OBJECTIVES

In HM clinical trials PROs are an important complement to their clinical endpoints and are key to understanding the overall treatment benefit and effectiveness of new interventions being tested. To guide researchers and clinicians in their choice of PRO assessment tools, this poster will provide an overview of HM specific PRO instruments, describe their content, and highlight commonalities and differences between measures.

METHODS

: A literature review was performed using PsycINFO and MEDLINE to identify HM studies published in English between 1990 and 2019 that reported on PROs assessing both symptoms and health related quality of life (HRQoL). Additional searches were conducted on clinicaltrials.gov to identify PROs used in HM clinical trials. A review of identified PROs was then conducted examining their content.

RESULTS

: 5 disease-specific symptom inventories and 12 HRQoL PROs were identified from 46 HM trials and 37 HM research studies. Most of the instruments were indication-specific versions of three general measures: the MD Anderson Symptom Inventory (MDASI) (n=3), the Functional Assessment of Cancer Therapy (FACT) (n=4), and the European Organisation for Research and Treatment of Cancer quality of life questionnaire (EORTC-QLQ) (n=6).

Some of the most common areas of investigation between the instruments include: symptom burden, physical condition/fatigue, worries/fears on health and functioning, and emotional impact. A fundamental difference was whether the instrument was limited to measuring symptom burden or attempted to assess HRQoL as well. The measures also differed in the weight placed on various symptoms and how optimal functioning or treatment benefit was conceptualised.

CONCLUSIONS

: This review highlighted a lack of disease-specific HR instruments as well as the limited availability of comprehensive and valid symptom instruments to capture the experiences of patients with HM during and after treatment. Further improvements to already exiting measures such as better coverage disease-specific symptoms are recommended, especially for myeloproliferative neoplasm and mycosis fungoides.