Pure red cell aplasia: The second hundred years

Pure red cell aplasia (PRCA) is a rare hematologic syndrome, characterized by an isolated normocytic anemia with severe reticulocytopenia, and defined by absence or near absence of erythroid precursors in the bone marrow. First described in 1922, PRCA may be a primary autoimmune or clonal myeloid or lymphoid disorder, but may also be secondary to other disorders of immune dysregulation/autoimmunity, to infections, to neoplasms, or to drugs. Insights from the study of PRCA have helped illuminate the understanding of the regulation of erythropoiesis. This review summarizes the classification, diagnostic, and therapeutic approach to PRCA as it begins its second century, with a particular focus on opportunities and challenges provided by new developments in the role of T-cells and T-cell regulatory mutations; the role of clonal hematopoiesis; and new developments in therapy for refractory PRCA and PRCA associated with ABO incompatible stem cell transplantation.