Objectives: We sought to evaluate the effectiveness of HspE7, a recombinant fusion protein of Hsp65 from Mycobacterium bovis BCG and E7 protein from human papillomavirus 16, to improve the clinical course of pediatric patients with recurrent respiratory papillomatosis.
Methods: An open-label, single-arm intervention study was conducted in 8 university-affiliated medical centers. Twenty-seven male and female patients with recurrent respiratory papillomatosis, ages 2 to 18 years, were enrolled and followed up to 60 weeks. Before enrollment, these patients required surgery on average every 55 days. After a baseline debulking surgery, the patients received HspE7 500 microg subcutaneously monthly, for 3 doses over 60 days. The primary end point was the length of the interval from the last surgery during the treatment period until the first debulking surgery in the posttreatment period, compared with the median intersurgical interval (ISI) of the 4 surgeries before the treatment.
Results: The mean of the first posttreatment ISI increased 93% (from 55 days to 106 days; p < .02). The median ISI for all surgeries after treatment was similarly prolonged (mean, 107 days; p < .02), indicating a sustained treatment effect, and was associated with a significant decrease in the number of required surgeries (p < .003). Unexpectedly, the treatment effect was most striking in the 13 female patients, who had statistically significant increases in both the first posttreatment ISI (142%; p < .03) and the median ISI (147%; p < .03). The most common adverse events were mild-to-moderate injection site reactions.
Conclusions: Treatment with HspE7 appears to significantly improve the clinical course in pediatric patients with RRP insofar as it reduces the frequency of required surgeries. These results warrant a confirmatory phase III trial.