%0 Journal Article
%T Understanding complex disease-related mechanisms: Rational therapies for Diamond-Blackfan anaemia.
%A Lipton JM
%J Br J Haematol
%V 204
%N 5
%D 2024 May 14
%M 38485153
%F 8.615
%R 10.1111/bjh.19404
%X 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.