Pediatrics

Weekly review: Profound genetic deafness gene therapy, measles increases, and more – Contemporary Pediatrics

Thank you for visiting the Contemporary Pediatrics website. Take a look at some of our top stories from last week (Monday, May 6, to Friday, May 10, 2024), and click on each link to read and watch anything you may have missed.

1.) DB-OTO improved hearing to normal in child with profound genetic deafness

Positive, phase 1/2 preliminary data for Regeneron Pharmaceuticals' DB-OTO, an investigational gene therapy for profound genetic deafness, was presented at the 2024 American Society of Gene and Cell Therapy (ASGCT) annual conference.

Click here for full commentary and data, in this discussion with Lawrence R. Lustig, MD, chair, Department of Otolaryngology-Head & Neck Surgery, Columbia University, and clinical trial investigator.

2.) Measles increase: Discussing vaccination with vaccine-hesitant parents

In a time when vaccine hesitancy is contributing to a rise in measles cases, an understanding of why parents are hesitant is key to help change their narrative.

Click here for the full article, part of the May issue of Contemporary Pediatrics.

3.) Child welfare: Now that we know better, lets do better

Improvement in access to mental health resources for children would decrease vicarious trauma of foster parents and social workers via improved living and working conditions.

Click here for the full article.

4.) Emergency department serves as equitable location for influenza vaccine delivery

Click here to watch the full interview with Courtney Nelson, MD, attending physician, director of Quality Division of Emergency Medicine, Nemours Children's Hospital Delaware; assistant professor of Pediatrics, Sidney Kimmel Medical College.

5.) The pediatrician's role in a multidisciplinary pediatric cardiology team

Carissa M. Baker-Smith, MD, MPH, explains how a multidisciplinary team works together to diagnose and treat hypertension, as well as obesity in children.

"We're not going to find the solutions to these problems by working in isolation within our respective fields or offices, we need to get out, include the patients, the community and the decision making, and very importantly, the general pediatricians."

Click here for the full interview.

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Weekly review: Profound genetic deafness gene therapy, measles increases, and more - Contemporary Pediatrics

Pediatrics

HPV vaccination coverage of US-children aged 9-17 years in 2022 – Contemporary Pediatrics

HPV vaccination coverage of US-children aged 9-17 years in 2022 | Image Credit: Tobias Arhelger- Tobias Arhelger - stock.adobe.com.

Vaccination prevents and controls Human papillomavirus (HPV), the most common sexually transmitted infection (STI) in men and women in the United States. According to data from a National Health Interview Survey and the Centers for Disease Control and Prevention (CDC), in 2022, 38.6% of children aged 9 to 17 years received 1 or more HPV vaccine doses.1

HPV vaccination has been recommended in the United States for girls since 2006 and for boys since 2011, with multiple doses required for boys. Vaccination can begin at age 9, and is targeted for children aged 11 to 12 years to prevent and control associated outcomes of HPV including: genital warts, precancerous lesions, and certain cancers, such as cervical, vaginal, vulvar, anal, penile, and oropharyngeal.1

Parent-reported data from the 2022 National Health Interview Survey is used in the CDC report to describe the percent change of children aged 9 to 17 years who received at least 1 dose of the HPV vaccine by "selected sociodemographic and health characteristics," the report stated.1

That National Health Interview Survey is a nationally representative household survey of the United States civilian noninstitutionalized population, and is conducted continuously throughout the year by the National Center for Health Statistics.1

Overall, for 2022, 38.6% of children received 1 or more HPV vaccine doses. That percentage increased with age, as 7.3% of children aged 9 to 10 years received 1 or more dose, 30.9% of children aged 11 to 12 years, 48.8% among children aged 13 to 14 years, and 56.9% among children aged 15 to 17 years.1

According to the report authors, girls were more likely to received 1 or more HPV vaccine doses compared to boys (42.9% vs 34.6%).1

The percentage of children who received 1 or more vaccine dose also varied by race and Hispanic origin, as Hispanic children were less likely to receive 1 or more doses compared to White non-Hispanic children (34.4% vs 39.9%). Observed differences between Asian non-Hispanic, Black non-Hispanic, White, and Hispanic children were not considered significant.1

Health insurance was also a factor in the percentage of children who received 1 or more vaccine doses, as was parental education and family income.1

Children with private health insurance (41.5%) were more likely to receive 1 or more HPV vaccinations compared to children with Medicaid (37%), other government sponsored coverages (30.2%), and those without insurance (20.7%). Children with Medicaid were more likely to receive vaccine doses compared to those without insurance.1

Increasing parental education was associated with higher vaccination percentages in children, as 31.1% of children who lived in households with parents whose highest education was high school or less. For those with parents who had an associate's degree or some college experience, 40.6% of children received 1 or more vaccine doses. For children with parents whose highest education was a bachelor's degree or higher, 42.1% received at least 1 HPV vaccine dose.1

For family income, 29.9% of children with a family income of less than 100% of the federal poverty level received 1 or more vaccine doses, compared to 45.7% among children with a family income of 400% or more of the federal poverty level.1

According to the US Department of Health and Human Services, $27,750 per year was the 100% federal poverty level in 2022 for a family or household size of 4 in the 48 contiguous states (not Alaska and Hawaii). The 400% poverty level for the same household was $111,000 per year.2

