Changes in weight status of caregivers of children and adolescents enrolled in a community-based healthy lifestyle programme: Five-year follow-up.

Whanau Pakari is a family-centred healthy lifestyle programme for children/adolescents with overweight/obesity in New Zealand. This secondary analysis from our randomised trial within the clinical service assessed 5-year BMI changes in accompanying caregivers (n = 23), mostly mothers. Overall, baseline and 5-year caregivers' BMI were similar (32.50 vs 31.42 kg/m2, respectively; p = 0.31) but two-thirds (65%) experienced BMI reductions. Five-year BMI change was similar in High-intensity and Low-intensity randomisation groups [-1.37 kg/m2 (-4.95, 2.21); p = 0.44]. Caregiver's BMI change was not associated with child's BMI change. Despite no overall BMI reduction, our findings contrast with upward BMI trajectories predicted for NZ adults with overweight/obesity.

A Multisource Process Evaluation of a Community-Based Healthy Lifestyle Programme for Child and Adolescent Obesity.

Whanau Pakari is a healthy lifestyle assessment and intervention programme for children and adolescents with obesity in Taranaki (Aotearoa/New Zealand), which, in this region, replaced the nationally funded Green Prescription Active Families (GRxAF) programme. We compared national referral rates from the GRxAF programme (age 5-15 years) and the B4 School Check (B4SC, a national preschool health and development assessment) with referral rates in Taranaki from Whanau Pakari. We retrospectively analysed 5 years of clinical data (2010-2015), comparing referral rates before, during, and after the Whanau Pakari clinical trial, which was embedded within the programme. We also surveyed programme referrers and stakeholders about their experiences of Whanau Pakari, analysing their responses using a multiple-methods framework. After the Whanau Pakari trial commenced, Taranaki GRxAF referral rates increased markedly (2.3 pretrial to 7.2 per 1000 person-years), while NZ rates were largely unchanged (1.8-1.9 per 1000 person-years) (p < 0.0001 for differences during the trial). Post-trial, Taranaki GRxAF referral rates remained higher irrespective of ethnicity, being 1.8 to 3.2 times the national rates (p < 0.001). Taranaki B4SC referrals for obesity were nearly complete at 99% in the last trial year and 100% post-trial, compared with national rates threefold lower (31% and 32%, respectively; p < 0.0001), with Taranaki referral rates for extreme obesity sustained at 80% and exceeding national rates for both periods (58% and 62%, respectively; p < 0.01). Notably, a referral was 50% more likely for referrers who attended a Whanau Pakari training half-day (RR = 1.51; p = 0.009). Stakeholders credited the success of Whanau Pakari to its multidisciplinary team, family-centred approach, and home-based assessments. However, they highlighted challenges such as navigating multidisciplinary collaboration, engaging with families with complex needs, and shifting conventional healthcare practices. Given its favourable referral trends and stakeholder endorsement, Whanau Pakari appears to be a viable contemporary model for an accessible and culturally appropriate intervention on a national and potentially international scale.

Ultrasound-guided Lipiodol® hysterosalpingography: A prospective study on pregnancy and complication rates.

BACKGROUND: Fluoroscopic hysterosalpingography (HSG) with Lipiodol® is safe and has a therapeutic effect on fertility: transient in endometriosis-related infertility and sustained in unexplained infertility. Ultrasound is replacing fluoroscopy as the preferred imaging modality for HSG due to comfort and radiation safety (no ionising radiation). The safety of ultrasound-guided Lipiodol® HSG is uncertain. AIMS: Prospectively observe pregnancy and complication rates after ultrasound-guided Lipiodol® HSG. MATERIALS AND METHODS: A single-centre prospective study of women with unexplained infertility undergoing ultrasound-guided Lipiodol® uterine bathing and tubal flushing after tubal patency confirmed with ExEm® Foam HyFoSy (hysterosalpingo-foam-sonography). Pregnancy outcomes at six months and serum and urinary thyroid function at one, three and eight weeks were recorded. Pain scores were recorded during and immediately after HSG. Descriptive statistics are reported. RESULTS: Fifty-two participants were enrolled between July 2019 and April 2021, median age 33 years (range 21-45). Only 45 (87%, 45/52) completed the Lipiodol® HSG; 5/7 experienced intravasation during initial HyFoSy. Of 30 women at follow-up, 57% had biochemical (17/30, 95% CI 37%-75%), 53% clinical (16/30 95% CI 34%-72%) and 35% ongoing pregnancies (11/30, 95% CI 20%-56%). The rate of subclinical hypothyroidism (SCH) at two months was 41% (7/17). One intravasation event occurred during Lipiodol® HSG (2%, 1/45). Median pain score was 5/10 (range 0-9, interquartile range 2.5-7). No anaphylaxis, infection or oil embolism was observed. CONCLUSION: Outpatient ultrasound-guided Lipiodol® HSG was safe, with pregnancy rates comparable to previous studies of fluoroscopic guidance. Rates of intravasation and SCH were also similar, confirming the need to monitor thyroid function.

Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial.

Background: TransCon CNP (navepegritide) is an investigational prodrug of C-type natriuretic peptide (CNP) designed to allow for continuous CNP exposure with once-weekly dosing. This 52-week phase 2 (ACcomplisH) trial assessed the safety and efficacy of TransCon CNP in children with achondroplasia. Methods: ACcomplisH is a global, randomised, double-blind, placebo-controlled, dose-escalation trial. Study participants were recruited between June 10, 2020, and September 24, 2021. Eligible participants were prepubertal, aged 2-10 years, with genetically confirmed achondroplasia, and randomised 3:1 to once-weekly subcutaneous injections of TransCon CNP (6, 20, 50, or 100 µg CNP/kg/week) or placebo for 52 weeks. Primary objectives were safety and annualised growth velocity (AGV). ACcomplisH is registered with ClinicalTrials.gov (NCT04085523) and Eudra (CT 2019-002754-22). Findings: Forty-two participants received TransCon CNP at doses of 6 µg (n = 10; 7 female), 20 µg (n = 11; 3 female), 50 µg (n = 10; 3 female), or 100 µg (n = 11; 6 female) CNP/kg/week, with 15 receiving placebo (5 female). Treatment-emergent adverse events (TEAEs) were mild or moderate with no grade 3/4 events reported. There were 2 serious TEAEs that were assessed as not related to TransCon CNP. Eleven injection site reactions occurred in 8 participants receiving TransCon CNP and no symptomatic hypotension occurred. TransCon CNP demonstrated a dose-dependent improvement in AGV. At 52 weeks, TransCon CNP 100 µg CNP/kg/week significantly improved AGV vs placebo (least squares mean [95% CI] 5.42 [4.74-6.11] vs 4.35 [3.75-4.94] cm/year; p = 0.0218), and improved achondroplasia-specific height SDS from baseline (least squares mean [95% CI] 0.22 [0.02-0·41] vs -0·08 [-0.25 to 0.10]; p = 0.0283). All participants completed the randomised period and continued in the ongoing open-label extension period receiving TransCon CNP 100 µg CNP/kg/week. Interpretation: This phase 2 trial suggests that TransCon CNP is effective, safe, with low injection site reaction frequency, and may provide a novel, once-weekly treatment option for children with achondroplasia. These results support TransCon CNP at 100 µg CNP/kg/week in the ongoing pivotal trial. Funding: Ascendis Pharma, A/S.

Feasibility study on a longer side-alternating vibration therapy protocol (15†min per session) in children and adolescents with mild cerebral palsy.

