Study of Hydroxyurea to Treat Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02868138
• Other study ID #: MEUKAEK-2016/27 - 16-AKD-19
• Status: Completed
• Phase: N/A
• First received: August 8, 2016
• Last updated: August 13, 2016
• Start date: May 2016
• Est. completion date: July 2016

Study information

• Verified date: August 2016
• Source: Mersin University
• Contact: n/a
• Is FDA regulated: No
• Health authority: Turkey: Ministry of Health
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 50
• Est. completion date: July 2016
• Est. primary completion date: July 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 7 Years to 22 Years

Eligibility

Inclusion Criteria:

1. Patients willing to participate in the study

2. Pediatric and young adult patients

3. Patients diagnosed with HbSS or HbSß0 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 HbSß0 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

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Locations

Country	Name	City	State
Turkey	Selma Unal	Mersin

Sponsors (1)

Lead Sponsor	Collaborator
Mersin University

Country where clinical trial is conducted

Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Effectiveness and acceptance of hydroxyurea therapy in patients		Up to 12 weeks	Yes
Secondary	Demographic characteristics of patients using Demographic Data Collection Form		Up to 12 weeks	Yes
Secondary	Clinical characteristics of patients using Case Report Form		Up to 12 weeks	Yes
Secondary	Health status of pediatric patients using Child Health Questionnaire-Parent Form		Up to 12 weeks	Yes
Secondary	Quality of life of young adult patients using Life Quality Survey Short Form-36		Up to 12 weeks	Yes
Secondary	Effectiveness of hydroxyurea therapy in patients using Case Report Form		Up to 12 weeks	Yes
Secondary	Acceptance of hydroxyurea therapy in patients using Hydroxyurea Satisfaction Survey		Up to 12 weeks	Yes