Compassionate use programs (CUPs) provide a treatment option for appropriate patients with unmet medical needs. We describe the trifluridine/tipiracil (TAS-102) CUP set up prior to marketing authorization for management of pretreated metastatic colorectal cancer (mCRC) in countries outside the USA and Japan.
Registration gave mCRC patients (pts) early access to 2 cycles of treatment, renewable as necessary. Pts' characteristics were collected at registration.
A total of 879 pts were registered in 21 countries by the 1st April 2016 cutoff (Argentina, Brazil, Australia, Austria, Czech Republic, Denmark, France, Germany, Hungary, Ireland, Israel, Italy, Latvia, Lithuania, Spain, The Netherlands, Portugal, Romania, South Africa, Switzerland, and UK). Of these, 725 pts (83%) were shipped enough treatment for the first 2 cycles. Mean age was 64 years and 60% were male. Oral trifluridine/tipiracil was initiated at 35 mg/m2 bid. Most pts had received 2, 3, or ≥4 lines of prior treatment for metastatic disease (28%, 32%, and 32%, respectively); 4% had received 1 line of treatment and 4% unknown. The main reasons for not initiating treatment included cancellation of request due to worsening condition and progressive disease. 37 pts (4%) permanently discontinued treatment before the end of cycle 1 or 2 due to disease progression (19 pts), death (8 pts), or other reasons (10 pts). A total of 184 pts (25% of pts in cycles 1or 2) went on to receive trifluridine/tipiracil for cycles 3 or 4. Of those, 28 pts had a dose reduction due to neutropenia (17 pts), diarrhea (3 pts), or other adverse events (8 pts; vomiting, dyspnea, creatinine increase, bowel obstruction, pleural effusion). 24 pts (3%) had treatment permanently discontinued before end of cycles 3or 4 due to progressive disease. 20 pts went on to receive treatment in cycles 5 or 6, 16 pts with no dose reduction.
Real-world treatment and safety data are consistent with those reported in phase 3 trials of trifluridine/tipiracil in pretreated mCRC.
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All authors have declared no conflicts of interest.