Long-term safety and efficacy of Oleogel-S10 (birch triterpenes) in epidermolysis bullosa wounds: 24-month open-label data from the EASE study

Objectives: The 90-day double-blind phase (DBP) of the Phase 3 EASE study demonstrated accelerated wound healing for Oleogel-S10 (birch triterpenes) versus control gel in epidermolysis bullosa (EB). Here, we report safety and total wound burden results from the 24-month open-label phase (OLP) in which all patients received treatment with Oleogel-S10. Method(s): Total wound burden was assessed using EB Disease Activity and Scarring Index (EBDASI) and Body Surface Area Percentage (BSAP). Data are reported without visit windows to reflect a realworld situation more accurately, particularly considering the COVID- 19 pandemic. Result(s): The patient population was made up of dystrophic EB (n = 178, 86.8%) and junctional EB (n = 25, 12.2%);71.7% (n = 147) of patients were aged <18 years. 141 patients (68.8%) completed the OLP. The mean (SD) treatment duration for all patients was 584.7 (246.1) days. Adverse events were reported in 77.1% of all patients in the OLP versus 81.7% of those receiving Oleogel-S10 in the DBP. Mean BSAP for patients treated with Oleogel-S10 in the DBP reduced from 12.1% at study entry to 6.1% with 27 months of treatment. Similarly, the mean EBDASI skin activity score in the Oleogel-S10 group improved from 19.6 to 15.1 after 27 months. In addition, reductions in both BSAP and EBDASI from OLP baseline were observed in patients who transitioned from control gel to Oleogel-S10 in the OLP. Discussion(s): These data support a reassuring long-term safety profile of Oleogel-S10. Furthermore, the reduction in wound burden previously reported with 15 months of Oleogel-S10 treatment is maintained to the end of OLP. This is encouraging given the nature of this chronic genetic disorder in which there is regular cycling of patients' fragile wounds.

Evaluation of teledermatology during a pandemic indicates high patient satisfaction, positive impact on patient-related costs and clinical effectiveness

Aim: Telehealth is becoming an increasingly important way of delivering health services accelerated by the COVID-19 pandemic. The aim of the study is to evaluate teledermatology by assessing patient satisfaction, patientrelated costs and clinical effectiveness. Method: This was a single-centre cross-sectional study. A three-part questionnaire-Dermatology life quality index (DLQI), patient satisfaction questionnaire (PSQ) and cost evaluation questionnaire-was sent out to all teledermatology patients attending the Royal Melbourne Hospital. The Spearman rank-order correlation coefficient was used to measure the association between the DLQI and PSQ. We evaluated the clinical effectiveness in psoriasis patients on biologic therapy as a sub-cohort by comparing PASI and DLQI pre and post implementation of teledermatology, using Wilcoxon signed-rank test. Results: 191/747 patients responded (51% male, 73% living in metropolitan victoria). The mean DLQI was 5.2 +/- 6.25. PSQ was divided into four categories (Positive attitude, hotel, photo anxiety and interaction). There is a negative correlation between DLQI and PSQ, with all categories showing significant correlation except the 'positive attitude' category. Analysis of the cost evaluation questionnaire showed that a majority of patients reported that telehealth appointments save time (99%), reduce time off work (41%) and help avoid costs of transport (96%). When assessing patients with psoriasis on biologic therapy, there was no significant difference in PASI scores before (mean of 1.59) and after (mean of 1.44) implementation of teledermatology (p-value 0.542). There was a significant reduction in DLQI in 59 psoriasis patients on biologic therapy, as an indicator of clinical effectiveness. The mean DLQI before implementation of teledermatology was 2.73 and after implementation was 1.69 (p-value 0.0126). Conclusions: Overall this study indicates high patient satisfaction, positive impact on patient-related costs and good clinical effectiveness of teledermatology which warrant further evaluation in larger prospective studies.

