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

Stephanie J. Lee, Brent Logan, Peter Westervelt, Corey Cutler, Ann Woolfrey, Shakila P. Khan, Edmund K. Waller, Richard Maziarz, Juan Wu, Bronwen E. Shaw, Dennis Confer, Mary M. Horowitz, Claudio Anasetti

Research output: Contribution to journalArticle

48 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1583-1589
Number of pages7
JournalJAMA oncology
Volume2
Issue number12
DOIs
StatePublished - Dec 1 2016

ASJC Scopus subject areas

  • Medicine(all)

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    Lee, S. J., Logan, B., Westervelt, P., Cutler, C., Woolfrey, A., Khan, S. P., Waller, E. K., Maziarz, R., Wu, J., Shaw, B. E., Confer, D., Horowitz, M. M., & Anasetti, C. (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. JAMA oncology, 2(12), 1583-1589. https://doi.org/10.1001/jamaoncol.2016.2520