title: Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.
abstract: We conducted a prospective, multicenter investigation of human-leukocyte antigen (HLA) identical sibling bone marrow transplantation (BMT) in children with severe sickle cell disease (SCD) between 1991 and 2000. To determine if children were protected from complications of SCD after successful BMT, we extended our initial study of BMT for SCD to conduct assessments of the central nervous system (CNS) and of pulmonary function 2 or more years after transplantation. In addition, the impact on gonadal function was studied. After BMT, patients with stroke who had stable engraftment of donor cells experienced no subsequent stroke events after BMT, and brain magnetic resonance imaging (MRI) exams demonstrated stable or improved appearance. However, 2 patients with graft rejection had a second stroke after BMT. After transplantation, most patients also had unchanged or improved pulmonary function. Among the 11 patients who had restrictive lung changes at baseline, 5 were improved and 6 had persistent restrictive disease after BMT. Of the 2 patients who had obstructive changes at baseline, 1 improved and 1 had worsened obstructive disease after BMT. There was, however, significant gonadal toxicity after BMT, particularly among female recipients. In summary, individuals who had stable donor engraftment did not experience sickle-related complications after BMT, and were protected from progressive CNS and pulmonary disease.
authors.affiliation: Blood and Marrow Transplantation Program, Children's Hospital & Research Center, Oakland, California 94609, USA. mwalters@mail.cho.org
authors.forename: Mark C
authors.initials: MC
authors.lastname: Walters
authors.forename: Karen
authors.initials: K
authors.lastname: Hardy
authors.forename: Sandie
authors.initials: S
authors.lastname: Edwards
authors.forename: Thomas
authors.initials: T
authors.lastname: Adamkiewicz
authors.forename: James
authors.initials: J
authors.lastname: Barkovich
authors.forename: Francoise
authors.initials: F
authors.lastname: Bernaudin
authors.forename: George R
authors.initials: GR
authors.lastname: Buchanan
authors.forename: Nancy
authors.initials: N
authors.lastname: Bunin
authors.forename: Roswitha
authors.initials: R
authors.lastname: Dickerhoff
authors.forename: Roger
authors.initials: R
authors.lastname: Giller
authors.forename: Paul R
authors.initials: PR
authors.lastname: Haut
authors.forename: John
authors.initials: J
authors.lastname: Horan
authors.forename: Lewis L
authors.initials: LL
authors.lastname: Hsu
authors.forename: Naynesh
authors.initials: N
authors.lastname: Kamani
authors.forename: John E
authors.initials: JE
authors.lastname: Levine
authors.forename: David
authors.initials: D
authors.lastname: Margolis
authors.forename: Kwaku
authors.initials: K
authors.lastname: Ohene-Frempong
authors.forename: Melinda
authors.initials: M
authors.lastname: Patience
authors.forename: Rupa
authors.initials: R
authors.lastname: Redding-Lallinger
authors.forename: Irene A G
authors.initials: IA
authors.lastname: Roberts
authors.forename: Zora R
authors.initials: ZR
authors.lastname: Rogers
authors.forename: Jean E
authors.initials: JE
authors.lastname: Sanders
authors.forename: J Paul
authors.initials: JP
authors.lastname: Scott
authors.forename: Keith M
authors.initials: KM
authors.lastname: Sullivan
mesh_terms: D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000150:complications / Q000503:physiopathology / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects*; D002493:Central Nervous System Diseases / Q000209:etiology* / Q000503:physiopathology; D002648:Child; D046148:Donor Selection; D005260:Female; D005500:Follow-Up Studies; D006058:Gonadal Disorders / Q000209:etiology* / Q000503:physiopathology; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000209:etiology / Q000401:mortality; D006680:HLA Antigens / Q000276:immunology; D006304:Health Status; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006650:Histocompatibility Testing; D006801:Humans; D008173:Lung Diseases, Obstructive / Q000209:etiology* / Q000503:physiopathology; D008297:Male; D035781:Siblings; D016019:Survival Analysis; D018183:Transplantation Chimera; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens