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30th January 2020
The Department for Education will be reviewing its statutory guidance “Supporting pupils with medical conditions at school” this year, and the article’s co-authors Dr Paul Turner, Prof Adam Fox and the CEOs of the charities Allergy UK and the Anaphylaxis Campaign are calling for a level of specific detail on supporting children with allergy to sit below the currently wholly generic guidance.
17% of fatal food-anaphylaxis reactions in school-age children happen while they are at school and 20% of anaphylactic reactions in schools are in children with no prior history of food allergy.
Coroners at the inquests into the deaths of Mohammad Ismaeel Ashraf and Karanbir Cheema (who both died as a result of anaphylaxis while at school) flagged up some key issues.
These focused on:
A lack of staff training which resulted in the delayed and incorrect administration of adrenaline, and
Policy implementation issues around autoinjectors which were out of date and not readily available.
The authors highlight the urgent need for clear and mandatory food allergy policies which recognise children at risk, include measures to minimise risk, and formalise adequate training for school staff to recognise the symptoms of anaphylaxis and administer adrenaline early and effectively in a ‘whole school’ approach.
The authors also review the current legislation which allows schools to purchase ‘spare’ AAIs. This legislation is supported by non-mandatory Department of Health and Social Care (DHSC) guidance on the management of children with anaphylaxis in schools. With take-up of spare pens at a very low level (linked in part due to the absence of any funding for this initiative) and poor staff training, they propose important changes which include funding for schools to purchase AAIs and for mandatory training for school staff. These initiatives could be cost-neutral, as pupils would no longer need to provide their own exclusive supply of AAIs to remain in school. Legislation in both the USA and Australia allows a supply of AAIs direct to schools, with a documented improvement in the management of allergic reactions.
The article’s lead author Dr Paul Turner says: “We are asking for the DHSC, the Department for Education and the schools inspectorates to work together to make schools a safer environment for food-allergic children. Two inquests into the death of school pupils have highlighted the urgent need for staff training, for systematic and consistent policies and procedures on the management of children with food allergy and an assurance through inspection that these are implemented in schools. We believe that the approach we will present to the Government departments in the next month is far-sighted and cost-effective. Families with children with food allergy live in fear of the consequences of anaphylaxis. They need to have confidence that schools will safeguard their children and that staff are trained to take correct and urgent action in the event of anaphylaxis. The current guidance is simply not adequate to inspire that confidence – parents, school staff and most importantly, our children deserve better.”
It is simply not possible to predict which medication will work for the individual patient, and it is therefore a matter of trying the different treatment options: glucocorticoids, low dose chemotherapeutic drugs or newer biological drugs. Until now.
A new research project from Aarhus University and Aarhus University Hospital in Denmark suggest that it is the composition of cells in the joint of the individual patient which determines whether the medicine is effective or not. The researcher behind the study, published in the journal of the American College of Rheumatology (ACR Open), is medical doctor and PhD Tue Wenzel Kragstrup from the Department of Biomedicine at Aarhus University and the Department of Rheumatology at Aarhus University Hospital.
“So far, we’ve examined the effect of nine drugs in three different in vitro models. We can see that the effect of a given drug depends on the cell compositions. This leads us to hope that the immunological cells and inflammatory signalling molecules in the joint of each individual patient will be able to predict the most effective treatment,” says Tue Wenzel Kragstrup.
The study’s in vitro models consist of cell cultures isolated from joint fluids aspirated from the joints of patients with severe arthritis as part of the treatment at Aarhus University Hospital. The researchers then used the diseased cells from the joint fluid to examine the effects of different types of medicine, explains Tue Wenzel Kragstrup’s colleague, PhD student Morten Aagaard Nielsen from the Department og Biomedicine, who is first author of the article.
