This document addresses hematopoietic stem cell transplantation in the treatment of the following conditions:

• Autoimmune Diseases
• Epithelial Ovarian Cancer
• Breast Cancer
• Malignant Astrocytomas and Gliomas
• Miscellaneous Solid Tumors in Adults

Note: For additional stem cell transplant information and criteria, see the applicable document(s):

• TRANS.00027 Hematopoietic Stem Cell Transplantation for Pediatric Solid Tumors
• TRANS.00030 Hematopoietic Stem Cell Transplantation for Germ Cell Tumors

Position Statement

Autoimmune Diseases

Medically Necessary:

A single autologous (ablative or non-myeloablative [mini-transplant]) hematopoietic stem cell transplantation is considered medically necessary for individuals with multiple sclerosis when all of the following criteria are met:

1. The transplant is used to treat relapsing-remitting multiple sclerosis; and
2. The individual is between 18 and 45 years of age with disease duration less than 10 years; and
3. The individual is disabled in at least one functional system but able to ambulate for 100 meters without aid or rest (expanded disability status scale [EDSS] score from 2.0 to 5.5); and
4. The individual has highly active and treatment resistant disease meeting criteria 1 and 2 below:
   1. Highly active disease as seen by 1.a or 1.b below:
      1. Two or more clinical relapses at separate times but within the previous 12 months; or
      2. One clinical relapse and one or more magnetic resonance imaging (MRI) lesions typical for MS (gadolinium-enhancing or T2-hyperintenase lesion), with the MRI lesion occurring at a separate time than the clinical relapse but both occurring within the previous 12 months; and
   2. Treatment resistant disease as seen by the disease activity meeting criteria D1 above occurring despite disease-modifying treatment (DMT) meeting all of the following requirements:
      1. Each relapse or episode of new MRI lesion(s) must occur after at least 3 months of treatment with a U.S. Food and Drug Administration-approved DMT; and
      2. At least 1 episode must be a clinical relapse, and MRI evidence of activity must include at least 2 unique or active lesions in the brain or spinal cord; and
      3. At least 1 of those episodes must occur after treatment with a DMT considered to be highly efficacious (natalizumab, ocrelizumab, rituximab, or alemtuzumab).

Investigational and Not Medically Necessary:

A single autologous (ablative or non-myeloablative [mini-transplant]) hematopoietic stem cell transplant is considered investigational and not medically necessary as a treatment of multiple sclerosis when the above criteria are not met, including for primary progressive or secondary progressive forms of multiple sclerosis.

A repeat autologous (ablative or non-myeloablative [mini-transplant]) hematopoietic stem cell transplant is considered investigational and not medically necessary as a treatment of relapsing-remitting multiple sclerosis.

An allogeneic (ablative or non-myeloablative [mini-transplant]) hematopoietic stem cell transplantation, or planned tandem is considered investigational and not medically necessary as a treatment of multiple sclerosis.

An autologous or allogeneic (ablative and non-myeloablative [mini-transplant]) hematopoietic stem cell transplantation, single or planned tandem is considered investigational and not medically necessary as a treatment of all other autoimmune diseases including, but not limited to:

• juvenile idiopathic arthritis;
• rheumatoid arthritis;
• systemic lupus erythematosus;
• systemic sclerosis

Epithelial Ovarian Cancer

Investigational and Not Medically Necessary:

An autologous or allogeneic (ablative and non-myeloablative [mini-transplant]) hematopoietic stem cell transplantation, single or planned tandem is considered investigational and not medically necessary as a treatment of epithelial ovarian cancer.

Breast Cancer

Investigational and Not Medically Necessary:

An autologous or allogeneic (ablative and non-myeloablative [mini-transplant]) hematopoietic stem cell transplantation, single or planned tandem is considered investigational and not medically necessary as a treatment of breast cancer.

Malignant Astrocytomas and Gliomas

Investigational and Not Medically Necessary:

An autologous or allogeneic (ablative and non-myeloablative [mini-transplant]) hematopoietic stem cell transplantation, single or planned tandem is considered investigational and not medically necessary as a treatment of malignant astrocytomas and gliomas, including both glioblastoma multiforme and oligodendroglioma.

Other Miscellaneous Solid Tumors in Adults

Investigational and Not Medically Necessary:

An autologous or allogeneic (ablative and non-myeloablative [mini-transplant]) hematopoietic stem cell transplantation, single or planned tandem is considered investigational and not medically necessary as a treatment of adult miscellaneous solid tumors, including but not limited to the following:

• Cancer of the bile duct;
• Cancer of the fallopian tubes;
• Cervical cancer;
• Colon cancer;
• Esophageal cancer;
• Gallbladder cancer;
• Lung cancer, any histology;
• Malignant melanoma;
• Neuroendocrine tumors;
• Nasopharyngeal cancer;
• Pancreas cancer;
• Paranasal sinus cancer;
• Prostate cancer;
• Rectal cancer;
• Renal cell cancer;
• Soft tissue sarcoma;
• Stomach cancer;
• Thyroid tumors;
• Tumors of the thymus;
• Uterine cancer;
• Undifferentiated tumors and tumors of unknown primary origin.