Haploidentical transplant, gene therapy, and standard care in sickle cell disease: a cost-effectiveness analysis.

Haploidentical stem cell transplantation is consistently more cost-effective than gene therapy for sickle cell disease, with a 100% probability across analyses.

• Why it matters: Sickle cell disease causes significant morbidity and reduced life expectancy, and current treatments are limited by high costs and limited access, creating a need for more affordable, effective options.
• What they did: A cost-effectiveness analysis compared three strategies—standard care, gene therapy, and haploidentical stem cell transplantation—using outcomes like QALYs and costs in a model with 10,000 Monte Carlo simulations.
• The result: Haploidentical transplantation provided the best value, with lower costs and higher QALYs, and gene therapy's maximum affordable price varied globally, supporting its potential for broader implementation.