Metabolism, Inborn Errors Clinical Trial
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by
allogeneic peripheral blood stem cell or bone marrow transplantation in combination with
cyclosporine and mycophenolate mofetil for establishing mixed chimerism in patients with
inherited disorders.
II. Determine whether this regimen can establish mixed chimerism in these patients.
III. Determine whether mixed chimerism is sufficient to reverse disease symptoms in these
patients.
IV. Determine the safety of donor lymphocyte infusions to eliminate persistent disease in
these patients with mixed chimerism.
PROTOCOL OUTLINE: Patients receive fludarabine IV over 2 hours on days -4 to -2 followed by
total body irradiation and peripheral blood stem cell or bone marrow transplantation on day
0. Patients also receive oral or IV cyclosporine 2-3 times daily on days -3 to 50 (related
donor) or 100 (unrelated donor) and oral mycophenolate mofetil twice daily on days 0 to 28
(related donor) or 40 (unrelated donor).
Patients may also receive donor lymphocyte infusion for continued treatment of symptoms in
the event of mixed chimerism and in the absence of graft-versus-host disease.
Patients are followed weekly for 1 month, monthly for 2 years, and then annually thereafter.
;
Primary Purpose: Treatment
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