Kaposiform Hemangioendothelioma
Conditions
Keywords
Kaposiform Hemangioendothelioma, Sirolimus, Discontinuation Strategies
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.
Interventions
DRUGSirolimus
Comparison of different sirolimus discontinuation strategies.
Sponsors
West China Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rebound growth rate of KHE | 12 months | The primary endpoint is the rebound growth rate of KHE during the follow-up period. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of disease sequelae | 12 months | 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. |
| Incidence of adverse events throughout the study. | 12 months | Incidence of adverse events during the follow-up period. |
Countries
China
Contacts
Primary ContactYi Ji, PhD
Backup ContactJiangyuan Zhou, Doctor
Outcome results
None listed