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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 Transplantation (2024)Cite this article

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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.

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Fig. 1: Opinions of survey participants regarding patient reported outcome (PRO) data collection after hematopoietic stem cell transplantation and cell therapy.
Fig. 2: Opinions of survey participants regarding Patient Active Involvement in Research (PAIR) in hematopoietic stem cell transplantation and cell therapy.

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Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.

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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

Author notes

  1. These authors contributed equally: Zinaida Peric, Nicolaus Kroeger.

Authors and Affiliations

  1. Department of Hematology, University Hospitals Leuven, Leuven, Belgium

    Hélène Schoemans

  2. Department of Public Health and Primary Care, ACCENT VV, KU Leuven - University of Leuven, Leuven, Belgium

    Hélène Schoemans & Sabina De Geest

  3. EBMT Transplant Complications Working Party, Paris, France

    Hélène Schoemans, Olaf Penack, Ivan Moiseev, William Boreland, Christophe Peczynski & Zinaida Peric

  4. Center for International Blood and Marrow Transplant Research (CIBMTR), Milwaukee, WI, USA

    Linda J. Burns

  5. Nursing Research and Development Office, Oncology Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona, Switzerland

    Sarah J. Liptrott

  6. Department of Nursing, Regional Hosptial of Bellinzona and Valli, Ente Ospedaliero Cantonale, Bellinzona, Switzerland

    Sarah J. Liptrott

  7. Haematology and Transplant Unit, The Christie NHS FT, Manchester, UK

    John Murray

  8. Department of Haematology, King’s College Hospital, London, UK

    Michelle Kenyon

  9. Massachusetts General Hospital, Boston, USA

    Anna Barata

  10. Harvard Medical School, Boston, USA

    Anna Barata

  11. EBMT, Chair, Patient Advocacy Committee, Paris, France

    Natacha Bolaños

  12. Lymphoma Coalition, Mississauga, ON, Canada

    Natacha Bolaños

  13. Universitätsklinikum Hamburg-Eppendorf, Institut und Poliklinik für Medizinische Psychologie, Hamburg, Germany

    Isabelle Scholl

  14. Blood and Marrow Transplant Program, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, USA

    Betty Hamilton

  15. Division of Pediatric Hematology, Oncology, Blood and Marrow Transplantation, Department of Pediatrics, Medical College of Wisconsin and Children’s Wisconsin, Milwaukee, WI, USA

    Rachel Phelan

  16. Division of Pediatric Hematology, Children’s Hospital of Orange County, Orange, CA, USA

    David Buchbinder

  17. Medical Clinic, Department for Haematology, Oncology and Tumorimmunology, Charité Universitätsmedizin Berlin, Berlin, Germany

    Olaf Penack

  18. First Pavlov State Medical University of St Petersburg, St Petersburg, Russia

    Ivan Moiseev

  19. EBMT Paris study office; Department of Haematology, Saint Antoine Hospital; INSERM UMR-S 938, Sorbonne University, Paris, France

    William Boreland & Christophe Peczynski

  20. Institute of Nursing Science, Department Public Health, University of Basel, Basel, Switzerland

    Sabina De Geest

  21. Clinical Hematology Department, Institut Català d’Oncologia-Hospitalet, Institut de Ciències Biomèdiques de Bellvitge (IDIBELL), Universitat de Barcelona, Barcelona, 08908, Spain

    Anna Sureda

  22. Sheffield Blood & Marrow Transplant and Cellular Therapy Programme, Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK

    John A. Snowden

  23. CIBMTR, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA

    Bronwen Shaw

  24. Department of Hematology, University Hospital Centre Rijeka, Rijeka, Croatia

    Zinaida Peric

  25. University Medical Center, Hamburg, Germany

    Nicolaus Kroeger

Authors
  1. Hélène Schoemans
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  2. Linda J. Burns
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  4. John Murray
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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

Correspondence to Hélène Schoemans.

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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.

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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

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