Allogeneic Hematopoietic Stem-Cell Transplantation for GATA2 Deficiency Using a Busulfan-Based Regimen

• Multiple phenotypes of disease complicate the choice of conditioning for stem cell transplantation in GATA2 deficiency.• Reduced intensity has been insufficient for engraftment or prevention of relapse of hematologic disorders or cytogenetic abnormalities.• A busulfan-based regimen targeted an AUC betwen 3600 and 4800 µMol*min and added post-transplant high dose cyclophosphamide for graft-versus-host disease prophylaxis in recipients from haploidentical donors.• Engraftment was achieved in all recipients.

Source: Biology of Blood and Marrow Transplantation - February 2, 2018 Category: Hematology Authors: Mark Parta, Nirali N. Shah, Kristin Baird, Hind Rafei, Katherine R. Calvo, Thomas Hughes, Kristen Cole, Meg Kenyon, Bazetta Blacklock Schuver, Jennifer Cuellar-Rodriguez, Christa S. Zerbe, Steven M. Holland, Dennis D. Hickstein Source Type: research