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Hematopoietic cell transplantation (HCT) is a potentially curative treatment for children and adults with malignant and non-malignant diseases. Despite increasing survival rates, long-term morbidity following HCT is substantial. Neurocognitive dysfunction is a serious cause of morbidity, yet little is known about neurocognitive dysfunction following HCT. To address this gap, collaborative efforts of the Center for International Blood and Marrow Transplant Research and the European Society for Blood and Marrow Transplantation undertook an expert review of neurocognitive dysfunction following HCT. In this review, we define what constitutes neurocognitive dysfunction, characterize its risk factors and sequelae, describe tools and methods to assess neurocognitive function in HCT recipients, and discuss possible interventions for HCT patients with this condition. This review aims to help clinicians understand the scope of this health-related problem, highlight its impact on well-being of survivors, and to help determine factors that may improve identification of patients at risk for declines in cognitive functioning after HCT. In particular, we review strategies for preventing and treating neurocognitive dysfunction in HCT patients. Lastly, we highlight the need for well-designed studies to develop and test interventions aimed at preventing and improving neurocognitive dysfunction and its sequelae following HCT.

Background Survival of neuroblastoma patients has improved over recent decades, but chronic health issues and treatment related late effects cause significant morbidity in survivors. Aims We aimed to describe late effects and long‐term toxicity in neuroblastoma patients treated at a tertiary, paediatric institution in Australia. Methods & Results Patients with neuroblastoma treated primarily at The Children's hospital at Westmead were eligible for inclusion. Retrospective analysis of 65 (45 with high‐risk and 20 with non‐high‐risk disease) neuroblastoma patients were performed via medical record review. Approximately 60% of patients were >5 years from diagnosis and termed the “full effects cohort” who had a range of medical and psychosocial late effects analysed through descriptive means. The remaining 26 patients who had not yet reached 5 years post treatment had audiometry analysis only. Of the 65 patients, 72% were alive at last follow‐up. The median length of follow‐up was 7 years from diagnosis amongst survivors. Therapy was according to contemporary protocols for neuroblastoma and ranged from standard cytotoxic therapies to intensive multimodal regimens and/or experimental therapy depending on risk group/relapse status. Of the 39 full effects cohort, 85% suffered from at least one late effect. Late effects were common in the endocrine, dental and audiometry domains with 38%, 49% and 72% of patients affected in these areas, respectively. Neuro‐cognitive domains were also notably affected with 46% of patients suffering a deficit. Two thirds of survivors were disease free at last follow‐up. Conclusion Survivors of high‐risk neuroblastoma suffer from a range of chronic illnesses, which lead to morbidity and affect quality of life of survivors.

IntroductionVaping among children and young people (CYP) has increased globally over the past decade, with rates stabilising in the UK in recent years. Factors such as curiosity, social influence, stress management and attractive flavours contribute to its popularity. Although the long-term health impacts are uncertain, vaping poses risks including nicotine dependence, cardiovascular and respiratory issues, and cognitive impairment, though evidence on long-term effects is still emerging. Despite established smoking cessation programmes for adults, tailored resources for vaping cessation among CYP remain scarce, particularly within healthcare settings, which offer unique opportunities for professional intervention and ongoing support. The objective of this review is to assess the extent and nature of available literature on interventions delivered in healthcare settings to support vaping cessation among CYP under the age of 18 years.Methods and analysisThis scoping review will include studies targeting CYP under the age of 18 years, specifically focusing on interventions delivered within healthcare settings. Studies outside healthcare contexts or those without healthcare provider involvement will be excluded. Additionally, interventions delivered solely to parents or carers will not be considered.A comprehensive search will be conducted in MEDLINE, Embase, Web of Science, Cochrane Library and CINAHL from January 2004 to present, with additional grey literature from sources including grey literature repositories and Google Scholar. Results will be imported into Rayyan for screening, with two independent reviewers assessing studies for inclusion. Data extraction will include study design, population characteristics (including explicit age ranges, specifically CYP under 18 years), intervention details and outcomes. A descriptive synthesis will map study characteristics, while thematic analysis will identify intervention themes and healthcare contexts.Ethics and disseminationEthics approval is not required for this secondary analysis. Findings will be disseminated through publication, conference presentations and shared with public health stakeholders.

