PMID- 21072825
OWN - NLM
STAT- MEDLINE
DCOM- 20110301
LR  - 20181201
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 56
IP  - 3
DP  - 2011 Mar
TI  - Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for 
      patients with transfusion-dependent beta-thalassemia.
PG  - 420-4
LID - 10.1002/pbc.22826 [doi]
AB  - BACKGROUND: Deferasirox is an oral iron-chelating agent taken once-daily by 
      patients with transfusion-dependent iron overload. However, some patients are 
      unresponsive or unable to tolerate once-daily deferasirox. The current study 
      evaluated whether twice-daily deferasirox treatment showed increased efficacy or 
      tolerability in unresponsive or intolerant patients. PROCEDURE: Patients from two 
      Taiwanese hospitals with transfusion-dependent beta-thalassemia, including those who 
      showed increasing serum ferritin levels for six consecutive months, with at least 
      one level >2,500 ng/dl, while treated with >30 mg/kg/day of once-daily 
      deferasirox (unresponsive) or developed deferasirox-related adverse events (AEs) 
      at the dosage required to maintain the iron burden balance (intolerant) and were 
      treated twice-daily with the same total daily dose of deferasirox since 2008, 
      were enrolled in the study and evaluated retrospectively by medical record 
      review. RESULTS: Eighteen patients were included for analysis. A statistically 
      significant median decrease in serum ferritin levels was detected in the 11 
      unresponsive patients after 6 months of continuous twice-daily deferasirox 
      treatment. Five out of the seven intolerant patients experienced either no 
      deferasirox-related AEs or less severe AEs. The 12 patients from both groups (11 
      unresponsive, 1 intolerant) who received continuous twice-daily deferasirox for 6 
      months showed a mild but significant median increase in serum creatinine levels. 
      CONCLUSIONS: Twice-daily deferasirox dosing is effective in iron chelation and 
      improves tolerability in transfusion-dependent beta-thalassemia patients who are 
      unresponsive to or intolerant of once-daily deferasirox. Future studies with 
      greater patient numbers will be required to confirm the results reported herein.
CI  - (c) 2010 Wiley-Liss, Inc.
FAU - Chang, Hsiu-Hao
AU  - Chang HH
AD  - Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
FAU - Lu, Meng-Yao
AU  - Lu MY
FAU - Liao, Yu-Mei
AU  - Liao YM
FAU - Lin, Pei-Chin
AU  - Lin PC
FAU - Yang, Yung-Li
AU  - Yang YL
FAU - Lin, Dong-Tsamn
AU  - Lin DT
FAU - Chiou, Shyh-Shin
AU  - Chiou SS
FAU - Jou, Shiann-Tarng
AU  - Jou ST
FAU - Lin, Kai-Hsin
AU  - Lin KH
FAU - Chang, Tai-Tsung
AU  - Chang TT
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Benzoates)
RN  - 0 (Iron Chelating Agents)
RN  - 0 (Triazoles)
RN  - 9007-73-2 (Ferritins)
RN  - V8G4MOF2V9 (Deferasirox)
SB  - IM
CIN - Expert Rev Hematol. 2011 Aug;4(4):411-4. PMID: 21801132
MH  - Administration, Oral
MH  - Adolescent
MH  - Adult
MH  - Benzoates/*administration & dosage
MH  - Child
MH  - Deferasirox
MH  - Drug Administration Schedule
MH  - Female
MH  - Ferritins/blood
MH  - Humans
MH  - Iron Chelating Agents/*administration & dosage
MH  - Iron Overload/blood/chemically induced/*drug therapy
MH  - Male
MH  - Maximum Tolerated Dose
MH  - Retrospective Studies
MH  - *Transfusion Reaction
MH  - Treatment Outcome
MH  - Triazoles/*administration & dosage
MH  - Young Adult
MH  - beta-Thalassemia/blood/*drug therapy
EDAT- 2010/11/13 06:00
MHDA- 2011/03/02 06:00
CRDT- 2010/11/13 06:00
PHST- 2010/11/13 06:00 [entrez]
PHST- 2010/11/13 06:00 [pubmed]
PHST- 2011/03/02 06:00 [medline]
AID - 10.1002/pbc.22826 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2011 Mar;56(3):420-4. doi: 10.1002/pbc.22826.