Key points are not available for this paper at this time.
QoL assessment is a crucial aspect for patients diagnosed with cancer. Over the years different tools have been developed to measure QoL, both generic and pathology-specific, but the inclusion of QoL among other indicators of efficacy in RCTs remains controversial. In this review we aim to review frequency and modality of QoL assessment in RCTs enrolling patients diagnosed with mesenchymal tumors. An electronic, systematic literature search of bone and soft tissue sarcoma and gastrointestinal stromal tumor (GIST)-related RCTs published between January 2000 and December 2023 was performed by two independent reviewers using PubMed®. English-language, phase II and III clinical trials enrolling at least more than 15 patients were included, regardless of disease stage. Studies involving patients under the age of 18 years or for which the full text was not available were excluded. For each study, data regarding journal and year of publication, study design, primary objective, and evaluation of QoL as endpoint with any type of patient reported outcomes used were extracted. Among 758 publications screened, 155 resulted eligible. QoL assessment was listed among endpoints in 31 trials and QoL results were reported in 15 (48.5%) primary publications. Of all these trials, 14 (45.2%) included patients with soft tissue sarcoma, 6 (19.3%) Kaposi sarcoma, 5 (16.1%) GIST, 4 (12.9%) desmoid tumor. Among all trials included, 8.1% on adjuvant/neoadjuvant setting and 23.7% on metastatic setting included QoL as endpoint. The proportion of trials including QoL was variable over time: 17.9% of trials in 2000-2007, 10.5% in 2008-2015 and 28.6% in 2016-2023. In 31 trials including QoL endpoints, 77.4% of them had a superiority design. In most trials (83.9%) tools for QoL assessment were generic and the most used were the EORTC QLQ-C30, the EQ-5D questionnaire and the modified Brief Pain Inventory-short form. QoL has not been assessed or published in many trials despite an improvement over time. QoL evaluation in RCTs should be considered even more carefully in patients with rare tumors, where the low number of patients who can be enrolled often makes it difficult to draw statistically significant conclusions on the effectiveness of treatments.
Paternostro et al. (Fri,) studied this question.