Abstract
Abstract 5295
In 2006, deferasirox was licensed in Canada for use in children over the age of 6 years. Deferasirox (Exjade â, ICL 670) is an orally active iron chelators which represents the new class of tridentate iron chelator. Deferasirox has been formulated as a dispersible tablet based on technical feasibility, stability, and adequate bioavailability. Deferasirox has been shown to be as effective as deferral (DFO) and has a clinically manageable safety profile (the most common side effects are gastrointestinal disturbances and rashes). Few studies have been conducted to compare the satisfaction, convenience, activity limitations, and patient preferences of deferasirox in comparison with DFO in sickle cell and thalassemia patients. The purpose of the present study was to evaluate the long-term response of deferosirox in iron-overloaded children aged 6 years or older treated with transfusion dependent anemia and secondary objective was to collect and analyze the following parameters such as serum ferrtin, SLT, AST, creatinine, audiology and ophthalmology, liver iron concentrations (LIC) and patient reported outcomes measures over a period of 2 years from the start of deferasirox.
The study population comprised of male and female patients aged 6 years or <17.99 with chronic iron overload related to blood transfusions in patients with Thalassemia and Sickle cell disease. Fifty-one patients were enrolled into this study at the Hematology Clinic, Hospital for Sick Children, Toronto, ON Canada. This study was observational and does not impose a therapy protocol, diagnostic/therapeutic interventions or a strict visit schedule. Patients were treated with deferasirox according to the local physician's judgment and in accordance with the local (country-specific) deferasirox prescribing information. Data were collected at study entry (baseline), and at 12 months. Patients were approached for the study after the ethics approval was obtained. All variables were explored and summarized using descriptive statistics such as means, standard deviation, median and ranges, counts and proportions, graphs as appropriate. Patients reported outcomes measures questionnaires were given after the written consent/assent was obtained. Statistical analysis was done by descriptive statistics and paired t-test.
Of the 51 subjects, 30 were females, 21 were males, and median age for the patients was 14 ± 5.3. Out of 51 patients, 49 patients had thalassemia and two had sickle cell disease. All of the subjects were on desferal chelation therapy before the start of the deferasirox, Analysis of patient reported outcomes measures showed that majority of the patients (73%) were very satisfied with deferasirox, when they started and after 12 months on the study, however 1 patient who reported dissatisfaction in 2009, reported satisfaction in year 2010. The main reason for the deferasirox, treatment preference were relief of pain associated with injections (30%) and more convenient (35%) in administration. Other reasons given were improved sleep patterns (2%) and less disruption to the family (6%).
At baseline and 12 months laboratory evaluations were as follows: Mean Creatinine values (88% of the patients) (baseline- 34.41±11.49, 12 months – 52.41 ± 19.82), Mean ALT (65% of the patients) (baseline- 41.73 ± 7.32, 12 months – 30.94 ± 5.30) and serum ferritin (baseline- 1995± 276, 12 month, 1833 ± 203). Mean LIC (baseline-11.08 ± 1.29, 12 months- 7.87 ± 1.12) evaluations showed the incremental decrease over time with 14/51 showed the values of <3 over a 12 months period. Chelation therapy was held for some patients for a short period to prevent toxicity related to the chelator. Mild (4/51) and moderate (1/51) hearing loss were observed in subjects over period from 2009–2010. Data will be collected after 24 months to evaluate their long-term responses and for comparative analysis. Further, we can conclude that given the high levels of satisfaction, it is likely that the quality of life and adherence to chelation treatment have improved for patients who are receiving the deferasirox treatment compared with previous subcutaneous chelation treatment.
Disclosures:
No relevant conflicts of interest to declare.
Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework
