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8th March 2024
Awarded an OBE in the New Year’s Honours list for services to paediatric surgery, Professor Simon Kenny is a consultant paediatric and neonatal surgeon at Alder Hey Children’s NHS Foundation Trust. He spoke to Saša Janković about how his varied leadership roles feed into one another, his work in advanced minimally invasive paediatric surgery and how addressing health inequalities can have the biggest impact on child health.
The first in his family to go to university, Professor Simon Kennytrained in the UK in Birmingham and Liverpool and in Melbourne, Australia, and has been a consultant paediatric and neonatal surgeon and urologist at Liverpool’s Alder Hey Children’s Hospital since 2003, with a specialist interest in urology and lower gastrointestinal surgery.
An honorary professor in child health, he has research interests including population level data and regenerative medicine, and is also the chair of the Women’s and Children’s Programme of Care in Specialised Commissioning.
If that wasn’t enough to keep him busy, Professor Kenny has also been NHS England’s national clinical director for children and young people since 2019.
As clinical director at Alder Hey Children’s Hospital – one of the biggest centres in Europe for children’s surgery – Professor Kenny played a significant role in redesigning services and moving into the brand new, and subsequently award-winning, Alder Hey in the Park building in 2015 (pictured below).
‘The first part of my career was about learning my craft and operating a lot, and it was at Liverpool that I developed the use laparoscopy in babies and children before taking on more of a clinical leadership role,’ explains Professor Kenny. ‘It then became clear that building a new children’s hospital would give us the opportunity to create a beacon for child health in the north-west of England.’
As a result, the old Victorian hospital was knocked down and its footprint made into a park, while the new Alder Hey was created in an open space next to the original buildings.
Alongside the physical change, Professor Kenny and his team were also able to redesign care processes. ‘We worked really closely with children and families as we were building the hospital and were able to realise a lot of the things that I’d wanted to see in terms of putting the child and family at the heart of care,’ he says.
A great example of this co-creation is the operating theatre – the design of which means no child going in sees a child coming out.
‘When a child comes for surgery, it could have life changing consequences for them,’ says Professor Kenny, ‘so we worked hard to make an environment which is welcoming, such as, for example, enabling parents to sleep alongside their baby after they’ve just had surgery.’
The success of this work, and the change management skills that it involved, gave Professor Kenny an appetite for more challenges. In 2017, this led to him taking on the role of clinical lead for the paediatric general surgery and urology workstream in the ‘Getting It Right First Time’ (GIRFT) programme.
This national programme, started by Tim Briggs at the Royal National Orthopaedic Hospital, is designed to improve the treatment and care of patients through in-depth, clinically led review of services, benchmarking and presenting a data-driven evidence base to support change.
Professor Kenny published his GIRFT report in February 2021 and made a series of recommendations to help make a difference in the lives of young patients. These have resulted in significant quality improvements in children’s surgery by modifying the existing model of care in England to ensure surgical expertise is concentrated in the right place for the right patients.
Among the most recent improvements is the release of the Summary Paediatric Indicator Table data tool – known as SPaedIT – which brings together provider-level data summarising demand, capacity, flow and outcomes in one place in an easily-accessible dashboard to support elective recovery.
In September 2023, Professor Kenny also led the publication of concise guidance offering 10 actions to help reduce waiting times for children.
Professor Kenny’s experience as one of the first surgeons nationally to develop minimally invasive laparoscopy in children’s surgery has also enabled him to share his knowledge of transformative practices.
‘There are still issues around the technical side of children’s surgery – for example, that robotics is not yet as advanced in children’s surgery as it is for adults,’ he says.
‘But, actually, one of the main learnings I’ve had in the last 25 years since I’ve been engaged in consultant practices is that the biggest determinants of outcome for what we do are the socio-economic backgrounds of the children and families that we serve.’
Alongside health inequalities, Dr Kenny says the main challenges in paediatric surgery now are around the long-term outcomes for children who’ve had surgery.
His current research is looking at how to use data to measure those outcomes, which includes an NIHR research programme in conjunction with Oxford University for children’s surgical outcome reporting, establishing the core outcomes that are really important to children and families that experience paediatric surgery.
The next stage will be integrating the data into the electronic patient record and then having it available at a national – and, possibly, international – level, in order to benchmark and compare outcomes.
