Low-Dose Total Body Irradiation and Fludarabine Followed by Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter Trial
I. To determine whether donor chimerism can be achieved in patients with Fanconi anemia
receiving marrow or peripheral blood stem cell (PBSC) grafts from unrelated donors following
low dose total body irradiation (TBI), fludarabine (fludarabine phosphate), mycophenolate
mofetil, and cyclosporine.
II. To determine the lowest dose of TBI necessary to achieve donor chimerism in at least 80%
III. To determine the incidence of severe regimen-related toxicity.
I. To determine the survival of Fanconi anemia patients transplanted with unrelated donor
marrow or PBSC grafts after conditioning with a non-myeloablative regimen.
II. To determine the incidence and severity of graft-vs-host disease (GVHD) incurred with
unrelated bone marrow or PBSC grafts transplant patients with Fanconi anemia.
III. To determine if mixed chimerism results in amelioration of symptoms associated with
bone marrow failure in patients with Fanconi anemia.
NON-MYELOABLATIVE CONDITIONING REGIMEN: Patients receive fludarabine phosphate intravenously
(IV) over 30 minutes on days -4 to -2, cyclosporine IV every 8-12 hours on days -3 to 0, and
undergo low-dose TBI on day 0.
TRANSPLANTATION: Patients undergo allogeneic bone marrow or PBSC transplantation on day 0.
IMMUNOSUPPRESSION: Patients receive cyclosporine orally (PO) or IV every 8-12 hours on days
1-100 with taper to day 177, and mycophenolate mofetil PO or IV every 8 hours on days 0-40
with taper to day 96.
After completion of study treatment, patients are followed up at 6 months and annually
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Engraftment, defined as donor chimerism (mixed or complete)
Patient data will be summarized using standard statistical methods.
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
United States: Federal Government
|Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium||Seattle, Washington 98109|
|Vanderbilt University||Nashville, Tennessee 37232-6305|
|Huntsman Cancer Institute/University of Utah||Salt Lake City, Utah 84112|
|Robert H. Lurie Comprehensive Cancer Center||Chicago, Illinois 60611|
|Riley Hospital for Children||Indianapolis, Indiana 46202|