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Donatella Baronciani

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Articles

Update of the decision-making algorithm on selecting transfusion-dependent β-thalassemic patients for gene therapy approaches: joint consensus report on behalf of EHA-specialized working group and EBMT hemoglobinopathies working party

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By Lucia De Franceschi, Mahmoud Aljurf, Christian Chabannon, Selim Corbacioglu, Josu de la Fuente, Gian Luca Forni, Antonis Kattamis, Franco Locatelli, Roland Meisel, Ali Taher, Paul Telfer, Isabelle Thuret, Emanuele Angelucci, Donatella Baronciani, Celeste Bento
| Nature Verified
Abstract β-thalassemia is a globally distributed hereditary red blood cell disorder. Up to now, clinical management of transfusion-dependent β-thalassemia (TDT) patients is still based on chronic transfusion combined with iron chelation therapy. Allogeneic hematopoietic cell transplantation potentially provides a cure, but few patients have an HLA-identical sibling, and optimal results are reported in patients ≤ 14 years.
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