Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry.
10.3346/jkms.2016.31.2.214
- Author:
Jun Ho JANG
1
;
Jin Seok KIM
;
Sung Soo YOON
;
Je Hwan LEE
;
Yeo Kyeoung KIM
;
Deog Yeon JO
;
Jooseop CHUNG
;
Sang Kyun SOHN
;
Jong Wook LEE
Author Information
1. Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
- Publication Type:Original Article ; Research Support, Non-U.S. Gov't
- Keywords:
Paroxysmal Nocturnal Hemoglobinuria;
PNH;
Risk Factors;
Mortality
- MeSH:
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
- From:Journal of Korean Medical Science
2016;31(2):214-221
- CountryRepublic of Korea
- Language:English
-
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.
