An Oral Iron Chelator and Quality of Life.
Authors: De Abreu Lourenco Richard , Osborne Richard , Dalton Andrew , Houltram Jennifer , Dowton David , Joshua Douglas , Ho Phoebe Joy , Lindeman Robert 
Published: April 2020

Abstract
The impact on patients’ quality of life (QoL) is an important consideration in both decisions of individual patient care, and in allocating health care resources between competing treatments. This study aims to better inform those decisions by investigating the differential impact on patients’ QoL of two equally safe and effective iron chelators: one a subcutaneous infusion, the other a dispersible tablet. The current standard of care in patients with chronic iron overload secondary to haematological conditions such as the thalassaemias, myelodysplastic syndrome and sickle cell disease is the subcutaneous administration of desferrioxamine (DFO), for 8 to 12 hours per day, 5 to 7 days a week. The burden of this treatment regimen in some cases leads not only to poor compliance, and hence a reduction in the extent of effective iron chelation, but also to a reduction in patients’ QoL. Novartis Pharmaceuticals AG has developed a once daily oral iron chelator (deferasirox, DFX) shown to be as safe and effective as DFO in treating iron overload. To investigate the differential impact of DFO and DFX on patients’ QoL, a community sample of 110 individuals were asked to participate in a time trade off (TTO) exercise to elicit community utility values, or preferences, for their different modes of administration. Following standard TTO methodology, participants were asked how they value ten years of full health compared with time in each of three health states for patients with thalassaemia:
an anchor/base state that described a patient who has iron chelation without describing the treatment itself; the anchor state plus iron chelation via a subcutaneous infusion; the anchor state plus iron chelation via a once daily oral medication.
To avoid ordering bias, respondents were randomised to respond to either scenario (2) or (3) first after the anchor state. Scenarios were not labelled in terms of the iron chelator included and differed only in their description of the mode of treatment administration. Maximum possible utility values are 1 for perfect health and 0 for death. Preliminary results based on the first 32 respondents showed an average utility value of 0.73 for the anchor state, 0.57 for the subcutaneously infused iron chelator and 0.82 for the once a day oral iron chelator. The difference of 0.25 in the utility value for the two treatments was statistically significant (Wilcoxon-signed rank test, p=0.002). Treatment with the subcutaneously infused agent was associated with a lower QoL value, being 0.16 below that for the anchor state (p=0.025). In contrast, treatment with a once daily oral iron chelator had a utility value that was 0.08 (p=0.002) higher than the anchor state. While data collection is ongoing (completion date late Sept), these preliminary results indicate that society associates oral administration of an iron chelator such as DFX with an improvement in the QoL of patients requiring such treatment. Assuming equal safety and efficacy, a once daily oral treatment may therefore be preferred to a burdensome subcutaneous infusion due to the benefits it confers on patients’ QoL.