Abstract

Introduction: Frequently transfused patients with SCD develop hemosiderosis, which, if untreated, results in multi-organ damage and early death. DFO is the only iron chelator (IC) approved by the FDA and is injected subcutaneously over 8–12 hours/day, 5–7 days/week. However, adherence to this therapy is difficult for patients. This report summarises patient satisfaction with deferasirox (DSX), an oral IC in development, compared to DFO in an open-label Phase II trial.

Methods: A total of 195 patients with SCD were randomized and treated with DSX (n=132) or DFO (n=63). Seventy-four patients had not received IC at baseline, of whom 25 were randomized to DFO and 49 to DSX. DSX was taken orally once/day while DFO was infused via a portable pump over 8–12 hours/day, 5–7 days/week. Patient reported outcome data were documented, including satisfaction and convenience with treatment, and number of hours lost/month for taking IC. Patients with previous experiences of DFO reported preferences for DSX, DFO, or neither. At end-of-study (EOS), patients reported their willingness to continue taking the drug they were on.

Results: At baseline, only 21% of patients previously treated with DFO reported that they were ‘very satisfied’ or ‘satisfied’ with DFO taken prior to the study, and only 16.5% reported that DFO was ‘very convenient’ or ‘convenient’. Further, 77.1% of them preferred DSX. Reasons included: more convenient to take (38.6%), less disruptive to their day (18.1%), less sore (15.7%) and less disruptive to sleep (6.0%). Table 1 indicates the greater satisfaction and convenience experienced by those on DSX versus DFO regardless of chelation status at baseline. At EOS, significantly more patients on DSX (84.3%) indicated they would be willing to continue DSX, compared to 10.5% of patients on DFO. Similarly, patients reported that time lost due to taking DSX ranged from 0.2–2.9 hours/month, while time lost due to taking DFO ranged from 3.2–37.4 hours/month.

Table 1.

Satisfaction and Convenience in Patients Previously Treated with DFO versus Treatment Naïve Patients

DSXDFO
Previous DFOTreatment naïvePrevious DFOTreatment naïve
Very Satisfied or Satisfied, % 
Wk 2 85.5 73.5 23.7 24.0 
Wk 12 89.2 63.3 42.1 36.0 
Wk 24 86.7 69.4 39.8 32.0 
EOS 84.3 59.2 23.7 24.0 
Very Convenient or Convenient, % 
Wk 2 79.5 73.5 15.8 32.0 
Wk 12 83.1 71.4 36.8 24.0 
Wk 24 79.5 71.4 34.2 24.0 
EOS 79.5 63.3 18.4 20.0 
DSXDFO
Previous DFOTreatment naïvePrevious DFOTreatment naïve
Very Satisfied or Satisfied, % 
Wk 2 85.5 73.5 23.7 24.0 
Wk 12 89.2 63.3 42.1 36.0 
Wk 24 86.7 69.4 39.8 32.0 
EOS 84.3 59.2 23.7 24.0 
Very Convenient or Convenient, % 
Wk 2 79.5 73.5 15.8 32.0 
Wk 12 83.1 71.4 36.8 24.0 
Wk 24 79.5 71.4 34.2 24.0 
EOS 79.5 63.3 18.4 20.0 

Conclusions: Results suggest satisfaction and convenience scores are significantly higher, and the impact on daily activities is less, for patients on DSX than those on DFO. Further studies are needed to explore whether higher satisfaction and convenience will lead to better adherence to DSX. As adherence to chelation therapy impacts on survival in transfusion-dependent patients with SCD, these data indicate that DSX may significantly improve the care of transfusion-dependent patients.

Author notes

Corresponding author