Patient and Caregiver Perspectives of Factors Associated with Adherence to and Satisfaction with Iron Chelation Therapy

Background

Adherence to iron chelation therapy (ICT) is critical in patients with transfusion dependent anemias (TDA) (e.g., sickle cell disease [SCD], thalassemia), myelodysplastic syndromes (MDS), aplastic anemia, and myelofibrosis (MF). One of the biggest challenges is poor adherence to therapy, which in turn could cause tissue damage in vital organs such as the heart, liver, and endocrine organs, leading to end-organ dysfunction and eventually organ failure. Some causes of poor adherence to ICT therapy in the literature include inconvenience and palatability issues. The literature also suggests that satisfaction with medication impacts adherence to medication regimens, and that satisfaction is an umbrella term for specific components such as convenience, and efficacy. There is limited information on the factors that influence adherence with ICT, however, from the patient and caregiver perspective.

Aim

The objective of this study was to explore the factors that influence adherence with ICT from the perspective of patients and caregivers of patients through qualitative research.

Methods

Semi-structured interviews including an open-ended component were conducted to elicit subjects’ (patients with TDA, MDS, aplastic anemia, or MF or caregivers thereof) experiences with ICT. Institutional Review Board approval was granted and subjects were screened by clinicians to meet pre-defined inclusion and exclusion criteria. Audio recordings of the interviews were transcribed and a code book that contained definitions of all codes was developed through a consensus process. Transcripts were coded and data were analyzed using Atlas ti, a qualitative data analysis software package. Several concepts related to adherence and satisfaction were identified.

Results

Four interviewers conducted 11 patient and 10 caregiver interviews in 6 US cities. The children of the caregivers were not interviewed as they were below the age of nine. Eight of the eleven patients were male and three were female, aged 14 to 81 years (mean=43). Nine of the ten caregivers were female, aged 35 to 65 years (mean=48). Six of the ten children of caregivers were female and four were male, with a mean age of 8.9 years (range 1.56-16.81 years). Conditions across pediatric and adult patients included: MDS (n=3), SCD (n=13), thalassemia (n=3), MF (n=1), and aplastic anemia (n=1). Reported reasons for adherence included perceived effect on health and longevity, support from clinicians and caregivers, and an established routine for taking their treatment. Reported reasons for non-adherence included palatability related issues of taste and aftertaste, texture of the ICT formulation, solubility, side effects such as GI symptoms, and mealtime restrictions. Caregivers reported better adherence if their children had a better understanding of treatment benefit, which usually “gets better as the child gets older”; physician involvement in explaining the importance of ICT; and patients witnessing the decrease in their iron levels. Caregivers indicated that if they were satisfied with the efficacy of the ICT, their children were more likely to adhere to their medications. Coping mechanisms included modifying how the medicine was taken to change or mask the taste, aftertaste, or texture and “dose splitting” during the day.

Conclusions

Factors associated with adherence and non-adherence to ICT and satisfaction with current therapies identified by patients and caregivers of patients provide information and insight for clinicians to provide optimal treatment with ICT and gain better adherence. These factors can be used to develop measures of adherence, palatability, GI symptoms, and satisfaction with ICT and design interventions to improve adherence to ICT.