Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: A Review
Author(s): Dinesh Pendharkar MD PhD FASCO* and Garima Nirmal MD DM
Sickle cell disease is an autosomal recessive disorder widely spread over the world. The disease is associated with continuing morbidity, mutiorgan damage, and mortality. Hematopoietic stem cell transplantation is the only established curative therapy. The probability of 5-year survival post HSCT extends beyond 90%. The therapy is continuously evolving in all aspects of transplantation. This review focuses on the recent trends in multiple facets of the procedure from indications to details of newer technologies. It reviews data related to age at transplantation, indications as proposed by various researchers, donor selection, multiplicity of conditioning regimens, various immunosuppressive methodologies in use and expected outcomes. Transplant at an early age is recommended if MSD is available. The complications profile differs, depending on the age. Practically every single serious complication requiring admission in SCD, is an indication for the transplant. The major challenges faced in HSCT include non-acceptance by the patients and non-availability of fully matched donors. The technology is completely gearing up to make haploidentical transplants more successful with lesser side effects like GVHD, rejections and infections. Different intensity levels of conditioning regimens are in use. Various manipulations with T cells also form the part of transplant. The review reinforces the current knowledge about methodologies, utility and feasibility of HSCT in SCD.