Sirolimus Treatment for Refractory PRCA

2017-12-13 08:34:08 | BioPortfolio


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 sirolimus 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.

Sirolimus (rapamycin) is an agent produced by the bacterium Streptomyces hygroscopicus, inhibits the mammalian target of rapamycin (mTOR). mTOR is a serine/threonine kinase that regulates cell growth, proliferation, metabolism and survival in eukaryotic cells, and is identified as two interacting complex, mTORC1 and mTORC2. Sirolimus primarily inhibits mTORC1, has been approved for prevent organ transplant rejection, especially in renal transplantation, sirolimus also promises to treat autoimmune, degenerative and hyperproliferative disorders. Recently, sirolimus 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 sirolimus on refractory PRCA so far.

In this study, It is anticipate to evaluate the effect of sirolimus on 30 patients with refractory PRCA, the side-effects was documented and plasma concentration of sirolimus will be monitor.

Study Design


Pure Red Cell Aplasia




Peking Union Medical College Hospital




Peking Union Medical College Hospital

Results (where available)

View Results


Published on BioPortfolio: 2017-12-13T08:34:08-0500

Clinical Trials [375 Associated Clinical Trials listed on BioPortfolio]

Pure Red Cell Aplasia in Patients With Chronic Kidney Disease and in Use of Epoetin Alfa

The purpose of this study is to determine occurrence of pure red cell aplasia in a group of participants with chronic renal insufficiency and with resistance criteria to epoetin alfa treat...

A Study to Observe Patients Who Have Developed a More Severe Form of Anemia (Pure Red Cell Aplasia) Which is Associated With the Erythropoietin Treatment for Their Original Anemia

The purpose of this study is to investigate a possible relationship between antibodies to recombinant erythropoietin and the clinical course of Pure Red Cell Anemia (PRCA) in patients curr...

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The purpose of this study is to record the number of patients with chronic renal disease who are receiving treatment with epoetin alfa or other recombinant erythropoietins who develop pure...

A Study to Collect Information About Risk Factors in the Development of a Rare Form of Anemia (Pure Red Cell Aplasia; PRCA) and Antibodies to Erythropoietin From Patients With Kidney Disease Treated With Epoetin Alfa

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Acquired pure red cell aplasia (PRCA) and autoimmune hemolytic anemia (AIHA) are rare complications of immunosuppression in pediatric solid organ transplant patients. We report a 14-month-old female c...

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Medical and Biotech [MESH] Definitions

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