Hormone Research in Paediatrics最新文献

Introduction: To address disparities in clinical research, we present strategies to optimize recruitment of underrepresented families into the Building the Evidence to Address Disparities in Type 1 Diabetes (BEAD-T1D) study.

Methods: A bilingual/bicultural Latino research assistant (RA) was hired to facilitate culturally congruent recruitment for pediatric type 1 diabetes families. The RA screened, approached, and consented families using their preferred language, time of contact, and answered personal concerns around research. Families were given the option to consent during outpatient clinic visits (in-person, or virtually via video/phone call) at a pace set by the parent/guardian to ensure understanding.

Results: Sixty-four families (Hispanic-65%, Non-Hispanic White [NHW]-17%, Non-Hispanic Black-1%, and Other-4%) were eligible. Of 49 approached, 32 consented (39 ± 7.9 years; female-81%; Hispanic-72%, NHW-28%, <50K income-69%, Spanish-speaking-50%). Clinic approaches were important to successful consent: 87% of the clinic approaches resulted in consent. Barriers to clinic approaches for RA included late/no response from clinicians, care team ending visit, and bandwidth/connectivity issues. Facilitators to clinic approaches included collaborative clinic care teams, flexible RA hours, and patient screening days in advance. We exceeded our recruitment goals for surveys (31/30), focus groups/interviews (26/20), and advisory board (22/10).

Conclusions: We identified that culturally and linguistically congruent staff, flexible recruitment practices, and prioritizing participant availability were solutions to recruit a diverse study cohort resulting exceeding recruitment goals. Cultural interpersonal relationships formed with families addressed barriers to research participation within and outside of the medical system. These strategies suggest equitable clinical trial recruitment is feasible in diabetes research.

Introduction: The COVID-19 pandemic necessitated worldwide lockdowns in 2020 and 2021, with restrictions on physical activity and changes in eating habits. The aim of this study was to investigate temporal trends in body mass index (BMI) and BMI Standard Deviation Score (SDS) in three international type 1 diabetes (T1D) registries between 2018 and 2021.

Methods: Data were extracted from DPV (Germany/Austria/Luxembourg/Switzerland), T1D Exchange Quality Improvement Collaborative (T1DX-QI, USA), and the Australasian Diabetes Data Network (ADDN, Australia/New Zealand). The period affected by the COVID-19 pandemic was defined as March to December 2020 and March to December 2021 and compared with the respective 9-month periods in 2018 and 2019. Estimated mean BMI (adults ≥19 years) and WHO BMI SDS (children and adolescents 5 to <19 years) were calculated, adjusted for sex, age, HbA1c, and diabetes duration. Adjusted mean proportions overweight (BMI ≥25 in adults or BMI SDS >1 in children and adolescents 5 to <19 years) and obese (BMI ≥30 kg/m2 or BMI SDS >2 in children and adolescents 5 to <19 years) were also calculated, adjusted for sex, age, HbA1c, and diabetes duration.

Results: The study population comprised: ADDN (n = 14,624, median age 15.7 years, 51% male); DPV (n = 62,732, 16.1 years, 53.3% male); and T1DX-QI (n = 22,942, 17.1 years, 52.1% male). In the DPV registry, BMI SDS in children and adolescents and BMI in adults increased consistently between 2018 and 2021 (p < 0.001). In ADDN and T1DX-QI, variable changes in BMI and BMI SDS were seen in adults and young people. Close to 50% of people in all registries were either overweight or obese. Proportions overweight remained relatively stable across the 4 years. The proportion of obesity increased in children 5 to <10 years.

Conclusions: A slight increase in BMI and BMI SDS observed before the pandemic continued during the pandemic years. The proportion of overweight and obesity was overall high. Healthy weight remains a priority for people with T1D.

Introduction: This survey investigates brain MRI practices for isolated GHD among Pediatric Endocrine Society (PES) members, focusing on gadolinium-based contrast agents (GBCAs) versus non-contrast MRI.

Methods: A 15-question survey was distributed to 1,553 PES members, capturing data on GBCA usage, non-contrast imaging access, and awareness of gadolinium retention.

Results: A total of 85% of respondents routinely order brain MRIs for isolated GHD, with 60% using GBCAs. Most respondents have access to high-resolution non-contrast imaging, though 54% are unaware of gadolinium retention risks.

Conclusion: High-resolution non-contrast MRI demonstrates diagnostic efficacy, suggesting a shift away from GBCAs in clinic practice for isolated GHD. The survey forms the basis to update PES guidelines in the evaluation of isolated GHD.

Background: Despite all the technological advances in treatment of patients with type 1 diabetes (T1D), glucose control remains suboptimal in most patients. In addition, a relatively high percentage of patients with T1D, including children, have obesity. Therefore, new interventions are required that focus their effects on weight loss, in order to help with associated insulin resistance and improve glycemic control.

Summary: GLP-1 receptor agonists (GLP-1 RAs) have proven to be effective and safe in adults with T1D, showing improvement in glycemic control, body weight and cardiorenal protection. GLP-1 RAs are also approved for children with obesity (above the age of 12 years) or type 2 diabetes (above the age of 10 years). However, currently these medications are not approved for use in children with T1D. Only a few published studies have evaluated their efficacy and safety for this indication.

Key message: This review presents the rationale and experience of add-on GLP-1 RA therapy to pediatric and adolescent patients with T1D, otherwise treated, from RCTs and real-world data. Results of studies of GLP-1 RA in children with T1D are still pending, while large multicenter randomized controlled trials (RCTs) in this population are lacking.

