Sickle Cell Disease Clinical Trial
Official title:
Evaluation of Clinical Effectiveness, Quality of Life, and Compliance in Patients With Sickle Cell Disease Receiving Hydroxyurea
The aim of this single-center observational study was to evaluate quality of life, clinical effectiveness, and satisfaction in pediatric and young adult patients with sickle cell disease receiving hydroxyurea.
In this study, 34 pediatric (HbSS: n= 5; HbSβ0: n= 29) and 16 (HbSS: n=5; HbSβ0: n= 11)
young adult adult patients with sickle cell disease receiving hydroxyurea for at least a
year were participated. Upon receipt of Informed Consent Form, Case Report Form, Demographic
Data Collection Form, Child Health Questionnaire-Parent Form, Life Quality Survey Short
Form-36, and Hydroxyurea Therapy Satisfaction Survey were used to obtain data for
effectiveness of hydroxyurea therapy and parameters that may affect compliance to treatment
and life quality of the participants.
Regarding the normal ranges, ferritin, hemoglobin A, A2, F, and S, platelet, mean
corpuscular volume, erythrocyte distribution width, basophil percentage, monocyte, monocyte
percentage, total bilirubin, direct bilirubin, and C-reactive protein values were higher
while hemoglobin, hematocrit, and erythrocyte values were lower in these patients. Our
findings regarding quality of life and satisfaction with hydroxyurea therapy indicated that
the patients with sickle cell disease had lower scores.
Demographic, clinical, and therapeutic variables as well as comorbid diseases and
concomitant drug use when considered together, these findings suggest that the health
quality and compliance of the pediatric and young adult patients to therapy might be low due
to not sufficiently effective hydroxyurea therapy in addition to comorbidities, concomitant
drug use, and side effects.
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Observational Model: Cohort, Time Perspective: Prospective
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