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

doi:10.1038/s41409-020-01179-5

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

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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.

Original language	English (US)
Pages (from-to)	1238-1247
Number of pages	10
Journal	Bone marrow transplantation
Volume	56
Issue number	6
DOIs	https://doi.org/10.1038/s41409-020-01179-5
State	Published - Jun 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.

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title = "Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders",

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.",

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

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: {\textcopyright} 2021, The Author(s), under exclusive licence to Springer Nature Limited.",

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AU - Gupta, Ashish O.

AU - Jan Boelens, Jaap

AU - Ebens, Christen L.

AU - Kurtzberg, Joanne

AU - Lund, Troy C.

AU - Smith, Angela R.

AU - Wagner, John E.

AU - Wynn, Robert

AU - Blazar, Bruce R.

AU - Orchard, Paul J.

N1 - 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.

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N2 - 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.

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

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