TY - JOUR
T1 - Blood and marrow transplantation for sickle cell disease
T2 - Overcoming barriers to success
AU - Bolaños-Meade, Javier
AU - Brodsky, Robert A.
PY - 2009/3
Y1 - 2009/3
N2 - Purpose of review Sickle cell disease (SCD) is a common health problem in the United States; yet, the only curative therapy, a bone marrow transplant (BMT), is seldom applied. The objective of this report is to review the most recent clinical trials involving blood and BMT for SCD and to discuss novel approaches to overcome the many barriers to successful use of BMT for SCD. Recent findings In select patients, disease-free and overall survival is greater than 80% following matched sibling BMT for SCD. Unfortunately, most patients with SCD do not have a suitable human lymphocyte antigen-matched sibling donor. In an attempt to expand the donor pool, several groups are beginning to explore the use of alternative sources of stem cells such as haploidentical donors and umbilical cord cell blood. Summary The curative potential of BMT in SCD is irrefutable, with outstanding results in children following a myeloablative conditioning regimen and a matched sibling donor transplant. Well tolerated and effective application of alternative sources of stem cells for BMT in SCD could greatly increase the cure rate for this devastating disease.
AB - Purpose of review Sickle cell disease (SCD) is a common health problem in the United States; yet, the only curative therapy, a bone marrow transplant (BMT), is seldom applied. The objective of this report is to review the most recent clinical trials involving blood and BMT for SCD and to discuss novel approaches to overcome the many barriers to successful use of BMT for SCD. Recent findings In select patients, disease-free and overall survival is greater than 80% following matched sibling BMT for SCD. Unfortunately, most patients with SCD do not have a suitable human lymphocyte antigen-matched sibling donor. In an attempt to expand the donor pool, several groups are beginning to explore the use of alternative sources of stem cells such as haploidentical donors and umbilical cord cell blood. Summary The curative potential of BMT in SCD is irrefutable, with outstanding results in children following a myeloablative conditioning regimen and a matched sibling donor transplant. Well tolerated and effective application of alternative sources of stem cells for BMT in SCD could greatly increase the cure rate for this devastating disease.
KW - Bone marrow transplantation
KW - Haploidentical bone marrow transplant
KW - Sickle cell disease
KW - Umbilical cord transplantation
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U2 - 10.1097/CCO.0b013e328324ba04
DO - 10.1097/CCO.0b013e328324ba04
M3 - Review article
C2 - 19532018
AN - SCOPUS:62349100621
SN - 1040-8746
VL - 21
SP - 158
EP - 161
JO - Current opinion in oncology
JF - Current opinion in oncology
IS - 2
ER -