BRADLEY MILLER, M.D.

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This research reviewed 219 studies on the effects of growth hormone therapy in children with growth hormone deficiency, focusing on which outcomes were most frequently reported over nearly two decades. The most commonly tracked results were changes in height and growth speed, with safety outcomes like injection site issues also noted. Out of the 342 different outcomes reported, only 9 were related to quality of life, indicating a gap in understanding how the treatment affects children's everyday lives. Who this helps: This information benefits doctors and researchers working with children who have growth hormone deficiency.

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The study looked at how children with congenital adrenal hyperplasia (CAH) grow differently than children without the condition. Researchers found that children with CAH have shorter heights and weights and an earlier increase in body fat compared to typical growth patterns, with girls starting this change at about 3.3 years and boys at 3.9 years, while the general population starts at 5-8 years. This is important because having specific growth charts for CAH will help doctors monitor these children's growth and evaluate new treatments more effectively. Who this helps: Patients with congenital adrenal hyperplasia and their healthcare providers.

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This study looked at the heart and blood vessel health of patients with Fanconi anemia (FA) who had undergone a type of stem cell transplant. Researchers found that patients with FA had higher artery thickness and stiffer blood vessels compared to healthy individuals—specifically, artery thickness was 0.56 mm for FA patients versus 0.43 mm for controls. This is important because stiffer arteries can increase the risk of heart disease, which is a major concern for FA patients as they age. Who this helps: This benefits patients with Fanconi anemia, as well as doctors managing their long-term health care.

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This study focused on a rare and aggressive cancer called adrenocortical carcinoma (ACC), which currently has few effective treatments. Researchers found that a protein called MMP-14 is crucial for the survival of ACC cells; when they inhibited or silenced this protein, the cancer cells could not survive well and showed increased DNA damage. Specifically, reducing MMP-14 led to a significant decrease in cell viability in lab-grown ACC cells, demonstrating it could be a promising target for new cancer therapies. Who this helps: This research benefits patients with adrenocortical carcinoma by providing potential new treatment options.

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This study looked at what patients and caregivers want in a once-weekly injection device for treating growth hormone deficiency. It involved 100 participants, including adults and caregivers of children and adolescents, who expressed that the most important features were lower injection pain (27%), fewer injections (23%), and easier preparation (16%). The findings help highlight that a device similar to one called somapacitan-beco was preferred by 82% of respondents, suggesting that better-designed injection options can improve treatment adherence and health outcomes. Who this helps: This benefits patients with growth hormone deficiency and their caregivers.

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This study focused on the effectiveness of a treatment called leuprolide acetate used for children with central precocious puberty (CPP), which causes early onset of puberty. The results showed that 84% of the children had low levels of a hormone called luteinizing hormone (LH) after 24 weeks, and this number increased to 86% by 48 weeks, indicating that the treatment was working well. It's important because successfully managing early puberty can help prevent growth and development issues in children. Who this helps: This benefits children with CPP and their families by ensuring effective treatment and healthier development.

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This study looked at how surgical reports written by trainees compared to those written by experienced doctors when it came to billing for endocrine surgeries, which include operations on glands like the thyroid and parathyroid. Researchers analyzed over 700 operations and found that while trainees sometimes missed important coding that could affect payment (like for special monitoring during surgery), the adjustments made by the supervising doctors prevented any loss in revenue for the hospital. This matters because it highlights the need for better training for trainees on billing practices to ensure they can capture all the necessary details in their reports. Who this helps: This helps trainees and hospitals by improving medical education and financial understanding.

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This study looked at how different patterns of a protein called aldosterone synthase might affect the health of patients with primary aldosteronism who had surgery. Researchers reviewed data from 43 patients and found five distinct staining patterns in the tissue samples. While 86% of patients experienced a complete biochemical cure after surgery, only about 23% achieved a complete clinical cure, and there was no relationship between the staining patterns and patient outcomes. Who this helps: Patients with primary aldosteronism and their doctors.

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This study focused on long-acting growth hormone (LAGH) treatments for children who have a deficiency in growth hormone. Experts from around the world reviewed existing research and established guidelines for using these new therapies, noting that LAGH could lead to fewer injections and better treatment adherence over time. However, they emphasized the need for more long-term data to ensure these treatments are safe and effective in everyday use. Who this helps: This helps pediatric patients with growth hormone deficiency and their doctors by providing clearer treatment options.

