Aplastic anemia: Quo vadis?

Abstract

In the last 30 years, the field of aplastic anemia (AA), and more generally bone marrow failure syndromes, has undergone a multitude of new discoveries. The application of modern and sophisticated sequencing techniques unveiled a variety of genes associated with these disorders and contributed to a better understanding of the disease pathobiology. This advancement was paralleled by the discovery, clinical testing and subsequent approval of new drugs for the treatment of AA and associated disorders. Several additional agents are currently under evaluation for possible therapies. Herein, we look at the potential future avenues of research in AA through a brief summary of an intergenerational Socratic dialogue between the mentor, who witnessed and actively contributed to the milestones achieved in the last 30 years, and his fellow, who would himself go on to become the mentor of a new generation of AA researchers.

Keywords: Aplastic anemia; Paroxysmal nocturnal hemoglobinuria.