β-Thalassemia intermedia : morbidity uncovered

Leiden Repository

β-Thalassemia intermedia : morbidity uncovered

Title: β-Thalassemia intermedia : morbidity uncovered
Author: Musallam, Khaled Mousa SalehTaher, Ali Taher
Publisher: Department of Clinical Epidemiology, Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University
Issue Date: 2012-06-21
Keywords: Hemoglobinopathy
Ineffective erythropoiesis
Iron
Overload
Hypercoagulability
Thrombosis
Stroke
Quality of life
Transfusion
Management
Abstract: The research presented in this thesis provides several novel insights regarding the β-thalassemia intermedia phenotype. Earlier studies observed that patients with β-thalassemia intermedia experience a clinical complications profile that is different from that in patients with β-thalassemia major; which was primarily attributed to their transfusion-independence. In this work, a variety of clinical morbidities were explored and their associations with the underlying disease pathophysiology and risk factors were examined. The morbidities evaluated throughout the studies involved several organs and organ systems including the vasculature (venous thrombosis, pulmonary artery hypertension, cerebrovascular disease, and leg ulcers), heart, liver, kidney, endocrine glands (diabetes mellitus, hypothyroidism, and hypogonadism), bone (osteoporosis), and the hematopoietic system (extramedullary hematopoietic tumors). Findi ngs confirm that β-thalassemia intermedia should no longer be regarded as a mild form of thalassemia as patients experience serious manifestations involving almost every organ system.
Description: Promotor: F.R. Rosendaal, Co-Promotor: F. Peyvandi
With Summary in Dutch
Faculty: LUMC
Citation: Musallam, K.M., Taher, A.T., 2012, Doctoral Thesis, Leiden University
Handle: http://hdl.handle.net/1887/19124
 

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