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The prevalence of disease- related complications in Egyptian children and young adults with sickle cell disease receiving hydroxycarbamide /

Ramy Loka Nageh

The prevalence of disease- related complications in Egyptian children and young adults with sickle cell disease receiving hydroxycarbamide / تأثير استخدام دواء الهيدروكسى كارباميد على مضاعفات مرض الأنيميا المنجليه فى الاطفال وصغار الشباب المصريين Ramy Loka Nageh ; Supervised Amina Abdelsalam Mahmoud , Mai Mohamed Mohamed Abdelsalam , Mona Ahmed Kamel - Cairo : Ramy Loka Nageh , 2020 - 145 P. : charts , facimiles ; 25cm

Thesis (M.Sc.) - Cairo University - Faculty of Medicine - Department of Pediatrics

Background: Although hydroxyurea has an established role in improving laboratory parameters and reducing acute clinical complications, its role in preventing organ damage in SCD is less well defined. Objectives: To assess the prevalence of disease- related complications in Egyptian patients with SCD receiving HU. Materials and Methods: This is a retrospective analytic study carried out on 60 SCD patients who were receiving HU therapy for at least 1 year. Details of HU therapy, clinical and laboratry data before and after HU were recorded. Results: All patients received HU at a starting dose 15 mg/kg/day with a maximum tolerated dose reached 35 mg/kg/day in 2 patients. Mean HU dose was 19 ± 4.9 mg/ kg/day and mean duration of HU treatment was 6.2 years.There was significant reduction in the frequency of vasoocclusive crises per year, frequency of blood transfusion and rate of hospitalization (p0.05). No significant difference was detected before and after using HU as regards the frequency infections, sequestration, aplastic crises, leg ulcers and cardiac complications (p0.05). One patient developed leg ulcers and another one had pulmonary hypertension while on HU. None of the studied patients had evidence of avascular necrosis at starting HU, however, eight (13.3%) patients developed bone changes while on HU. Two (3.4%) patients developed stroke while on HU and they were shifted to chronic transfusion regimen. There were a significant increases in total Hb, and significant decrease in TLC and reticulocytic count (p0.05). A statistically significant improvement of school scores among patients was seen after HU treatment (P=0.025). A marginally significant improvement of quality of life scores was detected among patients after using HU (P=0.05)



Disease related complications Egyptian children Hydroxycarbamide