Risultati per: Cardio-oncology: rivista open access

Introduction
Lafora disease (LD) is an ultrarare fatal progressive myoclonic epilepsy, causing drug-resistant epilepsy, myoclonus and psychomotor deterioration. LD is caused by mutations in EPM2A or NHLRC1, which lead to the accumulation of polyglucosans in the brain and neurodegeneration. There are no approved treatments for LD. VAL-1221 is a fusion protein comprising the Fab portion of a cell-penetrating antibody and recombinant human acid alpha glucosidase, and has demonstrated an ability to clear polyglucosans. We hypothesise that intravenous infusion of VAL-1221 might be able to degrade cerebral polyglucosans and stabilise or improve disease outcomes. The aim of this study is to assess the safety and preliminary efficacy of VAL-1221 in patients with LD.

Methods and analysis
The study is a phase 2, single-arm, open-label, baseline-controlled clinical trial which will be conducted in a single investigational study centre in Italy, namely the sponsor ‘IRCCS Istituto delle Scienze Neurologiche di Bologna—Azienda USL di Bologna’. The study will enrol six genetically confirmed patients with mid- to late-stage LD. The global duration of the study for each participant will be 18 months, including screening period, open-label treatment (12 months) and follow-up period. VAL-1221 20 mg/kg will be administered as an intravenous infusion every week for 3 weeks, then every other week. Patients will undergo full clinical assessments at baseline, at an intermediate and at the end-of-treatment visit. The primary objective is to evaluate the safety. The exploratory efficacy endpoints will be related to epilepsy, neuropsychological and motor functions, global assessment and disease burden, in addition to biomarkers. Statistical analyses will be primarily descriptive.

Ethics and dissemination
The study protocol was approved by the local ethics committee (number 232-2023-FARM-AUSLBO-23020, 22 March 2023). The results of this study will be disseminated by the investigators through presentations at international scientific conferences and reported in peer-reviewed scientific journals.

Trial registration number
European Union Clinical Trials Register (EudraCT 2023-000185-34).

Introduction
The role of sodium-glucose cotransporter 2 inhibitors (SGLT2is) in diabetes treatment is expanding; however, few studies have investigated the efficacy and safety of combining SGLT2is with insulin pump therapy. Notably, there is a scarcity of high-quality, multicentre, clinical trials. Therefore, we aim to conduct a prospective multicentre, randomised, controlled, study to investigate whether treatment of type 2 diabetes patients with continuous subcutaneous insulin infusion (CSII) combined with henagliflozin can reduce the time required for blood glucose control, decrease total insulin requirements, mitigate blood glucose fluctuations and enhance beta-cell function.

Methods and analysis
In this inpatient, open-label, multicentre, randomised, controlled trial, 200 patients with type 2 diabetes who have not received hypoglycaemic drugs will be randomly allocated at a 1:1 ratio to either the henagliflozin combined with CSII group or the CSII group. The efficacy and safety of treatment in both groups will be compared. We will use a real-time continuous glucose monitoring system for blood glucose monitoring. The primary aim of this study is to compare the time (% time in range (TIR)) in the range of 3.9~10.0 mmol/L blood glucose between the two treatment groups. The secondary outcome measures will include comparisons of the two treatment groups with respect to the (a) time at TIR >70%; (b) mean amplitude of glycaemic excursions; (c) time below range; (d) total insulin dosage; and (e) time above range.

Ethics and dissemination
This study was approved by the Ethics Committee of the First Affiliated Hospital of Guangxi Medical University and is to be conducted in accordance with the Declaration of Helsinki and Good Clinical Practice. The study will be disseminated through peer-reviewed publications and conference presentations.

Trial registration number
NCT05677334.

Introduction
First steps for knee osteoarthritis (OA) is a cluster randomised implementation trial examining the effect of an educational reminder message included in knee X-ray reports on the proportion of people subsequently referred to exercise professionals for their knee OA. Evaluating the processes supporting the completion of the study and the efficacy of the reminder message is essential to interpreting the outcomes of the study and aiding translation into practice.

Methods and analysis
We will conduct a concurrent process evaluation throughout the current study using a previously published framework for examining cluster randomised trials. This framework divides processes into those occurring at the cluster level and those at the target population level. For the current study, the cluster level is within radiology clinics. The target population is people with newly diagnosed radiologically evident, structural knee OA. A mixed methods design, incorporating survey data, administrative records, field notes and semi-structured interviews with representatives from radiology clinics and people with knee OA, will evaluate these processes. The focus of the evaluation will be recruitment and response processes of the radiology clinics and delivery and response processes for the people with knee OA. We will also describe the context and explore how the nudge theory of behavioural change influences the outcome of the study.

