The diagnosis and management of children with autoimmune cytopenias can be challenging. Children can present with immune-mediated destruction of a single-cell lineage or multiple cell lineages, including platelets (immune thrombocytopenia [ITP]), erythrocytes (autoimmune hemolytic anemia), and neutrophils (autoimmune neutropenia). Immune-mediated destruction can be primary or secondary to a comorbid immunodeficiency, malignancy, rheumatologic condition, or lymphoproliferative disorder. Treatment options generally consist of nonspecific immune suppression or modulation. This nonspecific approach is changing as recent insights into disease biology have led to targeted therapies, including the use of thrombopoietin mimetics in ITP and sirolimus for cytopenias associated with autoimmune lymphoproliferative syndrome.
Key points
- •
Most children with autoimmune cytopenias have idiopathic disease with no secondary cause and enter a spontaneous remission with time.
- •
Chronic or multi-lineage disease should prompt testing for secondary causes of autoimmune cytopenias, including human immunodeficiency virus, systemic lupus erythematosus, autoimmune lymphoproliferative syndrome (ALPS), and common variable immune deficiency.
- •
First-line treatments typically include corticosteroids and intravenous IgG. Second-line treatments vary depending on the cell lineage(s) affected and whether or not there is an underlying cause.
- •
Corticosteroids can cause significant long-term morbidity; therefore, their long-term use should be avoided, because numerous safe and effective alternatives exist.
- •
Recent advances have led to targeted therapies for select patients, including the use of thrombopoeitin (TPO) receptor agonists (TPO mimetics) for immune thrombocytopenia and sirolimus for ALPS.
Stay updated, free articles. Join our Telegram channel
Full access? Get Clinical Tree
