The challenges of adherence and persistence with iron chelation therapy

Abstract

Due to advances in medical sciences, many chronic diseases that formerly resulted in early death can now be effectively managed with long-term treatment regimens. Patients with potentially fatal anemias, for example, can be treated with ongoing blood transfusions and iron chelation therapy. Ensuring adherence and persistence is challenging, as the benefits of therapy are not perceived immediately. Poor adherence severely compromises the effectiveness of treatment and, therefore, improving compliance in terms of quality of life and health economics is critical. Although adherence to chelation therapy is generally poor, the availability of oral iron chelators may help to improve patient compliance. For chronic conditions such as thalassemia major, even when oral chelation therapy is available, support by an integrated team including a clinical psychologist and nurse specialist working with the treatment center is recommended to achieve optimal results.

Advances in iron chelation therapy: transitioning to a new oral formulation

“…In two studies of deferasirox that evaluated patient-reported outcomes, injection-site reactions were cited as being one of the reasons for preferring deferasirox to deferoxamine [36,37]. Tolerability issues, such as the injection-site reactions associated with deferoxamine and the GI side effects associated with deferasirox, are a concern for older patients with MDS that impact adherence [38]. …”

Impact of psychosocial status and disease knowledge on deferoxamine adherence among thalassaemia major adolescents

“…Deferoxamine (DFO) has been the principal iron chelating therapy for many years, and it has altered the prognosis of iron overload in patients with transfusion‐dependent β‐thalassaemia . DFO is effective only when administered parenterally, using infusion pumps that dispense an equal amount of drug over a 10–12 h period for 4–7 times a week.…”

“…DFO is effective only when administered parenterally, using infusion pumps that dispense an equal amount of drug over a 10–12 h period for 4–7 times a week. This modality of treatment, although effective, is very painful and locally irritating . Thus, it is not surprising that adherence with such a difficult treatment decreases over time …”

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

“…The economic, clinical, and humanistic impact of iron overload may become substantial, and poor adherence to ICTs has been shown to result in serum ferritin targets to not be achieved, which negatively impacts costs, morbitidy and mortality, and healthrelated quality of life [31,[40][41][42]. The long-term ramifications of nonadherence to ICTs in persons with iron overload may manifest in severe clinical complications, particularly hepatic failure, iron-induced cardiomyopathy, or pancreatic iron deposition [9].…”

“…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].…”