Sirolimus Discontinuation Strategies in Kaposiform Hemangioendothelioma

Sichuan University

Status and phase

Not yet enrolling

Phase 2

Conditions

Kaposiform Hemangioendothelioma

Treatments

Drug: Sirolimus

Study type

Interventional

Funder types

Other

Identifiers

NCT07131644

20250611

Details and patient eligibility

About

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.

Enrollment

120 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

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.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Single Blind

120 participants in 3 patient groups, including a placebo group

Direct discontinuation group

Placebo Comparator group

Description:

Sirolimus was gradually tapered and discontinued over a 2-month period.

Treatment:

Drug: Sirolimus

Low-dose treatment group

Experimental group

Description:

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.

Treatment:

Drug: Sirolimus

Intermittent treatment group

Experimental group

Description:

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.

Treatment:

Drug: Sirolimus