ENETS single center of excellence experience with the NETest: A real-world assessment of 565 patients.

2020 ◽  
Vol 38 (4_suppl) ◽  
pp. 605-605
Author(s):  
Anna Malczewska ◽  
Mateusz Rydel ◽  
Amanda Robek ◽  
Katarzyna Kusnierz ◽  
Izabela Les-Zielinska ◽  
...  
Keyword(s):  
Real World ◽  
Clinical Status ◽  
Unmet Need ◽  
Clinical Information ◽  
Disease Status ◽  
Diagnostic Sensitivity ◽  
Lung Cancers ◽  
Center Of Excellence ◽  
Mcnemar’S Test ◽  
Mcnemar's Test

605 Background: There is a substantial clinical unmet need for an accurate and effective blood biomarker of NET disease. We therefore evaluated under real-world conditions the clinical utility of the NETest in a NET Center of Excellence and compare it with the biomarker CgA. Methods: Cohorts: GEP-NET (253), BP-NET (49), colon cancer (37), lung cancers (80), benign lung disease (59) and controls (86). GEP-NETs: 164 (65%) had image-detectable disease or were resection-margin (R1) positive. Grading included G1 [106], G2 [49] and G3 [9]. BP-NETs, 28 of 49 (57%) had evidence of disease. Grading was TC [14], AC [14]. Disease status (stable [SD] or progressive [PD]) determined by RECIST 1.1. Blood sampling: NETest ( n= 565) and NETest/CgA matched samples (135). NETest (PCR) (0-100 score) with positive > 20; progressive > 40. CgA (ELISA). All samples deidentified, and measurement/ analyses blinded. Statistics: Mann-Whitney U-test, McNemar’s test and AUROC. Results: GEPNET: NETest was significantly higher (34.4±1.8, p< 0.0001) in NET disease versus no NET disease (10.5±1, p< 0.0001), non-NET disease (18±4, p= 0.0004) or controls (7±0.5, p< 0.0001). Diagnostic sensitivity was 89%, and specificity 94%. NETest levels were not related to grade (G1: 32±2 vs. G2: 38±3, p= 0.09). BPNET: NETest was significantly higher (30±1.3) vs no NET disease (24.1±1.3, p= 0.0049). Diagnostic sensitivity 100%. Levels were elevated vs controls ( p< 0.0001) and non-NET disease (20±2, p= 0.0001). NETest levels were not related to grade (TC 30±2 vs. AC: 30±2, p= NS). Levels were elevated in PD (55±5.5) vs SD (33.6±2, p= 0.0005). AUCs for detecting disease ranged between 0.89 (GEP-NET) to 1.0 (BE-NET) ( p< 0.0001). Matched GEP-NETS (135): NETest was significantly more accurate for detecting NETS (99%) than CgA (53%, McNemar’s test Chi2= 87, p= 0.0001). sensitivity (99%) and specificity (96%) were better than CgA (37% and 96% respectively). Conclusions: The NETest is an accurate diagnostic test for both GEP- and BP-NEN. It defines clinical status (stable or progressive disease). NETest is significantly more accurate than CgA. The multianalyte genomic blood assessment of NET disease provided clinical information of utility in management.

scholarly journals Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective

2021 ◽  
pp. 1-7
Author(s):  
Diane Stephenson ◽  
Reham Badawy ◽  
Soania Mathur ◽  
Maria Tome ◽  
Lynn Rochester
Keyword(s):  
Disease Progression ◽  
Digital Health ◽  
Low Cost ◽  
Unmet Need ◽  
Disease Status ◽  
Standard Test ◽  
Imaging Biomarkers ◽  
Health Technologies ◽  
Therapeutic Development ◽  
The Rich

The burden of Parkinson’s disease (PD) continues to grow at an unsustainable pace particularly given that it now represents the fastest growing brain disease. Despite seminal discoveries in genetics and pathogenesis, people living with PD oftentimes wait years to obtain an accurate diagnosis and have no way to know their own prognostic fate once they do learn they have the disease. Currently, there is no objective biomarker to measure the onset, progression, and severity of PD along the disease continuum. Without such tools, the effectiveness of any given treatment, experimental or conventional cannot be measured. Such tools are urgently needed now more than ever given the rich number of new candidate therapies in the pipeline. Over the last decade, millions of dollars have been directed to identify biomarkers to inform progression of PD typically using molecular, fluid or imaging modalities). These efforts have produced novel insights in our understanding of PD including mechanistic targets, disease subtypes and imaging biomarkers. While we have learned a lot along the way, implementation of robust disease progression biomarkers as tools for quantifying changes in disease status or severity remains elusive. Biomarkers have improved health outcomes and led to accelerated drug approvals in key areas of unmet need such as oncology. Quantitative biomarker measures such as HbA1c a standard test for the monitoring of diabetes has impacted patient care and management, both for the healthcare professionals and the patient community. Such advances accelerate opportunities for early intervention including prevention of disease in high-risk individuals. In PD, progression markers are needed at all stages of the disease in order to catalyze drug development—this allows interventions aimed to halt or slow disease progression, very early, but also facilitates symptomatic treatments at moderate stages of the disease. Recently, attention has turned to the role of digital health technologies to complement the traditional modalities as they are relatively low cost, objective and scalable. Success in this endeavor would be transformative for clinical research and therapeutic development. Consequently, significant investment has led to a number of collaborative efforts to identify and validate suitable digital biomarkers of disease progression.

