The Role of Minimal Residual Disease Testing Before and After Hematopoietic Cell Transplantation for Pediatric Acute Myeloid Leukemia
This is a prospective, non-therapeutic study, assessing the significance of minimal residual
disease (MRD) at three different time points in relation to allogeneic HCT for pediatric
AML. The study is a collaboration between the Pediatric Blood and Marrow Transplant
Consortium (PBMTC) and the Resource for Clinical Investigations in Blood and Marrow
Transplantation (RCI-BMT) of the Center for International Blood and Marrow Transplant
Research (CIBMTR). The study will enroll pediatric AML patients who undergo myeloablative
HCT at PBMTC sites. The eligibility criteria for this non-therapeutic study mirror widely
accepted criteria for allogeneic HCT in pediatric AML.
The study tests the hypothesis that assessment of pre-transplant and post-transplant MRD
predicts 2-year outcomes following transplant. Two MRD methodologies are being studied: flow
cytometry and WT1 PCR. The secondary hypothesis is that combining these 2 methodologies will
improve the accuracy in predicting 2-year outcomes following transplant.
It is well established that the level of minimal residual disease (MRD) during chemotherapy
is a strong predictor of relapse in children with acute lymphoblastic leukemia (ALL) [33,
34]. Within this population, MRD levels have the potential to predict those patients who
will respond well to standard therapy, thus allowing clinicians to tailor therapy and
minimize toxicity while ensuring maximal cure rates [10]. MRD levels before allogeneic
hematopoietic stem cell transplantation (HCT) also predict the risk of relapse post-HCT
[25], leading to the clinical practice of reducing MRD levels as much as possible before
transplant. By contrast, in children with acute myeloid leukemia (AML), the prognostic value
of MRD levels prior to HCT remains unclear.
Our long-term objective is to improve the cure rate for children with AML. The investigators
hypothesize that MRD levels before HCT will provide a powerful tool to select the best
candidates for transplant, guide decision making in stem cell source and preparative
therapy, and optimize the timing of the transplant. Measurements of MRD post-HCT will allow
informed decisions about withdrawal of immunosuppressive therapy, administration of donor
lymphocyte infusions, or alternative targeted therapies.
Observational
Observational Model: Cohort, Time Perspective: Prospective
David A. Jacobsohn, MD, ScM
Principal Investigator
Children's Research Institute
United States: Institutional Review Board
09-MRD
NCT01385787
October 2011
September 2015
Name | Location |
---|---|
University of Michigan | Ann Arbor, Michigan 48109-0624 |
Roswell Park Cancer Institute | Buffalo, New York 14263 |
University of Mississippi Medical Center | Jackson, Mississippi 39216-4505 |
Medical University of South Carolina | Charleston, South Carolina 29425-0721 |
Children's Hospital of Michigan | Detroit, Michigan 48201 |
Mount Sinai School of Medicine | New York, New York 10029 |
New York Medical College | Valhalla, New York 10595 |
University Hospitals of Cleveland | Cleveland, Ohio 44106 |
Hackensack University Medical Center | Hackensack, New Jersey 07601 |
Children's National Medical Center | Washington, District of Columbia 20010-2970 |
Miami Children's Hospital | Miami, Florida 33155-4069 |
All Children's Hospital | St. Petersburg, Florida 33701 |
Phoenix Children's Hospital | Phoenix, Arizona 85016-7710 |
Duke University Medical Center | Durham, North Carolina 27710 |
University of California San Francisco | San Francisco, California 941104206 |
Dana Farber Cancer Institute | Boston, Massachusetts 02115 |
University of Louisville | Louisville, Kentucky 40202 |
Children's Memorial Hospital | Chicago, Illinois 60614 |
Virginia Commonwealth University | Richmond, Virginia |
Children's Healthcare of Atlanta | Atlanta, Georgia 30342 |
Loma Linda University | Loma Linda, California 92354 |
Johns Hopkins | Baltimore, Maryland 21231 |
University of North Carolina at Chapel Hill | Chapel Hill, North Carolina 27599 |
Penn State Milton S. Hershey Medical Center | Hershey, Pennsylvania 17033 |
The Children's Hospital of Alabama, University of Alabama at Birmingham | Birmingham, Alabama 35233 |
The Children's Hospital Colorado | Aurora, Colorado 80045 |
Riley Hospital for Children/Indiana University | Indianapolis, Indiana 46202 |
Washington University, St. Louis Children's Hospital | St. Louis, Missouri 63110 |
Columbia University - The Morgan Stanley Children's Hospital of New York | New York, New York 10032 |
Oregon Health & Sciences University - Doerbecher Children's | Portland, Oregon 97239 |
Methodist Children's Hospital of South Texas/Texas Institute of Medicine and Surgery | San Antonio, Texas 78229 |
University of Utah - Primary Children's Medical Center | Salt Lake City, Utah 84108 |