Additionally, children with disability were more likely to receive HPV vaccination compared to those without disability (44.1% vs 37.7%). The same was true for children living in "large metropolitan areas (39.4%), large fringe metropolitan areas (41.1%), and medium and small metropolitan areas (39.4%) compared with those living in nonmetropolitan areas (30.0%)," stated the authors.1

According to the report, the CDC recommends a 2-dose series with 2 doses given 6 to 12 months apart for children aged 11 to 12 years, though vaccination series can start when children are at age 9 years. For those with a weakened immune system and those starting at age 15 years or older, a 3-dose series is recommended.1

References:

1. Villarroel MA, Galinsky AM, Lu PJ, Pingail C. Human Papillomavirus vaccination coverage in children ages 9-17 years; United States, 2022. National Center for Health Statistics. February 2024. Accessed May 10, 2024. https://www.cdc.gov/nchs/products/databriefs/db495.htm#Data%20sources%20and%20methods

2. 2022 poverty guidelines: 48 contiguous states (all states except Alaska and Hawaii). US Department of Health and Human Services. PDF. Accessed May 10, 2024. https://aspe.hhs.gov/sites/default/files/documents/4b515876c4674466423975826ac57583/Guidelines-2022.pdf

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HPV vaccination coverage of US-children aged 9-17 years in 2022 - Contemporary Pediatrics

Pediatrics

East Wenatchee Doctor Awarded For Boosting Child Vaccinations – KPQ

A Confluence Health East Wenatchee pediatrician is being credited with boosting the vaccination rate of children in the area.

Dr. Doug Eisert is being cited for improving the vaccination rates of children significantly at Confluence Health in Wenatchee and East Wenatchee.

According to the Washington Chapter of the American Academy of Pediatrics (WCAAP), Dr. Eisert has stood out for his leadership in raising vaccinations for children and adolescents by 24% at Confluence Health in Wenatchee and 15% at Confluence Health in East Wenatchee, impacting 6,215 patients.

The group says Dr. Eiserts leadership and work with teams at the two Wenatchee area clinics made a significant impact on the health of Hispanic children, improving their vaccination rates by 27.5%.

In his work on increasing HPV vaccination for 9 and 10 year olds, Dr. Eisert helped the clinics improve vaccination coverage by 31%, impacting 1,252 patients at both clinics. .

"Dr. Eisert is a true champion for Wenatchee Valley children and youth, improving public health and health for future generations, said Washington Chapter of the American Academy of Pediatrics executive director Sarah Rafton.

Dr. Eisert said his success is based on a group effort. We worked with several vaccine interest groups and tracked our successes and opportunities in making progress with increasing the vaccination rates of our patients," said Dr. Eisert.

The Leah Layne Memorial Health Leadership Award is named after the late Leah Layne, a long-time campaigner and activist for rural health.

Dr. Eisert stands out to me as exemplar of WCAAP and our members working every day to inspire and support his teammates in clinic to improve child and teen health, and public health, said Washington Chapter of the American Academy of Pediatrics executive director Sarah Rafton.

Gallery Credit: Parker Kane

Link:

East Wenatchee Doctor Awarded For Boosting Child Vaccinations - KPQ

Pediatrics

HCPs can play an important role in addressing the US infant formula shortage – Contemporary Pediatrics

HCPs can play a role in addressing the US infant formula shortage | Image Credit: 279photo - 279photo - stock.adobe.com.

In results from a 2020 survey conducted by the CDC, 45.3% of infants in the United States were exclusively breastfed at the age of 3 months, with this percentage dropping to 25.4% by the age of 6 months.1 The World Health Organization and the US Dietary Guidelines for Americans recommend that all infants should exclusively receive breast milk until the age of at least 6 months.2,3 Caregivers can begin incorporating other solid and liquid foods at this 6-month mark; however, children should continue breastfeeding until the age of 2 years. The CDC reports that 20.8% of breastfed infants receive additional formula supplementation within the first 2 days of life, which can be attributed to a multitude of factors, including lactation issues, scheduling conflicts, and stigma.1 When breastfeeding is not an option, caregivers will turn to infant formulas to meet their childs nutritional needs.

Infant formulas contain important macronutrients such as carbohydrates and proteins, which are crucial to growth and development.4 In addition to the macronutrients, the infant formula contains micronutrients such as vitamin D, iron, and zinc. Vitamin D is a fat-soluble vitamin essential for the development of bones and the prevention of conditions such as rickets by regulating calcium and phosphorus levels in the body.4-6 Iron is required for hemoglobin production and tissue oxygenation, with low levels of iron causing anemia and failure to thrive.4,7 Zinc is a mineral that contributes to growth and immune function, with deficiencies leading to growth failure and skin rashes.4,6 Due to potential complications, it is important for all pediatric patients to meet their nutritional requirements. Unfortunately, when infant formula shortages occur, access is limited, leading to varying conservation efforts or work-arounds that put the infant at risk for the deficiencies and complications noted previously.

Infant formula shortages can be a direct result of supply chain issues, natural disasters, and/or recalls.8,9 One such example is the infant formula shortage in the United States in April 2022. Several brands of powdered infant formulas were found to be contaminated with Cronobacter sakazakii, prompting a nationwide recall due to the risk of sepsis and meningitis after ingestion.10 Another more recent example is the infant formula recall in the United States in December 2023, also due to Cronobacter sakazakii contamination.11 Health care providers (HCPs) play an important role in shortages, as they can provide education and resources to caregivers in need. The resources that HCPs can provide include but are not limited to comparative formulations, imports, proper feeding practices, and milk banks.

