The Management of Paroxysmal Nocturnal Hemoglobinuria: Recent Advances in Diagnosis and Treatment and New Hope for Patients

Neal S. Young, Gabrielle Meyers, Hubert Schrezenmeier, Peter Hillmen, Anita Hill

    Research output: Contribution to journalArticle

    15 Scopus citations

    Abstract

    PNH is a rare clonal hematopoietic stem disorder clinically characterized by the triad of chronic complement-mediated hemolysis, thrombosis, and bone marrow failure. While median survival has improved when historical data are compared to more recent data, thrombosis, the major cause of death in PNH, is still observed in approximately 40% of patients. The symptoms associated with this disorder-including fatigue, pain, esophageal spasm, and erectile dysfunction-are often severe and disabling. While PNH may be a curiosity to the physician, it forces the majority of patients to significantly modify their lives. Transplantation represents a curative option; however, the risks associated with this option are not insignificant. Eculizumab has been shown to significantly reduce hemolysis, improve anemia, reduce transfusion requirements, and significantly improve fatigue and other QoL scores. Clearly, targeted complement inhibition by eculizumab has the promise to improve the lives of patients with PNH.

    Original languageEnglish (US)
    Pages (from-to)S1-S16
    JournalSeminars in hematology
    Volume46
    Issue numberSUPPL. 1
    DOIs
    StatePublished - Jan 2009

    ASJC Scopus subject areas

    • Hematology

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