TY - JOUR
T1 - Hematopoietic stem cell transplantation for sickle cell disease
T2 - current practice and emerging trends.
AU - Shenoy, Shalini
PY - 2011
Y1 - 2011
N2 - Allogeneic HSCT controls sickle cell disease (SCD)-related organ damage and is currently the only curative therapy available. Over the last 2 decades, HSCT has been limited largely to myeloablative matched sibling donor (MSD) procedures that are feasible only in a minority of patients. As the natural history of the disease has evolved, it is clear that subsets of patients with severe disease are at risk for sudden death, devastating CNS and pulmonary complications, and debilitating vasoocclusive crises. For these patients, the benefits of transplantation can outweigh the risks if HSCT can be safely and successfully performed with low early and late toxicities. This review describes advances and ongoing investigation of HSCT for SCD from the perspectives of recipient age and presentation, donor stem cell source, intensity of conditioning, family and medical perspectives, and other variables that influence outcome. Ultimately, HSCT should be viewed as a viable treatment option for SCD on par with other therapies for select patients who can benefit from the procedure.
AB - Allogeneic HSCT controls sickle cell disease (SCD)-related organ damage and is currently the only curative therapy available. Over the last 2 decades, HSCT has been limited largely to myeloablative matched sibling donor (MSD) procedures that are feasible only in a minority of patients. As the natural history of the disease has evolved, it is clear that subsets of patients with severe disease are at risk for sudden death, devastating CNS and pulmonary complications, and debilitating vasoocclusive crises. For these patients, the benefits of transplantation can outweigh the risks if HSCT can be safely and successfully performed with low early and late toxicities. This review describes advances and ongoing investigation of HSCT for SCD from the perspectives of recipient age and presentation, donor stem cell source, intensity of conditioning, family and medical perspectives, and other variables that influence outcome. Ultimately, HSCT should be viewed as a viable treatment option for SCD on par with other therapies for select patients who can benefit from the procedure.
UR - http://www.scopus.com/inward/record.url?scp=84859410473&partnerID=8YFLogxK
U2 - 10.1182/asheducation-2011.1.273
DO - 10.1182/asheducation-2011.1.273
M3 - Review article
C2 - 22160045
AN - SCOPUS:84859410473
VL - 2011
SP - 273
EP - 279
JO - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
JF - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
SN - 1520-4391
ER -