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A pooled safety and efficacy analysis of data from 3 clinical studies demonstrated that evolocumab was well tolerated, with no new safety concerns identified for pediatric patients with homozygous familial hypercholesterolemia (HoFH). View the complete article below ⤵️ #HomozygousFamilialHypercholesterolemia #HoFH #Pediatrics

📃Scientific paper: IPNA clinical practice recommendations for the diagnosis and management of children with steroid-sensitive nephrotic syndrome Abstract: Idiopathic nephrotic syndrome is the most frequent pediatric glomerular disease, affecting from 1.15 to 16.9 per 100,000 children per year globally. It is characterized by massive proteinuria, hypoalbuminemia, and/or concomitant edema. Approximately 85–90% of patients attain complete remission of proteinuria within 4–6 weeks of treatment with glucocorticoids, and therefore, have steroid-sensitive nephrotic syndrome (SSNS). Among those patients who are steroid sensitive, 70–80% will have at least one relapse during follow-up, and up to 50% of these patients will experience frequent relapses or become dependent on glucocorticoids to maintain remission. The dose and duration of steroid treatment to prolong time between relapses remains a subject of much debate, and patients continue to experience a high prevalence of steroid-related morbidity. Various steroid-sparing immunosuppressive drugs have been used in clinical practice; however, there is marked practice variation in the selection of these drugs and timing of their introduction during the course of the disease. Therefore, international evidence-based clinical practice recommendations (CPRs) are needed to guide clinical practice and reduce practice variation. The International Pediatric Nephrology Association (IPNA) convened a team of experts including pediatric nephrologists, an adult nephrologist, and a patient representative to develop comprehensive CPRs on the diagnosis and management of SSNS in children. Afte... Continued on ES/IODE ➡️ https://etcse.fr/Piz ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: IPNA clinical practice recommendations for the diagnosis and management of children with steroid-sensitive nephrotic syndrome Abstract: Idiopathic nephrotic syndrome is the most frequent pediatric glomerular disease, affecting from 1.15 to 16.9 per 100,000 children per year globally. It is characterized by massive proteinuria, hypoalbuminemia, and/or concomitant edema. Approximately 85–90% of patients attain complete remission of proteinuria within 4–6 weeks of treatment with glucocorticoids, and therefore, have steroid-sensitive nephrotic syndrome (SSNS). Among those patients who are steroid sensitive, 70–80% will have at least one relapse during follow-up, and up to 50% of these patients will experience frequent relapses or become dependent on glucocorticoids to maintain remission. The dose and duration of steroid treatment to prolong time between relapses remains a subject of much debate, and patients continue to experience a high prevalence of steroid-related morbidity. Various steroid-sparing immunosuppressive drugs have been used in clinical practice; however, there is marked practice variation in the selection of these drugs and timing of their introduction during the course of the disease. Therefore, international evidence-based clinical practice recommendations (CPRs) are needed to guide clinical practice and reduce practice variation. The International Pediatric Nephrology Association (IPNA) convened a team of experts including pediatric nephrologists, an adult nephrologist, and a patient representative to develop comprehensive CPRs on the diagnosis and management of SSNS in children. Afte... Continued on ES/IODE ➡️ https://etcse.fr/Piz ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

A recent study presented at NASPGHAN 2024 reveals that pediatric and adolescent patients who contracted COVID-19 were twice as likely to develop irritable bowel syndrome compared to those who did not. Analyzing data from over 115,000 patients in the Military Health System, researchers found that COVID-19 exposure significantly increased the risk of new-onset IBS, even after accounting for pre-existing conditions and other factors. The study highlights the long-term effects of COVID-19, underscoring the need for early diagnosis and intervention in pediatric patients. As the healthcare community continues to study the delayed impacts of COVID-19, this finding reinforces the importance of monitoring gastrointestinal health in children post-infection. Early intervention can improve quality of life for affected patients. #IBS #IrritableBowelSyndrome #PediatricCare #Gastroenterology #COVID19

