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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
120
Comparison of different sirolimus discontinuation strategies.
West China Hospital of Sichuan University
Chengdu, Sichuan, China
Rebound growth rate of KHE
The primary endpoint is the rebound growth rate of KHE during the follow-up period.
Time frame: 12 months
Incidence of disease sequelae
Incidence of disease sequelae, assessed by the number of participants who develop clinically confirmed sequelae during the follow-up period, as determined through questionnaires and clinical plus imaging evaluations.
Time frame: 12 months
Incidence of adverse events throughout the study.
Incidence of adverse events during the follow-up period.
Time frame: 12 months
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