Minihepcidin peptides as disease modifiers in mice affected by β-thalassemia and polycythemia vera

Carla Casu, Paraskevi Rea Oikonomidou, Huiyong Chen, Vijay Nandi, Yelena Ginzburg, Princy Prasad, Robert E. Fleming, Yatrik M. Shah, Erika V. Valore, Elizabeta Nemeth, Tomas Ganz, Brian MacDonald, Stefano Rivella

Research output: Contribution to journalArticlepeer-review

97 Scopus citations


In β-thalassemia and polycythemia vera (PV), disordered erythropoiesis triggers severe pathophysiological manifestations. β-Thalassemia is characterized by ineffective erythropoiesis, reduced production of erythrocytes, anemia, and iron overload and PV by erythrocytosis and thrombosis. Minihepcidins are hepcidin agonists that have been previously shown to prevent iron overload in murine models of hemochromatosis and induce iron-restricted erythropoiesis at higher doses. Here, we show that in young Hbbth3/+ mice, which serve as a model of untransfused β-thalassemia, minihepcidin ameliorates ineffective erythropoiesis, anemia, and iron overload. In older mice with untransfused β-thalassemia, minihepcidin improves erythropoiesis and does not alter the beneficial effect of the iron chelator deferiprone on iron overload. In PV mice that express the orthologous JAK2 mutation causing human PV, administration of minihepcidin significantly reduces splenomegaly and normalizes hematocrit levels. These studies indicate that drug-like minihepcidins have a potential as future therapeutics for untransfused β-thalassemia and PV.

Original languageEnglish
Pages (from-to)265-276
Number of pages12
Issue number2
StatePublished - 14 Jul 2016
Externally publishedYes


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