Publications
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Patient-Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non-Transfusion-Dependent β-Thalassemia Treated in the BEYOND Trial
Abstract In the phase 2, double‐blind, randomized controlled BEYOND trial (NCT03342404), luspatercept increased hemoglobin levels in patients with non‐transfusion‐dependent β‐thalassemia (NTDT). This study assessed long‐term effects of luspatercept on patient‐reported…
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Modelling the Cost-Effectiveness of Non-Invasive Prenatal Testing in the English Sickle Cell and Thalassaemia Screening Pathway
Background: Sickle cell disease (SCD) and thalassaemia are inherited conditions causing chronic anaemia, increased infection risk, and multi-organ failure. Standard of care (SoC) prenatal screening involves carrier blood testing for…
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Prevalence of Severe Thalassemia and Performance of Prenatal Screening Tests Among Pregnant Women at Siriraj Thalassemia Center in Thailand
Abstract Severe thalassemia remains a significant public health concern in Southeast Asia. Prenatal screening is an effective strategy for early detection and prevention. This study aimed to determine the prevalence…
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Systematic Review of Economic Evaluations in Thalassaemia Screening Programmes Globally: Developing Guidance for Low- and Middle-Income (LMIC) Settings
Objectives Thalassaemia, a genetic blood disorder, is a major public health burden. Most affected individuals reside in low-and-middle-income countries (LMICs). Screening programmes can reduce incidence, but in resource-constrained settings, cost-effectiveness…
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Outcomes With Luspatercept in Patients With Β-Thalassemia: A Systematic Review and Metaanalysis
Abstract Background: β-thalassaemia is a genetic blood disorder marked by ineffective erythropoiesis and chronic anaemia, often requiring lifelong red blood cell (RBC) transfusions. Luspatercept, an erythroid maturation agent, has emerged as…
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Evaluating Mitapivat for the Treatment of Alpha or Beta Thalassemia
INTRODUCTION Thalassemia is a group of diverse genetic disorders with worldwide distribution that affect hemoglobin synthesis. Until recently, the therapeutic approach to thalassemia was symptomatic, relying on red blood cell…
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Phase 2a Randomized Study to Evaluate Sapablursen in Patients With Non-Transfusion Dependent Β-Thalassemia Intermedia
Non-transfusion-dependent β-thalassemia intermedia (NTDT) is a genetically heterogeneous disorder characterized by decreased β-globin production in erythroid cells resulting from mutations in the β-globin gene that is not treated with transfusion.…
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2026 Update on Clinical Trials in β-Thalassemia
Abstract The therapeutic landscape of β-thalassemia has evolved rapidly over the past decade, shifting from a historical reliance on transfusion support and iron chelation toward disease-modifying and potentially curative therapies.…
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Long-Term Efficacy and Safety Results of Betibeglogene Autotemcel Gene Therapy for Transfusion-Dependent Β-Thalassemia
Abstract Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia (TDT) involves autologous transplantation of hematopoietic stem and progenitor cells transduced with a modified β-globin gene to produce functional adult hemoglobin…
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Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease
Background Exagamglogene autotemcel (exa-cel) is a cell therapy in which autologous CD34+ hematopoietic cells are engineered through ex vivo clustered regularly interspaced short palindromic repeats–Cas9 editing of the erythroid-specific enhancer…
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