Acquired Pure Red Cell Aplasia: Cyclosporin and T-Cell Dysregulation as Therapeutic Targets

Acquired pure red cell aplasia (PRCA) is a bone marrow failure syndrome marked by anemia, reticulocytopenia, and erythroid hypoplasia, predominantly affecting older adults. Its pathogenesis involves underlying T-cell dysregulation, frequently associated with clonality and/or STAT3 mutation among CD8+T-cells. This mechanism provides a strong rationale for directed immunosuppressive therapies like cyclosporin in the three most common subtypes: thymoma-associated PRCA, large granular lymphocytic leukemia-associated PRCA, and idiopathic PRCA. Current treatment options are limited for patients with relapsed or refractory disease after cyclosporin.

This review synthesizes current understanding of Acquired pure red cell aplasia (PRCA), detailing its pathophysiology, common subtypes, and established immunosuppressive treatment strategies. It examines the role of cyclosporin as a primary therapy and highlights the significance of maintenance therapy. The review also discusses the limited options for relapsed/refractory disease and anticipates results from a prospective randomized controlled trial of sirolimus, providing a comprehensive overview of the current therapeutic landscape.