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1 Increased Incidence of Chronic Graft-Versus-Host Disease (GVHD) and No Survival Advantage with Filgrastim-Mobilized Peripheral Blood Stem Cells (PBSC) Compared to Bone Marrow (BM) Transplants From Unrelated Donors: Results of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 0201, a Phase III, Prospective, Randomized TrialClinically Relevant Abstract

Program: Oral and Poster Abstracts
Type: Oral
Session: Plenary Scientific Session
Sunday, December 11, 2011: 2:05 PM
Hall AB (San Diego Convention Center)

Claudio Anasetti, MD1, Brent R. Logan, PhD2*, Stephanie J. Lee, MD, MPH3, Edmund K Waller, MD PhD4, Daniel J. Weisdorf, MD5, John R. Wingard, MD6, Corey S. Cutler, MD, MPH, FRCPC7, Peter Westervelt, MD, PhD8*, Ann Woolfrey, MD9, Stephen Couban, MD, FRCPC10, Laura Johnston, MD11, Richard T. Maziarz, MD12, Michael Pulsipher, MD13, Paolo Anderlini, MD14, William I Bensinger, MD15*, Susan F. Leitman, MD16, Scott D Rowley, MD17, Shelly L Carter, ScD18*, Mary M. Horowitz, MD, MS19 and Dennis L Confer, MD20

1H. Lee Moffitt Cancer Center & Research Institute, Tampa, FL
2Medical College of Wisconsin, Milwaukee, WI
3Fred Hutchinson Cancer Research Center, Seattle, WA
4Department of Heamtology/Medical Oncology, Winship Cancer Institute, Emory University, Atlanta, GA
5Blood and Marrow Transplant Program, University Of Minnesota Medical Center, Minneapolis, MN
6Shands Cancer Center, University of Florida, Gainesville, FL
7Dana-Farber Cancer Institute, Boston, MA
8Department of Medicine, Siteman Cancer Center, Washington University School of Medicine, St. Louis, MO
9Pediatric Hematologic Cell Transplant, Fred Hutchinson Cancer Research Center, Seattle, WA
10Hematology, Dalhousie University, Halifax, NS, Canada
11Stanford Univ. Med. Ctr. , Stanford, CA
12Oregon Health and Science University, Portland, OR
13University of Utah Medical Center, Salt Lake City, UT
14Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX
15Clinical Division, Fred Hutchinson Cancer Research Center, Seattle, WA
16Department of Transfusion Medicine, National Institutes of Health, Bethesda, MD
17The John Theurer Cancer Center at Hackensack University Medical Center, Hackensack, NJ
18The EMMES Corporation, Rockville, MD
19Medical College of Wisconsin, Center for International Blood and Marrow Transplant Research, Milwaukee, WI
20National Marrow Donor Program, Minneapolis, MN

Background: Randomized trials demonstrated that filgrastim-mobilized PBSC compared to BM from HLA-identical siblings improved engraftment kinetics, increased risks of acute and chronic GVHD, but also decreased relapse and improved survival in patients with high risk leukemia. Retrospective analyses of unrelated donor transplants did not appreciate the same PBSC protective effect.

Patients and Methods: The BMT CTN, sponsored by the NHLBI and NCI, conducted a Phase III, randomized, multicenter, trial of unrelated donor PBSC versus BM. The primary objective was to compare two-year survival probabilities in the two study arms using an intent-to-treat analysis. Both patients and donors provided informed consent. Fifty centers in the U.S. and Canada enrolled patients between January, 2004 and September 2009. Median follow up is 36 months (interquartile range 25 – 37 months). Randomization was performed in a 1:1 ratio to either PBSC or BM and stratified by transplant center and disease risk. Of the 278 subjects randomized to BM, 5% had no transplant, and 4.3% crossed over to PBSC; of the 273 randomized to PBSC, 4% had no transplant, and 0.4% crossed over to BM, so subjects on both arms had greater than 90% compliance with the assigned therapy. Patient primary disease (AML, ALL, CML, MDS, CMML, and MF), disease risk, gender, age, race, ethnicity, CMV serology, performance status, comorbidity, organ function, conditioning regimen, GVHD prophylaxis, use of growth factors, and donor characteristics were all well balanced between the two groups. Overall, 90% were adults over age 20, 47% had AML, 28% had high risk disease, 48% were conditioned with cyclophosphamide plus total body irradiation, and 71% received tacrolimus plus methotrexate for GVHD prophylaxis.

Results: There were no observed differences in outcomes between the two groups except for a higher incidence of overall chronic GVHD (see Table) and more common chronic extensive GVHD with PBSC (46% vs. 31%). There were no survival differences according to graft sources in planned subset analyses of low and high risk malignancy or in those received HLA-matched or mismatched grafts. Primary causes of death were relapse in 54% vs. 49%, graft failure in 7% vs. 0%, acute or chronic GVHD in 22% vs. 34%, others in 16% vs. 16% of the BM and PBSC arms, respectively.


Outcomes at 2 years




Overall survival, intent-to-treat

51% (45%-57%)

46% (40%-52%)


Overall survival, transplanted

52% (46%-58%)

48% (42%-54%)


Disease-free survival, transplanted

47% (40%-53%)

44% (38%-50%)



28% (22%-34%)

28% (23%-34%)


Non-relapse mortality

26% (20%-31%)

27% (22%-33%)


ANC > 500 by day 28

95% (80%-99%)

86% (78%-92%)


Acute GVHD II-IV by day 100

47% (40%-53%)

46% (39%-52%)


Acute GVHD III-IV by day 100

16% (12%-21%)

14% (10%-19%)


Any chronic GVHD

53% (45%-60%)

40% (33%-47%)







Conclusion: This large randomized trial shows that PBSC from unrelated donors is associated with higher rates of chronic GVHD compared to BM, although rates of acute GVHD, relapse, non-relapse mortality and overall survival are similar.


Disclosures: Off Label Use: Cyclophosphamide, busulfan, melphalan, fludarabine, anti-thymocyte globulin, irradiation were used to eradicate malignancy. Tacrolimus, cyclosporine, methotrexate were used for GVHD prophylaxis.. Weisdorf: Genzyme: Consultancy, Research Funding. Westervelt: Novartis: Speakers Bureau.

*signifies non-member of ASH