The Clinical Course of PNH in the USA and in JAPAN

摘要

Summary: PNH, which is characterized by intravascular hemofysis and venous thrombosis, is an uncommon disorder of hematopoietic stem cells with an acquired somatic mutation of PIG-A. An understanding of the natural history of PNH is essential to improve therapy. To determine and to directly compare the clinical courses of PNH patients from the USA and Japan, data were collected for 385 patients with PNH. There were 176 patients from Duke (DP) and 209 patients from Japan (JP). The mean age at diagnosis (mean + SE) was 32.8 + 1.2 years (4-80) in DP, and 45.1 + 1.3 years (10-86) in JP. The incidence of various clinical events was analyzed. These analyses provide important diagnostic and long-term data on two large cohorts of PNH patients, and identify important differences between Caucasian and Asian patients with PNH. Caucasians tend to be younger and have classical symptoms of PNH including thrombosis, while Asians tend to be older and have symptoms of aplasia. Multivariate analysis of factors influencingsurvival was also undertaken. By this study, estimates of PNH prognostic factors may provide a better understanding of the complications of the disease so that therapeutic interventions may be sought. Further, by noting differences in the two populations, possible genetic modifiers of the course of the disease may be identified for further investigation.