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Blood, 15 October 2008, Vol. 112, No. 8, pp. 3099-3106. Prepublished online as a Blood First Edition Paper on June 5, 2008; DOI 10.1182/blood-2008-01-133918. This Article Full Text (PDF) Supplemental Appendices and Figure All Versions of this Article: blood-2008-01-133918v1 112/8/3099    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Peffault de Latour, R. Articles by Socie, G. Search for Related Content PubMed PubMed Citation Articles by Peffault de Latour, R. Articles by Socie, G. Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted January 16, 2008 Accepted May 14, 2008 Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories R. Peffault de Latour, J. Y. Mary, C. Salanoubat, L. Terriou, G. Etienne, M. Mohty, S. Roth, S. de Guibert, S. Maury, J. Y. Cahn, and G. Socie* Service d'Hematologie Greffe de Moelle, Hopital Saint Louis, Paris, France Unite INSERM 717 – Universite Paris 7, DBIM, Hopital Saint Louis, Paris, France Hematologie clinique, Hotel Dieu, Paris, France Service des Maladies du Sang, Hopital Claude Huriez, Lille, France Service de Medecine Interne, Groupe Hospitalier Sud, Pessac, France Unite de Transplantation et de Therapie Cellulaire, Institut Paoli Calmettes, Marseille, France Service de Medecine Interne, Hopital Archet, Nice, France Service d'Hematologie Clinique, Centre hospitalier Pontchaillou, Rennes, France Service d'Hematologie Clinique, Hopital Henri-Mondor, Paris, France Service d'Onco-Hematologie, Hopital Michallon, Grenoble, France * Corresponding author; email: gerard.socie{at}sls.aphp.fr. The natural history of PNH clinical subcategories (Classic PNH and aplastic anemia (AA) /PNH syndrome) is still unknown. We retrospectively studied 460 PNH patients diagnosed in 58 French hematological centers from 1950 to 2005. The median (SE) follow-up time was 6.8 (0.5) years. The median survival time 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=0.01) and increasing age (p<0.001) were associated with worse survival prognoses, while use of androgens within the first year after diagnosis was protective (HR 0.17; p=0.01). Transfusions before 1996 (HR 2.7; p=0.007) led to lower survival rates in patients with AA-PNH syndrome, while immunosuppressive treatment was associated with better outcomes (HR 0.33; p=0.03). Evolution to thrombosis affected survival in both subcategories (Classic PNH: HR 7.8, p<0.001; AA-PNH syndrome HR 33.0, p<0.001). Evolution to bi- or pancytopenia for Classic PNH (HR 7.3, p<0.001) and malignancies for AA-PNH syndrome (HR 48.8; p<0.001) were associated with worse outcomes. Although clinical presentation and prognosis factors are different, Classic PNH and AA-PNH syndrome present roughly similar outcomes, mainly affected by complications. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: R. A. Brodsky How I treat paroxysmal nocturnal hemoglobinuria Blood, June 25, 2009; 113(26): 6522 - 6527. [Abstract] [Full Text] [PDF] D. Dingli, L. Luzzatto, and J. M. Pacheco Neutral evolution in paroxysmal nocturnal hemoglobinuria PNAS, November 25, 2008; 105(47): 18496 - 18500. [Abstract] [Full Text] [PDF]

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