Bortezomib treatment of steroid‐refractory Evans syndrome in children. Issue 12 (23rd September 2020)

• Record Type: Journal Article
• Authors: Beydoun, Serina B.; Persaud, Yogindra; Lafferty, Jennifer; Callaghan, Michael U.; Savaşan, Süreyya
• Is Part Of: Pediatric blood & cancer. Volume 67:Issue 12(2020)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2020-09-23
• Subjects: bortezomib -- children -- Evans syndrome -- recovery -- steroid‐refractory; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.28725
• Languages: English
• ISSNs: 1545-5009

Abstract

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 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.