Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

• Status: Recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Alemtuzumab; Busulfan; Cyclophosphamide; Fludarabine; Fludarabine phosphate; Melphalan; Mesna; Vidarabine

Criteria

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