Low-dose Versus High-dose Sirolimus Combined With Prednisolone for Kaposiform Hemangioendothelioma With Kasabach-Merritt Phenomenon: a Randomized Noninferiority Trial
This randomized clinical trial evaluates if low-dose sirolimus (target trough 4-8 ng/mL) combined with prednisolone is noninferior in efficacy but superior in safety compared to standard high-dose sirolimus (target trough 10-15 ng/mL) combined with prednisolone in pediatric patients with kaposiform hemangioendothelioma and Kasabach-Merritt phenomenon (KHE with KMP), with participants randomized 1:1 to receive the assigned regimen, undergo routine blood and imaging monitoring, and be evaluated for clinical response and adverse events.
• Presenting a KHE with the following characteristics:
‣ Clinical features and histological findings consistent with progressive, non-resectable KHE associated with KMP.
⁃ Patients must be 0 - 18 years of age at the time of study entry.
⁃ Without functional impairment requiring treatment of corticosteroid.
• Organ function requirements:
• 1 Adequate liver function:
⁃ Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN)for age, and
⁃ ALT and AST less than or equal to 2.5 x upper limit normal (ULN) for age.
• 2 Adequate renal function:
⁃ 0-5 years of age maximum serum creatinine (mg/dL) of 0.8
• Adequate bone marrow function: Absolute Neutrophil Count (ANC) greater than or equal to 1 x 10 to the ninth/Liter.
• Consent of parents (or the person having parental authority in families): Signed and dated written informed consent.