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


METHODS
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.


CONCLUSIONS
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.
Alvarez
Ofelia
O
Department of Pediatrics, Division of Pediatric Hematology-Oncology, University of Miami Miller School ofMedicine, Miami, FL 33136, USA. oalvarez2@-med.miami.edu
Rodriguez-Cortes
Hector
H
Robinson
Natasha
N
Lewis
Noeline
N
Pow Sang
Claudia Diaz
CD
Lopez-Mitnik
Gabriela
G
Paley
Carole
C
D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000150:complications / Q000188:drug therapy*; D001565:Benzoates / Q000008:administration & dosage*; D001803:Blood Transfusion; D002648:Child; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use; D019190:Iron Overload / Q000188:drug therapy / Q000209:etiology; D008297:Male; D010290:Parents; D010349:Patient Compliance / Q000706:statistics & numerical data*; D011795:Surveys and Questionnaires; D014230:Triazoles / Q000008:administration & dosage*
D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D000077588:Deferasirox