“As far as we know, this is the first description of a direct association between the composition of the immune system cells and the effect of medication,” Morten Aagaard Nielsen says. Tue Wenzel Kragstrup adds: “The findings are based on the assumption that the cells in the test tube behaving like they would behave in the patient. So of course, the research result must be validated in humans in a larger number of patients,” says Tue Wenzel Kragstrup, who concurrent with his biomedical research is in rheumatology specialist training.
It was at the hospital while meeting patients racked with pain, that Tue Wenzel Kragstrup got the idea and invented the project.
“Today, the first-choice drug has no effect in around one in three patients. So, doctors often have to try several different types of medicine before they find the right one. This is a process that may take years, because a drug typically takes three to four months to be effective,” explains Tue Wenzel Kragstrup.
“And while we wait, the patients continue to experience swelling and pain – not to mention the permanent damage that can occur in the joints as a result of persistent inflammation,” he says.
Tue Wenzel Kragstrup hopes that the research results in time can be translated into a simple test – such as a blood test – which can determine the correct medicine for the individual patient. According to Tue Wenzel Kragstrup, this type of individualised treatment could be realised within a decade.
“Prostate cancer is the most prevalent form of cancer in men throughout Europe, and the expanded approval of apalutamide marks a significant advancement for those living with mHSPC,” said Prof Dr med Axel S Merseburger, Chairman of the Department of Urology, Campus Lübeck, University Hospital Schleswig-Holstein, Kiel, Germany. “In prostate cancer treatment, our primary goal is always to delay progression of disease and prolong survival, to ensure the best possible outcomes for patients. Today’s news is therefore an encouraging development for patients within Europe, for whom the importance of an additional treatment option that can both delay progression and extend survival cannot be underestimated.”
The EC approval is based on data from the Phase III TITAN study, which assessed the addition of apalutamide to ADT in a broad range of patients with mHSPC, regardless of disease volume, prior treatment with docetaxel or staging at initial diagnosis. The dual primary endpoints of the study were overall survival (OS) and radiographic progression-free survival (rPFS).1 Apalutamide plus ADT significantly improved OS compared to placebo plus ADT with a 33% reduction in the risk of death (HR=0.67; 95% CI, 0.51-0.89; p=0.0053).1 In both study arms, median OS was not reached.1 Apalutamide plus ADT also significantly improved rPFS compared to placebo plus ADT with a 52% reduction in risk of radiographic progression or death compared to placebo plus ADT (HR=0.48; 95% CI, 0.39-0.60; p<0.0001).1 The median rPFS was 22.1 months for placebo plus ADT and not reached for apalutamide plus ADT.1 The two-year OS rates, after a median follow up of 22.7 months, were 82% for apalutamide plus ADT compared to 74% for placebo plus ADT.2 These results were published in The New England Journal of Medicine.1,2
References
1 Chi KN et al. Apalutamide for Metastatic, Castration-Sensitive Prostate Cancer. N Engl J Med 2019;381:13-24.
2 Chi KN. First results from TITAN: A phase III double-blind, randomized study of apalutamide versus placebo in patients with metastatic castration-sensitive prostate cancer receiving androgen deprivation therapy. American Society of Clinical Oncology Annual Meeting 2019. Abstract #5006.
Radioactive iodine is now to be recommended as the frontline treatment for patients with thyroid gland overactivity caused by conditions such as Graves’ disease, following an evidence review.
Hyperthyroidism is a condition in which the thyroid gland produces excess amounts of thyroid hormones. Affecting more than 1.5 million people in the UK, it can lead to a range of symptoms including weight loss, nervousness, irritability, heat intolerance, heart racing, tremor and muscular weakness. The most common type of hyperthyroidism is Graves’ disease, a condition where the body’s immune system mistakenly attacks the thyroid gland, triggering over-production of thyroid hormones.
Current recommendations are to treat patients with thionamides, and in the UK and Europe radioactive iodine is often only given if they suffer a relapse after this treatment. This is due to safety concerns over potential adverse effects of radioactive iodine such as worsening eye disease, and development of secondary cancers.
Research led by the Birmingham researchers has shown that radioiodine is a safe treatment and results in improved cardiovascular outcomes for patients with hyperthyroidism.
The NICE independent guideline committee surveyed clinical and economic evidence to conclude that radioactive iodine was the most effective treatment for the condition, curing 90 per cent of cases. They also concluded there was no evidence of a clinically important increase in cancer diagnoses or deaths between people treated with radioactive iodine and healthy controls.
Dr Kristien Boelaert, who led the guideline committee, says: “There has been uncertainty in the UK about the best treatment for hyperthyroidism despite radioactive iodine being the most common first line treatment for this condition in the US. We are very pleased to have been able to work with NICE to provide clear new guidance which we hope will improve outcomes for patients with this condition.”
Patients treated with radioactive iodine take single tablet which contains iodine and a low dose of radiation, which is absorbed by the thyroid. After taking the treatment patients are advised to avoid prolonged close contact with children and pregnant women for a few days or weeks and avoid getting pregnant or fathering a child for several months. The treatment is likely to lead to an underactive thyroid gland which will require ongoing treatment with thyroid hormone tablets.
A team at The Institute of Cancer Research, London, carried out the analysis as part of preparation for its pioneering new Centre for Cancer Drug Discovery, to help build the case for more streamlined regulations for the most exciting treatments.
Their study found that increasing numbers of drugs are being authorised – including rising numbers of highly innovative treatments. But it is taking an increasing amount of time for these new drugs to navigate the strict regulations on clinical trials and licensing, forcing patients to wait even longer for the latest treatments.
Innovative new treatments are designed to attack brand new weaknesses in cancer, and form a key part of The Institute of Cancer Research (ICR)’s strategy to overcome cancer’s lethal ability to evolve resistance to treatment. The study is published in the journal Drug Discovery Today, and provides an overview of access to all drugs licensed through the European Medicines Agency (EMA) from 2000 to the end of 2016.
The researchers assigned each drug to one of three categories of innovation – high, medium or low – with the high innovation category including drugs with a new target or mechanism of action, or representing a new class of treatment in an area of unmet need.
The most innovative cancer drugs took 3.2 years longer to go from the filing of the patent through to NHS patients than low-innovation treatments – suggesting various initiatives intended to prioritise the most innovative drugs had yet to be effective.
The researchers are calling for action to be taken to improve patients’ access to the most exciting new treatments by streamlining regulations on clinical trials and licensing and more strongly incentivising innovation in drug discovery and development.
Between 2000 and the end of 2016, the most innovative new drugs took 14.3 years to progress from patenting through to availability on the NHS – compared with 13.5 years for medium-innovation drugs and 11.1 years for the least innovative treatments.
Much of the delay seemed to occur in the period from the start of a phase I trial through to EMA authorisation – which lasted an average of 8.9 years for the most innovative drugs compared with 8.7 years for medium-innovation drugs and 6.8 years for the least innovative.
Among the highly innovative drugs to undergo delays, mifamurtide took 20 years to go from patent through trials and licensing to NICE approval for treating osteosarcoma, and it took trabectedin 22 years from patent to NICE approval for advanced soft tissue carcinoma. More recently, the EMA only authorised olaparib for breast cancer in April 2019, 15 months after the US Food and Drug Administration, and NICE has still not released its appraisal.
The average number of drugs to be licensed by the EMA increased from a median of six per year from 2000-08, to 13.5 per year from 2009-16. But the average time from a drug’s patent being filed to it being made available on the NHS increased over the study period – from 12.8 years for drugs first licensed between 2000 and 2008, up to 14.0 years for drugs licensed between 2009 and 2016.
The time taken from the filing of the patent to setting up a phase I trial rose from a median of two years for those licensed from 2000 to 2008, up to three years for those licensed from 2009 to 2016.
