Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories Export

@article{citeulike:3680078, abstract = {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 presenta-tion and prognosis factors are different, classic PNH and AA-PNH syndrome present roughly similar outcomes, affected mainly by complications. 10.1182/blood-2008-01-133918}, author = {de Latour, Regis P. and Mary, Jean Y. and Salanoubat, Celia and Terriou, Louis and Etienne, Gabriel and Mohty, Mohamad and Roth, Sophie and de Guibert, Sophie and Maury, Sebastien and Cahn, Jean Y. and Socie, Gerard and On}, citeulike-article-id = {3680078}, citeulike-linkout-0 = {http://dx.doi.org/10.1182/blood-2008-01-133918}, citeulike-linkout-1 = {http://bloodjournal.hematologylibrary.org/cgi/content/abstract/112/8/3099}, citeulike-linkout-2 = {http://view.ncbi.nlm.nih.gov/pubmed/18535202}, citeulike-linkout-3 = {http://www.hubmed.org/display.cgi?uids=18535202}, day = {15}, doi = {10.1182/blood-2008-01-133918}, journal = {Blood}, keywords = {pnh}, month = {October}, number = {8}, pages = {3099--3106}, posted-at = {2008-11-24 00:19:59}, priority = {2}, title = {Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories}, url = {http://dx.doi.org/10.1182/blood-2008-01-133918}, volume = {112}, year = {2008} }

TY - JOUR ID - citeulike:3680078 L3 - citeulike-article-id:3680078 N2 - 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 presenta-tion and prognosis factors are different, classic PNH and AA-PNH syndrome present roughly similar outcomes, affected mainly by complications. 10.1182/blood-2008-01-133918 IS - 8 JF - Blood EP - 3106 TI - Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories VL - 112 SP - 3099 KW - pnh AU - de Latour, Regis AU - Mary, Jean AU - Salanoubat, Celia AU - Terriou, Louis AU - Etienne, Gabriel AU - Mohty, Mohamad AU - Roth, Sophie AU - de Guibert, Sophie AU - Maury, Sebastien AU - Cahn, Jean AU - Socie, Gerard AU - On PY - 2008/10/15/ UR - http://dx.doi.org/10.1182/blood-2008-01-133918 ER -