scholarly journals Clinical-economic evaluation of a screening for Pompe disease in children in the local conditions

2021 ◽  
pp. 27-37
Author(s):  
A. S. Kolbin ◽  
I. A. Vilum ◽  
Yu. E. Balikina ◽  
M. A. Proskurin ◽  
O. I. Karpov
Keyword(s):  
Pompe Disease ◽  
Late Onset ◽  
Risk Group ◽  
Cost Effective ◽  
Muscular Diseases ◽  
Local Conditions ◽  
Economic Reason ◽  
Total Cost ◽  
Diagnostic Measures ◽  
Alglucosidase Alfa

Pompe disease with late onset (PDLO) can’t be diagnosed in time due to common symptoms with several neuro-muscular diseases. Screening and diagnostic measures could lead to efficacy enzyme replaced therapy (ERT) with alglucosidase alfa with aim of severe complications prediction. Screening has a nominal cost, so evaluation of it’s clinical-economic reason to use is important, especially in the local conditions. Materials and methods. Dynamic of expenditures for PDLO in case of screening in kids from risks groups has been performed, including treatment with alglucosidase on time in 12-months horizon — cost of illness. Direct and non-direct costs were calculated in case of diagnostic on time and for non-diagnosed patients, cost-effective ratios were calculated and compared in both cases. Results. The analysis showed a 10 % reduction in the total cost per year with 50 % coverage of children at risk group and 18 % (837 mln RUR) — in case of 100 %-coverage with screening on PDLO. These changes in costs are associated with a significant reduction in the burden on outpatient and inpatient care units. Cost-effective ratio in case of early treatment with alglucosidase alfa was less than in non-diagnosed group on 18,1 %. ERT had main cost in diagnosed group, and costs of complications and disability were main in nondiagnosed group. Conclusion: Health Care system expenditures are expecting less in case of screening on PDLO in risks groups.

Long-term efficacy of alglucosidase alfa in late-onset Pompe disease

2017 ◽  
Vol 120 (1-2) ◽  
pp. S134
Author(s):  
Ans van der Ploeg ◽  
Paula Clemens ◽  
Robert J Hopkin ◽  
Katherine Kacena ◽  
Bernd-Jan Sanson ◽  
...  
Keyword(s):  
Pompe Disease ◽  
Late Onset ◽  
Term Efficacy ◽  
Alglucosidase Alfa

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

2021 ◽  
Vol 20 (12) ◽  
pp. 1027-1037 ◽  
Author(s):  
Benedikt Schoser ◽  
Mark Roberts ◽  
Barry J Byrne ◽  
Sheela Sitaraman ◽  
Hai Jiang ◽  
...  
Keyword(s):  
Pompe Disease ◽  
Late Onset ◽  
Phase 3 ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Parallel Group ◽  
Alglucosidase Alfa

scholarly journals Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start

2020 ◽  
Vol 267 (10) ◽  
pp. 3038-3053
Author(s):  
David W. Stockton ◽  
Priya Kishnani ◽  
Ans van der Ploeg ◽  
Juan Llerena ◽  
Matthias Boentert ◽  
...  
Keyword(s):  
Prognostic Factors ◽  
Enzyme Replacement Therapy ◽  
Symptom Onset ◽  
Pompe Disease ◽  
Respiratory Function ◽  
Late Onset ◽  
Longitudinal Course ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa

Abstract Objective To examine respiratory muscle function among late-onset Pompe disease (LOPD) patients in the Pompe Registry (NCT00231400/Sanofi Genzyme) during enzyme replacement therapy (ERT) with alglucosidase alfa by assessing the longitudinal course of forced vital capacity (FVC), prognostic factors for FVC, and impact of time from diagnosis to ERT initiation. Methods Longitudinal FVC data from LOPD (symptom onset > 12 months or ≤ 12 months without cardiomyopathy) patients were analyzed. Patients had to have baseline FVC (percent predicted upright) assessments at ERT start and ≥ 2 valid post-baseline assessments. Longitudinal analyses used linear mixed-regression models. Results Among 396 eligible patients, median baseline FVC was 66.9% (range 9.3–126.0). FVC remained stable during the 5-year follow-up (slope = − 0.17%, p = 0.21). Baseline FVC was lower among various subgroups, including patients who were male; older at ERT initiation; had a longer duration from symptom onset to ERT initiation; and had more advanced disease at baseline (based on respiratory support use, inability to ambulate, ambulation device use). Age at symptom onset was not associated with baseline degree of respiratory dysfunction. Differences between subgroups observed at baseline remained during follow-up. Shorter time from diagnosis to ERT initiation was associated with higher FVC after 5 years in all patients and the above subgroups using a cut-off of 1.7 years. Conclusion FVC stability over 5 years suggests that respiratory function is preserved during long-term ERT in real-world settings. Early initiation of alglucosidase alfa was associated with preservation of FVC in LOPD patients with better respiratory function at the time of treatment initiation.

Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

2016 ◽  
Vol 117 (2) ◽  
pp. S117-S118
Author(s):  
Shafeeq Ladha ◽  
Pascal Laforet ◽  
Eugen Mengel ◽  
Alan Pestronk ◽  
Jean Pouget ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Phase 1 ◽  
The Novel ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa ◽  
Treatment Naïve

Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

2016 ◽  
Vol 117 (2) ◽  
pp. S92
Author(s):  
Loren Pena ◽  
Richard Baron ◽  
Barry Byrne ◽  
Claude Desnuelle ◽  
Ozlem Goker-Alpan ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Phase 1 ◽  
The Novel ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa ◽  
Treatment Naïve

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

2017 ◽  
Vol 122 (1-2) ◽  
pp. 80-85 ◽  
Author(s):  
Constantinos Papadopoulos ◽  
David Orlikowski ◽  
Hélène Prigent ◽  
Arnaud Lacour ◽  
Céline Tard ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

2016 ◽  
Vol 26 ◽  
pp. S109-S110 ◽  
Author(s):  
C. Papadopoulos ◽  
D. Orlikowski ◽  
H. Prigent ◽  
B. Perniconi ◽  
N. Taouagh ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

2021 ◽  
Vol 20 (12) ◽  
pp. 1012-1026 ◽  
Author(s):  
Jordi Diaz-Manera ◽  
Priya S Kishnani ◽  
Hani Kushlaf ◽  
Shafeeq Ladha ◽  
Tahseen Mozaffar ◽  
...  
Keyword(s):  
Pompe Disease ◽  
Multicentre Trial ◽  
Late Onset ◽  
Phase 3 ◽  
Safety And Efficacy ◽  
Alglucosidase Alfa
Sign in / Sign up

Export Citation Format

Share Document