Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders Review


Authors: Gupta, A. O.; Boelens, J. J.; Ebens, C. L.; Kurtzberg, J.; Lund, T. C.; Smith, A. R.; Wagner, J. E.; Wynn, R.; Blazar, B. R.; Orchard, P. J.
Review Title: Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders
Abstract: Hematopoietic stem cell transplantation (HCT) has been increasingly used for patients with inherited metabolic disorders (IMD). Immune mediated cytopenias (IMCs) after HCT, manifesting as hemolytic anemia, thrombocytopenia, and/or neutropenia, are recognized as a significant complication in this patient population, yet our understanding of the incidence, risk factors, and pathophysiology is currently limited. Review of the published literature demonstrates a higher incidence in younger patients who undergo HCT for a nonmalignant disease indication. However, a few reports suggest that the incidence is even higher among those with IMD (incidence ranging from 10 to 56%). This review summarizes the literature, provides an approach to better understanding of the possible etiology of IMCs, and proposes a diagnostic and management plan for patients with IMD who develop single or multi-lineage cytopenias after HCT. © 2021, The Author(s), under exclusive licence to Springer Nature Limited.
Journal Title: Bone Marrow Transplantation
Volume: 56
Issue: 6
ISSN: 0268-3369
Publisher: Nature Publishing Group  
Date Published: 2021-06-01
Start Page: 1238
End Page: 1247
Language: English
DOI: 10.1038/s41409-020-01179-5
PUBMED: 33441980
PROVIDER: scopus
PMCID: PMC8189901
DOI/URL:
Notes: Article -- Export Date: 1 July 2021 -- Source: Scopus
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  1. Jaap Jan Boelens
    97 Boelens