Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)
A Phase III Randomized, Multicenter Trial Comparing G-CSF Mobilized Peripheral Blood Stem Cell With Marrow Transplantation From Human Leukocyte Antigen (HLA) Compatible Unrelated Donors (BMT CTN #0201)
4 other identifiers
interventional
551
2 countries
45
Brief Summary
The study is designed as a Phase III, randomized, open label, multicenter, prospective, comparative trial of granulocyte colony stimulating factor (G-CSF)-mobilized peripheral blood stem cells (PBSC) versus marrow from unrelated donors for transplantation in patients with hematologic malignancies. Recipients will be stratified by transplant center and disease risk and will be randomized to either the PBSC or marrow arm in a 1:1 ratio.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3 leukemia
Started Jan 2004
Longer than P75 for phase_3 leukemia
45 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2004
CompletedFirst Submitted
Initial submission to the registry
January 9, 2004
CompletedFirst Posted
Study publicly available on registry
January 13, 2004
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2014
CompletedResults Posted
Study results publicly available
February 1, 2016
CompletedJanuary 4, 2023
December 1, 2022
9.3 years
January 9, 2004
January 22, 2015
December 7, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Two-year Overall Survival
Overall survival rate at 2 years according to an intention-to-treat analysis.
Measured at 2 years
Secondary Outcomes (16)
Neutrophil Engraftment
Measured at Day 28
Platelet Engraftment
Measured at Day 180
Graft Failure
Measured at 28 and 100 days
Extensive Chronic Graft-versus-host Disease (GVHD)
Measured at 730 days
Chronic GVHD
Measured at 2 years
- +11 more secondary outcomes
Study Arms (2)
Bone Marrow Transplant
ACTIVE COMPARATORAllogeneic bone marrow transplantation
Blood Stem Cell Transplant
ACTIVE COMPARATORPeripheral blood stem cell transplantation
Interventions
Bone marrow transplant from HLA compatible unrelated donors.
Peripheral blood transplant from HLA compatible unrelated donors.
Eligibility Criteria
You may qualify if:
- One of the following diagnoses:
- Acute myelogenous leukemia at the following stages: first remission, second remission, third or subsequent remission, or not in remission
- Acute lymphoblastic leukemia at the following stages: first remission, second remission, third or subsequent remission, or not in remission
- Chronic myelogenous leukemia at the following stages: chronic phase, accelerated phase, or blast phase
- Myelodysplastic syndromes (MDS) at the following stages: refractory anemia; refractory anemia with ringed sideroblasts; refractory cytopenia with multilineage dysplasia; refractory cytopenia with multilineage dysplasia and ringed sideroblasts; refractory anemia with excess blasts-1 (5-10% blasts); refractory anemia with excess blasts-2 (10-20% blasts); myelodysplastic syndrome, unclassified; or MDS associated with isolated del (5q)
- Myeloproliferative diseases: chronic myelomonocytic leukemia; agnogenic myeloid metaplasia with myelofibrosis (idiopathic myelofibrosis); juvenile myelomonocytic leukemia
- Therapy-related acute myelogenous leukemia (AML) or MDS with prior malignancy that has been in remission for at least 12 months. If the remission is less than 12 months, Medical Monitor or Protocol Chair approval is required for eligibility
You may not qualify if:
- Prior allogeneic or autologous transplants using any hematopoietic stem cell source; patients with secondary malignancies who have had a prior autologous transplant will be eligible; the prior autologous transplant must have been performed for the primary malignancy (such as lymphoma) and must have occurred 12 or more months prior to enrollment
- Lymphoma (11% of 2001 NMDP transplants), other malignant disorders (6%), and non-malignant disorders (9%)
- Matched for HLA-A, B, and DRB1 antigens
- One antigen mismatch at HLA-A, B, or DRB1 is acceptable with or without mismatch at HLA-C
- Typing is by DNA techniques: intermediate resolution for A, B, and C, and high resolution for DRB1. HLA-C typing is mandatory but will not count in the match.
