Hormone research in paediatrics
Audio Summaries

Real-world growth outcomes and safety of vosoritide in children with achondroplasia: a Brazilian single center 24 monhts experience

Jul 21, 2026

The authors aimed to evaluate the effects of vosoritide on growth, body proportionality, and safety in children with achondroplasia over a 24-month period in a real-world clinical setting. The study found significant increases in height velocity and height SDS, particularly at 24 months, but noted that individual responses varied and body proportionality remained unchanged. Despite some patients experiencing hypertrichosis, the treatment was well tolerated, indicating its potential for clinical use.

Use of teriparatide for treatment of a 3-year-old child with an activating calcium sensing receptor mutation

Jul 10, 2026

This case study explores the efficacy of teriparatide, an injectable PTH analogue, in treating a 3-year-old child with an activating calcium sensing receptor mutation and severe complications from standard treatment for congenital hypoparathyroidism. The authors aim to demonstrate that teriparatide can provide significant clinical improvement in young patients, addressing the limitations of current treatment options that increase the risk of kidney complications.

Screening and monitoring for presymptomatic type 1 diabetes: a time for action

Jul 9, 2026

The authors aim to highlight the importance of screening for presymptomatic type 1 diabetes (T1D) by discussing its potential benefits, such as reducing complications and enabling access to novel therapies. They also address the barriers to implementing widespread screening programs and emphasize the need for further research to optimize early detection strategies in clinical practice. Overall, the review advocates for adopting early-detection methods as a crucial component of T1D care.

Attitudes and Practices of Youth and Young Adults with Type 1 Diabetes Towards Self-Adjustments of Insulin Pump Settings

Jul 9, 2026

This study aimed to evaluate the frequency and practices of insulin pump self-adjustments among pediatric users of non-automated pumps and their association with glycemic outcomes. The findings revealed that many youth with Type 1 Diabetes rarely adjusted their insulin doses independently, but those who did showed better glycemic control, highlighting the need for enhanced patient education and support for self-management. Additionally, a significant portion of participants expressed interest in using automated insulin adjustment recommendations to improve their diabetes management.

Growth hormone therapy in children born small for gestational age with persistent short stature (SGA-SS): Lessons learned from real-world international databases and directions for future research

Jul 7, 2026

This paper explores the efficacy and safety of recombinant human growth hormone (rhGH) therapy in children born small for gestational age with persistent short stature (SGA-SS), a condition affecting about 10% of this population. The authors highlight the variability in treatment response due to the heterogeneous causes of SGA-SS and emphasize the importance of integrating genetic insights and next-generation patient databases to develop more personalized treatment strategies. Future research directions aim to refine treatment regimens and improve long-term outcomes for affected children.

Sleep Health and Barriers in Adolescents with Type 1 Diabetes, a Mixed Methods Study

Jul 6, 2026

This study investigates sleep patterns and barriers to sleep in adolescents with type 1 diabetes (T1D) to understand how these factors impact their overall health. The findings reveal that over half of the participants do not meet recommended sleep duration, with school demands and diabetes-related issues identified as significant barriers. The authors suggest the need for routine sleep assessments and tailored interventions to improve sleep health in this population.

Circulating mRNA and miRNA signatures used as predictive markers of lifestyle modification outcomes in pediatric obesity management

Jul 2, 2026

The authors aimed to identify molecular markers that could predict the outcomes of lifestyle modifications in children with obesity. By analyzing circulating mRNA and miRNA in a cohort of 159 children, they found significant differences in the expression of specific genes and miRNAs between responders and non-responders to lifestyle changes, highlighting the potential of these markers for personalizing obesity management strategies. Their findings suggest that the balance between STAT3 and PTEN, along with certain miRNAs, may play a critical role in predicting successful outcomes in pediatric obesity interventions.

Functional impact of a novel intronic TG variant in congenital goiter and thyroid dyshormonogenesis

Jun 30, 2026

The authors investigate the pathogenicity of a novel homozygous intronic variant in the TG gene, which is linked to congenital goiter and thyroid dyshormonogenesis. Through cDNA sequencing, they demonstrate that this variant leads to aberrant splicing and a nonfunctional TG protein, contributing to primary congenital hypothyroidism. This study highlights the significance of early genetic diagnosis for effective management and prevention of recurrence in affected families.

PREDICTORS OF RESPONSE TO GROWTH HORMONE TREATMENT AND FINAL HEIGHT IN MEDULLOBLASTOMA SURVIVORS

Jun 11, 2026

The authors investigate the growth characteristics and response to growth hormone treatment (GHT) in survivors of childhood medulloblastoma, aiming to identify factors influencing final height (FH) outcomes. Their findings indicate that a significant majority of patients experience growth retardation and GH deficiency, with early height-SDS at the start of treatment being the key predictor of FH success. The study emphasizes the importance of timely GH deficiency diagnosis and treatment while highlighting concerns regarding the long-term safety of GHT.

The Use Of EQ5D For Assessment Of Health Related Quality Of Life And Its Determinants In Children With Complex Endocrine Conditions

Jun 10, 2026

The authors aim to evaluate the utility of the EQ5D tool for assessing health-related quality of life (QoL) in children with complex endocrine conditions. Their study, involving 216 patients, reveals that the EQ5D scores correlate with the complexity of medical care, indicating that a higher number of specialties involved is associated with worse QoL outcomes. The findings suggest that EQ5D is a valuable instrument for routine QoL assessment in pediatric endocrinology.