This in itself brings a challenge: paediatric surgery is a small speciality, and the procedures are quite rare, which means that the experience of individual surgeons is quite low, especially as they are spread across many different surgical centres.
As such, one of the recommendations from the ‘Getting It Right First Time’ report was to concentrate expertise in fewer centres, resulting in NHS England now having 10 operational delivery networks across the country, which are working to deliver that concentration.
‘It’s definitely a gradual process,’ says Professor Kenny, ‘but we are seeing major improvements as time goes by.’
Looking ahead, his interest in stem cell and regenerative therapies for rare conditions has seen Professor Kenny subspecialise in Hirschsprung’s disease – a rare, congenital bowel disorder – around which he has a long record of publication and research.
‘We’ve looked at the potential to use stem cell therapy to treat Hirschsprung’s disease and, in 2007, we were the first group internationally to isolate the stem cells from newborn babies,’ he says. ‘That work continues but it’s still a long path to clinical application because it has to be very safe and moving to transplantation into newborns is quite a major step.’
Meanwhile, Professor Kenny is a firm believer that while research is crucial, ‘you can’t do this job if you’re not actually seeing patients’.
He says: ‘The bit that’s really important is both that contact with children and parents and seeing first-hand what the data is telling us and then the other bit is in terms of credibility as a clinical leader. I know what the challenges are of working with electronic patient records, and I know the frustrations of having huge waiting lists, so I can speak with authority about it and I can empathise with people because I’m in the same position.’
Professor Kenny also believes it is important for consultants to broaden their professional scope, saying: ‘The great joy of children’s surgery is that close relationship with the children and families and the impact you have on them, but the attraction of the wider roles is being able to influence whole population groups.’
His other career yardstick is, he says, that ‘if you’re faced with a decision in life that involves change, then you should probably take change’.
Indeed, taking these bold steps is in no small part what contributed to Professor Kenny being awarded an OBE for services to paediatric surgery in the King’s 2024 New Year Honours List.
Speaking at the time of the announcement, Professor Kenny said: ‘I am honoured and deeply humbled to be named in the King’s New Year Honours List 2024. Although this award is for me, none of this would have been possible without the hard work, commitment and support of the fantastic teams I work with both at Alder Hey Children’s Hospital and NHS England.’
While huge progress has been made, Professor Kenny is clear that the work he and his teams are undertaking locally and nationally is far from over. ‘There are so many challenges facing child health in this country in making sure that our children are emotionally and physically equipped for the life that’s coming,’ he says.
‘I’m really excited about how we can work with NHS England and across government to develop child-friendly policies that mean we guarantee the health and welfare of our nation – and that starts with the health and welfare of our children.’
9th February 2024
Declining health among children under five should be urgently addressed, according to alarming evidence from The Academy of Medical Sciences that suggests the UK is ‘presiding over a crisis in child health’ and failing many of its children.
High rates of infant mortality, obesity and tooth decay are highlighted in the report, entitled ‘Prioritising early childhood to promote the nation’s health, wellbeing and prosperity‘, outlining a gathering crisis across the early years of children’s lives.
Early years health, which starts in pre-conception and goes through pregnancy and the first five years of life, is often overlooked in current policy but is crucial for laying the foundations for lifelong mental and physical health.
The authors are calling on the Government to take urgent action to prioritise the health of babies and young children across the UK. They warn that inaction could cost the Government at least £16bn a year and ‘condemn’ children to a life of poorer health.
The report was written by child health experts from across the UK and chaired by Professor Helen Minnis, professor of child and adolescent psychiatry at the University of Glasgow, and Professor Sir Andrew Pollard, Ashall Professor of infection and immunity and director of the Oxford Vaccine Group, Department of Paediatrics, University of Oxford.
It includes perspectives from parents and carers with lived experience examining the positive impact of intervening in the early years on the health and future of the nation.
Child health in the UK has stalled in recent years. Infant survival rates are worse than in 60% of similar countries, and the demand for children’s mental health services has surged, the report highlights.
Current figures show over a fifth of five-year-old children are overweight or obese, and a quarter are affected by tooth decay. In addition, vaccination rates have plunged below World Health Organization safety thresholds, leading to the possibility of disease outbreaks such as measles.
External factors, such as the Covid-19 pandemic and the cost of living crisis, have exacerbated the situation and increased health inequalities. The number of children living in extreme poverty tripled between 2019 and 2022, and those living in the most deprived areas are more likely to be affected by conditions such as obesity.
The report findings highlight the urgent need for early years health interventions and more research into children’s health to help identify future interventions.
The authors would like to see a unified approach from the Government so that policies and resources can be coordinated, alongside an improvement in the collection and access to child health data to enable research to feed effectively into policies.
The experts also point to a decline in the workforce responsible for child and family health, including paediatricians, and call for this to be addressed so that effective services can be prioritised and delivered in the early years.
In addition, the report highlights the need to ensure that a diverse range of voices of children, parents and carers are represented in developing early years policies and interventions.
Professor Minnis said: ‘Every child has the right to a safe and healthy childhood. It is shameful that the UK is failing to provide this. Child deaths are rising, infant survival lags behind comparable countries, and preventable physical and mental health issues plague our youngest citizens.
‘The science is clear – we are betraying our children. Unless the health of babies and young children is urgently prioritised, we condemn many to a life of poorer health and lost potential. The time to act is now.’
The data in the report suggests that policymakers can positively benefit the future health of the UK by frontloading investment into early years healthcare.
The Royal College of Paediatrics and Child Health (RCPCH) also welcomed the review.
Dr Mike McKean, vice president for health policy at the RCPCH, said: ‘This report provides alarming evidence that the UK is failing too many of its children. We are presiding over a crisis in child health that demands urgent action.
‘As paediatricians, we witness daily the devastating consequences of these systemic failures. Without transformative intervention on child health, we condemn generations to a poorer future. The time for excuses is over – Government must act decisively to address this crisis.’
A version of this article was originally published by our sister publication Nursing in Practice.
8th December 2023
Reducing oxygen levels in critically ill children on mechanical ventilators in intensive care could save 50 lives each year, according to a new study by Great Ormond Street Hospital (GOSH) and University College London.
The Oxy-PICU study, published in The Lancet, is the largest randomised controlled trial ever conducted in paediatric intensive care units (PICUs).
The researchers recruited 2,040 children from 15 NHS PICUs across England and Scotland. Each of the children required a mechanical ventilator and extra oxygen on admission to the PICU.
The children, who ranged from newborn up to 16 years, were randomly allocated to one of two groups: they received oxygen to the standard target level of oxygen (saturation “SpO2” >94%), or a reduced oxygen target (SpO2 88-92%).
The researchers found that the children who received the lower level of oxygen were 6% more likely to have a better outcome, either in terms of survival or the number of days spent on machines supporting their organs.
In the UK, around 20,000 children are admitted to intensive care each year and roughly 75% will receive additional oxygen through a ventilator.
If the study were to be scaled up, the researchers anticipate that this approach could save 50 lives, free up 6,000 ICU bed days and save £20m annually in the UK alone.
Professor Mark Peters, consultant paediatric intensivist at GOSH, professor of paediatric intensive care at UCL Great Ormond St Institute of Child Health, and lead author on the study, said: ‘Giving the minimum safe dose of anything in intensive care appears to generate the best outcomes, so we wanted to test this approach with oxygen.
‘We found a small benefit of lower oxygen targets that is unlikely to have been due to chance. But because so many children are treated with oxygen, this has the potential to improve outcomes and reduce healthcare costs in the UK and around the world.
‘This could have particular implications in countries where oxygen is a scarce resource, or in situations as we have seen in recent years, where health needs change, and oxygen demand quickly peaks.’
Lauran O’Neill, senior critical care research nurse at GOSH, said: ‘This is a major milestone study, which was nurse-led, with research taking place at the bedside as part of normal clinical care. It’s a great example of a research-hospital vision as every child admitted to ICU was screened for inclusion into the study.
‘Although GOSH teams were managing the study and a major recruiter, undertaking the research process was part of the standard of care for many emergency teams across the country – so training and education was the focus. We had to work well together across all collaborating hospitals to deliver this huge success for our patients.’
The Oxy-PICU study also includes researchers from the Intensive Care National Audit & Research Centre and the Paediatric Critical Care Society Study Group.
It is funded by the National Institute for Health and Care Research (NIHR)’s Health Technology Assessment programme and supported by the NIHR’s Biomedical Research Centres at GOSH and UCLH.
3rd November 2023
A single, national standardised early warning system for paediatric teams in hospital settings is being rolled out by the NHS in England as a way of ensuring deterioration in a child’s condition is detected and escalated quickly.
Available from today (3 November), the new inpatient Paediatric Early Warning System (PEWS) chart allows paediatricians to measure things like blood pressure, heart rate, oxygen levels and levels of consciousness. The system tracks any changes and has different scores representing the level of concern.
If a parent or carer raises a concern that their child is getting ill or sicker than the score shows, this will immediately escalate the child’s care regardless of other clinical observations.
Unlike the similar universal system currently in place for adults, there are four separate charts for different age ranges, including 0-11 months, one to four years, five to 12 years, and those aged 13 and over.
The ultimate aim of the PEWS is to improve working methods and safety to support better health outcomes.
It is part of the System-wide Paediatric Observations Tracking (SPOT) programme, led by NHS England in collaboration and endorsement from the Royal College of Paediatrics and Child Health (RCPCH) and the Royal College of Nursing (RCN).
The partnership has been developing PEWS for three years, with pilots running across 15 sites demonstrating clear benefits for both patients and staff.
Updates to the system – such as those based on any guidance from Martha’s Rule, which gives families and patients the right to a second medical assessment – will continue to be implemented as required.
Professor Sir Stephen Powis, NHS national medical director, said: ‘We know that nobody can spot the signs of a child getting sicker better than their parents, which is why we have ensured that the concerns of families and carers are right at the heart of this new system with immediate escalation in a child’s care if they raise concerns and plans to incorporate the right to a second opinion as the system develops further.’
The NHS will develop and circulate a leaflet and video content for parents to explain how to communicate concerns to healthcare staff and encourage them to escalate if needed.
Dr Mike Mckean, vice president for policy, at the RCPCH, said: ‘The National PEWS chart is an extremely important new tool that will support all paediatricians and wider child health teams to spot deterioration in children in hospital. Alongside training and resources, it will help teams across the country improve patient safety, efficacy and quality of care.
‘The SPOT programme builds on systems already in place in many trusts and has the many advantages of becoming a national system. I’d strongly encourage all paediatricians to look at the chart and consider what they need to do to smoothly transition towards integration and embedding the National PEWS into their trusts.’
Standardised tools and e-learning for using national PEWS charts in a range of health settings are in development.
In 2023/24 trusts are expected to appoint an education/implementation lead who will begin to support their trust to roll out the national PEWS.
Plans are in place to expand the system to emergency departments as well as mental health, ambulance and community services in due course. It is also hoped that the national PEWS chart will become the standard of care in England, meaning all medical and nursing students will receive instruction on its use during their training.
5th July 2023
Hospital-level care at home will reach children in every region of England with the expansion of the NHS virtual wards services this month, NHS England has announced.
NHS chief executive Amanda Pritchard will announce the expansion today (5 July) to mark the 75th anniversary of the NHS.
The virtual wards service will treat conditions like respiratory illness and heart conditions, with children able to receive care in familiar surroundings, intended to speed up their recovery and free-up hospital beds.
In the last year, more than 6,400 children have been successfully treated during trials, including in Blackpool, Dudley and Dorset.
More than 160,000 adult patients have been successfully treated on virtual wards since April last year.
The rollout comes as part of the NHS’ plan to create 10,000 extra virtual beds by winter.
Announcing the expansion, Ms Pritchard said: ‘As the NHS celebrates its 75th anniversary today, it is amazing to see how services have changed since our foundation. Virtual wards are already providing excellent care to families when their children are sick, and this expansion will enable thousands more to receive high-quality care from home.
‘Being treated at home can have a hugely positive impact on patients – it means they receive hospital-level care, but it also means they are not separated from their families – providing peace of mind for loved ones.’
The NHS’ national clinical director for children and young people, Professor Simon Kenny, said: ‘The introduction of paediatric virtual wards means children can receive clinical care from home, surrounded by family and an environment they and their parents would rather they be – with nurses and doctors just a call away.
‘More than 6,400 children have already been treated on a virtual ward, which also means they spend less time in hospital and that paediatric beds are there for the children that need them most, when they need them.’
Patients on a virtual ward are called for by a multi-disciplinary team providing a range of tests and treatments, with patients reviewed daily by the clinical team.
This story was originally published by our sister publication Healthcare Leader.
3rd July 2023
The incidence of type 1 diabetes in children and adolescents significantly increased during the Covid-19 pandemic compared to pre-pandemic levels, according to a recent meta-analysis.
Suggestions of an association between infection with Covid-19 and a new diagnosis of of type 1 and type 2 diabetes emerged early in the pandemic. However, the causal mechanisms responsible are unclear. Moreover, understanding the nature of any relationship between diabetes and infection with Covid-19 is complicated by several factors including the seasonality of diagnoses and evidence of an estimated 3.4% annual increase in the incidence of the condition.
In trying to untangle the potential association between the rise in cases of type 1 diabetes and infection with Covid-19, a team of Canadian researchers, writing in JAMA Network Open, compared the incidence rates of paediatric diabetes during and before the Covid-19 pandemic.
The team undertook a systematic review and meta-analysis of all medical databases, using subject headings and text terms related to Covid-19, diabetes and diabetic ketoacidosis (DKA). Studies were included in the analysis if these reported differences in incident diabetes cases during compared to before the pandemic, among individuals under 19 years of age.
Researchers set the primary outcome as the change in the incidence rate of paediatric diabetes from before and during the pandemic. The secondary outcome was the change in the incidence rate of DKA among youths with new-onset diabetes during the pandemic.
In total, 42 studies with 102,984 incident diabetes cases were included in the analysis.
The type 1 diabetes incidence rate was 14% higher during the first year of the pandemic compared with the pre-pandemic period (incidence rate ratio, IRR = 1.14 95% CI 1.08 – 1.21). Nevertheless, this rate increased further during months 13 to 24 of the pandemic compared to the pre-pandemic level (IRR = 1.27, 95% CI 1.18 – 1.37). There was also a higher incidence of DKA compared to before the pandemic (IRR = 1.26, 95% CI 1.17 – 1.36).
The underlying mechanisms responsible for this observed increase are unclear and require further investigation.
15th May 2023
The number of children waiting for NHS hospital appointments has reached an all time high, the latest NHS figures show.
There are currently 403,955 children waiting for consultant-led care, of which 18,000 have been waiting for more than a year for essential treatment, the UK’s Royal College of Paediatrics and Child Health has warned.
The College notes that while there has been considerable progress made in shrinking the adult backlog, the children’s list ‘continues to rise at an unprecedented rate’, with ‘children not being prioritised’.
Long waits for children are of particular concern, given many treatments and interventions must be administered within specific age or developmental stages, a statement from the College said. And the data does not capture the full scale of the problem, it added, with hidden and growing waiting times for community care.
The RCPCH has called on the Government to set aside ringfenced funding for children’s service recovery at all community, elective, and urgent care levels, as well as publishing a fully-costed NHS workforce plan immediately.
The figures come as NHS England said the number of patients waiting more than 18 months fell to just 10,737 by April – down by more than 90% from 124,911 in September 2021 and by more than four-fifths since the start of January when there were 54,882.
RCPCH president Dr Camilla Kingdon said: ‘It is a national scandal that over 400,000 children are stuck in limbo on a list, waiting for treatment.
‘These children could fill Wembley stadium four times over. NHS England has a zero-tolerance policy for 52-week waits, so it is deeply concerning that these targets are being missed.
‘The clear regional variation in size of waiting lists also means that this is an equity issue for children and their families. Child health teams are working tirelessly to address the growing backlogs, but without proper support, their efforts are unable to make a meaningful dent in the problem.’
RCPCH officer for health services, Dr Ronny Cheung added: ‘It’s clear now that the voices of children are not being heard. It seems that the focus in the lead up to the next election is primarily on voting-aged adult issues.
‘Lengthy waits are unacceptable for any patient but for children and young people the waits can be catastrophic, as many treatments need to be given by a specific age or developmental stage.
‘In recent months we’ve heard about children missing school, quitting sports, and missing out on the important aspects of a healthy, happy childhood. This is not a trivial matter.’
Meanwhile, statistics published by NHS England showed that the overall elective waiting list has grown to a record high, with 7.3 million people now waiting for treatment.
A version of this story was originally published by our sister publication Pulse.
13th September 2022
The malaria vaccine, R21/Matrix-M has been shown to provide a high degree of efficacy against the disease in children, two years after the initial primary vaccination regime and a 12 month booster according to the findings of a study by a research team based at the Jenner Institute, University of Oxford and in collaboration with researchers from Burkina Faso.
Malaria is a life-threatening disease caused by Plasmodium falciparum (P falciparum), a parasite that is transmitted via the bite of infected female Anopheles mosquitoes that is both preventable and curable. According to the World Health Organisation (WHO), in 2020, there were an estimated 241 million global cases of malaria and 627 000 deaths.
Moreover, in a 2021 report WHO noted how Sub-Saharan Africa continues to carry the heaviest malaria burden, accounting for about 95% of all malaria cases and 96% of all deaths in 2020. Additionally, about 80% of deaths in the region are among children under 5 years of age.
WHO set a target in 2013 that a vaccine for malaria should show greater than or equal to 75 percent efficacy. Previous work demonstrated that three vaccination of low-dose R21/Matrix-M, administered at 4-week intervals before the malaria season, with a fourth dose 1 year later, provided an efficacy of 77%.
In the current study, the same research group who undertook the original, randomised, placebo-controlled trial with the R21/Matrix-M vaccine, have provided an update, reporting on the vaccine efficacy over the 12 months following the first booster, i.e. a 24 month period after the primary vaccination regime. The researchers used a primary case definition of clinical malaria as an axillary temperature of 37·5°C or greater.
R21/Matrix-M efficacy against malaria in children
The researchers included 409 children with a mean age of 11.6 years (49% male) at their first vaccine dose and who were followed after their 12 month booster dose, administered before the second malaria season.
The data set included 132 given the low dose, group 1 (5 μg R21 adjuvanted with 25 μg Matrix-M), 137 the higher dose, group 2 (5 μg R21 adjuvanted with 50 μg Matrix-M) and 140 who received a control vaccine. Adequate use of insecticide-treated nets before the second malaria season was 89% overall and indoor residual spraying was done in 43% of households.
Based on the primary case definition, there were 242 cases with at least one episode of malaria from 14 days to 12 months after the booster vaccination. Among those in group 1, this represented a vaccine efficacy of 70% (95% CI 59 – 78) and 80% (95% CI 72 – 85) in group 2 compared the control group, after adjustment for sex, age and adequate bednet use.
When researchers re-assessed R21/Matrix-M at 24 months after the primary vaccination regime, the vaccine efficacy was 66% (95% CI 55 – 74) for group 1 and 75% (95% CI 66 – 81) for group 2.
The authors concluded that when delivered seasonally, the R21/Matrix-M malaria vaccine maintained high efficacy and that these findings suggest the vaccine strategy could have a substantial impact in areas of highly seasonal malaria transmission in Africa.
Citation
Datoo MS et al. Efficacy and immunogenicity of R21/Matrix-M vaccine against clinical malaria after 2 years’ follow-up in children in Burkina Faso: a phase 1/2b randomised controlled trial Lancet Infect Dis 2022
5th September 2022
The presence of autoimmune thyroiditis is independently associated with a higher risk of thyroid cancer among children with thyroid nodules according to the findings of a cross-sectional study by researchers based at Harvard Medical School, US.
AIT, also known as Hashimoto’s thyroiditis, is frequently associated with papillary thyroid cancer and may indeed be a risk factor for developing this type of cancer. However, other work suggests that a correlation between AIT and a higher incidence of thyroid cancer is still undefined.
In children there is also some uncertainty of this relationship. Some data suggest that AIT seems to influence the development of thyroid nodules, but not cancer, whereas other data indicate a relationship with malignancy.
Whilst the presence of thyroid nodules are more common in children with AIT, whether this increases the risk of thyroid cancer is unclear, although there is a clear relationship between the presence of AIT, nodules and malignancy in adults.
In the present study, the researchers sought to better understand the relationship between AIT in children and thyroid cancer, among a cohort who underwent thyroid nodule evaluation over an extended period of time. Included participants were < 19 years of age and who had undergone fine needle aspiration of a nodule between 1998 and 2020.
The presence of thyroid cancer was defined by histopathology for resected nodules and the researchers set the primary outcome as the presence of thyroid cancer.
