1938P - Assessment of clinical benefit of cancer drugs recommended in National Comprehensive Cancer Network (NCCN) guidelines for advanced soft tissue sarcomas (STS) and bone sarcomas (BS)

Background

Magnitude of clinical benefit from drugs used in tumours must be evaluated using validated and reproducible tools. European Society for Medical Oncology-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS) and American Society for Medical Oncology (ASCO) Value Framework (ASCO-VF) are the most important. Here, we evaluate characteristics and outcomes of clinical trials supporting drugs used in STS and BS, and their association with ESMO-MCBS and ASCO-VF.

Methods

We reviewed publications between January 1988 and December 2022 that support the recommendations of NCCN guidelines version 1.2023 for STS and 3.2023 for BS (gastrointestinal stromal tumours were excluded). We collected data regarding trial characteristics, efficacy, toxicity and quality of life (QoL). ESMO-MCBS and ASCO-VF were applied. Substantial clinical benefit was defined as a grade 4 or 5 (in a scale from 1 to 5) for ESMO-MCBS and a ≥45 for ASCO-VF.

Results

We identified 119 studies (71 for STS and 48 for BS) that support the use of 49 drugs or combinations. ESMO-MCBS could be applied in 58 trials (48.7%). Of them, 37 (64%) were single arm and 21 (36%) randomized clinical trials (RCT; 13 phase III and 8 phase II). ASCO-VF could only be applied in 10 of the previously described (6 phase III and 4 phase II RCT). QoL was reported in 17.2% (10/58), all published beyond 2011. Among these, only 1 (10%) showed improvement in QoL. Only 33.3% of RCT (7/21) demonstrated improvement in overall survival. Ten (17.2%) trials, all of them RCT, met the ESMO-MCBS substantial benefit threshold (7 involving STS and 3 BS). Only 4 of these met the ASCO-VF criteria (all for STS). Spearman’s correlation coefficient between scales was 0.65.

Conclusions

Less than half of the studies supporting the NCCN recommendations for the prescription of drugs in advanced sarcomas can be evaluated using validated scales to assess the magnitude of their clinical benefit. Substantial clinical benefit was only achieved in a minority of those. QoL analysis should be incorporated in further studies to better evaluate their actual clinical benefit. Correlation among ESMO-MCBS and ASCO-VF was noteworthy.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.