Sirolimus led to complete and durable responses in a majority of children with refractory multi-lineage autoimmune cytopenias, according to a study of 30 patients treated with sirolimus as monotherapy. The study included 12 children with autoimmune lymphoproliferative syndrome (ALPS), 12 with secondary multi-lineage cytopenias, and 6 with single-lineage cytopenias. Researchers found:

• All patients with ALPS achieved a durable complete response, including rapid improvement in autoimmune disease.

• Double negative T cells were no longer detectable in most, while other lymphocyte populations were spared.

• 8 of 12 patients with multi-lineage cytopenias achieved a complete response, though the time to response was slower than in patients with ALPS.

• 2 of 6 patients with single-lineage cytopenias achieved a response.

• Sirolimus was well tolerated with very few side effects.

Citation: Bride KL, Vincent T, Smith-Whitley K, et al. Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial. [Published online ahead of print October 26, 2015]. Blood. doi: 10.1182/blood-2015-07-657981.