📃Scientific paper: Effects of aminophylline therapy on urine output and kidney function in children with acute kidney injury Abstract: Background Acute kidney injury (AKI) is a frequent complication of children admitted to the paediatric intensive care unit. One key management modality of AKI is the use of diuretics to reduce fluid overload. Aminophylline, a drug that is well known for its use in the treatment of bronchial asthma, is also purported to have diuretic effects on the kidneys. This retrospective cohort study assesses the effect of aminophylline in critically ill children with AKI. Methods A retrospective chart review of children admitted to the paediatric intensive care unit of the Red Cross War Memorial Children’s Hospital (RCWMCH) with AKI who received aminophylline (from 2012 to June 2018) was carried out. Data captured and analyzed included demographics, underlying disease conditions, medications, urine output, fluid balance, and kidney function. Results Data from thirty-four children were analyzed. Urine output increased from a median of 0.4 mls/kg/hr [IQR: 0.1, 1.1] at six hours prior to aminophylline administration to 0.6 mls/kg/hr [IQR: 0.2, 1.9] at six hours and 1.6 mls/kg/hr [IQR:0.2, 4.2] at twenty-four hours post aminophylline therapy. The median urine output significantly varied across the age groups over the 24-h time period post-aminophylline, with the most response in the neonates. There was no significant change in serum creatinine levels six hours post-aminophylline administration [109(IQR: 77, 227)—125.5(IQR: 82, 200) micromole/l] P-value = 0.135. However, there wer... Continued on ES/IODE ➡️ https://etcse.fr/eKQga ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

In a quest to better understand the side effects, particularly emergence phenomena, of using intravenous ketamine with or without midazolam for sedation in pediatric emergency departments, a comprehensive study was undertaken (https://lnkd.in/dy_jJuYd), including patients aged between 4.5 months and 16 years who required ketamine sedation. Each patient received a dose of ketamine (1 mg/kg) and glycopyrrolate (5 μg/kg) intravenously. They were then randomly assigned to receive either midazolam (0.1 mg/kg) or no additional medication. The study meticulously tracked the total duration of sedation, its efficacy, and any adverse effects. Researchers particularly focused on comparing the side effects between those who received only ketamine and those who received both ketamine and midazolam. Additionally, they looked at variations in side effects based on the patients' ages and the number of ketamine doses administered. The study included 266 patients in total, with 129 receiving only ketamine and 137 receiving both ketamine and midazolam. The results showed that the duration and effectiveness of sedation were similar across both groups. However, adverse effects did vary. Common side effects included respiratory events (4.5%), vomiting (18.7%), and emergence phenomena (26.7% in the ED and 22.4% at home). Significant emergence phenomena, such as nightmares, hallucinations, and severe agitation, were relatively low, occurring in 7.1% of the ketamine group and 6.2% of the ketamine-midazolam group. Interestingly, while the addition of midazolam did not significantly change the rate of emergence phenomena, it did lead to a higher incidence of oxygen desaturation events (1.6% with ketamine alone versus 7.3% with ketamine-midazolam) but a lower incidence of vomiting (19.4% with ketamine alone versus 9.6% with ketamine-midazolam). These side effects were more pronounced in children younger than 10 years. For instance, younger children in the ketamine-midazolam group experienced 6.3% more episodes of oxygen desaturation, whereas those in the ketamine-only group experienced 12.1% more vomiting episodes. For children aged 10 years and older, the addition of midazolam was associated with increased agitation in the pediatric ED (5.7% with ketamine alone versus 35.7% with ketamine-midazolam). Despite these findings, overall satisfaction among parents and physicians remained high for all patients receiving intravenous ketamine sedation. The study concluded that while ketamine alone and in combination with midazolam provided equally effective sedation, the addition of midazolam did not reduce the incidence of emergence phenomena and introduced additional risks such as oxygen desaturation. #PediatricCare #KetamineSedation #EmergencyMedicine #ClinicalResearch #HealthcareInnovation

