Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Based on diagnosis and clinical history, a determination of the most appropriate regimen will
be made based on the following prep plans:

Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative
Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen

Inclusion Criteria:

- Diagnosis of immunodeficiency or histiocytic disorder including the following:

- Severe combined immunodeficiency (SCID - all variants)

- Second bone marrow transplant (BMT) for SCID (after graft rejection)

- Omenn's Syndrome

- Reticular dysgenesis

- Wiskott-Aldrich syndrome

- Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte
syndrome)

- Hyper IgM Syndrome (CD40 Ligand Deficiency)

- Common variable immunodeficiency (CVID) with severe phenotype

- Chronic Granulomatous Disease (CGD)

- Other severe Combined Immune Deficiencies (CID)

- Hemophagocytic Lymphohistiocytosis (HLH)

- X-linked Lymphoproliferative Disease (XLP)

- Chediak-Higashi Syndrome (CHS)

- Griscelli Syndrome

- Langerhans Cell Histiocytosis (LCH)

- Acceptable stem cell sources include:

- HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow

- HLA identical or up to a 1 antigen mismatched unrelated BM donor

- Sibling donor cord blood with acceptable HLA match and cell dose as per current
institutional standards

- Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum
cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines

- Double unrelated umbilical cord blood units that are:

- up to 2 antigen mismatched to the patient

- up to 2 antigen mismatched to each other

- minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated
cells/kg

- combined dose of both units must provide a total cell dose of ≥ 5 x 10^7
nucleated cells/kg

- Age: 0 to 50 years

- Adequate organ function and performance status.

Exclusion Criteria

- pregnant or breastfeeding

- active, uncontrolled infection and/or HIV positive

- acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on
biopsy