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This study looked at the impact of newborn screening for a disease called adrenoleukodystrophy (ALD) in children in Minnesota over the past five years. The researchers found that of the 43 children diagnosed with ALD, six boys developed adrenal insufficiency, and two boys progressed to a more severe form of the disease called cerebral ALD, both of whom received stem cell transplants. This early screening has helped detect these conditions before symptoms appeared, potentially improving treatment outcomes for affected children. Who this helps: This benefits young patients with ALD and their families.

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This study examined how missing growth hormone injections affects the growth of children with growth hormone deficiency (GHD). Researchers found that children who received their daily injections regularly grew significantly taller, averaging an increase of 6.8 cm per year, compared to those who missed injections, who only grew 2.3 cm annually. This emphasizes the importance of sticking to the treatment schedule for improving growth outcomes in these children. Who this helps: This helps children with growth hormone deficiency and their caregivers.

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This study looked at how children with growth hormone (GH) deficiency respond to treatment with either daily or weekly GH therapies. The researchers found that children with the lowest initial GH levels (3 micrograms per liter or less) saw the best growth rates over a year, growing at an average rate of about 2.79 cm in height by week 104, compared to those with higher initial GH levels who saw less growth. This is important because it highlights that children with lower GH levels may require more aggressive treatment to achieve better growth outcomes. Who this helps: This helps children with GH deficiency and their doctors by guiding treatment choices for better growth.

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This study looked at how effective and safe a new treatment called somapacitan is for children with growth hormone deficiency (GHD) over a period of three years. Researchers found that both children who started with somapacitan and those who switched from daily growth hormone showed good growth rates, with average height increases of around 7.4 cm and 7.8 cm per year, respectively. Importantly, both groups had similar, healthy levels of insulin-like growth factor, which is important for growth, and experienced few side effects. Who this helps: This study benefits children with growth hormone deficiency and their families.

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This study focused on creating a standard list of data points that should be tracked when monitoring the safety and effectiveness of growth hormone treatment in adults with a condition known as growth hormone deficiency (AGHD). Researchers gathered information from 35 studies involving over 6,700 patients and concluded that 45 essential data items should be collected to ensure consistent and meaningful comparisons in future studies about AGHD treatment outcomes. This matters because having a standardized dataset allows for better assessment and understanding of how well growth hormone therapy works across different populations and settings. Who this helps: This helps patients with growth hormone deficiency and the doctors treating them.

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Researchers studied how effective growth hormone (GH) treatment is for children with growth hormone deficiency (GHD) based on their age when treatment begins, focusing on those under 2 years old versus those 2 years and older. They found that children treated before age 2 had a larger improvement in height after one year (an average increase of 1.4 units) compared to those treated at 2 or older (an increase of 0.75 units), and this growth advantage continued even after 10 years. However, younger children also experienced more serious side effects, with 3.3% facing serious adverse events compared to just 0.67% in older children. Who this helps: This information benefits doctors and caregivers of young children diagnosed with growth hormone deficiency.

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Researchers tested a new rapid COVID-19 test using tiny nanodiamonds that can detect the virus more accurately and quickly. They found that this test was 95.1% sensitive and 100% specific in detecting SARS-CoV-2 from 103 patient samples and could identify infections about 2 days earlier than standard tests. This faster detection can help reduce the spread of the virus and keep communities safer. Who this helps: This helps patients and public health officials by enabling quicker COVID-19 detection and response.

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This research looked at how accurately surgery residents evaluate their own skills in endocrine surgery compared to their teachers’ evaluations. They analyzed 698 assessments from various institutions and found that there was strong agreement between learners and teachers, with a correlation score of 0.66 overall. Specifically, learners were good at judging their technical skills during surgery but typically rated their pre-operative and post-operative skills lower than their teachers did. Who this helps: This study benefits surgery residents and their educators by improving training and assessment methods in endocrine surgery.

