Nonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding Study
I. Identify doses of TBI that lead to sufficient probability of donor engraftment (> 5%
donor cluster of differentiation [CD]3 chimerism) by day +200.
II. Evaluate the probability of severe acute graft-versus-host disease (GVHD).
I. Evaluate the probabilities of overall survival, regimen-related toxicity (RRT), and
recurrent hematopoietic malignancy in those patients with a prior underlying history of
II. Examine the degree to which mixed chimerism provides for amelioration of symptoms (i.e.,
infections due to neutropenia, hemorrhage due to thrombocytopenia) associated with bone
III. Determine if the FA complementation group and % initial mosaicism predict engraftment
and RRT outcomes.
OUTLINE: Patients are assigned to 1 of 4 treatment arms.
NOTE: Patients no longer receive pre-transplant cyclophosphamide as of February 2009.
After completion of study treatment, patients are followed up at 6 months and then annually
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Doses of TBI associated with acceptable levels of engraftment
By day 200
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
United States: Institutional Review Board
|Cleveland Clinic Foundation||Cleveland, Ohio 44195|
|Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium||Seattle, Washington 98109|
|Children's Hospital and Research Center at Oakland||Oakland, California 94609-1809|
|Vanderbilt University||Nashville, Tennessee 37232-6305|
|Children's Hospital of Wisconsin||Milwaukee, Wisconsin 53201|