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. 2022 Apr;29(4):1181-1186.
doi: 10.1111/ene.15222. Epub 2022 Jan 8.

Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID-19 pandemic

Céline Tard  1   2 , Emmanuelle Salort-Campana  3   4 , Maud Michaud  2   5 , Marco Spinazzi  2   6 , Aleksandra Nadaj Pakleza  2   7 , Hélène Durr  2   7 , Françoise Bouhour  3   8 , Claire Lefeuvre  2   9 , Romain Thomas  1   2 , Azzeddine Arrassi  2   9 , Nadjib Taouagh  2   9 ; Pompe Study Group; Guilhem Solé  10 , Pascal Laforêt  2   9
Collaborators, Affiliations

Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID-19 pandemic

Céline Tard et al. Eur J Neurol. 2022 Apr.

Abstract

Background and purpose: Data on interruption of enzyme replacement therapy (ERT) are scarce in late onset Pompe disease. Due to the COVID-19 crisis, eight neuromuscular reference centers in France were obligated to stop the treatment for 31 patients.

Methods: We collected the motor and respiratory data from our French registry, before COVID-19 and at treatment restart.

Results: In 2.2 months (mean), patients showed a significant deterioration of 37 m (mean) in the 6-min walk test and a loss of 210 ml (mean) of forced vital capacity, without ad integrum restoration after 3 months of ERT restart.

Conclusions: This national study based on data from the French Pompe Registry shows that the interruption of ERT, even as short as a few months, worsens Pompe patients' motor and respiratory function.

Keywords: alglucosidase alfa; enzyme therapy; stopping; suspension; treatment management.

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References

REFERENCES

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