Hematopoietic stem cell transplant therapy, clinical trials, complications, and quality of life for patients with Sickle cell anemia: Clinical potential and future perspectives

Abstract

Background: Sickle cell anemia (SCA) is an inherited monogenic disorder. The clinical symptoms of SCA are protean, including vaso-occlusion, hemolysis, early stroke, leg ulcers, multi-organ failure, and increased risk of premature death. Hematopoietic stem cell transplantation is the only treatment identified to reduce SCA-related organ damage. Unfortunately, graft rejection is a significant impediment to these strategies. Materials and Methods: The current standard of treatment for the past two decades is limited to myeloablative-matched sibling donors, which is likely to be only for minor patients and is feasible for non-malignant giant disease. Cumulative studies showed that HSCT increases overall survival and quality of life in patients with SCA. Results: Hematopoietic stem cell transplantation (HSCT) is significantly associated with a higher risk of graft versus host disease and moderate mortality risk. New strategy lacking standard donors includes cord blood, matched unrelated donors/ Haploidentical donors. Conclusion: This review summarized evidence from HSCT clinical trials from different transplantation methods, specific HSCT and HSCT-related health problems that need to be addressed in medical contexts with patients and family members, and other areas that enhance the quality of life in SCA.