The North American Society of Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) has infant formula comparison guides that clinicians can use to make safe interchange recommendations.12,13 For example, NASPGHAN states that Similac Alimentum powder (Abbott) is interchangeable with Extensive HA (Gerber) for infants who require extensively hydrolyzed or hypoallergenic formula.13 HCPs can also recommend alternative formulations such as liquid concentrate or ready-to-feed formulations and provide appropriate mixing instructions, as this varies based on the product formulation.14 For example, powder and liquid concentrate formulations require mixing with water in varied ratios whereas ready-to-feed formulations require no mixing. HCPs should also counsel the family on a use-by date after mixing or opening the formulation, as these vary between the different formulations.15 However, it is imperative to note that cost fluctuates between the different formulations, and financial barriers should be considered.

Furthermore, families can seek imported formulas that are authorized by the FDA and available in US stores as an alternative.12 Initially, the FDA temporarily approved imported formulas that met the nutritional requirements but may not have met the labeling requirements of products in the United States.16 Manufacturers who received temporary approval during the initial shortage have since been provided guidance on labeling requirements in order to continue to market their product in the United States. Imported infant formulas are approved by the FDA with a close examination of the nutrients provided by the individual formula and compared with those required by US standards. Clinicians are encouraged to access the FDAs website, which provides recommendations for appropriate substitutions when switching to an imported infant formula.13,17 Third-party imported formula websites may sell products that are not FDA authorized and do not undergo the scrutiny necessary to mirror the nutritional values of US standards. Accessing and/or ordering from these websites should be avoided.4,18 The FDA provides advice to avoid counterfeit infant formulas by confirming the lot number and use-by dates on the package, checking for damage or label tampering, or calling the manufacturers toll-free line.19 If caregivers have used a specific product in the past, they should look out for discoloration and changes in smell or taste.

If an FDA-authorized imported infant formula is chosen, there are unique considerations. There may be unfamiliar language in the patient-facing directions, such as using the word teats for the nipple of the baby bottle.15 Additionally, the definition of a special infant formulation may vary based on the country of origin when compared with that of the United States. Furthermore, labeling may contain different languages that may not be readily translated. Imported products may use the metric system, requiring unit conversions and subsequent relay of this information to the caregiver. This is a key counseling point, as mixing the formula incorrectly may lead to electrolyte imbalance, seizures, and poor weight gain.15,16

Human milk banks are an option for caregivers if alternative formulas cannot be obtained. HCPs can provide caregivers a contact number for a local certified human breast milk donation center through the Human Milk Banking Association of North America.18 Human milk donors are thoroughly screened prior to donation.Purchasing human milk from the internet or social media sites should be avoided, as the milk is not adequately screened or regulated and could unintentionally expose the infant to infectious diseases, illicit drugs, and chemical contaminants.12

Cows milk is normally not recommended for children until they are 12 months or older due to nutritional differences, such as low levels of bioavailable iron and higher amounts of protein.20 If both human milk and infant formulas are unavailable, the American Academy of Pediatrics (AAP) recommends that infants older than 6 months consume cows milk for no more than 1 week.21 Iron supplementation can be given to infants under the supervision of a physician in the form of pediatric drops if they are younger than 6 months.8 Infants can be introduced to solid food at approximately 6 months of age, so it is important to introduce iron-rich foods or cereals to avoid iron deficiency.7,20 There are 2 sources of iron: heme and nonheme iron.7 Heme iron is available in red meat, seafood, and poultry. It is more easily absorbed by the body than nonheme iron. Nonheme iron is available in iron-fortified infant cereals, tofu, and beans. Moreover, goats milk is not approved for infants in the United States and plant-based milk is not recommended in children younger than 12 months.21 Soy milk, which is fortified with calcium and protein, may be used for less than 1 week if other avenues are exhausted. Lastly, toddler formula is not interchangeable with infant formula due to its differing nutritional value.3,6 Toddler formula is intended to be supplemented with an oral diet for toddlers. These formulas should only be used for children 12 months or older for a few days if there is no other choice.6,12,21

HCPs should be prepared to discourage conservation practices that could lead to unintentional infant harm. For example, in an effort to make infant formulas last longer, caregivers may dilute the product with more water. Infant formula should never be overdiluted, as it will offset the nutritional concentration and cause electrolyte disturbances.6,12,21 These complications can be fatal. In addition, homemade formulas should be discouraged, as they do not meet the nutritional or safety standards that commercial products have.21 Online recipes for homemade formulas may contain inadequate or excess amounts of vitamins and minerals and have been linked to severe, life-threatening complications.12 A case series published by the AAP described 2 patients who were fed with organic homemade infant formulas after transitioning from exclusive breastfeeding by the age of 1 month.22 The most notable laboratory abnormalities were related to inadequate vitamin D supplementation. Low levels of vitamin D resulted in inadequate calcium and phosphorus absorption and subsequent breakdown of bone. Further complications experienced by the patients included bone demineralization, cardiac arrest, hepatotoxicity, and ischemia of multiple organs. A summary of the dos and donts for HCP recommendations during an infant formula shortage can be found in the Table.4,9,12,16,17,21

Click table to enlarge.

