title: Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease.
abstract: BACKGROUND
  Matched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a successful treatment for chronic granulomatous disease (CGD), but the safety and efficacy of HSCT from unrelated donors is less certain.
  
  
  OBJECTIVE
  We evaluated the outcomes and overall survival in patients with CGD after HSCT.
  
  
  METHODS
  We report the outcomes for 11 children undergoing HSCT from an MRD (n = 4) or an HLA-matched unrelated donor (MUD) (n = 7); 9 children were boys, and the median age was 3.8 years (range, 1-13 years). We treated both X-linked (n = 9) and autosomal recessive (n = 2) disease. Nine children had serious clinical infections before transplantation. The conditioning regimens contained busulfan, cyclophosphamide, cytarabine, or fludarabine according to the donor used. All patients received alemtuzumab (anti-CD52 antibody). Additional graft-versus-host disease (GvHD) prophylaxis included cyclosporine and methotrexate for MUD recipients and cyclosporine and prednisone for MRD recipients.
  
  
  RESULTS
  Neutrophil recovery took a median of 16 days (range, 12-40 days) and 18 days (range, 13-24 days) for MRD and MUD recipients, respectively. Full donor neutrophil engraftment occurred in 9 patients, and 2 had stable mixed chimerism; all patients had sustained correction of neutrophil oxidative burst defect. Four patients had grade I skin acute GVHD responding to topical treatment. No patient had grade II to IV acute GvHD or chronic GvHD. All patients are alive between 1 and 8 years after HSCT.
  
  
  CONCLUSIONS
  For CGD, equivalent outcomes can be obtained with MRD or MUD stem cells, and HSCT should be considered an early treatment option.
authors.affiliation: Center for Cell and Gene Therapy, Baylor College of Medicine, The Methodist Hospital, and Texas Children's Hospital, Houston, Tex 77030, USA. camartin@txch.org
authors.forename: Caridad A
authors.initials: CA
authors.lastname: Martinez
authors.forename: Sweta
authors.initials: S
authors.lastname: Shah
authors.forename: William T
authors.initials: WT
authors.lastname: Shearer
authors.forename: Howard M
authors.initials: HM
authors.lastname: Rosenblatt
authors.forename: Mary E
authors.initials: ME
authors.lastname: Paul
authors.forename: Javier
authors.initials: J
authors.lastname: Chinen
authors.forename: Kathryn S
authors.initials: KS
authors.lastname: Leung
authors.forename: Alana
authors.initials: A
authors.lastname: Kennedy-Nasser
authors.forename: Malcolm K
authors.initials: MK
authors.lastname: Brenner
authors.forename: Helen E
authors.initials: HE
authors.lastname: Heslop
authors.forename: Hao
authors.initials: H
authors.lastname: Liu
authors.forename: Meng-Fen
authors.initials: MF
authors.lastname: Wu
authors.forename: Imelda C
authors.initials: IC
authors.lastname: Hanson
authors.forename: Robert A
authors.initials: RA
authors.lastname: Krance
mesh_terms: D000203:Activities of Daily Living; D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004522:Educational Status; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control; D006105:Granulomatous Disease, Chronic / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011788:Quality of Life; D012801:Sibling Relations; D014019:Tissue Donors; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D061349:Unrelated Donors
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