Risultati per: Cardio-oncology: rivista open access

Objectives
Many patients presenting with acute exacerbations of chronic obstructive pulmonary disease (AECOPD) in primary care do not benefit from antibiotics. Excessive use wastes resources, promotes antimicrobial resistance and can harm patients.

Design
We conducted a within-trial economic evaluation, using a UK National Health Service perspective, as part of the multicentre, parallel-arm, open, individually randomised, controlled PACE trial.

Setting
Participating general practices in primary care.

Participants
PACE included 324 and 325 consenting participants presenting with AECOPD in the usual-care and CRP-guided groups, respectively.

Intervention
We assessed the cost-effectiveness (CE) of a C-reactive protein point-of-care-test (CRP-POCT) in addition to usual clinical assessment to guide antibiotic prescribing for AECOPD in primary care.

Primary and secondary outcome measures
A cost-effectiveness analysis (CEA) of incremental cost per 1% antibiotic consumption reduction at 4 weeks and a cost-utility analysis (CUA) at 6 months were performed, based on a modified intention-to-treat population. Sensitivity analyses assessed the impact of uncertainty on the results. CE acceptability curves represent the probability of CRP-POCT being cost-effective at different willingness-to-pay (WTP) thresholds.

Results
Both groups had similar clinical outcomes, but a 20% absolute reduction in antibiotic consumption was observed in the CRP-guided group. CRP-POCT costs of £11.31 per test were largely offset by savings in healthcare resource use related to COPD. The mean incremental CE ratios of CRP-POCT were £120 per 1% absolute reduction in antibiotic consumption at 4 weeks and £1054 per quality-adjusted life-year (QALY) gained at 6 months. Sensitivity analysis showed that the CEA results were most affected by changes in healthcare costs, while CUA was sensitive due to marginal differences in costs and outcomes. There is a 73% probability of CRP-POCT being cost-effective at WTP ≤£20 000 per QALY gained.

Conclusion
CRP-POCT is a cost-effective intervention for safely reducing antibiotic consumption in patients with AECOPD.

Trial registration number
ISRCTN24346473

This study identifies state policies authorizing law enforcement access to prescription drug monitoring program data and discusses the characteristics of those policies, particularly in states with laws banning gender-affirming care.

Introduction
The revision of International Federation of Gynaecology and Obstetrics staging in 2018 with recommendations to include cross-sectional imaging and a separate stage for node positive disease have opened a lot of uncertainties in implementing the correct treatment approach in these patients. While studies have suggested higher chances of occult para-aortic lymph node (PALN) even with advanced imaging, especially in pelvic node positive disease which tend to recur after pelvic radiation therapy. This study intends to study these patients and isolate the subset who will benefit most from elective PALN irradiation.

Methods and analysis
This is an ongoing multicentric phase III randomised controlled trial with a sample size of 274 subjects in two arms (137 in each arm) to determine the superiority of limited elective para-aortic irradiation compared with no irradiation. Arm one includes radiation to the lower PALN and pelvis; Arm two includes radiation to the pelvis. Concurrent chemotherapy followed by brachytherapy is standard in both arms. Patients with cervical cancer and radiologically positive pelvic LNs aged >18 years and

Introduction
Non-alcoholic fatty liver disease, now known as metabolic dysfunction-associated steatotic liver disease (MASLD), is a phenotype of the metabolic syndrome in the liver and is clearly associated with metabolic abnormalities such as hyperglycaemia and dyslipidaemia. Although the prevalence of MASLD is increasing worldwide, there is currently no consensus on the efficacy and safety of the drugs used to treat MASLD/metabolic dysfunction-associated steatohepatitis (MASH). Pemafibrate, a selective peroxisome proliferator-activated receptor alpha modulator, was designed to have higher peroxisome proliferator-activated receptor alfa (PPARα) agonist activity and selectivity than existing PPARα agonists, and in development trials, without increasing creatinine levels, lipid parameters and alanine aminotransferase (ALT) were significantly improved. Thus, pemafibrate may effectively ameliorate the pathogenesis and metabolic abnormalities in MASLD/MASH. In this trial, we evaluated the efficacy and safety of pemafibrate in patients with MASLD/MASH.

