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Clinical Trial
. 2016 Dec 1;2(12):1583-1589.
doi: 10.1001/jamaoncol.2016.2520.

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  1 , Brent Logan  2 , Peter Westervelt  3 , Corey Cutler  4 , Ann Woolfrey  5 , Shakila P Khan  6 , Edmund K Waller  7 , Richard T Maziarz  8 , Juan Wu  9 , Bronwen E Shaw  10 , Dennis Confer  11 , Mary M Horowitz  10 , Claudio Anasetti  12
Affiliations
Clinical Trial

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 et al. JAMA Oncol. .

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.

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Figures

Figure 1
Figure 1
Overall Survival
See this image and copyright information in PMC

References

    1. Anasetti C, Logan BR, Lee SJ, et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012;367(16):1487–1496. - PMC - PubMed
    1. McQuellon RP, Russell GB, Cella DF, et al. Quality of life measurement in bone marrow transplantation: development of the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) scale. Bone Marrow Transplant. 1997;19(4):357–368. - PubMed
    1. McQuellon RP, Russell GB, Rambo TD, et al. Quality of life and psychological distress of bone marrow transplant recipients: the ‘time trajectory’ to recovery over the first year. Bone Marrow Transplant. 1998;21(5):477–486. - PubMed
    1. Cella D. Manual of the Functional Assessment of Chronic Illness Therapy (FACIT) Scales. Northwestern University; Evanston: 1997.
    1. Veit CT, Ware JE., Jr. The structure of psychological distress and well-being in general populations. J Consult Clin Psychol. 1983;51(5):730–742. - PubMed

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