Tag: 29/9/25

Categories : Cancer Neurology PaediatricsTags : Leave a comment

Combination of Diet and Medication Reprograms Paediatric Neuroblastoma

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Credit: National Cancer Institute

Researchers at Children’s Hospital of Philadelphia (CHOP) found that combining a specialised diet with an approved medication interrupts the growth of high-risk neuroblastoma, a deadly paediatric cancer, by reprogramming tumour behaviour. The findings were published in the journal Nature.

Neuroblastoma originates from primitive cells meant to form nerve tissues but that remain “undifferentiated,” indicating cancer cells that haven’t specialized, often suggesting a more aggressive and unfavourable prognosis. These tumours rely on a steady supply of chemicals called polyamines that are essential for rapid cell growth and tumour progression. A medicine called difluoromethylornithine (DFMO) was approved by the Food and Drug Administration (FDA) to treat children with high-risk neuroblastoma, as DFMO blocks polyamine production. However, researchers sought to improve the effectiveness of the drug by using it at high doses and combining it with a diet that is depleted of the nutrients used by the body to make polyamines (arginine). This two-step approach was anticipated to lower polyamines substantially more than low dose DFMO alone.

“Our findings show that this treatment reduced polyamines in tumours to roughly 10% of their usual levels. This reduction greatly slowed tumour growth, and in many cases, completely eliminated the tumours,” said Michael D. Hogarty, MD, a lead author and an Attending Physician in the Division of Oncology at Children’s Hospital of Philadelphia. “Notably, the treatment altered the way the tumour cells make proteins, making it harder for them to grow and easier for them to mature, or differentiate.”

Hogarty and his team used a preclinical model to mimic MYCN-driven neuroblastoma, directly addressing the strong association between extra MYCN gene copies and aggressive neuroblastoma with poor prognosis. Animal models with tumours were divided into groups: one fed a normal diet and the other lacking amino acids for polyamine production. Each group either received DFMO in their drinking water or did not. The special diet or DFMO alone partially lowered polyamines and extended survival, but the combination had the most significant impact on tumours due to the profound polyamine depletion it caused.

The researchers plan to conduct additional preclinical studies, followed hopefully by clinical trials in children to determine the safety and efficacy of targeting this specific metabolic dependency of neuroblastoma cells. By complementing existing treatments, they hope to substantially improve patient outcomes, and because the therapy targets polyamines it may be effective in many other types of cancer that have frequent MYC gene activation. 

Source: Children’s Hospital of Philadelphia

Categories : Antibiotics PaediatricsTags : Leave a comment

‘Alarming’ Rise in Newborn Babies with Antibiotic-resistant Infections, Researchers Find

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Photo by Christian Bowen on Unsplash

Researchers are calling for an urgent overhaul of diagnostic and treatment guidelines for infections in newborn babies, after a University of Sydney-led study revealed frontline treatments for sepsis are no longer effective to treat the majority of bacterial infections. 

The study, published in The Lancet Regional Health – Western Pacific, analysed almost 15 000 blood samples collected from sick babies in 2019 and 2020 at 10 hospitals across five countries in Southeast Asia, including Indonesia and the Philippines. 

It found that most infections were caused by bacteria unlikely to respond to the currently applied WHO recommended treatments. These were developed using data from high-income countries, instead of using localised data which could be more accurate and therefore effective. 

“Our study highlights the causes of serious infections in babies in countries across Southeast Asia with high rates of neonatal sepsis, and reveals an alarming burden of AMR that renders many currently available therapies ineffective for newborns,” said senior author Associate Professor Phoebe Williams, a Senior Lecturer and NHMRC Fellow in the Sydney School of Public Health.

“Guidelines must be updated to reflect local bacterial profiles and known resistance patterns. Otherwise, mortality rates are only going to keep climbing.”

The problem is further compounded by a lack of new antimicrobial medications in development for infants and babies, added co-author Michelle Harrison, PhD candidate and Project Coordinator of NeoSEAP in the Sydney School of Public Health. 

“It takes about 10 years for a new antibiotic to be trialled and approved for babies,” Harrison said.

“With so few new drug candidates in the first place, we need a significant investment in antibiotic development.”

