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Updated: Sep 1, 2025
Establishment of a Primary Culture of Patient-derived Soft Tissue Sarcoma
Published on: April 11, 2018
Activity of Cabazitaxel in Metastatic or Inoperable Locally Advanced Dedifferentiated Liposarcoma: A Phase 2 Study of
Roberta Sanfilippo1, Richard L Hayward2, Jammbe Musoro3
1Medical Oncology Unit 2, Medical Oncology Dpt, Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milan, Italy.
Importance:
Treatment options for patients with unresectable and/or metastatic dedifferentiated liposarcoma (DDLPS) are limited. New drugs are required.
Objective:
To assess whether cabazitaxel demonstrated sufficient antitumor activity in patients with metastatic or inoperable locally advanced DDLPS to justify further investigation in a phase 3 setting.
Design, Setting, And Participants:
This international multicenter, open-label single-arm phase 2 trial was conducted at 10 institutions in 4 European countries from March 2015 to March 2019. Eligible patients had to have metastatic or locally advanced histologically proven DDLPS with evidence of disease progression within the past 6 months and had to have received no more than 1 previous line of chemotherapy.
Interventions:
After mandatory central review of tumor blocks, if the DDLPS diagnosis was confirmed, patients started treatment within 72 hours after registration. Cabazitaxel was administered at a dose of 25 mg/m2 IV infusion over 1 hour every 21 days until intolerance, progression, or withdrawal of consent.
Main Outcomes And Measures:
The primary end point was progression-free survival (PFS) rate at 12 weeks per RECIST 1.1. Based on a Simon 2-stage design, at least 4 of 17 (stage 1) and 11 of 37 (stage 2) eligible and evaluable patients who were progression free at 12 weeks were needed. The final analysis report was completed on November 17, 2021.
Results:
Forty patients were registered, with 2 patients being ineligible. The number of cycles ranged from 1 to 30, with a median of 5; 26 patients (65%) received at least 4 cycles of cabazitaxel. Progression-free survival at 12 weeks was 55%, achieving the primary study end point. At a median follow-up of 21.6 months, median PFS was 6 months and median OS 21 months. Response rate (RR) was 8% with 1 clinical response (CR) and 2 partial responses (PR). Twenty-three (60.5%) patients had a stable disease (SD). Disease control (PR+SD) was achieved in 26 patients (68%).
Conclusions And Relevance:
This nonrandomized phase 2 clinical trial met its primary end point, with 21 of 38 patients (55%) being progression free at 12 weeks. These results suggest important activity of cabazitaxel in patients with metastatic or inoperable locally advanced DDLPS. The drug is worth being further studied in these tumors in a phase 3 setting.
Insights
Cabazitaxel showed significant antitumor activity in patients with advanced dedifferentiated liposarcoma (DDLPS), with 55% progression-free survival at 12 weeks. This suggests cabazitaxel warrants further phase 3 investigation for DDLPS treatment.
Area of Science:
- Oncology
- Medical Research
- Clinical Trials
Background:
- Treatment options for unresectable or metastatic dedifferentiated liposarcoma (DDLPS) are limited.
- There is a critical need for novel therapeutic agents for DDLPS.
Purpose of the Study:
- To evaluate the antitumor activity of cabazitaxel in patients with metastatic or inoperable locally advanced DDLPS.
- To determine if cabazitaxel warrants further investigation in a phase 3 clinical trial setting.
Main Methods:
- An international, multicenter, open-label, single-arm phase 2 trial.
- Eligible patients with DDLPS received cabazitaxel (25 mg/m2 IV every 21 days) until progression or intolerance.
- Primary endpoint was 12-week progression-free survival (PFS) rate.
Main Results:
- The 12-week PFS rate was 55% (21/38 patients), meeting the primary endpoint.
- Median PFS was 6 months, and median overall survival (OS) was 21 months.
- Disease control (partial response + stable disease) was achieved in 68% of patients.
Conclusions:
- Cabazitaxel demonstrated significant antitumor activity in patients with advanced DDLPS.
- The study met its primary endpoint, supporting cabazitaxel's potential efficacy.
- Further investigation in a phase 3 trial is recommended for cabazitaxel in DDLPS.
