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Original Investigation
December 2016

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

Author Affiliations
  • 1Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington
  • 2Division of Biostatistics, Medical College of Wisconsin, Milwaukee
  • 3Department of Medicine, Division of Oncology, Washington University School of Medicine, St Louis, Missouri
  • 4Division of Hematologic Malignancies and Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts
  • 5Transplantation Biology Department, Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington
  • 6Division of Pediatric Hematology-Oncology, Mayo Clinic, Rochester, Minnesota
  • 7Winship Cancer Institute, Department of Hematology and Medical Oncology, Emory University, Atlanta, Georgia
  • 8Adult Blood and Marrow Stem Cell Transplant Program, Knight Cancer Institute, Oregon Health and Science University, Portland
  • 9Emmes Corporation, Rockville, Maryland
  • 10Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee
  • 11National Marrow Donor Program, Minneapolis, Minnesota
  • 12Department of Blood and Marrow Transplantation, Moffitt Cancer Center, Tampa, Florida
JAMA Oncol. 2016;2(12):1583-1589. doi:10.1001/jamaoncol.2016.2520
Key Points

Question  Should bone marrow or peripheral blood be used for hematopoietic cell transplantation from unrelated donors?

Findings  In this follow-up of a randomized clinical trial, recipients of unrelated donor bone marrow had the same 5-year survival with better psychological well-being, less burdensome chronic graft-vs-host disease symptoms, and a higher likelihood of returning to work than recipients of peripheral blood.

Meaning  Bone marrow should be used instead of peripheral blood for unrelated donor transplantation when calcineurin inhibitor/methotrexate-based graft-vs-host disease prophylaxis is used.


Importance  Bone marrow or peripheral blood from unrelated donors may be used for hematopoietic cell transplantation. Information about the relative success of transplantation with these 2 graft sources would help physicians and patients choose between them.

Objective  To compare patient-reported outcomes between patients randomized to receive 1 of 2 graft types for unrelated donor transplantation.

Design, Setting, and Participants  This follow-up of a randomized clinical trial included English- or Spanish-speaking patients 16 years or older participating in a multicenter randomized clinical trial of unrelated donor bone marrow (BM) vs peripheral blood (PB) (N = 551) in hematopoietic cell transplantation for hematologic neoplasms. Patient-reported outcomes were collected from patients at enrollment and 0.5, 1, 2, and 5 years after transplantation.

Interventions  Unrelated donor BM or PB hematopoietic cell transplantation.

Main Outcomes and Measures  Functional Assessment of Cancer Therapy–Bone Marrow Transplant, Mental Health Inventory, occupational functioning, Lee Chronic Graft-vs-Host Disease Symptom Scale.

Results  At 5 years after transplantation, 102 BM and 93 PB participants were alive and eligible for assessment (age ≥40 years or older: 104 [53.5%] male: 101 [51.8%]). The mean (SE) Mental Health Inventory Psychological Well-Being scores (78.9 [1.7] vs 72.2 [1.9]; P = .01; higher better) and Lee chronic graft-vs-host disease symptom scores (13.1 [1.5] vs 19.3 [1.6]; P = .004; lower better) were significantly better for BM recipients, adjusting for baseline scores and missing data. Recipients of BM were also more likely to be working full or part-time than recipients of PB (odds ratio, 1.5; 95% CI, 1.2-2.0; P = .002), adjusting for work status before transplantation. With a median follow-up of 73 months (range, 30-121 months) for survivors, no differences in survival (40% vs 39%; P = .84), relapse (32% vs 29%; P = .47), or treatment-related mortality (29% vs 32%; P = .44) between BM and PB were observed.

Conclusions and Relevance  Recipients of unrelated donor BM had better psychological well-being, less burdensome chronic GVHD symptoms, and were more likely to return to work than recipients of PB at 5 years after transplantation. Bone marrow should be the standard of care for these types of transplant procedures.

Trial Registration  clinicaltrials.gov Identifier: NCT00075816

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