Many homemade infant formula recipes can be found online and often contain ingredients that are harmful to infant growth and development. A 2020 study analyzed 149 homemade infant formula recipes distributed over 59 online blogs.23 A total of 24.3% of the recipes used whole unpasteurized cows milk, 23.6% used raw goats milk, and 14.5% used liver as the protein base for the homemade infant formula. Pasteurization is the process where raw milk is heated at a controlled temperature to reduce pathogens.24 Unpasteurized or raw milk is associated with outbreaks of foodborne illnesses such as Salmonella and Listeria infection. Liver contains high levels of vitamin A, which can lead to vomiting and bulging of the infants fontanelle.25 Additionally, only 84% of recipes included instructions for proper formula storage and 18.8% included shelf-life recommendations.23 Improper storage leads to an increased risk of bacteria proliferation and subsequent infection. Approximately 75% of the blogs did not encourage pediatrician consultations prior to the usage of a homemade formula.

Conclusion

HCPs are a vital source of information during the infant formula shortage and can provide reliable and safe resources to caregivers in need. It is imperative that HCPs discourage practices that can lead to unintentional infant harm. Education should be provided to all caregivers regarding proper feeding of infants and handling of infant formula.

Click here for more from the May issue of Contemporary Pediatrics.

References:

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HCPs can play an important role in addressing the US infant formula shortage - Contemporary Pediatrics

Pediatrics

The IT strategy behind a groundbreaking new $2B pediatric hospital – Healthcare IT News

Children's Healthcare of Atlanta Arthur M. Blank Hospital is scheduled to open this September. It was built from the ground up with the very latest healthcare information technology.

It is one of the largest and most advanced pediatric hospitals in the world. It cost more than $2 billion. It is 19 stories tall, with nearly two million square feet. It has 90 robots who have their own six elevators.

It features deep device integration, artificial intelligence, location awareness, video and patient engagement technology all focused on helping clinicians save steps and make better decisions while providing an excellent patient experience.

Jeremy T. Meller is chief information officer at Children's Healthcare of Atlanta Arthur M. Blank Hospital. We spoke with him to get the inside story of the creation of this facility's health IT strategy and the implementation of the very latest technologies.

Q. What was the overarching health IT strategy that went into creating the hospital from the ground up?

A. We are so proud of all the planning and work that has gone into Children's Healthcare of Atlanta Arthur M. Blank Hospital opening in September. The overall hospital visioning process began more than seven years ago. Children's worked with our Patient and Parent Advisory Council to understand what our families thought was most important for a new hospital.

We received so many valuable and creative responses that led to recommendations such as a second television in the room and washers and dryers on each floor. Our councils worked to understand the themes for what would make the most healing environment, with the best care possible for the kids we treat.

We also worked cross-functionally to learn about the leading practices in hospital design, understand pain points our staff were experiencing, and where we thought technology and innovation were headed in the future. But things move quickly. When we started the process in 2017, we assumed self-driving cars would need to be accommodated.

Meanwhile, we had no idea a global pandemic was on the horizon and we would be dealing with workforce issues. People were still confusing algorithmic bots with AI and nobody was talking about generative AI at all. We adapted our plans over the last few years to accommodate the new realities and needs we learned.

Our technology strategies started to form when we held massive-scale workflow simulations as part of our facility design process. Children's created "Cardboard City" by transforming a 100,000-square-foot warehouse into one of the largest, full-scale hospital mock-ups in the country. Using more than 12 miles of tape and 10,000 square feet of cardboard, our hospital planning and simulation teams built the mock-up to test drive the 3-D design plans and determine how the layouts might affect employee workflow, patient care and family experience.

Jeremy T. Meller, Children's Healthcare of Atlanta Arthur M. Blank Hospital

Through this process, we identified space requirements needed to be reworked in some areas, plug locations might be wrong, or we needed technology to help address an issue.

An example of this is the physical side of Arthur M. Blank Hospital. At nearly two million square feet, it rivals or exceeds many professional football stadiums in scale. This is in part because every patient room is spacious, with separate areas for parents. Parents will have a desk, a sofa bed, and their own television.

These amenities are sure to improve the quality of experience, but they also bring technology implications, such as increased network load. The physical size itself means moving around will simply take more time.

An extra 10,000 steps could impact the length of time it takes for a nurse to reach a patient room, so we were challenged to find ways that technology could help with communication, reduce steps (both physical and process steps), and improve the quality of care we were going to be providing.

Q. Were there special IT considerations because it is a pediatrics hospital?

A. Pediatric healthcare brings with it additional complexities in almost every way. Equipment must be sized for stages of anatomical childhood development. Care protocols are different and more complex.

Children respond differently to medical interventions, and most systems are not designed from the ground up to be oriented toward pediatric medicine. The electronic health record has more complexity, and providing access to patient records is more involved because a legal guardian relationship must be determined.

There are real-world reasons why pediatric hospitalizations are resource-intensive, but this doesn't mean finding experienced clinical staff and physicians is any easier. Our needs are more intensive, and we must do everything we possibly can to support our clinicians in providing the best care possible.

To help save physical steps and reduce time burden, we've integrated screens outside of each patient room with halo lights that change color depending on who is in the room. This is dependent on RTLS badges staff will wear once the hospital opens, and provides a visual clue that can reduce steps.

Locations are updated on status boards, and clinicians can review who is or was with the patient. This will help improve staff coordination, and in turn, patient care.

Q. You have 90 robots. Please talk about the place robotics holds in the hospital and what kinds of things they will be doing.

