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Hematopoietic Stem Cell Transplant in the Treatment of Autoimmune Endocrine Disease

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Stem Cells in Endocrinology

Part of the book series: Contemporary Endocrinology ((COE))

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Abstract

The recent successful application of hematopoietic stem cell transplantation (HSCT) to the treatment of severe or refractory rheumatic autoimmune diseases has led to speculation of whether stem cell transplantation might benefit patients with endocrine autoimmune diseases. Autoimmune type 1 diabetes mellitus is a prime candidate for hematopoietic stem cell therapies because of both the severity of the disease and associated long-term complications of chronic hyperglycemia. HSCT in patients with recent-onset type 1 diabetes may prevent further autoimmune-mediated destruction of islet β cells and thus decrease acute and chronic risks of hyperglycemia. In patients with type 1 diabetes, however, pancreas or islet transplant is necessary to restore endogenous insulin production, and current protocols require the use of chronic immunosuppressive therapies to control autoimmunity and prevent allograft rejection. HSCT may overcome limitations associated with pancreas and islet transplant by inducing immunologic tolerance to islet β cells. Nevertheless, autologous HSCT is associated with autoimmune disease relapse, and correction of genetic susceptibility to the development of type 1 diabetes would require allogeneic HSCT with human leukocyte antigen (HLA)-DQ or DR (HLA class II) mismatched donors, which leads to a high risk of acute graft versus host disease. In this chapter, we examine both the potential therapeutic benefits and risks of HSCT for treatment of autoimmune type 1 diabetes mellitus as a model for HSCT in the treatment of endocrine autoimmune diseases.

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  1. Whittier Institute, University of California San Diego, La Jolla, CA

    Jody Schumacher PhD

  2. Cancer Center, University of California San Diego, La Jolla, CA

    Ewa Carrier MD

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  1. Jody Schumacher PhD
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  1. Division of Endocrinology, Diabetes and Clinical Nutrition, Department of Medicine, Oregon Health and Sciences University, Portland, Oregon

    Linda B. Lester MS, MD

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Schumacher, J., Carrier, E. (2005). Hematopoietic Stem Cell Transplant in the Treatment of Autoimmune Endocrine Disease. In: Lester, L.B. (eds) Stem Cells in Endocrinology. Contemporary Endocrinology. Humana Press. https://doi.org/10.1385/1-59259-900-1:221

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  • DOI: https://doi.org/10.1385/1-59259-900-1:221

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