Patients randomised into Veregen® arm showed a significantly
better clinical response when compared to placebo
Clinical response was measured according to a protocol defined
evaluation criteria based on RECIST and responses were categorised into
stable disease, partial response, complete response and progressive
disease. Clinical responses were measured at 4, 8, 16, 32 and 52 weeks
after the start of treatment: 5 patients showed partial response in
Veregen® group when compared to only one patient in the placebo group at
four weeks; 7 patients showed clinical response in Veregen® group and 3
in the placebo group at eight weeks. At the end of treatment, 16 weeks,
10 patients in Veregen® group responded to treatment: 5 showed complete
response; 5 a partial response; 2 patients had stable disease; 1 patient
was lost to follow up but responded partially at four weeks. In the
placebo group, five patients showed clinical response: 2 complete
response, 3 partial response, 4 with stable disease and 1 had
progressive disease. Two patients were lost to follow up in the placebo
group. At 32 weeks, one patient had progressed in Veregen® treatment
arm, who previously had a partial response. This patient continued to
have disease progression at 52 weeks follow up. One other patient in the
Veregen® treatment arm, who already had a complete response at 16 weeks,
recurred at 52 weeks. In the placebo treatment arm, two patients
presented with progressive disease, one patient with previously complete
response at 16 weeks relapsed, and two further patient with previous
complete response and partial response remained disease-free, the
remaining patients had stable disease. In the Veregen® group, all
patients showed a significant clinical response as opposed to only five
patients showing clinical response following treatment (p=0.003) (Figure
1). Patients in the Veregen® group show a decreased time to clinical
resolution as compared to placebo, both when clinical resolution is
taken to be complete response and complete/partial response (Figure 1).
Biopsies were obtained at 16 and 32 weeks after treatment to evaluate
the histological resolution of uVIN and there was no observed difference
in best histological response in these two groups. Three patients in
each arm showed complete histology resolution; 7 and 6 patients in
placebo and active arm, respectively, had persistent disease
histologically; 3 and 4 patients in placebo and active arm,
respectively, refused post-treatment biopsy. One patient from the
placebo group developed early-stage squamous cell carcinoma at 16-week
biopsy, she was withdrawn from the study and managed according to our
local guideline for vulvar cancer.