Background Trifluridine/tiparacile is the second oral treatment approved for patients with mCRC who have received fluoropyrimidine, oxaliplin- and irinotecan-based chemotherapy, an anti-VEGF biologic therapy and, if RAS wild-type, an anti-EGFR.

Purpose To evaluate the efficacy and safety of patients treated with trifluridine plus tipiracil in a tertiary hospital in real-world data.

Material and methods Retrospective descriptive observational study was conducted. We included all patients from when the expanded access programme was introduced in our hospital (April 2016) to September 2017. Measured variables included: age, sex, KRAS status, ECOG performance status, number of cycles (minimum of two cycles), number of prior lines of treatment for CRCM, progression-free survival (PFS), adverse effects and dose reduction.

Response evaluation was performed according to RECIST version 1.1, and toxicity evaluation as defined by the NCI-CTCAE, version 4.0.

Results Thirty patients were included: 60% males and a median age of 64.2 years (41–77). 53.3% of cases were KRAS wild-type tumours and ECOG performance status was 0 in 15 patients. They had received a median of three lines of treatment prior to a median of 3.5 cycles of trifluridine/tipiracil.

Regarding effectiveness, the median PFS in 19 patients was 4.2 months, there were three patients that still continue treatment with a PFS of 3 months, four patients were not evaluated: three due to clinical progression and one was a case of exitus. Finally, four patients were awaiting PET scan evaluation.

Treatment-related adverse effects of any grade were reported in 83.3% of patients. The most common ones were fatigue (56.6%), neutropaenia (40%; grade IV: 13.3%), nausea (39.9%), diarrhoea (23.2%), neurotoxicity (10%) and gastrointestinal pain (10%). A total of 10 patients required dose reduction because of these events.

Conclusion Effectiveness evaluation revealed a much longer PFS during routine clinical practice in comparison to the result reported in the pivotal trials (4.2 vs 2 months in the RECOURSE study). Differences in study sample, number of prior lines of treatment and/or re-treatment rate may explain this fact. The safety profile, in contrast, was similar to that described in the data sheet. More experience in the use of trifluridine/tiparacile is needed to confirm these great data.

No conflict of interest