Identification of Patient Important Outcomes in Lung Transplantation

Lung transplantation (LT) is an exceptional treatment for severe patients. It is a lengthy process, and its outcomes may seem burdensome. LT is the subject of thriving research: a search on ClinicalTrials with the terms 'Lung Transplant' retrieves 85 ongoing studies. Outcome measures (OM) are used to assess the impact of an intervention during a prognostic study. They must be carefully chosen, relevant, reproducible, and validated. It is challenging for researchers to find a balance between these OM, which should be objective, reliable, easily evaluable, and OM that are important to the patient (patient-important outcomes - PIO) and integrate into their daily lives. The emergence of PIO has occurred in many fields (Dinglas Thorax 2018, Partridge Lancet 2004). They 'reflect what the patient feels, their functional state, or under what conditions they survive' (Wittes, Stat Med 1989) and cover various domains (pain, mobility, autonomy, quality of life, etc.). The role of PIO in LT has never been evaluated. We conducted a systematic review aiming to describe the role of PIO in LT research, published in 2019 (Weisenburger et al., Respir Med Res 2022). At least one PIO was used in 26 out of the 51 included studies, with only 11 evaluating a PIO other than mortality. Some transplant physicians are well aware of this gap (Lamas, NEJM 2018). Placing the patient's concerns at the center is considered an essential objective in patient care and clinical research, including in LT. PIO could encompass mortality and patient-reported OM (PROMS), but there could also be OM that we, clinicians and researchers, have not considered, which could be crucial for patients. Gathering the opinions of lung transplant patients, their families, and healthcare providers (physicians and paramedics) involved in LT will help define a set of PIO in LT. This work proposes an original approach to identify PIO in LT in order to define a 'core outcome set,' a recommended set of outcome measures to be used. The development of a 'core outcome set' incorporating PIO can only be achieved through the participation of patients (Needham, AJRCCM 2017) in collaboration with clinicians and researchers. A Delphi-like methodology allows for the establishment of such criteria. Involving patients and their families places their concerns at the center of research that affects them. The two participating LT centers complement each other in terms of patient recruitment (preferentially patients with chronic obstructive bronchopneumopathy or fibrosing pneumopathies at Bichat Hospital; preferentially younger patients with cystic fibrosis at Foch Hospital). The development of a 'core outcome set' will guide the LT research community towards the selection of relevant OM for patients and healthcare providers. Such work would also involve patients and their families, which is important in the field of LT research. Our results could be used to provide recommendations for future LT research and help standardize prognostic evaluation measures. The methods for assessing the performance of LT centers could be reviewed and not solely rely on the isolated measurement of mortality." Practical implementation: A Delphi survey will be conducted. This method aims to gather anonymous opinions from one or more groups of experts in order to reach a consensus on subjects with uncertain definitions (Linstone et al. 2002; Fink et al. Am J Public Health 1984).

This methodology has been used, among others, to identify important evaluation domains for survivors of acute respiratory distress syndrome and their families (Dinglas, Thorax 2018).

Formation of four panels:

• Patients followed in one of the two participating centers (Bichat Hospital, Paris; Foch Hospital, Suresnes).
• Relatives (spouses, partners, or, if not available, first-degree relatives) of patients in one of the participating centers.
• Clinician doctors and researchers from LT centers, belonging to the lung transplantation group of the French Language Pneumology Society or the francophone network of investigators.
• Paramedical caregivers (nurses, advanced practice nurses, transplantation coordination nurses, psychologists, physiotherapists, etc.) working in one of the French or identified French-speaking centers.