NYMC Faculty Publications

Efficacy, Safety Profile, and Immunogenicity of Alglucosidase Alfa Produced at the 4,000-Liter Scale in US Children and Adolescents with Pompe Disease: ADVANCE, A Phase IV, Open-Label, Prospective Study

DOI

10.1038/gim.2018.2

Journal Title

Genetics in Medicine

First Page

1284

Last Page

1294

Document Type

Article

Publication Date

January 2018

Department

Pediatrics

Disciplines

Medicine and Health Sciences

Abstract

Purpose: Pompe disease results from lysosomal acid alpha-glucosidase (GAA) deficiency and its associated glycogen accumulation and muscle damage. Alglucosidase alfa (recombinant human GAA (rhGAA)) received approval in 2006 as a treatment for Pompe disease at the 160 L production scale. In 2010, larger-scale rhGAA was approved for patients up to 8 years old without cardiomyopathy. NCT01526785 evaluated 4,000 L rhGAA efficacy/safety in US infantile- or late-onset Pompe disease (IOPD, LOPD) patients up to 1 year old transitioned from 160 L rhGAA.MethodsA total of 113 patients (87 with IOPD; 26 with LOPD) received 4,000 L rhGAA for 52 weeks dosed the same as previous 160 L rhGAA. Efficacy was calculated as the percentage of patients stable/improved at week 52 (without death, new requirement for invasive ventilation, left ventricular mass z-score increase >1 if baseline was >2, upright forced vital capacity decrease >/=15% predicted, or Gross Motor Function Measure-88 decrease >/=8 percentage points). Safety evaluation included an extension

Comments

Please see the work itself for the complete list of authors.

Link to Full Text

Plum Print visual indicator of research metrics
PlumX Metrics
  • Citations
    • Citation Indexes: 22
    • Patent Family Citations: 1
  • Usage
    • Abstract Views: 19
  • Captures
    • Readers: 66
  • Mentions
    • Blog Mentions: 1
see details

Share

COinS