Citations

Abstract Introduction Peyronie’s disease (PD) is a connective tissue disorder associated with the formation of fibrotic plaques in the tunica albuginea, leading to penile curvature. PD is underdiagnosed, and this may be related to the reluctance of some men to seek consultation with a health care provider (HCP). Objective The aim was to develop an English-language digital application (app) to increase PD awareness, give men concerned about penile curvature a self-screening tool, and provide a link for locating HCPs with expertise in PD. Methods The Peyronie’s Disease Self-Assessment App is Android- and iOS-compatible. It was developed as a self-assessment tool for screening purposes only. It is not intended to diagnose, treat, cure, or prevent any medical condition, disease, or health problem and should not be considered a replacement for a consultation with a physician. The app provides instructional videos on general features and how to perform a penile scanning procedure. Results The PD Self-Assessment App includes a questionnaire related to penile curvature, penetration difficulty, degree of bother for partner, and impact on sexual activity. An innovative feature of the app is a live scanning technology, which uses the phone’s camera to detect the contours of the penis from multiple angles and applies an algorithm that determines the degree of curvature. The resulting image is a 3D penile simulation in the app, which allows the user to mark the location of any “lumps.” The app will also produce a 2D outline to determine the maximum curvature. External links are provided to a PD education website and to locate an HCP. The patient can share both the questionnaire responses and penile curvature simulation with his HCP via the user’s native email. This tool has password protection features and encrypts sensitive data. The scan images are representations of the penis, not photographs. Images are stored in the app library and are not stored in the user’s photo gallery. To enhance security, all data are erased when the app is uninstalled. The app is adaptable and scalable as needed for future updates. Conclusions The PD Self-Assessment App will help improve patient knowledge of the condition, document penile deformity, and facilitate consultation with an HCP. For men with PD, the app will help ease their journey with PD. The app will assist HCPs with evaluation and diagnosis. Sequential documentation of penile curvature during medical treatment, during mechanical traction protocols, or following surgery will provide an evidence basis for tracking long-term outcomes in PD. Disclosure Yes, this is sponsored by industry/sponsor: Endo Pharmaceuticals Inc. Clarification Industry initiated, executed and funded study Any of the authors act as a consultant, employee or shareholder of an industry for: Endo Pharmaceuticals Inc.

10526 Background: Pro-angiogenic factors may represent therapeutic targets in sarcoma. Preclinical studies have demonstrated a potential additive or synergistic interaction between anti-angiogenic agents and chemotherapy. The purpose of this study was to determine the maximum tolerated dose (MTD), toxicities, pharmacokinetic (PK) and pharmacodynamics (PD) effects of PAZIT in patients with advanced sarcoma. Methods: Patients 6-30 years of age with relapsed/refractory sarcomas were eligible. In the initial dose escalation plan (A), patients received pazopanib PO (225-450 mg/m2/dose) on Days 1-21 of 21-day cycles. Pazopanib was combined with fixed doses of irinotecan (IV 50 or PO 90 mg/m2/dose) and temozolomide PO 100 mg/m2/dose on Days 1-5. Due to DLTs, an amendment was made to the dose escalation plan (B) and patients received fixed doses of pazopanib PO (225 mg/m2/dose) on Days 1-21 of 21-day cycles and reduced irinotecan doses (IV 25-37.5 or PO 45-67.5 mg/m2/dose) and temozolomide PO 100 mg/m2/dose on Days 1-5. Oral cephalosporin diarrhea prophylaxis was required. Dose escalation followed a 3+3 design. Correlative studies included PK (pazopanib, irinotecan) and PD (angiogenic factors, ctDNA) effects. Results: Sixteen patients were treated (median age 16 years, range 7-21). The dose levels in the table were evaluated. First cycle DLTs occurred at all dose levels (Table) and included diarrhea, pancreatitis, colitis, neutropenia, hypertension, deep vein thrombosis, and ALT increase. Due to excessive toxicity, an MTD could not be established. One patient with osteosarcoma had a partial response. Four patients had prolonged stable disease > 4 cycles, including 2 patients with Ewing sarcoma (5 and 6 cycles), rhabdomyosarcoma (9 cycles), and desmoplastic small round cell tumor (6 cycles). Mean±SD plasma exposures to pazopanib, irinotecan, and SN-38 in patients treated on dose level 1B (n = 4) on Day 4 were 601±83, 1.4±0.2 and 0.1±0.04 ug/mL*hr, respectively. Analyses of correlative studies are ongoing. Conclusions: Combination PAZIT therapy is not tolerable as evaluated at these doses/schedules. This study provides important toxicity data to inform future clinical trials using combination anti-angiogenic strategies in sarcoma. Clinical trial information: NCT03139331. [Table: see text]