Objective: Previous studies on side-alternating vibration therapy (sVT) have usually used a 9 min intervention protocol. We performed a feasibility study aimed at assessing the safety, acceptability, and potential effectiveness of a longer sVT protocol (15 min per session) in children and adolescents with cerebral palsy (CP). Methods: Fifteen participants aged 5.2-17.4 years (median = 12.4 years) with CP GMFCS level II underwent 20 weeks of sVT consisting of 15 min sessions 4 days/week. Participants were assessed at baseline and after the intervention period, including mobility (six-minute walk-test; 6MWT), body composition (whole-body dual-energy x-ray absorptiometry scans), and muscle function (force plate). Results: Adherence level to the 15 min VT protocol was 83% on average. There were no adverse events reported. After 20 weeks, there was some evidence for an increase in the walking distance covered in 6MWT (+43 m; p = 0.0018) and spine bone mineral density (+0.032 g/cm2; p = 0.012) compared to baseline. Conclusions: The 15 min sVT protocol is feasible and well tolerated. The results also suggest potential benefits of this protocol to mobility and bone health. Randomized controlled trials are needed to reliably ascertain the potential effectiveness of a longer sVT protocol on physical function and body composition in young people with CP.

Vibration Therapy as an Early Intervention for Children Aged 2-4 Years with Cerebral Palsy: A Feasibility Study.

Aims: To evaluate the feasibility and acceptability of vibration therapy (VT) in preschool children with cerebral palsy (CP) and obtain preliminary data on its potential effectiveness.Methods: Nine children aged 2.5-4.8 years (4 boys) with CP GMFCS levels I-III participated in a single-group feasibility study, undergoing a 12-week control period without intervention, followed by 12 weeks of home-based VT (four times/week, 9 min/day, frequency 20 Hz). We assessed adherence to VT protocol, adverse events, and family acceptability of VT. Clinical assessments included motor function (GMFM-66), body composition (DXA), mobility (10-meter walk/run test), and health-related quality of life (PedsQL).Results: VT was well tolerated and acceptable to families, with high adherence levels reported (mean = 93%). There were no observed between-period differences (ΔControl vs ΔVT) except for an improvement in the PedsQL "Movement & Balance" dimension with VT (p = 0.044). Nonetheless, changes after the VT but not the Control period were suggestive of potential treatment benefits for mobility, gross motor function, and body composition (lean mass and legs bone mineral density).Conclusion: Home-based VT is feasible and acceptable for preschool children with CP. Our preliminary data suggest potential health benefits from VT for these children, supporting larger randomized trials to assess its effectiveness properly. Clinical trial registration number: Australian New Zealand Clinical Trials Registry (ACTRN12618002027291).

Current Pubertal Development in Chinese Children and the Impact of Overnutrition, Lifestyle, and Perinatal Factors.

CONTEXT: Age of pubertal onset has been decreasing in many countries but there have been no data on pubertal development in Chinese children over the last decade. OBJECTIVE: The primary objective of the study was to evaluate the current status of sexual maturation in Chinese children and adolescents. Secondary objectives were to examine socioeconomic, lifestyle, and auxological associations with pubertal onset. METHODS: In this national, cross-sectional, community-based health survey, a multistage, stratified cluster random sampling method was used to select a nationally representative sample, consisting of 231 575 children and adolescents (123 232 boys and 108 343 girls) between 2017 and 2019. Growth parameters and pubertal staging were assessed by physical examination. RESULTS: Compared to 10 years previously, the median age of Tanner 2 breast development and menarche were similar at 9.65 years and 12.39 years respectively. However, male puberty occurred earlier with a median age of testicular volume ≥4 mL of 10.65 years. Pubertal onset did occur earlier at the extremes, with 3.3% of the girls with breast development at 6.5-6.99 years old, increasing to 5.8% by 7.5-7.99 years old. Early pubertal onset was also noted in boys, with a testicular volume ≥ 4 mL noted in 1.5% at 7.5-7.99 years, increasing to 3.5% at 8.5-8.99 years old. Obesity and overweight increased risk of developing earlier puberty relative to normal weight in both boys and girls. CONCLUSION: Over the past decade, pubertal development is occurring earlier in Chinese children. While the cause is multifactorial, overweight and obesity are associated with earlier puberty onset. The currently used normative pubertal data of precocious puberty may not be applicable to diagnose precocious puberty.

The impact of prolonged, maternal iodine exposure in early gestation on neonatal thyroid function.

Context: Hysterosalpingography (HSG) using oil-soluble contrast medium (OSCM) improves pregnancy rates but results in severe and persistent iodine excess, potentially impacting the fetus and neonate. Objective: To determine the incidence of thyroid dysfunction in newborns conceived within six months of OSCM HSG. Design: Offspring study of a prospective cohort of women who underwent OSCM HSG. Setting: Auckland region, New Zealand (2020-2022). Participants: Offspring from the SELFI (Safety and Efficacy of Lipiodol in Fertility Investigations) study cohort (n=57). Measurements: All newborns had a dried blood spot card for TSH measurement 48 hours after birth as part of New Zealand's Newborn Metabolic Screening Programme. Forty-one neonates also had a heel prick serum sample at one week to measure thyroid-stimulating hormone (TSH), free thyroxine (FT4), and free triiodothyronine (FT3). Maternal urine iodine concentration (UIC) and TSH in the six months after OSCM HSG were retrieved from the SELFI study for analyses. Primary outcome: Incidence of hypothyroidism in the neonatal period. Results: There was no evidence of primary hypothyroidism on newborn screening (TSH 2-10 mIU/L). All neonates tested at one week had normal serum TSH, FT4, and FT3 levels. However, increasing maternal peak UIC levels during pregnancy were associated with lower TSH levels (p= 0.006), although also associated with lower FT4 levels (p=0.032). Conclusions: While pre-conceptional OSCM HSG in women did not result in neonatal hypothyroidism, gestational iodine excess was associated with a paradoxical lowering of neonatal TSH levels despite lower FT4 levels. These changes likely reflect alterations in deiodinase activity in the fetal hypothalamic-pituitary axis from iodine excess. Trial registration: https://anzctr.org.au/Trial/Registration/TrialReview.aspx?ACTRN=12620000738921, identifier 12620000738921.

International Consensus Guideline on Small for Gestational Age: Etiology and Management From Infancy to Early Adulthood.

This International Consensus Guideline was developed by experts in the field of small for gestational age (SGA) of 10 pediatric endocrine societies worldwide. A consensus meeting was held and 1300 articles formed the basis for discussions. All experts voted about the strengths of the recommendations. The guideline gives new and clinically relevant insights into the etiology of short stature after SGA birth, including novel knowledge about (epi)genetic causes. Further, it presents long-term consequences of SGA birth and also reviews new treatment options, including treatment with gonadotropin-releasing hormone agonist (GnRHa) in addition to growth hormone (GH) treatment, as well as the metabolic and cardiovascular health of young adults born SGA after cessation of childhood GH treatment in comparison with appropriate control groups. To diagnose SGA, accurate anthropometry and use of national growth charts are recommended. Follow-up in early life is warranted and neurodevelopment evaluation in those at risk. Excessive postnatal weight gain should be avoided, as this is associated with an unfavorable cardiometabolic health profile in adulthood. Children born SGA with persistent short stature < -2.5 SDS at age 2 years or < -2 SDS at 3 to 4 years of age, should be referred for diagnostic workup. In case of dysmorphic features, major malformations, microcephaly, developmental delay, intellectual disability, and/or signs of skeletal dysplasia, genetic testing should be considered. Treatment with 0.033 to 0.067 mg GH/kg/day is recommended in case of persistent short stature at age of 3 to 4 years. Adding GnRHa treatment could be considered when short adult height is expected at pubertal onset. All young adults born SGA require counseling to adopt a healthy lifestyle.

Vibration therapy in young children with mild to moderate cerebral palsy: does frequency and treatment duration matter? A randomised-controlled study.

