Scientific Literature
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Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy
Abstract Gene therapy has emerged as a transformative treatment option for individuals with sickle cell disease (SCD), with recent regulatory approvals marking a pivotal shift in clinical care. However, the…
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Osteoporosis Risk in Alpha and Beta Thalassemia: An Age- and Sex-Specific Retrospective Cohort Study
Abstract Summary Rationale: Differential skeletal risks between alpha- and beta-thalassemia subtypes remain unclear. Main result: Beta-thalassemia correlates with higher fracture risk than alpha-thalassemia. Notably, males aged 18–50 with beta-thalassemia show…
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Addressing Obstetric and Gynecologic Needs in Thalassemia
Abstract Thalassemia represents a spectrum of rare, inherited blood disorders associated with a range of disease- and treatment-related complications. Thalassemia can have a significant effect on the female reproductive system,…
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Diagnosis and Management of Transfusion-Dependent Thalassemia: Evidence-Based Guidelines From the Pediatric Hematology Oncology Chapter of the Indian Academy of Pediatrics
Justification India bears a high burden of thalassemia, underscoring the need for enhanced awareness, systematic screening, and standardized, high-quality care. In the absence of indigenous guidelines, gaps remain in knowledge…
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Short- and Long-Term Effects of Transfusion in Β-Thalassemia: A Longitudinal Study of Transfusion Efficiency Factors
Abstract The complex interplay between donor and recipient factors likely influences transfusion outcomes in transfusion-dependent thalassemia (TDT). We investigated physiological responses to transfusion shortly after it and 1 week later,…
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Experiences of Families With Children Diagnosed With Beta-Thalassemia: A Meta-Synthesis of Qualitative Studies
Background and purpose This meta-synthesis aimed to integrate qualitative studies examining the emotional, social, and practical experiences of families caring for children diagnosed with thalassemia. Methods Following Thomas and Harden’s…
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Family Empowerment Education Module for Parents of Children With Thalassemia Major: A Randomized Controlled Study on Anxiety, Problem-Solving, and Psychological Resilience
Abstract Objective: This study determined the effect of the Family Empowerment in Pediatric Thalassemia Care (FE-PTC) Module on anxiety, problem-solving skills and psychological resilience in parents of children with thalassemia major.…
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Transition from Pediatric to Adult Care in Patients with Transfusion-Dependent Beta-Thalassemia in France: A National Study Concerning a Rare Disease
Abstract Background/Objectives: Transfusion-dependent β-thalassaemia (TDT) is a lifelong condition requiring coordinated multidisciplinary care. In France, where the disease is rare, transition from pediatric to adult care remains poorly structured, potentially…
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Dapagliflozin in Patients with Homozygous β-Thalassemia and Albuminuria
Abstract Patients with homozygous β-thalassemia show continuously improved survival, which has contributed to the development of kidney complications, predominantly expressed as increased albuminuria. In particular, patients with transfusion-dependent thalassemia (TDT)…
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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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