Hematopoietic cell transplantation for sickle cell disease: updates and future directions

Abstract

Excellent outcomes 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 applicability and acceptability of HCT for sickle cell disease (SCD). In young children with symptomatic SCD with an available HLA-identical related donor, HCT should be carefully considered. HCT from alternative donors is typically undertaken only in patients with severe symptoms, causing or likely to cause organ damage, and in the context of clinical trials. Patients undergoing HCT for SCD require careful counseling and preparation. They require careful monitoring of unique organ toxicities and complications during HCT. Patients must be prospectively followed for a prolonged time to determine the long-term outcomes and late effects of HCT for SCD. Thus, there is a need for a universal, longitudinal clinical registry to follow patients after HCT for SCD in conjunction with individuals who do not receive HCT to compare outcomes. Antibody-based conditioning and ex-vivo umbilical cord blood expansion are likely to improve the availability and acceptability of HCT. In addition, new disease-modifying drugs and the emerging option of the autologous transplantation of gene-modified hematopoietic progenitor cells are likely to expand the available therapeutic options and make decision-making by patients, physicians, and caregivers even more complicated. Future efforts must also focus on determining the impact of socioeconomic status on access to and outcomes of HCT and the long-term impact of HCT on patients, families, and society.

Hoping for a normal life: Decision‐making on hematopoietic stem cell transplantation by patients with a hemoglobinopathy and their caregivers

“…1,2 Currently, hematopoietic stem cell transplantation (HSCT) is the only established curative therapy option for patients with TDT and SCD. 3 Lately, gene therapy has emerged as a promising alternative 1,4 ; however, its accessibility still hampers its widespread clinical use. 5 A recent study showed that the appraisal of HSCT decision-making for patients with a hemoglobinopathy significantly varied among professionals in a scenario-based survey.…”

“…Currently, hematopoietic stem cell transplantation (HSCT) is the only established curative therapy option for patients with TDT and SCD 3 . Lately, gene therapy has emerged as a promising alternative 1,4 ; however, its accessibility still hampers its widespread clinical use 5 …”