MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

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to 50 Years old
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

SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis

Clinics and Surgery Center (CSC)

Christen Ebens -
Christen Ebens
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