title: Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry.
abstract: 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.
authors.affiliation: Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
authors.forename: Jun Ho
authors.identifier: 0000-0001-7423-4676
authors.initials: JH
authors.lastname: Jang
authors.affiliation: Divison of Hematology, Department of Internal Medicine, Yonsei University College of Medicine, Severance Hospital, Seoul Korea.
authors.forename: Jin Seok
authors.identifier: 0000-0001-8986-8436
authors.initials: JS
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, Seoul National University College of Medicine, Seoul, Korea.
authors.forename: Sung-Soo
authors.identifier: 0000-0003-2591-7459
authors.initials: SS
authors.lastname: Yoon
authors.affiliation: Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
authors.forename: Je-Hwan
authors.identifier: 0000-0002-7060-1675
authors.initials: JH
authors.lastname: Lee
authors.affiliation: Department of Hematology-Oncology, Chonnam National University Hwasun Hospital, Hwasun, Korea.
authors.forename: Yeo-Kyeoung
authors.identifier: 0000-0001-5447-4285
authors.initials: YK
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, Chungnam National University Hospital, Chungnam National University College of Medicine, Daejeon, Korea.
authors.forename: Deog-Yeon
authors.identifier: 0000-0002-8267-4214
authors.initials: DY
authors.lastname: Jo
authors.affiliation: Division of Hematology-Oncology, Department of Internal Medicine, Pusan National University Hospital, Pusan National University School of Medicine, Busan, Korea.
authors.forename: Jooseop
authors.identifier: 0000-0001-7008-245X
authors.initials: J
authors.lastname: Chung
authors.affiliation: Department of Hematology/Oncology, Kyungpook National University School of Medicine, Daegu, Korea.
authors.forename: Sang Kyun
authors.identifier: 0000-0003-1932-0429
authors.initials: SK
authors.lastname: Sohn
authors.affiliation: Division of Hematology, Department of Internal Medicine, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Jong Wook
authors.identifier: 0000-0003-2949-4166
authors.initials: JW
authors.lastname: Lee
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use; D019540:Area Under Curve; D002648:Child; D004417:Dyspnea / Q000209:etiology; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000175:diagnosis* / Q000188:drug therapy / Q000401:mortality; D006461:Hemolysis; D006801:Humans; D053208:Kaplan-Meier Estimate; D007674:Kidney Diseases / Q000150:complications / Q000175:diagnosis; D007770:L-Lactate Dehydrogenase / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D016017:Odds Ratio; D012372:ROC Curve; D012042:Registries; D056910:Republic of Korea; D012189:Retrospective Studies; D012307:Risk Factors; D013923:Thromboembolism / Q000150:complications / Q000175:diagnosis; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab; D007770:L-Lactate Dehydrogenase
keywords: Mortality; PNH; Paroxysmal Nocturnal Hemoglobinuria; Risk Factors
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