SOHO State of the Art Updates and Next Questions | Measuring Patient-Reported Outcomes (PROs) and Treatment Tolerability in Patients with Hematologic Malignancies

Abstract

There has been a rapid expansion of novel therapeutics for hematologic malignancies, including monoclonal antibodies, small molecules, and cellular therapies, which confer different treatment-related toxicities and symptomatic adverse events (AEs) than traditional cytotoxic chemotherapies. Given that patients with blood cancers are living longer with these newer treatments, with some therapies requiring indefinite or time-intensive administration, consideration of patient-reported tolerability and effects on health-related quality of life (HRQoL) are increasingly relevant. Historically, clinical trials have focused on the efficacy and safety of therapies. While related to safety and not intended to replace it, “treatment tolerability” is a distinct construct defined as the extent to which symptomatic and non-symptomatic AEs impact a patient's ability and desire to continue with current treatment dosing, which also encompasses how patients feel and function while undergoing anti-cancer therapies. Assessment of tolerability requires the systematic and rigorous measurement of patient-reported outcomes (PROs). In this review, we discuss the introduction of patient-reported outcomes measures (PROMs) into hematology clinical trials and how PROs inform the measurement of treatment tolerability, including symptomatic adverse events, physical and role functioning, and overall side effect burden. Selected PROMs for measurement of these core tolerability domains are outlined, with a focus on novel analytic tools that have been developed for the longitudinal analysis of tolerability data. Further, we outline ongoing studies to accelerate integration of PROs throughout the cancer care spectrum, from early-stage drug development to routine clinical care, with the goal of improving both HRQoL and survival.