Ethics and dissemination
The study protocol, inclusive of the process evaluation, was approved by Macquarie University Human Research Ethics Committee (#520221190343842). Findings will be disseminated through national and international conferences, national industry stakeholders and patient advocacy groups to reach all levels of healthcare. Staff at radiology clinics and people with knee OA involved in interviews provide written, informed consent to participate in the process evaluation. Specific findings will be incorporated into training modules aimed at radiology clinics and will be developed by our industry partners.

Trial registration
Prospectively registered with the Australian New Zealand Clinical Trials Registry (ACTRN12622001414707p). Registration occurred in December 2022

Palliative care is not new. It evolved in the 1960s and 1970s before many other medical specialties, such as medical oncology, emergency medicine, and critical care medicine. Although it would be unimaginable to conceive of hospitals, cancer centers, or universities without these 3 specialties, for palliative care, the road toward recognized specialty and adoption by organized medicine was much more difficult, at least in part because of its origins in community hospices and palliative care teams rather than major academic centers and its focus on care of the person rather than more traditional biomedical issues.

This Viewpoint discusses whether the federal EMTALA act requires that hospitals provide abortions when needed to protect the health of the pregnant person, even if prohibited by state law.

The villanelle is a highly structured, repeating and rhymed poetic form believed to have originated as song lyrics to accompany dancing in Renaissance France (from where its name derives). It consists of tercets with the first and final lines of the first stanza repeated in alternating fashion as the final lines of subsequent stanzas. These repeated lines, or refrains, rhyme with each other as well as with the initial line of each stanza; the middle lines of each stanza also rhyme. The enchanting, almost dizzying effect of these formal requirements is evident in “Chimeric Transplant Villanelle.” The dance of this poem, given its medical context of bone marrow transplantation, is as much interpersonal as it is melodious, as the sonic repetitions express a sense of vexed closeness between its physician-speaker and the patient. Inexorably intertwined by mortal illness and its treatment, through the villanelle they negotiate both their shared yet distinct emotions and the limits of healing. “You are not me, you are not mine,” the speaker begins, an insistence on separate selves that ironically is also the invitation to care; as the line is repeated through the poem, its attempt to maintain boundaries through the increasing intensity of side effects that become mutual experience (“We’re losing hair and teeth and mind”) is tested. Whether the music’s metaphor evokes more infusion cycles or oncology rounds’ repetitiveness, ultimately we feel both the resounding beauty of the therapeutic alliance here, and the reluctance, however necessary, to acknowledge its borders—and that it must end.

New England Journal of Medicine, Ahead of Print.

Stroke, Ahead of Print. BACKGROUND:Timely revascularization in acute arterial ischemic stroke (AIS) is paramount for optimal outcomes. However, factors causing treatment delays in pediatric AIS remain understudied. We investigated determinants affecting the time from symptom onset or last-known-well to the start of recanalization treatment in pediatric AIS.METHODS:We conducted an ancillary analysis of the French KID-CLOT study (The National Retrospective Study of Recanalization Treatments in Pediatric Arterial Ischemic Stroke), considering patients with pediatric AIS receiving recanalization treatments (IV thrombolysis IVT and mechanical thrombectomy) from 2015 to 2018. The study assessed prehospital triage’s impact, direct versus transferred admissions, and unit type (pediatric versus adult) on treatment delay and clinical outcomes using modified Rankin Scale at 1 year.RESULTS:Among 68 patients (median age, 11 [IQR, 4–16]; initial PedNIHSS, 13 [IQR, 7–19]), treatment modalities were IVT (n=31), and mechanical thrombectomy (n=23), and IVT+mechanical thrombectomy (n=14). Prehospital triage significantly reduced last-known-well to treatment delay (overall, 229 versus 270 minutes;P=0.01), most notably for and mechanical thrombectomy (P

Annals of Internal Medicine, Ahead of Print.

Annals of Internal Medicine, Ahead of Print.

JAMA Oncology is committed to publishing influential original research, opinions, and reviews that advance the science of oncology and improve the clinical care of patients with cancer.

This Viewpoint discusses possible outcomes of predictive analytic tool use in charity care determinations: hospital revenue and patient debt.

This cross-sectional study examines the proportion of people who are incarcerated in the US with health problems or pregnancy who used health services, changes in the prevalence of those conditions since 2004, and the association between their state’s standard prison co-payment and care receipt in 2016.

Circulation, Volume 150, Issue 14, Page 1072-1074, October 1, 2024.