Standardizing Patient-Reported Outcomes Assessment in Cancer Clinical Trials: A Patient-Reported Outcomes Measurement Information System Initiative

Abstract

Patient-reported outcomes (PROs), such as symptom scales or more broad-based health-related quality-of-life measures, play an important role in oncology clinical trials. They frequently are used to help evaluate cancer treatments, as well as for supportive and palliative oncology care. To be most beneficial, these PROs must be relevant to patients and clinicians, valid, and easily understood and interpreted. The Patient-Reported Outcomes Measurement Information System (PROMIS) Network, part of the National Institutes of Health Roadmap Initiative, aims to improve appreciably how PROs are selected and assessed in clinical research, including clinical trials. PROMIS is establishing a publicly available resource of standardized, accurate, and efficient PRO measures of major self-reported health domains (eg, pain, fatigue, emotional distress, physical function, social function) that are relevant across chronic illnesses including cancer. PROMIS is also developing measures of self-reported health domains specifically targeted to cancer, such as sleep/wake function, sexual function, cognitive function, and the psychosocial impacts of the illness experience (ie, stress response and coping; shifts in self-concept, social interactions, and spirituality). We outline the qualitative and quantitative methods by which PROMIS measures are being developed and adapted for use in clinical oncology research. At the core of this activity is the formation and application of item banks using item response theory modeling. We also present our work in the fatigue domain, including a short-form measure, as a sample of PROMIS methodology and work to date. Plans for future validation and application of PROMIS measures are discussed.

Patient‐reported acute gastrointestinal symptoms during concurrent chemoradiation treatment for rectal cancer

“…7,[19][20][21][22][23] Therefore, there is increasing recognition of the value of PROs in the evaluation of the treatment impact on patient QOL, in assessments of treatment tolerability, and as part of the decision-making process by physicians on whether to adopt new therapies for their patients. 7,24,25 This recognition has led to efforts by the National Cancer Institute, working with the cooperative groups, to encourage and facilitate the incorporation of PROs into phase 2, phase 3, and supportive therapy trials. …”

“…We were able to achieve a high completion rate of 95%, and only 2of 77 consecutive patients were excluded because of nonparticipation, probably attributable in part to the low response burden from the short questionnaire. 23,25,26 The instrument appeared to be appropriate for this patient population and treatment, and all items were sensitive to treatment-related changes. Anecdotally, we believe that the PRO measurement did not lengthen clinical visits and that having PROs available facilitated communication and the management of patient symptoms.…”

Rethinking fatigue in Gaucher disease

“…The measurement of fatigue using standard, validated instruments is common in many chronic illnesses, including cancer, chronic inflammatory diseases, depression, human immunodeficiency virus, and stroke, but generally not in GD [32][33][34]. The results of our literature review support fatigue as a core symptom in patients with Type 1 GD.…”

Evaluation of quality of life after breast reconstruction using an autologous latissimus dorsi myocutaneous flap

“…The mean score in our population was 3.36 [0][1][2][3][4][5]. Age (p=0.6116), BMI (p=0.4343), the severity of complications (p=0.0765), a history of an earlier lumpectomy (p=0.911), the type of reconstruction (p=0.…”