Paroxysmal Nocturnal Haemoglobinuria (PNH) Dr Conal McConville ...

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Thrombosis Chronic Kidney Disease Fatigue / Impaired QOL Pulmonary Hypertension Fatigue and Quality of Life Conclusions • 96% of patients report fatigue 1 – Fatigue/QoL independent of anemia/transfusion requirements • 76% of patients with PNH have disruptions in daily activities 1 – 17% of patients were unemployed due to PNH • Fatigue, QoL and abdominal pain are linked by underlying haemolysis and the threat of ischemia and mortality 2 – 66% of patients report shortness of breath – 57% of patients report abdominal pain • 2.2-fold increased risk of mortality • 3.6-fold increased risk of developing TE • PNH symptoms are independent of clone size 3 1. Meyers G et al. Blood. 2007;110(11):Abstract 3683. 2. Lee JW et al. Hematologica 2010. 95 (s2): Abstract #506. 3. Urbano-Ispizua A et al. Hematologica 2010. 95 (s2): Abstract #1022. August_12_2010Gbl 20
Thrombosis Chronic Kidney Disease Fatigue / / Impaired QOL Is Dyspnoea in PNH Linked to Pulmonary Hypertension? • 66% of PNH patients report dyspnoea 1 Pulmonary Hypertension • Haemolysis results in cell-free haemoglobin and NO consumption leading to pulmonary hypertension 2,3 1. Meyers G et al. Blood. 2007;110(11):Abstract 3683. 2. Rother R. et al. JAMA. 2005; 293: 1653-1662. 3. Hill A et al. Br J Haematol. 2010; May;149(3):414-25. August_12_2010Gbl 21
Page 1 and 2: Paroxysmal Nocturnal Haemoglobinuri
Page 3 and 4: Paroxysmal Nocturnal Haemoglobinuri
Page 5 and 6: The Defect in PNH The Somatic Mutat
Page 7 and 8: PNH is a Progressive Disease of Chr
Page 9 and 10: Thrombosis Chronic Kidney Disease F
Page 19: Thrombosis Chronic Kidney Disease F
Page 23 and 24: Common Symptoms of Haemolysis Signa
Page 25 and 26: Historical Management of PNH Bone M
Page 27 and 28: SOLIRIS ® Blocks Terminal Compleme
Page 29 and 30: Adverse Reactions Reported in ≥ 5
Page 31: August 12_10_2010Gbl Thank you Than

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