Hypersensitivity phenomena of various kinds occur in certain patients with late-onset Pompe disease receiving venous enzyme replacement therapy (ERT). The French consortium in charge of this myopathy, which includes clinicians from the Institut de Myologie, has compiled documented cases of intolerance to ERT using data from the French Pompe disease register:
- just over 10% of the 115 patients treated in France between 2006 and 2020 experienced this type of episode, which is far from negligible,
- this intolerance does not appear immediately (the first episode occurs on average after 15 months),
- in most cases, solutions have been found (either by adjusting the administration protocol or by introducing genuine desensitisation), enabling patients to resume ERT.
