Document Detail


Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories.
MedLine Citation:
PMID:  18535202     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
The natural history of paroxysmal nocturnal hemoglobinuria (PNH) clinical subcategories (classic PNH and aplastic anemia [AA]/PNH syndrome) is still unknown. We retrospectively studied 460 PNH patients diagnosed in 58 French hematologic centers from 1950 to 2005. The median (SE) follow-up time was 6.8 (0.5) years. The median survival time (SE) was 22 (2.5) years. We identified 113 patients with classic PNH, 224 patients with AA-PNH syndrome, and 93 (22%) intermediate patients who did not fit within these 2 categories. At presentation, classic PNH patients were older, with more frequent abdominal pain and displayed higher levels of GPI-AP-deficient granulocytes. A time-dependent improved survival was observed. In classic PNH, diagnoses before 1986 (hazard ratio [HR]: 3.6; P = .01) and increasing age (P < .001) were associated with worse survival prognoses, whereas use of androgens within the first year after diagnosis was protective (HR, 0.17; P = .01). Transfusions before 1996 (HR, 2.7; P = .007) led to lower survival rates in patients with AA-PNH syndrome, whereas immunosuppressive treatment was associated with better outcomes (HR, 0.33; P = .03). Evolution to thrombosis affected survival in both subcategories (classic PNH: HR, 7.8 [P < .001]; AA-PNH syndrome: HR, 33.0 [P < .001]). Evolution to bicytopenia or pancytopenia for classic PNH (HR, 7.3, P < .001) and malignancies for AA-PNH syndrome (HR, 48.8; P < .001) were associated with worse outcomes. Although clinical presentation and prognosis factors are different, classic PNH and AA-PNH syndrome present roughly similar outcomes, affected mainly by complications.
Authors:
Régis Peffault de Latour; Jean Yves Mary; Célia Salanoubat; Louis Terriou; Gabriel Etienne; Mohamad Mohty; Sophie Roth; Sophie de Guibert; Sebastien Maury; Jean Yves Cahn; Gerard Socié; ;
Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't     Date:  2008-06-05
Journal Detail:
Title:  Blood     Volume:  112     ISSN:  1528-0020     ISO Abbreviation:  Blood     Publication Date:  2008 Oct 
Date Detail:
Created Date:  2008-10-08     Completed Date:  2008-10-16     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  7603509     Medline TA:  Blood     Country:  United States    
Other Details:
Languages:  eng     Pagination:  3099-106     Citation Subset:  AIM; IM    
Affiliation:
Service d'Hématologie-Greffe, Université Paris 7, France.
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MeSH Terms
Descriptor/Qualifier:
Adult
Female
Hematology / methods
Hemoglobinuria, Paroxysmal / classification*,  complications,  diagnosis*
Humans
Immunosuppressive Agents / therapeutic use
Male
Middle Aged
Multivariate Analysis
Prognosis
Proportional Hazards Models
Risk
Thrombosis / genetics
Time Factors
Treatment Outcome
Chemical
Reg. No./Substance:
0/Immunosuppressive Agents

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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