Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma
Kaposiform Hemangioendothelioma, Kasabach-Merritt Syndrome
About this trial
This is an interventional treatment trial for Kaposiform Hemangioendothelioma focused on measuring Kaposiform Hemangioendothelioma, Kasabach-Merritt Syndrome, Sirolimus, Rapamycin
Eligibility Criteria
Inclusion Criteria:
- Participant diagnosed with KHE with or without KMP
- Participant age 0-12 years
- Participant with detailed medical records of the disease at the time of screening
- Participant with at least two years of remission of KHE and no previous toxicity or adverse events
- Participant with normal liver and kidney function
- Participant with signed and dated informed consent from the guardian(s)
Exclusion Criteria:
- Participant with other hematological diseases
- Participant with other solid tumor
- Participant with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
- Participant with infectious diseases
- Unwilling participant
Sites / Locations
- Children's Hospital of Fudan University
Arms of the Study
Arm 1
Arm 2
Active Comparator
Experimental
Maintenance treatment group
Guided discontinuation group
After at least 2 years of remission of KHE, the participant receives sirolimus as usual. The serum concentration is supposed to be 5-7 ng/ml. If the effect or side effects of sirolimus require discontinuation, it is allowed to modify intervention, and if so, the patient stays in the maintenance group.
After at least 2 years of remission of KHE, the discontinuation measurement should be guided by the clinician with the following principles: 10% monthly reduction of the previous dose at most. At least 5 half-lives between each reduction (2 weeks). Blood concentration should be monitored monthly. Adjustment can be suggested according to the linear relationship between the dose and the blood concentration. At least 6 months for the duration of guided discontinuation. Regular assessments and evaluations should be done. If the condition relapses or worsens during this process, dose of sirolimus should be adjusted to the previously effective dose. After a 3-month stabilization phase, 5% monthly reduction of the previous dose could be considered.