Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Editorial
. 2025 Dec 23;9(24):6547-6548.
doi: 10.1182/bloodadvances.2025017436.

Sickle cell disease and thalassemia go to the PROm

Miguel R Abboud  1 , Biree Andemariam  2
Affiliations
Editorial

Sickle cell disease and thalassemia go to the PROm

Miguel R Abboud et al. Blood Adv. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure: M.R.A. reports research support from Agios, Novartis Pharmaceuticals, Pfizer, AstraZeneca, Salix Pharmaceuticals, and Novo Nordisk; serves as a member of the data safety monitoring boards for trials with Vertex and CRISPR Therapeutics; reports direct speaker honoraria from Emmaus and Novo Nordisk;travel support from Roche; and serves as a member on the advisory board of Agios Pharmaceuticals. B.A. served as a consultant of Accordant, Afimmune, Agios, Beam Therapeutics, bluebird bio, Chiesi, Editas, Genentech, GlaxoSmithKline, Hema Biologics, Hemanext, Merck, Novartis, Novo Nordisk, Octapharma, Pfizer, Roche, Sanius Health, Sanofi Genzyme, and Vertex; reports research support from Afimmune, Agios, American Society of Hematology, American Thrombosis Hemostasis Network, Centers for Disease Control, Connecticut Department of Public Health, Hemanext, Health Resources and Services Administration, Novo Nordisk, Patient-Centered Outcomes Research Institute, and Pfizer; and served as a member of the data safety monitoring boards for trials with Editas and Fulcrum Therapeutics.

Comment on

References

    1. Locatelli F, Lang P, Wall D, et al. Exagamglogene autotemcel for transfusion dependent beta-thalassemia. N Engl J Med. 2024;390(18):1663–1676. - PubMed
    1. Frangoul H, Locatelli F, Sharma A, et al. Exagamglogene autotemcel for severe sickle cell disease. N Engl J Med. 2024;390(18):1649–1662. - PubMed
    1. de la Fuente J, Frangoul H, Lang P, et al. Improvements in health-related quality-of-life improvements in patients with transfusion-dependent beta-thalassemia after exagamglogene autotemcel. Blood Adv. 2025 doi: 10.1182/bloodadvances.2025016702. In press. - DOI - PMC - PubMed
    1. Sharma A, Locatelli F, Bhatia M, et al. Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel. Blood Adv. 2025 doi: 10.1182/bloodadvances.2025016701. In press. - DOI - PMC - PubMed
    1. Salcedo J, Bulovic J, Young CM. Cost-effectiveness of a hypothetical cell or gene therapy cure for sickle cell disease. Sci Rep. 2021;11(1) - PMC - PubMed