Abstract / Summary
Abstract Background The use of Clinical Outcome Assessments (COA) instruments in blood cancers such as myeloproliferative neoplasms (MPN) is widely documented, however little is known about Essential Thrombocythemia (ET). A targeted literature review was conducted to identify the concepts of interest to the ET population and to develop a conceptual framework. Using this framework, identification and review of COA instruments will help determine if there are existing instruments fit for purpose for use in ET studies. Methodology Targeted reviews in May and October 2023 synthesized the literature on ET symptoms and impacts to derive a conceptual model and identify suitable COAs. Eligible articles consisted of ET only populations and extended to MPN samples that included ET patients. A gap analysis was conducted on selected instruments. Results Of the 890 abstracts reviewed, 206 were included in the analysis. An ET conceptual model that encompassed disease-defining concepts (fatigue most common), proximal, distal, and general life impacts was created. MPN specific measures such as the Myelofibrosis Symptom Assessment Form v4.0 (MFSAF), and Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; MPN-10), European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core Questionnaire (EORTC QLQ-C30), Patient-Reported Outcomes Measurement Information System (PROMIS) - Fatigue, and Brief Fatigue Inventory (BFI) were deemed the most appropriate for the gap analysis. Each had strengths and limitations related to conceptual coverage, evidence for measurement properties, and feasibility for use in clinical trials. Conclusions The MFSAF v4.0, PROMIS-Fatigue, and EORTC QLQ-C30 are considered appropriate for use in deriving trial endpoints in ET; however further work is needed to assess the fitness-for-purpose in the ET population as most validation studies were performed in mixed MPN or oncology samples.