Un diagnostic est établi par l'analyse d'urine et des tests sanguins pour détecter l'hémoglobine libre.
HémoglobinurieAnémie hémolytique
#2
Quels tests sont utilisés pour confirmer le diagnostic ?
Les tests incluent l'examen microscopique des urines et des tests de Coombs.
Analyse d'urineTest de Coombs
#3
Quels signes indiquent une hémoglobinurie paroxystique ?
Des urines de couleur rougeâtre, fatigue, et signes d'anémie peuvent indiquer cette condition.
AnémieUrine
#4
L'hémoglobinurie paroxystique est-elle toujours visible ?
Non, elle peut être intermittente et ne pas toujours se manifester par des urines colorées.
HémoglobinurieSymptômes
#5
Quel rôle joue l'historique médical dans le diagnostic ?
L'historique médical aide à identifier des causes sous-jacentes comme des infections ou des médicaments.
Antécédents médicauxInfections
Symptômes
5
#1
Quels sont les symptômes principaux de l'hémoglobinurie paroxystique ?
Les symptômes incluent des urines foncées, fatigue, et douleurs abdominales.
SymptômesUrine
#2
L'hémoglobinurie paroxystique cause-t-elle des douleurs ?
Oui, des douleurs abdominales ou lombaires peuvent survenir en raison de l'hémolyse.
Douleur abdominaleHémolyse
#3
Peut-on avoir de la fièvre avec cette condition ?
Oui, une fièvre peut être présente, surtout si une infection est la cause sous-jacente.
FièvreInfection
#4
Les symptômes varient-ils d'une personne à l'autre ?
Oui, l'intensité et la fréquence des symptômes peuvent varier selon les individus.
VariabilitéSymptômes
#5
Y a-t-il des symptômes associés à l'anémie ?
Oui, des symptômes comme la pâleur, la faiblesse et des palpitations peuvent apparaître.
AnémieSymptômes
Prévention
5
#1
Comment prévenir l'hémoglobinurie paroxystique ?
Éviter les facteurs déclenchants comme certains médicaments et infections peut aider.
PréventionMédicaments
#2
Les vaccinations sont-elles utiles pour prévenir cette condition ?
Oui, les vaccinations contre certaines infections peuvent réduire le risque d'hémoglobinurie.
VaccinationInfection
#3
Faut-il surveiller les patients à risque ?
Oui, une surveillance régulière est recommandée pour les patients ayant des antécédents d'hémolyse.
SurveillanceHémolyse
#4
L'hydratation joue-t-elle un rôle préventif ?
Oui, une bonne hydratation peut aider à prévenir la concentration d'hémoglobine dans les urines.
HydratationPrévention
#5
Les changements alimentaires peuvent-ils aider ?
Une alimentation équilibrée peut soutenir la santé globale et réduire les risques d'hémolyse.
AlimentationSanté
Traitements
5
#1
Quel est le traitement principal de l'hémoglobinurie paroxystique ?
Le traitement vise à traiter la cause sous-jacente et peut inclure des transfusions sanguines.
Transfusion sanguineTraitement
#2
Des médicaments sont-ils utilisés pour traiter cette condition ?
Oui, des corticostéroïdes peuvent être prescrits pour réduire l'hémolyse dans certains cas.
CorticostéroïdesHémolyse
#3
Comment gérer les épisodes aigus d'hémoglobinurie ?
L'hydratation et le repos sont essentiels pour gérer les épisodes aigus et prévenir la déshydratation.
HydratationRepos
#4
Les transfusions sont-elles toujours nécessaires ?
Non, les transfusions ne sont nécessaires que si l'anémie est sévère ou symptomatique.
Transfusion sanguineAnémie
#5
Y a-t-il des traitements préventifs disponibles ?
Éviter les déclencheurs connus et traiter les infections peut aider à prévenir les épisodes.
PréventionInfection
Complications
5
#1
Quelles complications peuvent survenir avec l'hémoglobinurie paroxystique ?
Des complications incluent l'insuffisance rénale et des troubles électrolytiques.
Insuffisance rénaleÉlectrolytes
#2
L'hémoglobinurie peut-elle entraîner des problèmes rénaux ?
