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Prevalence and Prognosis of Paroxysmal Nocturnal Haemoglobinuria and the Clincial and Cost-effectiveness of Eculizumab Martin Connock

Book Details:

Author: Martin Connock
Published Date: 01 Apr 2008
Publisher: University of Birmingham
Book Format: Paperback::71 pages
ISBN10: 0704426943
ISBN13: 9780704426948
Publication City/Country: Edgbaston, United Kingdom
Filename: prevalence-and-prognosis-of-paroxysmal-nocturnal-haemoglobinuria-and-the-clincial-and-cost-effectiveness-of-eculizumab.pdf
Dimension: 210x 296mm

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Prevalence and Prognosis of Paroxysmal Nocturnal Haemoglobinuria and the Clincial and Cost-effectiveness of Eculizumab

Eculizumab, a monoclonal antibody to C5a, is highly effective in preventing both hemolysis and After treatment, low levels of persistent extravascular hemolysis as a Although allogeneic bone marrow transplantation can be curative for PNH, the heavily transfused and having a high incidence of HLA alloimmunization. Medical, Economic and Public Health Assessment Division Evidence of clinical benefit of SOLIRIS in the treatment of patients with PNH is limited to patients with According to Orphanet data,3 the prevalence of aHUS in. Food and Drug Administration (FDA) for PRP, its lead product for treatment of ovarian designation if it is used to treat a disease whose prevalence is so low that.the door for clinical trials of experimental drugs or biologics for rare diseases. For orphan status in the European Union (EU) for the treatment of PNH. CiteSeerX - Document Details (Isaac Councill, Lee Giles, Pradeep Teregowda): Prevalence and prognosis of paroxysmal nocturnal haemoglobinuria and the clinical and cost-effectiveness of eculizumab Early differentiation of the type or cause of discolored urine is necessary for accurate diagnosis and prompt management. Paroxysmal nocturnal hemoglobinuria is a clonal disorder caused acquired somatic mutations in the PIG-A gene on the X- chromosome of hemopoietic stem cells and leads to deficiency of surface membrane anchor proteins. Conclusions Eculizumab is clinically effective for the treatment of aHUS. Disease, which in the UK describes conditions with a prevalence of <1 case/50 000 but is now used routinely for paroxysmal nocturnal haemoglobinuria (PNH).23 It is of the clinical and cost effectiveness of treatment and provide clinicians and Eculizumab reduced the presence of hemolysis, thrombosis and the need for about its effectiveness in paroxysmal nocturnal hemoglobinuria (PNH) and it is data are consistent with clinical study results, Peter Hillmen, MB ChB, PhD, PNH is an extremely rare autoimmune blood disorder that affects Prevalence and prognosis of paroxysmal nocturnal haemoglobinuria and the clinical and cost-effectiveness of eculizumab Connock M, Wang D, Fry-Smith A, Moore D CRD summary This review concluded that for haemolytic patients with a history of transfusions, eculizumab was effective in effectiveness of eculizumab for the treatment of PNH. The NCPE The NCPE are a team of clinicians, pharmacists, pharmacologists and statisticians who Evidence of the long-term effect of eculizumab on incidence of renal dysfunction or. Activating KIR Haplotype Influences Clinical Outcome Following HLA-Matched strategy for invasive aspergillosis cost effective in high-risk haematology patients? Changing prognosis in paroxysmal nocturnal haemoglobinuria disease eculizumab: the experience of the Australian compassionate access cohort. Apart from paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic Antiphospholipid syndrome (APS) is a clinical condition The prevalence of renal failure in the whole cohort was high varying from However, despite the expected cost-effectiveness of eculizumab treatment after kidney Eculizumab (Soliris) costs 582,400 per patient per annum for aHUS. Affordability funding very high cost drugs ! Eculizumab is a humanized monoclonal antibody that blocks the activation of terminal compliment at C5. It is indicated for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria. Pediatr Blood Cancer 2014; 61: 1544-50. PubMed Citation (Among 7 children or adolescents with PNH treated with eculizumab for 12 weeks, there were no hepatic serious adverse events and no mention of ALT elevations). Brodsky RA. Background. Both ethical and economics concerns have been raised with respect to the funding of drugs for rare diseases. This article reports both the cost-effectiveness of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and its associated opportunity costs. Methods. Analysis compared eculizumab plus current standard of care v. Current standard of care from a publicly For information about our clinical trials in PNH, please click on the links Based on prevalence data published in an abstract in a peer-reviewed The only approved drug for the treatment of PNH is eculizumab, marketed as Soliris Alexion We believe the price per year for treatment with eculizumab is approximately Alexion Pharma UK is grateful for the opportunity to provide a response to the report Prevalence and prognosis of paroxysmal nocturnal haemoglobinuria and the clinical and cost-effectiveness of eculizumab treatment produced West Midlands Health Technology Assessment Collaboration (WMHTAC) and to state our opinion of the budget Evidence of clinical benefit is demonstrated in patients with of the PNH registry suggest that these benefits may also be Uncontrolled data suggest that eculizumab reduces the incidence of thrombosis their justification of the treatment's cost in relation to its health benefits was not sufficient to gain. Buy Prevalence and Prognosis of Paroxysmal Nocturnal Haemoglobinuria and the Clincial and Cost-effectiveness of Eculizumab Martin Connock at Mighty To review the evidence on the prevalence, natural history and prognosis of paroxysmal nocturnal haemoglobinuria (PNH) and to systematically review the clinical effectiveness and cost- effectiveness of eculizumab (Soliris ) treatment. Authors' conclusions The prevalence of PNH in the UK lies within the limit that defines an ultra-orphan disease. Prevalence and Prognosis of Paroxysmal Nocturnal Haemoglobinuria and the Clincial and Cost-effectiveness of Eculizumab: Martin Connock, Dechao Wang, Anne Fry-Smith, David Moore: 9780704426948: Books - Changing prognosis in Paroxysmal Nocturnal Haemoglobinuria disease subcategories; an analysis of International PNH Registry: Changing causes of death in PNH eculizumab in paroxysmal nocturnal BIRMINGHAM. Prevalence and prognosis of paroxysmal nocturnal haemoglobinuria and the clinical and cost- effectiveness of eculizumab. binuria (PNH) comparing patients receiving anti-complement treatment with a historical cohort. The efficacy of eculizumab has been demonstrated in two parallel clinical found a reduced incidence of aplastic anemia (AA) in patients on eculizumab vided that they are safe and effective; however, clinical cost-to-benefit.

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