Iron chelation adherence to deferoxamine and deferasirox in thalassemia

Abstract

The Thalassemia Clinical Research Network collected adherence information from 79 patients on deferoxamine and 186 on deferasirox from 2007 to 2009. Chelation adherence was defined as percent of doses administered in the last 4 weeks (patient report) out of those prescribed (chart review). Chelation history since 2002 was available for 97 patients currently on deferoxamine and 217 on deferasirox, with crude estimates of adherence from chart review. Self-reported adherence to both deferoxamine and deferasirox were quite high, with slightly higher adherence to the oral chelator (97 vs. 92%). Ninety percent of patients on deferasirox reported at least 90% adherence, compared with 75% of patients on deferoxamine. Adherence to both chelators was highest in children, followed by adolescents and older adults. Predictors of lower deferoxamine adherence were smoking in the past year, problems sticking themselves (adults only), problems wearing their pump, and fewer transfusions in the past year. Predictors of lower deferasirox adherence were bodily pain and depression. Switching chelators resulted in increased adherence, regardless of the direction of the switch, although switching from deferoxamine to deferasirox was far more common. As adherence to deferoxamine is higher than previously reported, it appears beneficial for patients to have a choice in chelators.

Toward optimizing the use of deferasirox: potential benefits of combined use with deferoxamine

“…With adherence to the use of DFO being a major issue in the management of b-thalassemia, 13,49 our results highlight the need for more patient-friendly chelation regimens. The issue, then, is how best to optimize the use of the oral agents.…”

Deferiprone (GPO‐L‐ONE®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

“…It is predicted that these oral medications should provide better overall outcome with chelation therapy compared with DFO due to their ease of use with better compliance [17,18]. Deferasirox, a once-daily oral chelator, showed efficacy with an acceptable safety profile in adult and pediatric populations with up-to 5-year follow up in a large scale prospective clinical studies [19].…”

Long-term persistency and costs associated with the use of iron chelation therapies in the treatment of Sickle cell disease within Medicaid programs

“…Therein, Gabutti and Piga reported an average adherence of 64% for DFO over a 30-year time horizon, while Weissman et al reported that 62% of persons with thalassemia or SCD were adherent to DFO treatment [27,28]. In more closely-related work comparing patterns of use with DFO and DFX, Trachtenberg et al measured adherence through patient self-reports and medical chart reviews from 2007-2009 among 265 individuals on DFO or DFX [29]. Results for the short-term nonadherence at one month were low, at 8% for DFO and 3% for DFX, though these values worsened with age; comparative results for treatment discontinuations in the current study using claims data were 17% for DFO and 15% for DFX.…”

“…It is also important to note that persons with SCD have reported significant barriers in access to health care, particularly those associated with delays in treatment [43]. Predictors of nonadherence in SCD remain complex and multifaceted, and current suggestions state that an integrated team approach involving healthcare providers, family caregivers, and treatment centers be utilized to achieve optimal results with chronic therapies [29,42].…”