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This study looked at somapacitan, a long-acting growth hormone treatment for people with growth hormone deficiency (GHD), to see how well it works compared to daily injections. Researchers found that patients using somapacitan experienced continued growth over time, similar to those on daily growth hormone, and it has been shown to be safe for nearly 4 years in kids. This is important because it offers a more convenient option for patients who need growth hormone, reducing the number of injections and improving treatment adherence. Who this helps: This helps patients with growth hormone deficiency, especially children and their families.

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This study looked at how well meniscus repairs hold up over six years in patients who had a second surgery to fix their knee ligaments after an initial surgery. Out of 221 patients studied, 16% needed another surgery due to failed meniscus repairs, with the failure rate being much higher for repairs on the inner side of the knee (20.6%) compared to the outer side (5.7%). These findings are important because they highlight the challenges of repairing the meniscus in patients who have already had knee surgery, guiding doctors on what to expect in terms of recovery. Who this helps: This information is valuable for doctors and patients undergoing knee surgeries, particularly those with previous ligament repairs.

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Researchers studied how to streamline the data collection process for children with growth hormone deficiency (GHD) receiving recombinant human growth hormone (rhGH) therapy. They worked with 12 clinical experts from various organizations and identified 63 important data points to monitor progress in these children, with specifics on how often to collect them: 20 points once, 14 every 6 months, and 9 every 12 months. This streamlined approach aims to simplify the work for doctors while ensuring safety and effectiveness in treating children with GHD. Who this helps: This benefits doctors and healthcare providers treating children with growth hormone deficiency.

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This study looked at a large group of over 240,000 patients to find specific changes (variants) in a protein called IGF-1, which is important for growth and development. Researchers found that 1,099 patients (about 0.45%) had these variants, with most changes occurring in certain parts of the protein. Understanding these variants is important because they can affect how well IGF-1 works in the body, potentially leading to health issues. Who this helps: This benefits patients with growth disorders and their doctors.

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This study explored how well a special imaging technique using near-infrared fluorescence could help surgeons see the thoracic duct during neck surgery. They found that this method allowed visualization of the thoracic duct in 64% of cases, and it was the only method that worked for 17% of patients. Using this technique was safe, with no long-lasting complications following surgery. Who this helps: This helps surgeons and patients undergoing neck surgery, especially those with previous surgeries or radiation treatment.

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This research focused on a new treatment called Somatrogon for children with growth hormone deficiency (GHD), which allows for a once-a-week injection instead of daily shots. The study found that Somatrogon worked equally well as daily growth hormone treatments in helping kids grow, with no serious side effects reported. This is important because it means that children can have a more convenient treatment option that may improve their overall quality of life. Who this helps: This benefits children with growth hormone deficiency and their families.

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This study looked at how children with Fanconi anemia (FA) grow compared to children without this condition. Researchers found that boys with FA often fall below normal growth percentiles, and girls show a drop in weight growth between 6 and 24 months but stabilize after that. Using these findings, the researchers created growth charts specifically for FA patients, which can help doctors and families understand and manage their growth better. Who this helps: This helps patients with Fanconi anemia and their doctors.

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This study focused on somapacitan, a long-acting growth hormone treatment designed to manage growth hormone deficiency in both children and adults. Researchers found that using somapacitan reduces the need for daily injections, which has been a major barrier for patients; with this treatment, there may be increased patient adherence and better health outcomes. The findings highlight somapacitan’s effectiveness and safety compared to other long-acting hormone treatments. Who this helps: This helps patients with growth hormone deficiency who struggle with daily injections.

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This study focused on how to better support children with growth hormone deficiency (GHD) due to brain cancer as they transition to adult healthcare. Experts found that many young adults face challenges, such as the prioritization of cancer treatment and a lack of knowledge about growth hormone therapy's benefits, which can lead to gaps in care. Improving education for patients and healthcare providers, as well as enhancing coordination among medical teams, is important to ensure these survivors receive the treatment they need during this critical transition. Who this helps: This helps patients who are childhood cancer survivors with growth hormone deficiency.

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This study looked at patients with adrenal hemorrhage, a condition where there is bleeding in the adrenal glands, affecting 193 individuals over 16 years. They found that most cases (47%) were due to trauma, and 62% of patients had bleeding outside the glands. Additionally, 12% of patients had adrenal nodules, but many weren't detected during initial scans, indicating the need for better follow-up imaging to identify these nodules. Who this helps: This research benefits doctors and patients with adrenal issues by improving diagnosis and management strategies.

