Molecular Targeted Therapy in the Treatment of Chordoma: A Systematic Review

Frontiers in Oncology, 2019 · DOI: 10.3389/fonc.2019.00030 · Published: February 1, 2019

Simple Explanation

Chordoma is a rare bone cancer that often recurs and spreads, making treatment difficult. Molecular targeted therapy (MTT) is a treatment option for advanced cases, but its effectiveness and safety haven't been systematically studied. This study reviewed clinical trials, case series, and reports on chordoma MTT to assess clinical outcomes like survival rates, disease progression, and side effects. The review found that imatinib and erlotinib were the most commonly used molecular targeted inhibitors (MTIs) for chordoma, showing benefits in patients with PDGFR-positive and/or EGFR-positive tumors with acceptable side effects. Monotherapy is typically the first line of treatment.

Study Duration

Not specified

Participants

Thirty-three eligible studies were selected for the systematic review

Evidence Level

Systematic Review

Key Findings

1
Imatinib and erlotinib were the most frequently used molecular targeted inhibitors (MTIs) for chordoma.
2
Clinical benefits were achieved with acceptable AEs for PDGFR-positive and/or EGFR-positive chordoma.
3
Monotherapy is preferred as the ﬁrst-line of treatment, and combined drug therapy as the second-line treatment.

Research Summary

This systematic review analyzed 33 studies on molecular targeted therapy (MTT) for chordoma, a rare bone malignancy affecting the spine and skull base. The review identified imatinib and erlotinib as the most frequently used molecular targeted inhibitors (MTIs), showing clinical benefits for PDGFR-positive and/or EGFR-positive chordoma with acceptable adverse events (AEs). The study recommends gene mutation screening and immunohistochemistry (IHC) to guide MTI selection, with monotherapy of TKIs as the first-line management and combination therapy for drug-resistant chordoma. Brachyury vaccine shows promise but needs more clinical trials.

Practical Implications

Personalized Treatment

Gene mutation screening and IHC should be used to guide the selection of MTIs for patients with advanced or relapsed chordoma.

Treatment Strategy

Monotherapy of TKIs is recommended as the first-line treatment, while combination therapy may be considered for drug-resistant chordoma.

Future Research

More clinical trials are needed to evaluate the safety and efficacy of the brachyury vaccine.

Study Limitations

1
Inclusion of case reports might overemphasize the ﬁnal results due to lack of strong results.
2
Only included English language publications which can also increase the selection bias.
3
The baseline conditions of the patients and the evaluation criteria were not consistent across studies which is another factor contributing to selection bias.

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