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Review
. 2021 Jun;56(6):1238-1247.
doi: 10.1038/s41409-020-01179-5. Epub 2021 Jan 13.

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

Ashish O Gupta  1 , Jaap Jan Boelens  2 , Christen L Ebens  3 , Joanne Kurtzberg  4 , Troy C Lund  3 , Angela R Smith  3 , John E Wagner  3 , Robert Wynn  5 , Bruce R Blazar  3 , Paul J Orchard  3
Affiliations
Review

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

Ashish O Gupta et al. Bone Marrow Transplant. 2021 Jun.

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.

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Conflict of interest statement

This expert consensus was based on information developed for and during a meeting supported by Magenta Therapeutics and subsequent data and literature review along with discussions. The content of this paper is solely that of the authors.

Figures

Fig. 1
Fig. 1
Proposed risk categories and treatment algorithm for immune cytopenias.
Fig. 2
Fig. 2
Current and future targets for intervention in immune mediated cytopenias.
See this image and copyright information in PMC

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