Use and Reporting of Patient-Reported Outcomes in Trials of Palliative Radiotherapy

Abstract

ImportanceApproximately 50% of all patients with cancer have an indication for radiotherapy, and approximately 50% of radiotherapy is delivered with palliative intent, with the aim of alleviating symptoms. Symptoms are best assessed by patient-reported outcomes (PROs), yet their reliable interpretation requires adequate reporting in publications.ObjectiveTo investigate the use and reporting of PROs in clinical trials of palliative radiotherapy.Evidence ReviewThis preregistered systematic review searched PubMed/Medline, EMBASE, and the Cochrane Center Register of Controlled Trials for clinical trials of palliative radiotherapy published from 1990 to 2020. Key eligibility criteria were palliative setting, palliative radiotherapy as treatment modality, and clinical trial design (per National Institutes of Health definition). Two authors independently assessed eligibility. Trial characteristics were extracted and standard of PRO reporting was assessed in adherence to the Consolidated Standards of Reporting Trials (CONSORT) PRO extension. The association of the year of publication with the use of PROs was assessed by logistic regression. Factors associated with higher CONSORT-PRO adherence were analyzed by multiple regression. This study is reported following the PRISMA guidelines.FindingsAmong 7377 records screened, 225 published clinical trials representing 24 281 patients were eligible. Of these, 45 trials (20%) used a PRO as a primary end point and 71 trials (31%) used a PRO as a secondary end point. The most prevalent PRO measures were the Numeric Rating Scale/Visual Analogue Scale (38 trials), European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire C30 (32 trials), and trial-specific unvalidated measures (25 trials). A more recent year of publication was significantly associated with a higher chance of PROs as a secondary end point (odds ratio [OR], 1.04 [95% CI, 1.00-1.07]; P = .03) but not as primary end point. Adherence to CONSORT-PRO was poor or moderate for most items. Mean (SD) adherence to the extension adherence score was 46.2% (19.6%) for trials with PROs as primary end point and 31.8% (19.8%) for trials with PROs as a secondary end point. PROs as a primary end point (regression coefficient, 9.755 [95% CI, 2.270-17.240]; P = .01), brachytherapy as radiotherapy modality (regression coefficient, 16.795 [95% CI, 5.840-27.751]; P = .003), and larger sample size (regression coefficient, 0.028 [95% CI, 0.006-0.049]; P = .01) were significantly associated with better PRO reporting per extension adherence score.Conclusions and RelevanceIn this systematic review of palliative radiotherapy trials, the use and reporting of PROs had room for improvement for future trials, preferably with PROs as a primary end point.

The impact of palliative radiotherapy on health-related quality of life in patients with head and neck cancer – Results of a multicenter prospective cohort study

“…These previous studies, however, mainly reported symptom improvement by any degree without reporting MID. In fact, reporting of PRO data in trials of palliative radiotherapy in general appears to be suboptimal as evidenced by a recent systematic review of our group [27] . Pain caused by the primary tumor per NRS is another common PRO measure [28] .…”

The value of patient reported outcomes in palliative radiotherapy: A discussion in light of current findings

“…In the September 2022 issue of JAMA Network Open, Fabian et al presented findings from their very relevant systematic review (1). They investigated the use of PROs as endpoints in palliative radiotherapy trials.…”

“…The scientific and clinical communities are on their way. As Fabian et al (1) reported, the use of PROs and the adherence to reporting guidelines are increasing, and in the long run, study investigators and sponsors will not be able to ignore, but to use the chance of guidance from regulatory authorities such as the European Medicines Agency (EMA) and the FDA, medical associations such as European Organisation for Research and Treatment of Cancer (EORTC) (7) and ESMO (18), and patient advocates (7).…”

“…Why patient reported outcomes (PROs) should be routinely captured in trials and the clinic Radiation therapy is commonly delivered with palliative intent (1,2). The aim is to either alleviate or prevent symptoms, such as pain and breathlessness, or to prolong life expectancy (3).…”

The use of electronic Patient Reported Outcomes in follow-up after palliative radiotherapy: A survey study in Belgium

“…However, in the advanced/palliative cancer setting, the importance of using (e)PROMs has been primarily proven for systemic therapy [ 23 , 24 ]: improving clinical effectiveness, treatment adherence, symptom reporting, quality of care and survival rates [ 19 , [24] , [25] , [26] , [27] , [28] ]. Far less research has been performed on ePROM use and implementation of ePROM in routine clinical care after palliative radiotherapy, especially regarding individual symptoms [ 22 , 29 , 30 ]. This could be because implementation of (e)PROMs after radiotherapy (in the palliative setting) is different than in systemic treatment: radiation courses are short with symptoms usually occurring after completion of treatment, compared to longer courses of systemic therapy where symptoms occur during treatment.…”