Methods and analysis
This trial was designed as an open-label, three-arm, randomised controlled study. After obtaining informed consent, patients aged 20–80 years who met the selection criteria were enrolled. Patients were randomised to receive pemafibrate 0.4 mg/day, 0.2 mg/day or fenofibrate (n=120 per group). The duration of treatment was 48 weeks. The primary endpoint was a change in ALT levels after 24 weeks of administration. Secondary endpoints included changes from baseline in liver fibrosis markers (fibrosis-4 index, type IV collagen 7s, enhanced liver fibrosis and Mac-2 binding protein glycosylation isomer) at 48 weeks as well as changes in liver fat mass and liver stiffness measured by MRI and ultrasound (US) at centres equipped with MRI and US capabilities.

Ethics and dissemination
Ethical approval was obtained from the Yokohama City University Certified Institutional Review Board before participant enrolment (CRB20-014). The results of this study will be submitted for publication in international peer-reviewed journals and the key findings will be presented at international scientific conferences. Participants wishing to understand the results of this study will be contacted directly on data publication.

Trial registration number
This trial was registered in the Japan Registry of Clinical Trials (number: jRCTs031200280).

Protocol version
V.1.9, 23 November 2023

Introduction
Necrotising enterocolitis (NEC) of the intestine of preterm infants leads to the risk of abdominal surgery, short bowel syndrome, neurodevelopmental disorders and death. Although the risks of NEC and its complications have been recognised in many countries, few countries have established NEC-specific registries to continuously monitor its aetiology and prognosis. In China, the understanding of risk factors and prognosis of NEC is incomplete, characterised by a lack of evidence from prospective and multicentre studies. Therefore, we designed a multicentre, prospective, open observational cohort study with the aim of investigating the risk factors and prognosis of NEC in a real-world setting in Shenzhen, Guangdong Province, by constructing an online registry of children with NEC and a bank of biospecimens.

Methods
This is a prospective, multicentre, open observational cohort study. From June 2024 to June 2028, more than 600 patients with NEC from 15 tertiary hospitals in Shenzhen, Guangdong Province, will be enrolled in the study. By constructing an online registry for NEC, clinical data will be collected during the prenatal and hospitalisation periods. Prospectively, biospecimens will be collected during the period of suspected NEC, at the time of confirmed NEC, and at the time of confirmed severe NEC, and filed in the online registry system. Follow-up data will include postdischarge healthcare needs, growth patterns measures, eye or vision examinations, cranial MRI findings, brainstem auditory evoked potentials or automated auditory brainstem responses, and the Chinese Griffith Developmental Scale at corrected age 18–24 months. Follow-up results were likewise recorded in an online registry system. Hospitalisation outcomes, including severe NEC, somatic growth and survival status, will be collected at discharge. Follow-up outcomes will include loss to visit, survival status, somatic growth measures and severe neurodevelopmental deficits at corrected age 18–24 months. This study will enhance our overall understanding of the risk factors and outcomes of NEC, ultimately helping to reduce the incidence of neonatal NEC and its poor prognosis.

Ethics and dissemination
Our programme has received approval from the Ethics Committee for Scientific Research Projects of the Longgang District Maternity & Child Healthcare Hospital in Shenzhen City (ethics approval number: LGFYKYXMLL-2024-47-01). We anticipate presenting our findings at various national conferences and submitting them to peer-reviewed paediatrics journals.

Trial registration number
ChiCTR2400085043.

Objective
Real-time access to test results on patient portals can have advantages and disadvantages for patients. It confronts patients with a complicated decision, namely whether to consult results before the medical consultation. To gain a deep understanding of patients’ decision-making processes, we unravelled three discourses about real-time access to test results, each of which articulates a different set of values, assumptions and arguments. Our research question was what patient discourses on real-time access to test results can be distinguished?

Design
We conducted discourse analysis on 28 semistructured interviews.

Setting
Interviews were conducted with patients who had (no) experience with real-time access to test results. Our participants were treated in different hospitals, and therefore, used different portals since Dutch hospitals can choose from suppliers for their patient portals.

Participants
Patients with experience (n=15) and without experience (n=13) of real-time access to test results on a patient portal.

