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Therapeutic Options for PNH
Helping Patients Manage Hemolysis and Achieve a Better Quality of Life
Historically, there have been few options for the treatment of PNH. However, major advances have occurred and options now include monoclonal antibodies, palliative therapies, like transfusions and steroids, and bone marrow transplantation (BMT).
Monoclonal Antibodies
Specific proteins that are normally found attached to cells via GPI anchors are absent from the surface of PNH cells. Monoclonal antibody therapy in PNH interrupts the complement cascade and helps preserve PNH cells, thereby significantly reducing chronic hemolysis (as measured by LDH).1,2
Palliative Therapies
Palliative therapeutic options: transfusions, corticosteroids, anticoagulants, androgen therapy, and supplements such as folic acid and iron.3,4
Bone Marrow Transplantation
Bone marrow transplantation (BMT) remains the only curative option for PNH, but is associated with high rates of morbidities and mortality.5-8
1. Brodsky RA. Blood. 2009;113:6522-6527. 2. Rother RP, Rollins SA, Mojcik CF, et al. Nat Biotechnol. 2007;25:1256-1264. 3. Parker C, Omine M, Richards S, et al; for the International PNH Interest Group. Blood. 2005;106:3699-3709. 4. Moyo VM, Mukhina GL, Garrett ES, Brodsky RA. Br J Haematol. 2004;126:133-138. 5. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. N Engl J Med. 1995;333:1253-1258. 6. Hegenbart U, Niederwieser D, Forman S, et al. Biol Blood Marrow Transplant. 2003;9:689-697. 7. Witherspood RP. In: Blume KG, Forman SJ, Appelbaum FR, eds. Thomas’ Hematopoietic Cell Transplantation. 3rd ed. Oxford, UK: Wiley-Blackwell;2004:1002-1006. 8. Santarone S, Bacigalupo A, Risitano AM, et al. Haematalogica. 2010;95:983-988.
Click on the links below to watch Dr Jeff Patten, MD, present videos of short case studies about patients with PNH.