Comparison of Patient-Reported Outcomes in 5-Year Survivors Who Received Bone Marrowvs Peripheral Blood Unrelated Donor Transplantation Long-term Follow-up of a Randomized Clinical Trial

作者：Lee Stephanie J^*; Logan Brent; Westervelt Peter; Cutler Corey; Woolfrey Ann; Khan Shakila P; Waller Edmund K; Maziarz Richard T; Wu Juan; Shaw Bronwen E; Confer Dennis; Horowitz Mary M; Anasetti Claudio

来源：JAMA Oncology, 2016, 2(12): 1583-1589.

DOI：10.1001/jamaoncol.2016.2520

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 BMand 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 PsychologicalWell-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 BMrecipients, 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.

• 出版日期2016-12-1