[Acquired pure red cell aplasia]

Acquired pure red cell aplasia (PRCA) is a bone marrow failure syndrome characterized by anemia, reticulocytopenia, and erythroid hypoplasia in the marrow that mostly affects older adults. Underlying T-cell dysregulation, often associated with clonality and/or STAT3 mutation among CD8+T-cells, provides a rationale for directed immunosuppressive therapies such as cyclosporin in the three most frequent disease subtypes: thymoma-associated PRCA, large granular lymphocytic leukemia-associated PRCA, and idiopathic PRCA. Some retrospective studies have demonstrated the significance of maintenance therapy for avoiding blood transfusion dependency, which is associated with poorer prognosis in patients with PRCA. While treatment options are currently scarce for patients with relapsed or refractory disease after CsA, results of a prospective randomized controlled clinical trial of sirolimus are expected in the near future.