Allogeneic hematopoietic cell transplantation is considered medically necessary for select patients with the following disorders.
Hemoglobinopathies
Sickle cell anemia for children or young adults with either a history of prior stroke or at increased risk of stroke or end-organ damage.
Homozygous b-thalassemia (ie, thalassemia major).
Bone Marrow Failure Syndromes
Aplastic anemia including hereditary (including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome, Diamond-Blackfan syndrome) or acquired (eg, secondary to drug or toxin exposure) forms.
Primary Immunodeficiencies
Absent or defective T-cell function (eg, severe combined immunodeficiency, Wiskott-Aldrich syndrome, X-linked lymphoproliferative syndrome) or
Absent or defective natural killer function (eg, Chédiak-Higashi syndrome) or
Absent or defective neutrophil function (eg, Kostmann syndrome, chronic granulomatous disease, leukocyte adhesion defect).
(See Guideline 1.)
Inherited Metabolic Diseases
Lysosomal and peroxisomal storage disorders except for Hunter, Sanfilippo, and Morquio syndromes.
(See Guideline 2.)
Genetic Disorders Affecting Skeletal Tissue
Infantile malignant osteopetrosis (Albers-Schonberg disease or marble bone disease).
