{Reference Type}: Journal Article
{Title}: Understanding complex disease-related mechanisms: Rational therapies for Diamond-Blackfan anaemia.
{Author}: Lipton JM;
{Journal}: Br J Haematol
{Volume}: 204
{Issue}: 5
{Year}: 2024 May 14
{Factor}: 8.615
{DOI}: 10.1111/bjh.19404
{Abstract}: 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.