How to measure success in indolent disease? Endpoints, toxicity and more

Graham Collins

Graham Collins, MA, MBBS, MRCP, FRCPath, DPhil of Oxford University Hospitals NHS Foundation Trust, Oxford, UK provides an overview of the session on how to measure success in indolent disease held at the 2017 British Society for Haematology (BSH) Annual Scientific Meeting in Brighton, UK. Dr Collins explains that the focus of the session is on the challenges in indolent disease that arise with long survival. Further, new agents such as BTK inhibitors and PI3K inhibitors are given for extended periods of time and are associated with side effects that have an impact on the patients’ quality of life. It is therefore important to consider how to judge the tolerability of these new drugs and realize that even grade I or II adverse events can significantly impact quality of life. Another topic of discussion is if there are any surrogate endpoints that can be used to measure success. Dr Collins discusses overall survival (OS), CR30, minimal residual disease (MRD) assessment, and time to next treatment. He also highlights the importance of reconsidering toxicities, how to measure them, and the importance of asking the patients for their opinion on what is important to them.

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