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Technology Insight: hematopoietic stem cell transplantation for systemic rheumatic disease

Nature Clinical Practice Rheumatology volume 4, pages 184–191 (2008)Cite this article

Abstract

Hematopoietic stem cells (HSCs) have the capacity for self-renewal and the potential to differentiate into all types of hematopoietic and immune system cells. These features have been successfully used to treat a multitude of hematologic malignancies and nonmalignant diseases such as aplastic anemia, hemoglobinopathies, inborn errors of metabolism and congenital immunodeficiency states. The application of HSC transplantation has been expanded over the past decade to include immune-mediated diseases such as multiple sclerosis, treatment-refractory rheumatoid arthritis, systemic lupus erythematosus and systemic sclerosis. Transplantation of HSCs for the treatment of autoimmune diseases aims to fundamentally correct the dysregulated immune system, which could result in sustained clinical remission or potential cure. The use of this approach is currently restricted to clinical research, as there is no standard conditioning regimen to attain these aims in autoimmune diseases. HSC transplantation is associated with inherent morbidity and mortality, both treatment-related and disease-related, and selecting the correct group of patients with the best risk:benefit ratio is a challenging task.

Key Points

  • Hematopoietic stem cell transplantation (HSCT) is a potential treatment option for patients with autoimmune disease

  • Defining the eligibility criteria for patient selection with optimal risk:benefit ratio remains a challenge

  • Early referral for consultation to experienced transplant centers of patients who are not critically ill but who have aggressive, refractory autoimmune disease is essential to improve outcomes

  • To assess efficacy and safety, the outcome measures in HSCT trials for autoimmune disease must be well defined

  • HSCT studies in autoimmune diseases must be coupled with basic research to understand the fundamental immunologic changes that occur during this treatment

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Figure 1: Mechanics of autologous hematopoietic stem cell transplantation (HSCT).

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Acknowledgements

We would like to thank GG Illei and I Alevizos for their critical review and suggestions on the manuscript.

Author information

Authors and Affiliations

  1. NP Nikolov is a Staff Clinician and Clinical Investigator at the Sjögren's Syndrome Clinic, Molecular Physiology and Therapeutics Branch of the National Institute of Dental and Craniofacial Research, NIH, Bethesda, MD, USA.,

    Nikolay P Nikolov

  2. SZ Pavletic is Head of the Graft-Versus-Host and Autoimmunity Unit in the Experimental Transplantation and Immunology Branch of the National Cancer Institute, Bethesda.,

    Steven Z Pavletic

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Correspondence to Nikolay P Nikolov.

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Nikolov, N., Pavletic, S. Technology Insight: hematopoietic stem cell transplantation for systemic rheumatic disease. Nat Rev Rheumatol 4, 184–191 (2008). https://doi.org/10.1038/ncprheum0756

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