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About the Study

Who runs this study?

This study is being conducted at the Division of Intramural Research in the NHLBI.

The Principal Investigator of this study is Richard Childs, MD. He is Chief of the Section of Transplantation Immunotherapy in the Cellular and Molecular Therapeutics Branch of the NHLBI and Clinical Director of Division of Intramural Research in the NHLBI.

What is this study about?

Our goal is to evaluate the safety and effectiveness of transplantation of using an unmanipulated GCSF mobilized peripheral stem cell allograft from a haploidentical donor and post-transplant cyclophosphamide for patients with SAA or SAA evolving to MDS that has proven to be refractory to conventional immunosuppressive therapy (IST) in patients who lack an HLA-matched donor and who do not have access to a good quality umbilical cord product.

The primary objective of the Phase II study is to evaluate the 1-year chronic GVHD-free survival rate of this modality. Secondary endpoints will include engraftment by day 42, 100 day and 200 day treatment related mortality (TRM), immune reconstitution and standard transplant outcome variables such as incidence and severity of acute and chronic GVHD, and relapse of disease. Health related quality of life will also be assessed as secondary outcome measure.

How can I ask questions or ask for help?

Email us at kristen.gunn@nih.gov (or click on Contact). We will respond within 2 business days.