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.
|Original language||English (US)|
|Number of pages||10|
|Journal||Bone marrow transplantation|
|Early online date||Jan 13 2021|
|State||Published - Jun 2021|
Bibliographical noteFunding Information:
Beth Kamp, PharmD, a medical writer supported by funding from Magenta Therapeutics provided initial editorial assistance to the authors during preparation of this paper.
© 2021, The Author(s), under exclusive licence to Springer Nature Limited.