Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
8
pubmed:dateCreated
2010-7-26
pubmed:abstractText
Paroxysmal nocturnal hemoglobinuria (PNH) is a debilitating and life-threatening disease in which lysis of PNH red blood cells frequently manifests with chronic hemolysis, anemia, and thrombosis. Renal damage in PNH is associated with chronic hemosiderosis and/or microvascular thrombosis. We determined the incidence of renal dysfunction or damage, defined by stages of chronic kidney disease (CKD), in a large cohort of PNH patients and evaluated the safety and efficacy of the complement inhibitor eculizumab in altering its progression. Renal dysfunction or damage was observed in 65% of the study population at baseline with 21% of patients with later stage CKD or kidney failure (glomerular filtration rate [GFR] <or=60 ml/min/1.73 m(2); Stage 3, 4, or 5). Eculizumab treatment was safe and well-tolerated in patients with renal dysfunction or damage and resulted in the likelihood of improvement as defined as categorical reduction in CKD stage (P < 0.001) compared with baseline and to placebo (P = 0.04). Improvement in renal function was more commonly seen in patients with baseline CKD Stages 1-2 (67.1% improvement, P < 0.001) although improvement was also observed in patients with CKD Stages 3-4 (P = 0.05). Improvements occurred quickly and were sustained for at least 18 months of treatment. Patients categorized at CKD Stages 3-5 did not worsen during treatment with eculizumab. Overall, 40 (21%) of 195 patients who demonstrated renal dysfunction or damage at baseline were no longer classified as such after 18 months of treatment. Administration of eculizumab to patients with renal dysfunction or damage was well tolerated and was usually associated with clinical improvement.
pubmed:commentsCorrections
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Aug
pubmed:issn
1096-8652
pubmed:author
pubmed:copyrightInfo
(c) 2010 Wiley-Liss, Inc.
pubmed:issnType
Electronic
pubmed:volume
85
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
553-9
pubmed:dateRevised
2011-11-17
pubmed:meshHeading
pubmed-meshheading:20658586-Adolescent, pubmed-meshheading:20658586-Adult, pubmed-meshheading:20658586-Aged, pubmed-meshheading:20658586-Aged, 80 and over, pubmed-meshheading:20658586-Antibodies, Monoclonal, pubmed-meshheading:20658586-Antibodies, Monoclonal, Humanized, pubmed-meshheading:20658586-Complement Activation, pubmed-meshheading:20658586-Complement C5, pubmed-meshheading:20658586-Complement Inactivating Agents, pubmed-meshheading:20658586-Female, pubmed-meshheading:20658586-Glomerular Filtration Rate, pubmed-meshheading:20658586-Hemoglobinuria, Paroxysmal, pubmed-meshheading:20658586-Hemolysis, pubmed-meshheading:20658586-Humans, pubmed-meshheading:20658586-Kidney, pubmed-meshheading:20658586-Kidney Failure, Chronic, pubmed-meshheading:20658586-Male, pubmed-meshheading:20658586-Metabolic Clearance Rate, pubmed-meshheading:20658586-Middle Aged, pubmed-meshheading:20658586-Pilot Projects, pubmed-meshheading:20658586-Severity of Illness Index, pubmed-meshheading:20658586-Treatment Outcome, pubmed-meshheading:20658586-Young Adult
pubmed:year
2010
pubmed:articleTitle
Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria.
pubmed:affiliation
Department of Hematology, St. James's Institute of Oncology, Leeds, United Kingdom. peter.hillmen@nhs.net
pubmed:publicationType
Journal Article, Randomized Controlled Trial, Multicenter Study, Clinical Trial, Phase II, Clinical Trial, Phase III