Related Experiment Video
Updated: Jun 12, 2026
Computer-Aided Three-Dimensional Visualization in the Treatment of Locally Advanced Thyroid Cancer
Published on: June 9, 2023
Systemic Treatment of Advanced Chordoma With Molecular Targeted Therapies: A Large Multicentre European Retrospective
Astrid Lipplaa1, Nadia Hindi2, Pawel Teterycz3
1Department of Medical Oncology, Leiden University Medical Center, Leiden, the Netherlands.
Background:
This retrospective series reports outcomes and prognostic factors for advanced chordoma (AC) treatment with molecular targeted therapies (MTTs) in different treatment lines.
Methods:
This is a retrospective series of 57 patients with AC treated between 2004 and 2023 at one of seven participating sarcoma centres. Demographics, previous treatment, treatment details and outcomes were recorded.
Results:
57 patients were treated with 7 different MTTs. Treatment was received in first (n = 57), second (n = 16), third (n = 5) or fourth (n = 1) line. The most frequently administered agent in first line was imatinib (84.2%) and in second line imatinib + sirolimus (35.7%). Overall median progression free survival (PFS) and overall survival (OS) in first-line treatment was 6.5 (95% CI 4.0-9.0) and 29.5 months (95% CI 24.0-40.4) and in second-line 10.0 (95% CI 4.0-22.0) and 37.2 months (95% CI 9.4-45.9). Partial response according to RECIST 1.1 was seen in 5/79 treatments (6.3%). Dose reductions and interruptions were reported in 19.0% and 27.8% of treatments.
Conclusions:
PFS and response rates with these MTTs were in line with previous phase II trials and retrospective series. Although the efficacy does not meet the European Society of Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale (MCBS) criteria for single arm studies in orphan diseases, MTTs are frequently used off-label due to the high unmet need and lack of other systemic treatment options. The toxicity profile and limited efficacy rate should be taken into account when counselling patients. Further research is needed to explore other systemic treatment options including (combinations with) immunotherapy.
Insights
Molecular targeted therapies (MTTs) show limited efficacy and toxicity in advanced chordoma (AC) treatment. Further research is needed for better systemic options, including immunotherapy, due to high unmet need.
Area of Science:
- Oncology
- Medical Science
Background:
- Advanced chordoma (AC) presents a significant treatment challenge.
- Molecular targeted therapies (MTTs) are explored for AC, but outcomes data are limited.
Purpose of the Study:
- To report outcomes and prognostic factors for advanced chordoma (AC) treated with molecular targeted therapies (MTTs).
- To evaluate the efficacy and toxicity of MTTs in different treatment lines for AC.
Main Methods:
- Retrospective series of 57 patients with AC treated between 2004 and 2023.
- Data collected included demographics, previous treatments, treatment details, and outcomes.
Main Results:
- 57 patients received 7 different MTTs across first to fourth-line treatment.
- Median progression-free survival (PFS) in first-line was 6.5 months, and overall survival (OS) was 29.5 months.
- Partial response (RECIST 1.1) was observed in 6.3% of treatments; dose modifications were frequent.
Conclusions:
- MTT efficacy and response rates align with previous studies but do not meet ESMO MCBS criteria.
- MTTs are used off-label due to high unmet need and lack of alternatives, despite toxicity and limited efficacy.
- Further research into novel systemic treatments, including immunotherapy combinations, is crucial for advanced chordoma.
Related Concept Videos
02:56Treatment Resistant Cancers
02:56Treatment Resistent Cancers
02:57Targeted Cancer Therapies
There are several types of targeted therapies against specific...
02:57Targeted Cancer Therapies
There are several types of targeted therapies against specific...