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±4.2y) 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 language | English |
|---|---|
| Pages (from-to) | 739-744 |
| Number of pages | 6 |
| Journal | Journal of Pediatric Hematology/Oncology |
| Volume | 31 |
| Issue number | 10 |
| DOIs | |
| State | Published - Oct 1 2009 |
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Keywords
- Adherence
- Children
- Deferasirox
- Sickle cell anemia
- Sickle cell disease
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Oncology
- Hematology
Cite this
Adherence to deferasirox in children and adolescents with sickle cell disease during 1-year of therapy. / Alvarez, Ofelia A; Rodriguez-Cortes, Hector; Robinson, Natasha; Lewis, Noeline; Pow Sang, Claudia Diaz; Lopez-Mitnik, Gabriela; Paley, Carole.
In: Journal of Pediatric Hematology/Oncology, Vol. 31, No. 10, 01.10.2009, p. 739-744.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Adherence to deferasirox in children and adolescents with sickle cell disease during 1-year of therapy
AU - Alvarez, Ofelia A
AU - Rodriguez-Cortes, Hector
AU - Robinson, Natasha
AU - Lewis, Noeline
AU - Pow Sang, Claudia Diaz
AU - Lopez-Mitnik, Gabriela
AU - Paley, Carole
PY - 2009/10/1
Y1 - 2009/10/1
N2 - 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.
AB - 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.
KW - Adherence
KW - Children
KW - Deferasirox
KW - Sickle cell anemia
KW - Sickle cell disease
UR - http://www.scopus.com/inward/record.url?scp=70349843630&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=70349843630&partnerID=8YFLogxK
U2 - 10.1097/MPH.0b013e3181b53363
DO - 10.1097/MPH.0b013e3181b53363
M3 - Article
C2 - 19734806
AN - SCOPUS:70349843630
VL - 31
SP - 739
EP - 744
JO - Journal of Pediatric Hematology/Oncology
JF - Journal of Pediatric Hematology/Oncology
SN - 1077-4114
IS - 10
ER -