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Comparative study on baseline clinical characteristics of Asian versus non-Asian patients with paroxysmal nocturnal hemoglobinuria

A difference in clinical manifestations of paroxysmal nocturnal hemoglobinuria (PNH) among different races has been suggested. The aim of this study was to clarify whether the clinical characteristics of patients with PNH in the International PNH Registry differ by ethnic background. Patients, who w...

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Published in:International journal of hematology 2019-10, Vol.110 (4), p.411-418
Main Authors: Sakurai, Masatoshi, Jang, Jun Ho, Chou, Wen-Chien, Kim, Jin Seok, Wilson, Amanda, Nishimura, Jun-ichi, Chiou, Tzeon-Jye, Kanakura, Yuzuru, Lee, Jong Wook, Okamoto, Shinichiro
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cited_by cdi_FETCH-LOGICAL-c399t-7b904317d3f45bc61b495ac78643dd4d89cd4313472ec07aaca721178669f4963
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container_title International journal of hematology
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creator Sakurai, Masatoshi
Jang, Jun Ho
Chou, Wen-Chien
Kim, Jin Seok
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Chiou, Tzeon-Jye
Kanakura, Yuzuru
Lee, Jong Wook
Okamoto, Shinichiro
description A difference in clinical manifestations of paroxysmal nocturnal hemoglobinuria (PNH) among different races has been suggested. The aim of this study was to clarify whether the clinical characteristics of patients with PNH in the International PNH Registry differ by ethnic background. Patients, who were eculizumab naïve at baseline and had ≥ 1% PNH clone size, were eligible for this analysis. Totally, 1793 patients were enrolled and divided into two cohorts, Asian ( N  = 246) and non-Asian ( N  = 1547). The Asian cohort was further divided into Asians in Asia cohort ( N  = 202) and Asians in non-Asia cohort ( N  = 44), based on geographical region. The Asian cohort had significantly higher PNH clone size in granulocytes, higher lactate dehydrogenase levels, and lower hemoglobin levels. However, the frequencies of symptoms including abdominal pain, backache, easy bleeding, fatigue and headache at baseline were significantly lower in the Asian cohort. The proportion of patients with a history of thromboembolism (TE) was significantly lower in the Asian than in the non-Asian cohort (3.6% vs. 8.9%, P 
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The aim of this study was to clarify whether the clinical characteristics of patients with PNH in the International PNH Registry differ by ethnic background. Patients, who were eculizumab naïve at baseline and had ≥ 1% PNH clone size, were eligible for this analysis. Totally, 1793 patients were enrolled and divided into two cohorts, Asian ( N  = 246) and non-Asian ( N  = 1547). The Asian cohort was further divided into Asians in Asia cohort ( N  = 202) and Asians in non-Asia cohort ( N  = 44), based on geographical region. The Asian cohort had significantly higher PNH clone size in granulocytes, higher lactate dehydrogenase levels, and lower hemoglobin levels. However, the frequencies of symptoms including abdominal pain, backache, easy bleeding, fatigue and headache at baseline were significantly lower in the Asian cohort. The proportion of patients with a history of thromboembolism (TE) was significantly lower in the Asian than in the non-Asian cohort (3.6% vs. 8.9%, P  &lt; 0.01); however, there was no difference between Asians in Asia and Asians in non-Asia (3.3% vs. 4.9%, P  = 0.61). These findings suggested that genetic factors may play a stronger role in developing TE than lifestyle factors in PNH patients.</description><identifier>ISSN: 0925-5710</identifier><identifier>EISSN: 1865-3774</identifier><identifier>DOI: 10.1007/s12185-019-02699-7</identifier><identifier>PMID: 31278635</identifier><language>eng</language><publisher>Tokyo: Springer Japan</publisher><subject>Abdominal Pain - epidemiology ; Abdominal Pain - etiology ; Adult ; Asian Continental Ancestry Group ; Asian people ; Back Pain - epidemiology ; Back Pain - etiology ; Bleeding ; Cloning ; Cohort Studies ; Comparative studies ; Continental Population Groups ; Fatigue - epidemiology ; Fatigue - etiology ; Female ; Genetic factors ; Granulocytes - pathology ; Headache ; Headache - epidemiology ; Headache - etiology ; Hematology ; Hemoglobin ; Hemoglobins ; Hemoglobinuria, Paroxysmal - blood ; Hemoglobinuria, Paroxysmal - complications ; Hemoglobinuria, Paroxysmal - genetics ; Hemoglobinuria, Paroxysmal - physiopathology ; Hemorrhage - epidemiology ; Hemorrhage - etiology ; Humans ; L-Lactate dehydrogenase ; L-Lactate Dehydrogenase - metabolism ; Lactate dehydrogenase ; Lactic acid ; Leukocytes (granulocytic) ; Male ; Medicine ; Medicine &amp; Public Health ; Middle Aged ; Oncology ; Original Article ; Pain ; Paroxysmal nocturnal hemoglobinuria ; Registries ; Signs and symptoms ; Thromboembolism ; Thromboembolism - epidemiology ; Thromboembolism - etiology</subject><ispartof>International journal of hematology, 2019-10, Vol.110 (4), p.411-418</ispartof><rights>Japanese Society of Hematology 2019</rights><rights>International Journal of Hematology is a copyright of Springer, (2019). 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The aim of this study was to clarify whether the clinical characteristics of patients with PNH in the International PNH Registry differ by ethnic background. Patients, who were eculizumab naïve at baseline and had ≥ 1% PNH clone size, were eligible for this analysis. Totally, 1793 patients were enrolled and divided into two cohorts, Asian ( N  = 246) and non-Asian ( N  = 1547). The Asian cohort was further divided into Asians in Asia cohort ( N  = 202) and Asians in non-Asia cohort ( N  = 44), based on geographical region. The Asian cohort had significantly higher PNH clone size in granulocytes, higher lactate dehydrogenase levels, and lower hemoglobin levels. However, the frequencies of symptoms including abdominal pain, backache, easy bleeding, fatigue and headache at baseline were significantly lower in the Asian cohort. The proportion of patients with a history of thromboembolism (TE) was significantly lower in the Asian than in the non-Asian cohort (3.6% vs. 8.9%, P  &lt; 0.01); however, there was no difference between Asians in Asia and Asians in non-Asia (3.3% vs. 4.9%, P  = 0.61). 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Public Health</subject><subject>Middle Aged</subject><subject>Oncology</subject><subject>Original Article</subject><subject>Pain</subject><subject>Paroxysmal nocturnal hemoglobinuria</subject><subject>Registries</subject><subject>Signs and symptoms</subject><subject>Thromboembolism</subject><subject>Thromboembolism - epidemiology</subject><subject>Thromboembolism - etiology</subject><issn>0925-5710</issn><issn>1865-3774</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2019</creationdate><recordtype>article</recordtype><recordid>eNp9kUtv1DAUhS1ERafT_gEWyBIbNgY_43hZjXhUqsSGriPHcTquEnvwdQqz5ZdjJgUkFmx8bZ_vHj8OQi8Zfcso1e-AcdYqQpkhlDfGEP0MbVjbKCK0ls_RhhquiNKMnqMLgAdKmaZSv0DngnHdNkJt0I9dmg822xIePYayDEecIu4t-ClEj10dg7MTdvsKueJzgBIc4DTiawg24kefYQEcUyTrxqF6-VgAfwtlX1c5fT_CXC1icmXJsc72fk73U-pDXHKwl-hstBP4q6e6RXcf3n_ZfSK3nz_e7K5viRPGFKJ7Q6VgehCjVL1rWC-Nsq6-Q4phkENr3FB1ITX3jmprndWcsao3ZpSmEVv0ZvU95PR18VC6OYDz02SjTwt0nCtRv0XUskWv_0Ef0unqJ4orRdsTxVfK5QSQ_dgdcphtPnaMdr8S6taEuppQd0qo07Xp1ZP10s9--NPyO5IKiBWAKsV7n_-e_R_bn-81neE</recordid><startdate>20191001</startdate><enddate>20191001</enddate><creator>Sakurai, Masatoshi</creator><creator>Jang, Jun Ho</creator><creator>Chou, Wen-Chien</creator><creator>Kim, Jin Seok</creator><creator>Wilson, Amanda</creator><creator>Nishimura, Jun-ichi</creator><creator>Chiou, Tzeon-Jye</creator><creator>Kanakura, Yuzuru</creator><creator>Lee, Jong Wook</creator><creator>Okamoto, Shinichiro</creator><general>Springer Japan</general><general>Springer Nature B.V</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7RV</scope><scope>7T5</scope><scope>7T7</scope><scope>7TM</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FD</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>BENPR</scope><scope>C1K</scope><scope>CCPQU</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>H94</scope><scope>K9.</scope><scope>KB0</scope><scope>M0S</scope><scope>M1P</scope><scope>NAPCQ</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>7X8</scope></search><sort><creationdate>20191001</creationdate><title>Comparative study on baseline clinical characteristics of Asian versus non-Asian patients with paroxysmal nocturnal hemoglobinuria</title><author>Sakurai, Masatoshi ; 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Public Health</topic><topic>Middle Aged</topic><topic>Oncology</topic><topic>Original Article</topic><topic>Pain</topic><topic>Paroxysmal nocturnal hemoglobinuria</topic><topic>Registries</topic><topic>Signs and symptoms</topic><topic>Thromboembolism</topic><topic>Thromboembolism - epidemiology</topic><topic>Thromboembolism - etiology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Sakurai, Masatoshi</creatorcontrib><creatorcontrib>Jang, Jun Ho</creatorcontrib><creatorcontrib>Chou, Wen-Chien</creatorcontrib><creatorcontrib>Kim, Jin Seok</creatorcontrib><creatorcontrib>Wilson, Amanda</creatorcontrib><creatorcontrib>Nishimura, Jun-ichi</creatorcontrib><creatorcontrib>Chiou, Tzeon-Jye</creatorcontrib><creatorcontrib>Kanakura, Yuzuru</creatorcontrib><creatorcontrib>Lee, Jong Wook</creatorcontrib><creatorcontrib>Okamoto, Shinichiro</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Nursing &amp; 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The aim of this study was to clarify whether the clinical characteristics of patients with PNH in the International PNH Registry differ by ethnic background. Patients, who were eculizumab naïve at baseline and had ≥ 1% PNH clone size, were eligible for this analysis. Totally, 1793 patients were enrolled and divided into two cohorts, Asian ( N  = 246) and non-Asian ( N  = 1547). The Asian cohort was further divided into Asians in Asia cohort ( N  = 202) and Asians in non-Asia cohort ( N  = 44), based on geographical region. The Asian cohort had significantly higher PNH clone size in granulocytes, higher lactate dehydrogenase levels, and lower hemoglobin levels. However, the frequencies of symptoms including abdominal pain, backache, easy bleeding, fatigue and headache at baseline were significantly lower in the Asian cohort. The proportion of patients with a history of thromboembolism (TE) was significantly lower in the Asian than in the non-Asian cohort (3.6% vs. 8.9%, P  &lt; 0.01); however, there was no difference between Asians in Asia and Asians in non-Asia (3.3% vs. 4.9%, P  = 0.61). These findings suggested that genetic factors may play a stronger role in developing TE than lifestyle factors in PNH patients.</abstract><cop>Tokyo</cop><pub>Springer Japan</pub><pmid>31278635</pmid><doi>10.1007/s12185-019-02699-7</doi><tpages>8</tpages></addata></record>
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subjects Abdominal Pain - epidemiology
Abdominal Pain - etiology
Adult
Asian Continental Ancestry Group
Asian people
Back Pain - epidemiology
Back Pain - etiology
Bleeding
Cloning
Cohort Studies
Comparative studies
Continental Population Groups
Fatigue - epidemiology
Fatigue - etiology
Female
Genetic factors
Granulocytes - pathology
Headache
Headache - epidemiology
Headache - etiology
Hematology
Hemoglobin
Hemoglobins
Hemoglobinuria, Paroxysmal - blood
Hemoglobinuria, Paroxysmal - complications
Hemoglobinuria, Paroxysmal - genetics
Hemoglobinuria, Paroxysmal - physiopathology
Hemorrhage - epidemiology
Hemorrhage - etiology
Humans
L-Lactate dehydrogenase
L-Lactate Dehydrogenase - metabolism
Lactate dehydrogenase
Lactic acid
Leukocytes (granulocytic)
Male
Medicine
Medicine & Public Health
Middle Aged
Oncology
Original Article
Pain
Paroxysmal nocturnal hemoglobinuria
Registries
Signs and symptoms
Thromboembolism
Thromboembolism - epidemiology
Thromboembolism - etiology
title Comparative study on baseline clinical characteristics of Asian versus non-Asian patients with paroxysmal nocturnal hemoglobinuria
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