Discontinuation of enzyme replacement therapy in adults with Pompe disease: evaluating the European POmpe Consortium stop criteria.

H.A. van Kooten,L. Harlaar,N.A.M.E. van der Beek,P.A. van Doorn,A.T. van der Ploeg,E. Brusse

Neuromuscular Disorders（2020）

引用 7|浏览28

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摘要

•The European POmpe Consortium stop criteria for enzyme replacement therapy.•We advise to add the (re)evaluation of continuing therapy in advanced disease.•After stopping enzyme replacement therapy, no rapid deterioration was observed.•Treatment decisions should be handled in a multidisciplinary expert team.

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关键词

Pompe disease,Enzyme replacement therapy,Adult patients,Treatment guidelines,Treatment discontinuation,Start and stop criteria

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