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

Background Over the last three 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. Objectives Patient-reported outcomes (PROs) and Health-related Quality of Life (HRQoL) instruments can capture the unique patient perspective on disease burden and impact of treatment. Methods Herein, we describe the basis and complexity of endpoints measuring patient-reported perceptions of efficacy and tolerability used in clinical practice and trials for patients with ADs undergoing autologous HSCT. Results Patient-reported outcome measures (PROMs) and patient-reported experience measures (PREM) are key tools to evaluate the impact and extent of disease burden for patients affected by ADs. For formal scientific assessment, it is essential that validated general instruments are used, while adaptations have resulted in disease specific instruments that may help guiding 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 pre-procedure expectations and investment in HSCT journey. Conclusions The complexity of AIDs advocates for individualized and multidisciplinary approach to impact positively the patient journey. PROs and HRQoL need to be collected using validated instruments in clinical practice and trials to enable robustness of data, ensure the impact of the intervention is comprehensively assessed, addressing the main questions and needs of the involved stakeholders.