Hematopoietic cell transplantation for sickle cell dis ease: Updates and future directions

Abstract

Excellent out comes in hematopoietic cell transplantation (HCT) from HLA - identical siblings, improvements in conditioning regimens, novel graft - versus- host disease prophylaxis, and the availability of alternative donors have all contributed to the increased appli ca bil ity and accept abil ity of HCT for sickle cell dis ease (SCD). In young chil dren with symp tom atic SCD with an avail able HLA - iden ti cal related donor, HCT should be care fully con sid ered. HCT from alter na tive donors is typ i cally under taken only in patients with severe symp toms, caus ing or likely to cause organ dam age, and in the con text of clin i cal tri als. Patients under go ing HCT for SCD require care ful coun sel ing and prep a ra tion. They require care ful mon itor ing of unique organ toxicities and com pli ca tions dur ing HCT. Patients must be pro spec tively followed for a prolonged time to deter mine the long - term out comes and late effects of HCT for SCD. Thus, there is a need for a uni ver sal, lon gitu di nal clin i cal reg is try to fol low patients after HCT for SCD in con junc tion with indi vid u als who do not receive HCT to com pare out comes. Antibody - based con di tion ing and ex - vivo umbil i cal cord blood expan sion are likely to improve the avail abil ity and accept abil ity of HCT. In addi tion, new dis ease - mod i fy ing drugs and the emerg ing option of the autologous transplantation of gene- modifi ed hematopoietic progenitor cells are likely to expand the avail able ther apeutic options and make deci sion - mak ing by patients, phy si cians, and care giv ers even more com pli cated. Future efforts must also focus on deter min ing the impact of socio eco nomic sta tus on access to and out comes of HCT and the long - term impact of HCT on patients, fam i lies, and society.