BACKGROUND: Vibration therapy (VT) has been increasingly studied in children with cerebral palsy (CP) over the last years, however, optimal therapeutic VT protocols are yet to be determined. The present study compared the effects of side-alternating VT protocols varying in frequency and treatment duration on the health of young children with mild-to-moderate CP. METHODS: Thirty-four participants aged 6.0 to 12.6 years with CP acted as their own controls and underwent two consecutive study periods: a 12-week lead-in (control) period prior to the intervention period of 20-week side-alternating VT (9 min/session, 4 days/week), with the frequency either 20 Hz or 25 Hz, determined by randomisation. Participants had 4 assessment visits: baseline, after the control period, after 12-week VT (12VT), and after further 8 weeks of VT (20VT). Assessments included 6-minute walk test (6MWT); dual-energy x-ray absorptiometry; gross motor function; muscle function testing on the Leonardo mechanography plate and by hand-held dynamometry, and a quality-of-life questionnaire (CP QOL). Analysis was carried out using linear mixed models based on repeated measures. RESULTS: Side-alternating VT was well-tolerated, with occasional mild itchiness reported. The median compliance level was 99%. VT led to improvements in 6MWT (+ 23 m; p = 0.007 after 20VT), gross motor function in standing skills (+ 0.8 points; p = 0.008 after 12VT; and + 1.3 points; p = 0.001 after 20VT) and in walking, running and jumping skills (+ 2.5 points; p < 0.0001 after 12VT; and + 3.7 points; p < 0.0001 after 20VT), spine bone mineral density z-score (+ 0.14; p = 0.015 after 20VT), velocity rise maximum of the chair rising test (+ 0.14 m/s; p = 0.021 after 20VT), force maximum of the single two-leg jump test (+ 0.30 N/kg; p = 0.0005 after 12VT; and + 0.46 N/kg; p = 0.022 after 20VT) and in the health module of CP QOL (+ 7 points; p = 0.0095 after 20VT). There were no observed differences between the two VT frequencies (i.e., 20 Hz vs 25 Hz) on study outcomes. CONCLUSIONS: The study confirms that side-alternating VT has positive effects on mobility, gross motor function, body composition, muscle function, and quality of life, independent of VT frequencies tested. Long-term, 20VT appears to be a more efficient treatment duration than a short-term, 12VT. TRIAL REGISTRATION: Australian New Zealand Clinical Trials Registry ACTRN12618002026202 ; 18/12/2018.

Transition from paediatric to adult care in young people with diabetes; A structured programme from a regional diabetes service, Auckland, New Zealand.

AIM: To assess participation with a structured transition programme for adolescents with diabetes. METHODS: Data from a regional cohort aged less than 16 years of age with type 1 (T1) and type 2 diabetes (T2D) in Auckland, New Zealand (2006-2016). Participation was defined as opting into a structured transition programme. RESULTS: Five hundrend and twelve adolescents who were to be transferred to adult care (476 type 1 (T1D) and 36 type 2 (T2D)), overall participation rate of 83%, 86% (408/476) with T1D compared to 47% (17/36) with T2D. Within the cohort of T1D, participation rates for Maori and Pacific were lower (74% and 77%, respectively) than New Zealand Europeans (88%, p = 0.020 and p = 0.039, respectively). Lower socio-economic status was associated with reduced participation (77%) compared to higher socio-economic status (90%, p = 0.002). Of the 476 T1D who participated, 408 (96%) subsequently attended at least one adult service clinic ("capture"). 42% attended an adult clinic within the planned 3 months, 87% at 6 months and retention in adult clinics over 5 years of follow-up was 78%. By contrast, the 68 young people with T1D who did not participate in the structured transition had a capture rate of 78% (p < 0.001) and retention of 63% (p = 0.036). CONCLUSIONS: In adolescents with diabetes, a formal transition from a paediatric service was associated with high rates of adult capture and subsequent retention in adult care over a 5-year follow-up period. Low socio-economic status, Maori or Pacific ethnicity and T2D were associated with reduced participation in the structured transition programme.

Comparison of percentile tables and algorithm-based calculators for classification of blood pressures in children and adolescents with obesity: A secondary analysis of a clinical trial.

