Bone marrow transplantation for sickle cell disease.
BACKGROUND
We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease.


METHODS
Twenty-two children less than 16 years of age who had symptomatic sickle cell disease received marrow allografts from HLA-identical siblings between September 1991 and April 1995. The indications for transplantation included a history of stroke (n = 12), recurrent acute chest syndrome (n = 5), and recurrent painful crises (n = 5). Patients were prepared for transplantation with busulfan, cyclophosphamide, and antithymocyte globulin.


RESULTS
Twenty of the 22 patients survived, with a median follow-up of 23.9 months (range, 10.1 to 51.0), and 16 patients had stable engraftment of donor hematopoietic cells. In three patients the graft was rejected and sickle cell disease recurred; in a fourth patient graft rejection was accompanied by marrow aplasia. In 1 of the 16 patients with engraftment, there was stable mixed chimerism. Two patients died of central nervous system hemorrhage or graft-versus-host disease. Kaplan-Meier estimates of survival and event-free survival at four years were 91 percent and 73 percent, respectively. Among patients with a history of acute chest syndrome, lung function stabilized; among patients with prior central nervous system vasculopathy who had engraftment, stabilization of cerebrovascular disease was documented by magnetic resonance imaging.


CONCLUSIONS
Allogeneic stem-cell transplantation can be curative in young patients with symptomatic sickle cell disease.
Walters
M C
MC
Division of Clinical Research, Fred Hutchinson Cancer Research Center, Seattle, WA 98104, USA.
Patience
M
M
Leisenring
W
W
Eckman
J R
JR
Scott
J P
JP
Mentzer
W C
WC
Davies
S C
SC
Ohene-Frempong
K
K
Bernaudin
F
F
Matthews
D C
DC
Storb
R
R
Sullivan
K M
KM
D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000150:complications / Q000401:mortality / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002543:Cerebral Hemorrhage / Q000209:etiology / Q000401:mortality; D002561:Cerebrovascular Disorders / Q000209:etiology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006084:Graft Rejection; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D008297:Male; D016019:Survival Analysis; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000150:complications / Q000628:therapy