314 Background: In a recent trial, we observed no statistically significant increase in engagement after a web-based decision aid (DA) was delivered to breast cancer patients prior to a surgical consult. However, a minority of the intervention group (44%) reviewed the DA. The objective is to assess the effect of the DA on engagement for the cohort of patients who reviewed it. Methods: A stepped wedge trial was conducted with 10 NCI Community Oncology Research Program clinics that care for a high proportion of socioeconomically disadvantaged patients (6/2019-12/2021). Clinics were randomized to time of transition from usual care (UC) to delivery of a DA. Patients with stage 0-3 breast cancer eligible for surgery provided consent prior to a surgical consult. Engagement was measured from audio-recorded surgical consults (Active Patient Behaviors, Street protocol). For this analysis, we identified a cohort of treatment compliant (TC) patients who reviewed the DA. Intervention effects comparing TC and UC groups were tested with linear mixed-effects models, accounting for time, enrollment post-COVID, and patient age, race, education, and socioeconomic disadvantage (assessed with Area Deprivation Index and dichotomized). Exploratory models were generated using Active Patient Behavior subcomponents (asking questions, assertive responses, expressions of concern) as the outcome. Results: The cohort includes 407 patients (UC 264; TC 143). TC patients were more likely to be white (TC 73%, UC 64%, p=0.03), have a college degree (TC 65%, UC 55%, p=0.05), and not be disadvantaged (TC 89%, UC 72%, p=0.001). TC was associated with increased engagement (8.2 [0.08, 16.2], p=0.048, Table). A college degree was associated with higher, while black race and socioeconomic disadvantage lower, engagement. On exploratory analyses, TC had a stronger association with higher levels of assertive responses (2.9 [-0.3, 6.1], p=0.07) compared with asking questions (5.2 [-1.4, 11.9], p=0.12) or expressions of concern (0.08 [-0.60, 0.76], p=0.82). Conclusions: We observed increased engagement in breast cancer patients who reviewed a DA prior to a surgical consult. However, we also observed differences based on race, education, and socioeconomic disadvantage. Further research will identify adjunct interventions to combine with the DA to improve cancer care delivery for diverse populations. Support: UG1 CA189823; AHRQ R01HS025194; https://acknowledgments.alliancefound.org. Clinical trial information: NCT03766009 .[Table: see text]
PurposeTo evaluate the safety of velaglucerase alfa in patients with type 1 Gaucher disease who received velaglucerase alfa in the US treatment protocol HGT-GCB-058 (ClinicalTrials.gov identifier NCT00954460) during a global supply shortage of imiglucerase.MethodsThis multicenter open-label treatment protocol enrolled patients who were either treatment naïve or had been receiving imiglucerase. Patients received intravenous velaglucerase alfa every other week at a dose of 60 U/kg (treatment naïve) or 15–60 U/kg (previously treated).ResultsA total of 211 (including six treatment-naïve) patients were enrolled. Among the 205 previously treated patients, 35 (17.1%) experienced an adverse event considered related to study drug. Among the six treatment-naïve patients, one had an adverse event considered related to study drug. Infusion-related adverse events occurred in 28 (13.3%) of the 211 patients and usually occurred during the first three infusions. De novo, nonneutralizing, anti–velaglucerase alfa antibodies developed during treatment in one (<1.0%) previously treated patient and none of the treatment-naïve patients.ConclusionThe currently observed safety profile was consistent with those previously reported for imiglucerase and velaglucerase alfa phase III clinical trials. These results support the safety of initiating treatment with velaglucerase alfa or transitioning patients from imiglucerase therapy to velaglucerase alfa therapy.
