Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Description

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

Conditions

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

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Study Overview

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Study Details

Study overview

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

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Condition
SCID
Intervention / Treatment

-

Contacts and Locations

Minneapolis

Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States, 55455

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * pregnant or breastfeeding
  • * active, uncontrolled infection and/or HIV positive
  • * acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy

Ages Eligible for Study

to 50 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Masonic Cancer Center, University of Minnesota,

Christen Ebens, MD, PRINCIPAL_INVESTIGATOR, Masonic Cancer Center, University of Minnesota

Study Record Dates

2026-12