Tacrolimus Treatment for Refractory PRCA

Pure Red Cell Aplasia

tacrolimus, refractory pure red cell aplasia, prospective study

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of tacrolimus for refractory PRCA.

Pure red cell aplasia (PRCA) is a rare normocytic normochromic anemia with reticulocytopenia, characterized by a reduction of erythroid precursors from the bone marrow, could be divided into congenital and acquired PRCA according to pathogenesis. Congenital PRCA, also known as Diamond-Blackfan syndrome, has been associated with pathogenic variant in GATA1 and TSR2 and gene encode ribosomal proteins. Acquired PRCA can be a primary disease which is usually mediated by immunology, or secondary to other diseases, such as lymphoproliferative diseases, autoimmune diseases, thymoma, infection, or drugs. The first line therapy of acquired PRCA is Cyclosporine A and steroids, the second line therapy are anti-CD20, ATG, immunosuppressive drugs like cyclophosphamide, bone marrow transplantation. Unfortunately, some patients did not response or tolerate the above treatments. Tacrolimus, also known as FK506, is an agent mainly used after allogeneic organ transplant to lower risk of organ rejection. Tacrolimus could inhibit the production the production of IL-2, and also used in the therapy of other T cell mediated diseases. Tacrolimus primarily has been approved for prevent organ transplant rejection, especially in renal transplantation, tacrolimus also promises to treat autoimmune, degenerative and hyperproliferative disorders. Recently, tacrolimus has been reported to be effective and well tolerated for many immune-mediated cytopenias, such as autoimmune lymphoproliferative syndrome, immune thrombocytopenia, EVANS syndrome, etc. However, due to the rare occurrence of PRCA and good response rate to cyclosporine, there are very few studies of tacrolimus on refractory PRCA so far. In this study, it is anticipate to evaluate the effect of tacrolimus on 30 patients with refractory PRCA, the side-effects was documented and plasma concentration of tacrolimus will be monitor.

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