Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma

  • End date
    Jul 1, 2023
  • participants needed
  • sponsor
    Children's Hospital of Fudan University
Updated on 16 August 2021


This randomized controlled trial aims to compare guided discontinuation with maintenance treatment of sirolimus in pediatric patients with KHE.


Kaposiform Hemangioendothelioma (KHE) is a rare vascular tumor that occurs in infants and children. KHE is characterized by sheets of spindle cells with an infiltrative pattern in the dermis, subcutaneous fat, and muscle. It is locally aggressive and can cause Kasabach-Merritt phenomenon, a serious life-threatening coagulopathy characterized by profound thrombocytopenia and hypofibrinogenemia. Sirolimus, one of the mTOR inhibitors, has become a new and very effective treatment, which is especially reliable for KHE with KMP and has acceptable side effects. However, there is yet no strong evidence on the best practice of treatment length of sirolimus. This randomized controlled trial aims to compare guided discontinuation with maintenance treatment in pediatric patients with KHE in order to provide a basis for the optimal treatment duration of sirolimus, as well as the clinical characteristics of pediatric patients who can safely reduce the dose till withdrawal.

Condition Kaposiform Hemangioendothelioma, Kasabach-Merritt Syndrome, kasabach-merritt phenomenon
Treatment Sirolimus
Clinical Study IdentifierNCT04448873
SponsorChildren's Hospital of Fudan University
Last Modified on16 August 2021


Yes No Not Sure

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
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