Catherine Skari

Intrabone Hematopoietic Stem Cell Gene Therapy for Adult and Pediatric Patients Affected by Transfusion-dependent ß-Thalassemia

ß-thalassemia is caused by ß-globin gene mutations resulting in reduced (β+) or absent (β0) hemoglobin production. Patient life expectancy has…

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Risk Factors for Mortality in Adult Patients with Sickle Cell Disease: A Meta-analysis of Studies in North America and Europe

Although recent studies show an improved survival of children with sickle cell disease in the US and Europe, for adult…

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Iron Metabolism Under Conditions of Ineffective Erythropoiesis in β-Thalassemia

β-Thalassemia (BT) is an inherited genetic disorder that is characterized by ineffective erythropoiesis (IE), leading to anemia and abnormal iron…

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Hepatocellular Carcinoma in β-Thalassemia Patients: Review of the Literature with Molecular Insight into Liver Carcinogenesis

With the continuing progress in managing patients with thalassemia, especially in the setting of iron overload and iron chelation, the…

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Evaluation of Endocrine Complications in Beta-Thalassemia Intermedia Patients: A Cross Sectional Multi-Center Study

The diagnosis of beta thalassemia intermedia (BTI) is mainly based on the severity of clinical phenotype. It is associated with…

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Nutritional Deficiencies Are Common in Patients with Transfusion-dependent Thalassemia and Associated with Iron Overload

Patients with thalassemia are frequently deficient in key micronutrients. Attempts to correct these inadequacies through nutritional supplementation have been met…

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Amustaline-glutathione Pathogen-reduced Red Blood Cell Concentrates for Transfusion-dependent Thalassaemia

Transfusion-dependent thalassaemia (TDT) requires red blood cell concentrates (RBCC) to prevent complications of anaemia, but carries risk of infection. Pathogen…

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An ICET-A Survey on Occult and Emerging Endocrine Complications in Patients with β-Thalassemia Major: Conclusions and Recommendations

In adult thalassemia major (TM) patients, a number of occult and emerging endocrine complications, such as: central hypothyroidism (CH), thyroid…

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Iron Chelators or Therapeutic Modulators of Iron Overload: Are We Anywhere Near Ideal One?

Iron overload is an important cause of mortality and morbidity in chronic transfusion-dependent states commonly exemplified by transfusion-dependent beta-thalassaemia syndromes…

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Estimating the Birth Prevalence and Pregnancy Outcomes of Congenital Malformations Worldwide

Congenital anomaly registries have two main surveillance aims: firstly to define baseline epidemiology of important congenital anomalies to facilitate programme,…

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