Results 31 to 40 of about 13,066 (196)

COVID‐19 infection presenting as paroxysmal nocturnal hemoglobinuria

Clinical Case Reports, 2021
A diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) was elicited during acute COVID‐19 infection. COVID‐19 spike proteins trigger the alternative pathway of complement. Acute SARS‐CoV‐2 infection possibly expanded an existing PIG‐A mutation.
Adam Hines, Nausheen Hakim, Jacqueline Barrientos   +2 more
doaj   +1 more source

Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency [PDF]

, 2018
Purpose: Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining ...
Ionova, Tatyana, Johns, Jeffrey, Oliva, Esther Natalie, Salek, Mir-Saeed Shayegan   +3 more
core   +2 more sources

PAROXYSMAL HEMOGLOBINURIA [PDF]

Journal of the American Medical Association, 1910
History. —The patient, a woman of French extraction, aged 30, had lived in New England for the past ten years. She had been always well and strong up to the time of the present illness. There was no history of syphilis or malaria. In the fall of 1905 after exposure to cold she was seized with a severe chill which lasted one hour.
openaire   +2 more sources

Paroxysmal nocturnal hemoglobinuria and pregnancy before the eculizumab era: the French experience

Haematologica, 2011
Background Pregnancy in women with paroxysmal nocturnal hemoglobinuria is rare, with few reports on maternal and fetal mortality rates.Design and Methods A specific questionnaire designed to solicit data on pregnancies in women with paroxysmal nocturnal ...
Sophie de Guibert, Régis Peffault de Latour, Nathalie Varoqueaux, Hélène Labussière, Bernard Rio, Dominique Jaulmes, Jean-Richard Eveillard, Stéphanie Dulucq, Anne-Marie Stoppa, Didier Bouscary, François Girodon, Bernard Bonnotte, Djamila Laskri, Gérard Socié, Thierry Lamy   +14 more
doaj   +1 more source

Renal hemosiderosis in paroxysmal nocturnal hemoglobinuria

Revista de Medicina da UFC, 2022
A previously healthy 38-year-old man presented with history of fever, adynamia and anemia for the last 5 months. Physical examination revealed pallor. Laboratory was marked by a hemoglobin level of 9.2g per deciliter (reference range, 14.0 to 18.0 g per ...
Italo Gustavo Lima Monteiro, Gabriela Studart Galdino, Raimundo Noberto de Lima Neto   +2 more
doaj   +1 more source

Thrombolytic therapy is effective in paroxysmal nocturnal hemoglobinuria: a series of nine patients and a review of the literature

Haematologica, 2012
Background Thrombosis is the major risk factor for death in patients with paroxysmal nocturnal hemoglobinuria. Previous case reports indicate that venous thrombosis in patients with paroxysmal nocturnal hemoglobinuria is amenable to thrombolysis.Design ...
David J. Araten, Rosario Notaro, Howard T Thaler, Nancy Kernan, Farid Boulad, Hugo Castro-Malaspina, Trudy Small, Andromachi Scaradavou, Heather Magnan, Susan Prockop, Sara Chaffee, Jason Gonsky, Raymond Thertulien, Roberto Tarquini, Lucio Luzzatto   +14 more
doaj   +1 more source

Stratification of responders towards eculizumab using a structural epitope mapping strategy [PDF]

, 2016
The complement component 5 (C5)-binding antibody eculizumab is used to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical haemolytic uremic syndrome (aHUS).
Berglund, Magnus M., Hu, Francis Jingxin, Nordling, Erik, Rockberg, Johan, Strömberg, Patrik, Uhlén, Mathias, Volk, Anna-Luisa   +6 more
core   +1 more source

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

eJHaem
Background Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive ...
Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour   +8 more
doaj   +1 more source

Polymorphism of the complement receptor 1 gene correlates with the hematologic response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria

Haematologica, 2014
Complement blockade by eculizumab is clinically effective in hemolytic paroxysmal nocturnal hemoglobinuria. However, the response is variable and some patients remain dependent on red blood cell transfusions.
Tommaso Rondelli, Antonio M. Risitano, Régis Peffault de Latour, Michela Sica, Benedetta Peruzzi, Patrizia Ricci, Wilma Barcellini, Anna Paola Iori, Carla Boschetti, Veronica Valle, Veronique Frémeaux-Bacchi, Maria De Angioletti, Gerard Socie, Lucio Luzzatto, Rosario Notaro   +14 more
doaj   +1 more source

Severity of effect considerations regarding the use of mutation as a toxicological endpoint for risk assessment: A report from the 8th International Workshop on Genotoxicity Testing (IWGT)

Environmental and Molecular Mutagenesis, EarlyView.
Abstract Exposure levels without appreciable human health risk may be determined by dividing a point of departure on a dose–response curve (e.g., benchmark dose) by a composite adjustment factor (AF). An “effect severity” AF (ESAF) is employed in some regulatory contexts.
Barbara L. Parsons, Marc A. Beal, Kerry L. Dearfield, George R. Douglas, Min Gi, B. Bhaskar Gollapudi, Robert H. Heflich, Katsuyoshi Horibata, Michelle Kenyon, Alexandra S. Long, David P. Lovell, Anthony M. Lynch, Meagan B. Myers, Stefan Pfuhler, Alisa Vespa, Andreas Zeller, George E. Johnson, Paul A. White   +17 more
wiley   +1 more source