Autoimmune thyroiditis and cancer in children
A total of 385 individuals with a mean age of 15.5 years (81% female) with 458 nodules, were included in the retrospective analysis.
Thyroid cancer was present in 108 nodules (24%) and AIT was seen 95 (25%) of the study population. The most common type of thyroid cancer was papillary (82%) followed by follicular carcinoma (14%).
The presence of clinical AIT was independently associated with an increased risk of thyroid cancer (odds ratio, OR = 2.19, 95% CI 1.32 – 3.62). Moreover, thyroid cancer was directly associated with the diameter of the nodules (OR = 1.05, 95% CI 1.03 – 1.06, p < 0.001) and inversely associated with age (OR = 0.90, 95% CI 0.83 – 0.97, p = 0.007).
Interestingly, female sex and the presence of multiple nodules were both independently associated with a lower risk of thyroid cancer.
The authors concluded that among children with thyroid nodules, the presence of AIT was associated with an increased risk of thyroid cancer. They suggested that a clinical diagnosis of AIT may inform the assessment of thyroid cancer risk and surgical decision-making in children who had thyroid nodules.
Citation
Keefe G et al. Autoimmune Thyroiditis and Risk of Malignancy in Children with Thyroid Nodules Thyroid 2022
Higher doses of the CAR T cell therapy tisagenlecleucel lead to better overall, event-free and relapse-free survival in children with relapsed or refractory acute lymphoblastic leukaemia, according to the findings of a retrospective study by US researchers.
Acute lymphoblastic leukaemia (ALL) is a malignant transformation and proliferation of lymphoid progenitor cells in the bone marrow, blood and extra-medullary sites and 80% of cases occur in children. While chemotherapy is used first line, in cases where this initial treatment is either refractory or if patients relapse, chimeric antigen receptor-modified T cells (CAR T-cells) against CD19, have been shown to be effective.
The target dose of transfused cells was 2.0 to 5.0 x 106 CAR transduced viable T-cells per kg, for those weighing less than 50 kg and 1.0 to 2.5 x 108 T-cells for those weighing over 50 kg. However, whilst it is an effective therapy, what remains unclear is whether the dose of tisagenlecleucel impacts on disease-related outcomes such as overall survival.
As a result, in the present study, the US researchers decided to retrospectively examine the impact of dose using data from the Pediatric Real World CAR Consortium which collects data from 15 paediatric oncology centres that are using commercial tisagenlecleucel.
The team included patients with relapsed or refractory ALL and divided the infused cell doses into quartiles, D1 (0.1341.300 x 106), D2 (1.301-1.700 x 106), D3 (1.701- 2.400 x 106) and D4 (2.401-5.100 x 106).
Overall survival (OS) was defined as the time from infusion to death from any cause, event-free survival (EFS), the time to the earliest non-response, relapse or death.
Patients who did not respond were considered to have had a EFS event on day 28. Relapse-free survival (RFS) was only defined among responders, as the time from day 28 to relapse.
Tisagenlecleucel and disease outcomes
A total of 180 children with a mean age of 12.6 years (40.3% female) were included in the analysis and the median cell dose was 1.7 x 106 cells/kg and overall, 82.8% of patients had a treatment relapse and 51% described as having a high disease burden.
Overall survival for the entire cohort after 3 years was 58.9% (95% CI 47.6 – 72.9%) and the median follow-up time was 402.5 days. In multivariable regression, those on the highest dose (D4) had a significantly improved survival compared to those in D1, the lowest dose (hazard ratio, HR = 0.22, 95% CI 0.08 – 0.61, p = 0.008).
For EFS, multivariable regression showed that again, those receiving the highest compared to the lowest dose, had improved survival (HR = 0.24, 95% CI 0.12 – 0.49, p < 0.001). Finally, RFS was also greater among those receiving the highest dose (HR = 0.18, 95% CI 0.07 – 0.49, p = 0.002). There were also no differences in toxicity across the different dose ranges.
The authors concluded that as higher doses were associated with superior survival in a real world setting, further work was required to examine the value of using higher tisagenlecleucel dosing.
Citation
Stefanski HE et al. Higher doses of tisagenlecleucel associate with improved outcomes: a report from the pediatric real-world CAR consortium Blood Ave 2022
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