PMID- 23460233
OWN - NLM
STAT- MEDLINE
DCOM- 20130510
LR  - 20181202
IS  - 1096-8652 (Electronic)
IS  - 0361-8609 (Linking)
VI  - 88
IP  - 4
DP  - 2013 Apr
TI  - Deferiprone (GPO-L-ONE((R)) ) monotherapy reduces iron overload in 
      transfusion-dependent thalassemias: 1-year results from a multicenter 
      prospective, single arm, open label, dose escalating phase III pediatric study 
      (GPO-L-ONE; A001) from Thailand.
PG  - 251-60
LID - 10.1002/ajh.23386 [doi]
AB  - Accessibility to iron chelators including deferoxamine and deferasirox remains 
      obscured in many developing countries. To provide an alternative, the government 
      pharmaceutical organization of Thailand (GPO) manufactured deferiprone which has 
      similar bioequivalent to the standard product. Seventy-three pediatric patients 
      with severe beta thalassemias, age range 3.2-19 years, were recruited to a 1-year 
      multicenter prospective, single arm, open label, dose escalating Phase III study 
      of deferiprone to determine its clinical efficacy and safety. Sixty-four patients 
      (87.6%) completed the study with good compliance (>94%). Average deferiprone dose 
      was 79.1+/-4.3 mg/kg/day. Overall, mean serum ferritin (SF) levels at 1 year were 
      not significantly changed from baseline. However, 45% of patients (response 
      group) had SF reduced >15% from baseline at 1 year with a median reduction of 
      1,065 ng ml(-1) . Baseline SF was the major factor that predicts clinical 
      efficacy; patients with baseline SF>3,500 ng ml(-1) had the most significant fall 
      of SF at 1 year. A subgroup analysis by MRI-T2* confirmed that the response group 
      had higher baseline liver iron and deferiprone could significantly reduce liver 
      iron overload and normalize levels of ALT at 1 year. Although, gastrointestinal 
      irritation (20.5%) was the most common drug-related adverse events (AEs) followed 
      by transaminitis (16.4%) and neutropenia (6.8%), all patients were well 
      tolerated. There was no mortality and agranulocytosis found in this trial. 
      Monotherapy of deferiprone with appropriate dose adjustment and monitoring for 
      adverse events appeared to be an effective chelation therapy in some patients 
      with good compliance and acceptable safety profiles.
CI  - Copyright (c) 2013 Wiley Periodicals, Inc.
FAU - Viprakasit, Vip
AU  - Viprakasit V
AD  - Hematology/Oncology Division, Department of Pediatrics and Thalassemia Center, 
      Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand. 
      vip.vip@mahidol.ac.th
FAU - Nuchprayoon, Issarang
AU  - Nuchprayoon I
FAU - Chuansumrit, Ampaiwan
AU  - Chuansumrit A
FAU - Torcharus, Kitti
AU  - Torcharus K
FAU - Pongtanakul, Bunchoo
AU  - Pongtanakul B
FAU - Laothamatas, Jiraporn
AU  - Laothamatas J
FAU - Srichairatanakool, Somdet
AU  - Srichairatanakool S
FAU - Pooliam, Julaporn
AU  - Pooliam J
FAU - Supajitkasem, Siriwat
AU  - Supajitkasem S
FAU - Suriyaphol, Prapat
AU  - Suriyaphol P
FAU - Tanphaichitr, Voravarn S
AU  - Tanphaichitr VS
FAU - Tuchinda, Soodsarkorn
AU  - Tuchinda S
LA  - eng
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20130305
PL  - United States
TA  - Am J Hematol
JT  - American journal of hematology
JID - 7610369
RN  - 0 (Iron Chelating Agents)
RN  - 0 (Pyridones)
RN  - 2BTY8KH53L (Deferiprone)
RN  - 9007-73-2 (Ferritins)
RN  - E1UOL152H7 (Iron)
SB  - IM
MH  - Acute Disease
MH  - Administration, Oral
MH  - Adolescent
MH  - Child
MH  - Child, Preschool
MH  - Deferiprone
MH  - Drug Administration Schedule
MH  - Female
MH  - Ferritins/blood
MH  - Humans
MH  - Iron/metabolism
MH  - Iron Chelating Agents/*administration & dosage/adverse effects
MH  - Iron Overload/*drug therapy/etiology/metabolism
MH  - Liver/metabolism
MH  - Male
MH  - Prospective Studies
MH  - Pyridones/*administration & dosage/adverse effects
MH  - Thailand
MH  - Transfusion Reaction
MH  - Treatment Outcome
MH  - Young Adult
MH  - beta-Thalassemia/*therapy
EDAT- 2013/03/06 06:00
MHDA- 2013/05/11 06:00
CRDT- 2013/03/06 06:00
PHST- 2012/10/11 00:00 [received]
PHST- 2012/12/19 00:00 [revised]
PHST- 2012/12/24 00:00 [accepted]
PHST- 2013/03/06 06:00 [entrez]
PHST- 2013/03/06 06:00 [pubmed]
PHST- 2013/05/11 06:00 [medline]
AID - 10.1002/ajh.23386 [doi]
PST - ppublish
SO  - Am J Hematol. 2013 Apr;88(4):251-60. doi: 10.1002/ajh.23386. Epub 2013 Mar 5.