Rapamune对肾移植有多大效果？

Rapamin () has shown good application prospects in vascular abnormal diseases. Most of the current studies are retrospective experience summaries, and larger phase II and phase III prospective studies are needed in the future to determine the treatment dose and reasonable medication duration. In addition, it is necessary to expand the sample size and extend the follow-up time to determine the therapeutic effect of Rapamune on diseases such as arteriovenous malformations or Kaposiform lymphangiomatosis, as well as possible adverse reactions that may occur with growth and development. Some of these trials are already underway. 

Kaposiform hemangioendothelioma (KHE) is a true vascular tumor in infants and young children that can occur in various parts of the body such as the limbs, head and neck, abdominal organs, and mediastinum. It is characterized by the presence of a large number of vascular structures composed of spindle-shaped endothelial cell bundles, which penetrate into the surrounding soft tissues and form nodules with irregular borders. Although pathologically benign, they are locally invasive. Especially when the lesions are located in the mediastinum and important abdominal organs, treatment is more difficult. When accompanied by consumption coagulopathy, Kasabach-Merritt syndrome (KMS), there is a considerable risk of death or disability. According to literature statistics, about 50%-60% of KHE will be accompanied by KMS, especially when the lesion is located in the abdominal cavity or chest and the diameter is >10cm. The pathological mechanism is lymphatic angiogenesis in the lesion area, which captures a large number of platelets in the circulation. The platelets aggregate in the blood vessels and the coagulation cascade is abnormally activated. The blood picture shows that the platelet count and hemoglobin are reduced, the prothrombin time (Pt) is prolonged, the partial thromboplastin time (APTT) is reduced, and the fibrinogen is reduced. This consumptive vicious cycle of coagulation often leads to rapid enlargement of lesions and tendency of systemic bleeding, leading to worsening of the condition. Past treatment experience recommends steroids + vincristine as the standard treatment plan, but 30%-40% of patients are ineffective or unable to achieve complete relief. Recently, some researchers reported that a 9-month-old child with KHE combined with KMS was initially treated with steroids, vincristine combined with Rapamune. After completing the initial 6-8 weeks of treatment, steroids and vincristine were no longer administered, and Rapamune was used as daily oral maintenance therapy for 20 months. After 15 days of treatment, the platelet count increased from 9×109/L to >50×109/L, and returned to normal (>150×109/L) 4 months later. Abdominal MR reexamination showed that the mass gradually shrank at 1, 9, and 16 months. A review of 42 relevant literature showed that in children with relapsed or refractory KHE, if there is an opportunity to use Rapamune for treatment, the prognosis can be significantly improved. In another study, rapamune was used as the initial and only drug in eight children. Among the 8 children, 3 had KHE and 5 had KHE combined with KMS. Before treatment, except for one child who received blood transfusion due to low platelet count, all children did not receive other drugs or treatments. The results showed that the range of lesions in all children was significantly reduced, and the blood images of children with KMS returned to normal. The drug withdrawal time was 6-22 months, and the size of the lesions was stable without recurrence. The experience of this study shows that rapamycin () can be used as the basis and only drug treatment option for KHE regardless of whether it is accompanied by KMS or not.