atorvastatin + acetylcysteine + danazol

atorvastatin + acetylcysteine + danazol is a combination therapy regimen being investigated by Peking University People's Hospital for the treatment of corticosteroid-resistant or relapsed immune thrombocytopenia (ITP). The regimen repurposes atorvastatin, a small molecule HMG-CoA reductase inhibitor typically used for cholesterol management, to enhance bone marrow endothelial progenitor cell (BM EPC) function. This enhancement supports megakaryocytopoiesis and platelet production by downregulating the p38 MAPK pathway and upregulating the Akt pathway. The combination also includes N-acetylcysteine (NAC), which acts as an antioxidant and reactive oxygen species scavenger to mitigate oxidative stress, and danazol, a synthetic androgen with immunomodulatory properties. This triple therapy is currently being evaluated in a randomized, open-label, multicenter Phase 2 clinical trial (NCT03460808) to compare its efficacy and safety against danazol monotherapy in patients who have failed first-line steroid treatments.

Other names

atorvastatin, acetylcysteine and danazol combination therapy