Clinical trial NCT02868138: Study of Hydroxyurea to Treat Sickle Cell Disease
Official title: Evaluation of Clinical Effectiveness, Quality of Life, and Compliance in Patients With Sickle Cell Disease Receiving Hydroxyurea
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Keywords: Sickle cell disease
Hydroxyurea
Compliance
Lead sponsor: Mersin University
Sponsor class: OTHER
First posted: 2016-08-16
Enrollment: 50
Sex: ALL
Minimum age (years): 7
Maximum age (years): 22
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric (7-17 years) and young adult (18-22 years) patients with sickle cell disease who received hydroxyurea

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Effectiveness and acceptance of hydroxyurea therapy in patients
timeFrame: Up to 12 weeks
Secondary outcomes:
measure: Demographic characteristics of patients using Demographic Data Collection Form
timeFrame: Up to 12 weeks
measure: Clinical characteristics of patients using Case Report Form
timeFrame: Up to 12 weeks
measure: Health status of pediatric patients using Child Health Questionnaire-Parent Form
timeFrame: Up to 12 weeks
measure: Quality of life of young adult patients using Life Quality Survey Short Form-36
timeFrame: Up to 12 weeks
measure: Effectiveness of hydroxyurea therapy in patients using Case Report Form
timeFrame: Up to 12 weeks
measure: Acceptance of hydroxyurea therapy in patients using Hydroxyurea Satisfaction Survey
timeFrame: Up to 12 weeks
Other outcomes:
Locations:
city: Mersin
country: Turkey
facility: Selma Unal
Study officials:
affiliation: MERSIN UNIVERSITY FACULTY OF PHARMACY DEPARTMENT OF PHARMACOLOGY
name: BAHAR TUNCTAN, Ph.D.
role: STUDY_CHAIR
References: