Clinical Edge

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Transplant Protocol Improves Engraftment in Severe Hemoglobinopathies

Key clinical point: An escalation in total body irradiation from 200 cGy to 400 cGy lowered graft failure without compromising safety in patients undergoing haploidentical hematopoietic cell transplantation for severe hemoglobinopathies.

Major finding: In total, 13 of 17 patients achieved full donor chimerism. Three patients achieved mixed chimerism and one patient had graft failure.

Study details: A prospective study of 17 patients with sickle cell disease or beta-thalassemia major.

Disclosures: The study was funded by the National Institutes of Health and the Maryland Stem Cell Research Fund. The researchers reported disclosures related to Aduro Biotech, Amgen, Alexion Pharmaceuticals, Celgene, Takeda, and others.


Bolaños-Meade FJ et al. Lancet Haematol. 2019 Mar 13. doi: 10.1016/S2352-3026(19)30031-6.