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American Academy of Pediatrics
Article

Hematopoietic Stem Cell Transplantation: A Neonatal Perspective

Erin Hall and Shalini Shenoy
NeoReviews June 2019, 20 (6) e336-e345; DOI: https://doi.org/10.1542/neo.20-6-e336
Erin Hall
*Department of Pediatrics, Division of Hematology/Oncology/Bone Marrow Transplantation, Children’s Mercy Hospital, Kansas City, MO
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Shalini Shenoy
†Department of Pediatrics, Division of Hematology/Oncology/Bone Marrow Transplantation, Washington University School of Medicine and St. Louis Children’s Hospital, St. Louis, MO
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  • Abbreviations:
    AML:
    acute myeloid leukemia
    ANC:
    absolute neutrophil count
    CAMT:
    congenital amegakaryocytic thrombocytopenia
    fHLH:
    familial (or primary) hemophagocytic lymphohistiocytosis
    GALC:
    galactocerebrosidase
    G-CSF:
    granulocyte colony-stimulating factor
    GVHD:
    graft-versus-host disease
    HLA:
    human leukocyte antigens
    HSCT:
    hematopoietic stem cell transplantation
    IMO:
    infantile malignant osteopetrosis
    IPEX:
    immune dysregulation, polyendocrinopathy, enteropathy X-linked
    MDS:
    myelodysplastic syndrome
    MHC:
    major histocompatibility complex
    NK:
    natural killer
    NMA:
    nonmyeloablative
    NMD:
    nonmalignant disorders
    RIC:
    reduced intensity conditioning
    SCID:
    severe combined immunodeficiency disorder
    SCN:
    severe congenital neutropenia
  • Abstract

    Allogeneic hematopoietic stem cell transplantation (HSCT) is indicated in various nonmalignant disorders that arise from genetic, hematopoietic, and immune system defects. Many of the disorders described here have life-threatening consequences in the absence of HSCT, a curative intervention. However, timing and approach to HSCT vary by disorder and optimum results are achieved by performing transplantation before irreversible disease-related morbidity or infectious complications. This article details the principles of HSCT in the very young, lists indications, and explores the factors that contribute to successful outcomes based on transplantation and disease-related nuances. It provides an overview into the HSCT realm from a neonatologist's perspective, describes the current status of transplantation for relevant disorders of infancy, and provides a glimpse into future efforts at improving on current success.

    • Copyright © 2019 by the American Academy of Pediatrics

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    Vol. 20, Issue 6
    1 Jun 2019
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    Hematopoietic Stem Cell Transplantation: A Neonatal Perspective
    Erin Hall, Shalini Shenoy
    NeoReviews Jun 2019, 20 (6) e336-e345; DOI: 10.1542/neo.20-6-e336

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    Hematopoietic Stem Cell Transplantation: A Neonatal Perspective
    Erin Hall, Shalini Shenoy
    NeoReviews Jun 2019, 20 (6) e336-e345; DOI: 10.1542/neo.20-6-e336
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    • Article
      • Abstract
      • Education Gaps
      • Objectives
      • Introduction
      • Donor Selection
      • Conditioning Therapy
      • GVHD Prophylaxis
      • Immune Reconstitution
      • Transplantation Approaches Based on Disease
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    • Gastroesophageal Reflux Disease in Neonates: Facts and Figures
    • Fetal Heart Rate Tracing Category II: A Broad Category in Need of Stratification
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