AI Article Synopsis
- Diamond-Blackfan anaemia (DBA) is a type of inherited anemia first identified in 1938 and has shaped the field of pediatric hematology over the years.
- The authors conducted a clinical trial testing eltrombopag, a drug that mimics thrombopoietin, to treat red cell failure associated with DBA.
- Despite a low response rate, the study underscores the significance of exploring new treatment options for this rare condition.
Article Abstract
The rich history surrounding Diamond-Blackfan anaemia (DBA), originally described in 1938 as congenital hypoplastic anaemia reflects the evolution of paediatric haematology. In their paper, the authors 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.
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| http://dx.doi.org/10.1111/bjh.19404 | DOI Listing |
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