Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan.

Published

Journal Article

: To determine and directly compare the clinical course of white and Asian patients with paroxysmal nocturnal hemoglobinuria (PNH), data were collected for epidemiologic analysis on 176 patients from Duke University and 209 patients from Japan. White patients were younger with significantly more classical symptoms of PNH including thrombosis, hemoglobinuria, and infection, while Asian patients were older with more marrow aplasia. The mean fraction of CD59-negative polymorphonuclear cells (PMN) at initial analysis was higher among Duke patients than Japanese patients. In both cohorts, however, a larger PNH clone was associated with classical PNH symptoms, while a smaller PNH clone was associated with marrow aplasia. Thrombosis was significantly more prevalent in white patients than Asian patients, and was associated with a significantly higher proportion of CD59-negative PMN. For individual patients, CD59-negative populations varied considerably over time, but a decreasing PNH clone portended hematopoietic failure. Survival analysis revealed a similar death rate in each group, although causes of death were different and significantly more Duke patients died from thrombosis. Japanese patients had a longer mean survival time (32.1 yr vs. 19.4 yr), although Kaplan-Meier survival curves were not significantly different. Poor survival in both groups was associated with age over 50 years, severe leukopenia/neutropenia at diagnosis, and severe infection as a complication; additionally, thrombosis at diagnosis or follow-up for Duke patients and renal failure for Japanese patients were poor prognostic factors. These data identify important differences between white and Asian patients with PNH. Identification of prognostic factors will help the design of prospective clinical trials for PNH.

Full Text

Duke Authors

Cited Authors

  • Nishimura, J-I; Kanakura, Y; Ware, RE; Shichishima, T; Nakakuma, H; Ninomiya, H; Decastro, CM; Hall, S; Kanamaru, A; Sullivan, KM; Mizoguchi, H; Omine, M; Kinoshita, T; Rosse, WF

Published Date

  • May 2004

Published In

Volume / Issue

  • 83 / 3

Start / End Page

  • 193 - 207

PubMed ID

  • 15118546

Pubmed Central ID

  • 15118546

International Standard Serial Number (ISSN)

  • 0025-7974

Digital Object Identifier (DOI)

  • 10.1097/01.md.0000126763.68170.46

Language

  • eng

Conference Location

  • United States