Data Submission Standards and Evidence Requirements

Presented are recommendations of the Data Submission Standards and Evidence Requirements panel from the Conference on Clinical Cancer Research along with the U.S. Food and Drug Administration's response to these recommendations

THE ASSOCIATION OF STEP-BASED METRICS WITH ADIPOSITY AND BLOOD PRESSURE IN THE HISPANIC COMMUNITY HEALTH STUDY/STUDY OF LATINOS

ABSTRACT Samantha Rose Schilsky: The Association of Step-based metrics and Adiposity and Blood Pressure in the Hispanic Community Health Study/Study of Latinos(Under the direction of Wayne D. Rosamond) Measures of adiposity and elevated blood pressure (BP) are established and highly prevalent cardiovascular disease (CVD) risk factors. Physical activity is a recommended non-pharmacologic intervention for alleviating overweight and obesity as well as high BP. Steps are an interpretable measure of physical activity. Research examining associations between step volume and cadence in relation to BP and measures of adiposity is limited and has not been examined in an ethnically diverse Hispanic/Latino population.This dissertation estimated associations of step volume (average daily total steps) and cadence (steps/min) with measures of adiposity and 6-year changes in measures of adiposity (weight, weight circumference (WC), body mass index (BMI) and weight maintenance) as well as measures of BP and 6-year changes in BP (systolic (SBP), diastolic (DBP)). The Hispanic Community Health Study/Study of Latinos (HCHS/SOL) (2008-2017) was used for these analyses. We included 12,353 and 12141 adults for the cross-sectional analyses of measures of adiposity and BP respectively and 8,427 and 9,077 adults for the longitudinal analyses of measures of changes in adiposity and BP, respectively. Engaging in lower step volume and intensity was cross-sectionally associated with higher measures of adiposity and higher odds of obesity and hypertension. Compared to those in the highest quartile of daily steps those in the lowest quartile had 1.4 and 1.5 times the odds of obesity and hypertension, respectively. Compared to those with the highest intensity levels, those in the lowest levels of average peak 30-minute cadence, brisk walking and faster ambulation and bouts of brisk walking and faster ambulation had a 1.6, 2.1 and 1.2 times the odds of obesity, respectively and a 1.4, 1.3 and 1.5 times the odds of hypertension, respectively. Engaging in less time spent sedentary (quartile 1) had a 0.74 times odds of hypertension compared to engaging in more time spent sedentary (quartile 4). Over a 6-year period, engaging in higher step intensity but not volume was associated with greater mean changes in weight and BMI.These findings suggest that engaging in higher daily step volume and cadences may be associated with more favorable obesity and hypertension profiles in Hispanic/Latino populations.Doctor of Public Healt

Costly choices for treating Wilson's disease

Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/110871/1/hep27663.pd

Magnetic resonance imaging radiomics to predict high-risk intraductal papillary mucinous neoplasms of the pancreas

BACKGROUND: Pancreatic cancer is one of the most lethal cancers. Despite enhanced understanding of the disease, the 5-year survival rate remains 8% due to the late stage of diagnosis and a lack of effective treatment options. Early detection of precancerous lesions, such as intraductal papillary mucinous neoplasms (IPMNs), is a strategy to prevent pancreas cancer related death. Standard qualitative imaging assessment cannot reliably distinguish between benign and malignant branch duct intraductal papillary mucinous neoplasms (BD-IPMNs). A more consistent risk prediction method is needed to inform clinical decision making such that patients with benign cysts may be spared from unnecessary surgical resection. OBJECTIVE: To assess whether a BD-IPMN malignancy risk prediction model which demonstrated strong potential on preoperative computed tomography (CT) images would show similar results on magnetic resonance imaging (MRI). METHODS: 19 pathologically proven BD-IPMN patients with preoperative contrast-enhanced CT and MRI and were included in the study. Five radiomics features were extracted from the portal-venous phase CT and MR images of the largest cyst. Associations between radiomics features extracted from CT and MR were assessed using Pearson correlations. RESULTS: Of the five radiomics features, average-weighted eccentricity (AWE) was most strongly correlated between imaging modalities in all patients (n=19, r=0.46, 95% CI=0.001-0.75, p=0.05), low-risk patients (r=0.63, 95% CI=0.09-0.88, p=0.028), and patients with a solid component or mural nodule (r=0.66, 95% CI=-0.32-0.96, p=0.15). However, when two outliers within the dataset were removed from analysis, AWE no longer correlated between MR and CT. None of the other radiomics features displayed significant correlations between the modalities. CONCLUSIONS: The CT-based risk prediction model cannot be applied to MR data suggesting that a new model should be created from MRI data alone.2021-06-17T00:00:00

A multicenter retrospective cohort analysis of therapeutic hypothermia in acute liver failure

Cerebral edema is a severe and life-threatening complication in acute liver failure (ALF). Concerns exist that therapeutic hypothermia (TH) may increase the risk of infection, worsen coagulopathy and inhibit hepatic regeneration. We therefore reviewed the experience in use of TH in participating US Acute Liver Failure Study Group (ALFSG) centers. The aims were to determine utilization of TH in ALF patients at high risk for cerebral edema (grade III or IV hepatic encephalopathy (HE)) and to determine its effect on survival and complication rates

Challenges and Opportunities to Updating Prescribing Information for Longstanding Oncology Drugs.

A number of important drugs used to treat cancer-many of which serve as the backbone of modern chemotherapy regimens-have outdated prescribing information in their drug labeling. The Food and Drug Administration is undertaking a pilot project to develop a process and criteria for updating prescribing information for longstanding oncology drugs, based on the breadth of knowledge the cancer community has accumulated with the use of these drugs over time. This article highlights a number of considerations for labeling updates, including selecting priorities for updating; data sources and evidentiary criteria; as well as the risks, challenges, and opportunities for iterative review to ensure prescribing information for oncology drugs remains relevant to current clinical practice

Provider-Initiated HIV Testing and Counseling: Increased Uptake in Two Public Community Health Centers in South Africa and Implications for Scale-Up

BACKGROUND: International guidance recommends the scale up of routinely recommended, offered, and delivered health care provider-initiated HIV testing and counseling (PITC) to increase the proportion of persons who know their HIV status. We compared HIV test uptake under PITC to provider-referral to voluntary counseling and testing (VCT referral) in two primary health centers in South Africa. METHODS: Prior to introducing PITC, clinical providers were instructed to refer systematically selected study participants to VCT. After PITC and HIV rapid test training, providers were asked to recommend, offer and provide HIV testing to study participants during the clinical consultation. Participants were interviewed before and after their consultation to assess their HIV testing experiences. RESULTS: HIV test uptake increased under PITC (OR 2.85, 95% CI 1.71, 4.76), and more patients felt providers answered their questions on HIV (104/141 [74%] versus 73/118 [62%] for VCT referral; p 0.04). After three months, only 4/106 (3.8%) HIV-positive patients had registered for onsite HIV treatment. Providers found PITC useful, but tested very few patients (range 0-15). CONCLUSION: PITC increased the uptake of HIV testing compared with referral to onsite VCT, and patients reported a positive response to PITC. However, providing universal PITC will require strong leadership to train and motivate providers, and interventions to link HIV-positive persons to HIV treatment centers