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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Direct discontinuation group | Placebo Comparator | Sirolimus was gradually tapered and discontinued over a 2-month period. |
|
| Low-dose treatment group | Experimental | 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 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sirolimus | Drug | Comparison of different sirolimus discontinuation strategies. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Rebound growth rate of KHE | The primary endpoint is the rebound growth rate of KHE during the follow-up period. | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 12 months |
| Incidence of adverse events throughout the study. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yi Ji, PhD | Contact | +862885423453 | jijiyuanyuan@163.com | |
| Jiangyuan Zhou, Doctor | Contact | +8613668491160 | 13668491160@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Yi Ji, PhD | Sichuan University West China Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| West China Hospital of Sichuan University | Chengdu | Sichuan | 610041 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40107509 | Result | Zhou J, Lan Y, Qiu T, Zhang Z, Gong X, Zhang X, Yang C, Zhou Z, Zhang Y, Yang M, Fu J, He C, Peng Q, Hu F, Xia C, Kong F, Chen S, Ji Y. Efficacy and safety of high-vs low-dose sirolimus in patients with kaposiform hemangioendothelioma: A randomized clinical trial. J Am Acad Dermatol. 2025 Jul;93(1):124-131. doi: 10.1016/j.jaad.2025.03.023. Epub 2025 Mar 17. | |
| 36916140 |
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| ID | Term |
|---|---|
| C537007 | Kaposiform Hemangioendothelioma |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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Incidence of adverse events during the follow-up period. |
| 12 months |
| Zhou J, Li Y, Qiu T, Gong X, Yang K, Zhang X, Zhang Z, Lan Y, Hu F, Peng Q, Zhang Y, Kong F, Chen S, Ji Y. Long-term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges. Int J Cancer. 2023 Aug 1;153(3):600-608. doi: 10.1002/ijc.34509. Epub 2023 Mar 22. |
| 35030255 | Result | Ji Y, Chen S, Zhou J, Yang K, Zhang X, Xiang B, Qiu T, Gong X, Zhang Z, Lan Y, Hu F, Kong F, Qiu Q, Zhang Y. Sirolimus plus prednisolone vs sirolimus monotherapy for kaposiform hemangioendothelioma: a randomized clinical trial. Blood. 2022 Mar 17;139(11):1619-1630. doi: 10.1182/blood.2021014027. |
| 34273457 | Result | Zhou J, Yang K, Dai S, Qiu T, Zhang X, Gong X, Chen S, Ji Y. Clinical features and management of kaposiform hemangioendothelioma and tufted angioma: Similarities and differences. J Am Acad Dermatol. 2022 Jul;87(1):172-174. doi: 10.1016/j.jaad.2021.07.012. Epub 2021 Jul 14. No abstract available. |
| 39831682 | Result | Zhou J, Qiu T, Zhang Z, Lan Y, Huo R, Xiang B, Chen S, Qiu L, Xia C, Xu X, Li J, Ma Y, Yao W, Wang Z, Dong C, Qin Z, Tai M, Guo L, He X, Gu S, Li L, Hou F, Cai Y, Wang H, Wang J, Jiang X, Zheng J, Li K, Ji Y. Consensus statement for the diagnosis, treatment, and prognosis of kaposiform hemangioendothelioma. Int J Cancer. 2025 May 15;156(10):1986-1994. doi: 10.1002/ijc.35344. Epub 2025 Jan 20. |