Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research.
We report outcomes after myeloablative haematopoietic cell transplantation (HCT) from human leucocyte antigen (HLA)-matched sibling donors in 67 patients with sickle cell disease transplanted between 1989 and 2002. The most common indications for transplantation were stroke and recurrent vaso-occlusive crisis in 38% and 37% of patients respectively. The median age at transplantation was 10 years and 67% of patients had received >10 red blood cell transfusions before HCT. Twenty-seven percent of patients had a poor performance score at transplantation. Ninety-four percent received busulfan and cyclophosphamide-containing conditioning regimens and bone marrow was the predominant source of donor cells. Most patients achieved haematopoietic recovery and no deaths occurred during the early post-transplant period. Rates of acute and chronic graft-versus-host disease were 10% and 22% respectively. Sixty-four of 67 patients are alive with 5-year probabilities of disease-free and overall survival of 85% and 97% respectively. Nine patients had graft failure with recovery of sickle erythropoiesis, eight of who had recurrent sickle-related events. This report confirms and extends earlier reports that HCT from HLA-matched related donors offers a very high survival rate, with few transplant-related complications and the elimination of sickle-related complications in the majority of patients who undergo this therapy.
Panepinto
Julie A
JA
Department of Hematology/Oncology/Bone Marrow Transplant, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226, USA. jpanepin@mcw.edu
Walters
Mark C
MC
Carreras
Jeanette
J
Marsh
Judith
J
Bredeson
Christopher N
CN
Gale
Robert Peter
RP
Hale
Gregory A
GA
Horan
John
J
Hows
Jill M
JM
Klein
John P
JP
Pasquini
Ricardo
R
Roberts
Irene
I
Sullivan
Keith
K
Eapen
Mary
M
Ferster
Alina
A
D000755:Anemia, Sickle Cell / Q000276:immunology / Q000401:mortality / Q000601:surgery*; D018572:Disease-Free Survival; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D007958:Leukocyte Count; D009504:Neutrophils / Q000276:immunology; D011336:Probability; D012042:Registries; D015996:Survival Rate; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous