Bortezomib treatment of steroid-refractory Evans syndrome in children

Serina B. Beydoun, Yogindra Persaud, Jennifer Lafferty, Michael U. Callaghan, Süreyya Savaşan

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


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.

Original languageEnglish
Article numbere28725
JournalPediatric Blood and Cancer
Issue number12
StatePublished - Dec 1 2020


  • Evans syndrome
  • bortezomib
  • children
  • recovery
  • steroid-refractory


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