Patient-Reported Outcomes of Deferasirox (Exjade®, ICL670) versus Deferoxamine in Sickle Cell Disease Patients with Transfusional Hemosiderosis

Abstract

Background/Aims: There is increasing evidence demonstrating the value of transfusions in sickle cell disease (SCD). However, resultant iron overload can be life threatening if untreated. Chelation therapy with deferoxamine requires parenteral infusions that can negatively impact quality of life and adherence to treatment. Methods: As part of a phase II trial, SCD patient-reported outcomes were evaluated. One hundred and ninety-five patients were randomized (2:1) to receive oral deferasirox (5–30 mg/kg/day) or deferoxamine (20–50 mg/kg, 5 days per week); 121 had previously received deferoxamine. Results: At each time point, significantly more patients who had previously received deferoxamine were ‘satisfied/very satisfied’ with deferasirox, or found treatment to be ‘convenient/very convenient’ compared with deferoxamine (p < 0.001). In these patients, fewer hours were lost from daily activities with deferasirox than deferoxamine treatment. Most patients (77%) preferred deferasirox, and more were willing to continue taking deferasirox than deferoxamine at end-of-study (84 vs. 11%, respectively). Conclusions: Patients with SCD are therefore more satisfied with deferasirox, which has a lower impact on daily activities than deferoxamine. Given the high levels of satisfaction, it is likely that quality of life will be improved. These results also suggest that treatment adherence with deferasirox may be better than with deferoxamine, which should lead to improved long-term outcomes.

New film‐coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower‐risk MDS: Results of the randomized, phase II ECLIPSE study

“…3,18 Patient satisfaction rates for deferasirox DT have been reported to be as high as 90%, 19 yet studies have shown that most patients dislike the mode of administration for deferasirox DT and would prefer to be able to take their medication with food. 20 Barriers such as these likely contribute to a reduced patient-reported adherence to deferasirox DT of 67-86%.…”

Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia

“…Recent advances have led to the development of new oral iron chelators, such as deferasirox, which has changed the clinical practice in several transfusiondependent anemias with iron overload. Vichinsky et al suggest that treatment adherence with deferasirox may be better than that of desferrioxamine and thus it should lead to improved long-term outcomes [18]. Deferasirox mobilizes iron stores by binding selectively to the ferric form of iron.…”

The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores

“…Numerous well-controlled clinical trials have demonstrated the efficacy of once-daily oral deferasirox in controlling liver iron concentration (LIC). [6][7][8][9][10][11] Deferasirox is preferred by patients, 12 improves compliance, 13 has a 12-to 16-hour half-life, 14,15 and is routinely prescribed 7 days per week. As a result, deferasirox chronically suppresses labile plasma iron levels better than intermittent, subcutaneous DFO therapy.…”