Cargando…

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

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 sympt...

Descripción completa

Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Korean Academy of Medical Sciences 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4729501/
https://www.ncbi.nlm.nih.gov/pubmed/26839475
http://dx.doi.org/10.3346/jkms.2016.31.2.214
_version_ 1782412263537246208
author 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
author_facet 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
author_sort Jang, Jun Ho
collection PubMed
description 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.
format Online
Article
Text
id pubmed-4729501
institution National Center for Biotechnology Information
language English
publishDate 2016
publisher The Korean Academy of Medical Sciences
record_format MEDLINE/PubMed
spelling pubmed-47295012016-02-02 Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry 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 J Korean Med Sci Original Article 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. The Korean Academy of Medical Sciences 2016-02 2016-01-26 /pmc/articles/PMC4729501/ /pubmed/26839475 http://dx.doi.org/10.3346/jkms.2016.31.2.214 Text en © 2016 The Korean Academy of Medical Sciences. http://creativecommons.org/licenses/by-nc/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Original Article
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
Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry
title Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry
title_full Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry
title_fullStr Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry
title_full_unstemmed Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry
title_short Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry
title_sort predictive factors of mortality in population of patients with paroxysmal nocturnal hemoglobinuria (pnh): results from a korean pnh registry
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4729501/
https://www.ncbi.nlm.nih.gov/pubmed/26839475
http://dx.doi.org/10.3346/jkms.2016.31.2.214
work_keys_str_mv AT jangjunho predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT kimjinseok predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT yoonsungsoo predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT leejehwan predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT kimyeokyeoung predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT jodeogyeon predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT chungjooseop predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT sohnsangkyun predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry
AT leejongwook predictivefactorsofmortalityinpopulationofpatientswithparoxysmalnocturnalhemoglobinuriapnhresultsfromakoreanpnhregistry