Paroxysmal nocturnal hemoglobinuria: pathophysiology, natural history and treatment options in the era of biological agents
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[1] S. Craigo,et al. Managing a pregnant patient with paroxysmal nocturnal hemoglobinuria in the era of eculizumab. , 2010, Leukemia research.
[2] B. Murtaza,et al. Paroxysmal nocturnal hemoglobinuria. , 2008, Journal of the College of Physicians and Surgeons--Pakistan : JCPSP.
[3] N. Young,et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. , 2008, Blood.
[4] A. Risitano,et al. 16: Long-Term Results of a Gitmo Retrospective Study on Hematopoietic Stem Cell Transplantation (HSCT) for Paroxysmal Nocturnal Hemoglobinuria (PNH) , 2008 .
[5] A. Zanella,et al. Paroxysmal nocturnal hemoglobinuria (PNH) in the eculizumab era: The bedside and beyond. , 2008 .
[6] N. Young,et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. , 2007, Blood.
[7] R. Brodsky,et al. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria , 2007, Nature Biotechnology.
[8] Lucio Luzzatto,et al. Highly homologous T-cell receptor beta sequences support a common target for autoreactive T cells in most patients with paroxysmal nocturnal hemoglobinuria. , 2007, Blood.
[9] Hong Chang,et al. Diagnosing PNH with FLAER and multiparameter flow cytometry , 2007, Cytometry. Part B, Clinical cytometry.
[10] S. Richards,et al. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria , 2007, British journal of haematology.
[11] Y. Maeda,et al. Targeted therapy for inherited GPI deficiency. , 2007, The New England journal of medicine.
[12] S. Richards,et al. Erythropoietin treatment during complement inhibition with eculizumab in a patient with paroxysmal nocturnal hemoglobinuria , 2007, Haematologica.
[13] B. Rotoli,et al. Glycosyl‐phosphatidyl‐inositol‐defective granulocytes from paroxysmal nocturnal haemoglobinuria patients show increased bacterial ingestion but reduced respiratory burst induction , 2007, American journal of hematology.
[14] S. Richards,et al. Erythopoietin treatment during complement inhibition with eculizumab in a patient with paroxysmal nocturnal hemoglobinuria. , 2007, Haematologica.
[15] S. Christmas,et al. Levels of expression of complement regulatory proteins CD46, CD55 and CD59 on resting and activated human peripheral blood leucocytes , 2006, Immunology.
[16] M. Mohty,et al. Paroxysmal Nocturnal Hemoglobinuria (PNH): Long-Term Epidemiological Study. , 2006 .
[17] Jichun Chen,et al. Soluble Urokinase Plasminogen Activator Receptor Is Increased in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Thrombosis and Inhibits Plasmin Generation In Vitro and Promotes Thrombosis in the Mouse Model. , 2006 .
[18] N. Young,et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. , 2006, The New England journal of medicine.
[19] R. Houlston,et al. Hypomorphic promoter mutation in PIGM causes inherited glycosylphosphatidylinositol deficiency , 2006, Nature Medicine.
[20] G. Smith,et al. Protection of erythrocytes from human complement-mediated lysis by membrane-targeted recombinant soluble CD59: a new approach to PNH therapy. , 2006, Blood.
[21] Neal Young,et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. , 2005, Blood.
[22] L. Luzzatto,et al. Patients with paroxysmal nocturnal hemoglobinuria have a high frequency of peripheral-blood T cells expressing activating isoforms of inhibiting superfamily receptors. , 2005, Blood.
[23] S. Richards,et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. , 2005, Blood.
[24] N. Young,et al. Differential gene expression in hematopoietic progenitors from paroxysmal nocturnal hemoglobinuria patients reveals an apoptosis/immune response in ‘normal’ phenotype cells , 2005, Leukemia.
[25] M. Gladwin,et al. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. , 2005, JAMA.
[26] C. O'keefe,et al. Large granular lymphocyte (LGL)-like clonal expansions in paroxysmal nocturnal hemoglobinuria (PNH) patients , 2005, Leukemia.
[27] S. Piantadosi,et al. PIG-A mutations in normal hematopoiesis. , 2004, Blood.
[28] H. Audebert,et al. Cerebral ischemic infarction in paroxysmal nocturnal hemoglobinuria report of 2 cases and updated review of 7 previously published patients. , 2005, Journal of neurology.
