Patient-reported outcomes - the missing link to advancing light chain (AL) amyloidosis clinical research

Systemic light chain (AL) amyloidosis often results in multi-organ dysfunction and significant morbidity. Clinical assessments may not capture the full impact of disease and treatment on patients. Patient-reported outcomes (PROs) can help fill this gap. Although evidence suggests that the use of PROs provides additional predictive value beyond established cardiac staging systems, their integration into standard AL amyloidosis management remains limited. Our review examines the prognostic and therapeutic value of PROs and their current use as endpoints in clinical trials. We also discuss practical considerations, including instrument selection and administration, data interpretation, and reporting. Finally, we present a roadmap for integrating PROs into routine AL amyloidosis management, focusing on the selection of appropriate ePRO platforms and implementation strategies. We advocate a framework for data sharing and a coordinated research agenda. By addressing evidence gaps and prioritizing the patient perspective, PROs have the potential to advance AL amyloidosis care and research.