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Original Paper
Acta Haematol 2010;123:220–225
DOI: 10.1159/000313447
Improved Treatment Satisfaction and
Convenience with Deferasirox in Iron-Overloaded
Patients with -Thalassemia: Results from the
ESCALATOR Trial
Ali Taher
a
Abdullah Al Jefri
b
Mohsen Saleh Elalfy
c
Kusai Al Zir
d
Shahina Daar
e
Diana Rofail
f
Jean François Baladi
g
Dany Habr
g
Ulrike Kriemler-Krahn
h
Amal El-Beshlawy
i
a
American University of Beirut, Beirut, Lebanon;
b
King Faisal Specialist Hospital and Research Center, Riyadh,
Saudi Arabia;
c
Ain Shams University, Cairo, Egypt;
d
National Thalassemia Center, Damascus, Syrian Arab Republic;
e
Sultan Qaboos University, Muscat, Oman;
f
Mapi Values, Bollington, UK;
g
Novartis Pharmaceuticals
Corporation, East Hanover, N.J., USA;
h
Novartis Pharma AG, Basel, Switzerland;
i
Cairo University, Cairo, Egypt
EOS] vs. 30.1 8 44.2 [baseline] h/month). Patients reported
greater satisfaction and convenience, and lower impact on
daily activities, with deferasirox than with previous ICT. This
may help improve adherence to lifelong ICT in iron-over-
loaded -thalassemia patients. Copyright © 2010 S. Karger AG, Basel
Introduction
It is estimated that more than 56,000 infants born
annually are affected by thalassemia major, including
30,000 who will require lifelong regular blood transfu-
sions to survive [1]. Dependence on frequent blood
transfusions results in an increased risk of accumulating
excess iron or developing iron overload. Excess iron can-
not be excreted naturally, leading to tissue damage and
Key Words
Convenience Iron chelation therapy Iron overload
-Thalassemia Deferasirox
Abstract
Patient-reported outcomes of once-daily oral deferasirox
(Exjade ) in iron-overloaded patients with -thalassemia
not achieving successful chelation with prior deferoxamine
and/or deferiprone were investigated in a prospective,
open-label, 1-year, multicenter study in the Middle East
(ESCALATOR). The initial dose of deferasirox was 20 mg/kg/
day, with subsequent dose adjustments. At baseline and the
end of study (EOS), patients (n = 237) completed a 5-point
rating scale for treatment satisfaction and convenience, and
recorded time lost to treatment. At EOS, 90.7% of patients
were ‘satisfied’/‘very satisfied’ with their iron chelation ther-
apy (ICT) versus 23.2% at baseline. 92.8% (EOS) versus 21.5%
(baseline) of patients considered their therapy to be
‘convenient’/‘very convenient’. Time lost to therapy for daily
activities was substantially reduced (3.2 8 8.6 [mean 8 SD;
Received: December 21, 2009
Accepted after revision: January 26, 2010
Published online: April 27, 2010
Ali Taher
Department of Internal Medicine, Hematology-Oncology Division
American University of Beirut Medical Center
Beirut 1107 2020 (Lebanon)
Tel. +961 1 749 230, ext. 5811, Fax +961 1 365 612, E-Mail ataher @ aub.edu.lb
© 2010 S. Karger AG, Basel
0001–5792/10/1234–0220$26.00/0
Accessible online at:
www.karger.com/aha
This study was funded by Novartis.