Patient-reported outcomes in hematology: is it time to focus more on them in clinical trials and hematology practice?

Abstract

Abstract In less than 2 decades, major clinical advances have been made in various areas of hematologic malignancies. Clinicians and patients now frequently face challenging choices regarding various treatments that are often similar in regard to safety or clinical effectiveness; hence, medical decision making has grown in complexity. For example, several novel drugs have been developed as oral agents, introducing an additional challenge in patient management, such as ensuring an optimal adherence to therapy in order to maximize drug effectiveness. This rapidly changing scenario provides a rationale for a more systematic collection of patient-reported outcomes (PRO) in clinical research and routine care. In the former case, PRO may help to better understand overall treatment effectiveness of a new drug being tested. In the latter case, it may aid in making more informed, individualized treatment decisions in daily practice by obtaining more accurate information on the actual symptom burden experienced by the patient. In any case, evaluating PRO requires making several, and often challenging, decisions depending, for example, on the population being studied and the specific setting. Therefore, if PROs are to fulfill their potential of generating clinically meaningful data that robustly inform patient care, special attention should be given to methodological rigor. We outline the value of a more systematic and rigorous implementation of PRO assessment in the current hematology arena, by providing some real world examples of how PRO data have contributed in better understanding the value of new therapies. We also discuss practical considerations in PRO assessment in clinical research.