Results
We identified three discourses: (1) real-time access as a source of stress, which highlighted how real-time access could cause stress due to the complexity of deciding whether to access test results, the incomprehensibility of medical language and the urge to repeatedly check if test results were available, (2) anxiety reduction through real-time access showed how real-time access can reduce stress by reducing waiting times and (3) real-time access for self-management showed how real-time access can give patients an opportunity for self-management because they can make informed decisions and are better prepared for the medical consultation.

Conclusion
Our study shows the plurality in opinions on real-time access, which helps in forming different strategies to inform and support patients in order to realise optimal use of real-time access.

Introduction
Bronchopulmonary dysplasia (BPD) in very preterm infants (VPIs) has adverse long-term outcomes and affects the quality of survival. There are no registry studies on BPD in VPIs in China. Our aim was to conduct a prospective, multicentre, open, longitudinal, observational cohort study to investigate the epidemiological characteristics, diagnosis, treatment, and short-term and long-term outcomes of BPD in a real-life setting in China and lay the grounds for establishing a nationwide registry with clinical data and biological specimens.

Methods
This study aims to recruit a minimum of 2000 VPIs and start research in January 2024 in Shenzhen, China. We will collect clinical data from the beginning of the life of VPIs and follow them up to 3 years old. Short-term outcomes, such as the incidence of BPD, necrotising enterocolitis, retinopathy of prematurity, intraventricular haemorrhage and porencephalic ventricular leukomalacia, as well as the cost of hospitalisation, are the major variables of concern. Bayley-III Scale assessment, gross motor function and pulmonary function evaluation will be performed at the age of correction, that is, 18–24 months and 30–36 months. The follow-up outcomes include loss to follow-up, survival status, moderate-to-severe neurodevelopmental deficits and severe respiratory complications. Cord blood, peripheral blood, tracheal aspirate, faeces and urine from VPIs, as well as mother’s milk, will be collected and stored at –80°C. All the data will be registered, stored and managed in a cloud-based database. This knowledge will be useful for establishing diagnostic criteria and predictive models for BPD in the Chinese population.

Ethics and dissemination
Our protocol has been approved by the Medical Ethics Committee of Shenzhen People’s Hospital (LL-KY-2023174-02) and the local ethics committee of each participating centre. Our goal is to present our findings at national conferences and in peer-reviewed paediatric journals.

Trial registration number
Chinese Clinical Trial Registry (ChiCTR2400081615).

Introduction
Efforts to improve access to essential medicines globally include the implementation and assessment of national medicine policies. Although existing assessment tools may identify gaps, they do not link these to health system functions and policy priority areas in a way that facilitates decision-making. This systematic review aims to identify and map out existing indicators assessing essential medicines policies; assess their strengths and limitations and understand the parameters required to develop a new instrument for assessing access to medicines in a way that better supports decision makers in identifying and addressing the gaps in their national medicine policies.

Methods and analysis
This systematic review is guided by the updated Preferred Reporting Items for Systematic Review and Meta-Analysis Protocol. Eligible studies will be identified across scholarly databases (MEDLINE, Embase and Scopus) and grey literature using relevant subject headings and text words. Title, abstract screening and full-text screening will be conducted by two independent reviewers. Indicators or assessment tools focused on the performance of national medicine policies with respect to improving access to essential medicines will be eligible. Data will be extracted using a pretested data abstraction form. Findings will be reported in the form of a descriptive narrative. This study will provide insights into the relevance of existing indicators and tools for assessing access to essential medicines policies. This review will also discuss the opportunities for the development of a new instrument for the assessment of national medicines policies in a way that better supports decision makers to improve access to essential medicines.

Ethics and dissemination
This systematic review does not require ethics approval as it only aims to synthesise evidence from the literature. Findings will be disseminated to key health stakeholders including researchers and global and public health organisations and published in a peer-review journal. This protocol has been registered on PROSPERO (CRD42023493052).

Objectives
The objective of this study is to examine the trends in geographical inequality of opportunity in maternal health services in India considering the Every Newborn Action Plan (ENAP) 2025 coverage targets.

Setting
India.

Participants
Women in the National Family Health Survey (NFHS)—NFHS-4 (2014–2015) and NFHS-5 (2019–2021).