- Willing to undergo both bone marrow harvest and G-CSF administration with apheresis
- Willing to be randomly assigned to either marrow or PBSC collection
- Adequate peripheral venous access for leukapheresis or willing to undergo placement of a central catheter
- Donor center affiliation with NMDP
- Pregnant (positive serum β-HCG) or uninterruptible breastfeeding
- Known allergy to G-CSF or to E. Coli-derived recombinant protein products
- History of autoimmune disorders
- History of deep vein thrombosis or venous thromboembolism
- History of iritis or episcleritis
- History of serious adverse reaction to anesthesia
- +4 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Medical College of Wisconsinlead
- National Heart, Lung, and Blood Institute (NHLBI)collaborator
- Blood and Marrow Transplant Clinical Trials Networkcollaborator
- National Cancer Institute (NCI)collaborator
- National Marrow Donor Programcollaborator
Study Sites (45)
University of Alabama at Birmingham
Birmingham, Alabama, 35294, United States
City of Hope National Medical Center
Duarte, California, 91010, United States
UCSD Cancer Center
La Jolla, California, 92093-0960, United States
University of California, San Francisco
San Francisco, California, 94143, United States
Stanford Hospital and Clinics
Stanford, California, 94305, United States
University of Florida College of Medicine (Shands)
Gainesville, Florida, 32610-100277, United States
Emory University
Atlanta, Georgia, 30322, United States
Loyola University
Maywood, Illinois, 60153, United States
IBMT (Indiana Blood and Marrow Transplant) at St Francis Franciscan Health
Indianapolis, Indiana, 46237, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
University of Kansas Hospital
Kansas City, Kansas, 66160, United States
University of Maryland
Baltimore, Maryland, 21228, United States
DFCI/Brigham & Women's
Boston, Massachusetts, 02114, United States
University of Michigan Medical Center
Ann Arbor, Michigan, 48109-0942, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Mayo Clinic Cancer Center
Rochester, Minnesota, 55905, United States
Washington University/Barnes Jewish Hospital
St Louis, Missouri, 63110, United States
Washington University/St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
University of Nebraska Medical Center
Omaha, Nebraska, 68198-3330, United States
Hackensack University Medical Center Cancer Center
Hackensack, New Jersey, 07601, United States
Roswell Park Cancer Institute
Buffalo, New York, 14263-0001, United States
Cohen Children's Hospital
New Hyde Park, New York, 11040, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157-1082, United States
Ohio State/Arthur G. James Cancer Hospital
Columbus, Ohio, 43210, United States
University of Oklahoma Medical Center
Oklahoma City, Oklahoma, 73104, United States
Oregon Health & Science University (Peds)
Portland, Oregon, 97239-3098, United States
Oregon Health Sciences University
Portland, Oregon, 97239, United States
University of Pennsylvania Cancer Center
Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh Cancer Institute
Pittsburgh, Pennsylvania, 15232, United States
Vanderbilt University
Nashville, Tennessee, 37232-6838, United States
Baylor University Medical Center
Dallas, Texas, 77030, United States
Baylor College of Medicine/The Methodist Hospital
Houston, Texas, 77030-2399, United States
University of Texas/MD Anderson CRC
Houston, Texas, 77030-4009, United States
Texas Transplant Institute
San Antonio, Texas, 78229, United States
Utah BMT/Primary Children's Medical Center
Salt Lake City, Utah, 84112, United States
Utah BMT/University of Utah Medical School
Salt Lake City, Utah, 84132, United States
Virginia Commonwealth University MCV Hospitals
Richmond, Virginia, 23298-0037, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98104, United States
Tom Baker Cancer Centre, Calgary
Calgary, Alberta, T2N 4N2, Canada
Vancouver General Hospital
Vancouver, British Columbia, V5Z1M9, Canada
Hamilton Health Sciences - McMaster Site
Hamilton, Ontario, Canada
Ottawa Hospital
Ottawa, Ontario, Canada
University of Toronto, Princess Margaret Hospital
Toronto, Ontario, M5G 2M9, Canada
Queen Elizabeth II Health Sciences Centre - Halifax
Halifax, Canada
Related Publications (8)
Anasetti C, Logan BR, Lee SJ, Waller EK, Weisdorf DJ, Wingard JR, Cutler CS, Westervelt P, Woolfrey A, Couban S, Ehninger G, Johnston L, Maziarz RT, Pulsipher MA, Porter DL, Mineishi S, McCarty JM, Khan SP, Anderlini P, Bensinger WI, Leitman SF, Rowley SD, Bredeson C, Carter SL, Horowitz MM, Confer DL; Blood and Marrow Transplant Clinical Trials Network. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012 Oct 18;367(16):1487-96. doi: 10.1056/NEJMoa1203517.