Medication Adherence Reminder System for Virtual Home Assistants: Mixed Methods Evaluative Study (Preprint)

2021 ◽  
Author(s):  
Cynthia F. Corbett ◽  
Elizabeth M. Combs ◽  
Peyton S. Chandarana ◽  
Isabel Stringfellow ◽  
Karen Worthy ◽  
...  
Keyword(s):  
Mixed Methods ◽  
Medication Adherence ◽  
Feasibility Study ◽  
Global Public Health ◽  
Constructive Feedback ◽  
The Past ◽  
Significant Difference ◽  
Mcnemar’S Test ◽  
Medication Reminder ◽  
Mcnemar's Test

BACKGROUND Medication non-adherence is a global public health challenge that results in sub-optimal health outcomes and increases healthcare costs. Forgetting to take medicines is one of the most common reasons for unintentional non-adherence. Research findings indicate that voice-activated virtual home assistants (VHAs), such as Amazon Echo and Google Home devices, may be useful in promoting medication adherence. OBJECTIVE Create a medication adherence app (skill) for Amazon Echo devices and measure the use, usability, and usefulness of that skill. METHODS A single-group mixed methods cohort feasibility study was conducted with females who took oral contraceptives (n=25). Participants were undergraduate students (mean age = 21.8, SD 6.2) at an urban university in the Southeast United States. Participants were given an Amazon Echo Dot with MedBuddy, a new medication reminder skill for Echo devices created by our team, attached to their study account, which they used for 60 days. Participants self-reported baseline and post-study medication adherence. MedBuddy use was objectively evaluated by tracking the participants’ interaction with MedBuddy through Amazon Alexa. The usability and usefulness of MedBuddy were evaluated through a post-study interview with participants responding to both quantitative and qualitative questions. RESULTS Participants’ interactions with MedBuddy, as tracked through Amazon Alexa, only occurred on half of the study days (mean of 50.97, SD 29.5). Compared to baseline, at study end participants reported missing their medication less in the past one and six months (χ 2 = .884 and .420 respectively, McNemar’s test p < .001 for both). However, there was no significant difference in participants’ reported adherence to consistently taking medication within the same two-hour time frame each day the past one or six months at the end of the study compared to baseline (χ 2 = 3.544 and 5.526 respectively, McNemar’s test p = .63 and p = .13 respectively). Overall feedback about usability was positive, and participants provided constructive feedback about features of the skill that could be improved. Participants’ evaluation of the usefulness of Medbuddy was overwhelmingly positive. Most participants (65.2%, n=15) said they would continue to use MedBuddy as a medication reminder in the future if provided the opportunity and the majority (91.3%, n=21) said they would recommend it to others. MedBuddy features that participants enjoyed were an external prompt separate from their phone, being able to hear the reminder prompt from a separate room, multiple reminders, and verbal responses as prompts. CONCLUSIONS The results of this feasibility study indicate the MedBuddy medication reminder skill may be useful in promoting medication adherence, but the skill could benefit from further usability enhancements.

scholarly journals The concept of control in chronic obstructive pulmonary disease: Development of the criteria and validation for use in clinical practice

2020 ◽  
Vol 30 (2) ◽  
pp. 135-141
Author(s):  
M. Miravitlles ◽  
B. Alcázar ◽  
J. J. Soler-Cataluña
Keyword(s):  
Chronic Obstructive Pulmonary Disease ◽  
Pulmonary Disease ◽  
Clinical Status ◽  
Disease Status ◽  
Chronic Obstructive ◽  
Cross Sectional ◽  
Obstructive Pulmonary Disease ◽  
Clinical Criteria ◽  
The Impact ◽  
Control Tool

