Hematopoietic Cell Transplantation for the Treatment of Patients with Bone Marrow Failure Syndromes

  • Andrew C. Dietz
  • Michael A. Pulsipher
Part of the Pediatric Oncology book series (PEDIATRICO)


Bone marrow failure, either acquired or inherited, is often treated by hematopoietic cell transplantation (HCT). The treatment algorithm for severe aplastic anemia is going through an evolution as newer reduced intensity approaches aimed at balancing and minimizing graft failure, graft-versus-host disease (GVHD), and transplant-related morbidity and mortality have resulted in excellent outcomes with limited late effects. Many patients with inherited bone marrow failure syndromes have increased sensitivity related to the underlying diagnosis or because of comorbidities due to previous therapies. Tailored approaches to each syndrome have been developed, with most patients now surviving. Only now are we starting to understand long-term sequelae that these patients face as a result of underlying diagnosis, previous treatments, and HCT combined. This chapter reviews the current approaches to transplantation in severe aplastic anemia, Fanconi anemia, dyskeratosis congenita, Diamond-Blackfan anemia, Shwachman-Diamond syndrome, severe congenital neutropenia, and congenital amegakaryocytic thrombocytopenia. The forthcoming role of gene therapy is also discussed.


Hematopoietic cell transplantation Fanconi anemia Dyskeratosis congenita Diamond-Blackfan anemia Shwachman-Diamond syndrome Severe congenital neutropenia Congenital amegakaryocytic thrombocytopenia 


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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  • Andrew C. Dietz
    • 1
  • Michael A. Pulsipher
    • 1
  1. 1.Children’s Center for Cancer and Blood Disorders, Children’s Hospital Los AngelesUniversity of Southern CaliforniaLos AngelesUSA

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