King, AA (2015), American Journal of Hematology(Link to Abstract)
Hematopoietic stem cell transplantation (HSCT) is curative for patients with hemoglobinopathies, but is often delayed or avoided because of the significant risks associated with it, particularly those associated to the myeloablative conditioning. The authors initiated a prospective multi-center trial of reduced intensity conditioning (RIC) with Alemtuzumab/ Fludarabine/ Melphalan in 52 children with hemoglobinopathies (sickles cell disease and thalassemia) who were to undergo HSCT with a matched sibling donor marrow or cord blood graft. Event free survival following RIC was 92%, which was comparable to myeloablative transplants (>85%), proving non-inferiority. Successful engraftment was maintained after withdrawal of immunosuppression in all but one case, and all cases with successful engraftment remained transfusion independent and without complications of their initial condition.
* Reduced intensity conditioning is emerging as an alternative option for patients with hemoglobinopathies undergoing matched sibling bone marrow transplantation.