Emerging Therapies and Strategies in Thalassemia: Toward a New Era in Management—Volume II

Dear Colleagues,

Until a few years ago, HSCT was the only cure able to modify the natural history of thalassemia syndromes; nevertheless, survival and quality of life in patients with thalassemia have been significantly improved with standard therapies comprising red blood cell transfusion and iron chelation, which represent a longstanding approach. On the other hand, aging and increase in life expectancy are disclosing a complex scenario of multiple disease-related morbidities, including osteoporosis, endocrine disorders, liver disease, renal dysfunction, and cancer. Therefore, new strategies are needed to achieve further control of disease burden. Looking at the pathophysiological mechanisms of beta-thalassemia, new drugs able to ameliorate globin synthesis, reducing ineffective erythropoiesis, chain imbalance, and iron overload, have been undergoing testing for a few years now. Furthermore, considering standard treatments, there is still the need to provide more strength of scientific evidence with long-term prospective observations or, if possible, with randomized trials. Similarly, to correctly manage the wide spectrum of thalassemia syndromes, biomarkers able to assess preventive and curative treatments in selected populations at increased risk of developing complications and to correctly evaluate interventional procedures in a homogeneous population are also needed.

This Special Issue on “Emerging Therapies and Strategies in Thalassemia: Toward a New Era in Management” aims to update researchers and clinicians by highlighting the main points that could represent a remarkable therapeutic advancement or improvement in the management made recently in the field of thalassemia syndromes.

Dr. Paolo Ricchi
Dr. Raffaella Origa
Guest Editors

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• HSCT
• gene therapy
• new drugs
• iron chelation
• aging
• long-term observation
• biomarkers
• new strategies

• Emerging Therapies and Strategies in Thalassemia: Toward a New Era in Management in Journal of Clinical Medicine (7 articles)