Introduction: Pediatric thyroid nodules exhibit higher malignancy rates compared to adults and are associated with increased incidences of metastases and recurrences. The American Thyroid Association recommends surgery for indeterminate thyroid biopsies in children based on these higher malignancy risks, though this approach may lead to overtreatment. However, there remains a lack of comprehensive pediatric data to inform clinical decisions. This study examines the risk of malignancy (ROM) in pediatric thyroid nodules using the Bethesda System for Reporting Thyroid Cytopathology (TBSRTC) and assesses the diagnostic accuracy of fine-needle aspiration (FNA) biopsy compared to histological outcomes.

Methods: A retrospective cross-sectional analysis was performed on patients under 19 years with thyroid nodules who underwent FNA and thyroidectomy at a tertiary care center. The sensitivity, specificity, positive predictive value, negative predictive value, and ROM of cytological biopsies were evaluated using TBSRTC criteria, with histology serving as the gold standard. Two analyses were conducted to assess diagnostic accuracy: (a) TBSRTC II as negative and TBSRTC VI as positive and (b) TBSRTC II as negative with TBSRTC V and VI as positive. For neoplasia detection, TBSRTC II was deemed negative, while TBSRTC IV, V, and VI were considered positive. TBSRTC categories III and I were excluded from the performance analysis and evaluated separately. Follicular neoplasm or lesions suspicious for follicular neoplasm (FN/SFN) were treated as positive outcomes, correlated with the presence of adenoma or carcinoma in the surgical specimen.

Results: Of 75 nodules from 73 patients, 28 (37.3%) were benign and 47 (62.6%) malignant. No significant differences in gender or age were noted between groups. The ROM in each TBSRTC was Bethesda I 0/2, 0%; II 0/13, 0%; III 2/7, 29%: IV 6/14, 43%; V 10/10, 100%, and VI 29/29, 100%. A sensitivity of 78.38% and specificity of 100% for FNA in detecting malignancy was found, with an even higher sensitivity (100%) for detecting neoplasia in TBSRTC IV.

Conclusions: This study reveals that indeterminate thyroid nodules in pediatric patients exhibit a higher rate of malignancy compared to adults, yet align with rates previously reported in the pediatric population. These findings highlight the critical need for guidelines tailored specifically to the management of thyroid nodules and thyroid cancer in children.

Introduction: Individuals with differences of sex development (DSD) experience complex, often competing, medical and psychosocial challenges surrounding fertility. The study aimed to characterize how "success" in fertility-related care is conceptualized and attained among individuals with a DSD, their parents or caregivers, healthcare providers, and other stakeholders.

Methods: As part of a larger study, DSD stakeholders (n = 110) participated in semi-structured interviews covering the clinical care of patients with DSD. Primary questions included "What is a successful outcome in DSD care?" and "How do you achieve it?" with fertility as either a spontaneous or suggested topic of discussion. Transcripts were analyzed utilizing a phenomenological approach. This analysis focuses on the extracted themes related to fertility.

Results: Fertility was discussed by 19/24 individuals with DSD, 12/19 parents or caregivers, 35/37 healthcare providers, and 19/30 other stakeholders. Components of successful fertility-related care included (1) specific discussions surrounding the relationship between DSD and fertility potential, options for fertility preservation, and options for non-biologic parenthood; (2) early and repeated introduction of these topics; and (3) consideration of age, developmental maturity, and cultural context on decisions around fertility. Challenges included the lack of fertility outcome data in this population and the irreversibility of gonadectomy. Trade-offs identified included anatomic typicality versus function, fertility preservation versus cancer risk reduction, and balancing the different priorities of stakeholders.

Discussion/conclusions: A wide range of DSD stakeholders highlighted the importance of addressing fertility concerns in achieving favorable outcomes for individuals with DSD. These stakeholder perspectives should inform fertility-related education, shared decision-making processes, and clinical care.

Introduction: Most children with adrenocortical tumors (ACTs) present with accelerated growth and skeletal maturation at diagnosis, which potentially compromises their adult heights (AHs). Knowledge about growth and pubertal patterns after ACT resection is scarce. This study presents the pubertal and auxological development of patients treated for ACT and followed up at a single pediatric endocrinology service in Brazil.

Methods: Retrospective cohort study including 63 patients (47 girls) followed up between 1966 and 2021. Pubertal and auxological data from ACT diagnosis to AH were analyzed.

Results: At diagnosis, the patients had median values of bone age (BA) more advanced than chronological age (CA), height standard deviation score greater than target height (TH-SDS), and predicted adult height (PAH-SDS) lower than TH-SDS. The difference between BA and CA decreased gradually during follow-up and the PAH-SDS moved closer to the TH-SDS 7 years after tumor resection. Puberty started at a median CA of 9.3 (8.3-11.3) years in girls and 9.9 (9.2-13.6) years in boys. Nine patients (6 girls) developed central precocious puberty (CPP), which was influenced by a CA >4 years at diagnosis. The difference between AH-SDS and TH-SDS was not significant (p = 0.3). The factors independently associated with AH below TH were CA >4 years at diagnosis, time between clinical manifestation and diagnosis >1 year, and development of CPP.

Conclusion: Most patients treated for ACT during childhood attained AH within the TH despite presenting with advanced skeletal maturation at diagnosis. Development of CPP was not infrequent.