Related Experiment Video

Updated: Jun 30, 2025

Author Spotlight: Advancing Erythropoiesis Research - A Simplified Pipeline for Assessing Hematopoietic Stem Cell Function in Myelodysplastic Syndromes
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Author Spotlight: Advancing Erythropoiesis Research - A Simplified Pipeline for Assessing Hematopoietic Stem Cell Function in Myelodysplastic Syndromes

Published on: January 10, 2025

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

Jeffrey M Lipton1

  • 1Division of Hematology Oncology Stem Cell Transplant/Cellular Therapy, Cohen Children's Medical Center, Northwell Health, New Hyde Park, New York, USA.

British Journal of Haematology
|March 14, 2024
PubMed

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.

Keywords:
Diamond–Blackfan anaemiaas congenital hypoplastic anemiaeltrombopag

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