Abstract 1552P
Background
The European Society for Medical Oncology-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS) is a validated and reproducible tool to assess the magnitude of clinical benefit from drugs for solid tumors. Here, we evaluate characteristics and outcomes of clinical trials supporting drugs used in advanced Soft Tissue Sarcomas (STS) and GIST and their association with ESMO-MCBS.
Methods
We reviewed publications between January 1990 and December 2020 that support the recommendations of NCCN (National Comprehensive Cancer Network) guidelines version 1.2021 for treatment of advanced STS and GIST. We collected data on trial characteristics, efficacy, toxicity and quality of life (QoL). For each trial, ESMO-MCBS grades were applied. Substantial clinical benefit was defined as a grade 4 or 5 (in a scale from 1 to 5).
Results
Among the 23 trials for which the ESMO-MCBS could be applied (23/57 -40.4%-), 13 (56.5 %) were randomized controlled trials (RCT; 8 phase III and 5 phase II trials), and 10 (43.5%) were single arm trials. Eighteen (78.2%) involved STS and 5 (21.8%) GIST. Six trials (26.1% of all trials and 46.1% of RCT) demonstrated improvement in overall survival. QoL was reported in 7 (30.4%) trials. Among these, only 3 (42.9%) showed improvement in QoL. Only 9 (39.1 %) trials met the ESMO-MCBS substantial benefit threshold (5/18 involving STS and 4/5 involving GIST). Of these 9 trials, 8 (88.8%) were RCT (4 phase III and 4 phase II). All 3 trials that demonstrated improvement in QoL showed substantial benefit according to the scale.
Conclusions
Only 39.1% of trials supporting the use of drugs in patients with advanced STS and GIST meet the threshold for substantial benefit using ESMO-MCBS. These results suggest that randomized trials with QoL assessment are more likely to achieve the ESMO-MCBS substantial benefit threshold. Only a quarter of the trials showed improvement in OS.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding
Disclosure
A. Sebio Garcia: Other, Institutional, Other, Travel grants, Research grant and Teaching Honoraria: Pharmamar; Other, Institutional, Other, Travel grants: Amgen; Other, Institutional, Other, Travel grants: Sanofi. All other authors have declared no conflicts of interest.