❓What can pediatricians learn from adult inherited metabolic diseases specialists ❓ Thanks to better diagnosis and pediatric care over the last 30 years, patients are living longer. 📈Adult metabolic medicine (AMM) is an expanding medical subspecialty, due to the increasing number of adult patients with inherited metabolic diseases (IMD) Now about 50% of patients with IMD are adults, and adult #metabolic #medicine (AMM) is getting more structured and AMM is an expanding medical specialty And there are several domains in which pediatricians can learn from AMM. ✅ long-term evolution of IMD patients ✅attenuated phenotypes in adults calling for caution in the development of newborn screening programs ✅adult-onset IMD are expanding our understanding of metabolism, including in children 🤝These observations underline the need of strong interdisciplinarity between #pediatricians and #adult specialists Learn more from Journal of Inherited Metabolic Disease report https://lnkd.in/gijkcyC9 Or the podcast of Dr Fanny Mochel AP-HP, Assistance Publique - Hôpitaux de Paris https://lnkd.in/gq8Q5x5a #raredisease #imd #nbs #healthcare Mirjam Langeveld THE SOCIETY FOR THE STUDY OF INBORN ERRORS OF METABOLISM Carla Hollak David Cassiman Wouter Meersseman

📃Scientific paper: Effects of aminophylline therapy on urine output and kidney function in children with acute kidney injury Abstract: Background Acute kidney injury (AKI) is a frequent complication of children admitted to the paediatric intensive care unit. One key management modality of AKI is the use of diuretics to reduce fluid overload. Aminophylline, a drug that is well known for its use in the treatment of bronchial asthma, is also purported to have diuretic effects on the kidneys. This retrospective cohort study assesses the effect of aminophylline in critically ill children with AKI. Methods A retrospective chart review of children admitted to the paediatric intensive care unit of the Red Cross War Memorial Children’s Hospital (RCWMCH) with AKI who received aminophylline (from 2012 to June 2018) was carried out. Data captured and analyzed included demographics, underlying disease conditions, medications, urine output, fluid balance, and kidney function. Results Data from thirty-four children were analyzed. Urine output increased from a median of 0.4 mls/kg/hr [IQR: 0.1, 1.1] at six hours prior to aminophylline administration to 0.6 mls/kg/hr [IQR: 0.2, 1.9] at six hours and 1.6 mls/kg/hr [IQR:0.2, 4.2] at twenty-four hours post aminophylline therapy. The median urine output significantly varied across the age groups over the 24-h time period post-aminophylline, with the most response in the neonates. There was no significant change in serum creatinine levels six hours post-aminophylline administration [109(IQR: 77, 227)—125.5(IQR: 82, 200) micromole/l] P-value = 0.135. However, there wer... Continued on ES/IODE ➡️ https://etcse.fr/eKQga ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

In a promising leap forward for #pediatric sickle cell disease (#SCD) treatment, the oral medication Oxbryta (#voxelotor) has been shown to reduce the #sickling and disintegration of #redbloodcells in #children, which could enhance oxygen delivery throughout the body, according to recent findings from the HOPE-KIDS 1 Phase 2a #clinicalstudy. Pfizer's Oxbryta is approved in the #US to treat SCD in adults and children as young as 4. and for patients 12 and older in the #EuropeanUnion, the #UK, and some countries in the #MiddleEast. https://lnkd.in/dnyp6Xt5... #SickleCellDisease #SickleCellAnemia #Oxbryta #InnovativeMedicines #ClinicalResearch #ClinicalNews #Pediatrics

#Fungemia in pediatric intensive care units (#PICU) presents a significant #mortality risk, with rates ranging from 20% to 50%. Key risk factors include prolonged use of broad-spectrum #antibiotics, central venous catheters, #immunosuppression, and underlying chronic conditions. Early identification and management are crucial for improving outcomes. The #Candidascore is an essential tool for predicting the risk of invasive #candidiasis. It considers factors like multifocal #colonization, surgery, total parenteral #nutrition, and severe #sepsis. A score above 2.5 indicates a high risk, guiding timely #antifungal #therapy. Addressing #fungemia requires a multifaceted approach, including stringent #infectioncontrol practices, judicious use of #antibiotics, and routine risk assessment using the #Candidascore. Collaboration across #Healthcare, #Pediatrics, #InfectionControl, and #CriticalCare teams is vital to reducing mortality rates in PICU settings. #PediatricICU #Fungemia #AntifungalTherapy #CandidaScore #PatientSafety #Sepsis #Immunocompromised #MedicalResearch #HospitalManagement #HealthcareInnovation