Treating Thalassemia Patients with Luspatercept: An Expert Opinion Based on Current Evidence

Longo, Filomena; Motta, Irene; Pinto, Valeria; Piolatto, Andrea; Ricchi, Paolo; Tartaglione, Immacolata; Origa, Raffaella

doi:10.3390/jcm12072584

Cited by 2 publications

(1 citation statement)

References 9 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Recent non-gene therapy drug treatments for thalassemia and sickle cell disease have witnessed significant progress, underscoring the continual evolution of treatment strategies that target specific facets of these blood disorders. Luspatercept, an activin receptor ligand trap, represents the first erythroid maturation agent approved for adult patients with transfusion-dependent β-thalassemia [ 50 ].…”

Section: Current Treatments For Rare Anemias: a Revolutionmentioning

confidence: 99%

Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment

Vives Corrons

2024

JCM

View full text Add to dashboard Cite

Background—This review provides a comprehensive overview of rare anemias, emphasizing their hereditary and acquired causes, diagnostic advancements, and evolving treatment strategies. It outlines the significance of rare anemias within public health, historical challenges in recognition and treatment, and the role of European initiatives like ENERCA and EuroBloodNet in advancing care. Content—This document discusses diagnostic technologies like next-generation sequencing and the impact of artificial intelligence, alongside the promising avenues of gene therapy, targeted drug treatments, and stem cell transplantation. It underscores the importance of a patient-tailored approach, advances in diagnostic tools, and the necessity for continued research, patient advocacy, and international collaboration to improve outcomes for individuals with rare anemias.

show abstract

Section: Current Treatments For Rare Anemias: a Revolutionmentioning

confidence: 99%

Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment

Vives Corrons

2024

JCM

View full text Add to dashboard Cite

show abstract

The Importance and Essentiality of Natural and Synthetic Chelators in Medicine: Increased Prospects for the Effective Treatment of Iron Overload and Iron Deficiency

Kontoghiorghes

2024

IJMS

View full text Add to dashboard Cite

The supply and control of iron is essential for all cells and vital for many physiological processes. All functions and activities of iron are expressed in conjunction with iron-binding molecules. For example, natural chelators such as transferrin and chelator–iron complexes such as haem play major roles in iron metabolism and human physiology. Similarly, the mainstay treatments of the most common diseases of iron metabolism, namely iron deficiency anaemia and iron overload, involve many iron–chelator complexes and the iron-chelating drugs deferiprone (L1), deferoxamine (DF) and deferasirox. Endogenous chelators such as citric acid and glutathione and exogenous chelators such as ascorbic acid also play important roles in iron metabolism and iron homeostasis. Recent advances in the treatment of iron deficiency anaemia with effective iron complexes such as the ferric iron tri-maltol complex (feraccru or accrufer) and the effective treatment of transfusional iron overload using L1 and L1/DF combinations have decreased associated mortality and morbidity and also improved the quality of life of millions of patients. Many other chelating drugs such as ciclopirox, dexrazoxane and EDTA are used daily by millions of patients in other diseases. Similarly, many other drugs or their metabolites with iron-chelation capacity such as hydroxyurea, tetracyclines, anthracyclines and aspirin, as well as dietary molecules such as gallic acid, caffeic acid, quercetin, ellagic acid, maltol and many other phytochelators, are known to interact with iron and affect iron metabolism and related diseases. Different interactions are also observed in the presence of essential, xenobiotic, diagnostic and theranostic metal ions competing with iron. Clinical trials using L1 in Parkinson’s, Alzheimer’s and other neurodegenerative diseases, as well as HIV and other infections, cancer, diabetic nephropathy and anaemia of inflammation, highlight the importance of chelation therapy in many other clinical conditions. The proposed use of iron chelators for modulating ferroptosis signifies a new era in the design of new therapeutic chelation strategies in many other diseases. The introduction of artificial intelligence guidance for optimal chelation therapeutic outcomes in personalised medicine is expected to increase further the impact of chelation in medicine, as well as the survival and quality of life of millions of patients with iron metabolic disorders and also other diseases.

show abstract

Treating Thalassemia Patients with Luspatercept: An Expert Opinion Based on Current Evidence

Cited by 2 publications

References 9 publications

Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment

Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment

The Importance and Essentiality of Natural and Synthetic Chelators in Medicine: Increased Prospects for the Effective Treatment of Iron Overload and Iron Deficiency

Contact Info

Product

Resources

About