QT Medical, Inc.’s Post

Congenital long QT syndrome (LQTS), occurring in 1 in 2,000 live births, is a genetic disorder characterized by a prolonged QT interval on the ECG. It is mostly inherited as an autosomal dominant trait and can manifest with syncope, ventricular arrhythmias, and sudden death. Studies have found that 10-15% of infants dying of sudden infant death syndrome (SIDS) carry known pathogenic mutations for LQTS. It is estimated that 300-400 infants may die of undiagnosed LQTS in the first few months of life in the U.S. each year and be labeled as SIDS deaths.   If the diagnosis of LQTS is made early, treatment by beta-blockers and/or placement of an implantable cardioverter defibrillator (ICD) is effective in preventing sudden death. The efficacy and cost-effectiveness of ECG screening of infants for LQTS have been demonstrated by studied in Italy. In the US, there have been debates for universal LQTS screening by ECG among pediatric cardiology professionals. While the pros and cons of ECG screening of all infants for LQTS for sudden death or SIDS prevention were well presented, gaps in technology and device designed specifically for this purpose were apparently the missing pieces of this debate.    With the initial funding from the National Institutes of Health (NIH), I led a team of engineers and clinical investigators to develop a 12-lead ECG system that parents can perform the test on their babies at home, and the results can be interpreted by cardiology experts immediately. QT Medical was founded to develop the product (PCA 500) and make it widely available and accessible. In a randomized controlled clinical trial of 2,582 infants, we found that over 94% of parents were able to use PCA 500 and complete a medical standard 12-lead ECG by themselves with no training.     Since its FDA clearance for use in adults in 2018, PCA 500 has been used by airlines, schools and in clinical trials. There are numerous stories about how PCA 500 has impacted or saved someone’s life, such as a passenger with chest pain on a long-haul flight was found to have a heart attack, or a student athlete had his/her heart condition diagnosed and treated in time because of ECG screening with PCA 500. While all of us at QT Medical are very proud of the work we do, nothing gets us more excited than coming back to our original mission to enable at-home screening for LQTS in infants and prevent sudden infant death. With the FDA clearance of PCA 500 for pediatric use, we can now focus on why we started the journey 10 years ago—providing ECG screening of infants for LQTS by parents at home. We are now launching the Pediatric Xpress ECG service in the US— a mail delivery at home 12-lead ECG test for infants.    More information at xpressecg.com

Roche announced that the primary analysis of the Phase III HAVEN 7 study reinforced the efficacy and safety of Hemlibra® (emicizumab) in previously untreated or minimally treated infants with severe haemophilia A without factor VIII inhibitors. Results showed that Hemlibra achieved meaningful bleed control in babies up to 12 months of age, and was well tolerated. The new data were presented at the 65th American Society of Hematology (#ASH23) Annual Meeting and Exposition taking place 9-12 December 2023, in San Diego, California, and included in the press programme. “Haemophilia A can have a devastating impact on any patient, but this is especially true for infants, where the emotional and physical stress due to frequent hospital visits, treatment administration and other worries can be distressing for babies and their parents and caregivers,” said Steven Pipe, MD, professor of paediatrics and pathology at the University of Michigan. “These results reinforce the benefit of starting prophylaxis as soon as possible after birth, as well as for the use of subcutaneous treatments, which are especially valuable in young babies where access to veins can be very difficult.” The burden of severe haemophilia A in babies and on their parents and caregivers is significant. The World Federation of Haemophilia treatment guidelines consider the standard of care in haemophilia to be regular prophylaxis initiated at a young age, as studies have shown this improves long-term outcomes, while reducing the risk of intracranial haemorrhage. However, for many babies with haemophilia A, prophylaxis is not started until after the first year of life because of the high treatment burden. Hemlibra, which is already approved and being used to treat babies with haemophilia A, provides a flexible treatment option that can be administered subcutaneously from birth at different dosing frequencies. “The results of HAVEN 7 provide additional confidence in the efficacy and safety profile of Hemlibra for babies with severe haemophilia A, and add to its extensive clinical and real-world evidence across all ages,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Conducted in collaboration with the haemophilia A community, this trial reflects our ongoing commitment to listen and respond to the needs of those impacted by this condition, in hopes of advancing treatment standards even further.” The HAVEN 7 study is a Phase III, descriptive, single-arm study, set up in collaboration with the haemophilia A community to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of subcutaneous Hemlibra in infants with severe haemophilia A without factor VIII inhibitors.  #hematology #haemophiliaA #clinicaltrials #emicizumab #Hemlibra #Roche #Genentech  👉 New data reinforce the benefit of early preventative treatment with Roche’s Hemlibra for babies with severe haemophilia A

