Clinical impact of uncontrolled complement activity in Japanese non-transfused patients with paroxysmal nocturnal hemoglobinuria

)–Mean SE FACIT-Fatigue scores increased from 39.0 1.86 and 32.5 13.50 at baseline to 42.3 1.54 and 46.5 4.50 at 12 weeks for non-transfused patients and transfused patients, respectively •Eculizumab treatment resulted in changes of 5 and 23 points (> 3-point improvement is considered clinically meaningful) over 12 weeks in the FACIT-Fatigue scores for the 2 non-transfused patients, respectively• Treatment with eculizumab resulted in improvements in QoL, as measured by EORTC QLQ-C30, in patients with and without a transfusion history (

[1]  K. Ozawa,et al.  Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS Clinical Trial , 2011, International journal of hematology.

[2]  W. Gregory,et al.  Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. , 2010, Blood.

[3]  G. Khursigara,et al.  Long‐term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria , 2010, American journal of hematology.

[4]  M. Gladwin,et al.  Effect of eculizumab on haemolysis‐associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria , 2010, British journal of haematology.

[5]  A. Risitano,et al.  Clinical Impact of Unregulated Terminal Complement Activity in Never-Transfused Patients with Paroxysmal Nocturnal Hemoglobinuria. , 2009 .

[6]  N. Young,et al.  Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. , 2008, Blood.

[7]  C. Parker The pathophysiology of paroxysmal nocturnal hemoglobinuria. , 2007, Experimental hematology.

[8]  N. Young,et al.  The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. , 2006, The New England journal of medicine.

[9]  M. Gladwin,et al.  The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. , 2005, JAMA.

[10]  S. Hall,et al.  Clinical Course and Flow Cytometric Analysis of Paroxysmal Nocturnal Hemoglobinuria in the United States and Japan , 2004, Medicine.

[11]  S M Lewis,et al.  Natural history of paroxysmal nocturnal hemoglobinuria. , 1995, The New England journal of medicine.

[12]  R. Hoffman,et al.  Hematology: Basic Principles and Practice , 1995 .