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This study looked at the experiences of children with growth hormone deficiency (GHD) and their caregivers, focusing on their journey from diagnosis to treatment. Researchers spoke with four patients and their caregivers, who shared their challenges, including varied times from when they suspected GHD to when they received a diagnosis, and issues with treatment access and administering medication. The study found that effective treatment led to significant growth benefits, motivating families to stick with daily injections, and it highlighted the need for better educational resources and support systems for patients and caregivers. Who this helps: This helps patients with GHD and their families by improving their understanding and experience with treatment.

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This study looked at how sticking to a daily growth hormone treatment affects the height growth of children with growth hormone deficiency over one year. Among 181 children evaluated, those who consistently took their medication grew an average of 10.2 centimeters in a year, while those who didn’t follow the treatment closely grew about 9.8 centimeters. Although there was a slight difference in growth, it wasn't significant, which could be due to various factors like the small number of participants and the short study duration. Who this helps: Patients with growth hormone deficiency and their healthcare providers.

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This study looked at how a protein called VEGF affects blood vessel leakage in boys with a severe brain condition called cerebral adrenoleukodystrophy (CALD), particularly focusing on those with adrenal insufficiency. Researchers found that treating six boys with a combination of a medication called bevacizumab, along with other drugs, resulted in some improvement in brain imaging, but the boys still continued to experience worsening symptoms. This matters because while some treatment effects were positive, it shows the complexity of CALD and the need for better therapies. Who this helps: This helps patients with cerebral adrenoleukodystrophy and their families.

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Researchers placed an underwater microphone near Heard Island to listen for different types of whales from September 2017 to March 2018. They found that sperm whales were rarely heard from September to January, with only 35 hours of sounds recorded, but their calls increased dramatically in February and March, totaling 684 hours. Understanding the presence and patterns of these whales helps scientists protect these animals and their habitat better. Who this helps: This helps marine biologists and conservationists working to protect whale populations.

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This study looked at how to treat primary aldosteronism (PA), a condition that causes high blood pressure and can lead to serious health problems. The researchers examined different surgical options and found that surgery can effectively lower blood pressure in patients with PA, which can significantly reduce their risk of other health issues. This is important because lowering blood pressure can lead to better health outcomes and longer lives for patients affected by this condition. Who this helps: This benefits patients with primary aldosteronism and their doctors.

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This research study examined the clinical and genetic features of 280 people with congenital disorders of glycosylation (CDG), a group of rare genetic conditions affecting how the body processes sugars. Key findings showed that most participants (50.4% female, 49.6% male) were diagnosed around 2.7 years after noticing symptoms, with 77% experiencing developmental delays, 56% having low muscle tone, and 42% facing feeding difficulties. Understanding these features helps improve care and future treatments for individuals with CDG. Who this helps: Patients with congenital disorders of glycosylation and their families.

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This study looked at Fanconi anemia, a rare genetic disorder that affects the body's ability to repair DNA and leads to serious health issues like bone marrow failure and cancer. The researchers reviewed various studies to understand how this condition affects different organs over time, revealing that improvements in treatment have increased life expectancy but also highlighted ongoing health challenges. This information is important because it helps improve the care and quality of life for those living with Fanconi anemia. Who this helps: Patients with Fanconi anemia and their healthcare providers.

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This study looked at what surgical techniques during revision ACL (anterior cruciate ligament) surgery can help improve patient outcomes six years later. Researchers found that using an interference screw instead of a cross-pin for bone fixation led to much better recovery results, doubling the chances of improved knee function scores and significantly reducing the risk of needing another surgery. Choosing specific surgical approaches, like a transtibial approach, also contributed to better long-term outcomes. Who this helps: This helps patients recovering from ACL surgeries, particularly those needing revision surgeries.

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This study compared weekly injections of somatrogon to daily injections of somatropin to treat children who have trouble growing due to growth hormone deficiency. The findings showed that children receiving weekly injections grew at a rate similar to those on daily injections, and both methods were equally safe for the children. This matters because it offers a more convenient treatment option for families without compromising effectiveness or safety. Who this helps: This helps children with growth hormone deficiency and their families.

