Successful sirolimus therapy of an aplastic anemia patient with chronic kidney disease A case report

Rationale: It is very difficult to treat patients with aplastic anemia accompanied by chronic kidney disease. The nephrotoxicity of cyclosporine limits its use in these patients. Most of these patients also lack suitable sibling donors. Sirolimus, as a new type of immunosuppressive agent, has good therapeutic effect, lower toxicity, especially lower nephrotoxicity, thus attracting the attention of hematologists. Patient concerns: This 55-year-old Chinese male patient suffered from pancytopenia and renal insufficiency and has a poor quality of life. Diagnosis: The patient was diagnosed as severe aplastic anemia with chronic kidney disease-G3a. Interventions: We started the sirolimus therapy with the initial dose of 1mg per day. Based on the good tolerability and clinical effect, we increased the dose of sirolimus to 2mg per day after 2 weeks. Outcomes: By taking sirolimus, the patient's peripheral blood cell count gradually increased, and he achieved blood transfusion independent, and eventually the blood cell count was completely normal. Lessons: We consider that sirolimus is a safe, effective, and well-tolerated oral drug that can be used as a treatment for aplastic anemia patients with chronic kidney disease.