Long-term efficacy and safety of Hydroxycarbamide for pediatric sickle cell disease: Research Project MEDS2138_1210 Non-lab based Project
| المؤلف الرئيسي: | Al Yahiwi, Hanan Eid (Author) |
|---|---|
| التاريخ الميلادي: |
2012
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| موقع: | ملبورن |
| الصفحات: | 1 - 28 |
| رقم MD: | 620150 |
| نوع المحتوى: | رسائل جامعية |
| اللغة: | الإنجليزية |
| الدرجة العلمية: | رسالة ماجستير |
| الجامعة: | RMIT University |
| الكلية: | School of Medical Science |
| الدولة: | أستراليا |
| قواعد المعلومات: | +Dissertations |
| مواضيع: | |
| رابط المحتوى: |
الناشر لهذه المادة لم يسمح بإتاحتها. |
| المستخلص: |
Sickle cell disease (SCD) is severe haematological disorders with substantial morbidity and early mortality. Most patients suffer from acute complications and life-threatening events beginning in the first year of life. An oral chemotherapeutic drug, hydroxycarbamide (Hydroxyurea) has emerged as an important therapeutic option for sickle cell patients specifically children and more recently infants and toddlers. Although its efficacy has been proven by prior studies, concern exits regarding long-term approach especially in young children. Therefore, numerous clinical trials have been conducted to assess the use of hydroxycarbamide for treatment sickle cell children and infants. The purpose of this project was to provide updated data in the literature regarding long -term efficacy and safety of hydroxycarbamide exposure for children and infants with SCD. In particular, the effects of the hydroxycarbamide on acute and chronic complications associated with SCD such as pain episodes, organ damage, stroke and iron-overload were included. Additional chromosomal stability and gene expression assessments were considered to highlight possible long-term toxicities. After analysis, hydroxyurea was safe and resulted in clinical and laboratory benefits among groups in recently multicenter randomised controlled trials and prospective observational cohort studies. Adverse toxicity effects were slow. On the basis of these findings, the use of hydroxyurea treatment for SCD infants is warranted. |
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