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).
Hematopoietic stem cell transplantation (autologous or allogeneic) for the treatment of autoimmune diseases, including, but not limited to, the following is considered investigational:
Juvenile idiopathic and rheumatoid arthritis
Systemic lupus erythematosus
Type 1 diabetes mellitus
Chronic inflammatory demyelinating polyneuropathy
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We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan, the express terms of the health plan will govern.
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 https://www.asbmt.org/practice-resources/practice-guidelines.
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. CMS.gov. NCD for stem cell transplantation (110.23). Retrieved July 3, 2017 from https://www.cms.gov/medicare-coverage-database/details/ncd-details.aspx?NCDId=366&ncdver=1&DocID=110.23&from2=search.asp&bc=gAAAABAAAAAAAA%3d%3d&.
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 http://www.nature.com/bmt/journal/v47/n6/full/bmt2011185a.html.
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 https://www.nature.com/articles/bmt2014251.
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)
ORIGINAL EFFECTIVE DATE: 4/10/2014
MOST RECENT REVIEW DATE: 8/9/2018
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