Treatment of refractory pure red cell aplasia with cyclosporine A: disappearance of IgG inhibitor associated with clinical response

Remissions were obtained in 6/9 evaluable patients with pure red cell asplasia (PRCA) refractory to other immunosuppressive agents who were treated with cyclosporine A (CsA). Four of these patients have remained in continuous remission off all treatment for 4-19 months. Another patient who stopped CsA abruptly relapsed, but responded to reinstitution of therapy. The sixth patient died of a cerebrovascular accident while in remission on a low dose of CsA. Acute side effects were minimal and were responsive to dose reduction. One patient developed a lymphoma while in an unmaintained remission, and one patient who did not respond to CsA was found to have a lymphoma approximately a year after stopping treatment. In vitro studies of autologous erythroid progenitors in a patient with an IgG inhibitor of erythropoiesis showed a reduction of autoantibody associated with the response to CsA. The antigen to which this inhibitor is directed was expressed only during the marrow erythroid burst-forming unit (BFU-E) period of erythroid differentiation. CsA can induce sustained remissions in cases of PRCA refractory to other multiple agents, and these remissions may be associated with a reduction in autoantibody to erythroid progenitor cells. Further studies of patients with PRCA who respond to CsA may lead to an improved understanding of this disorder.