Weekly review: Neffy pre-ordering launches, trofinetide data, and more

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Get caught up with our journal! Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

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Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from last week (Monday, September 2, to Friday, September 6, 2024), and click on each link to read and watch anything you may have missed.

ARS launches pre-ordering for neffy to treat type 1 allergic reactions

Pre-ordering for epinephrine nasal spray (neffy; ARS Pharmaceuticals) to treat allergic reactions, including anaphylaxis, in adults and pediatric patients weighing at least 66 pounds (30 kg) has launched. Patients can now pre-order through neffy.com and obtain the medication via a healthcare provider or virtual consultation. According to ARS Pharmaceuticals, the spray can be delivered within two days of availability in the U.S.

Richard Lowenthal, Co-Founder and CEO of ARS Pharmaceuticals, emphasized that this needle-free option addresses long-awaited demand. Through neffy.com, ARS offers assistance with insurance prior authorizations, potentially lowering co-pays to $25 for two devices.

The neffyConnect service and BlinkRx online pharmacy are accepting prescriptions, with BlinkRx offering free shipping. Sung Poblete, CEO of Food Allergy Research and Education, noted the importance of timely epinephrine administration and reducing injection hesitancy for effective anaphylaxis management.
Click here for full details.

Discussing trofinetide data, FDA approval for Rett syndrome with Alan Percy, MD

Alan Percy, MD, discussed key advancements in treating Rett syndrome, focusing on the FDA approval of trofinetide (Daybue; Acadia Pharmaceuticals) on March 13, 2023. This approval marked the first drug directly targeting Rett syndrome symptoms, unlike previous medications used only for symptomatic care.

Percy highlighted the LAVENDER trial, a 12-week, double-blind, placebo-controlled study that demonstrated significant improvements in the Rett Syndrome Behavioral Questionnaire (RSBQ) and Clinical Global Impression of Improvement (CGI-I). Two long-term open-label extension studies, LILAC-1 and LILAC-2, confirmed the safety and efficacy of trofinetide in patients aged 5 to 22 years.

While trofinetide has shown promise, Percy acknowledged diarrhea and vomiting as common side effects, with some patients needing to discontinue treatment. He noted, however, that gradual dose adjustments have improved tolerability over time. Despite challenges, this approval represents a milestone in Rett syndrome management.

Click here for the full video interview.

HIV Roundtable: Adolescents and Issues Around Care, Adherence, and Privacy

This is the third and final episode of our series on HIV care and management. This is a continuing collaboration working with our partners Contemporary Pediatrics and Contemporary OB/GYN.

This series discusses several aspects of HIV care including clinical management, therapies, vaccines, multidrug resistance, PrEP, and patient management in adolescents.

In this episode, the panel provides clinical feedback on the challenges for teens and young adults regarding staying in the continuum of care, adherence for PrEP, and potential adverse effects associated with it.

Click here to watch HIV Roundtable.

Study: Not all screen time before bed is associated with impacted sleep

A study published in JAMA Pediatrics found that not all screen time before bed negatively impacts sleep in children aged 11 to 15 years. The study used an objective, night-by-night approach, tracking screen time and sleep quality in 79 participants using video cameras and wrist-worn accelerometers.

The results showed that screen use in the two hours before bed had no significant impact on sleep duration or quality. However, using screens in bed, particularly interactive activities like gaming, was associated with delayed sleep onset and reduced total sleep time. Each additional 10 minutes of interactive screen time in bed resulted in a 10-minute delay in sleep onset and a decrease in total sleep time.

The study suggests that presleep recommendations should be adjusted, as not all screen time before bed impairs sleep. The findings align with concerns from the American Academy of Pediatrics about the impact of intense screen activities on sleep.

Click here for full study details.

Prenatal fish intake associated with reduced odds of autism diagnosis

A recent study published in The American Journal of Clinical Nutrition found that fish intake during pregnancy was linked to a reduced likelihood of autism diagnosis and autism-related traits in children. However, omega-3 supplementation did not show the same association. Fish is a primary source of omega-3 polyunsaturated fatty acids, which are important for brain development, though fish consumption remains generally low.

The study was part of the Child Health Outcomes (ECHO) Program, which analyzed data from 69 cohorts across the U.S. It included 3,939 participants for fish intake analysis and 4,537 for omega-3 supplement use. Results showed that pregnant women who consumed fish had lower odds of having a child diagnosed with autism, while omega-3 supplement use had no significant impact.

The findings support existing dietary guidelines that encourage fish consumption during pregnancy for its benefits, including reduced risks of autism-related outcomes. Researchers emphasized that fish with low toxicant levels should be prioritized.

Click here for more.

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