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Advances in Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria

31 Dec 2020

Abstract excerpt

In the era of eculizumab, the number of patients with paroxysmal nocturnal hemoglobinuria (PNH) who undergo hematopoietic stem cell transplantation (HSCT) has decreased significantly. However, owing to the possibility of severe aplastic anemia (AA) or a suboptimal response to eculizumab, HSCT still plays an important role in the treatment of patients with PNH combined with AA or recurrent hemolysis-related...

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PMID: 33840442
DOI: 10.1016/j.jtct.2020.11.004

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