Allogeneic Hematopoietic Cell Transplantation for Genetic Diseases and Acquired Anemia

Section: Surgery
Effective Date: November 01, 2019
Revised Date: October 14, 2019
Last Reviewed: September 26, 2019


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:

  • 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; or
    • Homozygous beta-thalassemia (i.e., thalassemia major); or
  • Bone marrow failure syndromes
    • Aplastic anemia including hereditary (including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond, Diamond-Blackfan) or acquired (e.g., secondary to drug or toxin exposure) forms; or
  • Primary immunodeficiencies
    • Absent or defective T-cell function (e.g., severe combined immunodeficiency, Wiskott-Aldrich syndrome, X-linked lymphoproliferative syndrome); or
    • Absent or defective natural killer function (e.g., Chediak-Higashi syndrome); or
    • Absent or defective neutrophil function (e.g., Kostmann syndrome, chronic granulomatous disease, leukocyte adhesion defect); or
  • Inherited metabolic disease
    • Lysosomal and peroxisomal storage disorders except Hunter, Sanfilippo, and Morquio syndromes; or
  • Genetic disorders affecting skeletal tissue
    • Infantile malignant osteopetrosis (Albers-Schonberg disease or marble bone disease).

Allo-HCT that does not meet the criteria of this policy is considered not medically necessary.

Procedure Codes

38205 38220 38221 38222 38230 38240 86812
86813 86816 86817 86821 S2140 S2142 S2150

The following guidelines list the immunodeficiencies that have been successfully treated by allo-HCT:

  • Lymphocyte immunodeficiencies
    • Adenosine deaminase deficiency
    • Artemis deficiency
    • Calcium channel deficiency
    • CD 40 ligand deficiency
    • Cernunnos/X-linked lymphoproliferative disease deficiency
    • CHARGE syndrome with immune deficiency
    • Common gamma chain deficiency
    • Deficiencies in CD45, CD3, CD8
    • DiGeorge syndrome
    • DNA ligase IV deficiency syndrome
    • Interleukin-7 receptor alpha deficiency
    • Janus-associated kinase 3 (JAK3) deficiency
    • Major histocompatibility class II deficiency
    • Omenn syndrome
    • Purine nucleoside phosphorylase deficiency
    • Recombinase-activating gene (RAG) 1/2 deficiency
    • Reticular dysgenesis
    • Winged helix deficiency
    • Wiskott-Aldrich syndrome
    • X-linked lymphoproliferative disease
    • Zeta-chain-associated protein-70 (ZAP-70) deficiency
  • Phagocytic deficiencies
    • Chediak-Higashi syndrome
    • Chronic granulomatous disease
    • Hemophagocytic lymphohistiocytosis
    • Griscelli syndrome, type 2
    • Interferon-gamma receptor deficiencies
    • Leukocyte adhesion deficiency
    • Severe congenital neutropenias
    • Shwachman-Diamond syndrome
  • Other immunodeficiencies
    • Autoimmune lymphoproliferative syndrome
    • Cartilage hair hypoplasia
    • CD25 deficiency
    • Hyper IgD and IgE syndromes
    • Immunodeficiency, centromeric instability, and facial dysmorphism syndrome (ICF syndrome)
    • Immunodysregulation polyendocrinopathy enteropathy X-linked syndrome (IPEX syndrome)
    • Nuclear factor –KB (NF-KB) essential modulator deficiency (NEMO deficiency)
    • NF-κB inhibitor, NF-KB-a deficiency
    • Nijmegen breakage syndrome

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).

Diagnosis Codes

D56.0 D56.1 D56.2 D56.3 D56.5 D56.8 D56.9
D57.00 D57.01 D57.02 D57.1 D57.20 D57.211 D57.212
D57.219 D57.40 D57.411 D57.412 D57.419 D57.80 D57.811
D57.812 D57.819 D60.0 D60.1 D60.8 D60.9 D61.01
D61.09 D61.1 D61.2 D61.3 D61.810 D61.811 D61.818
D61.82 D61.89 D61.9 D70.0 D81.0 D81.1 D81.2
D81.30 D81.31 D81.32 D81.39 D81.6 D81.7 D81.89
D81.9 D82.0 E75.21 E75.22 E75.240 E75.241 E75.242
E75.243 E75.248 E75.249 E75.3 E76.01 E76.02 E76.03
E76.1 E76.210 E76.211 E76.219 E76.22 E76.29 E76.3
E76.8 E76.9 E77.0 E77.1 E77.8 E77.9 Q78.2