Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

Brief Title

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies


Brief Summary

      OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation
      for patients with primary immunodeficiencies.

      II. Determine relevant outcomes of this treatment in these patients including quality of
      survival, extent of morbidity and mortality from complications of the treatment (e.g., graft
      versus host disease, regimen related toxicities, B- cell lymphoproliferative disease), and
      completeness of functional immunoreconstitution.
    

Detailed Description

      PROTOCOL OUTLINE: Patients with severe combined immunodeficiency (SCID) using a matched
      sibling donor receive allogeneic bone marrow or umbilical cord blood transplantation on day
      0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days
      1, 3, 6, and 11 and cyclosporine IV on days -3 to 50.

      Patients with SCID using donors other than histocompatible siblings, Wiskott Aldrich syndrome
      using a histocompatible sibling donor, Wiskott Aldrich syndrome and under 5 years of age
      using donors other than histocompatible siblings, X-linked CD40 ligand deficiency using a
      histocompatible sibling donor, X-linked CD40 ligand deficiency and under 5 years of age using
      donors other than histocompatible siblings, other primary immunodeficiencies without
      manifestations of hemophagocytosis using a histocompatible sibling donor, or other primary
      immunodeficiencies without manifestations of hemophagocytosis and under 5 years of age using
      donors other than histocompatible siblings receive busulfan IV over 2 hours every 6 hours on
      days -9 to -6, cyclophosphamide IV on days -5 to -2, and antithymocyte globulin (ATG) twice
      daily on days -4 to -1. Allogeneic bone marrow or umbilical cord blood transplantation takes
      place on day 0. Patients receive graft versus host disease (GVHD) prophylaxis with
      methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50.

      Patients with hemophagocytic lymphohistiocytosis, Chediak Higashi syndrome, X-linked
      lymphoproliferative syndrome, severe progressive Langerhans cell histiocytosis, or other
      primary immunodeficiencies with complications of hemophagocytosis receive busulfan IV over 2
      hours every 6 hours on days -9 to -6, cyclophosphamide IV over 2 hours on days -5 to -2,
      etoposide IV over 22 hours on days -5 to -3, and ATG IV twice daily on days -2, -1, 1, and 2.
      Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0. Patients
      receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days 1, 3, 6,
      and 11 and cyclosporine IV on days -3 to 50.

      Patients with Wiskott Aldrich syndrome or other primary immunodeficiencies without
      manifestations of hemophagocytosis, who are over 5 years of age and using donors other than
      histocompatible siblings, receive busulfan IV over 2 hours every 6 hours on days -6 and -5,
      cyclophosphamide IV over 2 hours on days -4 and -3, total body irradiation on day -2, and ATG
      IV over 2 hours twice daily on days -2, -1, 2, and 3. Allogeneic bone marrow or umbilical
      cord blood transplantation takes place on days 0 and 1. Patients receive GVHD prophylaxis
      with methylprednisolone IV every 12 hours on days 2-21, oral prednisone every 12 hours on
      days 22-100 and then tapered off over days 101 to 128, and cyclosporine IV over 2 hours every
      8-12 hours on days -3 to 100.

      All patients are followed as determined by their primary physician.
    


Study Type

Interventional




Condition

Immunologic Deficiency Syndromes

Intervention

anti-thymocyte globulin


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug


Start Date

March 2000

Completion Date

December 2002

Primary Completion Date

December 2002

Eligibility Criteria

        -  Severe combined immunodeficiency All ages with histocompatible sibling donors or with
             other donors OR Wiskott Aldrich syndrome All ages with histocompatible sibling donors
             or with other donors OR X-linked CD40 ligand deficiency All ages with histocompatible
             sibling donors OR Under 5 years of age with donors other than histocompatible siblings
             OR Other primary immunodeficiencies without manifestations of hemophagocytosis All
             ages with histocompatible sibling donors or with other donors OR Hemophagocytic
             lymphohistiocytosis (HLH) Familial erythrophagocytic lymphohistiocytosis (FEL),
             familial HLH (FHLH), recurrent virus-associated hemophagocytic syndrome (VAHS) All
             ages with related or unrelated donors OR Chediak Higashi syndrome All ages with
             related or unrelated donors OR X-linked lymphoproliferative syndrome All ages with
             related or unrelated donors OR Other primary immunodeficiencies with complication of
             hemophagocytosis All ages with related or unrelated donors OR Severe progressive
             Langerhans cell histiocytosis All ages with related or unrelated donors
      

Gender

All

Ages

N/A - 35 Years

Accepts Healthy Volunteers

No

Contacts

K. Scott Baker, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00006054

Organization ID

199/15104

Secondary IDs

UMN-MT-1995-26


Study Sponsor

Fairview University Medical Center


Study Sponsor

K. Scott Baker, Study Chair, Fairview University Medical Center


Verification Date

October 2003