This site is intended for US audiences only. If you are a resident of the European Union, please click here for the EU version of our site.
Bone Marrow Transplantation
Bone marrow transplantation (BMT) remains the only curative option for PNH. However, BMT is associated with high rates of morbidities and mortality.1-4 Data from a recent publication from Santarone et al show that of 26 PNH patients who underwent BMT, 42% died.4 Even those who undergo a successful BMT may experience long-term complications, including graft-versus-host disease (GVHD). Santarone et al showed that acute GVHD (≥ grade II) developed in 42% of patients and chronic GVHD developed in 50% of 20 evaluable patients.4
1. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. N Engl J Med. 1995;333:1253-1258. 2. Hegenbart U, Niederwieser D, Forman S, et al. Biol Blood Marrow Transplant. 2003;9:689-697. 3. Witherspoon RP. In: Blume KG, Forman SJ, Appelbaum FR, eds. Thomas’ Hematopoietic Cell Transplantation. 3rd ed. Oxford, UK: Wiley-Blackwell; 2004:1002-1006. 4. Santarone S, Bacigalupo A, Risitano AM, et al. Haematalogica. 2010;95:983-988.
Dr Wendell Rosse explains how the pathophysiology of PNH can lead to thrombosis.
Click here to watch a video of
Dr Wendell Rosse discussing the risk of pulmonary hypertension in PNH.