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This study looked at adrenocortical carcinoma (ACC), a rare and hard-to-treat cancer, to better understand its complexity at the cellular level. Researchers found different types of cells within tumors and discovered that some cells were experiencing replication stress, which could make them more vulnerable to certain treatments. Specifically, they identified that these stressed cells might be sensitive to a treatment targeting a protein called ATR, offering new possibilities for therapy in advanced ACC cases. Who this helps: This benefits patients with advanced adrenocortical carcinoma by providing insights that could lead to new treatment options.

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This study looked at a new machine learning model to predict the risk of failure in knee surgeries where the anterior cruciate ligament (ACL) is reconstructed, specifically focusing on patients who had previous surgeries. Out of 960 patients, 5.7% (55 patients) had their graft fail after six years. The model named AutoPrognosis was the most effective at predicting failure, which could help doctors better assess individual patient risks based on factors like previous surgery details and the type of graft used. Who this helps: This helps patients undergoing ACL surgeries and their doctors by providing better risk assessments.

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This study looked at how the location and depth of uterine fibroids affect the surrounding uterine tissue, specifically a layer called the endometrium. Researchers created a detailed computer model and found that fibroids located at the top of the uterus and deep within the tissue caused the most stress, with loads on the endometrium being 10-20% higher in these cases. Understanding these effects is important because it can help explain issues like abnormal bleeding and infertility, aiding doctors in making better treatment decisions. Who this helps: This research benefits patients experiencing complications from uterine fibroids, especially women dealing with abnormal bleeding or infertility.

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This study looked at the effectiveness and safety of a treatment called somapacitan, which is a long-lasting version of growth hormone, in children who have growth hormone deficiency (GHD). After two years, children given somapacitan grew at a rate of about 8.4 cm per year, similar to those who switched from daily growth hormone injections, which was 8.7 cm per year. This is important because it shows that somapacitan is a reliable alternative that is well-liked by patients and their families—90% preferred it over daily injections. Who this helps: Children with growth hormone deficiency and their caregivers benefit from this treatment option.

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This study created new growth charts that don't separate data by sex, which can help in caring for transgender and non-binary youth. Researchers analyzed information from over 39,000 young people aged 2-20 and found that these new charts can give better insights into growth patterns, especially after age 10 when growth differences between sexes become more noticeable. Having these charts is important because they provide a more appropriate way to monitor growth for transgender youth, ensuring healthcare providers can better support their development. Who this helps: This helps transgender and non-binary youth and their healthcare providers.

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This study looked at how children with growth hormone deficiency (GHD) respond to a new treatment called somapacitan, which is given once a week instead of daily. The researchers analyzed data from over 200 kids and found that somapacitan's dosing can effectively increase a hormone called IGF-I that helps track growth. The findings show that this treatment allows for flexible timing on when doses can be given while still supporting healthy growth in these children. Who this helps: This benefits children with growth hormone deficiency and their doctors.

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This study looked at a specific shoulder injury called Humeral Avulsion of the Glenohumeral Ligament (HAGL) in patients with anterior shoulder instability. Out of 915 patients, only 21 (about 2.3%) had HAGL lesions, and one-third of these were missed on MRI before surgery. Patients with HAGL lesions also had fewer common shoulder injuries compared to those without HAGL and were much more likely to require major surgery to fix the instability. Who this helps: This research is important for doctors treating shoulder injuries, as it highlights how easily HAGL lesions can be overlooked and the need for careful assessment to ensure proper treatment.

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This study compared how pediatric endocrinologists feel about using a once-weekly growth hormone treatment called somatrogon versus the standard daily treatment, which is recombinant human growth hormone (rhGH). Out of 24 physicians surveyed, 75% preferred somatrogon, saying it was more convenient and less burdensome; 62.5% reported being very satisfied with it compared to just 16.7% for the daily treatment. This matters because a weekly option can make treatment easier for both healthcare providers and families, potentially leading to better care for children with growth hormone deficiency. Who this helps: This helps patients with growth hormone deficiency and their families.