Goal-oriented monitoring of cyclosporine is effective for graft-versus-host disease prevention after Hematopoietic Stem Cell Transplantation in Sickle Cell Disease and Thalassemia major

Thalassemia major (TM) and sickle cell anemia (SCA) are the two most widespread inborn hemoglobinopathies worldwide. TM was the first congenital hemoglobinopathy to be cured by allogenic hematopoietic stem cell transplantation (HSCT),1 and SCA rapidly followed.2,3 Other than gene therapy,4 HSCT remains the only available curative treatment for these two diseases. Provided that an HLA-identical sibling donor is available, it should be performed as soon as possible for TM and in case of complications for SCA.

Source: Biology of Blood and Marrow Transplantation - January 29, 2020 Category: Hematology Authors: Alexandra Gauthier, Nathalie Bleyzac, Nathalie Garnier, Kamila Kebaili, Philippe Joly, Marie-Pierre Goutagny, Isabelle Mollet, Sylvain Goutelle, C écile Renard, Yves Bertrand Source Type: research