Oui, l'excès d'hémoglobine peut endommager les reins et entraîner une insuffisance rénale.
Insuffisance rénaleHémoglobinurie
#3
Y a-t-il un risque de thrombose ?
Oui, l'hémolyse peut augmenter le risque de thrombose dans certains cas.
ThromboseHémolyse
#4
Les complications sont-elles réversibles ?
Certaines complications peuvent être réversibles si traitées rapidement et efficacement.
RéversibilitéTraitement
#5
Comment surveiller les complications potentielles ?
Des tests sanguins réguliers et des analyses d'urine aident à surveiller les complications.
SurveillanceAnalyse d'urine
Facteurs de risque
5
#1
Quels sont les facteurs de risque de l'hémoglobinurie paroxystique ?
Les facteurs incluent des infections, des maladies auto-immunes et certains médicaments.
Facteurs de risqueInfections
#2
Les antécédents familiaux jouent-ils un rôle ?
Oui, des antécédents familiaux d'anémie hémolytique peuvent augmenter le risque.
Antécédents familiauxAnémie hémolytique
#3
Les personnes âgées sont-elles plus à risque ?
Oui, les personnes âgées peuvent être plus susceptibles en raison de maladies sous-jacentes.
Personnes âgéesMaladies sous-jacentes
#4
Les infections virales augmentent-elles le risque ?
Oui, certaines infections virales peuvent déclencher des épisodes d'hémoglobinurie.
Infections viralesHémoglobinurie
#5
Les médicaments peuvent-ils être un facteur de risque ?
Oui, certains médicaments, comme les antibiotiques, peuvent provoquer une hémolyse.
MédicamentsHémolyse
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"acceptedAnswer": {
"@type": "Answer",
"text": "Le traitement vise à traiter la cause sous-jacente et peut inclure des transfusions sanguines."
}
},
{
"@type": "Question",
"name": "Des médicaments sont-ils utilisés pour traiter cette condition ?",
"position": 17,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des corticostéroïdes peuvent être prescrits pour réduire l'hémolyse dans certains cas."
}
},
{
"@type": "Question",
"name": "Comment gérer les épisodes aigus d'hémoglobinurie ?",
"position": 18,
"acceptedAnswer": {
"@type": "Answer",
"text": "L'hydratation et le repos sont essentiels pour gérer les épisodes aigus et prévenir la déshydratation."
}
},
{
"@type": "Question",
"name": "Les transfusions sont-elles toujours nécessaires ?",
"position": 19,
"acceptedAnswer": {
"@type": "Answer",
"text": "Non, les transfusions ne sont nécessaires que si l'anémie est sévère ou symptomatique."
}
},
{
"@type": "Question",
"name": "Y a-t-il des traitements préventifs disponibles ?",
"position": 20,
"acceptedAnswer": {
"@type": "Answer",
"text": "Éviter les déclencheurs connus et traiter les infections peut aider à prévenir les épisodes."
}
},
{
"@type": "Question",
"name": "Quelles complications peuvent survenir avec l'hémoglobinurie paroxystique ?",
"position": 21,
"acceptedAnswer": {
"@type": "Answer",
"text": "Des complications incluent l'insuffisance rénale et des troubles électrolytiques."
}
},
{
"@type": "Question",
"name": "L'hémoglobinurie peut-elle entraîner des problèmes rénaux ?",
"position": 22,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, l'excès d'hémoglobine peut endommager les reins et entraîner une insuffisance rénale."
}
},
{
"@type": "Question",
"name": "Y a-t-il un risque de thrombose ?",
"position": 23,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, l'hémolyse peut augmenter le risque de thrombose dans certains cas."
}
},
{
"@type": "Question",
"name": "Les complications sont-elles réversibles ?",
"position": 24,
"acceptedAnswer": {
"@type": "Answer",
"text": "Certaines complications peuvent être réversibles si traitées rapidement et efficacement."
}
},
{
"@type": "Question",
"name": "Comment surveiller les complications potentielles ?",
"position": 25,
"acceptedAnswer": {
"@type": "Answer",
"text": "Des tests sanguins réguliers et des analyses d'urine aident à surveiller les complications."
}
},
{
"@type": "Question",
"name": "Quels sont les facteurs de risque de l'hémoglobinurie paroxystique ?",
"position": 26,
"acceptedAnswer": {
"@type": "Answer",
"text": "Les facteurs incluent des infections, des maladies auto-immunes et certains médicaments."
}
},
{
"@type": "Question",
"name": "Les antécédents familiaux jouent-ils un rôle ?",
"position": 27,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, des antécédents familiaux d'anémie hémolytique peuvent augmenter le risque."
}
},
{
"@type": "Question",
"name": "Les personnes âgées sont-elles plus à risque ?",
"position": 28,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, les personnes âgées peuvent être plus susceptibles en raison de maladies sous-jacentes."
}
},
{
"@type": "Question",
"name": "Les infections virales augmentent-elles le risque ?",
"position": 29,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, certaines infections virales peuvent déclencher des épisodes d'hémoglobinurie."
}
},
{
"@type": "Question",
"name": "Les médicaments peuvent-ils être un facteur de risque ?",
"position": 30,
"acceptedAnswer": {
"@type": "Answer",
"text": "Oui, certains médicaments, comme les antibiotiques, peuvent provoquer une hémolyse."
}
}
]
}
]
}
Institute of Transfusion Medicine, University of Ulm, Ulm, Germany.
Institute of Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Blood Transfusion Service, Baden-Württemberg-Hessen and University Hospital of Ulm, Ulm, Germany.
From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).
Institute of Transfusion Medicine, University of Ulm, Ulm, Germany.
Institute of Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Blood Transfusion Service, Baden-Württemberg-Hessen and University Hospital of Ulm, Ulm, Germany.
From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).
From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).
From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).
From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).
From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).
From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) - all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen (A.R., F.A.), the Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, University Hospital RWTH Aachen, and the Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf, Aachen (J.P.), the University of Ulm, the Institute of Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service Baden-Württemberg-Hessen, and University Hospital Ulm, Ulm (H.S., B. Höchsmann), the Department of Oncology, Hematology, and Bone Marrow Transplantation, Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg (P. Schafhausen), and Elblandklinikum Riesa, Riesa (J.S.) - all in Germany; King's College Hospital NHS Foundation Trust (A.G.K., S.G., R.T.), the National Institute for Health and Care Research and Wellcome King's Clinical Research Facility (A.G.K.), King's College London (A.G.K.), and Novartis Pharmaceuticals (C.T.), London, and St. James's University Hospital, Leeds (M.G., R.J.K.) - all in the United Kingdom; Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and Peking Union Medical College, Beijing (B. Han, C.Y.), the Department of Hematology, Tianjin Medical University General Hospital (R.F., H.L.), and the Department of Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (L.Z.), Tianjin, and China Novartis Institutes for BioMedical Research, Shanghai (Z.W., S.L.) - all in China; the Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo (P. Scheinberg), and ABC Medical School, Santo André (V.A.Q.M.) - both in Brazil; the Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland (J.P.M.); Osaka University Graduate School of Medicine, Suita (Y.U.), Kyoto University Hospital, Kyoto (T.K.), and Japanese Red Cross Society Suwa Hospital, Suwa (M.U.) - all in Japan; Duke University School of Medicine and Duke Cancer Institute, Durham, NC (C.M.C.); Unità Operativa Complessa Oncoematologia, AULSS7 Pedemontana, Bassano del Grappa (E.D.B.), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.), Division of Hematology, University of Turin, Città della Salute e della Scienza, Turin (E.B.), Instituto per lo Studio, la Prevenzione e la Rete Oncologica, and Azienda Ospedaliero-Universitaria Careggi, Florence (R.N.), AORN Moscati, Avellino (L.M., A.M.R.), and University of Naples Federico II, Naples (A.M.R.) - all in Italy; Radboud University Medical Center, Nijmegen, the Netherlands (S.M.C.L.); the Hematology Unit, Department of Medicine, Hospital Umum Sarawak, Kuching (L.P.C.), and the Hematology Unit, Queen Elizabeth Hospital, Kota Kinabalu (L.W.L.L.) - both in Malaysia; Hospital Clinic of Barcelona (A.G.) and the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (R.P.L., A.G.K., M.G., W.B., S.T., L.T., F.S.F., L.M., A.M.R.) - both in Barcelona; the Department of Hematology and Oncology and the Department of Clinical Pathology, Hualien Tzu Chi Hospital, the Buddhist Tzu Chi Medical Foundation, and the Institute of Medical Sciences, Tzu Chi University, Hualien, Taiwan (W.-H.H.); the Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (J.H.J.); Medical College of Georgia, Augusta (A.K.); City of Hope Medical Center, Duarte, CA (V.P.); the Department of Laboratory Medicine, National University Hospital, Singapore (E.-S.Y.); Novartis Pharma (C.K., R.L., M.D.) and Novartis BioMedical Research (A.V.) - both in Basel, Switzerland; and Novartis Healthcare Private, Hyderabad, India (P.B., R.K., S.M.).
From the Department of Haematology, St. James's University Hospital, Leeds (P.H., M.G.), and Lisa Tan Pharma Consulting, Cambridge (L.T.) - both in the United Kingdom; the Department of Clinical Haematology, Peter MacCallum Cancer Center and Royal Melbourne Hospital, Melbourne, VIC, Australia (J.S.); Jane Anne Nohl Division of Hematology, Keck School of Medicine of USC, Los Angeles (I.W.); the Department of Hematology, West German Cancer Center University Hospital Essen, University of Duisburg-Essen, Essen (A. Röth), the Institute of Transfusion Medicine, University of Ulm and Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and University Hospital Ulm, Ulm (B.H.), and the Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen (J.P.) - all in Germany; the Department of Hematology, NTT Medical Center Tokyo, Tokyo (K.U.), and the Department of Hematology and Oncology, Okayama University Hospital, Okayama (H.N.) - both in Japan; Centre d'Investigations Cliniques (J.-J.K.) and the French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (R.P.T.), Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris; the Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University, Durham, NC (C.C.); Apellis Pharmaceuticals, Waltham, MA (M.H., P.D., C.F., F.G., T.A.); and the Hematology and BMT Unit, AORN San Giuseppe Moscati, Avellino, Italy (A. Risitano).
Division of Hematology, Department of Internal Medicine, Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea. jwlee@catholic.ac.kr.
Paroxysmal cold hemoglobinuria (PCH) is a rare condition in childhood characterized by complement-mediated premature destruction of red blood cells. PCH is associated with intravascular hemolysis caus...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder, leading to various complications and impairments in patients' health-related quality of life (HRQOL). Limited research has been cond...
A cross-sectional study was conducted during 2022 to recruit patients with PNH in China. The study population was recruited from PNH China, one of the largest public welfare PNH patient mutual aid org...
A total of 329 valid questionnaires were collected. The mean (SD) age of the patients was 35.3 (10.0) years, with 52.3% of them being male. The patients reported more problems in Anxiety/Depression (8...
Our study manifested the HRQOL of PNH patients in China was markedly compromised, especially in two mental-health related dimensions, and revealed several socio-demographic and clinical factors of the...
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell disorder characterized by PIG-A mutations, leading to glycophosphatidylinositol (GPI)-anchored proteins deficiency that...
Out of 2790 first-time screened individuals, the presence of PNH clone in neutrophils was detected in 322 patients, including 49 children and 273 adults. Annual incidence was stable at a median of 31 ...
A significant underrepresentation of hemolytic patients was observed as compared to the published cohorts suggesting that these patients are missed in diagnostic process and classic PNH remains underd...
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated hemolysis and potentially life-threatening complications. Pegcetacoplan, an inhibitor of complement com...
Pegcetacoplan is a targeted complement component 3 (C3) therapy approved for adults with paroxysmal nocturnal hemoglobinuria (PNH; US) or PNH plus anemia despite C5-targeted therapy for ≥ 3 months (EU...
Patients from PEGASUS enrolled in the 307 OLE continued pegcetacoplan subcutaneous self-administration twice or three times weekly or every 3 days for an additional 48 weeks. ISRs were coded as advers...
