A Pediatric Case of Chronic Immune Thrombocytopenic Purpura Treated with Sirolimus

Few pediatric patients with Immune Thrombocytopenic Purpura (ITP) continue to have low platelets despite treatment with pooled gamma globulin (IVIG), corticosteroids, and anti-D immunoglobulin. Sirolimus (previously known as rapamycin) is an mTOR inhibitor that may improve platelet counts for this subset of patients. Here we present a pediatric case of chronic ITP refractoryto first-line pharmacotherapy. Following treatment with sirolimus, our patient'saverage platelet count increased from 41.9 x 109/L to 264.4 x 109/L (P=6.7 x 10-6). Furthermore, she experienced resolution of her bleeding symptoms withoutencountering side-effects related to treatment.