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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
5
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pubmed:dateCreated |
2007-4-26
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pubmed:abstractText |
This study was performed to determine whether adult male patients with Fabry disease who demonstrate a continuing decline in renal function despite 2 to 4 yr of conventionally dosed agalsidase alfa therapy (0.2 mg/kg every other week [EOW]) show an improved slope of decline with weekly administration using the same dosage. Eleven (27%) of 41 adult male patients with Fabry disease who participated in long-term agalsidase alfa clinical trials and who had demonstrated a slope of decline in estimated GFR (eGFR) of > or =5 ml/min per 1.73 m(2)/yr while receiving long-term treatment with agalsidase alfa at the currently recommended dosage of 0.2 mg/kg, infused EOW, were enrolled in this open-label, prospective study. Patients were switched from EOW to weekly infusions and followed for an additional 24 mo. Before switching to weekly dosing, eGFR was 53.7 +/- 6.3 ml/min per 1.73 m(2) (mean +/- SEM), and mean rate of change in eGFR was -8.0 +/- 0.8 ml/min per 1.73 m(2)/yr. During the 24-mo follow-up period after switching to weekly dosing, the mean rate of change in eGFR was observed to slow to -3.3 +/- 1.4 ml/min/1.73 m(2)/yr (P = 0.01 versus EOW). After switching to weekly dosing, three patients demonstrated an improvement in eGFR and six patients demonstrated a slowing in the rate of eGFR decline; only two patients failed to improve their eGFR slope. A multiple regression model confirmed that the weekly infusion regimen was the strongest explanatory variable for the change in eGFR (P = 0.0008), with a weaker contribution from the concomitant use of angiotensin converting enzyme inhibitors/angiotensin receptor blockers (P = 0.02). These results suggest that weekly infusions of agalsidase alfa at a dosage of 0.2 mg/kg may be beneficial in the subgroup of patients who have Fabry disease and whose kidney function continues to decline after 2 to 4 yr or more of standard EOW dosing.
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pubmed:grant |
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pubmed:commentsCorrections |
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-10618424,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11042029,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11042031,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11105184,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11386930,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11439963,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11694547,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11732485,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11889412,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-11929328,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-12068025,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-12421894,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-12427118,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-14505049,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-14639584,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-15149345,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-15154115,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-15303007,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-15327390,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-15687833,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-15713906,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-16027312,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-16204287,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-16298202,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-16481892,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-16939062,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-17073606,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-17179052,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-17206462,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-17429046,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-6023233,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-6803189,
http://linkedlifedata.com/resource/pubmed/commentcorrection/17409308-9918480
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
IM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
May
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pubmed:issn |
1046-6673
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pubmed:author |
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pubmed:issnType |
Print
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pubmed:volume |
18
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
1576-83
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pubmed:dateRevised |
2009-11-18
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pubmed:meshHeading |
pubmed-meshheading:17409308-Adult,
pubmed-meshheading:17409308-Antibodies,
pubmed-meshheading:17409308-Dose-Response Relationship, Drug,
pubmed-meshheading:17409308-Drug Administration Schedule,
pubmed-meshheading:17409308-Fabry Disease,
pubmed-meshheading:17409308-Glomerular Filtration Rate,
pubmed-meshheading:17409308-Humans,
pubmed-meshheading:17409308-Isoenzymes,
pubmed-meshheading:17409308-Kidney,
pubmed-meshheading:17409308-Male,
pubmed-meshheading:17409308-Middle Aged,
pubmed-meshheading:17409308-Proteinuria,
pubmed-meshheading:17409308-Time,
pubmed-meshheading:17409308-alpha-Galactosidase
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pubmed:year |
2007
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pubmed:articleTitle |
Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing.
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pubmed:affiliation |
Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Building 10, Room 3D03, 9000 Rockville Pike, Bethesda, MD 20892-1260, USA. rs4e@nih.gov
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pubmed:publicationType |
Journal Article,
Clinical Trial,
Research Support, N.I.H., Intramural
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