J Korean Med Sci.  2016 Feb;31(2):214-221. 10.3346/jkms.2016.31.2.214.

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

Affiliations
  • 1Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
  • 2Divison of Hematology, Department of Internal Medicine, Yonsei University College of Medicine, Severance Hospital, Seoul, Korea.
  • 3Department of Internal Medicine, Seoul National University College of Medicine, Seoul, Korea.
  • 4Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
  • 5Department of Hematology-Oncology, Chonnam National University Hwasun Hospital, Hwasun, Korea.
  • 6Department of Internal Medicine, Chungnam National University Hospital, Chungnam National University College of Medicine, Daejeon, Korea.
  • 7Division of Hematology-Oncology, Department of Internal Medicine, Pusan National University Hospital, Pusan National University School of Medicine, Busan, Korea.
  • 8Department of Hematology/Oncology, Kyungpook National University School of Medicine, Daegu, Korea.
  • 9Division of Hematology, Department of Internal Medicine, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea. jwlee@catholic.ac.kr

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] > or = 1.5 x 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 x 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.

Keyword

Paroxysmal Nocturnal Hemoglobinuria; PNH; Risk Factors; Mortality

MeSH Terms

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/drug therapy/mortality
Hemolysis
Humans
Kaplan-Meier Estimate
Kidney Diseases/complications/diagnosis
L-Lactate Dehydrogenase/metabolism
Male
Middle Aged
Odds Ratio
ROC Curve
Registries
Republic of Korea
Retrospective Studies
Risk Factors
Thromboembolism/complications/diagnosis
Young Adult
Antibodies, Monoclonal
Antibodies, Monoclonal, Humanized
L-Lactate Dehydrogenase
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