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.
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Jeffrey M. Lipton (2024) studied this question.
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