Journal
PEDIATRIC BLOOD & CANCER
Volume 67, Issue 1, Pages -Publisher
WILEY
DOI: 10.1002/pbc.28010
Keywords
autoimmune hemolytic anemia; daratumumab; HSCT; immune cytopenia
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Funding
- Australian Government Research Training Program Scholarship
- Hadassah Australia
- Deutsche Forschungsgemeinschaft (Discovery and Evaluation of New Combined Immunodeficiency Disease Entities) [DFGWA 1597/4-2]
- ERA-Net ERARE Consortium EURO-CID
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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.
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