Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders
- 13 January 2021
- journal article
- review article
- Published by Springer Science and Business Media LLC in Bone Marrow Transplantation
- Vol. 56 (6), 1238-1247
- https://doi.org/10.1038/s41409-020-01179-5
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.Keywords
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