Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food

Following the run-in phase, patients entered a three month, assessment phase. During the assessment phase, patients were given five general options for taking Deferasirox including with or without meals, crushed and added to a soft food or mixed in a liquid of choice.

• deferasirox: (Drug), also known as Exjade, Deferasirox: Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.

Primary outcomes

• Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 [Week 8 and Week 12]: Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver.

Secondary outcomes

• Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption [Day 1 up to Week 16]: Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Subjects who had permanently terminated from the treatment or kept the treatment on hold/deviated from protocol due to adverse event were defined as subjects with permanent discontinuation and temporary interruption, respectively.
• Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 [Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16]: Blood samples were drawn at every visit as close as possible to 24 hours post dose from each subject participating in the study and trough plasma concentrations were estimated.
• Change From Baseline in Serum Ferritin at Week 16 [Baseline, Week 16 (End of study)]: Ferritin protein stores iron and provides overall iron levels. Higher ferritin in blood showed higher iron content. Fluctuations from normal serum ferritin levels (500 ng/mL) observed at two consecutive visits led to dose adjustment of deferasirox.

Sex: All, ages: 2 Years to any

Inclusion Criteria:

* Male or female patients with thalassemia major, sickle cell disease (SCD), low or intermediate 1 (INT 1) risk myelodysplastic syndrome (MDS) or other anemias and transfusional hemosiderosis.
* Patients who were on, starting, or resuming treatment with Exjade.
* Patients who were >2 years (i.e., 2 years of age or older).

Exclusion criteria:

* Serum creatinine above the upper limit of normal (ULN) for age.
* Alanine aminotransferase (ALT) >2.5 times the ULN.-High risk intermediate-2 or high risk MDS or acute leukemia.

Participant flow

Outcome results

• Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 (Primary), Number, percentage of participants: Breakfast (Deferasirox With Soft Food) 10; Breakfast (Deferasirox With Liquid) 0; Dinner (Deferasirox With Soft Food) 0; Dinner (Deferasirox With Liquid) 0; No Meal (Deferasirox With Liquid) 0; No Meal Deferasirox With Soft Food) 0
• Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption (Secondary), Count of participants, Participants: Deferasirox (Run-in Phase) 39; Breakfast (Deferasirox With Soft Food) 10; Breakfast (Deferasirox With Liquid) 6; Dinner (Deferasirox With Soft Food) 6; Dinner (Deferasirox With Liquid) 6; No Meal (Deferasirox With Liquid) 24; No Meal Deferasirox With Soft Food) 11
• Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 (Secondary), Median, micromoles per litre (μmol/L): Deferasirox 36.30 (0.0 to 145.0)
• Change From Baseline in Serum Ferritin at Week 16 (Secondary), Mean, Nanogram/mlillilitre: Deferasirox -198.1 +/- 653.11

Adverse events

Time frame: First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV), up to 16 weeks

Serious adverse events by term

Other adverse events by term

4 more terms not shown.

• Results publication: Goldberg SL, Giardina PJ, Chirnomas D, Esposito J, Paley C, Vichinsky E. The palatability and tolerability of deferasirox taken with different beverages or foods. Pediatr Blood Cancer. 2013 Sep;60(9):1507-12. doi: 10.1002/pbc.24561. Epub 2013 Apr 23. PMID 23637051

• Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals