Changing prognosis in paroxysmal nocturnal haemoglobinuria disease subcategories: an analysis of the International PNH Registry.
Identifieur interne : 003710 ( Ncbi/Merge ); précédent : 003709; suivant : 003711Changing prognosis in paroxysmal nocturnal haemoglobinuria disease subcategories: an analysis of the International PNH Registry.
Auteurs : G. Socié [France] ; H. Schrezenmeier [Allemagne] ; P. Muus [Pays-Bas] ; I. Lisukov [Russie] ; A. Röth [Allemagne] ; A. Kulasekararaj [Royaume-Uni] ; J W Lee [Corée du Sud] ; D. Araten [États-Unis] ; A. Hill [Royaume-Uni] ; R. Brodsky [États-Unis] ; A. Urbano-Ispizua [Espagne] ; J. Szer [Australie] ; A. Wilson [États-Unis] ; P. Hillmen [Royaume-Uni]Source :
- Internal medicine journal [ 1445-5994 ] ; 2016.
Descripteurs français
- KwdFr :
- Adulte, Adulte d'âge moyen, Analyse de survie, Analyse multivariée, Anticorps monoclonaux humanisés (usage thérapeutique), Anémie aplasique (épidémiologie), Cause de décès, Enregistrements, Facteurs de risque, Femelle, France, Humains, Hémoglobinurie paroxystique (), Hémoglobinurie paroxystique (mortalité), Incidence, Mâle, Pronostic, Thrombose (épidémiologie), Transfusion d'érythrocytes, Études rétrospectives.
- MESH :
- mortalité : Hémoglobinurie paroxystique.
- usage thérapeutique : Anticorps monoclonaux humanisés.
- épidémiologie : Anémie aplasique, Thrombose.
- Adulte, Adulte d'âge moyen, Analyse de survie, Analyse multivariée, Cause de décès, Enregistrements, Facteurs de risque, Femelle, France, Humains, Hémoglobinurie paroxystique, Incidence, Mâle, Pronostic, Transfusion d'érythrocytes, Études rétrospectives.
English descriptors
- KwdEn :
- Adult, Anemia, Aplastic (epidemiology), Antibodies, Monoclonal, Humanized (therapeutic use), Cause of Death, Erythrocyte Transfusion, Female, France, Hemoglobinuria, Paroxysmal (classification), Hemoglobinuria, Paroxysmal (mortality), Hemoglobinuria, Paroxysmal (therapy), Humans, Incidence, Male, Middle Aged, Multivariate Analysis, Prognosis, Registries, Retrospective Studies, Risk Factors, Survival Analysis, Thrombosis (epidemiology).
- MESH :
- chemical , therapeutic use : Antibodies, Monoclonal, Humanized.
- classification : Hemoglobinuria, Paroxysmal.
- epidemiology : Anemia, Aplastic, Thrombosis.
- mortality : Hemoglobinuria, Paroxysmal.
- therapy : Hemoglobinuria, Paroxysmal.
- Adult, Cause of Death, Erythrocyte Transfusion, Female, France, Humans, Incidence, Male, Middle Aged, Multivariate Analysis, Prognosis, Registries, Retrospective Studies, Risk Factors, Survival Analysis.
Abstract
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare disease. Although much progress has been made in the understanding of the pathophysiology of the disease, far less is known with respect to the clinical outcomes of patients with PNH. Few retrospective studies provide survival estimates, and even fewer have explored the clinical heterogeneity of the disease. Haemolytic and aplastic anaemia (AA) forms of the disease have been recognised as main disease categories, with the haemolytic form being associated with the worst prognosis by the largest studied cohort some years ago.
DOI: 10.1111/imj.13160
PubMed: 27305361
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Socié</name><affiliation wicri:level="3"><nlm:affiliation>Hematology Transplantation, AP-HP, Hospital Saint Louis, University Paris VII Denis Diderot and Inserm UMR 1160, Paris, France. gerard.socie@sls.aphp.fr.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Hematology Transplantation, AP-HP, Hospital Saint Louis, University Paris VII Denis Diderot and Inserm UMR 1160, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Schrezenmeier, H" sort="Schrezenmeier, H" uniqKey="Schrezenmeier H" first="H" last="Schrezenmeier">H. 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Socié</name><affiliation wicri:level="3"><nlm:affiliation>Hematology Transplantation, AP-HP, Hospital Saint Louis, University Paris VII Denis Diderot and Inserm UMR 1160, Paris, France. gerard.socie@sls.aphp.fr.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Hematology Transplantation, AP-HP, Hospital Saint Louis, University Paris VII Denis Diderot and Inserm UMR 1160, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Schrezenmeier, H" sort="Schrezenmeier, H" uniqKey="Schrezenmeier H" first="H" last="Schrezenmeier">H. Schrezenmeier</name><affiliation wicri:level="3"><nlm:affiliation>Institute of Transfusion Medicine, University of Ulm Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen and University Hospital Ulm, Ulm, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>Institute of Transfusion Medicine, University of Ulm Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen and University Hospital Ulm, Ulm</wicri:regionArea><placeName><region type="land" nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Tübingen</region><settlement type="city">Ulm</settlement></placeName></affiliation></author><author><name sortKey="Muus, P" sort="Muus, P" uniqKey="Muus P" first="P" last="Muus">P. Muus</name><affiliation wicri:level="3"><nlm:affiliation>Department of Hematology, Radboud UMC, Nijmegen, the Netherlands.</nlm:affiliation><country xml:lang="fr" wicri:curation="lc">Pays-Bas</country><wicri:regionArea>Department of Hematology, Radboud UMC, Nijmegen</wicri:regionArea><placeName><settlement type="city">Nimègue</settlement><region type="province" nuts="2">Gueldre</region></placeName></affiliation></author><author><name sortKey="Lisukov, I" sort="Lisukov, I" uniqKey="Lisukov I" first="I" last="Lisukov">I. Lisukov</name><affiliation wicri:level="1"><nlm:affiliation>Institution of Children Hematology and Transplantation, St. Petersburg, Russia.</nlm:affiliation><country xml:lang="fr">Russie</country><wicri:regionArea>Institution of Children Hematology and Transplantation, St. Petersburg</wicri:regionArea><wicri:noRegion>St. Petersburg</wicri:noRegion></affiliation></author><author><name sortKey="Roth, A" sort="Roth, A" uniqKey="Roth A" first="A" last="Röth">A. Röth</name><affiliation wicri:level="1"><nlm:affiliation>University Hospital Essen, Essen, Germany.</nlm:affiliation><country xml:lang="fr">Allemagne</country><wicri:regionArea>University Hospital Essen, Essen</wicri:regionArea><wicri:noRegion>Essen</wicri:noRegion><wicri:noRegion>Essen</wicri:noRegion><wicri:noRegion>Essen</wicri:noRegion></affiliation></author><author><name sortKey="Kulasekararaj, A" sort="Kulasekararaj, A" uniqKey="Kulasekararaj A" first="A" last="Kulasekararaj">A. Kulasekararaj</name><affiliation wicri:level="3"><nlm:affiliation>Kings College Hospital, London, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Kings College Hospital, London</wicri:regionArea><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Lee, J W" sort="Lee, J W" uniqKey="Lee J" first="J W" last="Lee">J W Lee</name><affiliation wicri:level="3"><nlm:affiliation>Seoul St. Mary's Hospital, Seoul, Korea.</nlm:affiliation><country xml:lang="fr">Corée du Sud</country><wicri:regionArea>Seoul St. Mary's Hospital, Seoul</wicri:regionArea><placeName><settlement type="city">Séoul</settlement><region type="capital">Région capitale de Séoul</region></placeName></affiliation></author><author><name sortKey="Araten, D" sort="Araten, D" uniqKey="Araten D" first="D" last="Araten">D. Araten</name><affiliation wicri:level="1"><nlm:affiliation>Laura and Isaac Perlmutter Cancer Center, NYU Langone Medical Center, New York VA Medical Center, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Laura and Isaac Perlmutter Cancer Center, NYU Langone Medical Center, New York VA Medical Center</wicri:regionArea><wicri:noRegion>New York VA Medical Center</wicri:noRegion></affiliation></author><author><name sortKey="Hill, A" sort="Hill, A" uniqKey="Hill A" first="A" last="Hill">A. Hill</name><affiliation wicri:level="1"><nlm:affiliation>St James's University Hospital, Leeds, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>St James's University Hospital, Leeds</wicri:regionArea><wicri:noRegion>Leeds</wicri:noRegion></affiliation></author><author><name sortKey="Brodsky, R" sort="Brodsky, R" uniqKey="Brodsky R" first="R" last="Brodsky">R. Brodsky</name><affiliation wicri:level="1"><nlm:affiliation>Johns Hopkins University Medical Center, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Johns Hopkins University Medical Center</wicri:regionArea></affiliation></author><author><name sortKey="Urbano Ispizua, A" sort="Urbano Ispizua, A" uniqKey="Urbano Ispizua A" first="A" last="Urbano-Ispizua">A. Urbano-Ispizua</name><affiliation wicri:level="3"><nlm:affiliation>Hospital Clínic Barcelona, Barcelona, Spain.</nlm:affiliation><country xml:lang="fr">Espagne</country><wicri:regionArea>Hospital Clínic Barcelona, Barcelona</wicri:regionArea><placeName><settlement type="city">Barcelone</settlement><region nuts="2" type="region">Catalogne</region></placeName></affiliation></author><author><name sortKey="Szer, J" sort="Szer, J" uniqKey="Szer J" first="J" last="Szer">J. Szer</name><affiliation wicri:level="1"><nlm:affiliation>Royal Melbourne Hospital, Melbourne, Victoria, Australia.</nlm:affiliation><country xml:lang="fr">Australie</country><wicri:regionArea>Royal Melbourne Hospital, Melbourne, Victoria</wicri:regionArea><wicri:noRegion>Victoria</wicri:noRegion></affiliation></author><author><name sortKey="Wilson, A" sort="Wilson, A" uniqKey="Wilson A" first="A" last="Wilson">A. Wilson</name><affiliation wicri:level="2"><nlm:affiliation>Alexion Pharmaceuticals, New Haven, Connecticut, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Alexion Pharmaceuticals, New Haven, Connecticut</wicri:regionArea><placeName><region type="state">Connecticut</region></placeName></affiliation></author><author><name sortKey="Hillmen, P" sort="Hillmen, P" uniqKey="Hillmen P" first="P" last="Hillmen">P. Hillmen</name><affiliation wicri:level="1"><nlm:affiliation>St James's University Hospital, Leeds, UK.</nlm:affiliation><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>St James's University Hospital, Leeds</wicri:regionArea><wicri:noRegion>Leeds</wicri:noRegion></affiliation></author></analytic><series><title level="j">Internal medicine journal</title><idno type="eISSN">1445-5994</idno><imprint><date when="2016" type="published">2016</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term><term>Anemia, Aplastic (epidemiology)</term><term>Antibodies, Monoclonal, Humanized (therapeutic use)</term><term>Cause of Death</term><term>Erythrocyte Transfusion</term><term>Female</term><term>France</term><term>Hemoglobinuria, Paroxysmal (classification)</term><term>Hemoglobinuria, Paroxysmal (mortality)</term><term>Hemoglobinuria, Paroxysmal (therapy)</term><term>Humans</term><term>Incidence</term><term>Male</term><term>Middle Aged</term><term>Multivariate Analysis</term><term>Prognosis</term><term>Registries</term><term>Retrospective Studies</term><term>Risk Factors</term><term>Survival Analysis</term><term>Thrombosis (epidemiology)</term></keywords><keywords scheme="KwdFr" xml:lang="fr"><term>Adulte</term><term>Adulte d'âge moyen</term><term>Analyse de survie</term><term>Analyse multivariée</term><term>Anticorps monoclonaux humanisés (usage thérapeutique)</term><term>Anémie aplasique (épidémiologie)</term><term>Cause de décès</term><term>Enregistrements</term><term>Facteurs de risque</term><term>Femelle</term><term>France</term><term>Humains</term><term>Hémoglobinurie paroxystique ()</term><term>Hémoglobinurie paroxystique (mortalité)</term><term>Incidence</term><term>Mâle</term><term>Pronostic</term><term>Thrombose (épidémiologie)</term><term>Transfusion d'érythrocytes</term><term>Études rétrospectives</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Antibodies, Monoclonal, Humanized</term></keywords><keywords scheme="MESH" qualifier="classification" xml:lang="en"><term>Hemoglobinuria, Paroxysmal</term></keywords><keywords scheme="MESH" qualifier="epidemiology" xml:lang="en"><term>Anemia, Aplastic</term><term>Thrombosis</term></keywords><keywords scheme="MESH" qualifier="mortality" xml:lang="en"><term>Hemoglobinuria, Paroxysmal</term></keywords><keywords scheme="MESH" qualifier="mortalité" xml:lang="fr"><term>Hémoglobinurie paroxystique</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Hemoglobinuria, Paroxysmal</term></keywords><keywords scheme="MESH" qualifier="usage thérapeutique" xml:lang="fr"><term>Anticorps monoclonaux humanisés</term></keywords><keywords scheme="MESH" qualifier="épidémiologie" xml:lang="fr"><term>Anémie aplasique</term><term>Thrombose</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Adult</term><term>Cause of Death</term><term>Erythrocyte Transfusion</term><term>Female</term><term>France</term><term>Humans</term><term>Incidence</term><term>Male</term><term>Middle Aged</term><term>Multivariate Analysis</term><term>Prognosis</term><term>Registries</term><term>Retrospective Studies</term><term>Risk Factors</term><term>Survival Analysis</term></keywords><keywords scheme="MESH" xml:lang="fr"><term>Adulte</term><term>Adulte d'âge moyen</term><term>Analyse de survie</term><term>Analyse multivariée</term><term>Cause de décès</term><term>Enregistrements</term><term>Facteurs de risque</term><term>Femelle</term><term>France</term><term>Humains</term><term>Hémoglobinurie paroxystique</term><term>Incidence</term><term>Mâle</term><term>Pronostic</term><term>Transfusion d'érythrocytes</term><term>Études rétrospectives</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Paroxysmal nocturnal haemoglobinuria (PNH) is a rare disease. Although much progress has been made in the understanding of the pathophysiology of the disease, far less is known with respect to the clinical outcomes of patients with PNH. Few retrospective studies provide survival estimates, and even fewer have explored the clinical heterogeneity of the disease. Haemolytic and aplastic anaemia (AA) forms of the disease have been recognised as main disease categories, with the haemolytic form being associated with the worst prognosis by the largest studied cohort some years ago.</div></front></TEI><pubmed><MedlineCitation Status="MEDLINE" Owner="NLM"><PMID Version="1">27305361</PMID><DateCreated><Year>2016</Year><Month>09</Month><Day>16</Day></DateCreated><DateCompleted><Year>2017</Year><Month>08</Month><Day>24</Day></DateCompleted><DateRevised><Year>2017</Year><Month>08</Month><Day>24</Day></DateRevised><Article PubModel="Print"><Journal><ISSN IssnType="Electronic">1445-5994</ISSN><JournalIssue CitedMedium="Internet"><Volume>46</Volume><Issue>9</Issue><PubDate><Year>2016</Year><Month>Sep</Month></PubDate></JournalIssue><Title>Internal medicine journal</Title><ISOAbbreviation>Intern Med J</ISOAbbreviation></Journal><ArticleTitle>Changing prognosis in paroxysmal nocturnal haemoglobinuria disease subcategories: an analysis of the International PNH Registry.</ArticleTitle><Pagination><MedlinePgn>1044-53</MedlinePgn></Pagination><ELocationID EIdType="doi" ValidYN="Y">10.1111/imj.13160</ELocationID><Abstract><AbstractText Label="BACKGROUND" NlmCategory="BACKGROUND">Paroxysmal nocturnal haemoglobinuria (PNH) is a rare disease. Although much progress has been made in the understanding of the pathophysiology of the disease, far less is known with respect to the clinical outcomes of patients with PNH. Few retrospective studies provide survival estimates, and even fewer have explored the clinical heterogeneity of the disease. Haemolytic and aplastic anaemia (AA) forms of the disease have been recognised as main disease categories, with the haemolytic form being associated with the worst prognosis by the largest studied cohort some years ago.</AbstractText><AbstractText Label="AIMS" NlmCategory="OBJECTIVE">To describe mortality and causes of death in PNH overall and by PNH classification and to evaluate risk factors associated with mortality.</AbstractText><AbstractText Label="METHODS" NlmCategory="METHODS">We analysed data of 2356 patients enrolled in the International PNH Registry with multivariate analyses, using time-dependent covariates. Patients were classified into haemolytic, AA/PNH syndrome or intermediate PNH.</AbstractText><AbstractText Label="RESULTS" NlmCategory="RESULTS">Overall, 122 (5.2%) patients died after enrolment, the incidence according to subcategories being 5.1, 11.7, 2.0 and 4.8% for patients with haemolytic PNH, AA-PNH, intermediate and insufficient data respectively. Older age and decreased performance status also affected survival in multivariate analysis. Improved outcome of patients with haemolytic PNH suggests that eculizumab treatment in PNH may be associated with improved survival.</AbstractText><AbstractText Label="CONCLUSION" NlmCategory="CONCLUSIONS">A detailed analysis of clinical presentations and causes of death in patients with PNH, overall and by disease subcategories, provide evidence that in the current era, patients with haemolytic PNH are no longer those who harbour the worst prognosis. This finding differs sharply from what has been previously reported.</AbstractText><CopyrightInformation>© 2016 Royal Australasian College of Physicians.</CopyrightInformation></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Socié</LastName><ForeName>G</ForeName><Initials>G</Initials><AffiliationInfo><Affiliation>Hematology Transplantation, AP-HP, Hospital Saint Louis, University Paris VII Denis Diderot and Inserm UMR 1160, Paris, France. gerard.socie@sls.aphp.fr.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Schrezenmeier</LastName><ForeName>H</ForeName><Initials>H</Initials><AffiliationInfo><Affiliation>Institute of Transfusion Medicine, University of Ulm Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen and University Hospital Ulm, Ulm, Germany.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Muus</LastName><ForeName>P</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>Department of Hematology, Radboud UMC, Nijmegen, the Netherlands.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Lisukov</LastName><ForeName>I</ForeName><Initials>I</Initials><AffiliationInfo><Affiliation>Institution of Children Hematology and Transplantation, St. Petersburg, Russia.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Röth</LastName><ForeName>A</ForeName><Initials>A</Initials><AffiliationInfo><Affiliation>University Hospital Essen, Essen, Germany.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Kulasekararaj</LastName><ForeName>A</ForeName><Initials>A</Initials><AffiliationInfo><Affiliation>Kings College Hospital, London, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Lee</LastName><ForeName>J W</ForeName><Initials>JW</Initials><AffiliationInfo><Affiliation>Seoul St. Mary's Hospital, Seoul, Korea.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Araten</LastName><ForeName>D</ForeName><Initials>D</Initials><AffiliationInfo><Affiliation>Laura and Isaac Perlmutter Cancer Center, NYU Langone Medical Center, New York VA Medical Center, USA.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Hill</LastName><ForeName>A</ForeName><Initials>A</Initials><AffiliationInfo><Affiliation>St James's University Hospital, Leeds, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Brodsky</LastName><ForeName>R</ForeName><Initials>R</Initials><AffiliationInfo><Affiliation>Johns Hopkins University Medical Center, USA.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Urbano-Ispizua</LastName><ForeName>A</ForeName><Initials>A</Initials><AffiliationInfo><Affiliation>Hospital Clínic Barcelona, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Szer</LastName><ForeName>J</ForeName><Initials>J</Initials><AffiliationInfo><Affiliation>Royal Melbourne Hospital, Melbourne, Victoria, Australia.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Wilson</LastName><ForeName>A</ForeName><Initials>A</Initials><AffiliationInfo><Affiliation>Alexion Pharmaceuticals, New Haven, Connecticut, USA.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Hillmen</LastName><ForeName>P</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>St James's University Hospital, Leeds, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><CollectiveName>PNH Registry</CollectiveName></Author></AuthorList><Language>eng</Language><PublicationTypeList><PublicationType UI="D016428">Journal Article</PublicationType></PublicationTypeList></Article><MedlineJournalInfo><Country>Australia</Country><MedlineTA>Intern Med J</MedlineTA><NlmUniqueID>101092952</NlmUniqueID><ISSNLinking>1444-0903</ISSNLinking></MedlineJournalInfo><ChemicalList><Chemical><RegistryNumber>0</RegistryNumber><NameOfSubstance UI="D061067">Antibodies, Monoclonal, Humanized</NameOfSubstance></Chemical><Chemical><RegistryNumber>A3ULP0F556</RegistryNumber><NameOfSubstance UI="C481642">eculizumab</NameOfSubstance></Chemical></ChemicalList><CitationSubset>IM</CitationSubset><MeshHeadingList><MeshHeading><DescriptorName UI="D000328" MajorTopicYN="N">Adult</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D000741" MajorTopicYN="N">Anemia, Aplastic</DescriptorName><QualifierName UI="Q000453" MajorTopicYN="Y">epidemiology</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D061067" MajorTopicYN="N">Antibodies, Monoclonal, Humanized</DescriptorName><QualifierName UI="Q000627" MajorTopicYN="Y">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D002423" MajorTopicYN="N">Cause of Death</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D017707" MajorTopicYN="N">Erythrocyte Transfusion</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D005260" MajorTopicYN="N">Female</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D005602" MajorTopicYN="N">France</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D006457" MajorTopicYN="N">Hemoglobinuria, Paroxysmal</DescriptorName><QualifierName UI="Q000145" MajorTopicYN="N">classification</QualifierName><QualifierName UI="Q000401" MajorTopicYN="Y">mortality</QualifierName><QualifierName UI="Q000628" MajorTopicYN="Y">therapy</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D006801" MajorTopicYN="N">Humans</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D015994" MajorTopicYN="N">Incidence</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D008297" MajorTopicYN="N">Male</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D008875" MajorTopicYN="N">Middle Aged</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D015999" MajorTopicYN="N">Multivariate Analysis</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D011379" MajorTopicYN="N">Prognosis</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D012042" MajorTopicYN="N">Registries</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D012189" MajorTopicYN="N">Retrospective Studies</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D012307" MajorTopicYN="N">Risk