Relapse of aplastic anemia responsive to sirolimus combined with cyclosporine

Guangsheng He1, Xiang Zhang, Depei Wu

  • 1Key Laboratory of Thrombosis and Hematostasis of Ministry of Health, Jiangsu Insititute of Hematology, Department of Hematology of First Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, P.R. China. heguangsheng@medmail.com.cn

Aplastic anemia (AA) is an immune-mediated disease. Although most patients are responsive to immunosuppressive therapy (IST) with a combination of anti-thymocyte globulin (ATG) and cyclosporine (CsA), some patients relapse or are refractory to IST. Sirolimus (rapamysin) inhibits the serine-threonine kinase mammalian target of rapamysin (mTOR), and blocks CsA-resistant and calcium-independent pathways late in the progression of the T-cell cycle. We report two cases of AA which relapsed after CsA and ATG plus CsA, respectively. They achieved transfusion independence after retreatment with sirolimus in combination with a CsA.