A Phase III Randomized, Multicenter Trial Comparing G-CSF Mobilized Peripheral Blood Stem Cell With Marrow Transplantation From HLA Compatible Unrelated Donors (BMT CTN #0201)
BACKGROUND:
Many studies of allogeneic marrow transplantation have shown that a higher dose of marrow
cells correlates with more robust hematopoietic engraftment and lower mortality from
infectious complications. Peripheral blood stem cells (PBSC) collected after mobilization
with granulocyte colony stimulating factor (G-CSF) contain a larger number of CD34-positive
(CD34) progenitors and total cells than bone marrow. These observations led to the
hypothesis that transplantation of PBSC would lead to lower mortality compared to
transplantation of marrow. In addition, PBSC grafts have a higher T cell content,
predicting a possibly more powerful anti-leukemia effect. However, the higher T cell
content of PBSC may also lead to increased incidence and severity of acute and chronic
graft-versus-host disease (GVHD). This concern is especially serious when the donor is
unrelated to the recipient. This prospective, randomized, multicenter clinical trial of
unrelated donor transplantation will test the hypothesis that transplantation of PBSC leads
to similar patient survival compared to transplantation of marrow.
DESIGN NARRATIVE:
This is a Phase III randomized, open label, multicenter clinical trial sponsored by the NMDP
and the National Institutes of Health (NIH). The objective of the trial is to test the null
hypothesis that there is no difference in overall survival after PBSC versus marrow
transplants from HLA compatible unrelated donors. The study will compare G-CSF-mobilized
PBSC transplantation with bone marrow transplantation from HLA-compatible unrelated donors
for patients with leukemia, myelodysplastic or myeloproliferative syndromes. Conditioning
and GVHD prophylaxis regimens will vary by center and within centers, however, the center
must declare before randomization what regimens will be used for each patient. The primary
endpoint of this trial is 2-year survival following randomization. Secondary analyses will
consider neutrophil and platelet recovery, acute and chronic GVHD, time off all
immunosuppressive therapy, relapse, infections, adverse events and immune reconstitution.
The trial will include evaluation of patient and donor quality of life, composition of the
graft, and immune reconstitution. Accrual is anticipated for 3 years with a follow-up
period of 3 years.
Patient
Interventional
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Two-year survival
Measured at 2 years
No
William Vaughan, MD
Principal Investigator
University of Alabama at Birmingham
United States: Federal Government
418
NCT00075816
January 2004
April 2014
Name | Location |
---|---|
Roswell Park Cancer Institute | Buffalo, New York 14263 |
Fred Hutchinson Cancer Research Center | Seattle, Washington 98109 |
Mayo Clinic Cancer Center | Rochester, Minnesota 55905 |
University of Iowa Hospitals and Clinics | Iowa City, Iowa 52242 |
University of Pennsylvania Cancer Center | Philadelphia, Pennsylvania 19104 |
University of Pittsburgh Cancer Institute | Pittsburgh, Pennsylvania 15213 |
University of Nebraska Medical Center | Omaha, Nebraska 68198-3330 |
Schneider Children's Hospital | New Hyde Park, New York 11042 |
Hackensack University Medical Center Cancer Center | Hackensack, New Jersey 07601 |
City of Hope National Medical Center | Los Angeles, California 91010 |
Baylor University Medical Center | Dallas, Texas 75246 |
University of Minnesota | Minneapolis, Minnesota 55455 |
University of California, San Francisco | San Francisco, California 94143 |
University of Alabama at Birmingham | Birmingham, Alabama 35294-3300 |
Vanderbilt University | Nashville, Tennessee 37232-6305 |
Duke University Medical Center | Durham, North Carolina 27710 |
University of Michigan Medical Center | Ann Arbor, Michigan 48104-0914 |
Oregon Health Sciences University | Portland, Oregon |
Emory University | Atlanta, Georgia 30322 |
University of Maryland | Baltimore, Maryland 21201 |
Texas Transplant Institute | San Antonio, Texas 78229 |
UCSD Cancer Center | La Jolla, California 92093-0064 |
Loyola University | Maywood, Illinois 60153 |
Stanford Hospital and Clinics | Stanford, California 94305 |
University of Florida College of Medicine (Shands) | Gainesville, Florida 32610 |
Washington University/Barnes Jewish Hospital | St. Louis, Missouri 63110 |
Washington University/St. Louis Children's Hospital | St. Louis, Missouri 63110 |
Wake Forest University Health Sciences | Winston-Salem, North Carolina 27157 |
University of Texas/MD Anderson CRC | Houston, Texas 77030 |
Utah BMT/Primary Children's Medical Center | Salt Lake City, Utah 84132 |
Indiana BMT at Beech Grove | Beech Grove, Indiana 46107 |
University of Kansas Hospital | Kansas City, Kansas 66160 |
DFCI/Brigham & Women's | Boston, Massachusetts 02114 |
Ohio State/Arthur G. James Cancer Hospital | Columbus, Ohio 43210 |
University of Oklahoma Medical Center | Oklahoma City, Oklahoma 73104 |
Oregon Health & Science University (Peds) | Portland, Oregon 97239-3098 |
Baylor College of Medicine/The Methodist Hospital | Houston, Texas 77030-2399 |
Utah BMT/University of Utah Medical School | Salt Lake City, Utah 84132 |
Virginia Commonwealth University MCV Hospitals | Richmond, Virginia 23298-0037 |