[29] R. Rother,et al. Improvement in the symptoms of smooth muscle dystonia during eculizumab therapy in paroxysmal nocturnal hemoglobinuria. , 2005, Haematologica.
[30] N. Young,et al. In-vivo dominant immune responses in aplastic anaemia: molecular tracking of putatively pathogenetic T-cell clones by TCR β-CDR3 sequencing , 2004, The Lancet.
[31] E. S. Garrett,et al. Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays , 2004, British journal of haematology.
[32] S. Hall,et al. Clinical Course and Flow Cytometric Analysis of Paroxysmal Nocturnal Hemoglobinuria in the United States and Japan , 2004, Medicine.
[33] S. Neubauer,et al. Rapid Regulation of Platelet Activation In Vivo by Nitric Oxide , 2003, Circulation.
[34] C. O'keefe,et al. Shared and individual specificities of immunodominant cytotoxic T-cell clones in paroxysmal nocturnal hemoglobinuria as determined by molecular analysis. , 2004, Experimental hematology.
[35] S. Richards,et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. , 2004, The New England journal of medicine.
[36] S. Richards,et al. Primary prophylaxis with warfarin prevents thrombosis in paroxysmal nocturnal hemoglobinuria (PNH). , 2003, Blood.
[37] J. Maciejewski,et al. Application of the Molecular Analysis of the T-Cell Receptor Repertoire in the Study of Immune-Mediated Hematologic Diseases , 2003, Hematology.
[38] U. Jäger,et al. Fibrinolytic therapy with rt-PA in a patient with paroxysmal nocturnal hemoglobinuria and Budd-Chiari syndrome , 2003, Annals of Hematology.
[39] M. Okabe,et al. Inefficient response of T lymphocytes to glycosylphosphatidylinositol anchor-negative cells: implications for paroxysmal nocturnal hemoglobinuria. , 2002, Blood.
[40] L Luzzatto,et al. Dynamics of hematopoiesis in paroxysmal nocturnal hemoglobinuria (PNH): no evidence for intrinsic growth advantage of PNH clones , 2002, Leukemia.
[41] T. Roszman,et al. Lipid rafts, major histocompatibility complex molecules, and immune regulation. , 2002, Human immunology.
[42] D. Follmann,et al. Intracellular interferon-γ in circulating and marrow T cells detected by flow cytometry and the response to immunosuppressive therapy in patients with aplastic anemia , 2002 .
[43] H. Mitsuya,et al. Decreased susceptibility of leukemic cells with PIG-A mutation to natural killer cells in vitro. , 2002, Blood.
[44] N. Young,et al. Oligoclonal and polyclonal CD4 and CD8 lymphocytes in aplastic anemia and paroxysmal nocturnal hemoglobinuria measured by V beta CDR3 spectratyping and flow cytometry. , 2002, Blood.
[45] N. Young,et al. Changes in T-cell receptor VB repertoire in aplastic anemia: effects of different immunosuppressive regimens. , 2002, Blood.
[46] Y. Kanakura,et al. Long-term support of hematopoiesis by a single stem cell clone in patients with paroxysmal nocturnal hemoglobinuria. , 2002, Blood.
[47] V. Pistoia,et al. Interferon γ and tumour necrosis factor α are overexpressed in bone marrow T lymphocytes from paediatric patients with aplastic anaemia , 2001 .
[48] Y. Katayama,et al. Monitoring of CD59 expression in paroxysmal nocturnal hemoglobinuria treated with danazol , 2001, American journal of hematology.
[49] S. Jhanwar,et al. Cytogenetic and morphological abnormalities in paroxysmal nocturnal haemoglobinuria , 2001, British journal of haematology.
[50] S. Orkin,et al. GATA1-Cre mediates Piga gene inactivation in the erythroid/megakaryocytic lineage and leads to circulating red cells with a partial deficiency in glycosyl phosphatidylinositol-linked proteins (paroxysmal nocturnal hemoglobinuria type II cells). , 2001, Blood.
[51] P. Pandolfi,et al. Fes-Cre Targets Phosphatidylinositol Glycan Class a (Piga) Inactivation to Hematopoietic Stem Cells in the Bone Marrow , 2001, The Journal of experimental medicine.
[52] N. Young,et al. Limited heterogeneity of T cell receptor BV usage in aplastic anemia. , 2001, The Journal of clinical investigation.
[53] V. Pistoia,et al. Interferon gamma and tumour necrosis factor alpha are overexpressed in bone marrow T lymphocytes from paediatric patients with aplastic anaemia. , 2001, British journal of haematology.
