Year: 2018
Type: article
Abstract: BACKGROUND: ) gene could substitute for long-term red-cell transfusions in a patient with β-thalassemia, we wanted to evaluate the safety and efficacy of such gene therapy in patients with transfusion-dependent β-thalassemia. METHODS: , transfusion requirements, and average vector copy number. RESULTS: genotype or two copies of the IVS1-110 mutation, the median annualized transfusion volume was decreased by 73%, and red-cell transfusions were discontinued in 3 patients. Treatment-related adverse events were typical of those associated with autologous stem-cell transplantation. No clonal dominance related to vector integration was observed. CONCLUSIONS: Gene therapy with autologous CD34+ cells transduced with the BB305 vector reduced or eliminated the need for long-term red-cell transfusions in 22 patients with severe β-thalassemia without serious adverse events related to the drug product. (Funded by Bluebird Bio and others; HGB-204 and HGB-205 ClinicalTrials.gov numbers, NCT01745120 and NCT02151526 .).
Source: New England Journal of Medicine
Authors: Alexis A. Thompson, Mark C. Walters, Janet Kwiatkowski, John E.J. Rasko, Jean-Antoine Ribeil, Suradej Hongeng, Elisa Magrin, Gary J. Schiller, Emmanuel Payen, Michaela Semeraro, Despina Moshous, Francois Lefrere, Hervé Puy, Philippe Bourget, Alessandra Magnani, Laure Caccavelli, Jean-Sébastien Diana, Felipe Suarez, Fabrice Monpoux, Valentine Brousse, Catherine Poirot, Chantal Brouzes, Jean-François Meritet, Corinne Pondarré, Yves Beuzard, Stany Chrétien, Thibaud Lefebvre, David T. Teachey, Usanarat Anurathapan, P. Joy Ho, Christof von Kalle, Morris Kletzel, Elliott Vichinsky, Sandeep Soni, Gabor Veres, Olivier Negre, Robert W. Ross, David Davidson, Alexandria Petrusich, Laura Sandler, Mohammed Asmal, Olivier Hermine, Mariane De Montalembert, Salima Hacein-Bey-Abina, Stéphane Blanche, Philippe Leboulch, Marina Cavazzana
Institutions: Brigham and Women's Hospital, Lurie Children's Hospital, The University of Sydney, University of California, Los Angeles, University of California, San Francisco, Mahidol University, Institut Necker Enfants Malades, Assistance Publique – Hôpitaux de Paris, University of Pennsylvania, Stanford University
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: bronze
Awards: G1044916472, G471519714, G742032275
OpenAlex ID: W2802087233