Low- vs High-Dose Sirolimus With Prednisolone for KHE and KMP
Low-dose Versus High-dose Sirolimus Combined With Prednisolone for Kaposiform Hemangioendothelioma With Kasabach-Merritt Phenomenon: a Randomized Noninferiority Trial
Yi Ji
76 participants
Jun 16, 2026
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria15
- 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:
- 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.
- Adequate renal function:
- 0-5 years of age maximum serum creatinine (mg/dL) of 0.8
- 6-10 years of age maximum serum creatinine (mg/dL) of 1.0
- 11-15 years of age maximum serum creatinine (mg/dL) of 1.2
- 16-18 years of age maximum serum creatinine (mg/dL) of 1.5
- 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.
Exclusion Criteria7
- Allergy to sirolimus or other rapamycin analogues.
- Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of randomization.
- Patients must not be known to be Human Immunodeficiency Virus positive or known immunodeficiency. Testing is not required unless a condition is suspected.
- Other concurrent severe and/or uncontrolled medical disease which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).
- Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus.
- Patients who have a history of malignancy.
- Patients with an inability to participate or to follow the study treatment and assessment plan.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
Participants will receive oral sirolimus in combination with prednisolone. Patients will be randomized in a 1:1 ratio to either a low-dose sirolimus group, with dose adjustments to maintain a target plasma trough concentration of 4-8 ng/mL, or a high-dose sirolimus group, with dose adjustments to maintain a target plasma trough concentration of 10-15 ng/mL. Prednisolone will be administered according to the study protocol and tapered based on clinical response. The total treatment duration will be 12 months.
Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07656909