Publications
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Contemporary Global Burden of Sickle Cell Anaemia Under-5 and Under-20: A Systematic Review and Meta-Analysis
Abstract Sickle cell disease (SCD), a prevalent inherited non-communicable disease, remains a neglected public health priority, especially among children and adolescents in many low- and middle-income countries. The global burden…
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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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