And as with the delays to approval seen in the most innovative new drugs, there seemed to be hold-ups in the period between the start of the phase I trial and EMA authorisation – which lasted an average of 7.7 years for drugs first licensed from 2000 to 2008, but 9.0 years for those licensed from 2009 to 2016.
The researchers found that NICE had reduced the lag time between EMA authorisation and its beginning technology appraisals, from a mean of 21 months for drugs first licensed between 2000 and 2008, down to 6.5 months for drugs licensed between 2009 and 2016. But it was getting no faster at carrying out its appraisals, which took 16.7 months from 2000-08, and 16.0 months from 2009-16.
There was evidence that NICE had not been prioritising the most innovative treatments, with only 38% of highly innovative cancer drugs having received a positive recommendation at the time of the analysis, compared with 53% of drugs classed as moderately innovative and 40 per cent of low-innovation drugs.
However, this difference seemed to be because NICE had been less likely to start an appraisal for more highly innovative drugs – and it has since committed to appraising all new cancer drugs, which should address the discrepancy.
Overall, the findings suggest that there is a need to build stronger incentives into the whole system of drug discovery and development to encourage companies to take forward innovative new treatments that can deliver major advances for patients.
The study especially raises concerns over the regulation of clinical trials and drug licensing in the UK and Europe – and suggests that the Medicines and Healthcare Products Regulatory Agency (MHRA) and EMA should adapt their approaches to regulation to make it easier to take exciting new treatments to patients.
The study is published with the ICR now needing to raise less than £10 million for its new £75 million Centre for Cancer Drug Discovery, which will house the world’s first drug programme to be dedicated to combating cancer evolution and drug resistance.
Study leader Professor Paul Workman, Chief Executive of The Institute of Cancer Research, London, said: “Our study details the major progress being made against cancer, with the average number of drugs being licensed each year more than doubling over the last decade. But it also makes clear that our regulatory systems are not keeping pace with advances in the science. It is taking longer for new drugs to reach patients and, alarmingly, the delays are longest for the most exciting, innovative treatments, with the greatest potential to transform the lives of patients.
“At the moment the whole ecosystem for drug discovery and development – involving regulators, researchers and companies – is too risk averse. It’s crucial that academic researchers and pharmaceutical companies should feel that the regulatory systems for drug development support risk taking and innovation, rather than discouraging it and slowing it down. Our study raises questions in particular for the processes in the UK and Europe for regulating clinical trials and licensing, which need to do better at recognising and rewarding innovation.
“As the ICR begins an exciting new ‘Darwinian’ programme of drug discovery to combat cancer evolution and drug resistance, we are calling on Government, regulators and industry to together reshape the cancer therapeutics ecosystem, so the best new treatments can progress to patients much more quickly.”
28th January 2020
Lung cancer is the leading cause of cancer deaths in the UK and has a poor five year survival rate of around 13%. Research led by the University of Exeter Medical School, published in the British Journal of General Practice, aimed to improve potentially life-saving early diagnosis through analysing which symptoms patients present first to their doctor. The team examined 27,795 records of adults who were diagnosed with lung cancer between 2000 and 2017, at more than 600 UK GP practices.
Over the seven year period, the team found an increase in both cough and shortness of breath as the first symptom patients reported when they went on to be diagnosed with lung cancer. The study, funded by Cancer Research UK and supported by NIHR, found a decrease in patients who reported the first symptom to be coughing up blood, or loss of appetite, generally regarded as the headline symptom of lung cancer.
Professor Willie Hamilton, of the University of Exeter Medical School, who was an author on the paper, said: “Lung cancer is the biggest cancer killer in the UK. Our paper shows a rapid change in the first symptom doctors are seeing. That’s probably not caused by any change in basic biology, it’s more likely to be down to earlier detection.
“It means teaching must change — clinicians must be alert to the risks of cough and shortness of breath.”