Gram-negative bacteria responsible for 80% of infections

For the samples which tested positive for fungal or bacterial infections, the team analysed whether they were caused by gram-positive or gram-negative bacteria – referring to the structure of the bacteria’s cell wall which influences how likely it is to develop and acquire antibiotic resistance. 

Gram-negative bacteria like E. coli, Klebsiella and Acinetobacter were responsible for nearly 80% of infections and are more likely to develop (and spread) antibiotic resistance.

“These bugs have long been considered to only cause infections in older babies, but are now infecting babies in their first days of life,” said Associate Professor Williams. 

When treating babies, doctors don’t have time to wait for lab tests to confirm the exact cause of the infection, and often make an educated guess from published data, most often based on high-income populations, to guide treatment. These tests are also frequently delayed or falsely negative due to the difficulty of collecting blood samples.

Harrison explained that the findings showcase the importance of locally relevant data to guide routine medical decision-making.

“We need more region-specific surveillance to guide treatment decisions. Otherwise, we risk reversing decades of progress in reducing child mortality rates,” she said.

“Our results also revealed fungal infections caused nearly one in 10 serious infections in babies – a much higher rate than in high-income countries. 

“We need to ensure doctors are prescribing treatments that have the best chance at saving a baby’s life.”

Source: University of Australia

Categories : Injury & TraumaTags : Leave a comment

International Healthcare Workers Report on War Related Injuries Among Civilians in Gaza

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Findings suggest patterns of harm that exceed those reported in previous modern-day conflicts and provide critical insights to tailor humanitarian response

Photo by Mohammed Ibrahim on Unsplash

A British led study published by The BMJ provides detailed data on the pattern and severity of traumatic injuries and medical conditions seen by international healthcare workers deployed to Gaza during the ongoing military invasion.

Healthcare workers describe “unusually severe” traumatic injuries including complex blast injuries, firearm related injuries, and severe burns. Many respondents with previous experience of conflicts reported that the pattern and severity of injuries in Gaza were greater than those they had encountered in previous warzones.

It’s thought to be the first study to provide such detailed data from frontline clinicians during the conflict, which the authors say offers critical insights into the injuries and conditions most relevant to immediate management, rehabilitation, and long term health planning.

Since October 2023, Gaza has faced high intensity Israeli bombardment and ground military incursions. Publicly reported figures show that more than 59,000 Palestinians have been killed and over 143,000 wounded during the conflict, but other analyses suggest these figures may be higher.

To address this gap, healthcare workers were invited to take part in a survey about the nature and pattern of injuries and medical conditions they managed while in Gaza, ranging from explosive and firearm injuries to infections and chronic diseases.

A total of 78 doctors and nurses completed the survey using logbooks and shift records between August 2024 and February 2025, within 3 months of their deployment end date.

Participants represented 22 non-governmental organisations (NGOs) and were mainly from the US, Canada, the UK and European Union member states working in trauma surgery, emergency medicine, paediatrics, or critical care and anaesthesia.

Almost two thirds (65%) had prior experience working in an active conflict zone and their deployment to Gaza ranged from 2-12 weeks, contributing to a total of 322 weeks of frontline clinical care.

Overall, 23,726 trauma related injuries and 6,960 injuries related to weapons were reported. The most common traumas were burns (4,348, 18%), leg injuries (4,258, 18%), and arm injuries (3,534, 15%).

There were 742 obstetric cases reported, of which more than a third (36%) involved the death of the fetus, mother or both. Psychological trauma was also reported, with depression, acute stress reactions, and suicidal ideation being most common.

Some 70% of healthcare workers reported managing injuries across two or more anatomical regions and experiences of mass casualties were widespread, with 77% reporting exposure to 5-10 events and 18% managing more than 10 such scenarios.

Explosive injuries accounted for the majority of weapon related trauma (4,635, 67%), predominantly affecting the head (1,289, 28%) whereas firearm injuries targeted the legs (526, 23%).

The most common general medical conditions reported were malnutrition and dehydration, followed by sepsis and gastroenteritis. Healthcare workers also reported 4,188 people with chronic disease requiring long term treatment.

