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Objectives: The management of paediatric craniopharyngiomas was traditionally complete resection (CR), with better reported tumour control compared to that by partial resection (PR) or limited surgery (LS). The subsequent shift towards hypothalamic sparing, conservative surgery with adjuvant radiotherapy (RT) to any residual tumour aimed at reducing neuroendocrine morbidity, has not been systematically studied. Hence, we reviewed the sequelae of differing management strategies in paediatric craniopharyngioma across three UK tertiary centres over four decades. Methods: Meta-data was retrospectively reviewed over two periods before (1973–2000 (Group A: n = 100)) and after (1998–2011 (Group B: n = 85)) the introduction of the conservative strategy at each centre. Results: Patients had CR (A: 34% and B: 19%), PR (A: 48% and B: 46%) or LS (A: 16% and B: 34%), with trends reflecting the change in surgical approach over time. Overall recurrence rates between the two periods did not change (A: 38% vs B: 32%). More patients received RT in B than A, but recurrence rates were similar: for A, 28% patients received RT with 9 recurrences (32%); for B, 62% received RT with 14 recurrences (26%). However, rates of diabetes insipidus (P = 0.04), gonadotrophin deficiency (P < 0.001) and panhypopituitarism (P = 0.001) were lower in B than those in A. In contrast, post-operative obesity (BMI SDS >+2.0) (P = 0.4) and hypothalamic (P = 0.1) and visual (P = 0.3) morbidity rates were unchanged. Conclusion: The shift towards more conservative surgery has reduced the prevalence of hormone deficiencies, including diabetes insipidus, which can be life threatening. However, it has not been associated with reduced hypothalamic and visual morbidities, which remain a significant challenge. More effective targeted therapies are necessary to improve outcomes. [ABSTRACT FROM AUTHOR]

Congratulations on your March 12 issue, which I thought was truly over the top. I particularly appreciated Andrew Greeley's pertinent and deftly written article on 'Reforming the Church' (Hope you [...]

\nIntroduction: Quantifying differences in service provision for children and young people (CYP) living with congenital adrenal hyperplasia (CAH) across the UK. Methods: A national service evaluation using online questionnaires circulated to patients and clinicians from secondary and tertiary UK centres managing CYP with CAH and via the “Living with CAH” support group mailing list. Results: Total of 195 responses relating to patients aged 0–20 years attending 33 clinics (43 patients, 152 carers), as well as 34 clinicians from 18 trusts working across the 33 clinics. Only 12% of clinicians were “completely satisfied” with the service provided, compared to 68% of carers and 76% of patients. While 94% of clinicians reported providing formal training to families with CAH, over 80% of both patients and carers reported not attending what they considered formal training. Appetite for further training was higher in carers (86%) than patients (55%), although further “unsure” responses suggested formal training sessions would likely be well attended. Access to psychological services was difficult for 44% of clinicians. Biochemical monitoring of treatment was broadly in keeping with international guidelines, with 67% of clinicians reporting regular use of dried blood spots and 12% reporting regular urinary steroid metabolites. Conclusion: While there is overall good satisfaction with care provision among patients and carers with CAH in the UK, extra resources addressing the psychological and educational needs about the disease and its management would benefit patients and carers. Improved access to allied health professionals and psychologists will help support families and improve patient outcomes. Congenital adrenal hyperplasia (CAH) is a lifelong condition where the body does not produce enough steroids, and thus patients need to take daily steroid replacement. There are differences of opinion in how CAH is treated, and we aimed to find out about these differences in the UK. We surveyed children and young people living with CAH, their parents or carers, and doctors and nurses who look after them throughout the UK. We received 152 responses from carers, 43 from patients, and 34 from clinicians from a total of 33 different clinics. Overall satisfaction with services was high, although, while 94% of clinicians said they provided formal training to families with CAH, over 80% of both patients and carers said that they had not attended what they considered formal training. Most patients and carers said that they would like to engage with further training. Clinicians said they wanted better access to other support for patients, including psychologists. Treatment of CAH was generally in keeping with international guidelines. New methods of monitoring, such as dripping blood onto a card instead of attending hospital for a formal blood test, are increasing with two-thirds of clinicians reporting this method being used. In conclusion, there is good satisfaction with care provision among patients and carers with CAH in the UK. However, there are opportunities for increasing psychological support and family education about the condition to further improve the lives of those living with this disease. [ABSTRACT FROM AUTHOR]

