Adherence to Hydroxyurea in Children With Sickle Cell Anemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00672789
Recruitment Status : Completed
First Posted : May 6, 2008
Last Update Posted : May 27, 2013
National Institutes of Health (NIH)
Information provided by (Responsible Party):
Duke University

Brief Summary:

Medication non-adherence is a true public health problem. Despite advancements in the molecular understanding of disease and improvements in therapy, patient health outcomes will not improve unless patients take prescribed medications regularly. Decreasing the gap between efficacious and effective therapy for patients with SCD is an essential research agenda. Hydroxyurea has been shown to be safe and efficacious in children and infants. However, the effectiveness of the prophylaxis depends on adherence to the recommended regimen. Medication adherence in SCD has previously been found to be sub-optimal in patients taking penicillin, desferoxamine, and pain medication. Adherence to HU has been studied to some extent in children with SCD. Based on estimates of adherence in other chronic illness we expect approximately 50% of patients to be >80% adherent with their HU administration. There is no gold standard for improving adherence to treatment. There have been a few attempts in the SCD population to improve adherence. These include a day camp to promote education about desferoxamine and peer support, a combination of a slide-show about SCD and it complications, weekly phone calls by the clinic social worker and a calendar, and a seven-phase educational program. Given the striking improvements in the peripheral blood smear findings of patients with SCD on HU therapy, with reduction in the numbers of sickled cells, we hypothesize that viewing the peripheral blood smear of patients with poor adherence to HU compared to a blood smear of someone on HU can be used to improve adherence in non-adherent patients.

We will conduct a randomized trial between the intervention of regularly showing children and their parents the peripheral blood smear and standard care, including reminders of the importance of compliance and review of complete blood count parameters, including WBC, MCV, and Hgb concentration. The outcome measures will be increase in hemoglobin concentration and %HbF and increase in perceived QOL. QOL will be measured with age-appropriate and parent/proxy PedsQL™. Medication adherence will also be monitored throughout the study with pharmacy prescription refills, physician assessment, and self-report via a visual analogue scale. Adherence estimates, hemoglobin concentration, %HbF and QOL will be measured at baseline, 3 months and 6 months.

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Behavioral: Blood smear education Other: standard education Not Applicable

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 31 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Health Services Research
Official Title: Adherence to Hydroxyurea in Children With Sickle Cell Anemia
Study Start Date : April 2008
Actual Primary Completion Date : November 2012
Actual Study Completion Date : November 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia
Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Experimental: 1
Blood Smear Education
Behavioral: Blood smear education
education at baseline and three month follow-up

Active Comparator: 2
Standard education
Other: standard education
standard education

Primary Outcome Measures :
  1. treatment adherence [ Time Frame: six months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Sickle cell anemia
  • on Hydroxyurea for at least six months
  • age 2-17.9 years

Exclusion Criteria:

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00672789

United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Duke University
National Institutes of Health (NIH)
Principal Investigator: Courtney D Thornburg, MD, MS Duke University

Responsible Party: Duke University Identifier: NCT00672789     History of Changes
Other Study ID Numbers: Pro00000118
First Posted: May 6, 2008    Key Record Dates
Last Update Posted: May 27, 2013
Last Verified: November 2012

Keywords provided by Duke University:
Sickle cell anemia
Treatment Adherence

Additional relevant MeSH terms:
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors