Results 31 to 40 of about 15,613 (254)

Paroxysmal Nocturnal Hemoglobinuria [PDF]

Blood, 1956
Abstract 1. A fatal case of P.N.H. in a woman of 65 is presented. 2. The subject of P.N.H. is briefly discussed, and 3. The involvement of the bone marrow in the disease process is stressed.
A. M. NUSSEY, D. W. DAWSON
openaire   +1 more source

Crovalimab in the paroxysmal nocturnal hemoglobinuria treatment landscape. [PDF]

Immunotherapy
Röth A, Kulasekararaj AG, Scheinberg P, Nishimura JI.   +3 more
europepmc   +3 more sources

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

Technical advances in flow cytometry-based diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria

Einstein (São Paulo)
Objective: To discuss the implementation of technical advances in laboratory diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria for validation of high-sensitivity flow cytometry protocols. Methods: A retrospective study based on analysis of
Rodolfo Patussi Correia, Laiz Cameirão Bento, Ana Carolina Apelle Bortolucci, Anderson Marega Alexandre, Andressa da Costa Vaz, Daniela Schimidell, Eduardo de Carvalho Pedro, Fabricio Simões Perin, Sonia Tsukasa Nozawa, Cláudio Ernesto Albers Mendes, Rodrigo de Souza Barroso, Nydia Strachman Bacal   +11 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

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

Aplastic Crisis as Primary Manifestation of Systemic Lupus Erythematosus [PDF]

, 2011
Aplastic crisis is an unusual feature of systemic lupus erythematosus (SLE). We report the case of a 54-year-old woman presenting with both (extravascular) Coombs-positive hemolytic anemia and laboratory findings of bone marrow hyporegeneration with ...
Alger M, Andreas Völkl, Bailey FA, Brooks, Chute JP, Espinoza JL, Feng X, Fuat S. Oduncu, Hinterberger-Fischer M, Hirano N, Hirano N, Mestanza-Peralta M, Michael Bäuerle, Pavithran K, Philipp Baumann, Ralf Schmidmaier, Singh NP, Tagoe C, Takamatsu H, Tan EM, Torok-Storb B, Wolach B, Young NS, Young NS   +23 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

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

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