Signal Lorazepam confirmed as first-line treatment for stopping prolonged seizures in children

NIHR Signal Lorazepam confirmed as first-line treatment for stopping prolonged seizures in children


Published on 17 April 2018


Intravenous lorazepam is as effective as intravenous diazepam for stopping children’s tonic-clonic seizures in hospital. https://discover.dc.nihr.ac.uk/content/signal-00586/lorazepam-confirmed-as-first-line-treatment-for-stopping-prolonged-seizures-in-children
From the NIHR Dissemination Centre

Drug company says it will negotiate terms to allow children to access new treatment for rare genetic disorder

Drug company says it will negotiate terms to allow children to access new treatment for rare genetic disorder.


This preliminary decision, which is now the subject of public consultation until 5 March, comes as the company confirmed it is to negotiate with NHS England terms that could allow the drug to be made available while uncertainties in the evidence of its long term benefits are addressed.

Children with CLN2, which is also known as Batten Disease, live to between 8 years and early adolescence; the average life expectancy is 10 years. It is estimated that in the UK there are around 30 to 50 children living with the condition.

CLN2 is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. This results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.

Symptoms in children with CLN2 begin from around the age of 2 and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.

There is currently no cure or life-extending treatments for CLN2 and clinical management is limited to symptom relief and supportive and palliative care.

Costing over £500,000 for each year’s treatment, cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device.


See the full details from NICE at https://www.nice.org.uk/news/article/drug-company-says-it-will-negotiate-terms-to-allow-children-to-access-new-treatment-for-rare-genetic-disorder

UHCW publication: Clarithromycin as a steroid sparing agent for the management of infantile bullous pemphigoid

Clarithromycin as a steroid sparing agent for the management of infantile bullous pemphigoid Simon Tso, Gabriela Petrof, Simon Unter, Frances Humphreys
BMJ Case Reports 2018; doi:10.1136/bcr-2017-223507
http://casereports.bmj.com/content/2018/bcr-2017-223507.full



You can publish in BMJ Case Reports with the UHCW Fellowship Code. Ask in the CSB Library for more details.

Management of Infants born to Mothers who have used Opioids during Pregnancy

The incidence of infant opioid withdrawal has grown rapidly in many countries, including Canada, in the last decade, presenting significant health and early brain development concerns. Increased prenatal exposure to opioids reflects rising prescription opioid use as well as the presence of both illegal opiates and opioid-substitution therapies. Infants are at high risk for experiencing symptoms of abstinence or withdrawal that may require assessment and treatment.

This practice point focuses specifically on the effect(s) of opioid withdrawal and current management strategies in the care of infants born to mothers with opioid dependency.

Next-generation sequencing panel for solid tumour cancers in children [MIB133]

New:  Medtech innovation briefing

The next-generation sequencing (NGS)-based panel for solid tumour cancers in children is an in-house laboratory test, not a commercial product. It was developed at the Centre for Molecular Pathology at The Institute of Cancer Research and The Royal Marsden NHS Foundation Trust and validated in-house for clinical application, following the standardised framework published by Mattocks et al. (2010).

Common Paediatric Elbow Injuries

The Open Orthopaedics Journal, 11(Sup 8, M6) p1380-1393. DOI: 10.2174/1874325001711011380

Paediatric elbow injuries account for a large proportion of childrens’ fractures. Knowledge of common injuries is essential to understanding their assessment and correct management. Understanding of the ossification centres around the paediatric elbow is essential to correctly assessing and managing the common injuries that we have discussed in the review. Outcomes after these injuries are usually favourable with restoration of normal anatomy.

UHCW Research: Christopher E. Hill, Stephen Cooke

A regressing spindle cell tumour of Reed

BMJ Case Reports 2017; doi:10.1136/bcr-2017-222002

Spindle cell tumour of Reed is a benign melanocytic naevus which usually presents as a deeply pigmented mole. It is more commonly found on the lower extremities of young girls. It is an uncommon naevus but its incidence and prevalence are not known. A Reed naevus typically goes through a rapid initial growth phrase before stabilising in size and then regresses over time. Awareness of Reed naevus has been demonstrated to be low, even among dermatology doctors.

UHCW Research: Simon Tso, William Hunt and Joanna E Gach

Safe, sustainable and productive staffing for neonatal care and children and young people's services

Draft resources from NHS Improvement to help standardise safe, sustainable and productive staffing decisions in neonatal care and children and young people's services.

NIHR Signal Placing wet gauze on babies’ tummies speeds up urine collection

Almost a third of infants managed to urinate within five minutes after a painless, cheap technique that stimulates the skin, compared with 12% of infants observed only, as is standard practice.

The ‘Quick-Wee’ method involved rubbing the babies’ abdomens gently with gauze soaked in cold saline before collecting urine. This trial was carried out with 354 babies aged one to 11 months in one Australian paediatrics emergency room.

NICE guidelines recommend non-invasive ‘clean catch’ collection if possible but this can be difficult, especially in young children. They estimate that 20 minutes of staff time can be needed to obtain a sample. Hospital paediatrics settings and primary care may want to try this promising, cheap and safe technique.

