Podcast
Questions and Answers
When should patients with LOPD be initiated on Enzyme Replacement Therapy (ERT) according to the AANEM panel recommendations?
When should patients with LOPD be initiated on Enzyme Replacement Therapy (ERT) according to the AANEM panel recommendations?
- Only if they are symptomatic
- When they experience limb weakness and difficulty completing ADL
- When they have detectable proximal muscle weakness or reduced FVC (correct)
- When they use a wheelchair and invasive ventilation
What is the advice for patients who are asymptomatic but have signs of late-onset Pompe disease (LOPD) according to Brazilian clinicians?
What is the advice for patients who are asymptomatic but have signs of late-onset Pompe disease (LOPD) according to Brazilian clinicians?
- Monitor symptoms and start ERT at the onset of any symptoms
- Administer ERT for 1 year immediately
- Initiate ERT if there is difficulty with activities of daily living (ADL)
- Consider ERT if muscle weakness or reduced pulmonary function is observed (correct)
What is the recommended frequency for monitoring for the development of antibodies in patients undergoing ERT according to the AANEM panel recommendations?
What is the recommended frequency for monitoring for the development of antibodies in patients undergoing ERT according to the AANEM panel recommendations?
- Every month
- Every 6 months
- Every 3 months for up to 2 years, then annually (correct)
- Only if symptoms worsen
According to the recommendations, when should patients with severe symptoms be reevaluated after receiving ERT?
According to the recommendations, when should patients with severe symptoms be reevaluated after receiving ERT?
What should be considered before continuing ERT in patients requiring constant invasive ventilation?
What should be considered before continuing ERT in patients requiring constant invasive ventilation?
What is the primary factor determining the initiation of ERT in presymptomatic patients with objective signs according to the AANEM panel recommendations?
What is the primary factor determining the initiation of ERT in presymptomatic patients with objective signs according to the AANEM panel recommendations?
When is it recommended to initiate enzyme replacement therapy (ERT) for presymptomatic patients without objective signs of LOPD?
When is it recommended to initiate enzyme replacement therapy (ERT) for presymptomatic patients without objective signs of LOPD?
What should be done for symptomatic patients with LOPD who experience increased limb weakness and difficulty completing ADL?
What should be done for symptomatic patients with LOPD who experience increased limb weakness and difficulty completing ADL?
What is the recommendation for patients with severe symptoms such as wheelchair use and constant invasive ventilation?
What is the recommendation for patients with severe symptoms such as wheelchair use and constant invasive ventilation?
What is the protocol for presymptomatic patients with objective signs of LOPD regarding ERT initiation?
What is the protocol for presymptomatic patients with objective signs of LOPD regarding ERT initiation?
At what point should presymptomatic patients without objective signs initiate ERT according to the recommendations?
At what point should presymptomatic patients without objective signs initiate ERT according to the recommendations?
For which type of patient is the continuation of ERT considered on a case-by-case basis?
For which type of patient is the continuation of ERT considered on a case-by-case basis?
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.
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Description
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.
