BlueCross BlueShield of Tennessee Medical Policy Manual

Hematopoietic Stem Cell Transplantation for Autoimmune Diseases


Autoimmune diseases represent a heterogeneous group of immune-mediated disorders, including rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), systemic sclerosis/scleroderma, and chronic immune demyelinating polyneuropathy (CIDP).  The National Institute of Health (NIH) estimates that 5-8% of Americans have an autoimmune disorder.  Individuals with autoimmune disorders typically respond to conventional therapies.  However, these drugs are not curative, and a proportion of individuals will have severe, recalcitrant, or rapidly progressive disease.  It is in this group of individuals with severe autoimmune disease that alternate therapies have been sought, including stem-cell transplantation (HSCT).

Hematopoietic stem-cell transplantation (HSCT) refers to a procedure in which hematopoietic stem cells are infused to restore bone marrow function in individuals who receive bone-marrow-toxic doses of cytotoxic drugs with or without whole-body radiation therapy.  Hematopoietic stem cells may be obtained from the transplant recipient (autologous HSCT) or from a donor (alloegeneic HSCT). 




The field of hematopoietic stem cell therapy (HSCT) for the treatment of autoimmune diseases is evolving with multiple randomized and nonrandomized trials currently ongoing.  The results of these trials will assist in defining the role of HSCT in the management of these diseases.


American Society for Blood and Marrow Transplantation. (2015, November). Indications for autologous and allogeneic hematopoietic cell transplantation: guidelines from the American Society for Blood and Marrow Transplantation. Retrieved July 10, 2018 from

BlueCross BlueShield Association. Medical Policy Reference Manual. (1:2018). Hematopoietic cell transplantation for autoimmune diseases (8.01.25). Retrieved July 10, 2018 from BlueWeb. (40 articles and/or guidelines reviewed)

Centers for Medicare & Medicaid Services. NCD for stem cell transplantation (110.23). Retrieved July 3, 2017 from

El-Badawy, A., & El-Badri, N. (2016). Clinical efficacy of stem cell therapy for diabetes mellitus: a meta-analysis. PLOS ONE, 11 (4), e0151938. (Level 2 evidence)

European Group for Blood and Marrow Transplantation. (2012). Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Retrieved April 30, 2015 from

European Society for Blood and Marrow Transplantation. (2015). SCT for severe autoimmune diseases: consensus guidelines of the European society for blood and marrow transplantation for immune monitoring and biobanking. Retrieved July 10, 2018 from

Leng, X.M., Jiang, Y., Zhou, D.B., Tian, X.P., Li, T.S., Wang, S.J., et al. (2017). Good outcome of severe lupus patients with high-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation: a 10-year follow-up study. Clinical and Experimental Rheumatology, 35 (3), 494-499. Abstract retrieved July 10, 2018 from PubMed database.

Leone, A., Radin, M., Almarzooqi, A., Al-Saleh, J., Roccatello, D., Sciascia, S., & Khamashta, M. (2017). Autologous hematopoietic stem cell transplantation in systemic lupus erythematosus and antiphospholipid syndrome: a systematic review. Autoimmunity Reviews, 16 (5), 469-477. Abstract retrieved July 3, 2017 from PubMed database.

Snowden, J.A., Badoglio, M., Labopin, M., Giebel, S., McGrath, E., Marjanovic, Z., et al. (2017). Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases. Blood Advances, 1 (27), 2742-2755. (Level 2 evidence)

Van Laar, J. M., Farge, D., Sont, J., Naraghi, K., Marjanovic, Z., et al. (2014). Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis. The Journal of the American Medical Association, 311 (24), 2490-2498. (Level 1 evidence)

Young, A., & Khanna, D. (2015). Systemic sclerosis - a systematic review on therapeutic management from 2011-2014. Current Opinion Rheumatology, 27 (3), 241-248. (Level 2 evidence)




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