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- Title
Understanding complex disease‐related mechanisms: Rational therapies for Diamond–Blackfan anaemia.
- Authors
Lipton, Jeffrey M.
- Abstract
The rich history surrounding Diamond–Blackfan anaemia (DBA), originally described in 1938 as congenital hypoplastic anaemia2 reflects the evolution of paediatric haematology. In their paper, the authors1 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.
- Subjects
ANEMIA; APLASTIC anemia; ERYTHROCYTES; ELTROMBOPAG; HEMATOLOGY
- Publication
British Journal of Haematology, 2024, Vol 204, Issue 5, p1598
- ISSN
0007-1048
- Publication type
Article
- DOI
10.1111/bjh.19404