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

Ofelia Alvarez, Hector Rodriguez-Cortes, Natasha Robinson, Noeline Lewis, Claudia Diaz Pow Sang, Gabriela Lopez-Mitnik, Carole Paley

Research output: Contribution to journalArticlepeer-review

28 Scopus citations


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±4.2y) received DFX 20 to 30 mg/kg daily for 1-year while on chronic blood transfusions. Good adherence to DFX was defined as ≥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 ≤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.

Original languageEnglish (US)
Pages (from-to)739-744
Number of pages6
JournalJournal of Pediatric Hematology/Oncology
Issue number10
StatePublished - Oct 2009


  • Adherence
  • Children
  • Deferasirox
  • Sickle cell anemia
  • Sickle cell disease

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Oncology
  • Hematology


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