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

Journal Article (Journal Article;Review)

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.

Full Text

Duke Authors

Cited Authors

  • Gupta, AO; Jan Boelens, J; Ebens, CL; Kurtzberg, J; Lund, TC; Smith, AR; Wagner, JE; Wynn, R; Blazar, BR; Orchard, PJ

Published Date

  • June 2021

Published In

Volume / Issue

  • 56 / 6

Start / End Page

  • 1238 - 1247

PubMed ID

  • 33441980

Pubmed Central ID

  • PMC8189901

Electronic International Standard Serial Number (EISSN)

  • 1476-5365

Digital Object Identifier (DOI)

  • 10.1038/s41409-020-01179-5


  • eng

Conference Location

  • England