Purpose Four patients with Saul–Wilson syndrome were reported between 1982 and 1994, but no additional individuals were described until 2018, when the molecular etiology of the disease was elucidated. Hence, the clinical phenotype of the disease remains poorly defined. We address this shortcoming by providing a detailed characterization of its phenotype. Methods Retrospective chart reviews were performed and primary radiographs assessed for all 14 individuals. Four individuals underwent detailed ophthalmologic examination by the same physician. Two individuals underwent gynecologic evaluation. Z -scores for height, weight, head circumference and body mass index were calculated at different ages. Results All patients exhibited short stature, with sharp decline from the mean within the first months of life, and a final height Z -score between −4 and −8.5 standard deviations. The facial and radiographic features evolved over time. Intermittent neutropenia was frequently observed. Novel findings included elevation of liver transaminases, skeletal fragility, rod–cone dystrophy, and cystic macular changes. Conclusions Saul–Wilson syndrome presents a remarkably uniform phenotype, and the comprehensive description of our cohort allows for improved understanding of the long-term morbidity of the condition, establishment of follow-up recommendations for affected individuals, and documentation of the natural history into adulthood for comparison with treated patients, when therapeutics become available.

Background: The lived experience of children and adolescents diagnosed with cancer differs greatly from that of the adult cancer patient. A diagnosis of cancer disrupts almost every developmental life stage and continues to affect the child, and potentially their whole family, throughout adulthood. Objective: While it is important to recognise the potential for posttraumatic growth, a considerable proportion of children and adolescents will experience poorer psychological, social, educational and quality-of-life outcomes. Parents, particularly mothers, have been shown to experience levels of post-traumatic distress even greater than that of survivors. As such, there exists a critical need to provide family-centred support from diagnosis through to long-term survivorship or bereavement. Discussion: Ongoing surveillance, proactive management of chronic health conditions, and health behaviour education are critical to survivors' lifelong wellbeing and can be facilitated locally by general practitioners with support from tertiary healthcare teams in a shared-care arrangement.

Paediatric chronic pancreatitis (CP) is a relatively rare entity, but it can be accompanied by debilitating complications such as pseudocysts, chronic pain and pancreatic duct obstruction. Surgical drainage procedures, such as pancreaticojejunostomy or cystogastrostomy/jejunostomy to address these complications may be required; however, there is a paucity of evidence as to the efficacy and long-term outcomes of these operations in the paediatric population. A scoping review of contemporary (post-2000) studies detailing surgical pancreatic drainage procedures performed in children (< 18 years) was undertaken. After screening, 24 case series detailing a total of 248 patients met the inclusion criteria. Longitudinal pancreaticojejunostomy and cystogastrostomy were the most common surgical procedures performed in children with CP and pseudocysts, respectively. Overall generally favourable outcomes were reported, but all studies were considered to have a high risk of bias. Operative management for paediatric CP is infrequently required; therefore, large prospective studies or trials focusing on this population are infeasible, limiting the best available evidence on the topic to case series, level IV. Recommendations to improve the quality of surgical care in the paediatric CP population could include centralisation and the formation of registries to allow accurate long-term follow-up.

Aim To evaluate the population pharmacokinetics of unbound F-Ara-A (the circulating metabolite of fludarabine) in 211 patients (age range, 0.1–63.4 years) undergoing allogeneic haematopoietic stem cell transplantation conditioning. Methods Total ( n  = 2480) and unbound ( n  = 1403) F-Ara-A concentrations were measured in blood samples collected at timed intervals after fludarabine doses ranging from 10 to 50 mg/m 2 and infused over 0.42–1.5 h. A three-compartment population pharmacokinetic model was developed based on unbound plasma concentrations and used to estimate F-Ara-A unbound pharmacokinetic parameters and fraction unbound ( fu ). A number of covariates, including glomerular filtration rate (GFR) and post-menstrual age (PMA), were evaluated for inclusion in the model. Results The base population mean estimates ± relative standard error (%RSE) for unbound clearance from the central compartment (CLu) and inter-compartmental clearances (Q2u, Q3u) were 3.42 ± 3%, 6.54 ± 24% and 1.47 ± 16% L/h/70 kg, respectively. The population mean estimates (%RSE) for the unbound volume of distribution into the central (V1u) and peripheral compartments (V2u, V3u) were 9.65 ± 8%, 8.17 ± 9% and 16.4 ± 10% L/70 kg, respectively, and that for fu was 0.877 ± 1%. Covariate model development involved differentiating F-Ara-A CLu into non-renal (1.81 ± 9% L/h/70 kg) and renal components (1.02 ± 9%*GFR L/h/70 kg). A sigmoidal maturation factor was applied to renal CLu, with population mean estimates for the Hill exponent and PMA at 50% mature of 2.97 ± 4% and 69.1 ± 8% weeks, respectively. Conclusion Patient age and GFR are predictors of unbound F-Ara-A CLu. This has the potential to impact dose requirements. Dose individualisation by target concentration intervention will be facilitated by this model once it is externally validated.

