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Successful treatment with daratumumab for post-HSCT refractory hemolytic anemia


Autoimmune cytopenias (AIC) following allogeneic hematopoietic stem cell transplantation (HSCT) may cause significant morbidity and mortality and are often challenging to treat. We present a case of a pediatric patient with primary myelofibrosis of infancy caused by VPS45 protein deficiency, who developed severe refractory hemolytic anemia and immune-mediated thrombocytopenia 3.5 months following HSCT. After the failure of several treatments, he received daratumumab, an anti-CD38 specific antibody, and demonstrated fast and sustained response. The only side effect was delayed recovery of humoral immunity. Daratumumab, by targeting antibody-producing plasma cells, may be a valid treatment option for refractory post-HSCT AIC.

Type Journal
ISBN 1545-5017 (Electronic) 1545-5009 (Linking)
Authors Even-Or, E.; Naser Eddin, A.; Shadur, B.; Dinur Schejter, Y.; Najajreh, M.; Zelig, O.; Zaidman, I.; Stepensky, P.
Responsible Garvan Author Dr Bella Shadur
Published Date 2020-01-01
Published Volume 67
Published Issue 1
Published Pages e28010
Status Published in-print
DOI 10.1002/pbc.28010
URL link to publisher's version