Study of Hydroxyurea to Treat Sickle Cell Disease

Mersin University50 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Sickle Cell Disease

Eligibility

Sex: ALLMin age: 7 YearsMax age: 22 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Patients willing to participate in the study 2. Pediatric and young adult patients 3. Patients diagnosed with HbSS or HbSβ0 sickle cell disease 4. Patients receiving hydroxyurea for at least a year. Exclusion Criteria: 1. Patients not meeting the inclusion criteria 2. Patients not willing to participate in the study 3. Patients diagnosed with other types of anemia except HbSS or HbSβ0 sickle cell disease 4. Patients not receiving hydroxyurea for at least a year 5. Patients having other conditions such as physical and/or mental difficulties which may affect their quality of life 6. Patients having any contrindication against hydroxyurea

Locations (1)

Selma Unal

Mersin, Turkey (Türkiye)

Outcomes

Primary Outcomes

Effectiveness and acceptance of hydroxyurea therapy in patients

Time frame: Up to 12 weeks

Secondary Outcomes

Demographic characteristics of patients using Demographic Data Collection Form

Time frame: Up to 12 weeks

Clinical characteristics of patients using Case Report Form

Time frame: Up to 12 weeks

Health status of pediatric patients using Child Health Questionnaire-Parent Form

Time frame: Up to 12 weeks

Quality of life of young adult patients using Life Quality Survey Short Form-36

Time frame: Up to 12 weeks

Effectiveness of hydroxyurea therapy in patients using Case Report Form

Time frame: Up to 12 weeks

Acceptance of hydroxyurea therapy in patients using Hydroxyurea Satisfaction Survey

Time frame: Up to 12 weeks

Data from ClinicalTrials.gov

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