[54] S. Orkin,et al. GATA 1-Cre mediates Piga gene inactivation in the erythroid / megakaryocytic lineage and leads to circulating red cells with a partial deficiency in glycosyl phosphatidylinositol – linked proteins ( paroxysmal nocturnal hemoglobinuria type II cells ) , 2001 .
[55] H. Thaler,et al. Abnormal T-cell repertoire is consistent with immune process underlying the pathogenesis of paroxysmal nocturnal hemoglobinuria. , 2000, Blood.
[56] Shiyong Li,et al. Improved detection and characterization of paroxysmal nocturnal hemoglobinuria using fluorescent aerolysin. , 2000, American journal of clinical pathology.
[57] N. Young,et al. Genetic and environmental effects in paroxysmal nocturnal hemoglobinuria: this little PIG-A goes "Why? Why? Why?". , 2000, The Journal of clinical investigation.
[58] S. Richards,et al. Application of flow cytometry to the diagnosis of paroxysmal nocturnal hemoglobinuria. , 2000, Cytometry.
[59] F. Benvenuto,et al. Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria. , 2000, Haematologica.
[60] L. del Vecchio,et al. Glycosyl phosphatidylinositol (GPI)-anchored molecules and the pathogenesis of paroxysmal nocturnal hemoglobinuria. , 2000, Critical reviews in oncology/hematology.
[61] J. Freyssinet,et al. Elevated levels of circulating procoagulant microparticles in patients with paroxysmal nocturnal hemoglobinuria and aplastic anemia. , 1999, Blood.
[62] Taroh Kinoshita,et al. Genotypic, immunophenotypic and clinical features of Thai patients with paroxysmal nocturnal haemoglobinuria , 1999, British journal of haematology.
[63] H. Esperou,et al. Bone marrow transplantation for paroxysmal nocturnal haemoglobinuria , 1999, British journal of haematology.
[64] L Luzzatto,et al. Clonal populations of hematopoietic cells with paroxysmal nocturnal hemoglobinuria genotype and phenotype are present in normal individuals. , 1999, Proceedings of the National Academy of Sciences of the United States of America.
[65] H. Noji,et al. Complement sensitivity of erythrocytes in a patient with inherited complete deficiency of CD59 or with the Inab phenotype , 1999, British journal of haematology.
[66] L. Luzzatto,et al. Bone marrow transplants for paroxysmal nocturnal haemoglobinuria , 1999, British journal of haematology.
[67] H. Deeg,et al. New somatic mutation in the PIG-A gene emerges at relapse of paroxysmal nocturnal hemoglobinuria. , 1998, Blood.
[68] P. Pandolfi,et al. Murine embryonic stem cells without pig-a gene activity are competent for hematopoiesis with the PNH phenotype but not for clonal expansion. , 1997, The Journal of clinical investigation.
[69] W. Zeng,et al. Isolation of a T-cell clone showing HLA-DRB1*0405-restricted cytotoxicity for hematopoietic cells in a patient with aplastic anemia. , 1997, Blood.
[70] N. Young,et al. Expression and modulation of cellular receptors for interferon‐γ, tumour necrosis factor, and Fas on human bone marrow CD34+ cells , 1997, British journal of haematology.
[71] W. Rosse,et al. The natural history of paroxysmal nocturnal hemoglobinuria. , 1997, Revista de investigacion clinica; organo del Hospital de Enfermedades de la Nutricion.
[72] Y. Hasegawa,et al. Excess soluble urokinase-type plasminogen activator receptor in the plasma of patients with paroxysmal nocturnal hemoglobinuria inhibits cell-associated fibrinolytic activity. , 1997, International journal of hematology.
[73] N. Young,et al. Impaired hematopoiesis in paroxysmal nocturnal hemoglobinuria/aplastic anemia is not associated with a selective proliferative defect in the glycosylphosphatidylinositol-anchored protein-deficient clone. , 1997, Blood.
[74] L. Horstman,et al. Danazol for paroxysmal nocturnal hemoglobinuria , 1997, American journal of hematology/oncology.
[75] Lucio Luzzatto,et al. Somatic Mutations in Paroxysmal Nocturnal Hemoglobinuria: A Blessing in Disguise? , 1997, Cell.
[76] L Luzzatto,et al. The dual pathogenesis of paroxysmal nocturnal hemoglobinuria , 1997, Current opinion in hematology.
