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Matched Sibling Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease

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Sickle Cell Disease and Hematopoietic Stem Cell Transplantation

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Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) for sickle cell disease has been known to cure the sickle cell phenotype for over 30 years. The worldwide experience with HSCT for sickle cell disease is predominantly with matched-related donors (MRDs). Conditioning regimens have historically been myeloablative, still considered by many to be standard of care when HSCT is performed for this disease. Efforts are underway to reduce the acute and long-term toxicities with myeloablative agents—notably the risk of infertility—by studying reduced intensity conditioning approaches.

In this chapter, the initial reports of MRD HSCT for sickle cell disease are presented followed by subsequent larger multi-center clinical trials. The data describing both myeloablative and reduced intensity conditioning approaches will be discussed. The relevance of hematopoietic stem cell source, historic and current HSCT eligibility criteria for children and adolescents with sickle cell disease as well as future research directions will be reviewed.

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Abbreviations

AML:

Acute myelogenous leukemia

ATG:

Antithymocyte globulin

AUC:

Area under the curve

EFS:

Event-free survival

GVHD:

Graft-versus-host disease

HLA:

Human leukocyte antigen

HSCT:

Hematopoietic stem cell transplant

HU:

Hydroxyurea

MDC:

Mixed donor chimerism

MRD:

Matched-related donor

MRI:

Magnetic resonance imaging

PK:

Pharmacokinetics

rATG:

Rabbit antithymocyte globulin

SCD:

Sickle cell disease

SOS:

Sinusoidal obstruction syndrome

STAR:

Sickle Transplant Alliance for Research

TBI:

Total body irradiation

UCB:

Umbilical cord blood

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Author information

Authors and Affiliations

  1. Departments of Oncology and Paediatrics, Alberta Children’s Hospital, University of Calgary, Calgary, AB, Canada

    Gregory M. T. Guilcher

  2. Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, USA

    John T. Horan

Authors
  1. Gregory M. T. Guilcher
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  2. John T. Horan
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Correspondence to Gregory M. T. Guilcher .

Editor information

Editors and Affiliations

  1. Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA

    Emily Riehm Meier

  2. Division of Blood and Marrow Transplantation, Children’s National Medical Center, Washington, DC, USA

    Allistair Abraham

  3. Department of Pathology and Laboratory Medicine, Emory University School of Medicine, Atlanta, Georgia, USA

    Ross M. Fasano

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Guilcher, G.M.T., Horan, J.T. (2018). Matched Sibling Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease. In: Meier, E., Abraham, A., Fasano, R. (eds) Sickle Cell Disease and Hematopoietic Stem Cell Transplantation . Springer, Cham. https://doi.org/10.1007/978-3-319-62328-3_12

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  • DOI: https://doi.org/10.1007/978-3-319-62328-3_12

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  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-319-62327-6

  • Online ISBN: 978-3-319-62328-3

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