Abstract
The EBMT (European Blood and Marrow Transplantation Society) aims to connect patients, the scientific community, and other stakeholders to improve hematopoietic stem cell transplantation and cellular therapy outcomes. We performed a cross-sectional online survey to understand the perceptions regarding Patient Reported Outcomes (PROs) and Patient Active Involvement in Research (PAIR) in over 800 stakeholders (n = 813). Patients (n = 278) and health care professionals (HCPs) (n = 351) were compared. We observed high openness for EBMT PRO collection (n = 680, 84.5% across stakeholders’ groups; patients n = 256, 93.1% versus HCPs n = 273, 78.4% [p < 0.001]) and PAIR (n = 702, 87.3% across stakeholder groups; patients n = 256, 92.4% versus HCPs n = 296, 85.8% [p = 0.009]), with a significantly higher proportion of patients expressing interest compared to HCPs. Priority domains for PROs data-collection identified were the assessment of symptom experience, psychosocial and cognitive functioning. The most important issues for patients specifically were the data-collection of PROs reflecting cognitive function, the option of reporting data at home, the importance of identifying actionable targets to improve their recovery, and receiving feedback on their input when participating in research projects. Our multistakeholder approach suggests an added value to embracing patient engagement in the development of meaningful research and service design within the transplantation and cellular therapy community.
Graphical abstract
![](http://media.springernature.com/lw685/springer-static/image/art%3A10.1038%2Fs41409-024-02290-7/MediaObjects/41409_2024_2290_Figa_HTML.png)
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on SpringerLink
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
![](http://media.springernature.com/m312/springer-static/image/art%3A10.1038%2Fs41409-024-02290-7/MediaObjects/41409_2024_2290_Fig1_HTML.png)
![](http://media.springernature.com/m312/springer-static/image/art%3A10.1038%2Fs41409-024-02290-7/MediaObjects/41409_2024_2290_Fig2_HTML.png)
Similar content being viewed by others
Data availability
The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.
References
Devisch I. Progress in medicine: Autonomy, oughtonomy and nudging, vol. 17. 2011.
Batten LM, Bhattacharya IS, Moretti L, Haviland JS, Emson MA, Miller SE, et al. Patient advocate involvement in the design and conduct of breast cancer clinical trials requiring the collection of multiple biopsies. Res Involv Engagem. 2018;4:22. https://doi.org/10.1186/s40900-018-0108-0.
O’Donnell D, Ni She E, McCarthy M, Thornton S, Doran T, Smith F, et al. Enabling public, patient and practitioner involvement in co-designing frailty pathways in the acute care setting. BMC Health Serv Res. 2019;19:797. https://doi.org/10.1186/s12913-019-4626-8.
Deverka PA, Bangs R, Kreizenbeck K, Delaney DM, Hershman DL, Blanke CD, et al. A New Framework for Patient Engagement in Cancer Clinical Trials Cooperative Group Studies. J Natl Cancer Inst. 2018;110:553–9. https://doi.org/10.1093/jnci/djy064.
Sacristan JA, Aguaron A, Avendano-Sola C, Garrido P, Carrion J, Gutierrez A, et al. Patient involvement in clinical research: why, when, and how. Patient preference adherence. 2016;10:631–40. https://doi.org/10.2147/ppa.S104259.
Frank L, Morton SC, Guise JM, Jull J, Concannon TW, Tugwell P. Engaging Patients and Other Non-Researchers in Health Research: Defining Research Engagement. J Gen Intern Med. 2020;35:307–14. https://doi.org/10.1007/s11606-019-05436-2.
Grady PA, Gough LL. Self-management: a comprehensive approach to management of chronic conditions. Am J Public Health. 2014;104:e25–31. https://doi.org/10.2105/ajph.2014.302041.
Bevans M, El-Jawahri A, Tierney DK, Wiener L, Wood WA, Hoodin F, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Patient-Centered Outcomes Working Group Report. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2017;23:538–51. https://doi.org/10.1016/j.bbmt.2016.09.011.
Shaw BE, Brazauskas R, Millard HR, Fonstad R, Flynn KE, Abernethy A et al. Centralized patient-reported outcome data collection in transplantation is feasible and clinically meaningful. Cancer 2017. https://doi.org/10.1002/cncr.30936.
Shaw BE, Syrjala KL, Onstad LE, Chow EJ, Flowers ME, Jim H, et al. PROMIS measures can be used to assess symptoms and function in long-term hematopoietic cell transplantation survivors. Cancer. 2018;124:841–9. https://doi.org/10.1002/cncr.31089.
Wood WA, Le-Rademacher J, Syrjala KL, Jim H, Jacobsen PB, Knight JM, et al. Patient-reported physical functioning predicts the success of hematopoietic cell transplantation (BMT CTN 0902). Cancer. 2016;122:91–8. https://doi.org/10.1002/cncr.29717.
