Publications
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Impact of Imatinib on Reducing the Painful Crisis in Patients with Sickle Cell Disease
Introduction: Sickle cell disease (SCD) is a common hemoglobinopathy worldwide that causes painful crises and hospitalization of patients. These attacks decrease survival and cause chronic end-organ damage in these patients. Hypothesis: For this reason,…
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Health-Related Quality-of-Life Profile of Pediatric Patients with β-Thalassemia after Hematopoietic Stem Cell Transplantation
Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative treatment in pediatric patients with beta thalassemia major (β-TM). However, little data are available regarding patients and their parents’…
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Modelling the Public Health Impact of Voxelotor in the Management of Sickle Cell Disease in France
Sickle cell disease (SCD) is an inherited blood disorder in which sickle hemoglobin (HbS) polymerizes, leading to red blood cell sickling and chronic hemolytic anemia, vaso-occlusive crises, and end-organ damage…
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The Danish National Haemoglobinopathy Screening Programme: Report From 16 Years of Screening in a Low-prevalence, Non-endemic Region
The Danish national haemoglobinopathy screening programme seeks to determine parental haemoglobinopathy carrier state antenatally. In this retrospective register based study, we evaluated the 16-year trajectory of this programme, utilising the…
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Analysis of the Pregnancy Status and Outcomes of Pregnant Women with α-Thalassemia: A Retrospective Clinical Study
Background: The prevalence of α-thalassemia is high in Guangxi, and its co-occurrence with pregnancy may impede maternal system functions, with a consequent adverse effect on pregnancy progression and birth outcomes.…
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How I Τreat Non-Transfusion-Dependent β-Thalassemia
The intricate interplay of anemia and iron overload under the pathophysiological umbrella of ineffective erythropoiesis in non-transfusion-dependent β-thalassemia (NTDT) results in a complex variety of clinical phenotypes that are challenging…
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Pregnancy-related Thromboembolism in Women with Sickle Cell Disease: An Analysis of National Medicaid Data
Pregnancy and sickle cell disease (SCD) both individually carry a risk of thromboembolism (TE). Pregnancy in people with SCD may further enhance the prothrombotic effect of the underlying disease. The…
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Speckle Tracking Echocardiography and Β-Thalassemia Major: A Systematic Review
Heart disease is among the primary causes of morbidity and mortality in β-thalassemia major (β-TM). Conventional echocardiography has failed to identify myocardial dysfunction at an early stage among these patients,…
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SLN124, a GalNAc Conjugated 19‐mer siRNA Targeting tmprss6, Reduces Plasma Iron and Increases Hepcidin Levels of Healthy Volunteers
SLN124, an N-acetylgalactosamine conjugated 19-mer short interfering RNA, is being developed to treat iron-loading anemias (including beta-thalassemia and myelodysplastic syndromes) and myeloproliferative neoplasms (polycythemia vera). Through hepatic targeting and silencing…
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Management of the Sickle Cell Trait: An Opinion by Expert Panel Members
The number of individuals with the sickle cell trait exceeds 300 million worldwide, making sickle cell disease one of the most common monogenetic diseases globally. Because of the high frequency…
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