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.
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 anecdotal experiences. There is mounting evidence that some patients have genetic defects that could be targeted with promising preliminary results. Here, we describe three very refractory pediatric ES cases treated on bortezomib without adverse effects. Two of the three patients had dramatic and long-lasting recovery that started following the initial doses of the drug. Clinical trials to assess the role of bortezomib in ES treatment are warranted.
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