Paracetamol overdose is a leading cause of acute liver failure that can prove fatal. Establishing paracetamol concentration accurately and quickly is critical. Current detection methods are invasive, time-consuming and/or expensive. Non-invasive, rapid and cost-effective techniques are urgently required. To address this challenge, a novel approach, called Paper-Arrow Mass Spectrometry (PA-MS) has been developed. This technique combines sample collection, extraction, enrichment, separation and ionisation onto a single paper strip, and the entire analysis process, from sample to result, can be carried out in less than 10 min requiring only 2 μL of raw human saliva. PA-MS achieved a LOQ of 185 ng mL−1, mean recovery of 107 ± 7%, mean accuracy of 11 ± 8% and precision ≤5% using four concentrations, and had excellent linearity (r2 = 0.9988) in the range of 0.2–200 μg mL−1 covering the treatment concentration range, surpassing the best-in-class methods currently available for paracetamol analysis. Furthermore, from a panel of human saliva samples, inter-individual variability was found to be <10% using this approach. This technique represents a promising tool for rapid and accurate emergency diagnosis. [ABSTRACT FROM AUTHOR]

Background: Hydrocortisone is the preferred treatment for adrenal insufficiency in childhood. A small minority of children experience low cortisol concentrations and symptoms of cortisol insufficiency, poorly responsive to modifications in dosing. We speculated that treatment with modified-release hydrocortisone Plenadren® may be beneficial in these selected patients.Objective: The aim of this article was to report cortisol profiles during treatment with standard formulation hydrocortisone and Plenadren, and growth and weight gain during treatment with Plenadren in selected children with adrenal insufficiency.Patients and Methods: Data are reported as median (range). Eight patients (5 male) age 11.0 years (8.8-13.3), with adrenal insufficiency for 4.3 years (2.2-10.0) were treated with Plenadren in doses derived from cortisol concentrations measured during treatment with standard formulation hydrocortisone.Results: Plasma cortisol was 262 nmol/L (114-654) 2 h after the morning dose (hydrocortisone dose 6.1 mg/m2 [4.3-7.1]) of standard formulation hydrocortisone. After 4 h, cortisol concentration was 81 nmol/L (56-104) and was < 100 nmol/L in six patients. Two hours after Plenadren administration (hydrocortisone dose 12.1 mg/m2 [8.3-17.6]), plasma cortisol concentration was 349 nmol/L (150-466), and after 4 h it was 239 nmol/L (99-375) and < 100 nmol/L in one patient. Six hours after the Plenadren dose, cortisol concentration was < 100 nmol/L in four patients and after 8 h cortisol concentration was < 100 nmol/L in seven patients (sample not obtained in one patient). Six patients elected to continue treatment with Plenadren. After 4.2 years (2.7-6.0), change in height standard deviation score (SDS) was 0.1 SD (− 0.2 to 0.2) and body mass index SDS was 0.3 SD (0-1.1).Conclusion: Smoother cortisol profiles and more sustained cortisol exposure were achieved during treatment with Plenadren, which was the preferred treatment in most patients. Robust clinical trials are required to determine the place of this medication in paediatric practice. [ABSTRACT FROM AUTHOR]

Pituitary adenomas are rare in children and young people under the age of 19 (hereafter referred to as CYP) but they pose some different diagnostic and management challenges in this age group than in adults. These rare neoplasms can disrupt maturational, visual, intellectual and developmental processes and, in CYP, they tend to have more occult presentation, aggressive behaviour and are more likely to have a genetic basis than in adults. Through standardized AGREE II methodology, literature review and Delphi consensus, a multidisciplinary expert group developed 74 pragmatic management recommendations aimed at optimizing care for CYP in the first-ever comprehensive consensus guideline to cover the care of CYP with pituitary adenoma. Part 2 of this consensus guideline details 57 recommendations for paediatric patients with prolactinomas, Cushing disease, growth hormone excess causing gigantism and acromegaly, clinically non-functioning adenomas, and the rare TSHomas. Compared with adult patients with pituitary adenomas, we highlight that, in the CYP group, there is a greater proportion of functioning tumours, including macroprolactinomas, greater likelihood of underlying genetic disease, more corticotrophinomas in boys aged under 10 years than in girls and difficulty of peri-pubertal diagnosis of growth hormone excess. Collaboration with pituitary specialists caring for adult patients, as part of commissioned and centralized multidisciplinary teams, is key for optimizing management, transition and lifelong care and facilitates the collection of health-related quality of survival outcomes of novel medical, surgical and radiotherapeutic treatments, which are currently largely missing., (© 2024. Springer Nature Limited.)

