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Clinical Trial Shows Efficacy of a Once‑Weekly Growth Hormone Analog in Children With Noonan Syndrome

Dr. Tansey

A new multinational, phase 3 clinical trial published online ahead of print in the European Journal of Endocrinology studied the growth promoting effects of once‑weekly growth hormone analog versus standard daily growth hormone (GH) therapy for children living with Noonan syndrome. The results show that the weekly analog approach is superior to the daily approach in terms of enhancing growth rate. The study, titled “Once‑weekly Somapacitan in Children with Noonan Syndrome: Randomised Controlled Phase 3 Trial”. The trial authors included contributions from Dr. Tansey from our Division. A total of 77 children with Noonan syndrome were enrolled in the trial and randomized 2:1 to the once‑weekly standard or daily GH preparations over a one-year period. The children receiving the weekly analog grew a little faster (13% on average, statistically significant) compared with the daily GH group. Importantly, the weekly analog was well tolerated, showing a safety profile comparable to daily GH. It will be important to ascertain the long term impact of the weekly analog on final adult height in this population. These findings highlight a promising, less burdensome therapeutic option for families. We thank Dr. Tansey for his role in advancing this impactful work. The abstract of the work can be found at this pubmed link.

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Safety of One Year’s Treatment with Inhaled Insulin in Children with Type 1 Diabetes

Dr. Tansey

Our division has contribution to a clinical study recently published in Diabetes Technology & Therapeutics : “Inhaled Technosphere Insulin in Children with Diabetes: The INHALE-1 Extension Study.” One of our Division physicians, Dr. Tansey, served as a co-author on this important research. This paper describes an extension phase to the INHALE-1 trial to better examine long-term safety of an inhaled form of insulin. The results from the first 26-week phase of trial were published several months ago. Inhaled insulin demonstrated similar glucose levels to conventional therapy with injected insulin but greater treatment satisfaction. The extension phase of the trial allowed participants the option to continue inhaled insulin for another 26 weeks to further evaluate safety. Even after one year’s treatment, there were no serious pulmonary complications among the participants. There was a small increase in hemoglobin A1c from week 26 to week 52 (from 8.2 to 8.6%). The manuscript abstract is available on Pubmed. The results show the potential safety of inhaled insulin for children with type 1 diabetes. We especially wish to thank the research coordinators, research participants and their families who made this study possible.

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Muscle “Recycling Centers” Helps Prevent Type 2 Diabetes

Dr. Norris

A group of researchers have identified a key protein complex in muscle cells that regulates the body’s response to nutrients and helps maintain healthy blood sugar levels. The researchers focused on the role of this complex, known as LRRC8, in muscle lysosomes. Lysosomes are tiny compartments inside cells that act like recycling centers. The researchers found that lysosomal LRRC8 plays a critical role in governing cellular growth and metabolism. When LRRC8 was disrupted in lab-grown muscle cells, lysosomal function was impaired, weakening how muscle cells responded to nutrients and to insulin. Furthermore, mice lacking lysosomal LRRC8 developed increased body fat, insulin resistance, and poor glucose tolerance — conditions closely linked to type 2 diabetes. This research uncovers a previously unknown connection between LRRC8 lysosomal function in muscle cells and whole-body metabolic health, opening new avenues for understanding and potentially treating metabolic diseases such as type 2 diabetes. Dr. Norris from our division was part of the research team and is a co-author on the manuscript. The peer-reviewed findings are published in Science Advances under the title: “Lysosomal LRRC8 complex impacts lysosomal pH, morphology, and systemic glucose metabolism.” The abstract is available on PubMed: https://pubmed.ncbi.nlm.nih.gov/41004571/.

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Advancing Expert Diagnosis and Care for Pancreatogenic Diabetes

Dr. Larson Ode
Dr. Norris

Pancreatogenic diabetes is an underrecognized form of diabetes that develops as a consequence of diseases affecting the exocrine pancreas. Though often overshadowed by type 1 and type 2 diabetes, pancreatogenic diabetes is estimated to account for 1–9% of all diabetes cases, making it a significant contributor to global health burden. This condition can arise from a range of pancreatic disorders, including pancreatitis, pancreatic cancer, cystic fibrosis, surgical removal of the pancreas, and fibrocalculous pancreatic disease. Each of these conditions presents unique diagnostic and therapeutic challenges, underscoring the need for heightened clinical awareness. Two faculty members from our Division, Dr. Larson Ode and Dr. Norris, have authored a comprehensive clinical review on this topic, recently accepted for publication in the Journal of Clinical Endocrinology and Metabolism (JCEM), one of the leading journals in the field.

