Eculizumab has become the standard of care for patients with paroxysmal nocturnal hemoglobinuria (PNH). As more patients are treated, the long-term outcomes of these patients will become apparent. We recently treated a patient who developed PNH in the setting of aplastic anemia. The patient developed acute myeloid leukemia less than three years after initiating eculizumab. The patient also died suddenly from Scedosporium sepsis during induction therapy. This patient's course seemed more aggressive than would be expected. The possible effect of complement blockade is discussed.
Hanmantgad, M., Nog, R., & Seiter, K. (2014). Acute Myeloid Leukemia and Fatal Scedosporium prolificans Sepsis After Eculizumab Treatment for Paroxysmal Nocturnal Hemoglobinuria: a Case Report. Stem Cell Investigation, 4, 100. https://doi.org/10.21037/sci.2017.12.04
Originally published in Stem Cell Investigation, 4, 100. The original material can be found here.