Tumours of the anterior part of the pituitary gland represent just 1% of all childhood (aged <15 years) intracranial neoplasms, yet they can confer high morbidity and little evidence and guidance is in place for their management. Between 2014 and 2022, a multidisciplinary expert group systematically developed the first comprehensive clinical practice consensus guideline for children and young people under the age 19 years (hereafter referred to as CYP) presenting with a suspected pituitary adenoma to inform specialist care and improve health outcomes. Through robust literature searches and a Delphi consensus exercise with an international Delphi consensus panel of experts, the available scientific evidence and expert opinions were consolidated into 74 recommendations. Part 1 of this consensus guideline includes 17 pragmatic management recommendations related to clinical care, neuroimaging, visual assessment, histopathology, genetics, pituitary surgery and radiotherapy. While in many aspects the care for CYP is similar to that of adults, key differences exist, particularly in aetiology and presentation. CYP with suspected pituitary adenomas require careful clinical examination, appropriate hormonal work-up, dedicated pituitary imaging and visual assessment. Consideration should be given to the potential for syndromic disease and genetic assessment. Multidisciplinary discussion at both the local and national levels can be key for management. Surgery should be performed in specialist centres. The collection of outcome data on novel modalities of medical treatment, surgical intervention and radiotherapy is essential for optimal future treatment., (© 2024. Springer Nature Limited.)

In 1997 a survey identified a general lack of standardisation of blood pressure (BP) measurement and little consensus on the criteria for diagnosing hypertension amongst paediatricians. We have conducted a new online survey in 2021, to compare clinical practice between the two time periods. A national quality improvement survey was approved by the GAPRUKI committee and then circulated to consultant-grade general paediatricians. 125 analysable replies from 34 different sites were received and compared with the 1997 data. 106 (84.8%) reported clinic nurse involvement in BP measurement, more than twice than reported previously (40.6%). Most paediatricians (53.6%) now rely on oscillometric devices, whereas the mercury sphygmomanometer was favoured previously (82.7%). If assessing BP manually (n = 89), most (79.8%) now use Korotkoff phase V as the auscultatory endpoint for diastolic BP (phase IV was previously used (52.1%)). Diagnostic criteria of hypertension, the criteria (≥95 th centile for gender, age and height) were constant, and 100% of paediatricians diagnosed it using systolic BP, but only 43 (34.4%) used diastolic BP, a decrease from 79.4% previously. Ambulatory BP Monitoring was six times more available than in 1997 (81.6% vs 13.6%). Similar to previous findings, only 12 (9.6%) paediatricians would manage hypertensive patients themselves, however 82 (72.6%) would keep general paediatric input. There have been important changes in the assessment of BP in children, including increased nurse involvement and greater use of technology. However, fewer paediatricians are responding to high diastolic pressures than twenty years ago., (© 2023. The Author(s).)

Context: Somapacitan is a long-acting GH derivative for treatment of GH deficiency (GHD)., Objective: Evaluate the efficacy and tolerability of somapacitan in children with GHD after 2 years of treatment and after the switch from daily GH., Design: A randomized, multinational, open-labelled, controlled parallel group phase 3 trial, comprising a 52-week main phase and 3-year safety extension (NCT03811535)., Setting: Eighty-five sites across 20 countries., Patients: A total of 200 treatment-naïve prepubertal patients were randomized and exposed; 194 completed the 2-year period., Interventions: Patients were randomized 2:1 to somapacitan (0.16 mg/kg/wk) or daily GH (0.034 mg/kg/d) during the first year, after which all patients received somapacitan 0.16 mg/kg/wk., Main Outcome Measures: Height velocity (HV; cm/year) at week 104. Additional assessments included HV SD score (SDS), height SDS, IGF-I SDS, and observer-reported outcomes., Results: HV was sustained in both groups between 52 and 104 weeks. At week 104, mean (SD) for HV between weeks 52 and 104 was 8.4 (1.5) cm/year after continuous somapacitan treatment and 8.7 (1.8) cm/year after 1 year of somapacitan treatment following switch from daily GH. Secondary height-related endpoints also supported sustained growth. Mean IGF-I SDS during year 2 was similar between groups and within normal range (-2 to +2). Somapacitan was well tolerated, with no safety or tolerability issues identified. GH patient preference questionnaire results show that most patients and their caregivers (90%) who switched treatment at year 2 preferred once-weekly somapacitan over daily GH treatment., Conclusions: Somapacitan in children with GHD showed sustained efficacy and tolerability for 2 years, and after switching from daily GH. Patients/caregivers switching from daily GH expressed a preference for somapacitan., Clinical Trial Registration: NCT03811535., (© The Author(s) 2023. Published by Oxford University Press on behalf of the Endocrine Society.)