A new conditioning regimen for stem cell transplants in sickle cell disease is showing strong results, with high survival and low complication rates.
Researchers tested a combination of thymoglobulin, thiotepa, cyclophosphamide, fludarabine, and low-dose total body irradiation in 25 patients across three centers. Most received fully matched sibling transplants.
The results were striking: 2-year overall survival reached 100%, with a 5-year event-free survival of 96%. No graft failure was observed. Donor engraftment was robust, with median chimerism at 100% by day 28.
Safety outcomes were equally impressive. Only 4% developed severe acute graft versus host disease, and no chronic cases were reported. 81% of patients were able to stop immunosuppression after one year.
The study suggests this intensified yet tolerable regimen could become a new standard for adolescents and adults with severe sickle cell disease, including those needing repeat transplants.