Dr Botticelli talks to ecancer at ESMO 2024 about the ROME Trial, a phase 2 study comparing targeted therapy to the standard of care in patients with solid tumours carrying actionable genetic alterations, identified by comprehensive genome profiling and reviewed by a Molecular Tumor Board (MTB).

Tissue and blood samples from patients who had undergone up to two prior treatments were analyzed with comprehensive genome profiling tools, and those with targetable alterations were randomized (1:1) to either TT or standard of care. The primary goal was overall response rate (ORR), with secondary outcomes including progression-free survival (PFS), overall survival (OS), and safety.

Out of 1,794 patients screened, 400 were randomized. Common cancers included colorectal, breast, and gastric, with the most frequent mutations involving hTMB, PIK3CA/AKT/PTEN, and ERBB2. The targeted therapy group had an ORR of 17% compared to 9.5% in the SoC group, and a median PFS of 3.7 months vs. 2.8 months in SoC (p < 0.001). The group also had a higher 12-month PFS rate (22% vs. 7%).

Patients with hTMB/MSS tumours who received immunotherapy had notably improved PFS. The incidence of severe side effects (≥Grade 3) was slightly lower in the targeted therapy group (35% vs. 40% for standard of care).

Dr Botticelli states that the a mutation-based approach using comprehensive genome profiling and MTB review significantly improved ORR and PFS in patients with metastatic solid tumours, especially with immunotherapy.