Sara Hiom, director of early diagnosis at Cancer Research UK, said: “This important study indicates that people are now going to the doctor about different symptoms of lung cancer, such as cough and shortness of breath, possibly because of awareness campaigns in the past. Most people with these symptoms will not have lung cancer, but it’s well worth letting your GP decide if you need tests – because if it is cancer, a prompt diagnosis and speedy treatment make all the difference. And although smoking makes it much more likely, remember that non-smokers can have lung cancer too.”
In the study, from Queen Mary University of London and the University of Cambridge and published in the British Journal of General Practice (BJGP), the researchers describe a number of causes behind this. To do this, they took the results of a previous study looking at issues with returning to work after stroke at UK level to all stakeholders from a local community.
They found there was a mismatch between patient and carer needs and what is provided by primary care. This included lack of GP awareness of invisible impairments, uncertainty how primary care could help in time-limited consultations and complexity of return-to-work issues.
Primary care physicians were also not aware of relevant services they could refer patients to, such as occupational therapy support.
In addition, there was an overall lack of coordination between different stakeholders in the returning to work process. Linking with other services was considered important but challenging due to ongoing changes in service structure and the commissioning model.
A quarter of all strokes happen in working age, with a general practice of around 6000 patients containing on average 15 stroke survivors aged between 18 and 65 years. Stroke rates are increasing in people aged under 55 while it is known that enabling people with stroke to work has positive effects on health.
Dr Anna De Simoni, lead author of the study from Queen Mary University of London’s Institute of Population Health Sciences said: “Primary care is in a crucial position to support stroke survivors successfully returning to work and address inequalities in access to vocational rehabilitation support.
“Through group discussions with stakeholders from a local community, patients, carers, GPs, occupational therapists, employer representative and clinical commissioners we are able to put forward concrete proposals to address the barriers identified. “
Suggestions for improvement include a central contact in primary care for signposting to available services, a rehabilitation assessment integrated with the electronic record, and a patient-held share-care plan at discharge from stroke wards.
The Equality Act 2010 obliges employers to consider whether ‘reasonable adjustments’ could help stroke survivors return to work, provided there is an assessment of their impairments/disabilities.
However, the researchers found evidence of employers asking clinicians for sick notes rather than undertaking work adaptations.
Dr De Simoni added: “This is more straightforward for physical impairments. Primary care might be the only source of help for patients whose invisible impairments have not been highlighted and are exacerbated by return to employment and for self-employed or business owners.”
The researchers say that more work is needed to develop the three suggestions emerged from this study into interventions, addressing potential problems together with their evaluation in terms of cost-benefit.
Georgina Hill, Research Communications Manager at the Stroke Association, said: “Stroke can strike at any age, and about a quarter of stroke survivors are of working age. This study highlights the unique challenges that stroke survivors may face in getting the support they need when returning to work. While this study looked at a small number of people in the UK, it builds on existing evidence that too often, stroke survivors and those close to them, can’t get the support they need to rebuild their lives.
“Last year, our largest ever survey of people affected by stroke was published as the Lived Experience of Stroke report, which also showed that many stroke survivors experience fatigue and problems with memory and concentration which need consideration when stroke survivors return to work.
Bowel cancer screening tests look for hidden blood in the stool which can be an early sign of bowel cancer. But the new test, the faecal immunochemical test (FIT), does not provide a simple yes or no answer.
It is up to the individual health systems to decide what level of haemoglobin warrants further investigation, usually a colonoscopy. And this is where patients are missing out on potentially life-saving early diagnoses largely due to a lack of specialist staff.
NHS England and NHS Scotland have chosen different cut-off points for a referral following a screening test – 120 and 80 micrograms of haemoglobin per gram of faeces respectively. This means that in Scotland, the NHS refers people who have between 80 and 120 micrograms of haemoglobin, when the NHS in England does not.
And this equates to around 1100 bowel cancers that could be diagnosed through the bowel cancer screening programme each year in England but are not.
