Journal Publishes Exjade Food Study Results
April 30, 2013 – The medical journal Pediatric Blood and Cancer published online on April 23 a study entitled “The Palatability and Tolerability of Deferasirox Taken With Different Beverages or Food.”
This is known to many in the thalassemia community as “the Exjade food study;” as the official title suggests, it looks at the option of taking Exjade with various drinks other than those recommended on the label; with food; and without food.
The Exjade label currently specifies that the drug should be taken mixed with water, orange juice or apple juice and that it be taken on an empty stomach, 30 minutes before a meal. Some individuals find the limited choice of beverages and the need to take on an empty stomach inconvenient and would welcome an expanded range of options. This study provides clear indications that an expanded range of options are possible.
The study enrolled 65 patients with either sickle cell (38%), thalassemia (31%), Myelodysplastic syndromes(14%) or other anemias (17%).
In the study, patients were given the following options for administering deferasirox:
- with breakfast, crushed and added to soft food
- with breakfast, dispersed in a beverage of choice
- with dinner, crushed and added to soft food
- with dinner, dispersed in a beverage of choice
- With no meal, dispersed in a beverage of choice
These options began after a four-week “run-in” period during which patients administered deferasirox in the manner recommended on the label. The above options were implemented during the following 12-week period.
Among the findings:
- Favorable palatable ratings increased from 38% during the run-in period to 47% during the 12-week assessment period; unfavorable ratings decreased from 35% to 18%. (Patients like the taste of the food options and dislike the current beverage suggestions on the label!)
- Over the course of the study, serum ferritins remained stable among patients aged 18 to 60; ferritins trended downward in other age groups.
- 37% of patients experience gastrointestinal adverse events in the 4-week run-in phase as compared to 32% during the 12-week assessment phase.
- The authors state that the study “demonstrated that deferasirox could be administered by additional modes with potential improvements in palatability and tolerability and without obvious increases in toxicities.”
Ellis Neufeld, MD, Chair of CAF’s Medical Advisory Board, comments that “preliminary results of this study have been known for more than two years, and it is terrific to see the final published results. Many thalassemia treaters have already been passing on the initial results to patients, and this paper is encouraging news for patients, families, and the nurses and physicians who work with them.”
The study was authored by Stuart L. Goldberg, MD; Patricia J. Giardina, MS; Deborah Chirnomas, MD; Jason Esposito, MSHS; Carole Paley, MD; and Elliott Vichinsky, MD. The study was sponsored by Novartis Pharmaceuticals Corporation.