Hematopoietic Cell Transplantation for Sickle Cell Disease

Area of Science:

• Hematology
• Transplantation Medicine
• Genetic Medicine

Background:

• Sickle cell disease (SCD) is a severe inherited blood disorder causing red blood cell deformation and vaso-occlusion.
• Pathological changes in SCD lead to tissue damage, organ dysfunction, and reduced survival.
• Hematopoietic cell transplantation (HCT) is a curative or ameliorative treatment for SCD.

Purpose of the Study:

• To review the current landscape of HCT for sickle cell disease.
• To discuss evolving HCT strategies, including alternative donor sources and gene therapies.
• To highlight considerations for HCT performance and decision-making in SCD.

Main Methods:

• Review of existing literature on HCT for SCD.
• Analysis of outcomes with HLA-matched sibling, unrelated, and haploidentical donors.
• Evaluation of emerging gene-modified autologous cell therapies.

Main Results:

• Excellent outcomes are achieved with HLA-identical sibling HCT.
• HCT from unrelated or haploidentical donors is increasingly viable.
• Gene-modified cell therapies show promising early results.

Conclusions:

• HCT is a critical therapeutic option for sickle cell disease.
• Expanding donor options and novel gene therapies improve HCT accessibility and efficacy.
• Careful patient selection, multidisciplinary care, and shared decision-making are essential for successful HCT in SCD.