Stem-cell transplantation in children and adults with sickle cell disease: an update

Abstract

Sickle cell disease (SCD) is associated with significant morbidity, a decreased lifespan and a poor quality of life. While there is increasing evidence that hydroxyurea can improve the course of severe SCD, hematopoeitic stem-cell transplantation (HSCT) remains the only curative option for SCD. Multicenter trials have shown that HSCT after myeloablative conditioning has excellent outcomes in children with SCD, with an overall survival ranging from 93 to 97% and an event-free survival between 82 and 86%. With better understanding of the course of SCD in adulthood, there has been increasing interest in making HSCT a viable intervention in adults. Nonetheless, older patients with severe disease have not been considered suitable candidates because of the higher risks associated with myeloablative conditioning. Recently, reduced-intensity regimens have been used in adults with good results, albeit in a small number of patients. The main limitation of HSCT in both adults and children with SCD remains the lack of availability of fully matched HLA sibling donors for patients meeting transplant criteria.

Keywords::

• hematopoietic stem-cell transplantation
• reduced-intensity conditioning
• sickle cell disease
• umbilical cord blood

Acknowledgements

The authors would like to acknowledge Robert Habib for his expert statistical assistance in preparingFigure 1. Miguel Abboud and Ruby Khoury would also like to thank the Children’s Cancer Center of Lebanon and the International Outreach Program at St Jude’s Children’s Research Hospital, who supported them during the writing of this article.

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.