El-Jawahri A, Chen YB, Brazauskas R, He N, Lee SJ, Knight JM, et al. Impact of pre-transplant depression on outcomes of allogeneic and autologous hematopoietic stem cell transplantation. Cancer. 2017;123:1828–38. https://doi.org/10.1002/cncr.30546.
Muffly LS, Kocherginsky M, Stock W, Chu Q, Bishop MR, Godley LA, et al. Geriatric assessment to predict survival in older allogeneic hematopoietic cell transplantation recipients. Haematologica. 2014;99:1373–9. https://doi.org/10.3324/haematol.2014.103655.
Hamilton BK, Law AD, Rybicki L, Abounader D, Dabney J, Dean R, et al. Prognostic significance of pre-transplant quality of life in allogeneic hematopoietic cell transplantation recipients. Bone Marrow Transpl. 2015;50:1235–40. https://doi.org/10.1038/bmt.2015.122.
Chakraborty R, Sidana S, Shah GL, Scordo M, Hamilton BK, Majhail NS. Patient-Reported Outcomes with Chimeric Antigen Receptor T Cell Therapy: Challenges and Opportunities. Biol Blood Marrow Transpl: J Am Soc Blood Marrow Transplant. 2019;25:e155–62. https://doi.org/10.1016/j.bbmt.2018.11.025.
Burns LJ, Abbetti B, Arnold SD, Bender J, Doughtie S, El-Jawahiri A, et al. Engaging Patients in Setting a Patient-Centered Outcomes Research Agenda in Hematopoietic Cell Transplantation. Biol Blood Marrow Transpl: J Am Soc Blood Marrow Transplant. 2018. https://doi.org/10.1016/j.bbmt.2018.01.029.
Schoemans HM, Finn L, Foster J, Roche-Green A, Bevans M, Kullberg S, et al. A Conceptual Framework and Key Research Questions in Educational Needs of Blood and Marrow Transplantation Patients, Caregivers, and Families. Biol Blood Marrow Transpl: J Am Soc Blood Marrow Transplant. 2019;25:1416–23. https://doi.org/10.1016/j.bbmt.2019.02.017.
Pfadenhauer LM, Gerhardus A, Mozygemba K, Lysdahl KB, Booth A, Hofmann B, et al. Making sense of complexity in context and implementation: the Context and Implementation of Complex Interventions (CICI) framework. Implement Sci. 2017;12:21. https://doi.org/10.1186/s13012-017-0552-5.
Mielke J, Leppla L, Valenta S, Zullig LL, Zúñiga F, Staudacher S, et al. Unraveling implementation context: the Basel Approach for coNtextual ANAlysis (BANANA) in implementation science and its application in the SMILe project. Implement Sci Commun. 2022;3:102. https://doi.org/10.1186/s43058-022-00354-7.
Briefing notes for researchers - public involvement in NHS, health and social care research, April 2021. In: National Institute for Health and Care Research. 2021.
Goodman MS, Sanders Thompson VL. The science of stakeholder engagement in research: classification, implementation, and evaluation. Transl Behav Med. 2017;7:486–91. https://doi.org/10.1007/s13142-017-0495-z.
Gale NK, Heath G, Cameron E, Rashid S, Redwood S. Using the framework method for the analysis of qualitative data in multi-disciplinary health research. BMC Med Res Methodol. 2013;13:117. https://doi.org/10.1186/1471-2288-13-117.
Lee SJ, Cutler C, Blazar BR, Tu A, Yang Z, Pavletic SZ. Correlation of Patient-Reported Outcomes with Clinical Organ Responses: Data from the Belumosudil Chronic Graft-versus-Host Disease Studies. Transplant Cell Ther. 2022;28:700.e701–700.e706. https://doi.org/10.1016/j.jtct.2022.06.020.
King-Kallimanis BL, Wroblewski T, Kwitkowski V, De Claro RA, Gwise T, Bhatnagar V, et al. FDA review summary of patient-reported outcome results for ibrutinib in the treatment of chronic graft versus host disease. Qual Life Res: Int J Qual Life Asp Treat, care Rehab. 2020;29:1903–11. https://doi.org/10.1007/s11136-020-02448-y.
Zeiser R, Polverelli N, Ram R, Hashmi SK, Chakraverty R, Middeke JM, et al. Ruxolitinib for Glucocorticoid-Refractory Chronic Graft-versus-Host Disease. N Engl J Med. 2021;385:228–38. https://doi.org/10.1056/NEJMoa2033122.
