Children and young people with severe neurodisabling conditions (CYPSND)experience severe functional gastrointestinal symptoms and dependence on artificial nutrition. 'Gastrointestinal dystonia' (GID) has been applied by clinicians when symptoms become debilitating and potentially life-limiting. Evidence is lacking regarding the definition and appropriate management of GID. We therefore assembled a RAND appropriateness panel. We performed a systematic review, created an online survey and distributed this to a panel of 27 experts from five stakeholder groups from 13 UK specialist centres across the British Isles (gastroenterology, neurology/neurodisability, surgery, palliative care and allied health professionals). A Disagreement Index ≥1 indicated disagreement. The panel rated the appropriateness of 250 statements covering the following in GID: definition, clinical evaluation, nutritional assessment/feeding strategies, investigations, medications and prescribing, surgical interventions, safeguarding, palliative care and ethics. Agreement was reached except in selected statements regarding uncommon diagnostic features. There was uncertainty in specific clinical scenarios regarding: investigation, the use of blenderised diet, certain pharmacological agents and surgical interventions. The only intervention deemed inappropriate was antireflux surgery in the context of GID and gastrointestinal dysmotility without reflux disease. The remaining statements (198) were considered appropriate. We present a comprehensive review, agreement on the definition of GID and recommendations on management pathways agreed by a selected panel of multidisciplinary experts. Clear diagnostic criteria will enable important epidemiological work to record outcomes for this complex patient group. Identifying the associated morbidity, burden of care and mortality will help advocate for appropriate health resources and support to carers and families.

OBJECTIVES:We aimed to study the risks of relapse and long term disability in children with non-MS acquired demyelinating syndromes (ADS). METHODS:In this prospective, multi-centre study, from the 14 UK pediatric neurology centres, children (<16 years) experiencing a first episode of ADS were recruited from 2010 to 2014. Case report forms were collected prospectively. RESULTS:A total of 269 children were recruited and followed up for a median of 7.2 years. Median age at onset was 9y (IQR 9.5-14.5, 126 females). At last follow-up, 46 (18 %) had MS, 4 AQP4-Ab NMOSD and 206 (80 %) had other ADS, of which 27 (13 %) relapsed. Relapsing MOGAD was the diagnosis in 12/27, 6 were seronegative and 9 did not have antibodies tested. Frequency of relapse differed according to first presentation in non-MS ADS, being least likely in transverse myelitis (p = 0.025). In the non-MS group, MOG-Ab was predictive of relapse (HR = 8.42; p < 0.001) occurring 8 times as often decreasing over time. Long-term difficulties did not differ between children with monophasic vs relapsing diseases. CONCLUSION:The risk of relapse in non-MS ADS depends on initial diagnosis, and MOG-Ab positivity. Long-term difficulties are observed regardless of relapses and are determined by presenting phenotype.

BackgroundIdiopathic intracranial hypertension (IIH) is a potentially disabling condition. There is a lack of evidence and national guidance on how to diagnose and treat paediatric IIH, leading to variation in clinical practice. We conducted a national Delphi consensus via the Children’s Headache Network to propose a best-practice diagnostic and therapeutic pathway.MethodsThe Delphi process was selected as the most appropriate methodology for examining current opinion among experts in the UK. 104 questions were considered by 66 healthcare professionals, addressing important aspects of IIH care: assessment, diagnosis, treatment, follow-up and surveillance. General paediatricians, paediatric neurologists, ophthalmologists, opticians, neuroradiologists and neurosurgeons with a clinical interest or experience in IIH, were invited to take part.ResultsThe Delphi process consisted of three rounds comprising 104 questions (round 1, 67; round 2, 24; round 3 (ophthalmological), 13) and was completed between March 2019 and August 2021. There were 54 and 65 responders in the first and second rounds, respectively. The Delphi was endorsed by the Royal College of Ophthalmologists, which engaged 59 ophthalmologists for round 3.ConclusionsThis UK-based Delphi consensus process reached agreement for the management of paediatric IIH and has been endorsed by the Children’s Headache Network and more broadly, the British Paediatric Neurology Association. It provides a basis for a pragmatic clinical approach. The recommendations will help to improve clinical care while minimising under and over diagnosis.

Background For children and young people with severe neurodisabling conditions (CYPSND), the clinical constellation of pain behaviour, retching, bloating, abdominal distension and constipation/pseudo-obstruction can be referred to as gastrointestinal dystonia (GID). Nabilone, a synthetic analogue of the active component tetrahydrocannabinol found in cannabis, has a licence in paediatrics for the treatment of severe chemotherapy-induced nausea. We aim to describe our experience of using nabilone for CYPSND with GID. Methods Approval was sought on a named patient basis from the clinical directorate and lead pharmacist on the basis that; patients fulfilled our criteria for GID, patients had been trialled on medical therapies, jejunal feeding and blended diet, concomitant medication reviewed in multidisciplinary team including tone management and to review potential exacerbants to GID. Patients were admitted for 48 hours for monitoring of temperature, pulse, respiratory rate, oxygen saturations and symptoms using a modified paediatric pain score. Nabilone was commenced at 250 mu g daily and then incremented in 250 mu g doses to a dosage of <18 kg: 500 g two times per day and 18-27 kg: 500 mu g three times a day. Efficacy was assessed by parents' and clinicians' perception, PedsQL V.3.0 (gastrointestinal subset questions) and sleep diary, prior to treatment, 1 month and 6 months after stable dose, and median scores were analysed by paired Student's t-test (p<0.05). Results In four of five suitable patients, the PedsQL score was higher after commencing treatment and there was a sustained improvement at 6 months. Patient median total PedsQL Quality of Life (QOL) scores were 46 (28-50) prior to treatment and 68.5 (58-74) 1 month after commencing treatment (p=0.032.) After 6 months, the median PedsQL QOL score was 73 (51-82) (p=0.017.) Conclusions Nabilone shows promise in treating GID, a poorly understood debilitating complication of severe neurodisability. In particular, symptoms of nausea, retching and pain on feeding were all reduced in our cohort.

