Rapamune作用及效果

In addition to its significant role in true vascular tumors, rapamin () has also been tried by researchers to treat complex vascular malformations due to its ability to inhibit lymphatic angiogenesis. A recent report collected 35 patients with vascular malformations that failed to respond to conventional treatments, including 13 cases of lymphangiomatosis, 1 case of Kaposiform lymphangiomatosis, 11 cases of large cystic lymphatic malformations, 2 cases of lymphedema, 4 cases of venous malformations, and 4 cases of invasive arteriovenous malformations. After Rapamune treatment was given to these patients, except for all 4 cases of arteriovenous malformations and the only case of Kaposiform lymphangiomatosis, all other vascular malformation diseases showed a response to Rapamune treatment. It was manifested by the reduction of lesions on physical examination and imaging, as well as the improvement of clinical symptoms, and no patient's symptoms worsened after taking the drug. Rapamune undoubtedly provides a new option for refractory vascular malformations that are ineffective with conventional treatment options.  

At present, the dosage of Rapamune used in vascular tumors and vascular malformation diseases mostly refers to the dosage used to resist renal transplant rejection, which is 0.8mg/m2/12h or 2-3mg/m2/24h. The blood concentration reaches and is maintained at 7-15ng/ml. Its potential side effects and adverse reactions are similar to those used to combat immune rejection in organ transplants. In the articles that have been reported so far, the longest use time is 20-22 months, and no obvious adverse reactions have been seen in children. Most patients, even infants and young children, tolerate rapamune well. There are reports that 2 cases of children developed stomatitis. Because it lasted for a long time, the medication had to be stopped, and they gradually recovered after stopping the medication. No deaths related to side effects were reported. Compared with hormonal steroids, there is no growth retardation and no risk of Cushing's syndrome. A phase II randomized controlled trial is ongoing to compare the effects and adverse effects of rapamycin and vincristine in the treatment of KHE.