作者
Marc Nicolino, Barry Byrne, J Edmund Wraith, Nancy Leslie, Hanna Mandel, David R Freyer, Georgianne L Arnold, Eniko K Pivnick, CJ Ottinger, Peter H Robinson, John-Charles A Loo, Martin Smitka, Philip Jardine, Luciano Tatò, Brigitte Chabrol, Shawn McCandless, Shigemi Kimura, L Mehta, Deeksha Bali, Alison Skrinar, Claire Morgan, Lakshmi Rangachari, Deya Corzo, Priya S Kishnani
发表日期
2009/3
期刊
Genetics in medicine
卷号
11
期号
3
页码范围
210-219
出版商
Nature Publishing Group
简介
Purpose: A clinical trial was conducted to evaluate the safety and efficacy of alglucosidase alfa in infants and children with advanced Pompe disease.
Methods: Open-label, multicenter study of IV alglucosidase alfa treatment in 21 infants 3–43 months old (median 13 months) with minimal acid α-glucosidase activity and abnormal left ventricular mass index by echocardiography. Patients received IV alglucosidase alfa every 2 weeks for up to 168 weeks (median 120 weeks). Survival results were compared with an untreated reference cohort.
Results: At study end, 71%(15/21) of patients were alive and 44%(7/16) of invasive-ventilator free patients remained so. Compared with the untreated reference cohort, alglucosidase alfa reduced the risk of death by 79%(P< 0.001) and the risk of invasive ventilation by 58%(P= 0.02). Left ventricular mass index improved or remained normal in all patients evaluated beyond 12 …
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M Nicolino, B Byrne, JE Wraith, N Leslie, H Mandel… - Genetics in medicine, 2009