The impact is even higher when we consider the number of pre-cancerous growths that could be detected and removed during a colonoscopy.
Cancer Research UK has calculated that if the NHS in England referred people with the same hidden blood levels as Scotland, there could be an additional 2000 colonoscopies each month in England. Although many of these would not turn out to be cancer, the NHS in England does not have enough endoscopists, nurses and other specialist staff to handle this.
Already one in 10 diagnostic posts are vacant in England. And the demand for staff is rising. Around 363,000 people are diagnosed with cancer each year in the UK but by 2035, that is likely to increase to around half a million people.
When bowel cancer is diagnosed at the earliest stage, as it can be through screening, more than 9 in 10 people survive their disease for at least five years. But when it is detected in the late stages, survival falls to less than 1 in 10.
Sara Hiom, Cancer Research UK’s director of early diagnosis, said: “The UK’s bowel cancer screening programme is very effective at detecting cancer early. But we’re concerned that NHS staff shortages are having a direct impact on the ability to diagnose more patients at an early stage – something that the Government committed to doing last year. People shouldn’t be slipping through the net.
“Improvements to cancer screening in the UK need to be made quickly and safely to ensure the NHS can diagnose people earlier. Even though NHS staff on the ground are doing everything they can to diagnose people early, the Government needs to back them up with significant investment to train and recruit more staff so that doctors, nurses and other specialists can diagnose more people at an early stage, when treatment is more likely to be successful.”
FIT bowel screening is a more accurate way to test for early stage bowel cancer, than the previous test, known as gFOBT. It was introduced in England last year and has been running in Scotland for two years.
The latest figures show that around 42,000 people are diagnosed with bowel cancer each year in the UK which equates to more than 110 people every day. Around 16,300 people die from bowel cancer every year. And in England, more than half of bowel cancers with a known stage are diagnosed at a late stage.
Dr Ed Seward, consultant gastroenterologist and Cancer Research UK’s clinical adviser said: “When we treat bowel cancer patients who were diagnosed at an early stage, we have curative treatments we can offer, including surgery. It can be devastating to patients and their families when the disease is caught at a late stage, which is why the bowel screening programme is so important – finding cancers when they haven’t caused symptoms at all. So we really don’t want to see patients missing out on a potentially life-saving opportunity.”
24th January 2020
Time is critical with sepsis, but the tests currently used in hospitals can take up to 72 hours to provide a diagnosis.
Many scientists are working on this critical issue, including those at Abionic, an EPFL spin-off. Researchers at the Laboratory of Bionanophotonic Systems (BIOS) at EPFL’s School of Engineering have just unveiled a new technology.
They have developed an optical biosensor that slashes the sepsis diagnosis time from several days to just a few minutes. Their novel approach draws on recent developments in nanotechnology and on light effects at a nano scale to create a highly portable, easy-to-use device that can rapidly detect sepsis biomarkers in a patient’s bloodstream. And their device takes just a few minutes to deliver a result.
Because the biosensor uses a unique plasmonics technology, it can be built from small, inexpensive components, yet it can achieve an accuracy on par with gold-standard laboratory methods. The device can screen a large panel of biomarkers and be adapted for the rapid diagnosis of a number of diseases. It was installed at Vall d’Hebron University Hospital in Spain and used in blind tests to examine patient samples from the hospital’s sepsis bank. The researchers’ technology is patent-pending.
Trapping biomarkers in nanoholes
The device employs an optical metasurface – in this case a thin gold sheet containing arrays of billions of nanoholes. The metasurface concentrates light around the nanoholes so as to allow for exceptionally precise biomarker detection. With this type of metasurface, the researchers can detect sepsis biomarkers in a blood sample with nothing more than a simple LED and a standard CMOS camera.
