PMID- 35833019
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220716
IS  - 1663-9812 (Print)
IS  - 1663-9812 (Electronic)
IS  - 1663-9812 (Linking)
VI  - 13
DP  - 2022
TI  - A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa 
      in Chinese Patients With Infantile-Onset Pompe Disease.
PG  - 903488
LID - 10.3389/fphar.2022.903488 [doi]
LID - 903488
AB  - Background: A high prevalence of infantile-onset Pompe disease (IOPD) in the 
      Chinese population has been noted, but there are currently no reported clinical 
      trials of enzyme replacement therapy (ERT) for IOPD in this population. The 
      purpose of this study was to evaluate the efficacy and safety of alglucosidase 
      alfa in Chinese patients with IOPD. Materials and Methods: A multicentre, 
      single-arm, prospective, open-label clinical trial was performed at 4 sites in 
      China. Eligible Chinese subjects with IOPD received an infusion of alglucosidase 
      alfa at a dose of 20 mg/kg every 2 weeks for up to 52 weeks. The primary 
      endpoints of clinical efficacy were the survival rate and changes in the left 
      ventricular mass index (LVMI). The safety assessment was based on the incidence 
      of adverse events (AEs). Results: A total of 10 eligible subjects were enrolled 
      in the study. The mean age at the start of ERT was 5.36 +/- 1.56 months. Nine 
      subjects had survived after 52 weeks of treatment. One subject discontinued the 
      study and died after mechanical ventilation was withdrawn. The intent-to-treat 
      analysis demonstrated that the survival rate was 90.0% (95% confidence interval: 
      55.5-99.7%). The mean LVMI at week 52 was 70.59 +/- 39.93 g/m(2) compared to that 
      of 298.02 +/- 178.43 g/m(2) at baseline, with a difference of -227.60 +/- 
      155.99 g/m(2). All subjects had left ventricular mass (LVM) Z scores >10 at 
      baseline, and eight subjects (80%) achieved Z scores <5 at week 52. No 
      treatment-related AEs were observed, and no AEs led to the discontinuation of 
      treatment. Conclusions: This clinical trial is the first study of ERT for IOPD in 
      China, indicating that alglucosidase alfa has favourable efficacy and safety for 
      the treatment of Chinese patients with IOPD (ClinicalTrials.gov number, 
      NCT03687333).
CI  - Copyright (c) 2022 Zhu, Zhu, Qiu, Wang, Liu, Yu, Ou, Shan, Wang, Li, Chen, Liu, Li 
      and Fu.
FAU - Zhu, Diqi
AU  - Zhu D
AD  - Department of Cardiology, Shanghai Children's Medical Center, Shanghai Jiao Tong 
      University School of Medicine, Shanghai, China.
FAU - Zhu, Jiacong
AU  - Zhu J
AD  - Department of Cardiology, Shanghai Children's Medical Center, Shanghai Jiao Tong 
      University School of Medicine, Shanghai, China.
AD  - Department of Pediatrics, The Second Hospital of Jiaxing, Jiaxing, China.
FAU - Qiu, Wenjuan
AU  - Qiu W
AD  - Department of Pediatric Endocrinology and Genetic Metabolism, Xinhua Hospital, 
      Shanghai Institute of Pediatric Research, Shanghai Jiao Tong University School of 
      Medicine, Shanghai, China.
FAU - Wang, Benzhen
AU  - Wang B
AD  - Heart Center, Women and Children's Hospital, Qingdao University, Qingdao, China.
FAU - Liu, Lin
AU  - Liu L
AD  - Department of Pediatric Cardiology, Shenzhen Children's Hospital, Shenzhen, 
      China.
FAU - Yu, Xiaodan
AU  - Yu X
AD  - Department of Developmental and Behavioral Pediatrics, Shanghai Children's 
      Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 
      China.
FAU - Ou, Zhenheng
AU  - Ou Z
AD  - Department of Pediatric Cardiology, Shenzhen Children's Hospital, Shenzhen, 
      China.
FAU - Shan, Guangsong
AU  - Shan G
AD  - Heart Center, Women and Children's Hospital, Qingdao University, Qingdao, China.
FAU - Wang, Jian
AU  - Wang J
AD  - Research Division of Birth Defects, Institute of Pediatric Translational 
      Medicine, Shanghai Children's Medical Center, Shanghai Jiao Tong University 
      School of Medicine, Shanghai, China.
FAU - Li, Bin
AU  - Li B
AD  - Medical Department, Sanofi Investment Co., Ltd., Shanghai, China.
FAU - Chen, Xiaokang
AU  - Chen X
AD  - Medical Department, Sanofi Investment Co., Ltd., Shanghai, China.
FAU - Liu, Cong
AU  - Liu C
AD  - Department of Pediatric Cardiology, Shenzhen Children's Hospital, Shenzhen, 
      China.
FAU - Li, Zipu
AU  - Li Z
AD  - Heart Center, Women and Children's Hospital, Qingdao University, Qingdao, China.
FAU - Fu, Lijun
AU  - Fu L
AD  - Department of Cardiology, Shanghai Children's Medical Center, Shanghai Jiao Tong 
      University School of Medicine, Shanghai, China.
AD  - Shanghai Clinical Research Center for Rare Pediatric Disease, Shanghai, China.
LA  - eng
SI  - ClinicalTrials.gov/NCT03687333
PT  - Journal Article
DEP - 20220627
PL  - Switzerland
TA  - Front Pharmacol
JT  - Frontiers in pharmacology
JID - 101548923
PMC - PMC9271607
OTO - NOTNLM
OT  - Pompe disease
OT  - alglucosidase alfa
OT  - enzyme replacement therapy
OT  - glycogen storage disease type II
OT  - left ventricular mass index
OT  - survival rate
COIS- BL and XC were employed by the company Sanofi Investment Co., Ltd. The remaining 
      authors declare that the research was conducted in the absence of any commercial 
      or financial relationships that could be construed as a potential conflict of 
      interest.
EDAT- 2022/07/15 06:00
MHDA- 2022/07/15 06:01
PMCR- 2022/06/27
CRDT- 2022/07/14 02:33
PHST- 2022/03/25 00:00 [received]
PHST- 2022/05/05 00:00 [accepted]
PHST- 2022/07/14 02:33 [entrez]
PHST- 2022/07/15 06:00 [pubmed]
PHST- 2022/07/15 06:01 [medline]
PHST- 2022/06/27 00:00 [pmc-release]
AID - 903488 [pii]
AID - 10.3389/fphar.2022.903488 [doi]
PST - epublish
SO  - Front Pharmacol. 2022 Jun 27;13:903488. doi: 10.3389/fphar.2022.903488. 
      eCollection 2022.