Stem-Cell Transplant for Autoimmune Disorders
© Blue Cross and Blue Shield of Montana
Current Effective Date:
December 27, 2013
Original Effective Date:
April 18, 2013
September 27, 2013
September 16, 2013
The use of hematopoietic stem-cell transplantation (HSCT) has been investigated for treatment of patients with autoimmune disorders. Hematopoietic stem cells are infused to restore bone marrow function following cytotoxic doses of chemotherapeutic agents with or without whole body radiation therapy.
Autoimmune diseases represent a heterogeneous group of immune-mediated disorders, with the most common types being multiple sclerosis (MS), rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), and systemic sclerosis/scleroderma. These include over 40 disorders that are recognized as having an autoimmune pathogenesis, ranging from insulin dependent diabetes mellitus, rheumatoid arthritis and other connective tissue disorders to multiple sclerosis. Immune suppression is a common treatment strategy for many of these diseases, particularly the rheumatic diseases (such as RA, and scleroderma). The National Institutes of Health (NIH) estimates 5–8% of Americans have an autoimmune disorder. Some of the more common types of autoimmune disorders include but are not limited to:
- Multiple sclerosis (MS) is an autoimmune condition in which the immune system attacks the central nervous system, leading to demyelination (the destructive removal of myelin, an insulating and protective fatty protein which sheaths nerve cells).
- Juvenile idiopathic rheumatoid arthritis (JRA) may also be known as juvenile idiopathic arthritis (JIA), is a chronic, persistent, systemic autoimmune disorder of children, as it commonly occurs from the ages of 7 to 12, and before 16. JRA is a subset of arthritis seen in children and the most common form. Since there is no defined cause of the arthritis or inflammation of the synovium of the joint(s), it is referred to as idiopathic. Researchers sometimes refer JIA as juvenile chronic arthritis (JCA), which is a term that is not precise as JIA refers to all types of childhood arthritis. So, terminology for this condition is evolving.
- Rheumatoid arthritis (RA) is a chronic, systemic autoimmune disorder that causes the immune system to attack the joints, where it causes inflammation (arthritis) and destruction. It can also damage some organs, such as the lungs and skin.
- Systemic lupus erythematosus (SLE) may also be known as lupus, and is an acquired chronic autoimmune connective tissue disorder manifested by multi-organ involvement and potentially fatal. Women and minorities are disproportionately affected. SLE is most common in women of child-bearing age although it has been reported in both extremes of life (e.g., diagnosed in infants and in the tenth decade of life).
- Systemic sclerosis (scleroderma) is a chronic autoimmune disease characterized by a hardening or sclerosis in the skin or other organs.
- Type I diabetes mellitus (Type I DM, TIDM) is formerly known as insulin dependent or juvenile diabetes (IDDM) that results from autoimmune destruction of insulin-producing beta cells of the pancreas becoming a metabolic disease. TIDM can be further classified as immune-mediated or idiopathic. The majority of TIDM is of the immune-mediated nature, in which beta cell loss is a T-cell-mediated autoimmune attack.
The pathogenesis of autoimmune diseases is not well-understood but appears to involve underlying genetic susceptibility and environmental factors that lead to loss of self-tolerance, culminating in tissue damage by the patient’s own immune system (T cells).
Immune suppression is a common treatment strategy for many of these diseases, particularly the rheumatic diseases (e.g., RA, SLE, and scleroderma). Most patients with autoimmune disorders respond to conventional therapies, which consist of anti-inflammatory agents, immunosuppressants, and immunomodulating drugs. However, these drugs are not curative, and a proportion of patients will have severe, recalcitrant, or rapidly progressive disease. It is in this group of patients with severe autoimmune disease that alternative therapies have been sought, including hematopoietic stem-cell transplantation (HSCT). HSCT in autoimmune disorders raises the question of whether ablating and “resetting” the immune system can alter the disease process and sustain remission and possibly lead to cure. (1)
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||New 2013 BCBSMT medical policy. Considered investigational.|
||Policy formatting and language revised. Document updated with literature review. The following was added: 1) Donor leukocyte infusion and hematopoietic progenitor cell boost are considered experimental, investigational and unproven; and 2) Any use of short tandem repeat (STR) markers for the treatment of autoimmune disorders is considered experimental, investigational and unproven. Otherwise, coverage unchanged. Description and Rationale significantly revised. |