What can we learn from ineffective erythropoiesis in thalassemia?

Blood Rev. 2018 Mar;32(2):130-143. doi: 10.1016/j.blre.2017.10.001. Epub 2017 Oct 3.


Erythropoiesis is a dynamic process regulated at multiple levels to balance proliferation, differentiation and survival of erythroid progenitors. Ineffective erythropoiesis is a key feature of various diseases, including β-thalassemia. The pathogenic mechanisms leading to ineffective erythropoiesis are complex and still not fully understood. Altered survival and decreased differentiation of erythroid progenitors are both critical processes contributing to reduced production of mature red blood cells. Recent studies have identified novel important players and provided major advances in the development of targeted therapeutic approaches. In this review, β-thalassemia is used as a paradigmatic example to describe our current knowledge on the mechanisms leading to ineffective erythropoiesis and novel treatments that may have the potential to improve the clinical phenotype of associated diseases in the future.

Keywords: Ineffective erythropoiesis; Red blood cells; Thalassemia.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Biomarkers
  • Cell Differentiation
  • Erythrocytes / drug effects
  • Erythrocytes / metabolism
  • Erythropoiesis* / drug effects
  • Erythropoiesis* / genetics
  • Hematopoietic Stem Cells / cytology
  • Hematopoietic Stem Cells / metabolism
  • Humans
  • Iron / metabolism
  • Molecular Targeted Therapy
  • Stress, Physiological
  • Thalassemia / blood
  • Thalassemia / drug therapy
  • Thalassemia / etiology*
  • Thalassemia / metabolism*


  • Biomarkers
  • Iron