PMID- 21592110
OWN - NLM
STAT- MEDLINE
DCOM- 20110909
LR  - 20221118
IS  - 1365-2141 (Electronic)
IS  - 0007-1048 (Print)
IS  - 0007-1048 (Linking)
VI  - 154
IP  - 3
DP  - 2011 Aug
TI  - Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in 
      transfusional iron-overloaded patients with sickle cell disease.
PG  - 387-97
LID - 10.1111/j.1365-2141.2011.08720.x [doi]
AB  - To date, there is a lack of long-term safety and efficacy data for iron chelation 
      therapy in transfusion-dependent patients with sickle cell disease (SCD). To 
      evaluate the long-term safety and efficacy of deferasirox (a once-daily oral iron 
      chelator), patients with SCD completing a 1-year, Phase II, randomized, 
      deferoxamine (DFO)-controlled study entered a 4-year extension, continuing to 
      receive deferasirox, or switching from DFO to deferasirox. Average actual 
      deferasirox dose was 19.4 +/- 6.3 mg/kg per d. Of 185 patients who received at 
      least one deferasirox dose, 33.5% completed the 5-year study. The most common 
      reasons for discontinuation were withdrawal of consent (23.8%), lost to follow-up 
      (9.2%) and adverse events (AEs) (7.6%). Investigator-assessed drug-related AEs 
      were predominantly gastrointestinal [including nausea (14.6%), diarrhoea 
      (10.8%)], mild-to-moderate and transient in nature. Creatinine clearance remained 
      within the normal range throughout the study. Despite conservative initial 
      dosing, serum ferritin levels in patients with >/= 4 years deferasirox exposure 
      significantly decreased by -591 mug/l (95% confidence intervals, -1411, -280 mug/l; 
      P = 0.027; n = 67). Long-term deferasirox treatment for up to 5 years had a 
      clinically acceptable safety profile, including maintenance of normal renal 
      function, in patients with SCD. Iron burden was substantially reduced with 
      appropriate dosing in patients treated for at least 4 years.
CI  - (c) 2011 Blackwell Publishing Ltd.
FAU - Vichinsky, Elliott
AU  - Vichinsky E
AD  - Children's Hospital and Research Center at Oakland, Oakland, CA 94609, USA. 
      evichinsky@mail.cho.org
FAU - Bernaudin, Francoise
AU  - Bernaudin F
FAU - Forni, Gian Luca
AU  - Forni GL
FAU - Gardner, Renee
AU  - Gardner R
FAU - Hassell, Kathryn
AU  - Hassell K
FAU - Heeney, Matthew M
AU  - Heeney MM
FAU - Inusa, Baba
AU  - Inusa B
FAU - Kutlar, Abdullah
AU  - Kutlar A
FAU - Lane, Peter
AU  - Lane P
FAU - Mathias, Liesl
AU  - Mathias L
FAU - Porter, John
AU  - Porter J
FAU - Tebbi, Cameron
AU  - Tebbi C
FAU - Wilson, Felicia
AU  - Wilson F
FAU - Griffel, Louis
AU  - Griffel L
FAU - Deng, Wei
AU  - Deng W
FAU - Giannone, Vanessa
AU  - Giannone V
FAU - Coates, Thomas
AU  - Coates T
LA  - eng
PT  - Clinical Trial, Phase II
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20110519
PL  - England
TA  - Br J Haematol
JT  - British journal of haematology
JID - 0372544
RN  - 0 (Benzoates)
RN  - 0 (Iron Chelating Agents)
RN  - 0 (Triazoles)
RN  - V8G4MOF2V9 (Deferasirox)
SB  - IM
CIN - Br J Haematol. 2012 May;157(4):505-6; author reply 506-7. PMID: 22299817
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Anemia, Sickle Cell/*therapy
MH  - Benzoates/administration & dosage/adverse effects/*therapeutic use
MH  - Child
MH  - Child, Preschool
MH  - Deferasirox
MH  - Drug Administration Schedule
MH  - Female
MH  - Gastrointestinal Diseases/chemically induced
MH  - Humans
MH  - Iron Chelating Agents/administration & dosage/adverse effects/*therapeutic use
MH  - Iron Overload/*drug therapy/etiology
MH  - Male
MH  - Middle Aged
MH  - *Transfusion Reaction
MH  - Treatment Outcome
MH  - Triazoles/administration & dosage/adverse effects/*therapeutic use
MH  - Young Adult
PMC - PMC3170481
EDAT- 2011/05/20 06:00
MHDA- 2011/09/10 06:00
CRDT- 2011/05/20 06:00
PHST- 2011/05/20 06:00 [entrez]
PHST- 2011/05/20 06:00 [pubmed]
PHST- 2011/09/10 06:00 [medline]
AID - 10.1111/j.1365-2141.2011.08720.x [doi]
PST - ppublish
SO  - Br J Haematol. 2011 Aug;154(3):387-97. doi: 10.1111/j.1365-2141.2011.08720.x. 
      Epub 2011 May 19.