The researchers begin by adding a solution of special nanoparticles to the sample that are designed to capture the biomarkers. They then distribute this mixture on the metasurface. “Any nanoparticles that contain captured biomarkers are trapped quickly by antibodies on the nanoholes,” says Alexander Belushkin, the lead author of the study. When an LED is applied, those nanoparticles partially obstruct the light passing through the perforated metasurface. “These nano-scale interactions are imaged by the CMOS camera and digitally counted in real-time at high precision,” says Filiz Yesilkoy, the study’s co-author. The generated images are used to rapidly determine whether disease biomarkers are present in a sample and, if so, in what concentration. They used the new device to measure the blood serum levels of two important sepsis relevant biomarkers, procalcitonin and C-reactive protein. Doctors can use this information to accelerate the triage of sepsis patients, ultimately saving lives.
“We believe our low-cost, compact biosensor would be a valuable piece of equipment in ambulances and certain hospital wards,” says Hatice Altug, the head of BIOS. Scientists already have possible applications in mind. “There is an urgent need for such promising biosensors so that doctors can diagnosis sepsis accurately and quickly, thereby keeping patient mortality to a minimum,” say Anna Fàbrega and Juan José González, lead doctors at Vall d’Hebron University Hospital.
Ustekinumab was previously approved for use in adolescent and adult patients with plaque psoriasis, aged 12 years and older,and is now the first available biologic treatment in this patient population to selectively address the IL-23/IL-12 pathway, an important therapeutic target for the condition.
“This latest EC approval is a significant milestone for young children struggling to cope with the symptoms of psoriasis,” said Lloyd Miller, Vice President, Immunodermatology Disease Area Leader, Janssen Research & Development, LLC. “We’re delighted that this therapy, which has a well-established safety and efficacy profile in adults with plaque psoriasis and other immune diseases, is now expanded to children as young as six who are living with this chronic disease.”
The EC approval is based on results from the Phase III CADMUS Jr study, building on the prior Phase III CADMUS study, which found ustekinumab improved the signs and symptoms of plaque psoriasis,as well as health-related quality of life (HrQOL), in paediatric patients aged six to 11 years old. The primary endpoint was the proportion of patients who achieved a physician’s global assessment (PGA) score of Cleared (0) or Minimal (1) at week. Secondary endpoints included the proportion of patients achieving improvements in psoriasis area and severity index of ≥75% (PASI 75), ≥90% (PASI 90), and change from baseline in Children’s Dermatology Life Quality Index (CDLQI) at week 12.
In the study,44 patients (aged 6–11 years) from nine countries were enrolled and treated with at least one injection of ustekinumab. At baseline, the mean duration of psoriasis was 3.5 (standard deviation 2.49) years. At week 12, subjects treated with ustekinumab showed clinically meaningful improvements in their psoriasis and HrQOL. At week 12, 77.3% (95% confidence interval [CI]: 62.2%, 88.5%) achieved PGA 0/1, 84.1% (95% CI: 69.9%, 93.4%) achieved PASI 75, and 63.6% (95% CI: 47.8%, 77.6%) achieved PASI 90. The mean change from baseline in CDLQI was -6.3 (95% CI: -8.29; -4.28, lower is better). All patients were followed for up to 52 weeks after the first administration of ustekinumab. Improvements in PGA0/1, PASI 75, PASI 90 and CDLQI were maintained through to week 52 (75.6%, 87.7%, 70.7%, and 58.3%, respectively).
Safety data from CADMUS Jr were consistent with the known safety profiles reflected in respective current prescribing information labels and ustekinumab was generally well-tolerated by paediatric patients with plaque psoriasis. Overall, 34 patients had more than one adverse event (AE; [77.3%]) and three had more than one serious AE (6.8%). One patient had a serious infection (mononucleosis), 29 had infections (65.9%), and 12 had infections requiring treatment (27.3%). In general, the AEs and other safety data reported up to one year in two paediatric patient studies (CADMUS and CADMUS Jr) were similar to those seen in previous studies in adults with plaque psoriasis.
This marketing authorisation follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), issued on 12 December 2019.
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