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Understanding complex disease-related mechanisms: Rational therapies for Diamond-Blackfan anaemia.
British Journal of Haematology 2024 March 15
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 (Online ahead of print). doi: 10.1111/bjh.19357.
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