Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry

• Published in: Journal of Korean medical science Vol. 31; no. 2; pp. 214 - 221
• Main Authors: Jang, Jun Ho, Kim, Jin Seok, Yoon, Sung-Soo, Lee, Je-Hwan, Kim, Yeo-Kyeoung, Jo, Deog-Yeon, Chung, Jooseop, Sohn, Sang Kyun, Lee, Jong Wook
• Format: Journal Article
• Language: English
• Published: Korea (South) The Korean Academy of Medical Sciences 01.02.2016; 대한의학회
• Subjects: Adolescent; Adult; Aged; Aged, 80 and over; Antibodies, Monoclonal - therapeutic use; Antibodies, Monoclonal, Humanized - therapeutic use; Area Under Curve; Child; Dyspnea - etiology; Female; Hemoglobinuria, Paroxysmal - diagnosis; Hemoglobinuria, Paroxysmal - drug therapy; Hemoglobinuria, Paroxysmal - mortality; Hemolysis; Humans; Kaplan-Meier Estimate; Kidney Diseases - complications; Kidney Diseases - diagnosis; L-Lactate Dehydrogenase - metabolism; Male; Middle Aged; Odds Ratio; Original; Registries; Republic of Korea; Retrospective Studies; Risk Factors; ROC Curve; Thromboembolism - complications; Thromboembolism - diagnosis; Young Adult; 의학일반; PNH; Paroxysmal Nocturnal Hemoglobinuria; Mortality; Risk Factors
• ISSN: 1011-8934; 1598-6357; 1598-6357
• DOI: 10.3346/jkms.2016.31.2.214

Paroxysmal nocturnal hemoglobinuria (PNH) is a progressive, systemic, life-threatening disease, characterized by chronic uncontrolled complement activation. A retrospective analysis of 301 Korean PNH patients who had not received eculizumab was performed to systematically identify the clinical symptoms and signs predictive of mortality. PNH patients with hemolysis (lactate dehydrogenase [LDH] ≥ 1.5 × the upper limit of normal [ULN]) have a 4.8-fold higher mortality rate compared with the age- and sex-matched general population (P < 0.001). In contrast, patients with LDH < 1.5 × ULN have a similar mortality rate as the general population (P = 0.824). Thromboembolism (TE) (odds ratio [OR] 7.11; 95% confidence interval [CI] (3.052-16.562), renal impairment (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. The combination of hemolysis and 1 or more of the clinical symptoms such as abdominal pain, chest pain, or dyspnea, resulted in a much greater increased mortality rate when compared with patients with just the individual symptom alone or just hemolysis. Early identification of risk factors related to mortality is crucial for the management of PNH. This trial was registered at www.clinicaltrials.gov as NCT01224483.