Diagnosis and management of paroxysmal nocturnal hemoglobinuria.
Charles J. Parker,Mitsuhiro Omine,Stephen J. Richards,Junichi Nishimura,Monica Bessler,Russell E. Ware,Peter Hillmen,Lucio Luzzatto,Neal S. Young,Taroh Kinoshita,Wendell F. Rosse,Gérard Socié +11 more
TLDR
The primary clinical manifestations of paroxysmal nocturnal hemoglobinuria are hemolytic anemia, marrow failure, and thrombophilia.About:
This article is published in Blood.The article was published on 2005-12-01 and is currently open access. It has received 673 citations till now. The article focuses on the topics: Paroxysmal nocturnal hemoglobinuria & Eculizumab.read more
Citations
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Journal ArticleDOI
The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria.
Peter Hillmen,Neal S. Young,Jörg Schubert,Robert A. Brodsky,Gérard Socié,Petra Muus,Alexander Röth,Jeff Szer,Modupe O. Elebute,Ryotaro Nakamura,Paul Browne,Antonio M. Risitano,Anita Hill,Hubert Schrezenmeier,Chieh Lin Fu,Jaroslaw P. Maciejewski,Scott A. Rollins,Christopher F. Mojcik,Russell P. Rother,Lucio Luzzatto +19 more
TL;DR: Eculizumab is an effective therapy for PNH and Clinically significant improvements were found in the quality of life, as measured by scores on the Functional Assessment of Chronic Illness Therapy-Fatigue instrument and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire.
Journal ArticleDOI
Glycosylation in health and disease.
TL;DR: The broad role of glycans in immunity, cancer, xenotransplantation and glomerular filtration and the potential of ‘glycomedicine’ are discussed.
Journal ArticleDOI
Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria
TL;DR: The recent approval of eculizumab as a first-in-class complement inhibitor for the treatment of PNH validates the concept of complement inhibition as an effective therapy and provides rationale for investigation of other indications in which complement plays a role.
Journal ArticleDOI
Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria.
Robert A. Brodsky,Neal S. Young,Elisabetta Antonioli,Antonio M. Risitano,Hubert Schrezenmeier,Jörg Schubert,Anna Gaya,Luke Coyle,Carlos M. de Castro,Chieh Lin Fu,Jaroslaw P. Maciejewski,Monica Bessler,Henk André Kroon,Russell P. Rother,Peter Hillmen +14 more
TL;DR: The beneficial effects of eculizumab treatment in patients with PNH are applicable to a broader population of PNH patients than previously studied and were independent of baseline levels of hemolysis and degree of thrombocytopenia.
Journal ArticleDOI
Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria.
Peter Hillmen,Petra Muus,Ulrich Dührsen,Antonio M. Risitano,Jörg Schubert,Lucio Luzzatto,Hubert Schrezenmeier,Jeff Szer,Robert A. Brodsky,Anita Hill,Gérard Socié,Monica Bessler,Scott A. Rollins,Leonard Bell,Russell P. Rother,Neal S. Young +15 more
TL;DR: Results show that eculizumab treatment reduces the risk of clinical thromboembolism in patients with PNH.
References
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High-Resolution Genotyping by Amplicon Melting Analysis Using LCGreen
TL;DR: High-resolution melting analysis of PCR products amplified in the presence of LCGreen can identify both heterozygous and homozygous sequence variants, and is a promising method for mutation screening.
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Wintrobe's clinical hematology.
TL;DR: Wintrobe's clinical hematology is described in more detail in the book Wintrobe: The Anatomy of a Machine, 2nd Ed.
Journal ArticleDOI
Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria.
Junji Takeda,Toshio Miyata,Kazuyoshi Kawagoe,Yoshiyasu Iida,Yoshiyasu Iida,Yuichi Endo,Teizo Fujita,Minoru Takahashi,Teruo Kitani,Taroh Kinoshita +9 more
TL;DR: It is reported that PIG-A, which participates in the early step of GPI anchor biosynthesis, is the gene responsible for paroxysmal nocturnal hemoglobinuria.
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Hematopoietic Cell Transplantation
TL;DR: Hematopoietic cell transplantation is the IV infusion of hematopoetic stem and progenitor cells designed to establish marrow and immune function in patients with a variety of acquired and inherited malignant and nonmalignant disorders.
Journal ArticleDOI
Natural history of paroxysmal nocturnal hemoglobinuria.
TL;DR: PNH is a chronic disorder that curtails life, and a spontaneous long-term remission can occur, which must be taken into account when considering potentially dangerous treatments, such as bone marrow transplantation.
Related Papers (5)
The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria.
Peter Hillmen,Neal S. Young,Jörg Schubert,Robert A. Brodsky,Gérard Socié,Petra Muus,Alexander Röth,Jeff Szer,Modupe O. Elebute,Ryotaro Nakamura,Paul Browne,Antonio M. Risitano,Anita Hill,Hubert Schrezenmeier,Chieh Lin Fu,Jaroslaw P. Maciejewski,Scott A. Rollins,Christopher F. Mojcik,Russell P. Rother,Lucio Luzzatto +19 more