BACKGROUND: Right-side infective endocarditis (RSIE) is caused by microorganisms and develops into intracardiac and extracardiac complications with high in-hospital and 1-year mortality. Treatments involve antibiotic and surgical intervention. However, those presenting with extremes e.g. heart failure, or septic shock who are not ideal candidates for conventional medical therapy might benefit from minimally invasive procedures.
OBJECTIVE: This review summarizes existing observational studies that reported minimally invasive procedures to debulk vegetation due to infective endocarditis either on valve or cardiac implantable electronic devices.
METHODS: A targeted literature review was conducted to identify studies published in PubMed/MEDLINE, EMBASE, and Cochrane Central Database from January 1, 2015 to June 5, 2023. The efficacy and/or effectiveness of minimally invasive procedural interventions to debulk vegetation due to RSIE were summarized following PRISMA guidelines.
RESULTS: A total of 11 studies with 208 RSIE patients were included. There were 9 studies that assessed the effectiveness of the AngioVac system and 2 assessed the Penumbra system. Overall procedure success rate was 87.9%. Among 8 studies that reported index hospitalization, 4 studies reported no death, while the other 4 studies reported 10 deaths.
CONCLUSIONS: This study demonstrates that multiple systems can provide minimally invasive procedure options for patients with RSIE with high procedural success. However, there are mixed results regarding complications and mortality rates. Further large cohort studies or randomized clinical trials are warranted to assess and/or compare the efficacy and safety of these systems.

Regulatory guidance advises validation of patient-reported outcome (PRO) instruments prior to use in pivotal clinical studies, which may then be used to generate critical patient-centered evidence and support labelling claims. This targeted literature review aimed to determine if PRO instruments psychometrically validated in a phase 3 trial setting could support label claims from the same phase 3 study (i.e. PRO data were generated as an endpoint).
A targeted search of published studies (1 January 2006-3 June 2021) using the MEDLINE database identified PRO instruments validated during phase 3 trials. The search included instrument terms (e.g. patient-reported outcome measures, questionnaire, survey) and validation terms (e.g. reproducibility, minimal important difference), without filtering for therapeutic indications. Results were limited to phase 3 clinical trials or validation studies. The PROLABELS database was used to identify PROs validated in phase 3 trials and accepted in labelling claims.
Of 355 references identified, 68 studies with PRO psychometric validation in phase 3 studies were selected, covering 78 instruments. Of these, 20 were novel PRO instruments and 58 were existing instruments being validated for a new therapeutic indication/population. The psychometric properties most frequently validated were internal consistency reliability, known-group validity, responsiveness, minimal important difference, and concurrent validity. Five novel instruments obtained ten labelling claims for seven drugs/products.
These results suggest that quantitative validation of novel PRO instruments, and existing PROs for new indications, can occur within the context of phase 3 trials, and these PROs can also support label claims.

UNASSIGNED: Extraintestinal Pathogenic Escherichia coli (ExPEC) is a leading cause of invasive disease, including bacteremia and sepsis. Invasive ExPEC disease (IED) has the potential to complicate the clinical treatment of other conditions and is associated with an increased mortality, hospitalization, and worse outcomes. Older adults and individuals with comorbid conditions are at higher risk of IED. ExPEC is of particular concern in the Asia Pacific region due to aging populations and rising antimicrobial resistance.
UNASSIGNED: This study aimed to synthesize most recent data on the epidemiology, clinical and economic burden of IED in the elderly/high risk populations in China, Japan, South Korea, Taiwan, and Australia.
UNASSIGNED: A targeted literature review was conducted using Embase, Medline, as well as local scientific databases. We included studies published in English and local languages published from January 1, 2010 to October 7, 2020 that were relevant to the research objectives. Studies were narratively synthesized.
UNASSIGNED: A total of 1,047 studies were identified and 34 of them were included in this review. ExPEC accounted for 46.0% (1,238/2,692) of bacteria-related invasive diseases in patients aged above 60 years in South Korea, followed by China (44.4% (284/640)), Taiwan (39.0% (1,244/3,194)), and Japan (18.1% (581/3,206)), while Australia reported ExPEC out of all pathogens (54.7% (4,006/7,330)) in general adults. Comorbidities such as diabetes or cancer were common in these patients. Studies reported increases in length-of-stay, and in-hospital 30-day all-cause mortality related to ExPEC associated bacteremia was between 9% to 12%. From a cost perspective, a 3-fold increase in sepsis-associated cost was reported in South Korea between 2005 and 2012. In Australia, antimicrobial resistance contributed to an additional cost of AUD $5.8 million per year (95% uncertainty interval [UI], $2.2-$11.2 million) in the treatment of bloodstream infections (BSIs).
UNASSIGNED: ExPEC was a major cause of blood stream infection across China, Japan, South Korea, Taiwan, and Australia. Both the clinical and economic burden associated to ExPEC infections as well as the antimicrobial resistance observed in the elderly call for preventive and curative actions in these regions.
Extraintestinal Pathogenic Escherichia coli (ExPEC) is a leading cause of invasive disease, including bacteremia and sepsis.A targeted literature review included the most recent data from 34 published studies on the epidemiology and clinical and economic burden of IED in the elderly/high risk populations in China, Japan, South Korea, Taiwan, and Australia.ExPEC accounted for 46.0% (1,238/2,692) of bacteria-related invasive diseases in patients aged above 60 years in South Korea, followed by China (44.4% (284/640)), Taiwan (39.0% (1,244/3,194)), and Japan (18.1% (581/3,206)), while Australia reported ExPEC out of all pathogens (54.7% (4,006/7,330)) in general adults. Studies reported increases in length-of-stay and in-hospital 30-day all-cause between 9% to 12%. These factors, along with antimicrobial resistance observed in the elderly, call for preventive and curative actions in these regions.Data for costs associated with ExPEC induced BSI or sepsis in this region are limited, but evidence shows increasing expenditures.

