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NCT Number: NCT04448873

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

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

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Key information

Conditions

Kaposiform Hemangioendothelioma Blood Platelet Disorders Cytopenia Hemangioma Hematologic Diseases Hemic and Lymphatic Diseases Kasabach-Merritt Syndrome Neoplasms Neoplasms by Histologic Type Neoplasms, Vascular Tissue Thrombocytopenia

Age range

Up to 12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 210012, China

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

sirolimus

Drug

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

Other names: Rapamycin

Primary outcomes

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

    Time frame: From admission to follow-up one year

    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.

Secondary outcomes

  1. Remission of KHE and the dose of sirolimus at one year follow-up

    Time frame: From admission to follow-up one year

    At one year's follow-up, the participant may be on remission, but still taking sirolimus. The condition is a binary variable and the dose of sirolimus is a continuous variable.

  2. Relapse of KHE and the dose of sirolimus at one year follow-up

    Time frame: From admission to follow-up one year

    At one year's follow-up, the participant may suffer from relapse of KHE and still takes sirolimus. The period of time to the first relapse will be recorded as a time variable, and the cox regression survival analysis model will be used.The dose of sirolimus is a continuous variable. Whether this variable is normally distributed will be checked.

  3. Side effects of sirolimus

    Time frame: From admission to follow-up one year

    The outcome 4 will be described. Adverse events will be reported according to Common Terminology Criteria for Adverse Events, version 4.0 (CTCAE v4.0). Incidence of complications such as oral ulcers, abnormal liver enzymes, infections will be recorded. The blood concentration of sirolimus will be adjusted accordingly.

  4. Platelet count

    Time frame: From admission to follow-up one year

    Platelet count is one of the major indicators of response to treatment and will be used in a prognosis model to predict the clinical characteristics of patients with benefits. It is supposed to be greater than 100×10^9/L.

  5. Fibrinogen level

    Time frame: From admission to follow-up one year

    Fibrinogen level will be used in a prognosis model to predict the clinical characteristics of patients with benefits. It is supposed to be at 2-4g/L.

  6. Tumor volume

    Time frame: From admission to follow-up one year

    Tumor volume will be used in a prognosis model to predict the clinical characteristics of patients with benefits. The size of the tumor is supposed to shrink according to imaging evaluation. The tumor is supposed to be softer by palpation.

  7. Complaints

    Time frame: From admission to follow-up one year

    Complaints are subjective feelings. Whether there is pain, swelling, lameness or skin color change will be recorded as binary variables and used in a prognosis model to predict the clinical characteristics of patients with benefits.

Other outcomes

  1. Remission of KHE and no use of sirolimus at two or three year follow-up

    Time frame: From admission to follow-up two and three years

    This 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.

  2. Remission of KHE and the dose of sirolimus at two or three year follow-up

    Time frame: From admission to follow-up two and three years

    At two or three years' follow-up, the participant may be on remission, but still taking sirolimus. The condition is a binary variable and the dose of sirolimus is a continuous variable.

  3. Relapse of KHE and the dose of sirolimus at two or three year follow-up

    Time frame: From admission to follow-up two and three years

    At two or three years' follow-up, the participant may suffer from relapse of KHE and still takes sirolimus. The period of time to the relapse will be recorded as a time variable, and the cox regression survival analysis model will be used.The dose of sirolimus is a continuous variable. Whether this variable is normally distributed will be checked.

Sponsors and collaborators

Lead sponsor

Children's Hospital of Fudan University

Other

Registry information

Official study title

Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma: a Randomized Controlled Trial

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Jun 26, 2020
Registry last updated
Sep 26, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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