title: Reduced-intensity conditioning for hematopoietic cell transplantation of chronic granulomatous disease.
abstract: Hematopoietic cell transplantation (HCT) is the only available curative therapy for chronic granulomatous disease (CGD), but its use is limited by transplant-related mortality (TRM) in patients who often come to transplant with existing infections or organ dysfunction. Reduction in the intensity of the preparative regimen mitigates these risks, but increases the potential for mixed donor-recipient chimerism (MC) that may progress to graft loss. Recently a busulfan-based reduced-intensity conditioning (RIC) regimen has been described with excellent survival and little MC. We report our experience with a similar RIC regimen at our institution, demonstrating problems with donor chimerism and graft loss. Pediatr Blood Cancer 2015;62:359-361. © 2014 Wiley Periodicals, Inc.
authors.affiliation: Divisions of Oncology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Oshrine
authors.affiliation: Divisions of Immunology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
authors.forename: Megan
authors.initials: M
authors.lastname: Morsheimer
authors.affiliation: Divisions of Immunology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Heimall
authors.affiliation: Divisions of Oncology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
authors.forename: Nancy
authors.initials: N
authors.lastname: Bunin
mesh_terms: D000074323:Alemtuzumab; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002066:Busulfan / Q000627:therapeutic use*; D002675:Child, Preschool; D006086:Graft vs Host Disease / Q000401:mortality / Q000517:prevention & control*; D006105:Granulomatous Disease, Chronic / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008562:Membrane Glycoproteins / Q000235:genetics; D000074662:NADPH Oxidase 2; D019255:NADPH Oxidases / Q000235:genetics; D019172:Transplantation Conditioning / Q000379:methods*; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D008562:Membrane Glycoproteins; D000074323:Alemtuzumab; C056558:CYBB protein, human; D000074662:NADPH Oxidase 2; D019255:NADPH Oxidases; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine
keywords: chronic granulomatous disease; hematopoietic cell transplantation; primary immunodeficiency; reduced-intensity conditioning