Clinical guidelines recommend that parenteral nutrition (PN) is added to enteral nutrition (EN; supplemental parenteral nutrition (SPN)) in order to meet energy and protein needs in patients with cancer when EN alone is insufficient. However, although cancer-related malnutrition is common, there is poor awareness of the value of nutritional care, resulting in SPN being chronically underused.
We performed a targeted literature review and exploratory cost-utility analysis to gather evidence on the clinical effectiveness of SPN, and to estimate the potential cost-effectiveness of SPN versus EN alone in an example cancer setting.
The literature review identified studies linking SPN with malnutrition markers, and studies linking malnutrition markers with clinical outcomes. SPN was linked to improvements in body mass index (BMI), fat-free mass, phase angle (PhA) and prealbumin. Of these markers, BMI and PhA were strong predictors of survival. By combining published data, we generated indirect estimates of the overall survival HR associated with SPN; these ranged from 0.80 to 0.99 (mode 0.87). In patients with Stage IV inoperable pancreatic cancer, the incremental cost-effectiveness ratio versus EN alone was estimated to be £41 350 or £91 501 depending on whether nursing and home delivery costs for EN and SPN were combined or provided separately.
Despite a lack of direct evidence, the results of the literature review demonstrate that SPN may provide important clinical and quality of life benefits to patients with cancer. The potential for any improvement in outcomes in the modelled patient population is very limited, so cost-effectiveness may be greater in patients with less severe disease and other types of cancer.

In diseases where there is a large subjective component, such as celiac disease (CD), patient reported-outcomes (PRO) endpoints are highly relevant. However, there is a gap in knowledge about which PRO endpoints and instruments should be used for clinical trials for treatment of celiac disease.
To identify patient-centered symptom, impact, and health-related quality of life (HRQoL) concepts in CD and relevant PRO instruments, and to gather expert input on concepts and instruments to inform selection of PRO endpoints for use in clinical trials of new CD treatments.
A targeted literature review was conducted to identify symptom, impact, and HRQoL concepts, including those captured in PROs further reviewed against U.S. Food and Drug Administration standards for development and validation as endpoints. US and European clinicians, payers, and a patient advocate (n = 21) were interviewed to assess the identified concepts\' relative importance in measuring treatment benefit and to gauge the value of potential PROs as endpoints for market access/reimbursement.
Thirty-four published studies were identified: 27 elucidated patient-centered concepts and 7 detailed the development or validation of PRO instruments. The Celiac Disease Symptom Diary and Celiac Disease Patient Reported Outcome instrument were deemed most appropriate for use as endpoints; however, each had limitations related to conceptual coverage, evidence for measurement properties, and feasibility for use in clinical trials. Experts reported gastrointestinal symptoms as most important to treat, with extra-intestinal symptoms burdensome from the patient perspective as well. Payers emphasized measuring both frequency and severity of symptoms and targeting patients nonresponsive to the gluten-free diet for treatment.
With emerging treatment options for CD, further work is needed to operationalize PRO symptom endpoints that are meaningful to patients, valued by payers, and acceptable to regulators in demonstrating efficacy.