HEMATOPOIETIC CELL TRANSPLANTATION: A Curative Option for Sickle Cell Disease

Abstract

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.

Barriers to Hematopoietic Cell Transplantation Clinical Trial Participation of African American and Black Youth With Sickle Cell Disease and Their Parents

“…11–13 Barriers to widespread applicability of this treatment modality have included risks of early and long-term toxicity and uncertainty expressed by clinicians regarding appropriate selection of patients. 11,14 Lack of a suitable HLA-identical donor has also been identified as a major barrier to HCT.…”

How I use hydroxyurea to treat young patients with sickle cell anemia

“…The recent observation that mortality rates for older children with SCA have not changed in 20 years 96 indicates the need for new approaches to disease management, especially regarding therapeutic intervention. At the current time, curative therapy with stem cell transplantation remains an unavailable option to most patients with SCA, 97 although newer successful approaches may increase interest in this modality. 98 Until something better becomes available that has a similar wide spectrum of efficacy and safety, hydroxyurea appears to be the best available treatment option for children and adolescents with SCA.…”

Minor histocompatibility antigens on transfused leukoreduced units of red blood cells induce bone marrow transplant rejection in a mouse model

“…[9][10][11][12] One factor that potentially contributes to rejection is the requirement for chronic transfusion therapy. There is a strong correlation between the number of transfusions a patient has received and the likelihood of BMT rejection.…”