Molecular tumor board in advanced sarcoma: treatment implementation and clinical outcomes in a real-world cohort.

PURPOSE: Advanced sarcomas remain therapeutically challenging. We evaluated treatment implementation and clinical outcomes after molecular tumor board recommendations.

METHODS

We retrospectively analyzed 125 sarcoma patients discussed in our multidisciplinary Molecular and Familial Tumor Board between 2017 and 2023. Clinical benefit was assessed by treatment implementation and by the ratio of progression-free survival on molecularly guided therapy versus the preceding treatment line (PFS2/PFS1). Overall survival (OS) was measured from first board discussion.

RESULTS

A total of 252 pathogenic or likely pathogenic variant records across 133 genes were identified. Molecular profiling enabled therapy recommendations in 71 of 125 patients (56.8%), with treatment implementation in 41 (32.8%). PFS2/PFS1 evaluation was feasible in 29 patients, and nine (31.0%) had a documented ratio > 1.3. Median PFS2 was 102 days (95% CI 63-233). Two of seven patients (28.6%) receiving poly(ADP-ribose) polymerase (PARP) inhibitor-containing regimens had ratios > 1.3. Median OS in 124 patients was 14.6 months (95% CI 11.7-18.5; global log-rank p = 0.220).

CONCLUSION

Molecular tumor board-guided therapy identified treatment options for selected sarcoma patients. Prolonged disease control in individual PARP-treated cases provides a positive signal that warrants prospective evaluation.