Glycemic and Psychosocial Outcomes of Advanced Hybrid Closed-Loop Therapy in Youth With High HbA1c: A Randomized Clinical Trial.

Objective: To determine the efficacy of advanced hybrid closed-loop (AHCL) therapy in a high-risk cohort of youth on continuous subcutaneous insulin infusion (CSII) with or without continuous glucose monitoring (CGM) with suboptimal glycemia.

Research Design And Methods: In a 6-month multicenter clinical trial, youth with type 1 diabetes with mean and most recent HbA1c >8.5% (65 mmol/mol) were randomly assigned 1:1 to AHCL or treatment as usual (CSII ± CGM).

Outcomes of growth hormone treatment in children with Prader-Willi syndrome over a 30-year period: a single tertiary center experience.

Objectives: Clinical benefits of growth hormone (GH) in Prader-Willi syndrome (PWS) are proven and scoliosis is a known association of both PWS and GH therapy. The aims of this study were to assess GH prescribing practices and growth outcomes over time, the prevalence and predictors of scoliosis in GH-treated PWS children, and the near-final height of GH-treated PWS patients.

Design And Methods: This is a retrospective, descriptive study evaluating data from all clinic visits of patients aged 0-18 years with PWS, seen through the Children's Hospital at Westmead between March 1992 and May 2022 (n=75).

Developing a process to measure actual harm from medication errors in paediatric inpatients: From design to implementation.

Aims: The potential harm associated with medication errors is widely reported, but data on actual harm are limited. When actual harm has been measured, assessment processes are often poorly described, limiting their ability to be reproduced by other studies. Our aim was to design and implement a new process to assess actual harm resulting from medication errors in paediatric inpatient care.

Does insulin pump therapy offer benefits for behaviour, mood, cognition and HbA1c in children and adolescents with type 1 diabetes? A randomised controlled trial with observational follow-up.

Aims: Improved behaviour, mood, cognition and HbA1c have been reported with short-term use of continuous subcutaneous insulin infusion (CSII) in youth with type 1 diabetes (T1D). We sought to re-examine these findings in a randomised controlled trial (RCT), with longitudinal follow-up.

Methods: RCT of youth aged 7-15 years with T1D, at two tertiary paediatric centres.

Developmental Pathway Choices of Young People Presenting to a Gender Service with Gender Distress: A Prospective Follow-Up Study.

This prospective case-cohort study examines the developmental pathway choices of 79 young people (13.25-23.75 years old; 33 biological males and 46 biological females) referred to a tertiary care hospital's Department of Psychological Medicine (December 2013-November 2018, at ages 8.

Impact of Missing Data on the Accuracy of Glucose Metrics from Continuous Glucose Monitoring Assessed Over a 2-Week Period.

To explore the impact of missing data on the accuracy of continuous glucose monitoring (CGM) metrics collected for a 2-week period in a clinical trial. Simulations were conducted to examine the effect of various patterns of missingness on the accuracy of CGM metrics as compared with a "complete" data set. The proportion of missing data, the "block size" in which the data were missing, and the missing mechanism were modified for each "scenario.

Short- and long-term effects of an electronic medication management system on paediatric prescribing errors.

Electronic medication management (eMM) systems are designed to improve safety, but there is little evidence of their effectiveness in paediatrics. This study assesses the short-term (first 70 days of eMM use) and long-term (one-year) effectiveness of an eMM system to reduce prescribing errors, and their potential and actual harm. We use a stepped-wedge cluster randomised controlled trial (SWCRCT) at a paediatric referral hospital, with eight clusters randomised for eMM implementation.

Hybrid closed-loop therapy with a first-generation system increases confidence and independence in diabetes management in youth with type 1 diabetes.

Aim: Hybrid closed-loop (HCL) therapy improves glycaemic control in adolescents with type 1 diabetes; however, little is known about their lived experience using these systems. The aim of this study was to explore the lived experiences of youth with type 1 diabetes using HCL therapy, and their parents, to provide insight into their lived experiences.

