مورد إلكتروني
Atypical haemolytic uraemic syndrome treated with the complement inhibitor eculizumab: The experience of the Australian compassionate access cohort.
| العنوان: | Atypical haemolytic uraemic syndrome treated with the complement inhibitor eculizumab: The experience of the Australian compassionate access cohort. |
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| بيانات النشر: | Blackwell Publishing (E-mail: info@asia.blackpublishing.com.au) Australia 2015-10-07 |
| تفاصيل مُضافة: | Ford S. Burke J. Kausman J. Hewitt I. Parnham A. Isbel N. Mallett A. Hughes P. Szer J. Tuckfield A. Van Eps C. Cambell S.B. Hawley C. |
| نوع الوثيقة: | Electronic Resource |
| مستخلص: | Background/Aim: This study aimed to report the clinical characteristics and outcomes of Australian patients treated with eculizumab for atypical haemolytic uraemic syndrome (aHUS). Method(s): A retrospective cohort study was undertaken of all patients in Australia treated with eculizumab provided in a compassionate access programme for a clinical diagnosis of aHUS using prospectively collected clinical data. Result(s): A total of 10 patients with a median age of 23.5 years (interquartile range (IQR) 24.83 years) received compassionate access eculizumab for aHUS in Australia. Eight patients were female, and three had a family history of aHUS. Three received eculizumab for an initial acute aHUS presentation, three for relapsing and refractory acute aHUS, two for de novo aHUS post-renal transplantation, and one each for aHUS recurrence post-transplantation and facilitation of transplantation with a history of aHUS. The median duration of eculizumab therapy has been 911.5 days (IQR 569 days) with a cumulative exposure of 9184 days. At baseline all patients had renal and extra-renal aHUS involvement, with up to three non-renal organs affected. All but one patient, who died from uncontrollable gastrointestinal aHUS manifestations, have continued. The nine continuing patients achieved remission of aHUS. Two of the four patients requiring renal replacement therapy (RRT) at eculizumab commencement subsequently ceased RRT. Clinical events occurring in this cohort while on eculizumab treatment included neutropenia (two), posterior reversible encephalopathy syndrome (one), cardiomyopathy (one), pulmonary embolus (one), antibody-mediated rejection resulting in renal graft failure (one), iron deficiency (one), gastrointestinal haemorrhage (one) and death (one). Conclusion(s): Eculizumab has been an effective therapy for aHUS in this cohort, including when other therapies have failed.Copyright © 2015 Royal Australasian College of Physicians. |
| مصطلحات الفهرس: | genetic counseling, adult, article, Australia, Australian, cardiomyopathy/si [Side Effect], clinical article, cohort analysis, compassionate use, creatinine blood level, death, drug efficacy, drug fatality/si [Side Effect], female, gastrointestinal hemorrhage/si [Side Effect], hemolytic uremic syndrome/dt [Drug Therapy], hemolytic uremic syndrome/su [Surgery], hemolytic uremic syndrome/th [Therapy], human, infant, iron deficiency anemia/si [Side Effect], kidney function, kidney graft rejection/co [Complication], kidney transplantation, lung embolism/si [Side Effect], male, neutropenia/si [Side Effect], posterior reversible encephalopathy syndrome/si [Side Effect], priority journal, recurrent disease, remission, renal replacement therapy, retrospective study, thrombotic thrombocytopenic purpura, treatment response, complement inhibitor/dt [Drug Therapy], creatinine/ec [Endogenous Compound], eculizumab/ae [Adverse Drug Reaction], eculizumab/do [Drug Dose], eculizumab/dt [Drug Therapy], eculizumab/iv [Intravenous Drug Administration], Article |
| URL: | Internal Medicine Journal Click here for full text options LibKey Link |
| الإتاحة: | Open access content. Open access content Copyright 2016 Elsevier B.V., All rights reserved. |
| أرقام أخرى: | AUSHL oai:repository.monashhealth.org:1/40687 Internal Medicine Journal. 45 (10) (pp 1054-1065), 2015. Date of Publication: 01 Oct 2015. 1444-0903 https://repository.monashhealth.org/monashhealthjspui/handle/1/40687Test 26247170 [http://www.ncbi.nlm.nih.gov/pubmed/?term=26247170Test] 606257398 (Mallett) Department of Renal Medicine, Royal Brisbane and Women's Hospital, Brisbane, QLD, Australia (Mallett, Van Eps, Cambell, Hawley, Burke, Isbel) Centre for Kidney Disease Research, Centre for Chronic Disease, CKD.QLD, School of Medicine, University of Queensland, Brisbane, QLD, Australia (Van Eps, Cambell, Hawley, Burke, Isbel) Department of Nephrology, Princess Alexandra Hospital, Brisbane, QLD, Australia (Parnham) Department of Nephrology, Gold Coast Hospital, Gold Coast, QLD, Australia (Hughes) Department of Nephrology, Royal Melbourne Hospital, Melbourne, VIC, Australia (Szer, Tuckfield) Department of Clinical Haematology and BMT Service, Royal Melbourne Hospital, Melbourne, VIC, Australia (Kausman) Department of Nephrology, The Royal Children's Hospital Melbourne, Melbourne, VIC, Australia (Ford) Department of Nephrology, Monash Medical Centre, Melbourne, VIC, Australia (Hewitt) Department of Nephrology, Princess Margaret Hospital for Children, Perth, WA, Australia Mallett A.; andrew.mallett@health.qld.gov.au 1305139213 |
| المصدر المساهم: | MONASH HEALTH LIBRS From OAIster®, provided by the OCLC Cooperative. |
| رقم الانضمام: | edsoai.on1305139213 |
| قاعدة البيانات: | OAIster |
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