<p>Supplementary Figure S2. Kaplan Meier survival analysis evaluating association of PD-L1+ (SP142 assay with {greater than or equal to}1% IC+, in red) to PD-L1- tumors (SP142 assay <1% IC+ in black) with recurrence-free survival (RFS) (A) or overall survival (B) in the entire TNBC cohort.</p>
Introduction: Patients with familial adenomatous polyposis (FAP) are at markedly increased risk for duodenal polyps and cancer. Combination sulindac and erlotinib was previously shown to reduce duodenal polyp burden but was associated with a high adverse event (AE) rate. Current aim was to evaluate if a once weekly erlotinib intervention can reduce AEs while still providing efficacy with respect to reduced duodenal polyp burden in FAP participants. Methods: Single-arm trial conducted by the NCI Cancer Prevention Network, with FAP participants enrolled from October 2017 through September 2019 at eight academic medical centers. Participants self-administered 350 mg of erlotinib by mouth, once weekly for six months. Duodenal polyp burden (sum of polyp diameters) was assessed in the proximal duodenum by esophagogastroduodenoscopy (EGD) performed at baseline and at six months, with mean percent change in duodenal polyp burden defined as the primary outcome of interest. Rate of grade 2 or 3 AEs was evaluated as a co-primary outcome. Results: Forty-six participants (mean age 44.1 years [range 18-68]; 22 women [48%]) were enrolled; 42 completed intervention and were included in the per-protocol analysis. The total duodenal polyp burden was significantly reduced after six months of intervention, with a mean percent change of -29.6% (95% CI, -39.6% to -19.7%; P < 0.0001) (Table and Figure). Similar results were observed for participant subgroups defined by advanced (Spigelman stage III) duodenal polyp burden at baseline (mean -27%; 95% CI, -38.7% to -15.2%; P < 0.0001). Lower GI polyp numbers were assessed in 31 patients (9 IPAA, 16 IRA, 6 ileostomy) and were also moderately decreased after six months of intervention (median -30.8%; IQR -47.4% to 0.0%; P = 0.0256). Grade 2 or 3 AEs were reported in 71.7% of subjects, with only two experiencing grade 3 toxicity. The most common AE was an acneiform-like rash, which occurred in 56.5% of participants and was managed with topical cortisone and/or clindamycin therapy. Conclusion: In this single-arm, multi-center trial of FAP participants, erlotinib once weekly resulted in a significantly reduced duodenal and lower GI polyp burden after six months of intervention. While AEs were still reported by nearly three-quarters of participants, the events were generally of a low grade and well-tolerated. These findings support further investigation of erlotinib as an effective, acceptable cancer chemopreventive agent for FAP participants. NCT 02961374.Figure.: Per-protocol analysis of percent change in sum of duodenal polyp diameters for each participantTable.: Change in duodenal polyp burden from baseline. Abbreviations: SD, standard deviation; CI, confidence interval; FAP, familial adenomatous polyposis; APC, aerobic plate count 1. Paired t-test of observed mean change, %, against 0%
151 Background: Decision aids (DAs) for prostate cancer treatment can improve knowledge and reduce decisional conflict, but the relative effect of pre-visit and within-visit DAs is not known, and effect sizes for minority populations has not been estimated. Methods: We conducted a 3-arm, patient-level-RCT in specialty urology and radiation oncology practices in Ohio, South Dakota, and Alaska, test the effect of pre-consultation and with-in consultation decision aids on patient knowledge immediately after specialty consultation compared to usual care. We used linear regression to estimate effects of each intervention arm, including the respective standard error, two-sided 95% confidence interval, and two-sided P value for testing the study’s hypotheses. Results: 103 patients were recruited and randomized to receive either the pre-visit decision aid, within-visit decision aid, or neither decision aids (usual care). In 2017 and 20018, we accrued similar numbers of men to pre-consultation aid (n = 37), during-consultation aid (n = 33) and usual care arms, respectively (n = 33). The median (range) age in years was 64 [49, 81]; 67.6% were White, 15.7% were Black or African American, 16.7% were American Indian or Alaska Native, and 1% were not reported. 47.6%, 45.6%, and 6.8% had a baseline clinical stage of T1, T2, and T3, respectively. The median [range] prostate specific antigen (PSA) was 8.0 [2.4, 53.7]. There were no clinically notable imbalances. We obtained usable data on 102 of the 103 patient-participants. The pre-visit decision aid arm showed a mean knowledge effect of 0.694 (0.636, 0.753). The within-visit decision arm showed a very similar mean knowledge effect of 0.686 (0.625, 0.748). The usual care arm showed a mean knowledge effect of 0.644 (0.582,0.705). The linear regression model showed, that, compared with usual care, neither intervention effect achieved statistical significance in the primary analysis (p = 0.24, 0.330, for pre-visit and within-visit, respectively). Conclusions: Modest knowledge gains of pre-visit and during-visit decision aids for prostate cancer treatment were not statistically significant. Clinical trial information: NCT03182998 .