Aims Idiopathic Intracranial Hypertension (IIH) is associated with headaches and a potential loss of vision. The prevalence may rise with childhood obesity. As there is no strong evidence to support the way IIH is diagnosed or treated, it is important to establish consensus to guide management and identify areas of uncertainty for further research. We conducted a national Delphi consensus process to inform a national guideline for the management of IIH in children and young people. Methods The Delphi focused on all aspects of IIH including initial assessments (referral, assessments, laboratory tests, LP, ophthalmology assessments), diagnosis (criteria and terminology) and treatment (including conservative, drug and neurosurgical interventions), follow-up, and surveillance. General paediatricians, paediatric neurologists, ophthalmologists, opticians, neuroradiologists, and neurosurgeons known to have a clinical interest or experience in IIH were invited to take part. The charity IIH-UK contributed to represent patients and their families. A priori consensus was defined as 70% agreement. Results Recommendations are proposed, based on areas of consensus and relate to aspects of IIH patient management, including: timing of assessment, baseline assessments and investigations, recommended diagnostic criteria, LP and CSF pressure interpretation, neuroimaging, MDT meetings, ophthalmological assessments, method for LP (including use of local and general anaesthetic), dietary recommendations, acetazolamide use and neurosurgical management. (For detail please refer to table 2) Conclusion This new UK consensus for the management and surveillance of IIH provides a realistic and pragmatic approach, based on expert opinion for best clinical care for children and young people with IIH. We hope these recommendations will minimise under and over diagnosis, improve the care offered, and outcomes obtained.

Background and Aims Children and young people with severe neurosdisabling conditions (CYPWSND) experience an array of serious gastrointestinal symptoms beyond gastro-oesophageal reflux, constipation or dependence on artificial nutrition. When enteral feeds leads to disabling dystonia the term 'gastrointestinal dystonia of severe neurodisability' (GID) has been applied by clinicians. However a clear definition with criteria for entry point is lacking in the literature. We describe the methods for formal establishment of an agreed definition of GID. Methods After commissioning by BSPGHAN, systematic review1 and consultation with public bodies it was agreed, due to paucity of evidence that an appropriateness panel should be the forum for formulation of output on GID. A writers group structured the questions for the survey definition, based on the limited written evidence and added professional experience. A panel of 27 experts in their field were assembled from 5 stakeholder groups including: Gastroenterology, Neurology/Neurodisability, Surgery, Palliative Care and Allied Health Professionals. Geographic representation was from 13 UK specialist centres (including all 4 nations) and 1 centre from Republic of Ireland. The panel rated the appropriateness of definition, investigations and management of GID. A scale of 1–9 enabled scoring of 1–3 to indicate inappropriate, 4–6 uncertain, 7–9 appropriate as criteria for recommendation. Panel agreement index was calculated using a continuous likelihood ratio, with <1 indicated 'general agreement' and >1 'no agreement'. Results were discussed at a moderated. Results All of the panel completed all questions on 'common' (table 1) and 'uncommon' features of GID. The panel had strong concurrence that GID definition required patients have GMFCS 4–5 cerebral palsy or equivalent and that a temporal relationship between symptoms and enteral feeding had to be present (although this relationship may lessen or cease during progressive disease). Pain, distress, retching, autonomic activation and hypertonicity were seen as common features. Temporal relationship with bowel habit, involuntary movements were considered less common. The diagnosis should be a positive clinical diagnosis (not of exclusion) made by a specialist multi-disciplinary team with experience of feeding disorders in severe neuro-disability. Features suggesting patients feed intolerance has reached the threshold for GID would include malnutrition primarily due to feed cessation and GI symptoms being the greatest burden on QOL for patient/family on appropriate survey. Conclusions We present a coherent first definition for GID by consensus of a panel of identified experts drawn from 5 invested stakeholder groups. Clear entry point for diagnosing GID will allow for important epidemiological work to report investigations, interventions and outcomes for this complex group of patients. Identifying significant morbidity care burden and mortality in this patient group will help advocate for appropriate health resources, support to carers and families. The ongoing development of a management framework through completion of the RAND2 process in 2022 should assist navigation of the complex medical and ethical challenges of management of distressing and debilitating symptoms for patients with this condition. Reference McConnell N, Beattie LM, Richards CA Protheroe S, Barclay AR. JPGN; 2018: 1002 https://www.rand.org/pubs/monograph_reports/MR1269.html Acknowledgement BSPGHAN BiG funding 2020.