Can I read Recent Advances in the Diagnosis, Monitoring, and Management of Patients with Paroxysmal Nocturnal Hemoglobinuria on EtoBox?

Recent Advances in the Diagnosis, Monitoring, and Management of Patients with Paroxysmal Nocturnal Hemoglobinuria by Stephen J. Richards; Anita Hill; Peter Hillmen is a Medicine article available to read on EtoBox.

What is Recent Advances in the Diagnosis, Monitoring, and Management of Patients with Paroxysmal Nocturnal Hemoglobinuria about?

## Abstract Until recently, there has been no specific therapy for PNH with clinical management mainly supportive in terms of cytopenias and control of thrombotic risk. Currently, the only curative procedure for PNH is bone marrow transplantation although for the majority of patients the associated risks are too great to justify transplantation. The pioneering use of the therapeutic monoclonal antibody eculizumab, which binds to and prevents the activation of the complement protein C5, represents a significant advance in treatment for patients with PNH and is set to become the future standard therapy for hemolytic PNH. In both an initial pilot study and two phase III clinical trials, eculizumab has been shown to dramatically reduce intravascular hemolysis, hemoglobinuria, and transfusion requirements thus improving the quality of life in patients with PNH. As a clinical entity, PNH is synonymous with glycosylphosphatidylinositol (GPI) deficiency, and is an acquired clonal disorder associated with somatic mutations of the X‐linked __PIGA__ gene in hematopoietic stem cells. A recent study identified a novel autosomal recessively inherited form of GPI‐deficiency involving a mutation i

Who reads Recent Advances in the Diagnosis, Monitoring, and Management of Patients with Paroxysmal Nocturnal Hemoglobinuria?

Author

Stephen J. Richards; Anita Hill; Peter Hillmen

Publisher

John Wiley and Sons; Wiley (John Wiley & Sons); John Wiley & Sons Ltd.; Wiley (ISSN 1552-4949)

Published

2007

Language

EN

Field

Medicine (Health Sciences)