Background Childhood cancer survivors are vulnerable to long-term treatment-related health conditions, which can lead to poor quality of life. Little data exist on the overall health of long-term Australian and New Zealand childhood cancer survivors or on survivors’ motivations for attending survivorship clinics. Methods This study administers a cross-sectional questionnaire to long-term survivors ≥5 years from their primary diagnosis. We compared participant-reported number of late effects by a cancer diagnosis, and identified clinical (eg, treatment) and demographic (eg, age) factors that were associated with late effects burden and engagement in cancer survivorship care. Results A total of 634 participants completed questionnaires (48% male, mean age = 21.7 years). Most participants (79%) reported at least one cancer-related late effect, most commonly fatigue (40%) and memory/learning difficulties (34%). Brain tumor survivors reported a higher total number of late effects than survivors with other diagnoses (mean = 5.7 vs. 3.2, P < .001). Participants’ most commonly reported motivators for engaging in care were to understand problems that may occur later in life because of their cancer and/or treatment (98.5%) and to get reassurance about one’s health (97.4%). The proportion of survivors endorsing each motivating factor was similar across cancer diagnoses, with the exception of learning more about insurance and pensions (highest in brain tumor survivors = 80%). In multivariable analyses, survivors were more likely to report being engaged in survivorship care if they were younger (P < .001), less time had elapsed since their diagnosis (P < .001), or they reported a higher number of motivating factors (P = .016). Conclusion Survivors report a range of health problems decades after treatment completion. Understanding the burden of late effects, and motivators for seeking survivorship care to manage these health problems, is important for ensuring that tailored interventions or services are available to meet the needs of this growing population and to design effective models of survivorship care. Survivors of childhood cancer face unique and complex health problems in adulthood, known as late effects. Understanding survivors’ late effects and factors associated with engaging in survivorship care is critical to ensuring appropriate services are available to meet the needs of the growing survivor population. This study addressed this need.

The objective of the study was the analysis of clinical types, outcomes, and risk factors associated with the outcome of adenovirus (ADV) infection, in children and adults after allo-HCT. A total number of 2529 patients (43.9% children; 56.1% adults) transplanted between 2000 and 2022 reported to the EBMT database with diagnosis of ADV infection were analyzed. ADV infection manifested mainly as viremia (62.6%) or gastrointestinal infection (17.9%). The risk of 1-year mortality was higher in adults ( p  = 0.0001), and in patients with ADV infection developing before day +100 ( p  < 0.0001). The 100-day overall survival after diagnosis of ADV infections was 79.2% in children and 71.9% in adults ( p  < 0.0001). Factors contributing to increased risk of death by day +100 in multivariate analysis, in children: CMV seropositivity of donor and/or recipient ( p  = 0.02), and Lansky/Karnofsky score <90 ( p  < 0.0001), while in adults: type of ADV infection (viremia or pneumonia vs gastrointestinal infection) ( p  = 0.0004), second or higher HCT ( p  = 0.0003), and shorter time from allo-HCT to ADV infection ( p  = 0.003). In conclusion, we have shown that in patients infected with ADV, short-term survival is better in children than adults. Factors directly related to ADV infection (time, clinical type) contribute to mortality in adults, while pre-transplant factors (CMV serostatus, Lansky/Karnofsky score) contribute to mortality in children.