Stem Cell Transplantation in Patients with Sickle Cell Disease

Abstract

Hematopoietic stem cell transplantation (HSCT) is currently the only established cure for sickle cell disease (SCD). Replacement of the stem cell that has the defective beta globin allele with the normal gene decreases hemoglobin S and the risk of complications of SCD. The irst case reported was a girl with acute myeloid leukemia and SCD who received HSCT and achieved long-term SCD and leukemia-free survival. Given the favorable outcomes of HSCT with thalassemia major using myeloablative preparative regimens, this approach became widely used in the initial studies of HSCT in SCD. The current standard of care is to use a myeloablative stem cell transplantation in patients with severe disease who have human leukocyte antigen-identical sibling. HSCT improves organ function, quality of life, and overall and disease-free survival. However, this is associated with high risk of gonadal dysfunction and graft versus host disease in addition to the mortality associated with the myeloablative HSCT. Reduced-intensity HSCT has also been reported with high rates of engraftment and favorable outcomes. This has been introduced to lower the gonadal dysfunction, mortality, and graft versus host disease associated with myeloablative approaches. Other approaches include HSCT using matched unrelated donors, cord blood units, and human leukocyte antigen haploidentical donors. Unfortunately, graft rejection is a common complication with these approaches. In this chapter, we review the indications of HSCT for SCD and outcomes of diferent transplant strategies including alternative donor transplant, graft rejection, and infertility after transplantation.

Sickle Cell Disease: Advances in Treatment

“…Transplant has been performed, for the most part, in patients who have suffered a stroke, have had multiple episodes of acute chest syndrome, or have had recurrent vaso-occlusive crises (≥3 episodes requiring hospitalization per year), ie, patients considered to have the worst disease severity. 125 Controversies have arisen not only about whom to transplant but also about the optimal age to transplant, source of donor cells, and type of conditioning regimen. [126][127][128][129][130] Most stem cell transplants thus far have relied upon myeloablative conditioning regimens and have been bone marrow-derived with human leukocyte antigen (HLA)matched sibling donors as the source of stem cells.…”

Effectiveness of Nurse Led Intervention on Health Related Quality of Life among Children with Sickle Cell Disease in Oman: A Pilot Study

“…is study also reported promotion and educational programmes are needed in order to increase the public awareness on SCD and the value of Premarital Screening (PMS) [34]. A study conducted in Oman shows that Hematopoietic stem cell transplantation (HSCT) improves organ function, quality of life, and facilitates disease-free survival [35]. ough, nonnursing interventions supports the improvement of HRQOL of children with SCD, no nurse-led, culturally-tailored, interventional studies have been conducted in Oman in order to improve the HRQOL of Omani children.…”