Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

Graft Versus Host Disease Clinical Trial

Status Terminated

Clinical Trial 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.

Clinical Trial 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 Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Agammaglobulinemia
• Chediak-Higashi Syndrome
• Chronic Granulomatous Disease
• Common Variable Immunodeficiency
• Familial Erythrophagocytic Lymphohistiocytosis
• Genetic Diseases, X-Linked
• Graft Versus Host Disease
• Graft vs Host Disease
• Granuloma
• Granulomatous Disease, Chronic
• Hemophagocytic Lymphohistiocytosis
• Immunologic Deficiency Syndromes
• Leukocyte Adhesion Deficiency Syndrome
• Leukocyte-Adhesion Deficiency Syndrome
• Lymphohistiocytosis, Hemophagocytic
• Lymphoproliferative Disorders
• Severe Combined Immunodeficiency
• Syndrome
• Virus-Associated Hemophagocytic Syndrome
• Wiskott-Aldrich Syndrome
• X-linked Agammaglobulinemia
• X-linked Hyper IgM Syndrome
• X-Linked Lymphoproliferative Syndrome

• NCT number: NCT00006054
• Study type: Interventional
• Source: Office of Rare Diseases (ORD)
• Status: Terminated
• Phase: N/A
• Start date: March 2000
• Completion date: December 2002