Natural history of paroxysmal nocturnal hemoglobinuria Journal Article
| Authors: | Hillmen, P.; Lewis, S. M.; Bessler, M.; Luzzatto, L.; Dacie, J. V. |
| Article Title: | Natural history of paroxysmal nocturnal hemoglobinuria |
| Abstract: | Paroxysmal nocturnal hemoglobinuria (PNH), which is characterized by intravascular hemolysis and venous thrombosis, is an acquired clonal disorder associated with a somatic mutation in a totipotent hematopoietic stem cell. An understanding of the natural history of PNH is essential to improve therapy. We have followed a group of 80 consecutive patients with PNH who were referred to Hammersmith Hospital, London, between 1940 and 1970. They were treated with supportive measures, such as oral anticoagulant therapy after established thromboses, and transfusions. The median age of the patients at the time of diagnosis was 42 years (range, 16 to 75), and the median survival after diagnosis was 10 years, with 22 patients (28 percent) surviving for 25 years. Sixty patients have died; 28 of the 48 patients for whom the cause of death is known died from either venous thrombosis or hemorrhage. Thirty-one patients (39 percent) had one or more episodes of venous thrombosis during their illness. Of the 35 patients who survived for 10 years or more, 12 had a spontaneous clinical recovery. No PNH-affected cells were found among the erythrocytes or neutrophils of the patients in prolonged remission, but a few PNH-affected lymphocytes were detectable in three of the four patients tested. Leukemia did not develop in any of the patients. PNH is a chronic disorder that curtails life. A spontaneous long-term remission can occur, which must be taken into account when considering potentially dangerous treatments, such as bone marrow transplantation. Platelet transfusions should be given, as appropriate, and long-term anticoagulation therapy should be considered for all patients. © 1995, Massachusetts Medical Society. All rights reserved. |
| Keywords: | survival; adolescent; adult; human tissue; aged; survival analysis; leukemia; major clinical study; somatic mutation; disease course; flow cytometry; bone marrow cells; anticoagulant therapy; chronic disease; cause of death; vein thrombosis; anticoagulants; remission; blood cell count; bone marrow transplantation; lymphocyte; paroxysmal nocturnal hemoglobinuria; thrombocyte transfusion; hemoglobinuria, paroxysmal; middle age; remission, spontaneous; thrombophlebitis; platelet transfusion; human; male; female; priority journal; article; support, non-u.s. gov't; intravascular hemolysis |
| Journal Title: | New England Journal of Medicine |
| Volume: | 333 |
| Issue: | 19 |
| ISSN: | 0028-4793 |
| Publisher: | Massachusetts Medical Society |
| Date Published: | 1995-11-09 |
| Start Page: | 1253 |
| End Page: | 1258 |
| Language: | English |
| DOI: | 10.1056/nejm199511093331904 |
| PUBMED: | 7566002 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Article -- Export Date: 28 August 2018 -- Source: Scopus |
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