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

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.