Overview
In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.
Description
Vascular anomaly is a kind of rare disease. According to histology, biological behavior and clinical manifestations, it can be divided into two categories: vascular tumor and vascular malformation.
mTOR inhibitors are proved with the properties of anti-proliferation and anti-angiogenesis. Therefore, they have been used in the treatment of vascular anomalies. Sirolimus, by its ability to prevent downstream protein synthesis and subsequent cell proliferation and angiogenesis, has become a novel and effective treatment. However, after the children reach complete response, there may still be skin manifestations that affect the appearance and cause psychological shadows. Therefore, intervention is required.
Studies have reported that topical sirolimus is effective in treating Kaposiform Hemangioendothelioma (KHE). It is absorbed through the skin, avoiding the first pass elimination effect of the liver. Fewer adverse reactions have been observed. In this study, we investigate the efficacy and safety of percutaneous administration of sirolimus in the treatment of superficial complicated vascular anomalies.
Eligibility
Inclusion Criteria:
- Participant clinically or pathologically diagnosed with KHE, TA or complicated
superficial vascular anomolies involving lymphatic components.
- The case is initial, with a relatively limited superficial lesion.
- The participant has residual surface lesions after oral medication.
- Participant with no use of other medication or surgical treatment
- Participant with detailed medical records of the disease at the time of screening
- Participant with signed and dated informed consent from the guardian(s)
Exclusion Criteria:
- Participants with Kasabach-Merritt Phenomenon, with platelets <50×10 9 /L.
- Participants with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
- Participants with other hematological diseases or solid tumor.
- Participants allergic to sirolimus or dressing.