Evidence map›Paper›PMID 31582543›Full record

ArticleHaematologica2020

Minihepcidins improve ineffective erythropoiesis and splenomegaly in a new mouse model of adult β-thalassemia major.

Carla Casu, Roberta Chessa, Alison Liu, Ritama Gupta, Hal Drakesmith, Robert Fleming, Yelena Z Ginzburg, Brian MacDonald, Stefano Rivella

Open access · goldAbstract read
In one paragraph

Article in Haematologica, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed
4.6field-weighted citation impact, top 5% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

32 citing papers in PubMed, 46 citations in OpenAlex.

  1. Thalassaemia.Nature reviews. Disease primers · 2026
    Review
  2. Review
  3. Molecular and therapeutic frontiers in anemia therapy.The Journal of clinical investigation · 2026
    Review
  4. Review
  5. Review
  6. Current Landscape of Hepcidin Therapeutics.Advances in experimental medicine and biology · 2025
    Review
  7. Review
  8. Review
  9. Article
  10. Review
  11. Review
  12. Article
  13. Review
  14. The mutual crosstalk between iron and erythropoiesis.International journal of hematology · 2022
    Review
  15. Article
  16. Review
  17. Coordination of iron homeostasis by bone morphogenetic proteins: Current understanding and unanswered questions.Developmental dynamics : an official publication of the American Association of Anatomists · 2022
    Article
  18. Advancing the care of β-thalassaemia patients with novel therapies.Blood transfusion = Trasfusione del sangue · 2022
    Review
  19. Review
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

9 authors at 4 institutions in 2 countries.

Carla CasuDepartment of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
Roberta ChessaDepartment of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
Alison LiuDepartment of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
Ritama GuptaDepartment of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
Hal DrakesmithMRC Human Immunology Unit, MRC Weatherall Institute of Molecular Medicine, University of Oxford, John Radcliffe Hospital, Oxford, UK.
Robert FlemingDepartment of Pediatrics, Saint Louis University School of Medicine, St. Louis, MO, USA.
Yelena Z GinzburgDivision of Hematology and Medical Oncology, Tisch Cancer Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Brian MacDonaldMerganser Biotech Inc. King of Prussia, PA, USA.
Stefano RivellaDepartment of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA rivellas@email.chop.edu.
Children's Hospital of Philadelphia · USIcahn School of Medicine at Mount Sinai · USMRC Weatherall Institute of Molecular Medicine · GBSaint Louis University · US

Funding

Institutional Clinical and Translational Science AwardUL1TR000003 · NCATS · UNIVERSITY OF PENNSYLVANIA · PI FITZGERALD, GARRET A · 2012 to 2015
$38.9M
Regulatory Role of Transferrin in Erythropoiesis and Iron MetabolismR01DK095112 · NIDDK · SAINT LOUIS UNIVERSITY · PI Robert E Fleming, Yelena Ginzburg · 2012 to 2026
$5.4M
Hepcidin therapy for iron overload and hematologic disordersR01DK090554 · NIDDK · WEILL MEDICAL COLL OF CORNELL UNIV · PI RIVELLA, STEFANO · 2010 to 2021
$5.2M
The Role of Erythroferrone in Regulating Bone Metabolism in Beta-ThalassemiaR01DK107670 · NIDDK · NEW YORK BLOOD CENTER · PI GINZBURG, YELENA, YUEN, TONY · 2015 to 2025
$4.5M
Medical Research Council MC_UU_00008/10Medical Research Council MC_UU_12010/10NCATS NIH HHS UL1 TR000003NIDDK NIH HHS R01 DK090554NIDDK NIH HHS R01 DK095112NIDDK NIH HHS R01 DK107670
6 · The paper itself

Abstract

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.

Indexed as

beta-ThalassemiaIron OverloadSplenomegalyAnimalsDisease Models, AnimalErythropoiesisHepcidinsMiceHepcidins

Identifiers

PMID31582543
PMCPMC7327634
OpenAlexW2977564049

What OpenQuestion holds

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LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.