<unk>  is indicated for the prevention of organ transplant rejection and for the treatment of lymphangioleiomyomatosis (LAM).<unk>  (Fyarro), as protein-bound particles, is indicated for the treatment of adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). Prevention of transplant rejection The chief advantage<unk>  has over calcineurin inhibitors is its low toxicity toward kidneys. Transplant patients maintained on calcineurin inhibitors long-term tend to develop impaired kidney function or even kidney failure; this can be avoided by using<unk>  instead. It is particularly advantageous in patients with kidney transplants for hemolytic-uremic syndrome, as this disease is likely to recur in the transplanted kidney if a calcineurin-inhibitor is used. However, on 7 October 2008, the FDA approved safety labeling revisions for<unk>  to warn of the risk for decreased renal function associated with its use. In 2009, the FDA notified healthcare professionals that a clinical trial conducted by Wyeth showed an increased mortality in stable liver transplant patients after switching from a calcineurin inhibitor-based immunosuppressive regimen to<unk> . A 2019 cohort study of nearly 10,000 lung transplant recipients in the US demonstrated significantly improved long-term survival using<unk>  + tacrolimus instead of mycophenolate mofetil + tacrolimus for immunosuppressive therapy starting at one year after transplant.<unk>  can also be used alone, or in conjunction with a calcineurin inhibitor (such as tacrolimus), and/or mycophenolate mofetil, to provide steroid-free immunosuppression regimens. Impaired wound healing and thrombocytopenia are possible side effects of<unk> ; therefore, some transplant centers prefer not to use it immediately after the transplant operation, but instead administer it only after a period of weeks or months. Its optimal role in immunosuppression has not yet been determined, and it remains the subject of a number of ongoing clinical trials. Lymphangioleiomyomatosis In May 2015, the FDA approved<unk>  to treat lymphangioleiomyomatosis (LAM), a rare, progressive lung disease that primarily affects women of childbearing age. This made<unk>  the first drug approved to treat this disease. LAM involves lung tissue infiltration with smooth muscle-like cells with mutations of the tuberous sclerosis complex gene (TSC2). Loss of TSC2 gene function activates the mTOR signaling pathway, resulting in the release of lymphangiogenic growth factors.<unk>  blocks this pathway. The safety and efficacy of<unk>  treatment of LAM were investigated in clinical trials that compared<unk>  treatment with a placebo group in 89 patients for 12 months. The patients were observed for 12 months after the treatment had ended. The most commonly reported side effects of<unk>  treatment of LAM were mouth and lip ulcers, diarrhea, abdominal pain, nausea, sore throat, acne, chest pain, leg swelling, upper respiratory tract infection, headache, dizziness, muscle pain and elevated cholesterol. Serious side effects including hypersensitivity and swelling (edema) have been observed in renal transplant patients. While<unk>  was considered for treatment of LAM, it received orphan drug designation status because LAM is a rare condition. The safety of LAM treatment by<unk>  in people younger than 18 years old has not been tested. Coronary stent coating The antiproliferative effect of<unk>  has also been used in conjunction with coronary stents to prevent restenosis in coronary arteries following balloon angioplasty. The<unk>  is formulated in a polymer coating that affords controlled release through the healing period following coronary intervention. Several large clinical studies have demonstrated lower restenosis rates in patients treated with<unk> -eluting stents when compared to bare-metal stents, resulting in fewer repeat procedures. A<unk> -eluting coronary stent was marketed by Cordis, a division of Johnson & Johnson, under the tradename Cypher. However, this kind of stent may also increase the risk of vascular thrombosis. Vascular malformations<unk>  is used to treat vascular malformations. Treatment with<unk>  can decrease pain and the fullness of vascular malformations, improve coagulation levels, and slow the growth of abnormal lymphatic vessels.<unk>  is a relatively new medical therapy for the treatment of vascular malformations in recent years,<unk>  has emerged as a new medical treatment option for both vascular tumors and vascular malformations, as a mammalian target of rapamycin (mTOR), capable of integrating signals from the PI3K/AKT pathway to coordinate proper cell growth and proliferation. Hence,<unk>  is ideal for “proliferative” vascular tumors through the control of tissue overgrowth disorders caused by inappropriate activation of the PI3K/AKT/mTOR pathway as an antiproliferative agent.
Sirolimus
Tacrolimus
Temsirolimus
Pimecrolimus
Rituximab
Tofacitinib
Adalimumab
Lenalidomide