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

Children's Hospital of Fudan University30 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Kaposiform Hemangioendothelioma Kasabach-Merritt Syndrome

Interventions

SirolimusDRUG

After at least 2 years of remission of KHE, we compare guided discontinuation with maintenance treatment in pediatric patients with KHE.

Eligibility

Sex: ALLMax age: 12 Years

Medical Language ↔ Plain English

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

Locations (1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

Outcomes

Primary Outcomes

Remission of KHE and no use of sirolimus at one year follow-up.

The primary outcome is a binary variable. The primary outcome measure will be analyzed with binary logistic regression to estimate the odds ratio between the two groups.