Erythropoiesis in lower-risk myelodysplastic syndromes and beta-thalassemia
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Churchill Livingstone
Abstract
The hematologic disorders myelodysplastic syndromes and beta-thalassemia are characterized by ineffective erythropoiesis and anemia, often managed with regular blood transfusions. Erythropoiesis, the process by which sufficient numbers of functional erythrocytes are produced from hematopoietic stem cells, is highly regulated, and defects can negatively affect the proliferation, differentiation, and survival of erythroid precursors. Treatments that directly target the underlying mechanisms of ineffective erythropoiesis are limited, and management of anemia with regular blood transfusions imposes a significant burden on patients, caregivers, and health care systems. There is therefore a strong unmet need for treatments that can restore effective erythropoiesis. Novel therapies are beginning to address this need by targeting a variety of mechanisms underlying erythropoiesis. Herein, we provide an overview of the role of ineffective erythropoiesis in myelodysplastic syndromes and beta-thalassemia, discuss unmet needs in targeting ineffective erythropoiesis, and describe current management strategies and emerging treatments for these disorders. © 2022 The Authors
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Anemia, Erythropoiesis, Iron overload, Myelodysplastic syndromes, Novel therapies, Β-thalassemia, Beta-thalassemia, Erythrocytes, Hematologic diseases, Humans, Azacitidine, Cedazuridine plus decitabine, Deferasirox, Deferoxamine, Enasidenib, Erythropoietin, Galunisertib, Growth differentiation factor, Growth differentiation factor 11, Guadecitabine, Heat shock protein 70, Hemoglobin, Hepcidin, Hydroxyurea, Imetelstat, Iron, Iron chelating agent, Isocitrate dehydrogenase inhibitor, Ivosidenib, Janus kinase 2, Lenalidomide, Luspatercept, Mitapivat, Mitogen activated protein kinase 1, Mitogen activated protein kinase 3, Placebo, Recombinant erythropoietin, Roxadustat, Rusfertide, Ruxolitinib, Sapablursen, Sln 214, Stat5 protein, Tmprss6 lrx, Transcription factor gata 1, Transferrin, Unclassified drug, Vamifeport, Venetoclax, Allogeneic stem cell transplantation, Beta thalassemia, Blood transfusion, Caregiver burden, Cell differentiation, Cell proliferation, Cell survival, Drug targeting, Erythrocyte count, Erythrocyte transfusion, Erythroid precursor cell, Health care system, Hematopoietic stem cell, Hematopoietic stem cell transplantation, Human, International prognostic scoring system, Myelodysplastic syndrome, Overall survival, Pathogenesis, Patient care, Patient monitoring, Quality of life, Review, Risk assessment, Survival rate, Complication, Erythrocyte, Hematologic disease