Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in 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