In free text responses, healthcare workers frequently described injuries as unusually severe, including multi-limb trauma, open skull fractures, and extensive injuries to internal organs. Severe burns were also emphasised, particularly in children.

Respondents with previous experience of deployment in other conflict zones commented that the severity and pattern of injuries encountered in Gaza were greater than those they had previously managed.

Despite the strength of this data, the authors acknowledge limitations. For instance, relying on logbooks and shift records inevitably introduces uncertainty, especially during periods of large influxes of injured people. Nor can they rule out the possibility of duplication, although further analyses indicated minimal impact on overall estimates.

However, they say the volume, distribution, and severity of injuries seem to indicate patterns of harm that exceed those reported in previous modern-day conflicts.

“These findings highlight the urgent need for resilient, context specific surveillance systems, designed to function amid sustained hostilities, resource scarcity, and intermittent telecommunications, to inform tailored surgical, medical, psychological, and rehabilitation interventions,” they conclude.

Source: BMJ Group

Categories : Cardiovascular DiseaseTags : Leave a comment

Semaglutide and Tirzepatide Reduce Heart Failure Risk by More than 40%

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Right side heart failure. Credit: Scientific Animations CC4.0

Treatment with semaglutide or tirzepatide can reduce health risks for patients with heart failure by more than 40%. These finding come from a study by researchers at the Technical University of Munich (TUM). The medication can drastically reduce the risk of being hospitalised for heart failure or dying.

Semaglutide and related medications are widely used to treat diabetes and obesity. Their potential effects beyond weight loss are now being closely studied. Still, professional societies and regulatory authorities have urged caution. With regard to treating heart failure, cardiology societies have noted that the existing evidence base remains limited.

“Together with our colleagues at Harvard Medical School, we have created a solid evidence base for using these weight-loss medications in heart failure,” says Professor Heribert Schunkert, Director of the Department of Cardiovascular Diseases at the TUM University Hospital German Heart Center. “In patients with heart failure with preserved ejection fraction, both drugs have shown a clear protective effect that supports their use. Our analysis of around 100 000 patients provides a robust basis for reassessing an indication expansion and new indication approval in heart failure.“

The study focused on heart failure with preserved ejection fraction or HFpEF, in which the heart’s ability to pump remains intact but the stiffened heart muscle does not fill properly with blood. HFpEF affects more than 30 million people worldwide. To date, there are only a few effective treatment options for this form of the disease.

Large-scale database study provides solid evidence for use in heart failure

Published in JAMA, the study examined the effects of the drugs semaglutide and tirzepatidein patients with this specific form of heart failure. The researchers analysed three national US insurance claims databases. Their models first confirmed findings from earlier trials in patients with obesity or diabetes, then extended the analysis to populations excluded from clinical trials and to additional outcomes, such as hospitalisation for heart failure and mortality.

Treatment with both drugs led to a more than 40% reduction in the risk of hospitalisation for heart failure or death compared with another diabetes drug that had shown no effect on heart failure outcomes in previous studies.

“Currently, HFpEF can be treated with a few drugs only. At the same time, an increasing number of patients suffers from obesity and diabetes, which further worsens outcomes. In Germany, heart failure is the leading cause for hospitalizations and a major driver of health care expenditure. Our study shows that these drugs are highly effective, which expands treatment options and could prevent many hospital admissions,” says Dr Nils Krüger, resident physician at the TUM University Hospital German Heart Center and lead author of the study.

Data-driven approaches to drug approval

The study drew on patient populations nearly 20 times larger than those in traditional clinical trials. This allowed researchers to capture clinical practice and demonstrate that the benefits seen in pivotal trials also apply to broader patient groups. “The future belongs to such data‑driven approaches – alongside traditional trials, they can help ensure that findings from basic research feed into patient care more quickly,” explains Prof Schunkert.

From the researchers’ perspective, such analyses are also becoming increasingly relevant for Germany. The Health Data Utilization Act provides that anonymized health insurance data will in future be made systematically available for such research projects, while strictly protecting sensitive personal data. “We use these large data to investigate the safety and effectiveness of medications in clinical practice,” says Dr Krüger.

