Airway abnormalities and pulmonary complications in long‐term treated late‐onset Pompe disease: Diagnostic and interventional by flexible bronchoscopy

2021 ◽  
Author(s):  
Ting‐Hao Wang ◽  
Wen‐Jue Soong ◽  
Dau‐Ming Niu ◽  
Yen‐Ling Chu ◽  
M.S. Li‐Zhen Chen ◽  
...  
Keyword(s):  
Pompe Disease ◽  
Late Onset ◽  
Pulmonary Complications ◽  
Flexible Bronchoscopy ◽  
Airway Abnormalities

Long-term efficacy of alglucosidase alfa in late-onset Pompe disease

2017 ◽  
Vol 120 (1-2) ◽  
pp. S134
Author(s):  
Ans van der Ploeg ◽  
Paula Clemens ◽  
Robert J Hopkin ◽  
Katherine Kacena ◽  
Bernd-Jan Sanson ◽  
...  
Keyword(s):  
Pompe Disease ◽  
Late Onset ◽  
Term Efficacy ◽  
Alglucosidase Alfa

Long-term whole-body vibration training in two late-onset Pompe disease patients

2016 ◽  
Vol 37 (8) ◽  
pp. 1357-1360 ◽  
Author(s):  
Federica Montagnese ◽  
Simone Thiele ◽  
Stephan Wenninger ◽  
Benedikt Schoser
Keyword(s):  
Pompe Disease ◽  
Late Onset ◽  
Whole Body Vibration ◽  
Whole Body ◽  
Body Vibration ◽  
Vibration Training ◽  
Whole Body Vibration Training

Safety and efficacy of short- and long-term inspiratory muscle training in late-onset Pompe disease (LOPD): a pilot study

2018 ◽  
Vol 266 (1) ◽  
pp. 133-147 ◽  
Author(s):  
Stephan Wenninger ◽  
Eva Greckl ◽  
Haris Babačić ◽  
Kristina Stahl ◽  
Benedikt Schoser
Keyword(s):  
Pilot Study ◽  
Pompe Disease ◽  
Late Onset ◽  
Inspiratory Muscle ◽  
Inspiratory Muscle Training ◽  
Muscle Training ◽  
Safety And Efficacy ◽  
Short And Long Term

Effect of long term enzyme replacement therapy in late onset Pompe disease: a single-centre experience

2020 ◽  
Author(s):  
George K. Papadimas ◽  
Christoforos Anagnostopoulos ◽  
Sophia Xirou ◽  
Helen Michelakakis ◽  
Gerasimos Terzis ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Single Centre ◽  
Enzyme Replacement ◽  
Single Centre Experience

scholarly journals Late-Onset, Noninfectious Pulmonary Complications following Allogeneic Hematopoietic Stem Cell Transplantation: A Nationwide Cohort Study of Long-Term Survivors

Respiration ◽  
2021 ◽  
pp. 1-9
Author(s):  
Ole Henrik Myrdal ◽  
Trond Mogens Aaløkken ◽  
Phoi Phoi Diep ◽  
Ellen Ruud ◽  
Lorentz Brinch ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Pulmonary Function ◽  
Late Onset ◽  
Adult Survivors ◽  
Pulmonary Complications ◽  
Healthy Controls ◽  
Pathological Findings ◽  
Hematopoietic Stem

<b><i>Background:</i></b> Survivors of allogeneic hematopoietic stem cell transplantation (allo-HSCT) are at risk for pulmonary adverse events. Data on late-onset noninfectious pulmonary complications in long-term adult survivors of allo-HSCT are limited and incomplete. <b><i>Objectives:</i></b> This study aimed (1) to determine occurrence and degree of pulmonary sequelae in adult survivors of allo-HSCT and (2) to identify associations between pulmonary function, high-resolution CT (HRCT), and clinical characteristics. <b><i>Method:</i></b> In a nationwide, single-center cross-sectional study, 103 survivors (aged median [range] 35 [17–58] years, 53% females) were examined 17 (6–32) years after allo-HSCT and compared with healthy controls (<i>n</i> = 105). Methods included pulmonary function tests and HRCT. <b><i>Results:</i></b> Chronic graft-versus-host disease was diagnosed in 33% of survivors, including 12% with bronchiolitis obliterans syndrome (BOS). Mean lung volumes (TLC, FVC, and FEV<sub>1</sub>) and gas diffusing capacity were &#x3e;80% of predicted for the survivors as a group, but significantly lower than in healthy controls. Pathological HRCT findings were detected in 48% of the survivors (71% airways disease, 35% interstitial lung disease, and 24% apical subpleural interstitial thickening). Air trapping (%) on HRCT correlated with % predicted FEV<sub>1</sub>, <i>p</i> &#x3c; 0.001. In a multiple logistic regression model, both BOS and pathological findings on HRCT were associated with chemotherapy prior to allo-HSCT, <i>p</i> &#x3c; 0.05. <b><i>Conclusions:</i></b> Long-term allo-HSCT survivors had significantly lower pulmonary function than age- and gender-matched healthy controls and nearly half had pathological findings on HRCT. Longitudinal data will determine if pulmonary sequelae will remain stable or progress. We recommend lifelong monitoring of pulmonary function in allo-HSCT survivors. HRCT provides additional information, but is not suited for surveillance.

Airway abnormalities in very early treated infantile‐onset Pompe disease: A large‐scale survey by flexible bronchoscopy

2020 ◽  
Vol 182 (4) ◽  
pp. 721-729 ◽  
Author(s):  
Chia‐Feng Yang ◽  
Dau‐Ming Niu ◽  
Shyh‐Kuan Tai ◽  
Ting‐Hao Wang ◽  
Hsiao‐Ting Su ◽  
...  
Keyword(s):  
Pompe Disease ◽  
Large Scale ◽  
Flexible Bronchoscopy ◽  
Large Scale Survey ◽  
Airway Abnormalities

Long term follow-up of cerebrovascular abnormalities in late onset Pompe disease (LOPD)

2017 ◽  
Vol 264 (3) ◽  
pp. 589-590 ◽  
Author(s):  
Matteo Garibaldi ◽  
Sabrina Sacconi ◽  
Giovanni Antonini ◽  
Claude Desnuelle
Keyword(s):  
Pompe Disease ◽  
Late Onset ◽  
Long Term Follow Up
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