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Paroxysmal Nocturnal Hemoglobinuria (PNH) Research Studies

Stem Cell Transplant as Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) - Protocol Description

Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) have an abnormal blood stem cell population which produces faulty red blood cells which are prematurely destroyed in the circulation. This protocol is designed to determine whether healthy stem cells can replace abnormal PNH stem cells in patients with severe PNH. Blood stem cells which grow to become mature blood cells will be collected from a tissue compatible family member. The patient with PNH will be given chemotherapy followed by a transfusion of stem cells from their donor. The procedure will replace the patients abnormal or "PNH" stem cells with normal healthy stem cells from the donor. 

Patients on this study live in the area for approximately four months. We pay for all medical costs related to the transplant. Patients are responsible for living expenses while here, although we provide some daily financial assistance while on study.

To inquire about eligibility, contact our research coordinator at (301) 594-8013 or by e-mail at 

Last Updated: July 31, 2015