Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab
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, there...
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Wiley
2012-01-01
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| Series: | Case Reports in Hematology |
| Online Access: | http://dx.doi.org/10.1155/2012/106182 |
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| author | Radha Raghupathy Olga Derman |
| author_facet | Radha Raghupathy Olga Derman |
| author_sort | Radha Raghupathy |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-b6adfa4ea0124d35b08cf09e38cdaebf |
| institution | OA Journals |
| issn | 2090-6560 2090-6579 |
| language | English |
| publishDate | 2012-01-01 |
| publisher | Wiley |
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| series | Case Reports in Hematology |
| spelling | doaj-art-b6adfa4ea0124d35b08cf09e38cdaebf2025-08-20T02:23:11ZengWileyCase Reports in Hematology2090-65602090-65792012-01-01201210.1155/2012/106182106182Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to RituximabRadha Raghupathy0Olga Derman1Division of Hematology, Department of Medicine, Montefiore Medical Center, 111E 210th Street, Bronx, NY 10467, USADepartment of Oncology, Montefiore Medical Center, 111E 210th Street, Bronx, NY 10467, USAParoxysmal 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.http://dx.doi.org/10.1155/2012/106182 |
| spellingShingle | Radha Raghupathy Olga Derman Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab Case Reports in Hematology |
| title | Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab |
| title_full | Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab |
| title_fullStr | Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab |
| title_full_unstemmed | Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab |
| title_short | Response of Paroxysmal Nocturnal Hemoglobinuria Clone with Aplastic Anemia to Rituximab |
| title_sort | response of paroxysmal nocturnal hemoglobinuria clone with aplastic anemia to rituximab |
| url | http://dx.doi.org/10.1155/2012/106182 |
| work_keys_str_mv | AT radharaghupathy responseofparoxysmalnocturnalhemoglobinuriaclonewithaplasticanemiatorituximab AT olgaderman responseofparoxysmalnocturnalhemoglobinuriaclonewithaplasticanemiatorituximab |