Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.
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.
Walters
Mark C
MC
Blood and Marrow Transplantation Program, Children's Hospital & Research Center, Oakland, California 94609, USA. mwalters@mail.cho.org
Hardy
Karen
K
Edwards
Sandie
S
Adamkiewicz
Thomas
T
Barkovich
James
J
Bernaudin
Francoise
F
Buchanan
George R
GR
Bunin
Nancy
N
Dickerhoff
Roswitha
R
Giller
Roger
R
Haut
Paul R
PR
Horan
John
J
Hsu
Lewis L
LL
Kamani
Naynesh
N
Levine
John E
JE
Margolis
David
D
Ohene-Frempong
Kwaku
K
Patience
Melinda
M
Redding-Lallinger
Rupa
R
Roberts
Irene A G
IA
Rogers
Zora R
ZR
Sanders
Jean E
JE
Scott
J Paul
JP
Sullivan
Keith M
KM
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
D006680:HLA Antigens