report the clinical characteristics of two patients with KHE involving the right upper arm. The patients demonstrated rapid enlargement of the lesion with severe KMP shortly after vaccination. Sirolimus was used to treat the KHE with KMP. The patients showed a quick normalization of the platelet level. The follow‐up examination revealed that the size of the mass was significantly decreased. This report raises the intriguing possibility that extrinsic factors may contribute to the development of KMP in the context of an already existing KHE. [ABSTRACT FROM AUTHOR]

Purpose: Musculoskeletal complications have been associated with kaposiform hemangioendothelioma (KHE) and can lead to disability and reduced quality of life. We aimed to determine the clinical characteristics of musculoskeletal complication in patients with KHE without Kasabach–Merritt phenomenon (KMP) in order to identify features that may aid clinicians in KHE treatment.Patients and methods: We conducted a cohort study of KHE without KMP associated with musculoskeletal complication between January 2006 and February 2017 at three tertiary medical centers in China. The study included 29 nonthrombocytopenic patients with KHE and musculoskeletal complication.Results: The mean age at diagnosis of KHE was 4.5 years (range, 0.3–50.0 years). The mean follow-up was 4.1 years (range, 0.5–9.0 years). In most cases (72.4%), decreased range of motion (ROM) appeared within 2 years of KHE onset. Associated chronic pain was reported in 12 patients. Bone–joint changes were common in patients with decreased ROM (75.9%). All the patients received at least one medical therapy including corticosteroids, vincristine, propranolol, and sirolimus. Sirolimus demonstrated the highest efficacy rate, with 94.7% of patients showing improvements in ROM and chronic pain.Conclusion: Musculoskeletal complication can occur early in the disease course of KHE without KMP. Although no uniformly effective treatment modality was found, sirolimus demonstrated the best response in patients with KHE with decreased ROM and chronic pain. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]