Abstracts

HspE7 Treatment of Pediatric Recurrent Respiratory Papillomatosis (RRP): Interim Results utilizing a Laryngeal Staging and Severity Scale (LSSS)

Oral presentation at SENTAC (Society for Ear, Nose, and Throat Advances in Children) on October 31 2003

Derkay CS (1), Arnold J (2), Bower C (3), McClay J (4), Smith R (5), Van Burik J (6), Wiatrak B (7), Wohl D (8), Berger B (9), Neefe J (9)

1) Eastern Virginia Med Sch, VA
2) Rainbow Babies & Children’s Hosp, Cleveland, OH
3) Little Rock, AK
4) Univ of Texas, Dallas, TX
5) Univ of Iowa Hosp & Clin, Iowa City, IA
6) Fairview Univ Med Ctr, Univ of Minnesota, Minneapolis, MN
7) Children’s Hosp of Alabama, Birmingham, AL
8) Univ of Florida, Jacksonville, FL
9) Stressgen Biotechnologies, Collegeville, PA

Background:

HspE7, a recombinant fusion protein of heat shock protein (Hsp65) from M. bovis -BCG and E7 protein from HPV-16, has been reported to increase the interval between required debulking surgeries in an open-label, single-arm intervention study conducted in 8 university-affiliated medical centers.

Patients:

27 male and female patients with RRP, ages 2-18 were enrolled and followed up to 60 weeks. Severity of disease ranged from mild to severe, including 13 patients (48%) with severe disease (pulmonary disease and/or baseline anatomical LSSS >20).

Methods:

After a baseline debulking surgery, patients received HspE7 500 mcg subcutaneously monthly, 3 doses over 60 days. The primary endpoint was the length of the interval to the next debulking surgery after treatment compared with the median inter-surgical interval of four surgeries prior to treatment. Because the decision for surgery is influenced by a number of factors, a protocol-defined standardized LSSS for RRP (Derkay/Coltrera Laryngeal Staging and Severity Scale) was utilized to provide an objective measure of the indication for surgery.

Results:

The mean (±S.D.) pre-treatment inter-surgical interval for the entire cohort was 55.3 ± 24.0 days, compared with 95.6 ± 81.3 days after treatment (paired t-test p=0.0098), a mean increase of 78.6% (t-test p=0.015). The mean staging/severity score at baseline was 18.4 ± 10.7. The mean score at the time of first debulking surgery was 17.5 ± 9.2 (NS). Among the 8 patients with at least 100% increase in inter-surgical interval, the mean score at baseline and first debulking surgery were 14.4 ±10.3 and 12.8 ±9.0, respectively (NS).

Conclusions:

These data suggest that 1) HspE7 is active in the treatment of RRP; and 2) because the severity scores indicate that debulking surgery was conducted for similar severity of disease during the study, the observed increase in inter-surgical interval is not due to bias in the choice of surgery date.

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