Their article, titled “Approach to the Patient with Pancreatogenic Diabetes”, synthesizes current evidence and offers practical guidance for clinicians managing these complex cases. The work was co-authored by adult endocrinologist Dr. Yumi Imai. The review emphasizes the importance of timely diagnosis and tailored treatment. For instance, diabetes can be an early warning sign of pancreatic cancer—a disease with poor prognosis and no reliable biomarkers—making clinical suspicion critical. In cystic fibrosis, even mild diabetes can worsen lung function and increase mortality risk, highlighting the need for proactive screening. The authors also discuss the frequent coexistence of exocrine pancreatic insufficiency in these patients, noting that in these cases pancreatic enzyme replacement therapy may improve glycemic control.

We are glad that these authors have been able to bring their recognized expertise in pancreatogenic diabetes to this work, providing clinicians with actionable insights into evolving screening and treatment strategies. The article’s abstract can be found on PubMed (https://pubmed.ncbi.nlm.nih.gov/41252284/), where there are links to the open access full manuscript.

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What Mechanisms Drive Diabetes-Risk in Youth with Pancreatitis?

Dr. Larson Ode
Dr. Catherina Pinnaro

Why does diabetes happen in some children who have experienced pancreatitis? A new study from Dr. Larson Ode and Dr. Pinnaro, along with a former research fellow Dr. Parra Villasmil, delves into this very question, uncovering unexpected patterns. Their work has been just published in the Journal of Pediatric Gastroenterology and Nutrition. The manuscript, titled “Early mechanisms of diabetes development in pediatric pancreatitis – a pilot study”, explores the complex interplay between pancreatitis and diabetes risk in children. Diabetes is a serious complication for children with pancreatitis, affecting nearly 9% during childhood and up to half over a lifetime. Despite this high risk, little is known about the underlying mechanisms or biomarkers that predict which children are most vulnerable. This study takes an important step toward filling that gap. Their findings revealed that 35% of participants exhibited dysglycemia, often linked to insulin resistance. Could insulin resistance be the key mechanism that drives diabetes risk? Another surprise finding was that dysglycemia was associated with higher pancreatic polypeptide levels. These results will help inform future studies that could pave the way for earlier detection and/or targeted interventions to prevent diabetes in this high-risk population. The manuscript is indexed on PubMed at this URL.

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Inhaled Insulin Shows Some Benefits for Children with Type 1 Diabetes in Landmark Clinical Trial

Dr. Tansey

We are proud to announce our division’s contribution to a major clinical study recently published in Diabetes Care: “INHALE-1, A Multicenter Randomized Trial of Inhaled Technosphere Insulin in Children With Type 1 Diabetes.” One of our Division physicians, Dr. Tansey, served as a co-author on this important research. The INHALE-1 trial examined the safety and effectiveness of an inhaled form of insulin compared to standard rapid-acting insulin injections in 230 children and adolescents aged 4 to 17 living with diabetes. Over a 26-week period, participants continued their long-acting basal insulin and used continuous glucose monitoring while receiving either inhaled or injected rapid acting insulin. Compared to the injected insulin group, the inhaled insulin group experienced a similar hemoglobin A1c, similar continuous glucose monitor readings, and lung function. Children using TI reported greater treatment satisfaction and experienced less weight gain compared to those on injected insulin. These findings suggest that inhaled insulin could become a valuable option for some pediatric patients. It is important to note that the study did not compare inhaled insulin to insulin pump-based therapy. The manuscript abstract is available on Pubmed. We especially wish to thank the research coordinators, research participants and their families who made this study possible.