As of August 27, 2021, 58/64 patients from PEGASUS completed an additional 48 weeks of treatment in the 307 OLE (median treatment duration 337.0 [range 55-344] days); 95.3% (61/64) of patients achieve...
Though ISRs were common, most were mild, and the percentage of patients reporting ISRs declined from PEGASUS through the 307 OLE. Patient compliance remained high, and no patients discontinued because...
ClinicalTrials.gov identifiers: NCT03500549 (PEGASUS) and NCT03531255 (307 OLE)....
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder characterized by uncontrolled terminal complement activation. Eculizumab, a monoclonal antibody C5 inhibitor was introduced in ...
This study assessed the long-term efficacy/safety of eculizumab in PNH using real-world data from the Korean Health Insurance Review and Assessment Service. Eighty patients who initiated eculizumab fr...
At eculizumab initiation, the median age was 51.5 years, lactate dehydrogenase (LDH) 6.8 × upper limit of normal, and granulocyte clone size 93.0%. All patients had at least one PNH-related complicati...
These data provided evidence for the long-term efficacy and safety of eculizumab in Korean PNH patients with high disease burdens....
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder. Symptoms such as fatigue can have a substantial impact on patients' physical activity levels, sleep, quality of life, and w...
REVEAL was a 32-week prospective observational cohort study in individuals with PNH receiving ravulizumab in the USA. A wrist-worn Fitbit™ collected data on resting heart rate, daily step count, and s...
Twenty-eight ravulizumab-treated patients were included (median age: 34 years; 54% female). PRO scores were within US general population normative values, including FACIT-Fatigue (40.0), PROMIS Global...
This was the first study to use digital monitoring technology to collect data on physical activity and sleep in patients with PNH. The findings indicate that ravulizumab treatment enables patients wit...
Although complement component 5 inhibitors (C5is) eculizumab and ravulizumab improve paroxysmal nocturnal hemoglobinuria (PNH) outcomes, patients may experience persistent anemia. This post hoc analys...
Patients with PNH and hemoglobin ≥10.0 g/dL at baseline of PADDOCK (N = 6), PRINCE (N = 8), and PEGASUS (N = 11) were included. Before receiving pegcetacoplan, PADDOCK and PRINCE patients were C5i-nai...
From baseline to week 16, mean (SD) hemoglobin concentrations increased in C5i-naive patients (PADDOCK: 10.5 [0.4] to 12.7 [1.1] g/dL; PRINCE: 11.3 [1.0] to 14.0 [1.3] g/dL) and those with suboptimal ...
Patients with PNH and mild/moderate anemia who were C5i-naive or who had suboptimal hemoglobin concentrations despite eculizumab treatment had improved hematologic outcomes and reduced fatigue after i...
Trial registration numbers: PADDOCK (NCT02588833), PRINCE (NCT04085601; EudraCT, 2018-004220-11), PEGASUS (NCT03500549)....
Standard-of-care first-line treatments for paroxysmal nocturnal hemoglobinuria (PNH) include the anti-C5 therapies eculizumab and ravulizumab. However, persistent anemia, likely due to extravascular h...
In patients previously treated with anti-C5 therapies or naive to complement inhibitors, iptacopan 200 mg twice daily resulted in clinically meaningful results in the pivotal phase 3 APPLY-PNH (NCT045...
APPLY-PNH and APPOINT-PNH reported clinically meaningful improvements in hemoglobin, bilirubin, and lactate dehydrogenase levels; transfusion avoidance; reticulocyte count; and fatigue. Iptacopan's sa...
The elevated mortality risk among patients with paroxysmal nocturnal hemoglobinuria (PNH) has been suggested to derive from a high risk of thromboembolism (TE); however, the risks of coexisting cardio...
Patients with PNH (n=115) were identified in the 1977-2016 Danish National Patient Register (DNPR). For each patient with PNH, we identified 50 age- and sex-matched general population comparators. Usi...
One-year survival among patients and comparators was 92.2% and 99.4%, and after 10 years, it was 68.4% and 85.8%, respectively. Early mortality was associated with older age, higher levels of comorbid...
We conclude that early mortality in PNH is associated with older age, cardiovascular comorbidity, and hematological malignancies....
