Compliance and clinical benefit of deferasirox granule and dispersible tablet formulation in pediatric patients with transfusional iron overload: in a randomized, open-label, multicenter, phase II study
Ali T. Taher,
Yasser Wali,
Maria Cecilia Cruz,
Pimlak Charoenkwan,
Yesim Aydinok,
Olena Werner,
Sameera Govindaraju,
Fabian Romen,
Vip Viprakasit
Affiliations
Ali T. Taher
American University of Beirut Medical Center, Beirut
Yasser Wali
Sultan Qaboos University Hospital, Muscat
Maria Cecilia Cruz
Philippine Children’s Medical Center, Quezon City, Republic of the Philippines
Pimlak Charoenkwan
Faculty of Medicine, Chiang Mai University, Chiang Mai
Yesim Aydinok
Ege University, Faculty of Medicine, Izmir
Olena Werner
Novartis Pharma AG, Basel
Sameera Govindaraju
Novartis Healthcare Pvt Ltd, Hyderabad
Fabian Romen
Novartis Pharma AG, Basel
Vip Viprakasit
Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok
CALYPSO (clinicaltrials gov. Identifier: NCT02435212), a randomized, open-label, multicenter, phase II study evaluated the compliance, clinical benefits, and safety of deferasirox granules and dispersible tablets (DT) in pediatric patients with iron overload. Iron chelation therapy-naive and iron chelation therapy-pretreated patients aged 2 to 0.5 mg/mg; 24.5% and 34.2%), upper respiratory tract infection (28.2% and 29.7%), and pyrexia (26.4% and 23.4%). In iron chelation therapy-naive patients, mean compliance and change from baseline in serum ferritin with both deferasirox formulations were not significantly different. The safety profile was comparable between granule and DT formulations, and was consistent with the general safety profile of deferasirox.
