[Clinical features and quality of life assessment in Japanese PNH patients enrolled in the International PNH Registry].

The aim of the present study was to clarify the clinical characteristics and quality of life (QOL) of Japanese patients (N=116) enrolled in the International PNH Registry, compared to the whole registry cohort (N=3,457), censored as of March 2015. The proportion of patients treated with eculizumab was comparable between the two cohorts; the Japanese cohort showed higher levels of lactate dehydrogenase and lower hemoglobin values at baseline. Compared to the whole cohort, the Japanese cohort had a greater incidence of bone marrow failure (67.4% vs 56.8%), but fewer episodes of thrombotic events in their medical history (6.4% vs 13.3%), and lower reported rates of abdominal pain (11.9% vs 33.9%), dysphagia (1.7% vs 16.4%), and erectile dysfunction (5.6% vs 25.3%). Additionally, assessment of QOL using FACIT-Fatigue and EORTC QLQ-C30 confirmed that the Japanese cohort had better QOL scores in most of the QOL subscales at baseline. These data are the first report of QOL outcomes for the Japanese cohort in the International PNH Registry; further assessments are ongoing.