Guidelines of treatment of chronic obstructive pulmonary disease (COPD) identify symptom reduction and prevention of exacerbations as the main goals of therapy. Initial pharmacological treatment must be guided by these parameters, and effectiveness must be assessed at each clinical visit. However, there is no clear guidance as to how this assessment must be performed. The concept of control has been well developed in asthma, but it has been elusive in COPD. Patients with COPD may not be completely free from symptoms or exacerbations even under optimized therapy; therefore, control in COPD does not mean cure or absence of symptoms, but rather reaching the best clinical status possible according to the level of disease severity. A control tool has been developed based on a cross sectional evaluation of the impact of the disease and a longitudinal evaluation of stability. Low impact is a disease status defined by at least 3 of the following: low levels of dyspnoea, absence of or white sputum, low use of rescue medication and self-declared walking time of more than 30 minutes a day, and stability is the absence of moderate or severe exacerbations in the previous 3 months. Control can also be defined by COPD Assessment Test (CAT) scores ≤ 10 units for patients with FEV1 ≥ 50% and 16 for patients with FEV1 < 50% and stability as a change in CAT ≤ 2 units. Control of COPD is then defined as a status of low impact and stability. The control tool has been validated prospectively in several studies and has demonstrated to be sensitive to clinical changes and to have a good predictive value for poor outcomes. Clinical criteria are more reliable than CAT scores for the evaluation of control. The control tool is a quick and inexpensive method to evaluate clinical status and future risk of exacerbations that can be used at all levels of healthcare. 

Treatment patterns among patients with advanced urothelial carcinoma following discontinuation of PD1/L1 inhibitor therapy.

2021 ◽  
Vol 39 (6_suppl) ◽  
pp. 414-414
Author(s):  
Alicia K. Morgans ◽  
Simrun Kaur Grewal ◽  
Zsolt Hepp ◽  
Rupali Fuldeore ◽  
Shardul Odak ◽  
...  
Keyword(s):  
Urothelial Carcinoma ◽  
Real World ◽  
Chart Review ◽  
Group Performance ◽  
Performance Status ◽  
Locally Advanced ◽  
Eastern Cooperative Oncology Group ◽  
Unmet Need ◽  
Case Series ◽  
Treatment Patterns

414 Background: There are a lack of published real-world data on treatment patterns for patients with locally advanced or metastatic urothelial carcinoma (la/mUC) previously treated with programmed death 1/ligand 1 inhibitor (PD-1/L1i) therapy. The objective of this study was to characterize the clinical characteristics and treatments among patients with la/mUC following discontinuation of first-line (1L) or second-line (2L) PD-1/L1i therapy. Methods: We performed a retrospective chart review at 26 geographically diverse clinical sites in the US. Patients aged ≥18 years with histologically or cytologically confirmed urothelial carcinoma and radiographic evidence of metastatic or locally advanced disease were identified. Included patients had initiated and subsequently discontinued PD-1/L1i therapy in the 1L or 2L setting for la/mUC between May 15, 2016-July 31, 2018. All patients had follow-up through October 31, 2019. Data were summarized using descriptive statistics. Results: Among the 300 patients included in the chart review, 198 (66%) received PD-1/L1i therapy as 1L and 102 (34%) as 2L therapy. Mean (SD) age at la/mUC diagnosis was 69.4 (8.7) years, and a majority of patients were male (66.0%) and White (74.7%). Consistent with age, most patients (82.7%) had comorbidities at la/mUC diagnosis; 39.7% hypertension, 23.7% coronary artery disease, 17.7% pulmonary disease, and 9.3% renal disease. At initiation of therapy, a higher proportion of patients who received 1L PD-1/L1i therapy had an Eastern Cooperative Oncology Group performance status of 2 or more than patients who received 2L PD-1/L1i therapy (36.8% vs 22.5%, respectively). Following discontinuation of PD-1/L1i therapy, 34% (n = 68) received subsequent therapy in 2L and 29% (n = 30) in third-line (3L). The most common subsequent therapies in 2L were gemcitabine monotherapy (24%), gemcitabine plus cisplatin or carboplatin (22%), PD-1/L1i therapy (22%), and taxane monotherapy (19%). The most common subsequent therapies received in 3L were taxane monotherapy (50%), pemetrexed (17%), and PD-1/L1i therapy (16%). Overall, switching from one PD-1/L1i therapy to another distinct PD-1/L1i therapy occurred in approximately 20% of patients, with “better efficacy/survival” noted by treatment teams as the most common reason for switching therapy among this subgroup. Conclusions: In this real-world case series, only a minority of patients with la/mUC who discontinued PD-1/L1i therapy received subsequent therapy. Among those that did, no clear standard of care was observed and approximately one-fifth of patients were treated with a second PD-1/L1i therapy after the first failed to control disease. Collectively, the data highlight significant unmet need for patients with la/mUC who discontinue PD-1/L1i therapy.

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