Therapeutic approaches in common pediatric bleeding disorders

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Absztrakt

Although both morbidity and mortality have been improved due to advancements in management possibility, bleeding disorders in pediatric population are one of the most threatening diseases that bring concerns to the patients and their families as the diseases are often diagnosed when serious bleeding conditions or repetitive bleeding episodes present. Upon diagnosis, following management is the key to not only save patients’ lives in a timely manner, but also to provide appropriate and planned management to prevent subsequent complications and to support the patient’s later life. To achieve prompt diagnosis and provide optimal pharmacological therapy, classification of disease based on the common age of onset and corresponding approach to the patients are of especial importance because age-specific manifestations of diseases determine needs in treatments in each developmental stage. Accordingly, in this thesis, the pediatric population is subclassified into three categories: neonates, infants/ school-age, and adolescents. Among wide ranges of diseases that possibly confront the pediatric patients, particularly characteristic disorders to each age group will be discussed in separate chapters. In neonates, ITP caused by maternal ITP, DIC, VKDB, congenital factor deficiency (i.e. FXIII, α2-AP deficiency, afibrinogenemia) are focused on. In infants and school-age, hemophilia, DIC, VKDB, and platelet dysfunctional disorder (i.e. Glanzmann thrombasthenia), and VWD and ITP are attended to in adolescent period. Following brief overview of pathomechanisms behind the diseases, currently available pharmacological management options with advantages and disadvantages, and in particular cases where potentially novel therapies are to be noted, they are further discussed with the current studies to provide insights in future options. With the managements comprehended, discussion is dedicated to unfold the perspectives of needs in pediatric management and their families in addition to pharmacological management to deepen understanding of the issues to be addressed along with the pharmacological management of pediatric bleeding disorders.

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pediatric, bleeding disorder, Therapeutic approach
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