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Cord Blood Transplants for Nonmalignant Disorders: Data, Consensus, and Challenges

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Abstract

Successful use of cord blood transplant (CBT) in the treatment of children with a variety of nonmalignant disorders has been well documented in the last 25 years. Because of their relatively smaller size and weight, most children are able to have access to cord blood units with excellent cell dose. A large electronically searchable worldwide inventory of pretested cord blood units (CBU) allows for rapid donor identification and selection and timely transplantation. Additionally, lower graft-versus-host disease (GvHD), absence of risk to the donor, and higher probability of finding a donor for ethnic and racial minorities make CBT an attractive option in patients with nonmalignant diseases. This chapter will review the current status and cumulative experience of CBT for hemoglobinopathies, bone marrow failure syndromes, primary immunodeficiency diseases, and inherited metabolic disorders. Additionally, areas of consensus, novel therapeutic applications, and future challenges will be reviewed. Finally, the chapter will conclude that CBT is a proven therapeutic option for most children requiring transplantation for nonmalignant diseases and should be performed early in the course of the disease to achieve the best outcomes.

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  1. The Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, 1400 Morreene Road, Durham, NC, 27710, USA

    Vinod K. Prasad MD, FRCP (London)

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  1. Duke Cancer Center, Duke University, Durham, North Carolina, USA

    Mitchell Horwitz

  2. Duke Cancer Center, Duke University, Durham, North Carolina, USA

    Nelson Chao

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Prasad, V.K. (2017). Cord Blood Transplants for Nonmalignant Disorders: Data, Consensus, and Challenges. In: Horwitz, M., Chao, N. (eds) Cord Blood Transplantations. Advances and Controversies in Hematopoietic Transplantation and Cell Therapy. Springer, Cham. https://doi.org/10.1007/978-3-319-53628-6_7

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