Adherence to deferasirox in children and adolescents with sickle cell disease during 1-year of therapy.

Abstract

BACKGROUND:

Adherence to long-term treatment is challenging. Deferasirox (DFX) is a daily oral iron chelator approved in the United States for transfusional iron overload.

PROCEDURES:

Twenty-one subjects with sickle cell disease (mean age 13.8+/-4.2 y) received DFX 20 to 30 mg/kg daily for 1-year while on chronic blood transfusions. Good adherence to DFX was defined as > or =80% intake of prescribed dose. Adherence was assessed by monthly pill counts and calendars, and questionnaires at 1, 3, 6, and 12 months follow-up.

RESULTS:

Fifteen of 21 subjects (71%) were adherent to DFX according to self-reports, with 83% of the patients being adherent at 1 month, 89% at 3 months, 65% at 6 months, and 78% at 12 months. We were only able to document continuous good adherence in 43% of patients by pill counts because of poor bottle return. The discrepancy between pill counts and self-reports may be related to over-reporting, with the real adherence being lower. Parental involvement with DFX administration (P=0.03) and age < or =16 years (P=0.0055) correlated with adherence. We could not detect a significant correlation between serum ferritin declines and adherence.

CONCLUSION:

Seventy-one percent of patients adhered to DFX according to questionnaire responses but only 43% did according to pill counts. Adherence was poorer in adolescents older than 16 years of age and in patients who had no parental supervision of medications.

• Authors: Ofelia Alvarez, Hector Rodriguez-Cortes, Natasha Robinson, Noeline Lewis, Claudia Diaz Pow Sang, Gabriela Lopez-Mitnik, Carole Paley
• Journal: Journal of pediatric hematology/oncology (J Pediatr Hematol Oncol) Vol. 31 Issue 10 Pg. 739-44 (Oct 2009) ISSN: 1536-3678 [Electronic] United States
• PMID: 19734806 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Benzoates
• Iron Chelating Agents
• Triazoles
• Ferritins
• Deferasirox

Topics

• Adolescent
• Anemia, Sickle Cell (complications, drug therapy)
• Benzoates (administration & dosage)
• Blood Transfusion
• Child
• Deferasirox
• Female
• Ferritins (blood)
• Humans
• Iron Chelating Agents (therapeutic use)
• Iron Overload (drug therapy, etiology)
• Male
• Parents
• Patient Compliance (statistics & numerical data)
• Surveys and Questionnaires
• Triazoles (administration & dosage)