Factors</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D016019" MajorTopicYN="N">Survival Analysis</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D013927" MajorTopicYN="N">Thrombosis</DescriptorName><QualifierName UI="Q000453" MajorTopicYN="Y">epidemiology</QualifierName></MeshHeading></MeshHeadingList><KeywordList Owner="NOTNLM"><Keyword MajorTopicYN="N">PNH</Keyword><Keyword MajorTopicYN="N">PNH Registry</Keyword><Keyword MajorTopicYN="N">causes of death</Keyword></KeywordList></MedlineCitation><PubmedData><History><PubMedPubDate PubStatus="received"><Year>2016</Year><Month>03</Month><Day>15</Day></PubMedPubDate><PubMedPubDate PubStatus="revised"><Year>2016</Year><Month>05</Month><Day>17</Day></PubMedPubDate><PubMedPubDate PubStatus="accepted"><Year>2016</Year><Month>05</Month><Day>29</Day></PubMedPubDate><PubMedPubDate PubStatus="entrez"><Year>2016</Year><Month>6</Month><Day>16</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="pubmed"><Year>2016</Year><Month>6</Month><Day>16</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="medline"><Year>2017</Year><Month>8</Month><Day>25</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate></History><PublicationStatus>ppublish</PublicationStatus><ArticleIdList><ArticleId IdType="pubmed">27305361</ArticleId><ArticleId IdType="doi">10.1111/imj.13160</ArticleId></ArticleIdList></PubmedData></pubmed><affiliations><list><country><li>Allemagne</li><li>Australie</li><li>Corée du Sud</li><li>Espagne</li><li>France</li><li>Pays-Bas</li><li>Royaume-Uni</li><li>Russie</li><li>États-Unis</li></country><region><li>Angleterre</li><li>Bade-Wurtemberg</li><li>Catalogne</li><li>Connecticut</li><li>District de Tübingen</li><li>Grand Londres</li><li>Gueldre</li><li>Région capitale de Séoul</li><li>Île-de-France</li></region><settlement><li>Barcelone</li><li>Londres</li><li>Nimègue</li><li>Paris</li><li>Séoul</li><li>Ulm</li></settlement></list><tree><country name="France"><region name="Île-de-France"><name sortKey="Socie, G" sort="Socie, G" uniqKey="Socie G" first="G" last="Socié">G. Socié</name></region></country><country name="Allemagne"><region name="Bade-Wurtemberg"><name sortKey="Schrezenmeier, H" sort="Schrezenmeier, H" uniqKey="Schrezenmeier H" first="H" last="Schrezenmeier">H. Schrezenmeier</name></region><name sortKey="Roth, A" sort="Roth, A" uniqKey="Roth A" first="A" last="Röth">A. Röth</name></country><country name="Pays-Bas"><region name="Gueldre"><name sortKey="Muus, P" sort="Muus, P" uniqKey="Muus P" first="P" last="Muus">P. Muus</name></region></country><country name="Russie"><noRegion><name sortKey="Lisukov, I" sort="Lisukov, I" uniqKey="Lisukov I" first="I" last="Lisukov">I. Lisukov</name></noRegion></country><country name="Royaume-Uni"><region name="Angleterre"><name sortKey="Kulasekararaj, A" sort="Kulasekararaj, A" uniqKey="Kulasekararaj A" first="A" last="Kulasekararaj">A. Kulasekararaj</name></region><name sortKey="Hill, A" sort="Hill, A" uniqKey="Hill A" first="A" last="Hill">A. Hill</name><name sortKey="Hillmen, P" sort="Hillmen, P" uniqKey="Hillmen P" first="P" last="Hillmen">P. Hillmen</name></country><country name="Corée du Sud"><region name="Région capitale de Séoul"><name sortKey="Lee, J W" sort="Lee, J W" uniqKey="Lee J" first="J W" last="Lee">J W Lee</name></region></country><country name="États-Unis"><noRegion><name sortKey="Araten, D" sort="Araten, D" uniqKey="Araten D" first="D" last="Araten">D. Araten</name></noRegion><name sortKey="Brodsky, R" sort="Brodsky, R" uniqKey="Brodsky R" first="R" last="Brodsky">R. Brodsky</name><name sortKey="Wilson, A" sort="Wilson, A" uniqKey="Wilson A" first="A" last="Wilson">A. Wilson</name></country><country name="Espagne"><region name="Catalogne"><name sortKey="Urbano Ispizua, A" sort="Urbano Ispizua, A" uniqKey="Urbano Ispizua A" first="A" last="Urbano-Ispizua">A. 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