TY - JOUR
T1 - New benchmarks to design clinical trials with advanced or metastatic liposarcoma or synovial sarcoma patients
T2 - An EORTC – Soft Tissue and Bone Sarcoma Group (STBSG) meta-analysis based on a literature review for soft-tissue sarcomas
AU - Kantidakis, Georgios
AU - Litière, Saskia
AU - Neven, Anouk
AU - Vinches, Marie
AU - Judson, Ian
AU - Blay, Jean Yves
AU - Wardelmann, Eva
AU - Stacchiotti, Silvia
AU - D'Ambrosio, Lorenzo
AU - Marréaud, Sandrine
AU - van der Graaf, Winette T.A.
AU - Kasper, Bernd
AU - Fiocco, Marta
AU - Gelderblom, Hans
N1 - Copyright © 2022 The Author(s). Published by Elsevier Ltd.. All rights reserved.
PY - 2022/10
Y1 - 2022/10
N2 - Background: Recently, we performed a meta-analysis based on a literature review for STS trials (published 2003–2018, ≥10 adult patients) to update long-standing reference values for leiomyosarcomas. This work is extended for liposarcomas (LPS) and synovial sarcomas (SS). Materials and methods: Study endpoints were progression-free survival rates (PFSRs) at 3 and 6 months. Trial-specific estimates were pooled per treatment line (first-line or pre-treated) with random effects meta-analyses. The choice of the therapeutic benefit to target in future trials was guided by the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Results: Information was acquired for 1030 LPS patients (25 trials; 7 first-line, 17 pre-treated, 1 both) and 348 SS patients (13 trials; 3 first-line, 10 pre-treated). For LPS, the overall pooled first-line PFSRs were 69% (95%-CI 60–77%) and 56% (95%-CI 45–67%) at 3 and 6 months, respectively. These rates were 49% (95%-CI 40–57%)/28% (95%-CI 22–34%) for >1 lines. For SS, first-line PFSRs were 74% (95%-CI 58–86%)/56% (95%-CI 31–78%) at 3 and 6 months, and pre-treated rates were 45% (95%-CI 34–57%)/25% (95%-CI 16–36%). Following ESMO-MCBS guidelines, the minimum values to target are 79% and 69% for first-line LPS (82% and 69% for SS) at 3 and 6 months. For pre-treated LPS, recommended PFSRs at 3 and 6 months suggesting drug activity are 63% and 44% (60% and 41% for SS). Conclusions: New benchmarks are proposed for advanced/metastatic LPS or SS to design future histology-specific phase II trials. More data are needed to provide definitive thresholds for the different LPS subtypes.
AB - Background: Recently, we performed a meta-analysis based on a literature review for STS trials (published 2003–2018, ≥10 adult patients) to update long-standing reference values for leiomyosarcomas. This work is extended for liposarcomas (LPS) and synovial sarcomas (SS). Materials and methods: Study endpoints were progression-free survival rates (PFSRs) at 3 and 6 months. Trial-specific estimates were pooled per treatment line (first-line or pre-treated) with random effects meta-analyses. The choice of the therapeutic benefit to target in future trials was guided by the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Results: Information was acquired for 1030 LPS patients (25 trials; 7 first-line, 17 pre-treated, 1 both) and 348 SS patients (13 trials; 3 first-line, 10 pre-treated). For LPS, the overall pooled first-line PFSRs were 69% (95%-CI 60–77%) and 56% (95%-CI 45–67%) at 3 and 6 months, respectively. These rates were 49% (95%-CI 40–57%)/28% (95%-CI 22–34%) for >1 lines. For SS, first-line PFSRs were 74% (95%-CI 58–86%)/56% (95%-CI 31–78%) at 3 and 6 months, and pre-treated rates were 45% (95%-CI 34–57%)/25% (95%-CI 16–36%). Following ESMO-MCBS guidelines, the minimum values to target are 79% and 69% for first-line LPS (82% and 69% for SS) at 3 and 6 months. For pre-treated LPS, recommended PFSRs at 3 and 6 months suggesting drug activity are 63% and 44% (60% and 41% for SS). Conclusions: New benchmarks are proposed for advanced/metastatic LPS or SS to design future histology-specific phase II trials. More data are needed to provide definitive thresholds for the different LPS subtypes.
KW - Advanced or metastatic population
KW - Efficacy
KW - Liposarcoma
KW - Meta-analysis
KW - Study design
KW - Synovial sarcoma
KW - Humans
KW - Sarcoma/drug therapy
KW - Benchmarking
KW - Bone Neoplasms
KW - Lipopolysaccharides/therapeutic use
KW - Osteosarcoma
KW - Soft Tissue Neoplasms/drug therapy
KW - Adult
KW - Sarcoma, Synovial/therapy
KW - Advanced or metastatic population
KW - Efficacy
KW - Liposarcoma
KW - Meta-analysis
KW - Study design
KW - Synovial sarcoma
UR - http://www.scopus.com/inward/record.url?scp=85138100622&partnerID=8YFLogxK
UR - https://www.mendeley.com/catalogue/53851687-cc7a-31e5-879a-896bd21ec946/
U2 - 10.1016/j.ejca.2022.07.010
DO - 10.1016/j.ejca.2022.07.010
M3 - Review article
C2 - 36116829
AN - SCOPUS:85138100622
SN - 0959-8049
VL - 174
SP - 261
EP - 276
JO - European Journal of Cancer
JF - European Journal of Cancer
ER -