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Centralized patient-reported outcome data collection in transplantation is feasible and clinically meaningful. Cancer 2017 Dec 01;123(23):4687-4700 PMID: 28817182 PMCID: PMC5693638

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BACKGROUND: Allogeneic hematopoietic cell transplantation (HCT) cures many patients, but often with the risk of late effects and impaired quality of life. The value of quantifying patient-reported outcomes (PROs) is increasingly being recognized, but the routine collection of PROs is uncommon. This study evaluated the feasibility of prospective PRO collection by an outcome registry at multiple time points from unselected HCT patients undergoing transplantation at centers contributing clinical data to the Center for International Blood and Marrow Transplant Research (CIBMTR), and then it correlated the PRO data with clinical and demographic data.

METHODS: The Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT), 36-Item Short Form Health Survey (SF-36), and Pediatric Quality of Life Inventory measures were administered before HCT, on day 100, and at 6 and 12 months. Patients were recruited by the transplant center, but posttransplant PRO collection was managed centrally by the CIBMTR.

RESULTS: There were 580 eligible patients, and 390 (67%) enrolled. Feasibility was shown by high time-specific retention rates (176 of 238 at 1 year or 74%) and participant satisfaction. Factors associated with higher response rates were an age > 50 years (odds ratio [OR], 1.58; 95% confidence interval [CI], 1.03-2.41; P = .0355), white race (OR, 4.61; 95% CI, 2.66-7.99; P < .0001), and being married (OR, 2.28; 95% CI, 1.42-3.65; P = .0006) for adults and a higher family income for children (OR, 4.99; 95% CI, 2.12-11.75; P = .0002). Importantly, pre-HCT PRO scores independently predicted survival after adjustments for patient-, disease-, and transplant-related factors. The adjusted probabilities of 1-year survival were 56%, 67%, 75%, and 76% by increasing quartiles of the pre-HCT FACT-BMT score and 58%, 72%, 62%, and 82% by increasing quartiles of the pre-HCT SF-36 physical component score.

CONCLUSIONS: A hybrid model of local consent for centralized PRO collection is feasible, and pretransplant PROs provide critical prognostic information for HCT outcomes. Cancer 2017;123:4687-4700. © 2017 American Cancer Society.

Author List

Shaw BE, Brazauskas R, Millard HR, Fonstad R, Flynn KE, Abernethy A, Vogel J, Petroske C, Mattila D, Drexler R, Lee SJ, Horowitz MM, Rizzo JD


Ruta Brazauskas PhD Associate Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Kathryn Eve Flynn PhD Associate Professor in the Medicine department at Medical College of Wisconsin
Mary M. Horowitz MD, MS Center Director, Professor in the Medicine department at Medical College of Wisconsin
J. Douglas D. Rizzo MD, MS Director, Ctr Associate Director, Professor in the Medicine department at Medical College of Wisconsin
Bronwen E. Shaw MBChB, PhD Professor in the Medicine department at Medical College of Wisconsin


2-s2.0-85034085598   4 Citations

MESH terms used to index this publication - Major topics in bold

Aged, 80 and over
Child, Preschool
Data Collection
Feasibility Studies
Follow-Up Studies
Hematologic Neoplasms
Hematopoietic Stem Cell Transplantation
Middle Aged
Neoplasm Staging
Patient Reported Outcome Measures
Prospective Studies
Quality of Life
Transplantation Conditioning
Transplantation, Homologous
Young Adult
jenkins-FCD Prod-332 f92a19b0ec5e8e1eff783fac390ec127e367c2b5