Evaluating the European Pompe Consortium stop criteria for discontinuing enzyme replacement therapy


  • The European Pompe Consortium (EPOC) criteria for stopping enzyme replacement therapy (ERT) in those with Pompe disease facilitates the decision-making process well.

Why this matters

    This study indicates that the EPOC guidelines are appropriate for continued decision making regarding ERT and suggests that rationale for continuing treatment in the advanced stages of the disease should be addressed in future criteria.