Bortezomib Treatment of Steroid-Refractory Evans Syndrome in Children

Abstract

Treatment of refractory Evans syndrome (ES) remains a challenge in Hematology practice. Due to rarity of this condition, evidence-based approaches are limited and often treatment choices stem from small case series or anectodal experiences. Here, we describe three very refractory pediatric ES cases treated on bortezomib without adverse effects. Two of the three patients had dramatical and long-lasting recovery that started following the first doses of the drug. Clinical trials to assess bortezomib role in ES treatment are warrented and results may lead to inclusion of this drug as an option, even as a first-line therapy.

Role of Proteasomes in Inflammation

“…This effect is unfortunately not limited to pathogenic PCs but also depletes protective PCs [ 164 ]. Bortezomib has furthermore successfully been used in different cases of autoimmunity including autoimmune cytopenia, refractory primary SjS and encephalitis [ 165 , 166 , 167 , 168 ]. As all beneficial effects of bortezomib on the SLE disease course cannot be attributed to the reduction in autoantibodies [ 162 ], an additional anti-inflammatory effect is suspected.…”