Primary and secondary measures
District-level coverages of 4+antenatal care (ANC) visits, institutional delivery with skilled birth attendant, postnatal care (PNC) services within 48 hours of delivery, continuum of care (CoC) services for women with most recent live births were considered. Human Opportunity Index (HOI) documented the opportunities for equitable access to these services, ranging from 0 (high inequality) to 100 (universal access). HOI was compared between the survey rounds and wealth index (WI) quintiles that the women belonged to.

Results
Coverages of 4+ANC visits, institutional delivery, PNC and CoC in India increased by 22.8% (95% CI 22.1% to 23.5%), 12.6% (95% CI 12.2% to 12.9%), 28.5% (95% CI 28.0% to 29.0%) and 38.6% (95% CI 37.6% to 39.6%) between NFHS-4 and NFHS-5, respectively. The HOI for 4+ANC visits was 48.4 in NFHS-5, ranging from 11.3 to 92.4 in states and from 31.1 to 70.5 for WI I–V. The HOI for institutional delivery was 80.4 in NFHS-5, ranging from 37.4 to 99.7 in the states and from 21.0 to 100 for WI I–V. The HOI for PNC services was 73.5 in NFHS-5, ranging from 37.5 to 95.6 in the states and from 61.2 to 88.3 for WI I–V. The HOI for CoC was 37.1, ranging from 6.5 to 88.8 in the states and from 19.8 to 62.7 for WI I–V for CoC in NFHS-5.

Conclusion
Though significant improvements in the geographical inequity of maternal health services have been made in India, the geographical inequity for 4+ANC visits coverage lags significantly behind resulting in CoC coverage inequity to achieve the ENAP targets for these services.

Objective
The Person Empowered Asthma Relief (PREPARE) study found that as-needed inhaled corticosteroid (ICS) supplementation combined with participants’ usual controller and rescue therapy reduced asthma exacerbations for Black and Hispanic/Latinx individuals. We aimed to determine whether treatment assignment to the intervention group (Patient Activated Reliever-Triggered ICS (PARTICS)) versus the control group (usual care) influenced controller therapy based on clinicians’ written prescriptions.

Design
Secondary data analysis of electronic health record data of a pragmatic, open-label, patient-level randomised trial.

Setting
Practices treating asthma.

Participants
PREPARE study participants— Black and Hispanic/Latinx individuals with asthma.

Interventions
Effects of adding ICS to rescue therapy among black and Hispanic adults with moderate-to-severe asthma.

Outcome measures
For PARTICS therapy impact on patients, each month is the 28-month period (12 months prior to enrolment, the month of enrolment and 15 months after enrolment), a patient was assigned to a controller step based on a six-step classification scheme. A linear mixed effect spline model was completed for before and after enrolment data to determine controller changes over a 28-month period between the two study arms.

Results
This analysis included 713 participants. Of these, 49.1% were usual care patients and 50.9% were PARTICS patients. Throughout the study, the majority of patients changed asthma controller medications in both arms. By the end of the study, the usual care patients were at a significantly higher asthma controller medication step (0.20 step higher) than the PARTICS patients.

Conclusions
Clinicians’ prescribing patterns showed significant changes over time. Compared with usual care patients, PARTICS patients were on lower doses of asthma controller medications by the end of the study.

Trial registration number
NCT02995733.

Objective
This qualitative study aimed to analyse rectal cancer survivors’ lived experiences to identify facilitators and barriers to support access.

Design
We conducted one-on-one semi-structured interviews and employed thematic analysis to identify key themes and insights.

Setting/participants
Participants included eight rectal cancer survivors and three caregivers recruited at Texas Colorectal Collaborative sites.

Results
Results showed that adequate hospital resources, high health literacy and close connections with clinicians and peers who share similar experiences facilitate survivors’ access to social support. Conversely, ineffective healthcare team communication, financial challenges and low self-motivation hindered access.

Conclusion
Survivorship experiences were shaped by varying degrees of social support access, influenced by internal and external factors. We aim to establish a cross-institutional survivorship support network to address these factors, ensuring equitable access to support services and enhancing survivorship experiences.

