Sirolimus Discontinuation Strategies in Kaposiform Hemangioendothelioma

Not Applicable

Not yet recruiting

• Conditions: Kaposiform Hemangioendothelioma
• Interventions: Drug: Sirolimus

• Registration Number: NCT07131644
• Lead Sponsor: West China Hospital

Brief Summary

Sirolimus has demonstrated efficacy in the treatment of kaposiform hemangioendothelioma (KHE); however, a high rate of rebound growth following discontinuation has been reported, highlighting the clinical importance of establishing an appropriate withdrawal regimen.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-08
• Study Start: 2025-08-13
• Study First Submitted: 2025-08-13
• Study First Submitted QC: 2025-08-13
• Last Update Submitted: 2025-08-13
• Study First Posted: 2025-08-20
• Last Update Posted: 2025-08-20
• Primary Completion: 2027-09-01
• Study Completion: 2028-02-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

• KHE patients who have received sirolimus treatment and met the criteria for drug discontinuation.
• With evaluable clinical or imaging parameters to monitor disease activity or recurrence.
• Patients or their guardians are able to understand the study and provide written informed consent.
• Hematologic and hepatic/renal function meet the safety criteria for drug administration.

Exclusion Criteria

• Patients who are unable to comply with the follow-up or treatment schedule, potentially affecting the integrity of study data.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Low-dose treatment group	Sirolimus	Low-dose sirolimus treatment was administered for 6 months (with blood levels maintained at 2-5 ng/mL), followed by gradual tapering and discontinuation over 2 months.
Intermittent treatment group	Sirolimus	Sirolimus was administered using a 3-days-on/4-days-off weekly schedule for 6 months, followed by gradual tapering and discontinuation over 2 months.
Direct discontinuation group	Sirolimus	Sirolimus was gradually tapered and discontinued over a 2-month period.

Primary Outcome Measures

Name	Time	Method
Rebound growth rate of KHE	12 months	The primary endpoint is the rebound growth rate of KHE during the follow-up period.

Secondary Outcome Measures

Name	Time	Method
Incidence of disease sequelae	12 months	Incidence of disease sequelae during the follow-up period.
Incidence of adverse events throughout the study.	12 months	Incidence of adverse events during the follow-up period.

Trial Locations

Locations (1)

West China Hospital of Sichuan University; 🇨🇳 Chengdu, Sichuan, China