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Can we agree on patient-reported outcome measures for assessing hematopoietic cell transplantation patients? A study from the CIBMTR and BMT CTN

Bone Marrow Transplantation volume 51, pages 1173–1179 (2016)Cite this article

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Abstract

Much research into the impact of hematopoietic cell transplantation (HCT) on recipients’ symptoms, functioning and health-related quality of life uses diverse patient-reported outcome (PRO) measures. Robust conclusions regarding PROs in HCT patients are constrained by methodological issues, including the use of multiple different and noncomparable assessment measures. We reviewed 114 publications addressing PROs in HCT patients. Although three multi-item measures were most frequently used (FACT-BMT, n=28; European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30, n=26; and SF-36, n=26), 25 additional measures were used in more than one study. Another 50 measures were used in single studies. Over 50% of studies used more than one measure. We recommend that the field agrees upon a set of measures to address the core domains important to patients, to reduce heterogeneity and allow comparisons across studies and between different populations. Measures should be available in a free and easily accessible manner internationally. We discuss the relative benefits of the National Institutes of Health-supported Patient-Reported Outcomes Measurement Information System (PROMIS) system to achieve these goals. To further address these issues, the Blood and Marrow Transplant Clinical Trials Network has recently created a task force to implement PROMIS measures alongside traditional PRO measures in future clinical trials. Robust comparisons between measures in this setting may allow for the development of a standard for HCT patients.

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Acknowledgements

Support for this study was provided by a grant to the BMT CTN: #U10HL069294 from the National Heart, Lung and Blood Institute and the National Cancer Institute, and the Health Resources and Services Administration Contract No. HHSH234200637020C. Dr Flynn was funded in part by the Research and Education Program Fund, a component of the Advancing a Healthier Wisconsin endowment at the Medical College of Wisconsin. Any views, opinions, findings, conclusions or recommendations expressed in this material are those of the author(s) and do not reflect the views or the official policy or position of the above mentioned parties.

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Authors and Affiliations

  1. Department of Medicine, CIBMTR (Center for International Blood and Marrow Transplant Research), Froedert and the Medical College of Wisconsin, Milwaukee, WI, USA

    B E Shaw, M M Horowitz & J D Rizzo

  2. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA

    S J Lee

  3. Division of Hematology/Oncology, Department of Medicine, University of North Carolina, Chapel Hill, NC, USA

    W A Wood

  4. Department of Medicine, Center for Patient Care and Outcomes Research, Medical College of Wisconsin, Milwaukee, WI, USA

    K E Flynn

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  1. B E Shaw
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  2. S J Lee
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  3. M M Horowitz
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  4. W A Wood
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  5. J D Rizzo
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  6. K E Flynn
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Correspondence to B E Shaw.

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Shaw, B., Lee, S., Horowitz, M. et al. Can we agree on patient-reported outcome measures for assessing hematopoietic cell transplantation patients? A study from the CIBMTR and BMT CTN. Bone Marrow Transplant 51, 1173–1179 (2016). https://doi.org/10.1038/bmt.2016.113

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