Methods: Adolescents and young adults aged 12-25 years, who used Medtronic MiniMed™ 670G HCL system during a 6-month randomised clinical trial, and their parents, were invited to participate in a semi-structured interview at the end of the study.

Effect of a Hybrid Closed-Loop System on Glycemic and Psychosocial Outcomes in Children and Adolescents With Type 1 Diabetes: A Randomized Clinical Trial.

Importance: Hybrid closed-loop (HCL) therapy has improved glycemic control in children and adolescents with type 1 diabetes; however, the efficacy of HCL on glycemic and psychosocial outcomes has not yet been established in a long-term randomized clinical trial.

Objective: To determine the percentage of time spent in the target glucose range using HCL vs current conventional therapies of continuous subcutaneous insulin infusion or multiple daily insulin injections with or without continuous glucose monitoring (CGM).

Design, Setting, And Participants: This 6-month, multicenter, randomized clinical trial included 172 children and adolescents with type 1 diabetes; patients were recruited between April 18, 2017, and October 4, 2019, in Australia.

Gene expression signatures predict response to therapy with growth hormone.

Recombinant human growth hormone (r-hGH) is used as a therapeutic agent for disorders of growth including growth hormone deficiency (GHD) and Turner syndrome (TS). Treatment is costly and current methods to model response are inexact. GHD (n = 71) and TS patients (n = 43) were recruited to study response to r-hGH over 5 years.

Attachment Patterns in Children and Adolescents With Gender Dysphoria.

The current study examines patterns of attachment/self-protective strategies and rates of unresolved loss/trauma in children and adolescents presenting to a multidisciplinary gender service. Fifty-seven children and adolescents (8.42-15.

Investigating paediatric hypoglycaemia: Dynamic studies at a tertiary paediatric hospital.

Aim: Paediatric hypoglycaemia often requires specific investigations to determine aetiology. Samples from the time of hypoglycaemia may not be available and a diagnostic fasting test may be required. Additionally, fasting studies can determine safe fasting intervals and prolonged oral glucose challenges can assess hypoglycaemia due to abnormal post-prandial glucose handling.

Effect of frequency of sensor use on glycaemic control in individuals on sensor-augmented pump therapy with and without Predictive Low Glucose Management System.

Improved frequency of sensor use improves glycaemic control. Furthermore, there is no deterioration of glycaemic control with increased sensor use in individuals on Predictive Low Glucose Management (PLGM) system. Younger children are more likely to have better sensor uptake than older children.

Positive impacts of changes to a tertiary hospital after-hours endocrine and diabetes on-call service.

Aim: To examine the impact of changes to the endocrine/diabetes after-hours service model of care at a major tertiary children's hospital in Australia. The model aimed to enhance the independence of families and reduce dependency on after-hours calls to health professionals.

Methods: The after-hours activity was captured prospectively using an iPad with a customised FileMaker database.

Diagnosis, Genetics, and Therapy of Short Stature in Children: A Growth Hormone Research Society International Perspective.

The Growth Hormone Research Society (GRS) convened a Workshop in March 2019 to evaluate the diagnosis and therapy of short stature in children. Forty-six international experts participated at the invitation of GRS including clinicians, basic scientists, and representatives from regulatory agencies and the pharmaceutical industry. Following plenary presentations addressing the current diagnosis and therapy of short stature in children, breakout groups discussed questions produced in advance by the planning committee and reconvened to share the group reports.

Requirements for improving health and well-being of children with Prader-Willi syndrome and their families.

Prader-Willi syndrome (PWS) is a rare genetic condition with multi-system involvement. The literature was reviewed to describe neurodevelopment and the behavioural phenotype, endocrine and metabolic disorders and respiratory and sleep functioning. Implications for child and family quality of life were explored.

Abnormal Cortical and Trabecular Bone in Youth With Type 1 Diabetes and Celiac Disease.

