12 Questions
When should patients with LOPD be initiated on Enzyme Replacement Therapy (ERT) according to the AANEM panel recommendations?
When they have detectable proximal muscle weakness or reduced FVC
What is the advice for patients who are asymptomatic but have signs of late-onset Pompe disease (LOPD) according to Brazilian clinicians?
Consider ERT if muscle weakness or reduced pulmonary function is observed
What is the recommended frequency for monitoring for the development of antibodies in patients undergoing ERT according to the AANEM panel recommendations?
Every 3 months for up to 2 years, then annually
According to the recommendations, when should patients with severe symptoms be reevaluated after receiving ERT?
After 1 year of treatment
What should be considered before continuing ERT in patients requiring constant invasive ventilation?
Symptom improvement during the first year of treatment
What is the primary factor determining the initiation of ERT in presymptomatic patients with objective signs according to the AANEM panel recommendations?
Proximal muscle weakness
When is it recommended to initiate enzyme replacement therapy (ERT) for presymptomatic patients without objective signs of LOPD?
When they have objective signs of proximal muscle weakness
What should be done for symptomatic patients with LOPD who experience increased limb weakness and difficulty completing ADL?
Consider ERT if they have reduced FVC
What is the recommendation for patients with severe symptoms such as wheelchair use and constant invasive ventilation?
Consider initiation of ERT on a case-by-case basis
What is the protocol for presymptomatic patients with objective signs of LOPD regarding ERT initiation?
Administer ERT immediately
At what point should presymptomatic patients without objective signs initiate ERT according to the recommendations?
At the onset of symptoms or detectable proximal muscle weakness
For which type of patient is the continuation of ERT considered on a case-by-case basis?
For patients requiring constant invasive ventilation
Study Notes
- A panel of neuromuscular specialists and a pulmonologist developed treatment recommendations for Late-Onset Pompe Disease (LOPD).
- Recommendations for initiating Enzyme Replacement Therapy (ERT) in LOPD patients are based on the onset and severity of symptoms.
- ERT initiation criteria include different scenarios for presymptomatic patients without objective signs, presymptomatic patients with objective signs, symptomatic patients, and patients with severe symptoms.
- Continuation of ERT can be considered on a case-by-case basis for patients requiring constant invasive ventilation, based on symptom stabilization or improvement.
- Regular monitoring, evaluation of ERT effectiveness annually, and testing for IgG antibodies development are recommended by the panel.
Test your knowledge on treatment recommendations for Late-Onset Pompe Disease (LOPD) developed by an AANEM panel of specialists. Learn about when to initiate enzyme replacement therapy (ERT) based on the onset and severity of symptoms.
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