%0 Randomized Controlled Trial
%T Neoadjuvant Chemotherapy in High-Grade Myxoid Liposarcoma: Results of the Expanded Cohort of a Randomized Trial From Italian (ISG), Spanish (GEIS), French (FSG), and Polish Sarcoma Groups (PSG).
%A Gronchi A
%A Palmerini E
%A Quagliuolo V
%A Martin Broto J
%A Lopez Pousa A
%A Grignani G
%A Brunello A
%A Blay JY
%A Tendero O
%A Diaz Beveridge R
%A Ferraresi V
%A Lugowska I
%A Pizzamiglio S
%A Verderio P
%A Fontana V
%A Donati DM
%A Palassini E
%A Sanfilippo R
%A Bianchi G
%A Bertuzzi A
%A Morosi C
%A Pasquali S
%A Stacchiotti S
%A Bagué S
%A Coindre JM
%A Miceli R
%A Dei Tos AP
%A Casali PG
%J J Clin Oncol
%V 42
%N 8
%D 2024 Mar 10
%M 38232337
%F 50.717
%R 10.1200/JCO.23.00908
%X <B>OBJECTIVE: </B>A randomized trial was conducted to compare neoadjuvant standard (S) anthracycline + ifosfamide (AI) regimen with histology-tailored (HT) regimen in selected localized high-risk soft tissue sarcoma (STS). The results of the trial demonstrated the superiority of S in all STS histologies except for high-grade myxoid liposarcoma (HG-MLPS) where S and HT appeared to be equivalent. To further evaluate the noninferiority of HT compared with S, the HG-MLPS cohort was expanded.<BR><B>METHODS: </B>Patients had localized high-grade (cellular component >5%; size ≥5 cm; deeply seated) MLPS of extremities or trunk wall. The primary end point was disease-free survival (DFS). The secondary end point was overall survival (OS). The trial used a noninferiority Bayesian design, wherein HT would be considered not inferior to S if the posterior probability of the true hazard ratio (HR) being >1.25 was <5%.<BR><B>RESULTS: </B>From May 2011 to June 2020, 101 patients with HG-MLPS were randomly assigned, 45 to the HT arm and 56 to the S arm. The median follow-up was 66 months (IQR, 37-89). Median size was 107 mm (IQR, 84-143), 106 mm (IQR, 75-135) in the HT arm and 108 mm (IQR, 86-150) in the S arm. At 60 months, the DFS and OS probabilities were 0.86 and 0.73 (HR, 0.60 [95% CI, 0.24 to 1.46]; log-rank P = .26 for DFS) and 0.88 and 0.90 (HR, 1.20 [95% CI, 0.37 to 3.93]; log-rank P = .77 for OS) in the HT and S arms, respectively. The posterior probability of HR being >1.25 for DFS met the Bayesian monitoring cutoff of <5% (4.93%). This result confirmed the noninferiority of trabectedin to AI suggested in the original study cohort.<BR><B>CONCLUSIONS: </B>Trabectedin may be an alternative to standard AI in HG-MLPS of the extremities or trunk when neoadjuvant treatment is a consideration.