Source: Technical University Munich

Categories : Lab Tests and ImagingTags : Leave a comment

Pocket Ultrasound Reduces Hospital Stays for Patients with Shortness of Breath

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Credit: Rutgers Health/RWJBarnabas Health

When hospitalised patients struggle to breathe, doctors typically reach for their stethoscopes, but results from a clinical study in JAMA Network Open suggest they should diagnose the problem with portable ultrasounds instead.

The study, by Rutgers Universitty and RWJBarnabas Health, found initial exams with portable ultrasounds led to better diagnoses, shorter hospital stays and big cost savings. However, the findings revealed a need for additional training and workflow integration to help clinicians transition from traditional tools to this promising new technology.

“The study clearly shows that ultrasound is the superior diagnostic technology, even for long-time stethoscope users who get a few hours of ultrasound training,” said senior study author Partho Sengupta, Henry Rutgers professor and chief of cardiology at Rutgers Robert Wood Johnson Medical School (RWJMS), chief of cardiology at Robert Wood Johnson University Hospital (RWJUH) and member of the RWJBarnabas Health Medical Group.

The explanation here is simple. Ultrasound gives you more information, and more concrete information, about what’s going on

Partho Sengupta, Henry Rutgers professor

The study enrolled 208 patients admitted with shortness of breath to Robert Wood Johnson University Hospital in New Brunswick. About half of them underwent diagnosis via point-of-care ultrasound devices that attach to smartphones. The rest underwent diagnosis via existing standards of care. 

Initial diagnosis with ultrasound trimmed a patient’s average length of hospital stay from 11.9 days to 8.3 days. In all, initial diagnosis with ultrasound saved 246 bed-days and about $751 000 in direct costs across the cohort, while 30-day readmissions were similar between groups.

“The explanation here is simple. Ultrasound gives you more information, and more concrete information, about what’s going on,” Sengupta said.  “When clinicians can see fluid in the lungs, a failing heart or a stiff inferior vena cava in minutes, they can target therapy sooner or rule out a cardiopulmonary cause and look elsewhere.”

To keep things simple and encourage buy-in, scans focused on a handful of cardiac views and a six-zone lung sweep. The exam was designed to be quick and binary: signs of congestion or not, systolic function reduced or not.

The study provided several hours of ultrasound training to participating hospitalists (doctors who oversee and coordinate hospital patient care) so that each could perform and interpret the ultrasound exam in 10 to 15 minutes. 

Nevertheless, most hospitalists who underwent the training let sonographers perform the exams and cardiologists interpret them. Only 20% of patients received an ultrasound diagnosis from one of the hospitalists.

Sengupta said time pressure on rounds and a lack of incentives make it hard to add a 10-minute procedure, even if it changes care.

“This is consistent with what we observe in day-to-day clinical practice,” Sengupta said. “Although the ultrasound probe fits in your pocket and attaches to the back of a smartphone, its use in clinical settings remains inconsistent. This study overcame those barriers by leveraging a multidisciplinary framework.”

Kameswari Maganti, professor of cardiology at RWJMS and section chief for non-invasive cardiology at RWJUH, led the image interpretation with the sonography team that worked closely with the RWJUH hospitalist team, led by RWJMS faculty Catherine Chen and Payal Parikh, as well as the engineering and data science team, headed by Naveena Yanamala. 

“This coordinated team effort was key to developing and delivering a streamlined protocol that significantly reduced hospital stays and healthcare costs,” Sengupta said.

The researchers reported that ultrasound findings altered medical decisions in roughly a third of cases, including new diagnoses and changes in therapy. They also noted that longer-stay patients appeared to benefit the most, a hint that ultrasound-guided triage and treatment may pay particular dividends when cases are complex.

As with most single-center implementations, caveats apply. The model relied on trained sonographers and cardiology reads, which may not be available everywhere. Broader studies across multiple hospitals will be needed to confirm the cost and length-of-stay benefits, and to test strategies that make adoption stick.

Still, the argument for seeing more and guessing less is gaining ground. A bedside view of the heart and lungs, delivered early in admission, may help the right treatments arrive sooner.

Source: Rutgers University