Hematopoietic cell transplantation: a curative option for sickle cell disease.
Sickle cell disease is associated with considerable morbidity and premature mortality. Hematopoietic cell transplantation offers the possibility of cure and is associated with excellent results in pediatric patients receiving stem cell transplantation from a matched sibling donor. Reduced intensity conditioning regimen have the potential to further reduce regimen related morbidity and mortality. Improved understanding of the natural history of complications such as stroke and pulmonary hypertension, effects of treatments, such as hydroxyurea and blood transfusions, as well as the impact of transplantation on organ damage are likely to influence the timing and indication of transplantation. Improvements in preparative regimen may enable the safe use of alternate source of stem cells such as unrelated matched donors and further improve the applicability and acceptability of this treatment.
Krishnamurti
Lakshmanan
L
Division of Hematology/Oncology/BMT Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System, Pittsburgh, Pennsylvania 15213, USA. krislx@chp.edu
D000755:Anemia, Sickle Cell / Q000150:complications / Q000401:mortality / Q000628:therapy*; D000986:Antisickling Agents / Q000627:therapeutic use; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D006976:Hypertension, Pulmonary / Q000209:etiology / Q000401:mortality / Q000628:therapy; D008297:Male; D020521:Stroke / Q000209:etiology / Q000401:mortality / Q000628:therapy; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous
D000986:Antisickling Agents; D006918:Hydroxyurea