Long-term survival following a phase I/II trial with VETOPEC for solid tumors in childhood

The objective of this study was to evaluate long-term survival after treatment during a phase I/II trial with a specific regimen of vincristine, etoposide, and escalating cyclophosphamide (VETOPEC). Fifty-six children with poor-prognosis solid tumors were enrolled on study between May 1991 and May 1994. All had tumors that had relapsed on, or were refractory to, conventional treatment, or for whom existing treatment options were considered ineffective. The records of all surviving patients were reviewed to ascertain their disease and health status. Of the 56 patients, 10 patients (18%) remain alive with no further disease progression at a median follow-up of 11 years (range 7-13 years). Eight patients (14%) remain completely free of disease. None of the patients show long-term side effects directly attributable to the VETOPEC regimen, apart from one patient with ovarian failure. The VETOPEC regimen can offer not only good tumor responses but also the chance of cure for a surprisingly large number of children with very-poor-prognosis solid tumors. This regimen warrants continuing development and consideration for use in future trials.