Circulation, Ahead of Print. Background: In the phase 3 randomized controlled study, ATTRibute-CM, acoramidis, a transthyretin (TTR) stabilizer, demonstrated significant efficacy on the primary endpoint. Participants with transthyretin amyloid cardiomyopathy (ATTR-CM) who completed ATTRibute-CM were invited to enroll in an open-label extension study (OLE). We report efficacy and safety data of acoramidis in participants who completed ATTRibute-CM and enrolled in the ongoing OLE.Methods: Participants who previously received acoramidis through Month 30 (M30) in ATTRibute-CM continued to receive it (continuous acoramidis), and those who received placebo through M30 were switched to acoramidis (placebo to acoramidis). Participants who received concomitant tafamidis in ATTRibute-CM were required to discontinue it to be eligible to enroll in the OLE. Clinical efficacy outcomes analyzed through Month 42 (M42) included time to event for all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH), ACM alone, first CVH alone, ACM or recurrent CVH, change from baseline in N-terminal pro-B-type natriuretic peptide (NT-proBNP), 6-minute walk distance (6MWD), serum TTR, and the Kansas City Cardiomyopathy Questionnaire Overall Summary score (KCCQ-OS). Safety outcomes were analyzed through M42.Results: Overall, 438 of 632 participants in ATTRibute-CM completed treatment and 389 enrolled in the ongoing OLE (263 continuous acoramidis, 126 placebo to acoramidis). The hazard ratio (HR) (95% CI) for ACM or first CVH was 0.57 (0.46, 0.72) at M42 based on a stratified Cox proportional hazards model (P-value < 0.0001) favoring continuous acoramidis. Similar analyses were performed on ACM alone and first CVH alone, with HRs (95% CI) of 0.64 (0.47, 0.88) and 0.53 (0.41, 0.69), respectively, at M42. Treatment effects for NT-proBNP and 6MWD also favored continuous acoramidis. Upon initiation of open-label acoramidis in the placebo-to-acoramidis arm there was a prompt increase in serum TTR. Quality of life assessed by KCCQ-OS was well preserved in continuous acoramidis participants compared with the placebo to acoramidis participants. No new clinically important safety issues were identified in this long-term evaluation.Conclusions: Early initiation and continuous use of acoramidis in the ATTRibute-CM study through M42 of the ongoing OLE study was associated with sustained clinical benefits in a contemporary ATTR-CM cohort, with no clinically important safety issues newly identified.

Circulation, Ahead of Print. Background: The optimal duration of anticoagulation therapy for patients with cancer and acute low-risk pulmonary embolism (PE) is clinically relevant, but evidence is lacking. Prolonged anticoagulation therapy could have a potential benefit for prevention of thrombotic events; however, it could also increase the risk of bleeding.Methods: In a multicenter, open-label, adjudicator-blinded, randomized clinical trial at 32 institutions in Japan, we randomly assigned patients with cancer and acute low-risk PE of the simplified version of the Pulmonary Embolism Severity Index score of 1, in a 1:1 ratio, to receive either an 18-month or a 6-month rivaroxaban treatment. The primary end point was recurrent venous thromboembolism (VTE) at 18 months. The major secondary end point was major bleeding at 18 months according to the criteria of the International Society on Thrombosis and Hemostasis. The primary hypothesis was that an 18-month treatment was superior to a 6-month treatment in terms of the primary end point.Results: From February 2021 to March 2023, 179 patients were randomized, and after the exclusion of one patient who withdrew consent, 178 were included in the intention-to-treat population: 89 patients in the 18-month rivaroxaban group and 89 in the 6-month rivaroxaban group. The mean age was 65.7 years; 47% of the patients were men, and 12% had symptoms of PE at baseline. The primary end point of recurrent VTE occurred in 5 of the 89 patients (5.6%) in the 18-month rivaroxaban group and in 17 of the 89 (19.1%) in the 6-month rivaroxaban group (odds ratio, 0.25 [95% CI, 0.09–0.72];P=0.01). Among 22 recurrent VTE, 5 patients presented with a symptomatic recurrent VTE; recurrent PE occurred in 11 patients, including 2 with main and 4 with lobar PEs; and recurrent deep vein thrombosis was seen in 11 patients, including 3 with proximal deep vein thromboses. The major secondary end point of major bleeding occurred in 7 of the 89 patients (7.8 %) in the 18-month rivaroxaban group and in 5 of the 89 patients (5.6%) in the 6-month rivaroxaban group (odds ratio, 1.43 [95% CI, 0.44–4.70];P=0.55).Conclusions: In patients with cancer and acute low-risk PE of the simplified version of the Pulmonary Embolism Severity Index score of 1, the 18-month rivaroxaban treatment was superior to the 6-month rivaroxaban treatment with respect to recurrent VTE events.

