Ineffective erythropoiesis and regulation of iron status in iron loading anaemias

Clara Camaschella, Antonella Nai

Research output: Contribution to journalArticle

42 Citations (Scopus)

Abstract

The definition 'iron loading anaemias' encompasses a group of inherited and acquired anaemias characterized by ineffective erythropoiesis, low hepcidin levels, excessive iron absorption and secondary iron overload. Non-transfusion-dependent β-thalassaemia is the paradigmatic example of these conditions that include dyserythropoietic and sideroblastic anaemias and some forms of myelodysplasia. Interrupting the vicious cycle between ineffective erythropoiesis and iron overload may be of therapeutic benefit in all these diseases. Induction of iron restriction by means of transferrin infusions, minihepcidins or manipulation of the hepcidin pathway prevents iron overload, redistributes iron from parenchymal cells to macrophage stores and partially controls anaemia in β-thalassaemic mice. Inhibition of ineffective erythropoiesis by activin ligand traps improves anaemia and iron overload in the same models. Targeting iron loading or ineffective erythropoiesis shows promise in preclinical studies; activin ligand traps are in clinical trials with promising results and may be useful in patients with ineffective erythropoiesis.

Original languageEnglish
Pages (from-to)512-523
Number of pages12
JournalBritish Journal of Haematology
Volume172
Issue number4
DOIs
Publication statusPublished - Feb 1 2016

Fingerprint

Erythropoiesis
Iron Overload
Anemia
Iron
Hepcidins
Activins
Sideroblastic Anemia
Ligands
Thalassemia
Transferrin
Macrophages
Clinical Trials

Keywords

  • Anaemia
  • Erythropoiesis
  • Iron
  • Iron overload
  • Thalassaemia

ASJC Scopus subject areas

  • Hematology

Cite this

Ineffective erythropoiesis and regulation of iron status in iron loading anaemias. / Camaschella, Clara; Nai, Antonella.

In: British Journal of Haematology, Vol. 172, No. 4, 01.02.2016, p. 512-523.

Research output: Contribution to journalArticle

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