Dittrich Tobias, Weinert Lina, D'Souza Anita
Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg University, Heidelberg, Germany; Institute of Medical Informatics, Heidelberg University Hospital, Heidelberg University, Heidelberg, Germany.
Heidelberg Institute of Global Health, Section for Oral Health, Heidelberg University, Heidelberg, Germany.
Blood Rev. 2025 May 20:101303. doi: 10.1016/j.blre.2025.101303.
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.
系统性轻链(AL)淀粉样变性常导致多器官功能障碍和严重发病。临床评估可能无法全面反映疾病和治疗对患者的影响。患者报告结局(PROs)有助于填补这一空白。尽管有证据表明,使用PROs除了已有的心脏分期系统外还能提供额外的预测价值,但其在标准AL淀粉样变性管理中的整合仍然有限。我们的综述探讨了PROs的预后和治疗价值以及它们目前在临床试验中作为终点指标的应用。我们还讨论了实际考虑因素,包括工具选择与应用、数据解读和报告。最后,我们提出了将PROs整合到常规AL淀粉样变性管理中的路线图,重点关注合适的电子PRO平台选择和实施策略。我们倡导数据共享框架和协调的研究议程。通过填补证据空白并优先考虑患者视角,PROs有潜力推动AL淀粉样变性的治疗和研究。
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