For adults with sickle cell disease (SCD), a cost-effectiveness analysis suggests that treatment with nonmyeloablative haploidentical (NMAC-HID) allogeneic stem cell transplantation (allo-SCT) may be more cost effective than gene therapy or standard of care (SOC). Study results were reported in the journal Blood.1
The study investigators conducted a model-based analysis to assess the cost-effectiveness of NMAC-HID allo-HSCT, gene therapy, and SOC (including hydroxyurea, blood transfusions, and pain management). Base-case scenarios reflected US costs, with some evaluations including other countries. Primary outcomes included the incremental cost-effectiveness ratio and net monetary benefits (NMBs) across the 3 approaches.1
In the base case, treatment with SOC was associated with accruing 14.3 quality-adjusted life-years (QALYs), with a cost of $1.22 million, while NMAC-HID allo-HSCT was associated with accruing 20.1 QALYs, with a cost of $1.15 million, and gene therapy was associated with accrual of 22.1 QALYs and a cost of $2.75 million.1