Background Bae applied and evaluated between March and July 2016 by an expanded access to the drug, and the patients were followed until 15 October. Clinical data were obtained from the electronic history CernerMillenium and the variables were: age, gender, progression date, death date and adverse effects.
Results Treatment was requested for 32 patients; 30 were authorised to receive TAS-102. Of them, only 24 patients started treatment. 5 patients did not start treatment due to worsening of disease during the process of authorisation, and another 1 because of supply problems.
There were 15 men and 9 women, and median age was 66.3 years. Most patients progressed, and only 6 continued receiving TAS-102. Median PFS was 2.25 months (range 0.47–6.47). During treatment, 6 patients died. Median OS was 3.27 months (range 0.70–6.47). The toxicity profile showed haematologic effects such as: neutropenia (70.3%), anaemia (50%), leukopenia (37.5%) and thrombopenia (25%). Other adverse effects were less frequent: diarrhoea (8.3%), asthenia (8.3%), vomiting (8.3%) and neuropathy (4.2%).
Conclusion Although treatments were approved and received for all patients, 20% never started TAS-102. OS was lower than in the pivotal study because the study was not long enough to achieve better results. On the other hand, PFS was higher than in the pivotal study which was 2.0 months. Adverse effects confirmed haematologic toxicity.
References and/or acknowledgements N Engl J Med2015;372:1909–19. doi:10.1056/NEJMoa1414325.
No conflict of interest
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