AIM: Obesity as a major risk factor for childhood hypertension necessitates careful blood pressure (BP) monitoring of those affected. This study aimed to compare BP classification in a cohort of children affected by obesity using tables versus digital calculations in two sets of guidelines. METHODS: This study was a secondary analysis of data collected from a randomised clinical trial of a multidisciplinary life-style assessment and intervention program. Baseline data from 237 children with a body mass index >99th percentile or >91st percentile with weight-related comorbidities and available BP measurements were analysed. We assessed agreement between tables and algorithms in classification of elevated BP/pre-hypertension and hypertension based on the American Academy of Paediatrics (AAP) clinical practice guidelines (CPG) and the older Fourth Report using Cohen's weighted kappa. The prevalence of hypertensive diagnoses was also compared between the two guidelines. RESULTS: Agreement between BP tables and algorithmic calculation of percentiles was discordant, though improved in the AAP CPG compared to the Fourth Report (Cohen's kappa = 0.70 vs. 0.57, respectively). None (0%) were missed diagnoses, and 59 (24.9%) were false positives for the Fourth Report, and 0 (0%) were missed diagnoses, and 49 (20.9%) were false positives for the AAP CPG. Under the recent guidelines, there was an increase in prevalence of 6.0% (95% confidence interval (CI) 2.5-9.4%; P = 0.0001) for BP ≥90th percentile, and of 3.0% (95% CI 0.4-5.6%; p = 0.016) for hypertension (BP ≥ 95th percentile) in the cohort (18.0% and 6.8%, respectively, increased from 12.0% and 3.8%). CONCLUSIONS: Digital calculators over tables in clinical practice are recommended where possible to improve the accuracy of paediatric BP classification. Substantial rates of elevated BP/Hypertension were found in this cohort of children and adolescents with overweight and obesity.

The Impact of a Family-Based Assessment and Intervention Healthy Lifestyle Programme on Health Knowledge and Beliefs of Children with Obesity and Their Families.

Objective: To determine the impact of a family-based assessment-and-intervention healthy lifestyle programme on health knowledge and beliefs of children and families affected by obesity. Second, to compare the health knowledge of the programme cohort to those of a national cohort in Aotearoa/New Zealand (NZ). Design: This mixed-methods study collected health knowledge and health belief data in a questionnaire at baseline and 12-, 24-, and 60-month follow-up assessments. Health knowledge over time was compared with baseline knowledge and with data from a nationally representative survey. A data-driven subsumption approach was used to analyse open-text responses to health belief questions across the study period. Setting: Taranaki region, a mixed urban−rural setting in NZ. Participants: Participants (caregiver/child dyads) from the Whanau Pakari randomised trial. Results: A greater proportion of the cohort correctly categorised foods and drinks as healthy or unhealthy at 12 months compared to baseline for most questionnaire items. Retention of this health knowledge was evident at 24- and 60-month follow-ups. More than twice as many participants correctly reported physical activity recommendations at follow-up compared to baseline (p < 0.001). Health knowledge of participants was similar to the national survey cohort at baseline, but surpassed it at 12 and 24 months. Participant beliefs around healthy lifestyles related to physical functioning, mental and emotional wellbeing, and enhancement of appearance, and gained greater depth and detail over time. Conclusions: This study demonstrates the important role that community-level healthy lifestyle programmes can have in knowledge-sharing and health promotion.

Evaluation of a New Laboratory Protocol for Newborn Screening for Congenital Adrenal Hyperplasia in New Zealand.

Between 2005 and 2021, 49 cases of classical congenital adrenal hyperplasia were diagnosed in New Zealand, 39 were detected in newborns and 10 were not detected by screening. Currently, for every case of CAH detected by screening, 10 false-positive tests are encountered. Second-tier liquid chromatography-tandem mass spectrometry (LCMSMS) has the potential to improve screening sensitivity and specificity. A new laboratory protocol for newborn screening for CAH was evaluated. Birthweight-adjusted thresholds for first- and second-tier 17-hydroxyprogesterone, second-tier 21-deoxycortisol and a steroid ratio were applied to 4 years of newborn screening data. The study was enriched with 35 newborn screening specimens from confirmed CAH cases. Newborn screening was conducted on 232,542 babies, and 11 cases of classical CAH were detected between 2018 and 2021. There were 98 false-positive tests (specificity 99.96%, PPV = 10.1%) using the existing protocol. Applying the new protocol, the same 11 cases were detected, and there were 13 false-positive tests (sensitivity > 99.99%, PPV = 45.8%, (X2 test p < 0.0001). Incorporating the retrospective specimens, screening sensitivity for classical CAH was 78% (existing protocol), compared to 87% for the new protocol (X2 test p = 0.1338). Implementation of LCMSMS as a second-tier test will improve newborn screening for classical CAH in New Zealand.

