Study of Hydroxyurea to Treat Sickle Cell Disease

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Evaluation of Clinical Effectiveness, Quality of Life, and Compliance in Patients With Sickle Cell Disease Receiving Hydroxyurea

Conditions
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Sickle Cell Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients willing to participate in the study Pediatric and young adult patients Patients diagnosed with HbSS or HbSβ0 sickle cell disease Patients receiving hydroxyurea for at least a year. Exclusion Criteria: Patients not meeting the inclusion criteria Patients not willing to participate in the study Patients diagnosed with other types of anemia except HbSS or HbSβ0 sickle cell disease Patients not receiving hydroxyurea for at least a year Patients having other conditions such as physical and/or mental difficulties which may affect their quality of life Patients having any contrindication against hydroxyurea

Ages Eligible for Study
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Minimum Age: 7 Years
Maximum Age: 22 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effectiveness and acceptance of hydroxyurea therapy in patients |  | Up to 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Demographic characteristics of patients using Demographic Data Collection Form |  | Up to 12 weeks | 
| Clinical characteristics of patients using Case Report Form |  | Up to 12 weeks | 
| Health status of pediatric patients using Child Health Questionnaire-Parent Form |  | Up to 12 weeks | 
| Quality of life of young adult patients using Life Quality Survey Short Form-36 |  | Up to 12 weeks | 
| Effectiveness of hydroxyurea therapy in patients using Case Report Form |  | Up to 12 weeks | 
| Acceptance of hydroxyurea therapy in patients using Hydroxyurea Satisfaction Survey |  | Up to 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Sickle cell disease, Hydroxyurea, Compliance