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Thalassemia Reports
Volume 15
Issue 1
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Thalass. Rep., Volume 15, Issue 1 (March 2025) – 3 articles

Cover Story (view full-size image): Acute transfusion reactions are transfusion-related events that can significantly affect patient safety and therapeutic management. APTRs pose a significant challenge in transfusion medicine, requiring a multifaceted approach to therapy and prevention. Through hemovigilance procedures, healthcare professionals can enhance patient outcomes and transfusion safety by fostering awareness, promoting research, improving surveillance and reporting mechanisms, and prioritizing patient education and follow-up care. Such efforts will deepen the understanding of APTRs and support the development of more effective strategies for their management and prevention. View this paper
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6 pages, 197 KiB  
Perspective
The Changing Landscape of Opportunity for Cure of Severe Hemoglobinopathies in Middle-Income Regions
by Lawrence Faulkner
Thalass. Rep. 2025, 15(1), 3; https://doi.org/10.3390/thalassrep15010003 - 6 Mar 2025
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Abstract
Thalassemia and sickle cell disease remain the most common life-threatening non-communicable diseases in children worldwide and an increasing burden on affected families and health services. Significant progress has been made in terms of technologies to improve access to a cure by both allogeneic [...] Read more.
Thalassemia and sickle cell disease remain the most common life-threatening non-communicable diseases in children worldwide and an increasing burden on affected families and health services. Significant progress has been made in terms of technologies to improve access to a cure by both allogeneic and autologous gene-modified hematopoietic stem cell transplantation (HSCT). However, the high cost of cutting-edge treatments often places them beyond the reach of individual families or even national healthcare systems. Advances in frugal innovation and simplified HSCT procedures for low-risk transplants have significantly reduced the costs and complexities associated with HSCT without compromising on quality and outcomes. Because of the geographical distribution of hemoglobinopathies, i.e., largely in low- and middle-income countries (LMICs), HSCT cost optimization has the potential to impact a huge number of patients, increasing hope for a cure and health-related quality of life normalization, which in turn may affect supportive care compliance. Furthermore, because of the high burden of disease, LMIC transplant centers are rapidly increasing in number and developing unique expertise for the cure of thalassemia and sickle cell disease, particularly in India, where the Sankalp India Foundation with the support of DKMS and Cure2Children has implemented several cost-conscious transplant services. In fact, the very high success rate, increasing cost-effectiveness of transplantation, as well as the chronic nature of these conditions make them ideal initial candidates for start-up transplant centers, so it is likely that the global capacity for a cure for severe hemoglobinopathies will substantially increase in the years to come. Full article
(This article belongs to the Section Conventional Treatment of Thalassemia)
10 pages, 487 KiB  
Review
New Perspectives on the Impact of Iron Chelation Therapy on the Gut Microbiome in Thalassemia Patients
by Sara Deumić, Neira Crnčević, Mirsada Hukić, Muamer Dizdar and Monia Avdić
Thalass. Rep. 2025, 15(1), 2; https://doi.org/10.3390/thalassrep15010002 - 10 Feb 2025
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Abstract
Thalassemia, a genetic condition characterized by defective hemoglobin synthesis, is often managed with transfusion therapy, which can lead to iron overload—a significant contributor to morbidity and mortality due to organ damage and pathogenic infections. Iron chelation therapy, the cornerstone of managing iron toxicity, [...] Read more.
Thalassemia, a genetic condition characterized by defective hemoglobin synthesis, is often managed with transfusion therapy, which can lead to iron overload—a significant contributor to morbidity and mortality due to organ damage and pathogenic infections. Iron chelation therapy, the cornerstone of managing iron toxicity, may inadvertently influence the gut microbiome, a critical modulator of immunity and metabolism. This review provides new insights into the interplay between iron chelation therapy and gut microbiome dynamics in thalassemia patients. It synthesizes findings on how chelators such as deferoxamine, deferasirox, and deferiprone influence microbial composition, iron availability, and systemic inflammation. Emerging evidence highlights alterations in gut microbial diversity, with reduced beneficial taxa and increased pathogenic populations, driven by changes in luminal iron levels. This imbalance contributes to immune dysregulation, systemic inflammation, and susceptibility to infections. The review advocates for tailored treatment strategies that integrate microbiome-targeted interventions alongside traditional chelation therapy to improve patient outcomes. By combining genetic profiling, dietary adjustments, and microbiome modulation, this approach offers a promising avenue for personalized medicine in thalassemia care. Full article
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8 pages, 654 KiB  
Review
Acute Painful Transfusion Reactions (APTRs): A Comprehensive Review of Clinical Features, Pathophysiology, Diagnosis, and Management
by Sophia Delicou, Aspasia Argyrou, Sophia Mellou, Aikaterini Xydaki, Anthippi Gafou and Constantina Politis
Thalass. Rep. 2025, 15(1), 1; https://doi.org/10.3390/thalassrep15010001 - 31 Jan 2025
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Abstract
It is important to recognize the significance of acute painful transfusion reactions (APTRs) as a complication of blood transfusions. These adverse reactions are characterized by the onset of acute pain after the administration of blood components. Despite their potential to cause significant discomfort [...] Read more.
It is important to recognize the significance of acute painful transfusion reactions (APTRs) as a complication of blood transfusions. These adverse reactions are characterized by the onset of acute pain after the administration of blood components. Despite their potential to cause significant discomfort and distress to patients, these reactions are frequently disregarded and under-reported in clinical practice. This review aims to provide a comprehensive analysis of the clinical features, pathophysiology, diagnosis, incidence, and management of APTRs, emphasizing the necessity for heightened awareness and further research in this field. Full article
(This article belongs to the Section Innovative Treatment of Thalassemia)
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