Epidemiology in PNH: Clinical Epidemiological Studies in Korea

Abstract

A retrospective analysis was performed on 301 PNH patients from the South Korean National PNH Registry to describe disease burden and identify the associated risk factors for thromboembolism (TE) and mortality. A multivariate analysis showed that PNH patients with elevated hemolysis (LDH levels ≥1.5 times the upper limit of normal [ULN]) at diagnosis were at significantly higher risk for TE than patients with LDH <1.5 × ULN (odds ratio [OR] 7.0; P = 0.013). The combination of LDH ≥1.5 × ULN at diagnosis with the clinical symptoms of abdominal pain, chest pain, dyspnea, or hemoglobinuria was associated with a greater increased risk for TE than elevated hemolysis or clinical symptoms alone. TE (OR 7.11; 95 % confidence interval [CI] (3.052–16.562), impaired renal function (OR 2.953; 95 % CI 1.116–7.818), and PNH-cytopenia (OR 2.547;95 % CI 1.159–5.597) are independent risk factors for mortality, with mortality rates 14-fold (P < 0.001), 8-fold (P < 0.001), and 6.2-fold (P < 0.001) greater than that of the age- and sex-matched general population, respectively.

Early identification of risk factors related to TE and mortality is crucial for the management of high-risk PNH patients.