A. We are really excited about opening Arthur M. Blank Hospital with the world's largest fleet of robots. We will have two types of robots, autonomous mobile robots and Robos. About a third will be patient facing, helping to deliver meals or medications and pick up labs or other items that cannot go through the tube system.

Our back-hall "tugs" are designed with a platform to slide under specially designed carts that will haul heavy linen and trash. Arthur M. Blank Hospital has six elevators designed specifically for the robots. The system will include advanced algorithms and camera technology to determine location and bin utilization.

Logistical planning for the new hospital has been key for our teams as well. Our intelligent supply chain management system uses RFID technology to better automate stock and billing processes. Our pneumatic tube system has traffic-control monitoring, and our pressurized trash-chute system even has radiation monitoring.

Everything is connected. We have nearly 60 facility- and supply chain-related systems most of them are systems we have in other facilities, but at our new hospital they are networked, automated and monitored.

Q. You told me the hospital will feature deep device integration and artificial intelligence. Please elaborate.

A. Like many organizations, we have a long-term investment in predictive analytics but are more recently looking at how generative AI can be used in meaningful and safe ways. Everyone talks about how AI will change the world, change healthcare. But for AI to be useful, it needs to have content, it needs data. For predictive deterioration algorithms to be most effective, timely bio-physio data is needed.

Many hospitals have IT systems and medical equipment, but they aren't deeply integrated. A nurse will be standing at a computer, looking at a patient monitor (another computer) and keying in hourly vitals. This is an outmoded paradigm that needs to shift to real time.

We are integrating virtually every type of device that can provide this data, pulling it into our analytics systems, and currently are developing 11 predictive models to assist our clinical teams, helping them decide where to focus or make better, safer decisions.

Waveforms will be available to clinicians in real time on their mobile clinical smartphones, allowing them to better triage alarm response, such as seeing when a lead is disconnected. The nurse call system is integrated into our RTLS so alarms will auto-silence when a nurse enters the room.

Q. What kinds of patient engagement technologies do you have and what are the expected outcomes?

A. We know patients would probably rather be home than in the hospital, and that is why we've designed rooms that are all large and designed for the greatest comfort and connection for the whole family.

We will have multiple screens in each room. We will have the traditional patient education and entertainment screen, as well as a second TV for parents and a vertically mounted virtual whiteboard. The RTLS system will be integrated into the room, and when a clinician enters, a pop-up will appear on the television and whiteboard showing who just entered.

The whiteboard will provide information useful to the patient and family, including a daily schedule and a list of care team members. When the physician or nurse enters the room, the display will change, allowing the caregiver to pull up relevant information and radiology images to communicate with the patient.

A high-quality pan-tilt-zoom camera also is installed in every room and will allow caregivers, interpreters or even family members to dial into the room. It will turn away for privacy when not in use and all calls will be accepted or declined from the pillow-speaker.

Q. In the end, what are your goals as the CIO of this new high-tech hospital?

A. The new Arthur M. Blank Hospital will be one of the most advanced facilities in the world when it opens in September. My goal is we use technology to help clinicians provide the best care possible through more intelligent systems, save steps through improved communication and technology-supported visual processes, and improve care and experience through improved patient engagement.

Of course, we can't do anything in IT without an eye on cybersecurity, so that has been an integral part of everything we have done in advance of opening the hospital. All the new systems and capabilities have been carefully evaluated and implemented to provide the greatest safety and security possible.

Follow Bill's HIT coverage on LinkedIn: Bill Siwicki Email him:bsiwicki@himss.org Healthcare IT News is a HIMSS Media publication.

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The IT strategy behind a groundbreaking new $2B pediatric hospital - Healthcare IT News

Pediatrics

DB-OTO improved hearing to normal in child with profound genetic deafness – Contemporary Pediatrics

Positive, phase 1/2 preliminary data for Regeneron Pharmaceuticals' DB-OTO, an investigational gene therapy for profound genetic deafness, was presented at the 2024 American Society of Gene and Cell Therapy (ASGCT) annual conference.

The gene therapy improved hearing to normal levels in a child born with profound genetic deafness, who was dosed at 11 months of age, within 24 weeks. Additionally, initial hearing improvements were observed in another child, who was dosed at 4 years of age, at a 6-week assessment.

"Both [of the children] received their treatment in the United Kingdom," Lawrence R. Lustig, MD, chair, Department of Otolaryngology-Head & Neck Surgery, Columbia University, and clinical trial investigator, told Contemporary Pediatrics in the video interview above.

"What we saw early on [in the child dosed at 11 months], was a gradual improvement of their hearing thresholds, their auditory thresholds. The most recent data that we captured at about 24 weeks shows that the hearing in the ear that was dosed with the gene therapy, particularly in the speech frequencies, is at a normal to mild hearing loss range, which honestly is jaw-dropping," added Lustig. "I think these results surpassed even our wildest imaginations in this 1 particular child."

Watch the video at the top of this article for Lustig's full interview with Contemporary Pediatrics.

Data stems from the ongoing phase 1/2 CHORD trial, a first-in-human, multicenter, open-label trial to evaluate the safety, tolerability, and preliminary efficacy of DB-OTO in infants, children, and adolescents with otoferlin variants.

Each child received a single intracochlear injection of DB-OTO in 1 ear, with a surgical procedure that leverages the same approach used for cochlear implants. Pure tone audiometry (PTA) and auditory brainstem response (ABR) assessed the hearing improvements. According to Regeneron, PTA is considered to be the "gold standard" measurement of hearing, measured through behavioral confirmation of sound, such as turning the head toward a sound. ABR, as an objective confirmation of hearing function, corroborates behavioral responses by measuring electrical brainstem responses to sound emitted at different decibels.

