Paroxysmal Nocturnal Haemoglobinuria (PNH) Dr Conal McConville ...
Paroxysmal Nocturnal Haemoglobinuria (PNH) Dr Conal McConville ...
Paroxysmal Nocturnal Haemoglobinuria (PNH) Dr Conal McConville ...
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Historical Management of <strong>PNH</strong><br />
Bone Marrow Transplant<br />
BMT is associated with significant morbidity and mortality<br />
Haemolysis and thrombosis are risk factors for poor outcomes<br />
• In a recent retrospective study in France examining <strong>PNH</strong><br />
patients: 1<br />
– 54% had GVHD<br />
• In another study examining <strong>PNH</strong> patients (n=23 )2<br />
– 50% chronic GVHD; 42% acute GVHD<br />
• BMT has a significant impact on quality of life post<br />
transplant 3,4<br />
• Allogeneic BMT recommended for <strong>PNH</strong> patients with life-<br />
threatening cytopenias or possibly the rare patient with<br />
disabling haemolysis or thrombosis not controlled with<br />
existing therapy 5<br />
1. De Latour PF et al. Abstract #316. EBMT 2009. 2. Santarone S et al. Haematologica. 2010; Jun;95(6):983-8 . 3. Bieri S et al. Bone Marrow Transplantation.<br />
2008; 42: 819–827. 4. Fraser CJ et al. Blood. 2006;108: 2867-2873. 5. Brodsky RA. Blood. 2009 113: 6522-6527<br />
August_12_2010Gbl<br />
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