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

13 Citations (Scopus)

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)
JournalSeminars in Hematology
Volume46
Issue numberSUPPL. 1
DOIs
StatePublished - Jan 2009
Externally publishedYes

Fingerprint

Hope
Paroxysmal Hemoglobinuria
Hemolysis
Fatigue
Thrombosis
Diffuse Esophageal Spasm
Exploratory Behavior
Erectile Dysfunction
Anemia
Cause of Death
Therapeutics
Transplantation
Bone Marrow
Physicians
Pain
Survival
eculizumab

ASJC Scopus subject areas

  • Hematology

Cite this

The Management of Paroxysmal Nocturnal Hemoglobinuria : Recent Advances in Diagnosis and Treatment and New Hope for Patients. / Young, Neal S.; Meyers, Gabrielle; Schrezenmeier, Hubert; Hillmen, Peter; Hill, Anita.

In: Seminars in Hematology, Vol. 46, No. SUPPL. 1, 01.2009.

Research output: Contribution to journalArticle

@article{6969c6c156924d5c99e7ddd2f4b9aed3,
title = "The Management of Paroxysmal Nocturnal Hemoglobinuria: Recent Advances in Diagnosis and Treatment and New Hope for Patients",
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.",
author = "Young, {Neal S.} and Gabrielle Meyers and Hubert Schrezenmeier and Peter Hillmen and Anita Hill",
year = "2009",
month = "1",
doi = "10.1053/j.seminhematol.2008.11.004",
language = "English (US)",
volume = "46",
journal = "Seminars in Hematology",
issn = "0037-1963",
publisher = "W.B. Saunders Ltd",
number = "SUPPL. 1",

}

TY - JOUR

T1 - The Management of Paroxysmal Nocturnal Hemoglobinuria

T2 - Recent Advances in Diagnosis and Treatment and New Hope for Patients

AU - Young, Neal S.

AU - Meyers, Gabrielle

AU - Schrezenmeier, Hubert

AU - Hillmen, Peter

AU - Hill, Anita

PY - 2009/1

Y1 - 2009/1

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=58549084856&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=58549084856&partnerID=8YFLogxK

U2 - 10.1053/j.seminhematol.2008.11.004

DO - 10.1053/j.seminhematol.2008.11.004

M3 - Article

VL - 46

JO - Seminars in Hematology

JF - Seminars in Hematology

SN - 0037-1963

IS - SUPPL. 1

ER -