https://scholars.lib.ntu.edu.tw/handle/123456789/195624
DC Field | Value | Language |
---|---|---|
dc.contributor | 小兒科 | en |
dc.contributor.author | HWU, WUH-LIANG | en |
dc.creator | 胡務亮 | zh-tw |
dc.creator | HWU, WUH-LIANG | en |
dc.date | 2008 | en |
dc.date.accessioned | 2010-06-29T02:55:18Z | - |
dc.date.accessioned | 2018-07-11T18:02:49Z | - |
dc.date.available | 2010-06-29T02:55:18Z | - |
dc.date.available | 2018-07-11T18:02:49Z | - |
dc.date.issued | 2008 | - |
dc.identifier.uri | http://ntur.lib.ntu.edu.tw//handle/246246/186804 | - |
dc.description.abstract | Background: Pompe’s disease is caused by a deficiency of acid alpha-glucosidase (GAA). Severe GAA deficiency manifests during infancy with rapidly progressing muscle weakness, and cardiomyopathy that typically results in death by 1 year. Aim and methods: Two open-label studies were conducted in patients 6 months (S1, n=18) or >6–36 months (S2, n=21) of age with rapidly progressing disease. S1 patients received alglucosidase alpha at 20 or 40 mg/kg qow; S2 patients started at 20 mg/kg qow. Results: Mean age at treatment and median duration of treatment were: 5.1 months and 121 weeks (S1), 15.7 months and 120 weeks (S2), respectively. Cox regression analyses comparing study patients to historical controls; (S1 n=61; S2 n=84) indicated that in patients treated at 6 months, treatment reduced risk of death by 95%, death or invasive ventilation by 91%, and death or any ventilation by 87% (all p<0.0001). In patients >6–36 months, treatment reduced risk of death by 79% (p=0.0009) and death or invasive ventilation by 58% (p=0.02). Sustained decrease in LVM occurred in 94% (S1) and 81% (S2) of patients. Normal growth occurred in >80% of patients, clinically significant motor gains in 61%. Infusion-associated reactions occurred in 56%; IgG antibodies developed in 92%. Low IgG titres or trending towards decreasing titres occurred with continued treatment in 74% of those seroconverting. Patients with two null GAA mutations and high, sustained IgG titres more often had poor long-term clinical outcomes. Conclusion: These findings demonstrate the clinical benefit of alglucosidase alfa in this population and emphasize need for early treatment. | en |
dc.language | en-us | en |
dc.language.iso | en_US | - |
dc.relation | EUROPEAN JOURNAL OF NEUROLOGY v.15 SUPPL. 3 pp.176-176 | en |
dc.relation.ispartof | EUROPEAN JOURNAL OF NEUROLOGY | - |
dc.title | Alglucosidase Alpha in Infants and Children with Pompe's Disease | en |
dc.type | journal article | en |
dc.relation.pages | 176-176 | - |
dc.relation.journalvolume | v.15 | - |
dc.relation.journalissue | 3 | - |
item.openairecristype | http://purl.org/coar/resource_type/c_6501 | - |
item.openairetype | journal article | - |
item.languageiso639-1 | en_US | - |
item.grantfulltext | none | - |
item.cerifentitytype | Publications | - |
item.fulltext | no fulltext | - |
Appears in Collections: | 醫學系 |
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