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Vitamin D Repletion in People with Cystic Fibrosis

Dr. Larson Ode

Cystic fibrosis often disrupts gastrointestinal function, impairing the absorption of fat-soluble vitamins such as vitamin D. As a result, individuals with cystic fibrosis are at increased risk for vitamin D deficiency. While daily supplementation is a common corrective strategy, it adds to the already significant treatment burden faced by these patients.

An alternative approach known as stoss dosing involves administering a single, very high dose of vitamin D. This method is effective because vitamin D remains active in the body for several weeks, potentially reducing the need for daily supplementation.

To evaluate the effectiveness of stoss dosing, researchers analyzed the medical records of 58 individuals with cystic fibrosis who received this treatment. Their findings showed that vitamin D levels were increased by the therapy, as measured three months after the dose administration. However, only about half of the patients reached the target vitamin D levels, suggesting that while stoss dosing is likely beneficial, further research and optimization are necessary.

Dr. Larson Ode from our division contributed to the research, which has been peer-reviewed, published in Health Science Report and is publicly available in the PubMed Central repository (link).

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Sugar Surge: Rates of Pediatric Type 2 Diabetes More than Doubled During the Pandemic

Dr. Catherina Pinnaro

Early in the pandemic, pediatric endocrinologists began noticing a rise in new cases of diabetes among youth. To investigate this trend, a group of specialists formed a national consortium representing 23 pediatric centers across the country. Their findings revealed a significant increase in new diagnoses of type 2 diabetes during the pandemic, with cases more than doubling. This increase was observed across all sampled regions of the United States. In contrast, the rise in new-onset type 1 diabetes was less pronounced and did not reach statistical significance.

Encouragingly, the number of new type 2 diabetes cases appeared to return to baseline by the end of the pandemic’s second year.

While the study was not designed to determine the exact causes of the type 2 diabetes surge, the results suggest that the major contributing factors were transient in nature. These temporary factors may have included reduced physical activity, disrupted daily routines and eating habits, and increased psychosocial stress. Perhaps less likely to play a major role would be the physiological effects of the SARS-CoV2 virus itself.

The consortium has now published these findings in the Journal of Clinical Endocrinology and Metabolism. Dr. Pinnaro, from our division, was involved with the consortium’s creation, this research and the writing of the published manuscript. The abstract can be found on PubMed at this link.

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Adrenal Insufficiency after Pediatric ICU

Dr. Radig

Children admitted to the Pediatric Intensive Care Unit (PICU) often receive glucocorticoid steroids as part of their treatment. While these medications can be highly effective in managing critical illness, prolonged use may lead to adrenal insufficiency—a dangerous but treatable condition in which the body cannot produce adequate levels of cortisol. To better understand the risks, researchers reviewed the medical records of 530 pediatric patients who received steroids during their PICU stay. Among these patients, 2.3% were diagnosed with adrenal insufficiency. The study identified several key risk factors including the following: younger age, higher cumulative dose of steroids and the use of steroids specifically for treating excess inflammation. These findings aim to improve the identification of pediatric patients at risk for adrenal insufficiency following critical illness. The study has been published in the Journal of Intensive Care Medicine. The lead author, Dr. Radig, will soon join our Division as a pediatric endocrinology fellow. Also contributing to the the research was Dr. Curtis, also from our Division. The abstract is available on PubMed at the following link.

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Long-Term Study Links Elevated Glucose Levels to Persistent Brain Changes in Children with Type 1 Diabetes

Dr. Tsalikian
Dr. Tansey

In 2018, faculty from our division participated in a multicenter, observational study that added to growing evidence linking elevated glucose levels in children with type 1 diabetes to changes in brain structure. While the initial findings highlighted these associations, they could not determine how such changes might evolve over time. Now, the research team—including Drs. Tsalikian and Tansey from our division—has completed a long-term follow-up of the original study participants. The results, recently published in the journal Diabetes, are available via PubMed (link here) in abstract form. The follow-up spanned 6 to 8 years and revealed that the previously observed brain differences largely persisted. Although the magnitude of some differences diminished slightly, higher average blood glucose levels continued to correlate with more pronounced structural changes. These findings reinforce the importance of maintaining tight glycemic control to potentially mitigate long-term neurological impacts. As the cohort transitions into adulthood, continued monitoring will be essential to fully understand the long-term implications of these early brain changes.