Im A, Pusic I, Onstad L, Kitko CL, Hamilton BK, Alousi AM, et al. Patient-reported treatment response in chronic graft-versus-host disease. Haematologica. 2024;109:143–50. https://doi.org/10.3324/haematol.2023.282734.
Calvert M, Blazeby J, Altman DG, Revicki DA, Moher D, Brundage MD. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. Jama. 2013;309:814–22. https://doi.org/10.1001/jama.2013.879.
Calvert M, Kyte D, Mercieca-Bebber R, Slade A, Chan AW, King MT, et al. Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols: The SPIRIT-PRO Extension. Jama. 2018;319:483–94. https://doi.org/10.1001/jama.2017.21903.
Long CR, Stewart MK, McElfish PA. Health research participants are not receiving research results: a collaborative solution is needed. Trials. 2017;18:449. https://doi.org/10.1186/s13063-017-2200-4.
Hemphill R, Forsythe LP, Heckert AL, Amolegbe A, Maurer M, Carman KL, et al. What motivates patients and caregivers to engage in health research and how engagement affects their lives: Qualitative survey findings. Health Expectations: Int J Public Participation health care health policy. 2020;23:328–36. https://doi.org/10.1111/hex.12979.
Bombard Y, Baker GR, Orlando E, Fancott C, Bhatia P, Casalino S, et al. Engaging patients to improve quality of care: a systematic review. Implement Sci: IS. 2018;13:98. https://doi.org/10.1186/s13012-018-0784-z.
Acknowledgements
We thank all respondents who have taken the time to fill in the survey and share their insights. We are grateful to the EBMT and CIBMTR staff who facilitated its dissemination through virtual channels and at the 2020 EBMT Annual Meeting organized by Professor Rafael Duarte. We express very special thanks to all members of the EBMT patient engagement task force for their help with this project and more specifically to Bregje Verhoeven (patient advocate from the Netherlands and EBMT Patient Advocacy Committee member), Guy Bouguet (patient advocate from France and EBMT Patient Advocacy Committee member), Chris Lewis (patient advocate from the United Kingdom), Dirk Pretzel (informal caregiver advocate from Germany), Paul Johnson (Anthony Nolan staff member), Deborah Buk (DKMS staff member), Anja Van Biezen (EBMT staff member), Iris Bargallo (EBMT staff member), Marianne Mol (EBMT staff member), Emmanuelle Polge (EBMT staff member), Melanie Chaboissier (EBMT staff member) and Lia Vazquez (EBMT staff member) for their invaluable help with this project. HS also acknowledges the support of the UZ Leuven Klinische onderzoeks- en opleidingsraad (KOOR).
Author information
Authors and Affiliations
Contributions
HS, BV, NB, BS, CL, SJL, SDG, JAS, LJB, NK, the EBMT Transplant Complications Working Party, the Patient Advocacy Committee of the EBMT and the EBMT Patient Engagement Task Force designed the study protocol. The survey was reviewed by all participants. CP and WB prepared the data analysis. Results were discussed with all contributing authors, and initially presented as an oral abstract to the 2021 EBMT Annual Meeting. HS wrote the manuscript, which was reviewed and accepted by all collaborating authors.
Corresponding author
Ethics declarations
Competing interests
HS reports having received personal fees from Incyte, Janssen, Novartis, Sanofi and from the Belgian Hematological Society (BHS), as well as research grants from Novartis and the BHS, all paid to her institution and not directly related to this work. She has also received non-financial support (travel grants) from Gilead, Pfizer, the EBMT (European Society for Blood and Marrow transplantation) and the CIBMTR (Center for International Bone Marrow Transplantation Research). SJL reports receiving received non-financial support (travel grants) as secretary of the Haematology Nurses and Healthcare Professionals Group, not directly related to this work. JM reports receiving personal payments from Therakos, Sanofi and Gilead, not directly related to this work. BKH reports adhoc advisory board for Incyte, Sanofi, Nkarta, Rigel; Data Safety Monitoring Board for Angiocrine; adjudication committee for CSL Behring; speaker fees Mallinkrodt. JAS reports consulting fees from Vertex, Jazz and Medac; Data Safety Monitoring Board for Kiadis; unpaid leadership positions for EBTL, BSBMTCT and BSH. BS reports consulting fees for OrcaBio and Mallinkrodt. The other authors have no relevant competing interests to disclose.
Additional information
Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Supplementary information
Rights and permissions
Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.
About this article
Cite this article
Schoemans, H., Burns, L.J., Liptrott, S.J. et al. Patient engagement in hematopoietic stem cell transplantation and cell therapy: a survey by the EBMT patient engagement task force & transplantation complications working party. Bone Marrow Transplant (2024). https://doi.org/10.1038/s41409-024-02290-7
Received:
Revised:
Accepted:
Published:
DOI: https://doi.org/10.1038/s41409-024-02290-7