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

Abstract

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.

Kaposiform hemangioendothelioma with Kasabach–Merritt phenomenon in an infant: Successful treatment with prednisolone, vincristine, and addition of sirolimus

“…This is consistent with other literature that has shown effectiveness of mTOR inhibition in patients with KHE refractory to standard or alternative treatment modalities . In a multicenter retrospective analysis, patients who received sirolimus as part of treatment tended to have improvement in clinical symptoms and tumor size, suggesting that sirolimus therapy should be considered …”

Development of Kasabach–Merritt phenomenon following vaccination: More than a coincidence?

“…Evidence for the efficacy of sirolimus in treatment of KHE is plentiful, albeit no controlled studies exist . However, it is noteworthy that patients should not receive a live vaccine while being treated with sirolimus.…”

Clinical Review: Management of Adult Kasabach-Merritt Syndrome Associated with Hemangiomas

“…Another study showed that Sirolimus is a well-tolerated. The common grade three side effects bone marrow toxicity in 27%, gastrointestinal toxicity in 3% and metabolic toxicity in 3% [74,75].…”