Durvalumab in frail and older patients with stage iv non-small cell lung cancer: The randomized phase ii duration trial

Introduction: Checkpoint inhibitors have recently been established as first-line treatment for metastatic NSCLC. Notably, a phenomenon known as immunosenescence, i.e. the progressive remodeling of the immune system with aging, as well as the inflammatory status, may interfere with the activity of PD-1/PD-L1 inhibitors. Although frail and older patients represent a major fraction of non-small cell lung cancer (NSCLC) patients in routine clinical practice, this population is still underrepresented in most clinical trials. Thus, clinical evidence about tolerability and efficacy of these novel agents in the elder and frail population is urgently required. Methods/Design: DURATION (NCT03345810) is a prospective, open label, treatment stratified, randomized, multicenter phase II study. The protocol foresaw the enrollment of 200 patients diagnosed with stage IV NSCLC ≥70 years old and/or with Charlson-Comorbidity Index > 1 and/ or with a performance status ECOG > 1. Patients were stratified according to Cancer and Age Research Group (CARG) score in 'fit' and 'less fit'. Patients were then randomized 1:1 to receive either CT or two cycles of CT followed by two cycles of durvalumab and durvalumab maintenance. Details are shown in figure 1. The primary endpoint is the rate of treatment-related grade III/IV adverse events. Secondary endpoints are progression-free survival, response rate and overall survival and quality of life assessment. The trial is accompanied by a biomaterial repository to explore potential biomarkers. Results: The last patient was enrolled in January 2021. Forty-nine, 48, 51, and 52 patients were allocated to arm A, arm B, arm C, and arm D, respectively. Currently, 145 patients have completed the study, 13 patients are under treatment (12 in total in durvalumab-maintenance arms and 1 in the CT arm), while 42 are in follow-up. Conclusions: Inspite of the COVID-19 pandemic, the DURATION trial successfully completed the planned enrollment of 200 patients, underlying the urgent need for evidence of efficacy and tolerability of immunotherapy in the challenging population of frail and elderly patients.

Investigating decreased positive airway pressure compliance in a veteran affairs sleep medicine clinic during the 2020 pandemic

Introduction: Positive airway pressure (PAP) compliance for the treatment of sleep apnea at the Albuquerque Veterans Affairs (VA) Sleep Medicine clinic has been observed to be lower in new setup patients after the onset of the COVID-19 pandemic. The reasons for decreased PAP compliance during the COVID-19 pandemic are unclear. The primary outcome will be to identify if there is a common reason that patients at the Albuquerque VA were less compliant with PAP after the onset of the COVID-19 pandemic. Methods: Compliance data for 4/1/2020 through 9/30/2020 was compared to compliance data for 4/1/2019 through 9/30/2019. Compliance after PAP machine setup was confirmed to be lower during the 2020 time period. Noncompliant patients will be selected by setup type, new versus machine replacement, and surveyed for reasons for noncompliance. The survey will be conducted at the Albuquerque VA Sleep Center and will include questions regarding beliefs, barriers, and challenges with the use of PAP therapy during the coronavirus pandemic. The definition for initial PAP compliance will be the use of PAP therapy for greater than or equal to four hours per night on at least 70% of nights. Results: For the 6-month time period of 4/1/2019 through 9/30/2019, there were 758 PAP setups at the Albuquerque VA. The 30-day compliance for the 758 setups was found to be 61.4%. Comparatively, for the six-month period of 4/1/2020 through 9/30/2020, there were 462 setups with a 30-day compliance result of 49.7%. A survey consisting of questions designed to elicit barriers to use as well as beliefs regarding PAP and COVID-19 will be administered to 20% (n = 46) of the non-compliant patients who were set up with a PAP machine during the 2020 study period. Conclusion: PAP compliance after machine setup was lower at the Albuquerque VA sleep center in 2020 versus 2019 (49.7% versus 61.4%). The reasons for the lower observed compliance are attributed to the effects of the coronavirus pandemic. A random sampling of the non-compliant patients during the 2020 time period will be performed and the results will be presented once available.