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

Ashish O. Gupta, Jaap Jan Boelens, Christen L. Ebens, Joanne Kurtzberg, Troy C. Lund, Angela R. Smith, John E. Wagner, Robert Wynn, Bruce R. Blazar, Paul J. Orchard

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


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 languageEnglish (US)
JournalBone marrow transplantation
Early online dateJan 13 2021
StateE-pub ahead of print - Jan 13 2021

Bibliographical note

Funding 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.

Publisher Copyright:
© 2021, The Author(s), under exclusive licence to Springer Nature Limited.

Copyright 2021 Elsevier B.V., All rights reserved.

PubMed: MeSH publication types

  • Journal Article
  • Review

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