Both participants had behavioral (PTA) or electrophysiological (ABR) responses at maximum sound levels of 100 decibels or greater at baseline. After treatment with DB-OTO, both children showed auditory responses at the first efficacy assessment of 4 weeks.

The first participant was 16 months of age at the 24-week assessment. Data presented at ASGCT showed the child had improvement of hearing to normal levels among key speech frequencies. The child had an average of 84 dB improvement from baseline, and 1 frequency measure reaching 10 dB in hearing level per PTA. Among all tested frequencies, an average 80 dB improvement from baseline was observed.

The second trial participant was 4 years of age at the 6-week assessment and experienced consistent results to the first participant at the same timepoint, stated Regeneron in a press release.

The second participant demonstrated initial improvement of hearing with responses to loud sounds observed across key speech frequencies, with an average of 19 dB improvement from baseline. One frequency measure reached 80 dB in hearing level per PTA. In this child, an average 16 dB improvement from baseline was observed.

"What we have right now, is the ability to measure hearing on a hearing test to determine what level they can detect sounds," said Lustig. "We don't really know what they're hearing. It's not going to be until the children are older that we see how their speech and language and speech comprehension come along and to listen to how they hear sounds. That's when we are really going to know, but that's not going to be for several years down the road."

Congenital deafness impacts approximately 1.7 out of every 1000 children born in the United States according to Regeneron, and approximately half of these cases have genetic causes. Otoferlin-related hearing loss is "ultra-rare," as the condition is "caused by variants in the otoferlin gene, which impairs the production of the OTOF protein that is critical for the communication between the sensory cells of the inner ear and the auditory nerve," stated Regeneron in the press release.

"I think this has profound impact on the treatment of children in general with hearing loss and how we approach them," said Lustig. "All children born with hearing loss really need to be genetically tested, because this particular form of deafness is relatively rare. Understanding which gene is involved in the deafness in children, if it can be identified, is going to be important."

Watch the video at the top of this article for Lustig's full interview with Contemporary Pediatrics.

Reference:

Latest DB-OTO results show dramatically improved hearing to normal levels in a child with profound genetic deafness within 24 weeks and initial hearing improvements in a second child at 6 weeks. Regeneron Pharmaceuticals. Press release. May 8, 2024. Accessed May 8, 2024. https://investor.regeneron.com/news-releases/news-release-details/latest-db-oto-results-show-dramatically-improved-hearing-normal

Link:

DB-OTO improved hearing to normal in child with profound genetic deafness - Contemporary Pediatrics

Pediatrics

Pediatric Telehealth Platform Market is expected to Expand at a Massive CAGR of 26.6% through 2030 – openPR

Pediatric Telehealth Platform Market

The global Pediatric Telehealth Platform market research is predicted to record a Massive CAGR of +26.6 % during the review period 2024-2031.

Get Sample Report + All Related Graphs & Charts: https://www.researchcognizance.com/sample-request/163062?utm_source=openpr.com

Market Overview: A Pediatric Telehealth Platform provides remote medical care for children, leveraging digital technologies. It offers real-time consultations with pediatricians through video calls or messaging. The platform facilitates timely diagnosis, treatment, and monitoring of various pediatric conditions. It enhances accessibility to healthcare, especially for families in remote or underserved areas. Additionally, it supports parental education and engagement in managing children's health. By promoting convenience and efficiency, it aims to improve pediatric healthcare outcomes while ensuring patient confidentiality and security.

Top Key Players in Global Pediatric Telehealth Platform Market, TytoCare, Alpha Medical, Blueberry Pediatrics, Anytime Pediatrics, NightLight Connect, Maven, Vsee, Anytime Telehealth, Amwell, Sesame, CallOnDoc, PM Pediatrics, Teladoc Health, K Health, Vivify Health, Southdale Pediatrics, KID-DOC Pediatrics, InSync Healthcare Solutions, CareXM,

The main goal for the dissemination of this information is to give a descriptive analysis of how the trends could potentially affect the upcoming future of Pediatric Telehealth Platform market during the forecast period. This markets competitive manufactures and the upcoming manufactures are studied with their detailed research. Revenue, production, price, market share of these players is mentioned with precise information.

In the geographic segmentation, the regions such as North America, Middle East & Africa, Asia Pacific, Europe and Latin America are given major importance. The top key driving forces of the Pediatric Telehealth Platform market in every particular market is mentioned with restraints and opportunities. The restraints are also given a counter act which prove to be an opportunity for this market during the forecast period of 2024 to 2030 respectively.

The Pediatric Telehealth Platform market is also explained to the clients as a holistic snapshot of a competitive landscape within the given competitive forecast period. A comparative analysis of regional players and segmentations, which helps readers get a better understanding of the areas and resources with better understanding.

Global Pediatric Telehealth Platform Market Segmentation:

Market Segmentation: By Type Cloud-based On-premises

Market Segmentation: By Application Respiratory Cold, Flu & Fever Gastroenterology Behavioural Health Dermatology Other

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An assessment of the market attractiveness with regard to the competition that new players and products are likely to present to older ones has been provided in the publication. The research report also mentions the innovations, new developments, marketing strategies, branding techniques, and products of the key participants present in the global Pediatric Telehealth Platform market. To present a clear vision of the market the competitive landscape has been thoroughly analysed utilizing the value chain analysis. The opportunities and threats present in the future for the key market players have also been emphasized in the publication.

