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• W3207225008 abstract "This study evaluates the whole airway abnormalities of long-term treated late-onset Pompe disease (LOPD) patients, with interventions using the flexible bronchoscope (FB). As a retrospective study, we follow up with our five LOPD patients treated with Myozyme from 2012 to 2021 regularly, but with a focus on the whole airway abnormalities of these patients visualized through FB. The long-term clinical outcomes and relevant airway symptoms were assessed. Pulmonary function test and polysomnography were performed to evaluate the degree of respiratory compromise. All patients in the study had varying degrees of airway collapsibility, pulmonary complications, sleep apnea syndrome, and facial anomalies. Pulmonary function could preserve after Myozyme treatment, but potential deterioration thereafter. This is the first study that focuses on airway abnormalities and pulmonary complications in long-term treated LOPD patients using FB. Despite years of Myozyme treatment, we still observed airway abnormalities in these patients. In our series, the pulmonary complications seem more obvious than those observed in patients with infantile-onset Pompe disease, which might be related to the late diagnosis and treatment. We might recommend that FB could provide dynamic evaluation and interventions of airway abnormalities simultaneously. Early diagnosis of respiratory dysfunction is a critical prognostic factor of the long-term outcome of treated LOPD patients." @default.
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• W3207225008 date "2021-10-19" @default.
• W3207225008 modified "2023-10-16" @default.
• W3207225008 title "Airway abnormalities and pulmonary complications in long‐term treated late‐onset Pompe disease: Diagnostic and interventional by flexible bronchoscopy" @default.
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• W3207225008 doi "https://doi.org/10.1002/ppul.25725" @default.
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