Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial.

ABSTRACT: Patients with autoimmune multilineage cytopenias are often refractory to standard therapies requiring chronic immunosuppression with medications with limited efficacy and high toxicity. We present data on 30 patients treated on a multicenter prospective clinical trial using sirolimus as monotherapy. All children (N = 12) with autoimmune lymphoproliferative syndrome (ALPS) achieved a durable complete response (CR), including rapid improvement in autoimmune disease, lymphadenopathy, and splenomegaly within 1 to 3 months of starting sirolimus. Double-negative T cells were no longer detectable in most, yet other lymphocyte populations were spared, suggesting a targeted effect of sirolimus. We also treated 12 patients with multilineage cytopenias secondary to common variable immunodeficiency (C

SUBMITTER: Bride KL 

PROVIDER: S-EPMC4705607 | biostudies-literature | 2016 Jan

REPOSITORIES: biostudies-literature