Objective: This study compared bone health in youth with type 1 diabetes and celiac disease (CD) versus type 1 diabetes alone.

Research Design And Methods: This was a case-control study of 42 youth with coexisting type 1 diabetes and CD and 40 with type 1 diabetes matched for age, sex, diabetes duration, and HbA. Bone mineral density (BMD), bone mineral content (BMC), and BMC-to-lean tissue mass (LTM) ratio were measured using DXA and reported as -scores for height.

Characteristics of Automated Insulin Suspension and Glucose Responses with the Predictive Low-Glucose Management System.

Background: The Predictive Low-Glucose Management (PLGM) system suspends basal insulin when hypoglycemia is predicted and reduces hypoglycemia. The aim of this analysis was to explore the characteristics of automated insulin suspension and sensor glucose (SG) responses following PLGM-initiated pump suspension.

Research Design And Methods: Children and adolescents with type 1 diabetes used the Medtronic MiniMed™ 640G pump as part of a randomized controlled trial.

A will and a way to fund medicines for rare diseases: the story of human growth hormone replacement for adults with growth hormone deficiency.

Growth hormone (GH) replacement therapy was recently recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme for adults with severe GH deficiency and impaired quality of life. This approval was significant for two reasons. First, the application was initiated and coordinated by a health professional working group, who prepared a 'public interest' submission to PBAC.

Unexpected Management Behaviors in Adolescents With Type 1 Diabetes Using Sensor-Augmented Pump Therapy.

Background: Continuous glucose monitoring can improve glycemic outcomes in individuals with type 1 diabetes. However, the constant exposure to real-time glucose levels can sometimes lead the individual to make some risky choices to address the glycemic excursions. Hence, the purpose of this study was to explore the aberrant management behaviors of youth with type 1 diabetes on sensor-augmented pump therapy (SAPT).

Reduction in Hypoglycemia With the Predictive Low-Glucose Management System: A Long-term Randomized Controlled Trial in Adolescents With Type 1 Diabetes.

Objective: Short-term studies with automated systems that suspend basal insulin when hypoglycemia is predicted have shown a reduction in hypoglycemia; however, efficacy and safety have not been established in long-term trials.

Research Design And Methods: We conducted a 6-month, multicenter, randomized controlled trial in children and adolescents with type 1 diabetes using the Medtronic MiniMed 640G pump with Suspend before low (predictive low-glucose management [PLGM]) compared with sensor-augmented pump therapy (SAPT) alone. The primary outcome was percentage time in hypoglycemia with sensor glucose (SG) <3.

Insulin regimens for newly diagnosed children with type 1 diabetes mellitus in Australia and New Zealand: A survey of current practice.

Aim: There is no consensus on the optimal insulin treatment for children newly diagnosed with type 1 diabetes mellitus (T1DM). The aims of this study were (i) to describe the insulin regimens used at diagnosis by patient age and geographical region and (ii) to explore differences between and within Australia (AU) and New Zealand (NZ) with regards to other aspects of patient management and education.

Methods: An online survey of medical professionals caring for children with T1DM in AU and NZ was undertaken.

Low dose growth hormone treatment in infants and toddlers with Prader-Willi syndrome is comparable to higher dosage regimens.

Objective: Evaluate benefit and risk of low dose growth hormone treatment (GHT, 4.5mg/m/week) in very young children with Prader-Willi Syndrome (PWS).

Design: Prospective longitudinal clinical intervention.

Greater postprandial glucose excursions and inadequate nutrient intake in youth with type 1 diabetes and celiac disease.

The gluten free diet (GFD) has a high glycemic index and low-fiber content, which potentially influences glycemic excursions in type 1 diabetes (T1D) and celiac disease (CD). Participants in this case-control study of youth with T1D+CD (n = 10) and T1D only (n = 7) wore blinded continuous glucose monitoring systems for six days. Blood glucose levels (BGLs) were compared between groups for each meal, including pre-meal, peak, 2-hour postprandial and time-to-peak.