Dimana Dimitrova, Juan Gea-Banacloche, Seth M Steinberg, Jennifer L Sadler, Stephanie N Hicks, Ellen Carroll, Jennifer S Wilder, Mark Parta, Lauren Skeffington, Thomas E Hughes, Jenny E Blau, Miranda M Broadney, Jeremy J Rose, Amy P Hsu, Rochelle Fletcher, Natalia S Nunes, Xiao-Yi Yan, William G Telford, Veena Kapoor, Jeffrey I Cohen, Alexandra F Freeman, Elizabeth Garabedian, Steven M Holland, Andrea Lisco, Harry L Malech, Luigi D Notarangelo, Irini Sereti, Nirali N Shah, Gulbu Uzel, Christa S Zerbe, Daniel H Fowler, Ronald E Gress, Christopher G Kanakry, Jennifer A Kanakry
Allogeneic blood or marrow transplantation (BMT) is a potentially curative therapy for patients with primary immunodeficiency (PID). Safe and effective reduced-intensity conditioning (RIC) approaches that are associated with low toxicity, utilize alternative donors, and afford good immune reconstitution are needed to advance the field. Twenty PID patients, ranging in age from 4 to 58 years, were treated on a prospective clinical trial of a novel, radiation-free and serotherapy-free RIC, T-cell-replete BMT approach using pentostatin, low-dose cyclophosphamide, and busulfan for conditioning with post-transplantation cyclophosphamide-based graft-versus-host-disease (GVHD) prophylaxis...
September 4, 2019: Biology of Blood and Marrow Transplantation