PRE ASSESSMENT REBROADCAST - Optimizing Outcomes in Late-Onset Pompe Disease: Integrating New Therapies, Whole-person Markers of Disease Monitoring, and Shared Decision-making into Practice

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AKH and Catalyst Medical Education are dedicated to developing educational initiatives that meet the needs of busy healthcare providers. As such, we are seeking your opinion and input to help us create the most effective education that will improve care for patients.

This brief survey should only take a few minutes to complete. All information provided will be de-identified and reported in aggregate. We truly appreciate your valuable feedback.

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Which of the following best describes your primary profession?

Physician

Physician assistant

Nurse/nurse practitioner

Genetic counselor

Laboratory director/technician

Other:

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Which of the following best describes your primary specialty?

Cardiology

Clinical genetics

Genetic counseling

Hepatology

Inherited metabolic disease/lysosomal storage disorder specialist

Neurology

Neuromuscular specialist

Orthopedics

Pediatrics

Physical medicine and rehabilitation specialist

Primary care

Pulmonology

Other:

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Where do you practice?

United States

Asia

Canada

European Union

Middle East

United Kingdom

Other:

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Approximately how many patients with Pompe disease (PD) do you see each year?

None

1-5

6-10

More than 10

Unsure

NA, I am not currently practicing

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How confident are you in your ability to use strategies, such as patient education materials or patient quality of life assessments, to support shared decision-making when developing treatment plans for PD?

Not confident

Somewhat confident

Moderately confident

Very confident

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How often do you use at least 2 or more measures of disease severity (eg, 6-minute walk distance [6MWD], quick motor function test, forced vital capacity [FVC]) and patient-reported outcomes (eg, Pompe Disease Symptom Scale) to monitor PD progression and inform treatment decision-making?

Rarely

Sometimes

Often

Always

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Which of the following is emerging as a biomarker to assess physiologic changes and inform treatment decision-making in patients with late-onset PD (LOPD)?

Cardiac muscle heat measurements

Joint imaging using magnetic resonance imaging

Culturing muscle cells obtained from biopsy

Skeletal muscle ultrasound

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Which of the following results was observed in the PROPEL study after treatment of PD with cipaglucosidase alfa/miglustat for 104 weeks?

Enzyme replacement therapy (ERT)-experienced patients who were treated with cipaglucosidase alfa/miglustat throughout the study period experienced improvements in 6MWD and FVC.

ERT-naive patients who were treated with cipaglucosidase alfa/miglustat throughout the study period experienced greater improvements in 6MWD and FVC than patients who were switched from alglucosidase alfa/placebo.

ERT-experienced patients who were treated with cipaglucosidase alfa/miglustat throughout the study period experienced an improvement in 6MWD and a slight decline in FVC.

ERT-naive patients who were treated cipaglucosidase alfa/miglustat throughout the study period experienced a slight decline in 6MWD and an improvement in FVC.

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Which of the following results is TRUE regarding the safety of cipaglucosidase alfa/miglustat in comparison to alglucosidase alfa at the end of the 52-week PROPEL study?

Cipaglucosidase alfa/miglustat had a better safety profile than alglucosidase alfa/placebo.

Alglucosidase alfa/placebo had a better safety profile than cipaglucosidase alfa/miglustat.

Cipaglucosidase alfa/miglustat and alglucosidase alfa/placebo had similar safety profiles.

Additional information was needed to compare safety profiles.

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A 53-year-old woman with a history of LOPD treated with alglucosidase alfa presents for a routine evaluation. After 12 years of treatment, her 6MWD has declined from 320 m to 100 m with the use of a walker and FVC results show a decline from 70% to 46% predicted compared with previous testing. She also complains that she has less energy and no longer goes out to visit her friends. Based on this presentation, what is the next BEST step for the management of this patient?

Continue first-generation ERT

Continue first-generation ERT and provide a wheelchair

Perform a muscle biopsy and refer for sleep apnea testing

Consider a switch to second-generation ERT

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