Allogeneic transplantation strategies including haploidentical transplantation in sickle cell disease

Area of Science:

• Hematology
• Transplantation Medicine
• Genetic Blood Disorders

Background:

• Sickle cell disease (SCD) is a prevalent inherited blood disorder causing significant morbidity and mortality.
• Current treatments are limited, with hematopoietic stem cell transplantation (HSCT) being the only curative option.
• HSCT for SCD is not globally established due to awareness, prognostic, and risk-benefit challenges.

Purpose of the Study:

• To review the current status and outcomes of HSCT for sickle cell disease.
• To identify barriers to wider HSCT implementation for SCD.
• To discuss future directions for improving HSCT accessibility and efficacy in SCD.

Main Methods:

• Analysis of data from European Blood and Marrow Transplant (EBMT) and Centre for International Blood and Marrow Transplant Research (CIBMTR) registries.
• Review of outcomes for patients undergoing HSCT for SCD, particularly focusing on HLA-identical sibling donors.
• Assessment of conditioning regimens and donor types in HSCT for SCD.

Main Results:

• Over 90% cure rates are achieved with HLA-identical sibling HSCT post-myeloablative conditioning, with minimal complications.
• EBMT and CIBMTR registries report over 600 HSCT cases for SCD each.
• Key barriers include physician/patient awareness, lack of prognostic factors, and perceived transplant risks.

Conclusions:

• HLA-identical sibling HSCT is a highly effective curative treatment for SCD.
• Reduced-toxicity conditioning and alternative donor sources (cord blood, haploidentical) are crucial for expanding HSCT.
• Addressing awareness and prognostic challenges will facilitate earlier and broader HSCT application for sickle cell disease.