Successful treatment with daratumumab for post-HSCT refractory hemolytic anemia.
Anemia, Hemolytic
/ drug therapy
Anemia, Refractory
/ drug therapy
Antibodies, Monoclonal
/ therapeutic use
Antineoplastic Agents
/ therapeutic use
Child, Preschool
Hematopoietic Stem Cell Transplantation
/ adverse effects
Humans
Male
Primary Myelofibrosis
/ pathology
Prognosis
Transplantation, Homologous
Vesicular Transport Proteins
/ deficiency
HSCT
autoimmune hemolytic anemia
daratumumab
immune cytopenia
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
01 2020
01 2020
Historique:
received:
07
07
2019
revised:
20
08
2019
accepted:
08
09
2019
pubmed:
24
9
2019
medline:
10
4
2020
entrez:
24
9
2019
Statut:
ppublish
Résumé
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.
Substances chimiques
Antibodies, Monoclonal
0
Antineoplastic Agents
0
VPS45 protein, human
0
Vesicular Transport Proteins
0
daratumumab
4Z63YK6E0E
Types de publication
Case Reports
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e28010Informations de copyright
© 2019 Wiley Periodicals, Inc.
Références
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