Gray Baby Syndrome (Part 3) Gray Baby Syndrome Treatment Management of chloramphenicol toxicity centers primarily around emergency measures and intensive supportive therapy in severe cases with cardiorespiratory impairment. The general approach to the ashen-gray hemodynamically unstable neonate starts with aggressive resuscitation and an early call to the pediatric intensive care unit or extracorporeal life support team, as some of these patients may be ideal candidates. These patients should be hemodynamically stabilized, appropriately oxygenated and ventilated, and intubated early. Checking a core temperature is critical as hypothermia is common in the gray neonate. Aggressive rewarming should be considered. A point-of-care glucose should also be checked, and hypoglycemia should be reversed if present. The differential diagnosis for an ashen-gray, cyanotic neonate should include chloramphenicol toxicity, congenital heart disease, adrenal insufficiency/hypothyroidism, inborn errors of metabolism, trauma, seizures, and of course, sepsis. Empiric administration of broad-spectrum antibiotics such as vancomycin, ampicillin (targeting Listeria), and a third-generation cephalosporin such as ceftriaxone or cefotaxime is recommended. Additional consideration should also be given to empiric prostaglandin administration in gray/cyanotic neonates, especially if a duct-dependent congenital cardiac lesion is present. Modalities that have been used for the treatment of gray-baby syndrome are primarily aimed towards direct removal of the parent chloramphenicol molecule. This has been achieved through charcoal hemoperfusion and exchange transfusion. There have also been reports of phenobarbital being used for induction of the UDP-glucuronyltransferase enzyme. Consideration for cardiopulmonary bypass including extracorporeal membrane oxygenation may also be considered . Exchange transfusion This lifesaving procedure involves removing some of your baby’s blood and replacing the blood with freshly donated blood or plasma. The procedure is completed using a catheter. Hemodialysis This procedure uses a dialysis machine to cleanse toxins from your baby’s bloodstream. It also balances potassium and sodium levels and helps control your baby’s blood pressure. In addition to the above treatments, your baby may be given oxygen therapy to improve breathing and oxygen delivery to the body. Your baby’s doctor may also recommend hemoperfusion. This treatment is similar to dialysis and helps remove toxins from the blood. Your baby’s blood will be monitored during treatment. References:

Children are more likely to experience organ rejection after transplant than adults, so monitoring their new organ’s function is key to estimate if there is rejection and if the treatment for rejection is working. If the organ function drops, a biopsy is done to confirm if there’s rejection. New evidence highlights the importance of follow-up biopsies to detect persistent rejection in pediatric kidney transplant. Dr. Tom Blydt-Hansen, investigator at BC Children's Hospital Research Institute (BCCHR) and associate professor in the department of pediatrics at UBC, is involved in the study. The new evidence is the result of the data analysis from two pediatric centres — BC Children’s Hospital and University of Alabama at Birmingham (UAB) in the US — where follow-up biopsies were done routinely on 58 children after treatment for acute kidney rejection. Read the full story at https://bcchr.info/4atvJAS BC Children's Hospital Foundation BC Children's Hospital, Vancouver Faculty of Medicine at the University of British Columbia UAB Medicine #kidney #transplant #pediatrics #research