Liraglutide for Weight Management in Children and Adolescents With Prader-Willi Syndrome and Obesity.

CONTEXT: Prader-Willi syndrome (PWS) is characterized by lack of appetite control and hyperphagia, leading to obesity. Pharmacological options for weight management are needed. OBJECTIVE: To determine whether liraglutide treatment for weight management is superior to placebo/no treatment in pediatric individuals with PWS. METHODS: This was a multicenter, 52-week, placebo-controlled trial with a 16-week double-blinded period. Adolescents (n = 31, aged 12-17 years; Tanner stage 2-5) and children (n = 24, aged 6-11 years; Tanner stage <2) with PWS and obesity were included. Patients were randomized 2:1 to liraglutide 3.0 mg (or maximum-tolerated dose) or placebo for 16 weeks, after which placebo was stopped. Liraglutide was continued for 52 weeks. All patients followed a structured diet and exercise program throughout the trial. The coprimary endpoints were change in body mass index (BMI) standard deviation score (SDS) from baseline to 16 and 52 weeks. Secondary endpoints included other weight-related parameters, hyperphagia, and safety. RESULTS: Change in BMI SDS from baseline to weeks 16 and 52 was not significantly different between treatments in adolescents (estimated treatment difference: -0.07 at week 16 and -0.14 at week 52) and children (-0.06 and -0.07, respectively). Changes in other weight-related parameters between treatments were not significant. At week 52, hyperphagia total and drive scores were lower in adolescents treated with liraglutide vs no treatment. The most common adverse events with liraglutide were gastrointestinal disorders. CONCLUSION: Although the coprimary endpoints were not met, changes in hyperphagia total and drive scores in adolescents warrant further studies on liraglutide in this population.

The SELFI Study: Iodine Excess and Thyroid Dysfunction in Women Undergoing Oil-Soluble Contrast Hysterosalpingography.

CONTEXT: Hysterosalpingography (HSG) with oil-soluble contrast medium (OSCM) improves pregnancy rates. However, OSCM has high iodine content and long half-life, leading to potential iodine excess. OBJECTIVE: This work aimed to determine the pattern of iodine excess after OSCM HSG and the effect on thyroid function. METHODS: A prospective cohort study was conducted of 196 consecutive consenting eligible women without overt hypothyroidism or hyperthyroidism. All completed the study with compliance greater than 95%. Participants underwent OSCM HSG (Auckland, 2019-2021) with serial monitoring of thyrotropin (TSH), free thyroxine (FT4), and urine iodine concentration (UIC) for 24 weeks. The main outcome measure was the development of subclinical hypothyroidism (SCH), defined as a nonpregnant TSH greater than 4 mIU/L with normal FT4 (11-22 pmol/L) in those with normal baseline thyroid function. RESULTS: Iodine excess (UIC ≥ 300 µg/L) was almost universal (98%) with UIC peaking usually by 4 weeks. There was marked iodine excess, with 90% and 17% of participants having UIC greater than or equal to 1000 µg/L and greater than 10 000 µg/L, respectively. Iodine excess was prolonged with 67% having a UIC greater than or equal to 1000 µg/L for at least 3 months. SCH developed in 38%; the majority (96%) were mild (TSH 4-10 mIU/L) and most developed SCH by week 4 (75%). Three participants met the current treatment guidelines (TSH > 10 mIU/L). Thyroxine treatment of mild SCH tended to improve pregnancy success (P = .063). Hyperthyroidism (TSH < 0.3 mIU/L) occurred in 9 participants (5%). CONCLUSION: OSCM HSG resulted in marked and prolonged iodine excess. SCH occurred frequently with late-onset hyperthyroidism occasionally. Regular thyroid function tests are required for 6 months following this procedure.

Feasibility, safety, and efficacy of 12-week side-to-side vibration therapy in children and adolescents with congenital myopathy in New Zealand.

