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Bortezomib treatment of steroid‐refractory Evans syndrome in children
Author(s) -
Beydoun Serina B.,
Persaud Yogindra,
Lafferty Jennifer,
Callaghan Michael U.,
Savaşan Süreyya
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28725
Subject(s) - bortezomib , medicine , refractory (planetary science) , adverse effect , hematology , evans syndrome , clinical trial , oncology , intensive care medicine , multiple myeloma , physics , autoimmune hemolytic anemia , astrobiology , anemia
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.