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Friday 20 September 2019
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Topic: Paediatrics

September 20, 2019
The FAD is part of the final guidance to the NHS in England and Wales expected to be published in October 2019
Takeda has announced that the National Institute for Health and Care Excellence (NICE) has issued its Final Appraisal Determination (FAD) recommending Takhzyro®▼ (lanadelumab) subcutaneous injection as an option for preventing recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.
 
The recommendation is only if:
  • patients are eligible for preventive C1-esterase inhibitor (C1-INH) treatment in line with NHS England’s commissioning policy
September 3, 2019
For fuss-free measuring of lung function, the phone connects to a wireless spirometer and the app can register respiratory symptoms and provide visual feedback on treatment

A study by researchers at Karolinska Institutet shows that a treatment adjustment algorithm based on lung function and symptoms in a mobile phone can be an efficient tool in managing uncontrolled asthma.

The study is published in the European Respiratory Journal.

August 8, 2019
Dupixent is the first and only biologic to show positive results in this paediatric atopic dermatitis population.
A pivotal Phase III trial evaluating Dupixent® (dupilumab) in severe atopic dermatitis in children has met its primary and secondary endpoints. 
 
August 7, 2019
Dupixent is now the first biologic medicine approved in the EU to treat these patients.

The European Commission has extended the marketing authorisation for Dupixent® (dupilumab) in the European Union to include adolescents 12 to 17 years of age with moderate-to-severe atopic dermatitis who are candidates for systemic therapy.

Dupixent is now the first biologic medicine approved in the EU to treat these patients.
 

August 6, 2019
Although previous studies have found many genes that exert an effect on these diseases, research have been unable to explain the whole genetic background to the origin of asthma, hay fever and eczema
In a new study from SciLifeLab at Uppsala University, researchers have found a total of 141 genes that largely explain the genetic risk underlying asthma, hay fever and eczema. 
 
As many as 41 of the genes identified have not previously been linked to an elevated risk for these diseases. The results are published in the scientific journal Human Molecular Genetics.1
 
August 5, 2019
A significant increase in the incidence of allergies has been registered in developed countries, first beginning in the 1960s and steadily progressing thereafter
A study from NHS Digital – data from which were reported on the BBC website in 20161 –  showed that there had been a 33% increase in admissions for different forms of allergy and an increase of 19% of cases of anaphylactic shock in hospital facilities in the UK in the preceding five years.
 
And the UK is not alone in this, in that a significant increase in the incidence of allergies has been registered in developed countries, first beginning in the 1960s and steadily progressing thereafter. 
July 29, 2019
The discovery adds further evidence to support the prenatal sex steroid theory of autism first proposed 20 years ago
Scientists have identified a link between exposure to high levels of oestrogen sex hormones in the womb and the likelihood of developing autism. 
 
The findings have been published in the journal Molecular Psychiatry.1
 
July 15, 2019
Researchers have identified new biomarkers for IBS in urine, which could lead to better treatments and reduce the need for costly and invasive colonoscopy procedures currently used for diagnosis
Scientists at McMaster University in Canada have identified new biomarkers for irritable bowel syndrome (IBS) in urine, which could lead to better treatments and reduce the need for costly and invasive colonoscopy procedures currently used for diagnosis.
 
July 8, 2019
Carefully designed, integrated multi-'omic' studies could accelerate the use of precision medicine for asthma patients, according to researchers from the Icahn School of Medicine at Mount Sinai, USA
In an invited review article published in the Journal of Allergy and Clinical Immunology, Scott Tyler, PhD, and Supinda Bunyavanich, MD, MPH, report that numerous studies have shown the value of applying transcriptomics and other 'omic' approaches for defining asthma subtypes - but they also cite the need for more studies aimed at pulling together these disparate data streams for a more comprehensive view of the disease.
 
July 2, 2019
'Back to school asthma' - a seasonal peak in cases associated with the start of the school year in September - is linked to a tripling in the rate of GP appointments across England

'Back to school asthma’- a seasonal peak in cases associated with the start of the school year in September - is linked to a tripling in the rate of general practitioner (GP) appointments across England, reveals research published online in the Journal of Epidemiology & Community Health.1

July 1, 2019
Even when rhinitis is mild, it is important to accurately diagnose its pathology, understand which allergens trigger it, and follow the prescribed therapy to avoid the condition deteriorating and becoming worse
Allergic rhinitis is one of the most frequently neglected and underappreciated diseases. When the symptoms are limited to sneezing and itchy eyes, many sufferers decide to treat themselves with do-it-yourself remedies or remedies recommended by friends and relatives; however, this is a mistake.
 
July 1, 2019
Research suggests that fats can help to aggravate the T-cell activated inflammation in the lungs that is seen in asthma
Researchers at Karolinska Institutet in Sweden have studied which genes are expressed in overactive immune cells in mice with asthma-like inflammation of the airways. Their results, which are published in the journal Immunity, suggest that the synthesis and breakdown of fats plays an important part in the process.
 
Th2 cells plays a vital part in asthma-related inflammation, but the rarity of these cells and a lack of sensitivity technology has made these cells hard to study in any detail.
 
July 1, 2019
New Danish research may help direct focus towards the serious complications that on average every fifth haematological cancer patient suffers
New Danish research may help direct focus towards the serious complications that on average every fifth haematological cancer patient suffers. 
 
This is according to medical doctor and PhD Kasper Adelborg from Aarhus University and Aarhus University Hospital, who has studied the cases of 32,000 haematological cancer patients between the years 2000-2013. Haematological cancer includes leukaemia, bone marrow cancer and cancers of the lymph nodes.
 
June 20, 2019
The purpose of the study was to assess the drug’s pharmacokinetics, efficacy and safety for treatment of MAS and to confirm the proposed dose regimen
SOBI has announced that new research demonstrating the effects of emapalumab in patients with macrophage activation syndrome (MAS), a form of secondary haemophagocytic lymphohistiocytosis (HLH) complicating systemic juvenile idiopathic arthritis (sJIA), was presented at the EULAR/Paediatric Rheumatology European Society (PReS) Scientific Congress in Madrid.  
 
June 13, 2019
Study reports no vaccine infections in children given live-attenuated booster vaccine while on immune suppressing therapies
A study presented at EULAR 2019, jointly organised with the Paediatric Rheumatology Society (PReS), demonstrate no vaccine infections, and no disease flare, in the 234 rheumatic patients who received live-attenuated booster vaccination while taking immune suppressing therapies.1
 
June 7, 2019
Experts from Norway found that the risk of paediatric coeliac disease was 8% lower per 10g increase in fibre intake during pregnancy
High fibre intake during pregnancy is linked with a decreased risk of coeliac disease in children, research presented at the 52nd Annual Meeting of the European Society of Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) has shown.1 
 
June 6, 2019
Scientists have found that young children with severe eczema infected with Staphylococcus aureus are at a higher risk of developing a food allergy
In a study published in the Journal of Allergy and Clinical Immunology, scientists from King's College London have found that young children with severe eczema infected with Staphylococcus aureus (SA) bacterium, are at a higher risk of developing a food allergy.1
 
Staphylococcus aureus (SA) is found in the nose and the skin of healthy individuals; however, SA is more common in sufferers of eczema, especially severe eczema.
 
May 29, 2019
Among 20 children with EoE who wore the Viaskin Milk patch, nine saw an improvement in their symptoms and normalisation of their biopsies after 11 months

A study from Children's Hospital of Philadelphia (CHOP) finds that a skin patch may be useful in treating children with a painful, chronic condition called eosinophilic oesophagitis (EoE) triggered by milk.1

May 20, 2019
Over 870,000 people have taken part in clinical trials in 2018/2019 and this number marks a significant step towards the NHS Long Term Plan’s goal of reaching one million participants by 2023/24
The past 12 months has shown a positive rise in people benefiting from clinical research in England, as numbers have reached record highs with over 870,000 participants involved in clinical research studies supported by the National Institute for Health Research (NIHR) over the last year.
 
May 17, 2019
Faecal microbiota transplant, or the transfer of stool from a healthy donor to a patient, has been found highly effective in reversing severe Clostridiodes difficile diarrheal infections in adults
A new study in the journal Clinical Gastroenterology and Hepatology, the largest faecal microbiota transplant (FMT) study in children to date, found it both safe and effective and also identifies predictors of success.
 
April 29, 2019
The diagnosis of the causes of urticaria is very challenging, because very many physiological or pathological conditions are associated with the appearance of hives
Urticaria is a clinical condition characterised by the presence of skin lesions, called wheals. Typically, these are intensely itchy and surrounded by an area of erythema. Wheals can have a diameter from a few millimetres or appear as confluent lesions with a diameter of several centimetres. In some cases, urticaria is associated with angioedema, which appears as a swelling of soft tissues from fluid accumulation due to dermal oedema.
 
April 25, 2019
A report describes how a new battery of tests enables researchers to distinguish patients with IBS from healthy children and identifies correlations between certain microbes and metabolites with abdominal pain
To improve the treatment of children with IBS, investigators have developed a sophisticated way to analyse the microbial and metabolic contents of the gut. 
 
April 24, 2019
Research illuminates the relationship between eczema and food allergy

Scratching the skin triggers a series of immune responses culminating in an increased number of activated mast cells in the small intestine, according to research conducted in mice.

This newly identified skin-gut communication helps illuminate the relationship between food allergy and atopic dermatitis. The study was supported by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and led by researchers at Boston Children's Hospital.1

April 15, 2019
Identification of the allergic triggers of rhinitis is crucial in order to implement adequate avoidance measures in both atopic and non-atopic patients; moreover, this identification can help administer specific therapies, such as allergen immunotherapy
Rhinitis is an inflammatory disorder of the nasal mucosa clinically defined by two or more symptoms of nasal itching, sneezing, anterior or posterior rhinorrhoea, and nasal blockage.1