Can sirolimus treat thrombocytopenia?

Immune thrombocytopenia(ITP) is an autoimmune disease caused by the self-destruction of circulating platelets. Failure to respond or maintenance of response to first-line therapy can lead to refractory/relapsed (R/R) ITP. Its mechanism remains complex and standard clinical treatments are lacking. Sirolimus, a mammalian target of rapamycin (mTOR) inhibitor, has been shown to inhibit lymphocyte activity, suggesting the potential of SRL in the treatment of ITP. Activation of the mTOR pathway in autoimmune diseases suggests that SRL may be a useful drug for the treatment of ITP.

An open-label, prospective clinical trial using SRL to treat patients with R/R ITP. The trial recruited 86 patients, each taking 2-4 mg of SRL per day. By the third month, 40% of patients achieved complete response (CR) and 45% achieved partial response (PR), establishing an 85% overall response rate (ORR). After 6 months of treatment, 41% of patients achieved CR and 29% achieved PR, with an ORR of 70% and no serious side effects. After 12 months of follow-up, the ORR remained at 65%. In summary, sirolimus shows its effectiveness as a second-line drug for R/R ITP.

SirolimusThe original drug has been launched in China and has entered the scope of Class B medical insurance. SpecificationsThe price of each box of 1mg*10 tablets is about 400 yuan. The domestic drug is only available to patients with anti-rejection reactions in organ transplantation. Patients who do not meet the conditions may not be able to purchase this drug. The original sirolimus drug has also been launched overseas. The price of the Turkish version of Specifications1mg*100 tablets per box is around RMB 2,000 (the price may fluctuate due to exchange rates). The ingredients of the original drugs marketed in China are basically the same as those of foreign original drugs. Currently, there are no generic sirolimus drugs produced in other countries.