In this last decade, a deeper understanding of the pathophysiology of hereditary red cell disorders and the development of novel classes of pharmacologic agents have provided novel therapeutic approaches to thalassemias, sickle cell disease (SCD) and other red cell disorders. Here, we analyze and discuss the novel therapeutic options according to their targets and taking into consideration the complex process of erythroid differentiation, maturation, and survival of erythrocytes in the peripheral circulation. We focus on active clinical exploratory and confirmatory trials on thalassemias, SCD and other red cell disorders. Beside -thalassemia and SCD, we found that the development of new therapeutic strategies has allowed the design of clinic studies also for hereditary red cell disorders still lacking valuable therapeutic alternative such as -thalassemias, congenital dyserythropoietic anemia or Blackfan Diamond anemia. In addition, reduction of heme synthesis, which can be achieved by the repurposed anti-psychotic drug Bitopertin, might affect not only hematological disorders but multiorgan diseases such as erythropoietic protoporphyria. Finally, our review highlights the current state of therapeutic scenarios, in which multiple indications targeting different red cell disorders are being considered for a single agent. This is welcome change which will hopefully expand therapeutic option for patients affected by thalassemias, SCD and other red cell disorders.
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Review Article|
May 31, 2024
NOVEL THERAPEUTIC APPROACHES IN THALASSEMIAS, SICKLE CELL DISEASE AND OTHER RED CELL DISORDERS
Valeria Maria Pinto,
Valeria Maria Pinto
IRCCS Ospedale Policlinico San Martino, Italy
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Filippo Mazzi,
Filippo Mazzi
Azienda Ospedaliera Universitaria Integrata Verona, Valeggio Sul Mincio, Italy
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Lucia De Franceschi
DIMI, University of Verona, Verona, Italy
* Corresponding Author; email: lucia.defranceschi@univr.it
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Blood blood.2023022193.
Article history
Submitted:
December 22, 2023
Revision Received:
May 16, 2024
Accepted:
May 19, 2024
Citation
Valeria Maria Pinto, Filippo Mazzi, Lucia De Franceschi; NOVEL THERAPEUTIC APPROACHES IN THALASSEMIAS, SICKLE CELL DISEASE AND OTHER RED CELL DISORDERS. Blood 2024; blood.2023022193. doi: https://doi.org/10.1182/blood.2023022193
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