Clinical trial NCT04448873: Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma
Official title: Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma: a Randomized Controlled Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Kaposiform Hemangioendothelioma
Kasabach-Merritt Syndrome
Keywords: Kaposiform Hemangioendothelioma
Kasabach-Merritt Syndrome
Sirolimus
Rapamycin
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
First posted: 2020-06-26
Enrollment: 30
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed description: 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.

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

Primary outcomes:
measure: Remission of KHE and no use of sirolimus at one year follow-up.
timeFrame: From admission to follow-up one year
Secondary outcomes:
measure: Remission of KHE and the dose of sirolimus at one year follow-up
timeFrame: From admission to follow-up one year
measure: Relapse of KHE and the dose of sirolimus at one year follow-up
timeFrame: From admission to follow-up one year
measure: Side effects of sirolimus
timeFrame: From admission to follow-up one year
measure: Platelet count
timeFrame: From admission to follow-up one year
measure: Fibrinogen level
timeFrame: From admission to follow-up one year
measure: Tumor volume
timeFrame: From admission to follow-up one year
measure: Complaints
timeFrame: From admission to follow-up one year
Other outcomes:
measure: Remission of KHE and no use of sirolimus at two or three year follow-up
timeFrame: From admission to follow-up two and three years
measure: Remission of KHE and the dose of sirolimus at two or three year follow-up
timeFrame: From admission to follow-up two and three years
measure: Relapse of KHE and the dose of sirolimus at two or three year follow-up
timeFrame: From admission to follow-up two and three years
Locations:
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Kai Li, MD, PhD
role: PRINCIPAL_INVESTIGATOR
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