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Case Reports in Hematology
Volume 2012, Article ID 106182, 5 pages
http://dx.doi.org/10.1155/2012/106182
Case Report

Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab

1Division of Hematology, Department of Medicine, Montefiore Medical Center, 111E 210th Street, Bronx, NY 10467, USA
2Department of Oncology, Montefiore Medical Center, 111E 210th Street, Bronx, NY 10467, USA

Received 19 December 2011; Accepted 1 February 2012

Academic Editors: D. Galanakis, M. Nagasawa, and P. Tsirigotis

Copyright © 2012 Radha Raghupathy and Olga Derman. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Abstract

Paroxysmal nocturnal hemoglobinuria is caused by expansion of a hematopoietic stem cell clone with an acquired somatic mutation in the PIG-A gene. This mutation aborts the synthesis and expression of the glycosylphosphatidylinositol anchor proteins CD55 and CD59 on the surface of blood cells, thereby making them more susceptible to complement-mediated damage. A spectrum of disorders occurs in PNH ranging from hemolytic anemia and thrombosis to myelodysplasia, aplastic anemia and, myeloid leukemias. Aplastic anemia is one of the most serious and life-threatening complications of PNH, and a PNH clone is found in almost a third of the cases of aplastic anemia. While allogeneic bone marrow transplantation and T cell immune suppression are effective treatments for aplastic anemia in PNH, these therapies have significant limitations. We report here the first case, to our knowledge, of PNH associated with aplastic anemia treated with the anti-CD20 monoclonal antibody rituximab, which was associated with a significant reduction in the size of the PNH clone and recovery of hematopoiesis. We suggest that this less toxic therapy may have a significant role to play in treatment of PNH associated with aplastic anemia.