Minihepcidins improve ineffective erythropoiesis and splenomegaly in a new mouse model of adult β-thalassemia major
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Minihepcidins are hepcidin agonists that have been previously shown to reverse iron overload and improve erythropoiesis in mice affected by non-transfusion-dependent thalassemia. Given the extreme anemia that occurred with the previous model of transfusion-dependent thalassemia, that model was inadequate for investigating whether minihepcidins can improve red blood cell quality, lifespan and ineffective erythropoiesis. To overcome this limitation, we generated a new murine model of transfusion-dependent thalassemia with severe anemia and splenomegaly, but sufficient red cells and hemoglobin production to test the effect of minihepcidins. Furthermore, this new model demonstrates cardiac iron overload for the first time. In the absence of transfusions, minihepcidins improved red blood cell morphology and lifespan as well as ineffective erythropoiesis. Administration of a minihepcidin in combination with chronic red blood cell transfusion further improved the ineffective erythropoiesis and splenomegaly and reversed cardiac iron overload. These studies indicate that drugs such as minihepcidins have therapeutic potential for patients with transfusion-dependent thalassemia.
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By inducing iron restriction, hepcidin agonists ameliorated anemia and iron overload in preclinical studies of thalassemia models.
“By inducing iron restriction hepcidin agonists ameliorated anemia and iron overload in preclinical studies of thalassemia models;106,121”
From Iron metabolism and iron disorders revisited in the hepcidin era (2020) · cites this paper as [121]
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Narahari, J. M., Guruswamy, P., Jagadeesha, N. M., Shivashakar, K. K., Kumar, D. P., Narayana, P. S., Vishwanath, P. M., & Prashant, A. (2024) · Gene Expression · doi:10.14218/ge.2023.00128 · Cited by 4 · see citing works
Control of Systemic Iron Homeostasis: Insights Gained from Studying Mouse Models
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Hepcidin as a Molecular Hub of Iron Homeostasis: From BMP–SMAD Signaling to Therapeutic Modulation
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- Authors
- Casu, C., Chessa, R., Liu, A., Gupta, R., Drakesmith, H., Fleming, R., Ginzburg, Y. Z., MacDonald, B., & Rivella, S.
- Affiliations
- Children's Hospital of Philadelphia (US)
- John Radcliffe Hospital (GB)
- Saint Louis University (US)
- Tisch Cancer Institute (US)
- Corresponding author
- Rivella, Stefano
- Venue
- Haematologica
- Published
- 2019-10-03
- Volume
- 105
- Issue
- 7
- Pages
- 1835-1844
- ISSN
- 0390-6078 (ISSN-L)
- 1592-8721
- PMC
- PMC7327634
- OpenAlex
- W2977564049
- Cited by
- 50
- References
- 56
- Citations by year
- 2026: 6 · 2025: 3 · 2024: 3 · 2023: 6 · 2022: 10 · 2021: 13 · 2020: 9
- FWCI
- 4.39
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- Haematologica (publishedVersion)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
- Funders
- National Institutes of Health (UL1TR000003)
- Medical Research Council (MC_UU_00008/10)
- National Heart, Lung, and Blood Institute
- National Institute of Diabetes and Digestive and Kidney Diseases (R01DK090554, R01DK095112, R01DK107670)
- National Center for Advancing Translational Sciences
- Topics
- Health Sciences › Medicine › Genetics › Hemoglobinopathies and Related Disorders
- Health Sciences › Medicine › Hematology › Iron Metabolism and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cell Biology › Hemoglobin structure and function
- Keywords
- beta-thalassemia major, transfusion-dependent thalassemia, ineffective erythropoiesis, splenomegaly, cardiac iron overload, red blood cell lifespan, erythrocyte morphology, chronic transfusion, murine disease models, iron overload
- MeSH terms
- Animals, Disease Models, Animal, Erythropoiesis, Splenomegaly, beta-Thalassemia, Iron Overload, Mice, Hepcidins
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