Recent progress in the diagnosis and management of acquired pure red cell aplasia

Acquired pure red cell aplasia (PRCA) is characterized by normocytic anemia, reticulocytopenia, and a marked decrease in erythroid cell count in the bone marrow. PRCA develops in the context of various backgrounds, including recently recognized immune checkpoint inhibitor-associated PRCA, that need careful differential diagnoses. Besides humoral abnormalities such as major ABO-incompatible allogeneic hematopoietic stem cell transplantation-related PRCA, dysregulations of T cells have been shown. STAT3 gene mutations of cytotoxic T cells were identified in 40% of PRCA patients, which might suggest their use as novel molecular markers for PRCA. As initial management options for PRCA, red blood cell transfusion and immunosuppressive therapy (IST) drugs, such as cyclosporin, are usually selected. Roughly 80% of patients respond to IST; however, some relapse afterward or are refractory to IST. When patients with PRCA become refractory to two or three lines of IST, allogeneic hematopoietic stem cell transplantation (HCT) would become an appropriate choice, although the optimal procedures for allogeneic HCT have not been determined. A prospective study of PRCA in Japan has been ongoing since 2016 to solve the myriad clinical issues of PRCA.