PMID- 25219292
OWN - NLM
STAT- MEDLINE
DCOM- 20151028
LR  - 20150131
IS  - 1096-7206 (Electronic)
IS  - 1096-7192 (Linking)
VI  - 114
IP  - 2
DP  - 2015 Feb
TI  - Safety and efficacy of enzyme replacement therapy with idursulfase beta in 
      children aged younger than 6 years with Hunter syndrome.
PG  - 156-60
LID - S1096-7192(14)00274-1 [pii]
LID - 10.1016/j.ymgme.2014.08.009 [doi]
AB  - Idursulfase beta (Hunterase(R)) has been used for enzyme replacement therapy (ERT) 
      of patients with mucopolysaccharidosis II (MPS II, Hunter syndrome) aged 6 years 
      or older since 2012 in Korea. The objective of this study was to evaluate the 
      safety and efficacy of ERT with idursulfase beta in Hunter syndrome children 
      younger than 6 years. This study was a 52-week, single center, single arm, 
      open-label clinical trial (NCT01645189). Idursulfase beta (0.5mg/kg/week) was 
      administered intravenously for 52 weeks. The primary endpoint was safety assessed 
      by adverse events (AEs). Secondary endpoints included vital signs, physical 
      examination, ECG, laboratory tests, anti-idursulfase antibodies, and efficacy 
      represented by changes in urinary glycosaminoglycan (GAG) at week 53 from 
      baseline. In addition, growth indices and developmental milestones (Denver II 
      test) were evaluated as exploratory variables. All six patients experienced at 
      least one AE. A total of 109 AEs were reported. One patient experienced a serious 
      AE (hospitalization due to gastroenteritis) that was considered not to be 
      treatment related. One patient (16.7%) experienced infusion-related adverse drug 
      reactions (ADRs), developing urticaria six times and a cough five times. There 
      were no serious ADRs and no clinically significant changes in vital signs, 
      physical exam, laboratory parameters, or ECG. Of the six patients, four (66.7%) 
      showed anti-idursulfase antibodies and neutralizing antibodies on at least one 
      occasion during the study. At week 53, urinary GAG was significantly reduced by 
      -35.1+/-30.6mgGAG/g creatine from baseline (P=0.038). This study indicates that the 
      safety and efficacy of idursulfase beta are similar to those reported in Hunter 
      syndrome patients aged 6 years or older.
CI  - Copyright (c) 2014 Elsevier Inc. All rights reserved.
FAU - Sohn, Young Bae
AU  - Sohn YB
AD  - Department of Medical Genetics, Ajou University Hospital, Ajou University School 
      of Medicine, Suwon, Republic of Korea.
FAU - Cho, Sung Yoon
AU  - Cho SY
AD  - Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School 
      of Medicine, Seoul, Republic of Korea.
FAU - Lee, Jieun
AU  - Lee J
AD  - Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School 
      of Medicine, Seoul, Republic of Korea.
FAU - Kwun, Yonghee
AU  - Kwun Y
AD  - Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School 
      of Medicine, Seoul, Republic of Korea.
FAU - Huh, Rimm
AU  - Huh R
AD  - Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School 
      of Medicine, Seoul, Republic of Korea.
FAU - Jin, Dong-Kyu
AU  - Jin DK
AD  - Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School 
      of Medicine, Seoul, Republic of Korea. Electronic address: jindk@skku.edu.
LA  - eng
PT  - Clinical Trial
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20140830
PL  - United States
TA  - Mol Genet Metab
JT  - Molecular genetics and metabolism
JID - 9805456
RN  - 0 (Antibodies)
RN  - 0 (Antibodies, Neutralizing)
RN  - 0 (Glycosaminoglycans)
RN  - EC 3.1.6.13 (Iduronate Sulfatase)
RN  - EC 3.1.6.13 (idursulfase)
SB  - IM
MH  - Administration, Intravenous/adverse effects
MH  - Antibodies/blood
MH  - Antibodies, Neutralizing/blood
MH  - Child
MH  - Child, Preschool
MH  - *Enzyme Replacement Therapy
MH  - Glycosaminoglycans/urine
MH  - Humans
MH  - Iduronate Sulfatase/*administration & dosage/immunology/*therapeutic use
MH  - Infant
MH  - Male
MH  - Mucopolysaccharidosis II/*drug therapy
MH  - Republic of Korea
MH  - Treatment Outcome
OTO - NOTNLM
OT  - Children
OT  - Enzyme replacement therapy
OT  - Hunter syndrome
OT  - Idursulfase beta
OT  - Mucopolysaccharidosis II
EDAT- 2014/09/16 06:00
MHDA- 2015/10/29 06:00
CRDT- 2014/09/16 06:00
PHST- 2014/07/08 00:00 [received]
PHST- 2014/08/26 00:00 [revised]
PHST- 2014/08/26 00:00 [accepted]
PHST- 2014/09/16 06:00 [entrez]
PHST- 2014/09/16 06:00 [pubmed]
PHST- 2015/10/29 06:00 [medline]
AID - S1096-7192(14)00274-1 [pii]
AID - 10.1016/j.ymgme.2014.08.009 [doi]
PST - ppublish
SO  - Mol Genet Metab. 2015 Feb;114(2):156-60. doi: 10.1016/j.ymgme.2014.08.009. Epub 
      2014 Aug 30.