Allogeneic hematopoietic cell transplantation (allo-HCT) involves the intravenous (IV) infusion of allogeneic (donor) stem cells to reestablish hematopoietic function in individuals whose bone marrow or immune system is damaged or defective. They can be harvested from bone marrow, peripheral blood, or umbilical cord blood and placenta shortly after delivery of neonates.
Allo-HCT may be considered medically necessary for selected individuals with the following disorders:
Allo-HCT that does not meet the criteria of this policy is considered not medically necessary.
The following guidelines list the immunodeficiencies that have been successfully treated by allo-HCT:
For inherited metabolic disorders, allo-HCT has been proven effective in some cases of Hurler, Maroteaux-Lamy, and Sly syndromes, childhood onset cerebral X-linked adrenoleukodystrophy, globoid-cell leukodystrophy, metachromatic leukodystrophy, alpha-mannosidosis, and aspartylglucosaminuria. Allogeneic HCT is possibly effective for fucosidosis, Gaucher types 1 and 3, Farber lipogranulomatosis, galactosialidosis, GM1, gangliosidosis, mucolipidosis II (I-cell disease), multiple sulfatase deficiency, Niemann-Pick, neuronal ceroid lipofuscinosis, sialidosis, and Wolman disease. Allogeneic HCT has not been effective in Hunter, Sanfilippo, or Morquio syndromes.
The experience with reduced-intensity conditioning (RIC) and allo-HCT for the diseases listed in this policy has been limited to small numbers of individuals and have yielded mixed results, depending upon the disease category. In general, the results have been most promising in the bone marrow failure syndromes and primary immunodeficiencies. In the hemoglobinopathies, success has been hampered by difficulties with high rates of graft rejection, and in adult individuals, severe graft-versus-host-disease (GVHD).