Year: 2026
Type: article
Abstract: Beta-thalassemia is an inherited anemia characterized by a broad spectrum of clinical manifestations. The most severe form is transfusion-dependent β-thalassemia, in which patients need regular blood transfusions to survive, since no adequate amount of hemoglobin is produced by the bone marrow. The therapeutic landscape for this disease is constantly evolving, and new therapies have been recently approved. Luspatercept is the first and only approved drug for treating anemia in transfusion-dependent β-thalassemia. Most available information regarding its safety and efficacy is derived from clinical trials, with limited data on real-world experiences. Thus, a significant gap remains in the literature concerning patient management in everyday clinical settings, particularly in terms of assessing efficacy and the challenges that arise when managing luspatercept. Indeed, effectiveness evaluation in the real-world presents a much more complex scenario compared to clinical trials. In this paper, we present five clinical cases that drive us through the complexity of luspatercept management and highlight the following topics: (1) the role of genotype in the patient selection, (2) the importance of patient empowerment and the psychological aspects when introducing a new therapy, (3) efficacy assessment in the real-world, including improvement of iron balance, optimization of pretransfusion hemoglobin, and (4) the importance of the constant monitoring for safety and for adverse events. Emerging evidence and insights from real-world settings play a crucial role in shaping best practices for everyday clinical practice.
Source: HemaSphere
DOI: 10.1002/hem3.70315
Authors: Daniele Lello Panzieri, Natalia Scaramellini, Simona Leoni, Ali Taher, Maria Domenica Cappellini, Irene Motta
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,480
OpenAlex ID: W7130534275