[77] J. Mary,et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors , 1996, The Lancet.
[78] W. Rosse. Epidemiology of PNH , 1996, The Lancet.
[79] L. del Vecchio,et al. Blood cell flow cytometry in paroxysmal nocturnal hemoglobinuria: a tool for measuring the extent of the PNH clone. , 1996, Leukemia.
[80] R. Ware,et al. Molecular basis of the heterogeneity of expression of glycosyl phosphatidylinositol anchored proteins in paroxysmal nocturnal hemoglobinuria. , 1996, Blood.
[81] L. Luzzatto,et al. Serum erythropoietin levels in paroxysmal nocturnal haemoglobinuria: implications for therapy , 1996, British journal of haematology.
[82] S. B. Olsen,et al. Enhancement of platelet deposition by cross-linked hemoglobin in a rat carotid endarterectomy model. , 1996, Circulation.
[83] K. Nafa,et al. Factor V Leiden mutation investigated by amplification created restriction enzyme site (ACRES) in PNH patients with and without thrombosis. , 1996, Haematologica.
[84] H. Heimpel,et al. Emergence of CD52-, phosphatidylinositolglycan-anchor-deficient T lymphocytes after in vivo application of Campath-1H for refractory B-cell non-Hodgkin lymphoma. , 1995, Blood.
[85] L. Luzzatto,et al. Genomic organization of the X-linked gene (PIG-A) that is mutated in paroxysmal nocturnal haemoglobinuria and of a related autosomal pseudogene mapped to 12q21. , 1994, Human molecular genetics.
[86] P. Ganly,et al. Tissue plasminogen activator for hepatic vein thrombosis in paroxysmal nocturnal haemoglobinuria , 1994, Journal of internal medicine.
[87] K. Bourantas. High-dose recombinant human erythropoietin and low-dose corticosteroids for treatment of anemia in paroxysmal nocturnal hemoglobinuria. , 1994, Acta haematologica.
[88] Bourantas Kl. High-Dose Recombinant Human Erythropoietin and Low-Dose Corticosteroids for Treatment of Anemia in Paroxysmal Nocturnal Hemoglobinuria , 1994 .
[89] E. Gómez-Morales,et al. TRATAMIENTO DE LA HEMOGLOBINURIA PAROXISTICA NOCTURNA CON GLOBULINA ANTILINFOCITARIA , 1993 .
[90] L. del Vecchio,et al. Membrane proteins in paroxysmal nocturnal haemoglobinuria. , 1993, Blood reviews.
[91] Teizo Fujita,et al. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria , 1993, Cell.
[92] T. Miyata,et al. The cloning of PIG-A, a component in the early step of GPI-anchor biosynthesis. , 1993, Science.
[93] T. Miyata,et al. Deficient Biosynthesis of N-acetylglucosaminyl- Phosphatidylinositol, the First Hte~iiiediate of Glycosyl Phosphatidylinositol Anchor Biosynthesis, in Cell Lines Established from Patients with Paroxysmal Nocturnal Hemoglobinuria Materials and Methods , 2022 .
[94] T. Ortel,et al. Complement-induced vesiculation and exposure of membrane prothrombinase sites in platelets of paroxysmal nocturnal hemoglobinuria. , 1993, Blood.
[95] G. Gutiérrez-Espíndola,et al. [Treatment of paroxysmal nocturnal hemoglobinuria with antilymphocyte globulin]. , 1993, Revista de investigacion clinica; organo del Hospital de Enfermedades de la Nutricion.
[96] M. Rosenfeld,et al. Synthesis of mannosylglucosaminylinositol phospholipids in normal but not paroxysmal nocturnal hemoglobinuria cells. , 1992, Proceedings of the National Academy of Sciences of the United States of America.
[97] M. Borowitz,et al. Defective glycosylphosphatidylinositol anchor synthesis in paroxysmal nocturnal hemoglobinuria granulocytes. , 1992, Blood.
[98] A. Gratwohl,et al. Morphology in patients with severe aplastic anemia treated with antilymphocyte globulin. , 1992, Blood.
[99] C. Parker,et al. Molecular basis of the enhanced susceptibility of the erythrocytes of paroxysmal nocturnal hemoglobinuria to hemolysis in acidified serum. , 1991, Blood.
[100] A. Gratwohl,et al. High-dose recombinant human erythropoietin for treatment of anemia in myelodysplastic syndromes and paroxysmal nocturnal hemoglobinuria: a pilot study. , 1990, Experimental hematology.