There was no difference in rates of contamination and it may reduce the number of babies who need to go on to the less desirable collection by pad samples or invasive techniques.

From the NIHR Dissemination Centre

NIHR Signal Simple preventive actions by parents linked to fewer child injuries

Education is promoted as a way to tackle the scale of avoidable injuries to young children. Children have two to five times the risk of an accident leading to injury if a parent leaves them on a raised surface, places hot drinks within reach, or does not put medicines away straight after use.
For example, children are also more than twice as likely to attend hospital for falling on stairs if their parent leaves stair gates open or does not use them.


From the NIHR Dissemination Centre

NICE approves gene therapy for rare ‘bubble baby syndrome’

Strimvelis, a treatment for an ultra-rare inherited immune deficiency condition that has been dubbed ‘bubble baby syndrome’ has been approved by NICE in draft guidance.

Immunosuppressive therapy for kidney transplant in children and young people [TA481]

New Guidance from NICE:

Evidence-based recommendations on immunosuppressive therapies for preventing kidney rejection in children and young people. The therapies are basiliximab (Simulect), immediate-release tacrolimus (Adoport, Capexion, Modigraf, Prograf, Tacni, Vivadex), mycophenolate mofetil (Cellcept and non-branded versions), rabbit anti-human thymocyte immunoglobulin (Thymoglobuline), prolonged-release tacrolimus (Advagraf, Envarsus), mycophenolate sodium (Myfortic, Ceptava), sirolimus (Rapamune), everolimus (Certican) and belatacept (Nulojix).

This guidance makes recommendations on using basiliximab, rabbit anti-human thymocyte immunoglobulin, tacrolimus (immediate-release and prolonged-release), mycophenolate mofetil, mycophenolate sodium, sirolimus, everolimus and belatacept after kidney transplant in children and young people. The recommendations apply only to the initial immunosuppressive therapy (induction and maintenance therapy) started around the time of kidney transplant.
It was outside the scope of the appraisal to make recommendations on using azathioprine or corticosteroids after kidney transplant in children and young people.
Under an exceptional directive from the Department of Health, the appraisal committee was allowed to make recommendations about using drugs outside the terms of their marketing authorisations if there was compelling evidence of their safety and effectiveness.

Urinary tract infection in under 16s: diagnosis and management [CG54] / QS36

Updated. Guidanca from NICE. It covers diagnosing and managing first or recurrent upper or lower urinary tract infections in infants, children and young people. It aims to achieve more consistent clinical practice, based on accurate diagnosis and effective management.

This guideline includes recommendations on:

• investigations and diagnosis
• acute management
• imaging tests
• surgical intervention
• follow-up
• information and advice for children, young people and parents or carers

See also Urinary tract infection in children and young people quality standard.

Thora-3Di for assessing asthma in children [MIB122]

New NICE Advice Medtech innovation briefing

The technology described in this briefing is Thora‑3Di for assessing respiratory function in children with asthma.

The innovative aspects are that the measurements are taken non-invasively without the need for special breathing manoeuvres, and provide information on right-versus-left lung function.

Risk of severe harm and death from infusing total parenteral nutrition too rapidly in babies

A warning alert has been issued by NHS Improvement on the risk of harm and death from infusing total parenteral nutrition too rapidly in babies.

New guidance on radiological investigations of suspected child abuse

The guidance, entitled “The Radiological Investigation of Physical Abuse in Children”, has been jointly produced by The Royal College of Radiologists (RCR) and the Society and College of Radiographers (SCoR), with input and endorsement from the Royal College of Paediatrics and Child Health (RCPCH).

It revamps initial guidance from 2008 and incorporates new evidence on child imaging techniques and follow-up protocols, as well as a series of new procedural templates to help radiologists, radiographers and referring paediatricians.

Quality Standards for Paediatric Gastroenterology, Hepatology & Nutrition

The Royal College of Paediatrics and Child Health and the British Society of Paediatric Gastroenterology, Hepatology and Nutrition have worked together to develop these new standards.

The standards apply to children receiving gastroenterology, hepatology and nutrition care in the secondary and tertiary care settings across the UK.

End of life care for infants, children and young people [QS160]

New quality standard covers end of life care for infants, children and young people (from birth to 18 years) who have a life-limiting condition. Life-limiting conditions are those that are expected to result in an early death for the person. It also covers support for family members and carers. It describes high-quality care in priority areas for improvement.

Ability of a fecal occult blood test in ruling out intussusception in children.

A negative fecal occult blood test cannot reliably rule out the diagnosis of intussusception.

Prescribing regular drugs to prevent febrile convulsions risks more harm than benefit

The benefits of giving anti-epileptic medication to children aged between six months and seven years who have had a convulsion while feverish, do not outweigh the harms. While diazepam given when a child becomes feverish reduced the chance of a convulsion from about 25% to 18% after a year, one in three children experienced adverse effects. Therefore, the authors suggest benefits do not seem to outweigh the harms.Prescribing regular drugs to prevent febrile convulsions risks more harm than benefit

From the NIHR Dissemination Centre