PMID: 23075175RESULTFoley B, Cooley S, Verneris MR, Curtsinger J, Luo X, Waller EK, Anasetti C, Weisdorf D, Miller JS. Human cytomegalovirus (CMV)-induced memory-like NKG2C(+) NK cells are transplantable and expand in vivo in response to recipient CMV antigen. J Immunol. 2012 Nov 15;189(10):5082-8. doi: 10.4049/jimmunol.1201964. Epub 2012 Oct 17.
PMID: 23077239RESULTSwitzer GE, Bruce JG, Harrington D, Haagenson M, Drexler R, Foley A, Confer D, Bishop M, Anderlini P, Rowley S, Leitman SF, Anasetti C, Wingard JR. Health-related quality of life of bone marrow versus peripheral blood stem cell donors: a prespecified subgroup analysis from a phase III RCT-BMTCTN protocol 0201. Biol Blood Marrow Transplant. 2014 Jan;20(1):118-27. doi: 10.1016/j.bbmt.2013.10.024. Epub 2013 Nov 1.
PMID: 24184336RESULTWaller EK, Logan BR, Harris WA, Devine SM, Porter DL, Mineishi S, McCarty JM, Gonzalez CE, Spitzer TR, Krijanovski OI, Linenberger ML, Woolfrey A, Howard A, Wu J, Confer DL, Anasetti C. Improved survival after transplantation of more donor plasmacytoid dendritic or naive T cells from unrelated-donor marrow grafts: results from BMTCTN 0201. J Clin Oncol. 2014 Aug 1;32(22):2365-72. doi: 10.1200/JCO.2013.54.4577. Epub 2014 Jun 30.
PMID: 24982459RESULTKhera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ. Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial. Biol Blood Marrow Transplant. 2015 Oct;21(10):1815-22. doi: 10.1016/j.bbmt.2015.06.004. Epub 2015 Jun 11.
PMID: 26071866RESULTYoung JH, Logan BR, Wu J, Wingard JR, Weisdorf DJ, Mudrick C, Knust K, Horowitz MM, Confer DL, Dubberke ER, Pergam SA, Marty FM, Strasfeld LM, Brown JWM, Langston AA, Schuster MG, Kaul DR, Martin SI, Anasetti C; Blood and Marrow Transplant Clinical Trials Network Trial 0201. Infections after Transplantation of Bone Marrow or Peripheral Blood Stem Cells from Unrelated Donors. Biol Blood Marrow Transplant. 2016 Feb;22(2):359-370. doi: 10.1016/j.bbmt.2015.09.013. Epub 2015 Sep 25.
PMID: 26409243RESULTBurns LJ, Logan BR, Chitphakdithai P, Miller JP, Drexler R, Spellman S, Switzer GE, Wingard JR, Anasetti C, Confer DL; Blood and Marrow Transplant Clinical Trials Network. Recovery of Unrelated Donors of Peripheral Blood Stem Cells versus Recovery of Unrelated Donors of Bone Marrow: A Prespecified Analysis from the Phase III Blood and Marrow Transplant Clinical Trials Network Protocol 0201. Biol Blood Marrow Transplant. 2016 Jun;22(6):1108-1116. doi: 10.1016/j.bbmt.2016.02.018. Epub 2016 Mar 21.
PMID: 27013014RESULTLee SJ, Logan B, Westervelt P, Cutler C, Woolfrey A, Khan SP, Waller EK, Maziarz RT, Wu J, Shaw BE, Confer D, Horowitz MM, Anasetti C. Comparison of Patient-Reported Outcomes in 5-Year Survivors Who Received Bone Marrow vs Peripheral Blood Unrelated Donor Transplantation: Long-term Follow-up of a Randomized Clinical Trial. JAMA Oncol. 2016 Dec 1;2(12):1583-1589. doi: 10.1001/jamaoncol.2016.2520.
PMID: 27532508DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Adam Mendizabal
- Organization
- The EMMES Corporation
Study Officials
- STUDY DIRECTOR
Mary Horowitz, MD
Center for International Blood and Marrow Transplant Research
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 9, 2004
First Posted
January 13, 2004
Study Start
January 1, 2004
Primary Completion
April 1, 2013
Study Completion
April 1, 2014
Last Updated
January 4, 2023
Results First Posted
February 1, 2016
Record last verified: 2022-12
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, ICF
- Time Frame
- Within 6 months of official study closure at participating sites.
- Access Criteria
- Available to the public
Results will be published in a manuscript