Medical College of Wisconsin
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Hematopoietic stem cell transplantation for sickle cell disease: state of the science. Eur J Haematol 2015 May;94(5):391-9

Date

09/10/2014

Pubmed ID

25200500

DOI

10.1111/ejh.12447

Scopus ID

2-s2.0-84926509714 (requires institutional sign-in at Scopus site)   31 Citations

Abstract

Sickle cell disease (SCD) is an inherited disorder secondary to a point mutation at the sixth position of the beta chain of human hemoglobin resulting in the replacement of valine for glutamic acid. This recessive genetic abnormality precipitates the polymerization of the deoxygenated form of hemoglobin S inducing a major distortion of red blood cells (S-RBC), which decreases S-RBC deformability leading to chronic hemolysis and vaso-occlusion. These processes can result in severe complications including chronic pain, end-organ dysfunction, stroke, and early mortality. The only proven curative therapy for patients with SCD is myeloablative conditioning and allogeneic stem cell transplantation from HLA-matched sibling donors. In this review, we discuss the most recent advances in allogeneic stem cell transplantation in patients with SCD including more novel approaches such as reduced toxicity conditioning and the use of alternative allogeneic donors, including matched unrelated donors (MUDs), unrelated cord blood donors (UCBT), and familial haploidentical (FHI) donors. The results to date are very encouraging regarding allogeneic stem cell transplantation for patients with SCD including high survival rates and enabling a greater number of patients suffering from this chronic and debilitating condition to receive curative allogeneic stem cell therapies. However, we still have several areas to investigate and barriers to overcome to successfully cure the majority of patients with severe SCD through allogeneic stem cell therapies.

Author List

Talano JA, Cairo MS

Author

Julie-An M. Talano MD Professor in the Pediatrics department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Anemia, Sickle Cell
Chronic Pain
Cord Blood Stem Cell Transplantation
Hematopoietic Stem Cell Transplantation
Hemoglobin, Sickle
Histocompatibility Testing
Humans
Myeloablative Agonists
Protein Aggregation, Pathological
Siblings
Transplantation Conditioning
Transplantation, Homologous
Unrelated Donors