[101] T. Kinoshita,et al. Inherited complete deficiency of 20-kilodalton homologous restriction factor (CD59) as a cause of paroxysmal nocturnal hemoglobinuria. , 1990, The New England journal of medicine.
[102] W. Rosse. Phosphatidylinositol-linked proteins and paroxysmal nocturnal hemoglobinuria , 1990 .
[103] H. Okada,et al. Erythrocytes of patients with paroxysmal nocturnal haemoglobinuria acquire resistance to complement attack by purified 20‐kD homologous restriction factor , 1990, Clinical and experimental immunology.
[104] W. Rosse,et al. Phosphatidylinositol-linked proteins and paroxysmal nocturnal hemoglobinuria. , 1990, Blood.
[105] W. Rosse,et al. Relationship between the membrane inhibitor of reactive lysis and the erythrocyte phenotypes of paroxysmal nocturnal hemoglobinuria. , 1989, The Journal of clinical investigation.
[106] L. Luzzatto,et al. 7 Paroxysmal nocturnal haemoglobinuria , 1989 .
[107] T. Springer,et al. The major Fc receptor in blood has a phosphatidylinositol anchor and is deficient in paroxysmal nocturnal haemoglobinuria , 1988, Nature.
[108] S. Issaragrisil,et al. Corticosteroids therapy in paroxysmal nocturnal hemoglobinuria , 1987, American journal of hematology.
[109] H. Müller-Eberhard,et al. Deficiency of the homologous restriction factor in paroxysmal nocturnal hemoglobinuria , 1987, The Journal of experimental medicine.
[110] H. Müller-Eberhard,et al. Isolation of a human erythrocyte membrane protein capable of inhibiting expression of homologous complement transmembrane channels. , 1986, Proceedings of the National Academy of Sciences of the United States of America.
[111] V. Hu,et al. Membrane factors responsible for homologous species restriction of complement-mediated lysis: evidence for a factor other than DAF operating at the stage of C8 and C9. , 1985, Journal of immunology.
[112] N. Young,et al. Circulating activated suppressor T lymphocytes in aplastic anemia. , 1985, The New England journal of medicine.
[113] J. Leddy,et al. Enhanced reactive lysis of paroxysmal nocturnal hemoglobinuria erythrocytes by C5b-9 does not involve increased C7 binding or cell-bound C3b. , 1985, Journal of immunology.
[114] V. Nussenzweig,et al. Inhibition of complement activation on the surface of cells after incorporation of decay-accelerating factor (DAF) into their membranes , 1984, The Journal of experimental medicine.
[115] N. Scarpato,et al. Two populations of erythroid cell progenitors in paroxysmal nocturnal hemoglobinuria. , 1984, Blood.
[116] K. Austen,et al. Affected erythrocytes of patients with paroxysmal nocturnal hemoglobinuria are deficient in the complement regulatory protein, decay accelerating factor. , 1983, Proceedings of the National Academy of Sciences of the United States of America.
[117] W. Rosse. Treatment of paroxysmal nocturnal hemoglobinuria , 1982 .
[118] L. Luzzatto,et al. Decreased number of circulating BFU-Es in paroxysmal nocturnal hemoglobinuria. , 1982, Blood.
[119] W. Rosse. Treatment of paroxysmal nocturnal hemoglobinuria. , 1982, Blood.
[120] K. Austen,et al. Isolation of C4-binding protein from guinea pig plasma and demonstration of its function as a control protein of the classical complement pathway C3 convertase. , 1981, Journal of immunology.
[121] J. Dacie. Paroxysmal nocturnal haemoglobinuria. , 1972, The Scientific basis of medicine annual reviews.
[122] L Luzzatto,et al. Paroxysmal nocturnal hemoglobinuria: evidence for monoclonal origin of abnormal red cells. , 1970, Blood.
[123] R. Dourmashkin,et al. IMMUNE LYSIS OF NORMAL HUMAN AND PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) RED BLOOD CELLS , 1966, The Journal of experimental medicine.
[124] J. Dacie,et al. Immune lysis of normal human and paroxysmal nocturnal hemoglobinuria (PNH) red blood cells. I. The sensitivity of PNH red cells to lysis by complement and specific antibody. , 1966, The Journal of clinical investigation.
[125] A. Risitano. PAROXYSMAL nocturnal hemoglobinuria. , 2012, Journal of the American Medical Association.