Key questions answered in the report include: What are the main factors likely to encourage the growth of the global Pediatric Telehealth Platform Market? Which factors are expected to limit the development of the global Pediatric Telehealth Platform Market? Which application and product segments are anticipated to top in the forecast period? Which geographical segment is expected to lead and hold the main share of the global Pediatric Telehealth Platform Market in the next few years? What are the projected values and growth rate of the global Pediatric Telehealth Platform Market? Which are the key players operating in the global Pediatric Telehealth Platform Market?

Table of Contents Global Pediatric Telehealth Platform Market Research Report 2024 - 2030 Chapter 1 Pediatric Telehealth Platform Market Overview Chapter 2 Global Economic Impact on Industry Chapter 3 Global Market Competition by Manufacturers Chapter 4 Global Production, Revenue (Value) by Region Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions Chapter 6 Global Production, Revenue (Value), Price Trend by Type Chapter 7 Global Market Analysis by Application Chapter 8 Manufacturing Cost Analysis Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers Chapter 10 Marketing Strategy Analysis, Distributors/Traders Chapter 11 Market Effect Factors Analysis Chapter 12 Global Pediatric Telehealth Platform Market Forecast

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Pediatric Telehealth Platform Market is expected to Expand at a Massive CAGR of 26.6% through 2030 - openPR

Family Medicine

Weith, Wiewel and Wittels Wrap Up Medical School, Share a Common Destination – University of Missouri School of Medicine

The MU School of Medicine Class of 2024 will soon walk across the stage at graduation, a symbol of how far theyve come in their medical careers and education. But theyre only just beginning.

Most of these graduates will begin orientation for residency soon, though the exact date depends on the program. For Katelyn Weith, Brooke Wiewel and Andruw Wittels, they will officially start their residency July 1 at the Fulton Family Health Clinic. All three students are part of the family medicine integrated residency program and happen to be great friends.

I've known Andruw and Brooke all through medical school, and they both have similar backgrounds to me from smaller towns, Weith said. We've gotten pretty close -- I'm really excited to be working with them in Fulton.

Learn more about the trio.

Katelyn Weiths been around health care for as long as she can remember. Her mom works in medical imaging and her aunt is a nurse, but not in Weiths hometown of Wardsville, Missouri. Both currently commute about 20 minutes to Jefferson City.

Its not a lengthy drive, but Weiths met people who havent seen a specialist in years because of the distance. Her grandfather, for example, had to travel more than two hours to see a cardiologist for heart problems. Her experiences from growing up and working in rural clinics affirmed her choice to go into rural medicine.

Those primary care offices end up being a one-stop shop for anything a patient might need, and those doctors are really well-rounded physicians, Weith said.

Part of what drew Weith to family medicine was the ability to develop a wide base of knowledge and skills, across multiple medical fields, to provide preventative care. Its whats needed to serve patients in rural communities, and to be that one-stop shop Weith wants.

Another reason was the friendships she saw curated between a patient and physician, and hopes shell be able to do her part in connecting rural areas to long-term, primary care.

I want to be with patients throughout their health care journey, Weith said. I didn't want to see just a small snapshot of what's going on with them. I want to work alongside them and walk them through the health care process.

Similar to Weith, Brooke Wiewel was also exposed to healthcare at a young age, though not in the same way. When she was 12, her older sister had a traumatic accident and needed lifesaving surgery.

After that, it just really opened my eyes to how incredible the human body is, with how she was still able to survive and function after all of the organ damage, and how the surgeons were able to save her, Wiewel said.

Watching her sister survive and later make a full recovery sparked her interest in becoming a doctor. Wiewel was initially drawn to emergency medicine, and even worked as an EMT during college and at the former MU Women and Childrens Hospital Emergency Room. It was her experiences in the ER, though, that ultimately led her to family medicine.

You often see a lot of patients with chronic medical conditions and people who come into the emergency room with things that could have been addressed or prevented if they had adequate preventative and primary care, Wiewel said.

Doing rural family medicine would let her provide that preventative care. Plus, the location meant she could do obstetrics, see pediatric patients and even do emergency medicine, when cases presented themselves fields she still wants to pursue. She looks forward to learning more in her coming residency.

I just am really grateful and excited to be continuing residency here at Mizzou, because Mizzou Family Medicine is what ultimately solidified my passion for family medicine and my passion for rural health care, Wiewel said. I can't think of a better place to be able to continue along with some of my classmates, who I really admire.

Unlike his two friends, Andruw Wittels never dreamed of being a doctor. If it wasnt for his high school anatomy class, which required shadowing a physician, his life would look very different.

I had no idea that somebody from where I grew up or how I grew up could go into medicine, Wittels said. Where I went to high school, the biggest thing that they pushed was trade school, as well as just entering the workforce in general. There was really no mention at all about pursuing medicine. And I don't think, if it wasnt for that class, I would've never thought about doing medicine in the first place.

Wittels grew up near Osage Beach and knows what it was like to rely on one person for your medical needs. As he learned more about medically underserved areas and the physician shortage, he couldnt just sit idle while knowing he could help.

Its one reason Wittels went into family medicine, but he also enjoys providing intergenerational care and challenging himself with the ever-changing nature of a rural clinic.

