Abstract | PURPOSE: METHODS: The long-term clinical efficacy of ERT was assessed in eight late-onset GSDII patients using home mechanical ventilation (HMV) by comparing their outcomes with those of six historical control patients (GSDII patients) who had received HMV alone. The number of hospitalizations due to pulmonary exacerbations and of hours of daily use of HMV were considered the study's primary efficacy endpoints. RESULTS: The treatment group showed an increased tendency toward shorter follow-up compared to the control group (35.8 ± 29.2 vs. 52.6 ± 8.55 months; p = 0.04). At the end of the study period, the daily use of HMV (12.5 ± 7.6 vs. 19 ± 14.3 h; p = 0.004) and the hospitalization rate [incidence rate ratio = 0.43 (95 % confidence interval 0.18-0.93); p = 0.03] were significantly lower in the patients receiving ERT. The differences in the forced vital capacity absolute value and percentage change from baseline were not significantly different in the two groups. CONCLUSIONS: ERT reduces ventilator dependency in late-onset GSDII patients and the need for hospitalization due to respiratory exacerbations.
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Authors | Andrea Vianello, Claudio Semplicini, Luciana Paladini, Alessandra Concas, Sabrina Ravaglia, Serenella Servidei, Antonio Toscano, Tiziana Mongini, Corrado Angelini, Elena Pegoraro |
Journal | Lung
(Lung)
Vol. 191
Issue 5
Pg. 537-44
(Oct 2013)
ISSN: 1432-1750 [Electronic] United States |
PMID | 23839583
(Publication Type: Controlled Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
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Chemical References |
- GAA protein, human
- alpha-Glucosidases
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Topics |
- Adolescent
- Adult
- Age of Onset
- Aged
- Ambulatory Care
- Comorbidity
- Enzyme Replacement Therapy
- Female
- Follow-Up Studies
- Glycogen Storage Disease Type II
(drug therapy, epidemiology)
- Hospitalization
(statistics & numerical data)
- Humans
- Incidence
- Lung Diseases
(epidemiology, physiopathology, therapy)
- Male
- Middle Aged
- Respiration, Artificial
- Respiratory Mechanics
(drug effects, physiology)
- Treatment Outcome
- alpha-Glucosidases
(pharmacology, therapeutic use)
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