Patient-reported outcomes in HSCT for autoimmune diseases: Considerations on behalf of the EBMT ADWP, PAC, and Nurses Group

Tobias Alexander, MD; Noëlle Tassy, PhD(c); Ariadna Domenech, RN; Ellen Kramer, MD; Helen Jessop, RN; Michelle Kenyon, RN; Basil Sharrack, MD, PhD; Riccardo Saccardi, MD; Natacha Bolanos; John A. Snowden, MD; Raffaella Greco, MD

Journal of Allergy and Clinical Immunology: Global (Aug 2024)

Patient-reported outcomes in HSCT for autoimmune diseases: Considerations on behalf of the EBMT ADWP, PAC, and Nurses Group

Tobias Alexander, MD,
Noëlle Tassy, PhD(c),
Ariadna Domenech, RN,
Ellen Kramer, MD,
Helen Jessop, RN,
Michelle Kenyon, RN,
Basil Sharrack, MD, PhD,
Riccardo Saccardi, MD,
Natacha Bolanos,
John A. Snowden, MD,
Raffaella Greco, MD

Affiliations

Tobias Alexander, MD: Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and the Department of Rheumatology and Clinical Immunology, Berlin Institute of Health, Berlin, Germany
Noëlle Tassy, PhD(c): EBMT Patient Advocacy Committee (PAC), EBMT Executive Office, Barcelona, Spain; Philosophy Department, University of Rouen, Rouen, France
Ariadna Domenech, RN: Bone Marrow Transplant Unit, Department of Hematology, Hospital Clínic of Barcelona, Barcelona, Spain
Ellen Kramer, MD: EBMT Patient Advocacy Committee (PAC), EBMT Executive Office, Barcelona, Spain; Department of Hematology, Amsterdam UMC, Amsterdam, The Netherlands
Helen Jessop, RN: Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom
Michelle Kenyon, RN: Department of Haematology, King’s College Hospital, London, United Kingdom
Basil Sharrack, MD, PhD: Department of Neuroscience and Sheffield NIHR Translational Neuroscience BRC, Sheffield Teaching Hospitals NHS Foundation Trust and University of Sheffield, Sheffield, United Kingdom
Riccardo Saccardi, MD: Cellular Therapies and Transfusion Medicine Unit, Careggi University Hospital, Florence, Italy
Natacha Bolanos: EBMT Patient Advocacy Committee (PAC), EBMT Executive Office, Barcelona, Spain; Lymphoma Coalition, Mississauga, Ontario, Canada
John A. Snowden, MD: Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom; Division of Clinical Medicine, School of Medicine and Population Health, The University of Sheffield, Sheffield, United Kingdom
Raffaella Greco, MD: Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy; Corresponding author: Raffaella Greco, MD, Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, via Olgettina 60, Milan 20132, Italy.

Journal volume & issue: Vol. 3, no. 3
p. 100283

Abstract

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Background: Over the last 3 decades, hematopoietic stem cell transplantation (HSCT) has been successfully used to treat severe and refractory autoimmune diseases (AIDs). A multidisciplinary appraisal of potential benefits and risks by disease and transplant specialists is essential to determine individual suitability for HSCT. Objective: Our aim was to observe that patient-reported outcomes (PROs) and health-related quality of life instruments can capture the unique patient perspective on disease burden and impact of treatment. Methods: Herein, we describe the basis and complexity of end points measuring patient-reported perceptions of efficacy and tolerability used in clinical practice and trials for patients with AIDs undergoing autologous HSCT. Results: PRO measures and patient-reported experience measures are key tools to evaluate the impact and extent of disease burden for patients affected by AIDs. For formal scientific assessment, it is essential that validated general instruments are used, whereas adaptations have resulted in disease-specific instruments that may help guide tailored interventions. An additional approach relates to qualitative evaluations, from carefully structured qualitative research to informal narratives, as patient stories. The patients’ subjectively reported responses to HSCT may be influenced by their preprocedure expectations and investment in the HSCT journey. Conclusions: The complexity of AIDs advocates for individualized and multidisciplinary approach to positively affect the patient journey. PROs and health-related quality of life need to be collected using validated instruments in clinical practice and trials to enable robustness of data and to ensure the impact of the intervention is comprehensively assessed, addressing the main questions and needs of the involved stakeholders.

Published in Journal of Allergy and Clinical Immunology: Global

ISSN: 2772-8293 (Online)
Publisher: Elsevier
Country of publisher: United States
LCC subjects: Medicine: Internal medicine: Specialties of internal medicine: Immunologic diseases. Allergy
Website: https://www.sciencedirect.com/journal/journal-of-allergy-and-clinical-immunology-global

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