title: Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide in a Patient with Chronic Granulomatous Disease and Active Infection: A First Report.
abstract: OBJECTIVE
  We describe haploidentical hematopoietic cell transplantation (HCT) with high-dose post-transplant cyclophosphamide (PTCy) in a boy with x-linked chronic granulomatous disease (CGD).
  
  
  METHODS
  A persistent and life-threatening fungal infection was the indication for HSCT. Non-myeloablative conditioning with PTCy (50 mg/kg days 3 and 4) was used in the absence of fully matched donors.
  
  
  RESULTS
  Engraftment occurred on day 24. The patient experienced Grade 2 graft-versus-host disease of the skin and gastrointestinal tract and CMV infection, both of which were controlled. Chimerism was 100 % at days 30 and 6 months. Cessation of antifungal therapy was consistent with cure of the infection.
  
  
  CONCLUSIONS
  Haploidentical HCT with high-dose PTCy for CGD is feasible and succeeded even in the context of active infection.
authors.affiliation: Leidos Biomedical Research, Inc, Frederick National Laboratory for Cancer Research, 10 Center Dr., Rm. 6-3754, MSC 1763, Bethesda, MD, 20892-1456, USA. mark.parta@nih.gov.
authors.forename: Mark
authors.initials: M
authors.lastname: Parta
authors.affiliation: National Institute of Allergy and Infectious Diseases/National Institutes of Health, Bethesda, MD, USA.
authors.forename: Dianne
authors.initials: D
authors.lastname: Hilligoss
authors.affiliation: National Institute of Allergy and Infectious Diseases/National Institutes of Health, Bethesda, MD, USA.
authors.forename: Corin
authors.initials: C
authors.lastname: Kelly
authors.affiliation: National Institute of Allergy and Infectious Diseases/National Institutes of Health, Bethesda, MD, USA.
authors.forename: Nana
authors.initials: N
authors.lastname: Kwatemaa
authors.affiliation: National Institute of Allergy and Infectious Diseases/National Institutes of Health, Bethesda, MD, USA.
authors.forename: Narda
authors.initials: N
authors.lastname: Theobald
authors.affiliation: National Institute of Allergy and Infectious Diseases/National Institutes of Health, Bethesda, MD, USA.
authors.forename: Harry
authors.initials: H
authors.lastname: Malech
authors.affiliation: National Institute of Allergy and Infectious Diseases/National Institutes of Health, Bethesda, MD, USA.
authors.forename: Elizabeth M
authors.initials: EM
authors.lastname: Kang
mesh_terms: D001804:Blood Transfusion, Autologous; D046528:Chimerism / Q000187:drug effects; D003520:Cyclophosphamide / Q000008:administration & dosage*; D054796:Drug Dosage Calculations; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control*; D006105:Granulomatous Disease, Chronic / Q000150:complications / Q000628:therapy*; D006680:HLA Antigens / Q000276:immunology; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D007223:Infant; D008297:Male; D009181:Mycoses / Q000209:etiology / Q000628:therapy*; D009316:National Institutes of Health (U.S.); D011183:Postoperative Complications / Q000517:prevention & control*; D021681:Scedosporium; D019172:Transplantation Conditioning; D014481:United States
chemical_list: D006680:HLA Antigens; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide
keywords: Chronic granulomatous disease; graft versus host disease; haploidentical; hematopoietic cell transplantation; post-transplant cyclophosphamide; scedosporium apiospermum