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NCT Number: NCT02868138

Study of Hydroxyurea to Treat Sickle Cell Disease

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Behavior Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Health Behavior Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Patient Acceptance of Health Care Patient Compliance Treatment Adherence and Compliance

Age range

7 year–22 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Selma Unal

Mersin, 33169, Turkey (Türkiye)

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Effectiveness and acceptance of hydroxyurea therapy in patients

    Time frame: Up to 12 weeks

Secondary outcomes

  1. Demographic characteristics of patients using Demographic Data Collection Form

    Time frame: Up to 12 weeks

  2. Clinical characteristics of patients using Case Report Form

    Time frame: Up to 12 weeks

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

    Time frame: Up to 12 weeks

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

    Time frame: Up to 12 weeks

  5. Effectiveness of hydroxyurea therapy in patients using Case Report Form

    Time frame: Up to 12 weeks

  6. Acceptance of hydroxyurea therapy in patients using Hydroxyurea Satisfaction Survey

    Time frame: Up to 12 weeks

Sponsors and collaborators

Lead sponsor

Mersin University

Other

Registry information

Official study title

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

Important dates

Study start
2016
Primary completion
2016
Study completion
2016
First posted
Aug 16, 2016
Registry last updated
Aug 16, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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