New insights into paroxysmal nocturnal hemoglobinuria

William J. Savage, Robert A Brodsky

Research output: Contribution to journalArticle

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is an uncommon acquired hemolytic anemia that manifests with abdominal pain, esophageal spasm, fatigue, and thrombosis. The hallmark of PNH at the cellular level is a deficiency in cell surface glycosylphosphatidylinositol anchored proteins; this deficiency on erythrocytes leads to intravascular hemolysis. Free hemoglobin from hemolysis leads to circulating nitric oxide depletion and is responsible for many of the clinical manifestations of PNH, including fatigue, erectile dysfunction, esophageal spasm, and thrombosis. The recently FDA approved complement inhibitor eculizumab has been shown to decrease hemolysis, decrease erythrocyte transfusion requirements, and improve quality of life for PNH patients.

Original languageEnglish (US)
Pages (from-to)371-376
Number of pages6
JournalHematology
Volume12
Issue number5
DOIs
StatePublished - Oct 2007

Fingerprint

Paroxysmal Hemoglobinuria
Diffuse Esophageal Spasm
Hemolysis
Complement Inactivating Agents
Fatigue
Thrombosis
Erythrocyte Transfusion
Glycosylphosphatidylinositols
Hemolytic Anemia
Erectile Dysfunction
Abdominal Pain
Nitric Oxide
Hemoglobins
Erythrocytes
Quality of Life
Proteins

Keywords

  • Eculizumab
  • Nitric oxide
  • Paroxysmal nocturnal hemoglobinuria
  • PIG-A

ASJC Scopus subject areas

  • Hematology

Cite this

New insights into paroxysmal nocturnal hemoglobinuria. / Savage, William J.; Brodsky, Robert A.

In: Hematology, Vol. 12, No. 5, 10.2007, p. 371-376.

Research output: Contribution to journalArticle

Savage, William J. ; Brodsky, Robert A. / New insights into paroxysmal nocturnal hemoglobinuria. In: Hematology. 2007 ; Vol. 12, No. 5. pp. 371-376.
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