Toward dual hematopoietic stem-cell transplantation and solid-organ transplantation for sickle-cell disease

Abstract

Sickle-cell disease (SCD) leads to recurrent vaso-occlusive crises, chronic end-organ damage, and resultant physical, psychological, and social disabilities. Although hematopoietic stem-cell transplantation (HSCT) is potentially curative for SCD, this procedure is associated with well-recognized morbidity and mortality and thus is ideally offered only to patients at high risk of significant complications. However, it is difficult to identify patients at high risk before significant complications have occurred, and once patients experience significant organ damage, they are considered poor candidates for HSCT. In turn, patients who have experienced long-term organ toxicity from SCD such as renal or liver failure may be candidates for solid-organ transplantation (SOT); however, the transplanted organs are at risk of damage by the original disease. Thus, dual HSCT and organ transplantation could simultaneously replace the failing organ and eliminate the underlying disease process. Advances in HSCT conditioning such as reduced-intensity regimens and alternative donor selection may expand both the feasibility of and potential donor pool for transplantation. This review summarizes the current state of HSCT and organ transplantation in SCD and discusses future directions and the clinical feasibility of dual HSCT/SOT.

Management of Sickle Cell Intrahepatic Cholestasis: An Argument in Favor of Automated Exchange Transfusion

“…SCT is likewise associated with the risk of graft versus host disease, as well as hepatic veno-oclusive disease [ 37 ]. Dual SCT/OLT has been described in other disease processes, and may be an avenue explored in SCD patients in the coming years [ 1 ].…”

“…Sickle cell disease (SCD) is the most common hemoglobinopathy, and there are approximately 100,000 patients in the United States with the disease [ 1 , 2 ]. This results in a major impact on healthcare, with between 68 and 75,000 hospitalizations a year, and approximately 100,000 emergency department visits, with an estimated total cost of care of $2.4 billion [ 3 ].…”

The Role of CTLA4 and Its Polymorphisms in Solid Organ and Haematopoietic Stem Cell Transplantation

“…Moreover, some rarer serious complications involve specific groups of patients, such as children [ 4 , 5 , 6 ]. Recipients of dual transplantations might be an even greater challenge for doctors [ 7 , 8 ]. Those challenges indicate that there is an immense demand for progress in the field of transplantology [ 3 ].…”

Conceptual DFT study of the chemical reactivity of four natural products with anti-sickling activity

“…It was reported that Africa possesses the major affected patients estimated to be about 50 millions [3,4]. Nowadays, bone marrow transplant, also known as stem cell transplant, is mostly considered as potential cure for sickle cell anemia [8][9][10][11][12][13]. Stem cell transplant from a compatible donor was found more recently to be successful for sicklers patients becoming SCD-free after treatment [8][9][10].…”