This pilot study explored the feasibility and effectiveness of vibration therapy (VT) on muscle and bone health, motor performance, and respiratory function in patients with congenital myopathy (CM). Eleven participants with CM (11.5 ± 2.8 years) underwent 12 weeks of side-alternating VT at 20 Hz for nine minutes per session, four days a week. VT was preceded by a 12-week control period. Assessments included dual-energy X-ray absorptiometry scans, 6-minute walk and 10-meter run tests, muscle function and motor performance assessment, dynamometry, and pulmonary function. VT was well-tolerated, with occasional mild itchiness reported. The median compliance level with VT treatment was 75%. 12 weeks of VT improved the total score of motor function performance by 2.4 units (p=0.006) and velocity rise maximum of the chair rising test by 0.11 m/s (p=0.029). VT was shown to be feasible, safe, and associated with improving motor function performance. Our findings support further exploration of VT's potential health benefits to patients with CM in larger studies involving a longer intervention period.

Caregiver survey of preschool children with obesity referred to Whanau Pakari-a multidisciplinary healthy lifestyle intervention programme.

AIM: To examine caregiver perceptions relating to the acceptability of weight screening at New Zealand's B4 School Check (B4SC), and the accessibility and acceptability of a healthy lifestyle programme (Whanau Pakari) for preschool children (Whanau Pakari preschool programme) identified with weight issues. METHOD: An online survey was designed to assess agreement with statements relating to the B4SC healthy weight check and Whanau Pakari programme. Eligible participants (n=125) were caregivers of preschool children identified with obesity (BMI ≥98th centile), or overweight (BMI >91st centile) with weight-related co-morbidities, at the B4SC and referred to Whanau Pakari over the period July 2016 to March 2019. RESULTS: Twenty-nine caregivers responded to the survey (23%). The majority (76%, n=22) were open to discussing their child's weight. However, whilst most caregivers were comfortable receiving a weight referral to a healthy lifestyle programme for their child, some were ambivalent (24%, n=7) or disagreed (21%, n=6) to feeling comfortable about this. Furthermore, only 38% (n=11) of caregivers were concerned about their child's weight. CONCLUSIONS: Findings reveal a reasonable level of acceptability by caregivers to aspects of the B4SC healthy weight check. However, caregiver perceptions may not always be in alignment with the support offered by B4SC health professionals. Regular healthy lifestyle messaging by health professionals, and positive referral experiences, are key to subsequent engagement with healthy lifestyle programmes.

Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results.

PURPOSE: The objectives of the ongoing, Phase 3, open-label extension trial enliGHten are to assess the long-term safety and efficacy of weekly administered long-acting growth hormone lonapegsomatropin in children with growth hormone deficiency. METHODS: Eligible subjects completing a prior Phase 3 lonapegsomatropin parent trial (heiGHt or fliGHt) were invited to participate. All subjects were treated with lonapegsomatropin. Subjects in the United States switched to the TransCon hGH Auto-Injector when available. Endpoints were long-term safety, annualized height velocity, pharmacodynamics [insulin-like growth factor-1 SD score (SDS) values], and patient- and caregiver-reported assessments of convenience and tolerability. RESULTS: Lonapegsomatropin treatment during enliGHten was associated with continued improvements in height SDS through week 104 in treatment-naïve subjects from the heiGHt trial (-2.89 to -1.37 for the lonapegsomatropin group; -3.0 to -1.52 for the daily somatropin group). Height SDS also continued to improve among switch subjects from the fliGHt trial (-1.42 at fliGHt baseline to -0.69 at week 78). After 104 weeks, the average bone age/chronological age ratio for each treatment group was 0.8 (0.1), showing only minimal advancement of bone age relative to chronological age with continued lonapegsomatropin treatment among heiGHt subjects. Fewer local tolerability reactions were reported with the TransCon hGH Auto-Injector compared with syringe/needle. CONCLUSIONS: Treatment with lonapegsomatropin continued to be safe and well-tolerated, with no new safety signals identified. Children treated with once-weekly lonapegsomatropin showed continued improvement of height SDS through the second year of therapy without excess advancement of bone age.