Anything can walk through the door in a family medicine clinic, Wittels said. You could have a toenail removal, then an IUD insertion and then you could talk to somebody about their diabetes.

As Wittels and his friends prepare for the next phase of their medical journey, one word describes them all theyre ready.

This feels like its something that I was meant to do, Wittels said.

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Weith, Wiewel and Wittels Wrap Up Medical School, Share a Common Destination - University of Missouri School of Medicine

Family Medicine

Beebe’s Residency Program One of 25 in the U.S. Selected for Pilot Project Led by the Society for Teachers of Family … – Beebe Healthcare

Beebe Healthcares R. Randall Rollins Center for Medical Education is proud to announce that its Family Medicine Residency program is one of just 25 across the country that has been selected to participate in a special pilot project facilitated by the Society for Teachers of Family Medicine (STFM).

The project is designed to elevate the importance of competency-based medical education while integrating a more individualized and personalized approach to learning for medical residents.

There is so much benefit in developing individualized learning plans for our residents, with consideration for each persons special needs, strengths, and goals, said Joyce Robert, MD, FAAFP, Program Director. We are incredibly excited to be part of this project and to have the opportunity to lead the way and set an example for other residency programs to follow.

In addition to implementing new hands-on approaches in the clinical training environment, the STFM program will put new technologies in the hands of residency program leaders, who will be spending more time on direct observation as they are working with clinical residents. The goal is to allow for real-time assessments in medical training environments. Additionally, the program promotes the importance of communication, teamwork, and leadership from a professional development standpoint, and work/life balance and wellness on a personal level.

This project encourages us to look at what we are doing through a different lens and take a more holistic approach as we grow our people, and therefore our program, said Dr. Robert. Its a chance to be at the forefront of positive change and we are excited to be part of it.

Beebes Family Medicine Residency program welcomed it inaugural cohort of residents in summer 2023, and recently announced the second group of residents, who will come onboard this July. The program is led by Dr. Robert and Miri Shlomi, MD, Associate Program Director, both of whom will participate in a series of conferences sponsored by STFM, as Beebe adopts and implements its competency based medical education curriculum. They and their colleagues will have opportunities in the future to share their experiences and learned best practices through their activism in the Delaware Academy of Family Physicians (DAFP).

Recently, Dr. Robert was inducted as DAFPs President-Elect and Ryan Arias, DO, primary care physician and faculty member within Beebes Family Medicine Residency program, was named DAFP Teacher of the Year.

We have come a long way in a short period of time, said Dr. Robert. So many wonderful things are happening because our team is made up of a group of people who are passionate about the work, dedicated to doing things the right way, and committed to growing in a way that positions Beebe as a premier place to come for family medicine residency training.

Caption: From left to right, Wendi Schirvar, PhD, Ryan Arias, DO, Miri Shlomi, MD, Joyce Robert, MD, Tanya Ray, MS, Jeffrey Hawtof, MD, and Cynthia Lamour, DO.

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Beebe's Residency Program One of 25 in the U.S. Selected for Pilot Project Led by the Society for Teachers of Family ... - Beebe Healthcare

Family Medicine

UVA Health expands footprint in Northern Virginia | News – Prince William Times

UVA Health will acquire Piedmont Family Practice, a private family medicine clinic in Warrenton, this summer.

Piedmont Family Practice is the largest primary care practice in Warrenton and has served the area since 1993. In addition to general family medicine, it offers women's health, in-office surgeries and includes Piedmont Urgent Care and the Bariatric & Metabolic Weight Loss Center.

It employs about 90 staff members, including seven physicians, 17 nurse practitioners or physician assistants, a certified diabetic educator and a registered dietitian.

The acquisition will expand UVA Health's primary care provider footprint 61% in the area, according to a news release.

Dr. K. Craig Kent, CEO of UVA Health and executive vice president for health affairs at the University of Virginia, said the agreement helps fulfill key goals for the health system.

The acquisition of Piedmont Family Practice helps UVA Health address many components of our 10-year strategic plan, including the expansion of our statewide care network and access to primary care, by providing a geographic connection to our primary care network, which will now extend from Culpeper through Warrenton and throughout Northern Virginia, Kent said in a news release. Piedmont Family Practice is a group of outstanding physicians and allied health providers, and we are so thrilled they are joining our UVA Health family.

UVA Health officials say they plan to continue employing all the current Piedmont Family Practice team members and "support the practices growth ambitions over time."

The academic health system already boasts a surgical care center, cardiology and obstetrics and gynecology services in Warrenton.

The closest UVA Health family medicine or primary care offices are currently in Gainesville and Haymarket. UVA Community Health also offers services in Culpeper.

The Piedmont Family Practice team is a well-known, high-quality group of care providers with a longstanding commitment to serving the community, Erik Shannon, chief executive officer of UVA Community Health, said in a news release Tuesday. This partnership provides both Piedmont Family Practice and UVA Health an opportunity to benefit from each organizations best practices as we strive for excellence in our primary care offering.

Dr. Steven W. von Elten, a founding member and physician partner inPiedmont Family Practice, praised the merger.

Personalized, quality health care is a key shared value of Piedmont Family Practice and UVA Health, von Elten said in a news release. Joining forces with UVA Health will enable us to enhance the care we provide by providing a valuable investment in the latest technology as well as making it easier for our patients to access subspecialty care.

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UVA Health expands footprint in Northern Virginia | News - Prince William Times