Understanding complex disease-related mechanisms: Rational therapies for Diamond-Blackfan anaemia.

Autor: Lipton JM; Division of Hematology Oncology Stem Cell Transplant/Cellular Therapy, Cohen Children's Medical Center, Northwell Health, New Hyde Park, New York, USA.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2024 May; Vol. 204 (5), pp. 1598-1599. Date of Electronic Publication: 2024 Mar 14.
DOI: 10.1111/bjh.19404
Abstrakt: The rich history surrounding Diamond-Blackfan anaemia (DBA), originally described in 1938 as congenital hypoplastic anaemia 2 reflects the evolution of paediatric haematology. In their paper, the authors 1 present the results of a clinical trial using the thrombopoietin-mimetic agent eltrombopag to treat red cell failure in DBA. A low response rate belies the importance of this work. Commentary on: Duncan et al. Treatment of refractory/relapsed Diamond-Blackfan anaemia with eltrombopag. Br J Haematol 2024;204:2077-2085.
(© 2024 British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
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