Pediatric Oncologist

❗ New Approach May Prevent Deadly Intestinal Disease in Preemies Excerpt: Scientists from Stanley Manne Children's Research Institute’s at Ann & Robert H. Lurie Children's Hospital of Chicago and colleagues found that an investigational protein replacement – #recombinant #human #insulin-like growth factor 1 and its binding protein-3 (rhIGF-1/BP3) – protected neonatal mice from #necrotizing #enterocolitis (NEC), a deadly intestinal disease that often strikes extremely premature infants. “Our preclinical evidence is encouraging and paves the way to a clinical trial of rhIGF-1/BP3 for prevention of NEC,” said senior author Isabelle De Plaen, MD, a scientist at Manne Research Institute, neonatologist at Lurie Children’s, and Professor of Pediatrics at Northwestern University Feinberg School of Medicine. “The rhIGF-1/BP3 is already in Phase 2 clinical trials for preventing bronchopulmonary dysplasia (BPD) and retinopathy of prematurity (ROP), both of which are severe complications affecting the lungs and eyes in extremely premature babies. Our results could help add NEC to these clinical trials.” Previous studies have shown that low levels of the naturally occurring insulin-like growth factor 1 (IGF-1) are associated with increased risk of BPD, ROP and NEC in preemies. In Dr. De Plaen’s study, supplementing with rhIGF-1/BP3 protected intestinal microvasculature development and decreased inflammation, which might be how NEC was prevented. Her group’s previous research has shown that defective intestinal microvascular development significantly contributes to NEC. “Currently we don’t have curative therapy for NEC, so prevention is a highly promising approach,” said Dr. De Plaen. “Our goal is to spare premature infants from this devastating disease and other complications of prematurity.” Results were published in the journal Pediatric Research: Daniel York et al, Nailfold Capillaroscopy: A Promising, Noninvasive Approach to Predict Retinopathy of Prematurity, The Journal of Pediatrics (2023). DOI: 10.1016/j.jpeds.2023.113478 https://lnkd.in/eHGiPYcn ➡️ https://lnkd.in/epaTv5hm #growthfactor1 #intestinaldisease #necrotizingenterocolitis #clinicaltrials #phase2 #prematurebabies

TOP Pediatric SECRETS: 28. In children with simple obesity (e.g., familial), linear growth is typically enhanced; in children with endocrinopathies (e.g., Cushing syndrome, hypothyroidism), linear growth is usually impaired. 29. The most common cause of persistent seizures in children is an inadequate serum antiepileptic level. 30. Genetic influences are strong in pediatric nocturnal enuresis. If both parents were enuretic, a child's likelihood is about 75%; if one parent was involved, the likelihood is about 50% 31. During the first year of life, hypotonia is more common than hypertonia in Infants who are ultimately diagnosed with cerebral palsy. 32. In patients with sickle cell disease, use of transcranial Doppler ultrasound to measure intracranial blood flow and regular transfusions to reduce the hemoglobin S content for those with abnormal values can significantly lower the likelihood of stroke. 33. in infants with significant gastroesophageal reflux, 25% to 50% spontaneously resolve by 6 months of age, 75% to 85% by 12 months of age, and 95% to 98% by 18 months of age. infant or in the deltoic muscle in 34. When vaccinations are given as recommended in the anterior lateral thigh in toddlers 18 months, aspiration (which increases pain and time to administer) is not required because no larje blood vessels are located at those preferred sites. 35. An infant with vomiting, lethargy, hypoglycemia, and no ketones on urinalysis should be evaluated for a fatty-acid oxidation defect. 36. Treatment failures are more common in osteomyelitis than in septic arthritis because antibiotic concentrations are much greater in joint fluid than in inflamed bone, devitalized bone may serve as an ongoing nidus for infection, and diagnosis of osteomyelitis is more likely to be delayed than diagnosis of septic arthritis. 37. Acute kidney Injury (AK) has replaced the term acute renal failure (ARF) to reflect the more appropriate concept that smaller reductions in kidney function (short of complete organ failure) have significant clinical repercussions in terms of morbidity and mortality. 38. An infant with nonsyndromic sensorineural hearing loss should be tested for mutations in the connexin 26 gene. Mutations in that gene contribute to at least 50% of autosomal recessive hearing loss and about 10% to 20% of all prelingual hearing loss.

Combined liver-kidney transplantation (CLKT) is a significant and successful surgical procedure, particularly in pediatric cases where congenital diseases drive the need for transplantation, according to a recent review. View the complete article below ⤵️ #pediatrics #livertransplant #kidneytransplant

Can we rely on #pEEG findings to guide us on hemodynamic management during cardiogenic shock in pediatrics? Seemingly yes, please read our latest case report in Journal of Clinical Monitoring and Computing https://lnkd.in/dZuKeTBR