Efficacy and Safety of Different Concentrations of Sirolimus in the Treatment of Kaposiform Hemangioendothelioma.

  • STATUS
    Recruiting
  • End date
    Aug 20, 2023
  • participants needed
    30
  • sponsor
    West China Hospital
Updated on 5 May 2022

Summary

The purpose of this study is to compare the efficacy and safety of different concentration gradients of sirolimus in the treatment of Kaposiform hemangioendothelioma.

Description

Kaposiform hemangioendothelioma (KHE) is a rare aggressive vascular neoplasm that occurs predominantly in infancy or early childhood, with an incidence of approximately 0.71/100,000. Currently, sirolimus is a promising treatment modality for KHE. Most scholars consider sirolimus blood concentration of 5-15 ng/ml to be an effective therapeutic concentration.

However, long-term higher dose sirolimus treatment can cause some common complications such as oral mucositis which affects the quality of life of the patient. Finer control of the plasma concentration of sirolimus may contribute to the efficacy of treatment and reduce the incidence of complications. Therefore, we conducted this study to see if low-dose sirolimus is beneficial to the prognosis of patients.

Details
Condition Kaposiform Hemangioendothelioma
Treatment Sirolimus
Clinical Study IdentifierNCT04775173
SponsorWest China Hospital
Last Modified on5 May 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Presenting a KHE with the following characteristics
Clinical features and histological findings consistent with progressive, non-resectable KHE
Patients must be 0 - 18 years of age at the time of study entry
Adequate liver function: a. Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN) for age, and; b. ALT and AST less than or equal to 2.5 x upper limit normal (ULN) for age
Adequate renal function: a. 0-5 years of age maximum serum creatinine (mg/dL) of 0.8
6-10 years of age maximum serum creatinine (mg/dL) of 1.0; c. 11-15 years of age maximum serum creatinine (mg/dL) of 1.2; d. 16-18 years of age maximum serum creatinine (mg/dL) of 1.5
Adequate bone marrow function: Absolute Neutrophil Count (ANC) greater than or equal to 1 x 10 to the ninth/Liter
Consent of parents (or the person having parental authority in families): Signed and dated written informed consent

Exclusion Criteria

Allergy to sirolimus or other rapamycin analogues
Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of randomization
Patients must not be known to be Human Immunodeficiency Virus positive or known immunodeficiency. Testing is not required unless a condition is suspected
Other concurrent severe and/or uncontrolled medical disease which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration)
Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus
Patients who have a history of malignancy
Patients with an inability to participate or to follow the study treatment and assessment plan
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