Anthem Blue Cross Connecticut TRANS.00029 Hematopoietic Stem Cell Transplantation for Genetic Diseases and Aplastic Anemias Form

This document addresses hematopoietic stem cell transplantation for genetic diseases and aplastic anemias. The document also addresses autologous hematopoietic stem cell mobilization and pheresis when conducted as part of the development of an ex vivo gene therapy for the treatment of a genetic disease.

Note: For additional information and criteria for umbilical cord transplants see:

• MED.00140 Gene Therapy for Beta Thalassemia
• MED.00142 Gene Therapy for Cerebral Adrenoleukodystrophy
• TRANS.00016 Umbilical Cord Blood Progenitor Cell Collection, Storage and Transplantation

Position Statement

Medically Necessary:

Allogeneic (ablative and non-myeloablative) hematopoietic stem cell transplantation is considered medically necessary for individuals with the following disorders. In addition, individuals with Aplastic Anemia, Sickle Cell Disease or Thalassemia should meet the Disease Specific Criteria below.

1. Bone Marrow Failure Syndromes
   1. Acquired aplastic anemia (drug, idiopathic, immune disorder, toxin or viral infection)
   2. Heritable bone marrow syndromes:
      1. Congenital amegakaryocytic thrombocytopenia (CAMT)
      2. Diamond-Blackfan anemia (DBA)
      3. Dyskeratosis congenita
      4. Fanconi’s anemia (FA)
      5. Schwachman-Diamond syndrome (SDS)
   3. Paroxysmal nocturnal hemoglobinuria (PNH)
2. Immunodeficiencies
   1. Hemophagocytic Lymphohistiocytosis (HLH)
   2. Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome
   3. Severe Combined Immunodeficiency (SCID)
   4. Wiskott-Aldrich Syndrome (WAS)
   5. X-linked lymphoproliferative syndrome
   6. Chediak-Higashi syndrome
   7. Primary granulocyte dysfunction
   8. Chronic granulomatous disease
3. Storage disorders
   1. Hurler Syndrome (MPS I)
   2. Hunter Syndrome (MPS II)
   3. Maroteaux-Lamy Syndrome (MPS VI)
   4. SanFilippo’s (MPS III)
   5. Gaucher disease
4. Leukodystrophies
   1. Adrenoleukodystrophy (ALD)
   2. Globoid Cell Leukodystrophy (GBL; Krabbe's disease)
   3. Metachromatic Leukodystrophy (MLD)
5. Hemoglobinopathies
   1. Sickle Cell Disease
   2. Thalassemia (homozygous beta-thalassemia)
6. Infantile malignant osteopetrosis (Albers-Schonberg disease or marble bone disease)
7. Other autosomal recessive disorders
   1. Leukocyte adhesion deficiencies
   2. Kostmann’s syndrome (severe congenital neutropenia, infantile genetic agranulocytosis)

A repeat allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplantation due to primary graft failure or failure to engraft is considered medically necessary.

Autologous hematopoietic stem cell mobilization and pheresis is considered medically necessary for the treatment of a genetic disease as part of the development of an FDA-approved ex vivo gene therapy (for example, betibeglogene autotemcel or elivaldogene autotemcel).

Investigational and Not Medically Necessary:

Allogeneic (ablative and non-myeloablative) hematopoietic stem cell transplantation is considered investigational and not medically necessary for the treatment of all genetic diseases not listed above including, but not limited to cystic fibrosis, and all acquired anemias not specifically identified above as medically necessary.

Autologous hematopoietic stem cell transplantation is considered investigational and not medically necessary for the treatment of all genetic diseases including, but not limited to cystic fibrosis, and all acquired anemias.

A planned tandem allogeneic hematopoietic stem cell transplantation or autologous hematopoietic stem cell transplantation is considered investigational and not medically necessary for all genetic diseases and for all acquired anemias.

A second or repeat allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplant due to persistent, progressive or relapsed disease is considered investigational and not medically necessary.

Hematopoietic stem cell harvesting for a future but unscheduled transplant is considered investigational and not medically necessary.

Autologous hematopoietic stem cell mobilization and pheresis is considered investigational and not medically necessary for the treatment of a genetic disease when the medically necessary criteria above are not met.

Disease Specific Criteria

Aplastic Anemia Criteria:

• One of the following:
  • 55 years of age or younger do not need to have failed immunosuppressive therapy
  • Older than 55 years of age, or non-HLA identical donor, have to have failed immunosuppressive therapy

Sickle Cell Criteria:

• Children less than 16 years of age with homozygous SS disease or S-B thalassemia and who have had at least one of the following complications:
  • Stroke or CNS hemorrhage
  • Clinical evidence of progressive neurologic deterioration, for example, abnormal cerebral MRI and arteriogram and impaired neuropsychiatric testing
  • Sickle cell lung disease, recurrent acute chest syndrome or a combination of both requiring hospitalization and exchange transfusions
  • Sickle cell nephropathy

Thalassemia Criteria:

• Thalassemia major only and
• Individuals less than or equal to 30 years of age and
• The presence of minimal or no portal fibrous or active hepatitis