Hematopoietic stem cell transplantation (HSCT) refers to a procedure in which hematopoietic stem cells are infused to restore bone marrow function in patients who receive bone-marrow-toxic doses of drugs. Hematopoietic stem cells may be obtained from the transplant recipient (autologous HCT) or from a donor (allogeneic HCT). These cells are harvested from bone marrow, peripheral blood, or umbilical cord blood shortly after delivery of neonates.

Autoimmune diseases represent a heterogeneous group of immune-mediated disorders; some of the most common types include multiple sclerosis (MS), rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), and systemic sclerosis/scleroderma. The National Institutes of Health estimate that 5–8% of Americans have an autoimmune disorder. The pathogenesis of autoimmune diseases is not well understood but appears to involve underlying genetic susceptibility and environmental factors that lead to loss tissue damage caused by the patient’s own immune system.

Most patients with autoimmune disorders respond to conventional therapies, which consist of anti-inflammatory agents, immunosuppressants, and immune-modulating 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 HSCT.

Scleroderma: involves the hardening and tightening of skin and connective tissue. It is not a single condition but a spectrum of rare diseases that vary widely in severity. In some patients, it may affect only the skin. In others it may affect additional organs including blood vessels. Systemic sclerosis refers to forms of scleroderma that affect internal organs.

I.    Systemic Sclerosis/Scleroderma

• Autologous hematopoietic stem cell transplantation may be considered MEDICALLY NECESSARY AND APPROPRIATE for treatment of systemic sclerosis/scleroderma when all of the following are met:
  • Adult patients age 18 to 69 years; AND
  • Disease duration ≤ 4 years; AND
  • Rapidly progressing systemic sclerosis/scleroderma at risk of organ failure; AND
  • ONE of the following:
    • Patient meets transplanting institution's selection criteria for HSCT in treatment of systemic sclerosis/scleroderma;
    •  OR
    • If center does not have selection criteria for this condition, NONE of the following comorbid conditions are present:
      1. Cardiac disease: left ventricular ejection fraction (LVEF) of less than 50%
      2. Pulmonary arterial hypertension: mean pulmonary artery pressure > 20 mm Hg
      3. Renal disease: creatinine clearance of less than 40 mL/min
• Allogeneic hematopoietic stem cell transplantation as a treatment of systemic sclerosis/scleroderma is considered EXPERIMENTAL/INVESTIGATIVE due to a lack of evidence demonstrating an impact on improved health outcomes.

II.   Other Autoimmune Conditions

Autologous or allogeneic hematopoietic stem cell transplantation is considered EXPERIMENTAL/ INVESTIGATIVE as a treatment of all other autoimmune diseases, including but not limited to the following, due to a lack of evidence demonstrating an impact on improved health outcomes:

• Multiple sclerosis (MS);
• Rheumatoid arthritis (RA);
• Juvenile idiopathic arthritis;
• Systemic lupus erythematosus (SLE);
• Type 1 diabetes mellitus;
• Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

III.   Repeat Transplant

A second (allogeneic or autologous) hematopoietic stem-cell transplantation (HSCT) is considered EXPERIMENTAL/INVESTIGATIVE for all indications, due to the lack of clinical evidence demonstrating an impact on improved health outcomes. 

Documentation Submission:

Link to Transplant Pre-Authorization Form: https://www.bluecrossmn.com/sites/default/files/DAM/2022-06/x16519r04-transplant-prior-authorization-request-form.pdf