Introduction
Janus kinase (JAK) inhibitors are an important therapeutic option in the treatment of rheumatoid arthritis, but increase the risk of developing herpes zoster. Although a dry recombinant zoster vaccine (RZV) that can be used under immunosuppressive conditions has recently been developed, its optimal use and appropriate timing in patients scheduled to start JAK inhibitors is still unclear. The present study is designed to clarify the appropriate timing of JAK inhibitor initiation to measure varicella zoster virus (VZV)-specific IgG titers and VZV-specific T cell response in patients with rheumatoid arthritis who start tofacitinib at the first RZV vaccination or at the second one.

Methods and analysis
STOP HZ (Effectiveness and S afe T y O f P rophylactic Recombinant H erpes Z oster Virus Vaccination for Rheumatoid Arthritis Patients with Tofacitinib Treatment) study is a multicentre, open-label, randomised, comparative study in patients with rheumatoid arthritis who are scheduled to start tofacitinib. This study enrols 60 study subjects in 12 sites. Enrolled subjects receive RZV two times on day 1 and week 8 and initiate tofacitinib 5 mg two times a day at the time of their first RZV (day 1, group A) or second RZV (week 8, group B) based on randomisation. The random assignment is performed centrally in a 1:1 ratio. Patients in Group B continue the same treatment until the start of tofacitinib treatment. Primary endpoint is VZV-specific IgG antibody titers at week 12 compared with those at baseline in each group. Secondary endpoints include comparison of VZV-specific IgG antibody between the groups, changes in disease activity of rheumatoid arthritis, VZV-specific T cell response and adverse events.

Ethics and dissemination
The study has been approved by the Certified Review Board of Keio (No. 2022008), and conforms to the Declaration of Helsinki and good clinical practice guidelines. Written informed consent is obtained from participants prior to enrolment. The results of this study are planned to be submitted for publishment in relevant peer-review journals.

Trial registration number
jRCTs031230329.

The purpose of this American Gastroenterological Association (AGA) Clinical Practice Update is to facilitate understanding and improve the clinical practice of endoscopic enteral access.

Purpose
The FUPEC (Follow-Up Pre-EClampsia) study aims to investigate the presence and development of cardiovascular risk factors, cardiovascular disease, as well as cardiovascular health following a pregnancy complicated by severe pre-eclampsia.

Participants
The FUPEC study is an open-cohort study conducted within routine care at the FUPEC clinic at Erasmus Medical Center in the Netherlands. This clinic is specifically designed for the cardiovascular follow-up of patients who have experienced severe pre-eclampsia. Women with a history of severe pre-eclampsia are invited to the FUPEC clinic at 6 weeks, 3 months, 1 year and every 2 years thereafter postpartum until they are 50 years of age. Clinical and biochemical data are routinely collected, encompassing pregnancy characteristics and outcomes, anthropometric measurements, cardiovascular risk factors, cardiovascular health scores, carotid intima-media thickness—including vascular age and ambulatory blood pressure measurements. Additionally, blood and urine samples are collected and stored in a biobank.

Findings to date
The first patient was enrolled in April 2011. As of March 2024, a total number of 1268 women have been enrolled in the FUPEC study, with an annual enrolment rate of 100–150 new patients. At inclusion, women had a median age of 33.5 years (IQR 30.1–37.9). At their first FUPEC visit, women were a median of 4.9 months (1.9–29.4) after delivery. At the first visit, the median body mass index was 25.7 (IQR 23.0–29.9) kg/m2, 23.4% of participants were using antihypertensive medication and 6.4% were smoking. Preliminary analyses of 24-hour blood pressure patterns and carotid intima-media thickness have previously been conducted on a subset of the cohort, with details provided in the ‘Findings to Date’ section.

Future plans
The FUPEC cohort serves as a robust clinical data source and biobank that can be used for future studies and collaborative research answering, for example, questions on the aetiology, risk factors and short-term and long-term complications of pregnancies complicated by severe pre-eclampsia. Since the FUPEC cohort is integrated with routine care, there is no strict completion of data collection, allowing for flexible data acquisition.