A total of 20 patients were recruited between three sites in the UK (Oxford, Glasgow, Cheltenham) and followed up for a median of 27 months. One patient was replaced because of non-compliance with the study protocol. In total, 19 patients (demographics shown in ) completed at least one cycle of treatment and were assessable for response and safety evaluation. Two patients remain on study, 4 years after enrolment.
Capecitabine was generally well tolerated (). Single episodes of grade IV toxicity were recorded for infection, raised liver function tests (LFT's), thrombocytopenia, leucopenia and neutropenia. The most common grade III toxicities were diarrhoea (26% patients), fatigue (21% patients) and raised LFTs (21% patients). Most patients, as expected, experienced a rash and/or hand-foot syndrome (HFS), but all were of grade I-II toxicity. The Stuart–Maxwell test showed no statistically significant increase in the grade of diarrhoea between baseline and subsequent cycles of capecitabine therapy.
A total of 13 (68%) patients achieved a radiological SD of whom two (11%) also had biochemical PR by Chromogranin A. Three (16%) patients had radiological PD and three (16%) were not evaluable radiologically. There were no radiological PR or CR, thus the overall disease control rate was 68%. Four (21%) patients maintained SD for >12 months. Of these, two remain well in the follow-up phase of study and two discontinued treatment because of toxicity. Overall seven patients came off study due to PD (37%), five due to toxicity (26%), three by patient choice and two by investigator choice. The investigators' reason in both cases was to defer treatment until symptomatic disease progression.
The median PFS was 9.9 months (lower quartile: 4.4, upper quartile: 36.7) and median OS was 36.5 months (lower quartile: 19.9, upper quartile not yet reached).