Journal Title
Title of Journal: Lung
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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
Publish Date: 2013/07/10
Volume: 191, Issue: 5, Pages: 537-544
Abstract
Type II glycogenosis GSDII is a rare and often fatal neuromuscular disorder caused by acid alphaglucosidase deficiency Although alglucosidase alfa enzyme replacement therapy ERT significantly improves outcomes in subjects with the infantile form its efficacy in patients with the lateonset one is not entirely clear The longterm efficacy of ERT in lateonset GSGII complicated by severe pulmonary impairment causing high mechanical ventilation dependency was investigated in this studyThe longterm clinical efficacy of ERT was assessed in eight lateonset 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 endpointsThe treatment group showed an increased tendency toward shorter followup compared to the control group 358 ± 292 vs 526 ± 855 months p = 004 At the end of the study period the daily use of HMV 125 ± 76 vs 19 ± 143 h p = 0004 and the hospitalization rate incidence rate ratio = 043 95 confidence interval 018–093 p = 003 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
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