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

Bone Marrow Transplantation volume 56, pages 1238–1247 (2021)Cite this article

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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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Acknowledgements

This expert consensus was based on information developed for and during a meeting supported by Magenta Therapeutics. The content of this paper is solely that of the authors. Beth Kamp, PharmD, a medical writer supported by funding from Magenta Therapeutics provided initial editorial assistance to the authors during preparation of this paper.

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Authors and Affiliations

  1. Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA

    Ashish O. Gupta, Christen L. Ebens, Troy C. Lund, Angela R. Smith, John E. Wagner, Bruce R. Blazar & Paul J. Orchard

  2. Stem Cell Transplantation and Cellular Therapy, MSK Kids, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Jaap Jan Boelens

  3. Marcus Center For Cellular Cures, Duke University School of Medicine, Durham, NC, USA

    Joanne Kurtzberg

  4. Department of Blood and Marrow Transplantation, Royal Manchester Children’s Hospital, Manchester, UK

    Robert Wynn

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Contributions

AG, JB, CE, TL, PO, AS, JW, and RW provided review of the literature and contributed to the development of the content of the paper. BB provided critical input regarding pathophysiology of IMC and critical review of paper. AG wrote the paper with contributions from all authors.

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Correspondence to Ashish O. Gupta.

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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.

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Gupta, A.O., Jan Boelens, J., Ebens, C.L. et al. Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders. Bone Marrow Transplant 56, 1238–1247 (2021). https://doi.org/10.1038/s41409-020-01179-5

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