Low-Dose Sirolimus as Effective as High-Dose for Kaposiform Hemangioendothelioma Treatment, suggests study

It remains unknown whether low-dose sirolimus can replace high-dose sirolimus for the treatment of kaposiform hemangioendothelioma (KHE) without the Kasabach–Merritt phenomenon (KMP). A study was done to evaluate the noninferiority and safety of low-dose versus high-dose sirolimus in Kaposiform Hemangioendothelioma patients.

This randomized, multicenter, open-label, noninferiority trial was conducted from February 2021 to August 2022. Participants received either a low-dose sirolimus regimen (blood trough concentration 5-8 ng/mL) or a high-dose sirolimus regimen (blood trough concentration 10-15 ng/mL). The primary endpoint was the difference in the proportion of patients between groups who achieved an objective response, defined as a ≥20% reduction in Kaposiform Hemangioendothelioma volume at month 12.

Results: In this study, 39 were in the low-dose group, and 40 were in the high-dose group. At 1 year of treatment, 90.0% in the high-dose group and 89.7% in the low-dose group achieved an objective response (difference, 0.3%; 95% confidence interval -13.1 to 13.6). The incidences of total adverse events (AEs) and serious adverse events were similar between the two groups, but respiratory, skin and mucosal adverse events were less common in the low-dose group.

Low-dose sirolimus is noninferior to high-dose sirolimus in treating Kaposiform Hemangioendothelioma.

Reference:

Efficacy and Safety of High- vs Low-Dose Sirolimus in Patients with Kaposiform Hemangioendothelioma: A Randomized Clinical Trial Zhou, Jiangyuan et al. Journal of the American Academy of Dermatology, Volume 0, Issue 0