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.