In a new survey released by Ann & Robert H. Lurie Children’s Hospital of Chicago and the Chicago Department of Public Health (CDPH), Chicago parents identified gun violence, bullying and poverty as the biggest social problems for children and adolescents in the city. The survey included parents from all 77 community areas in Chicago.
An open label Phase 3 study conducted at Ann & Robert H. Lurie Children’s Hospital of Chicago and other centers established that a combination of cystic fibrosis drugs lumacaftor and ivacaftor is safe and effective in children aged 2-5 years, whose disease is caused by two copies of F508del-CFTR gene mutation – the most common and severe form of cystic fibrosis.
Researchers at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago, and colleagues, discovered a promising direction toward understanding the development of necrotizing enterocolitis (NEC), a devastating intestinal disease commonly affecting premature infants, in order to treat it.
Researchers at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago, and colleagues, used a blood test and microarray technology to identify distinct molecular signatures in children with cystic fibrosis. These patterns of gene expression ultimately could help predict disease severity and treatment response, and lead to therapies tailored to each patient’s precise biology.
Chicago adults identified drug abuse, obesity, and child abuse and neglect as the top three big health problems for children and adolescents in the city, according to results from a new survey developed by Ann & Robert H. Lurie Children’s Hospital of Chicago and the Chicago Department of Public Health (CDPH).
While probiotics are often used to treat acute gastroenteritis (also known as infectious diarrhea) in children, the latest evidence shows no significant differences in outcomes, compared to a placebo.
About a year after receiving daily oral immunotherapy for severe peanut allergy, 67 percent of children in a Phase 3 trial were able to tolerate eating at least two peanuts (600 mg) without an allergic reaction, while 50 percent tolerated eating three to four peanuts (1,000 mg) without symptoms. At the start of the study, all of these children had allergic reactions after ingesting just 1/10 of a peanut (30 mg). These results of an international, randomized, double-blind, placebo-controlled trial conducted at 66 sites, including Ann & Robert H. Lurie Children’s Hospital of Chicago, were published in the New England Journal of Medicine.
Researchers from Ann & Robert H. Lurie Children’s Hospital of Chicago and colleagues estimate that nearly 8 percent of U.S. children (about 5.6 million) have food allergies, with nearly 40 percent allergic to more than one food. These findings were based on their latest national food allergy prevalence survey, which assessed over 38,000 children.
Project LifeSkills, a behavioral intervention to prevent HIV in young transgender women, was designated by the HIV/AIDS Prevention Research Synthesis (PRS) project at the U.S. Centers for Disease Control and Prevention (CDC) as showing the best evidence of efficacy.
Scientists from Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago and colleagues identified promising new genetic pathways associated with severe lung disease in extremely premature infants, as well as pathways linked to faster recovery from lung disease in this population. The study is the largest to date to perform whole exome sequencing – or examine all the genes that code for proteins – in relation to respiratory outcomes of prematurity.
In the first publication from the U.S. on surgical techniques and outcomes of single ovary removal for fertility preservation in girls, surgeons from Ann & Robert H. Lurie Children’s Hospital of Chicago report that the procedure caused no complications and can be performed laparoscopically, on an outpatient basis, without delaying treatment for cancer or other therapies posing high risk of infertility.
To help clinicians maximize nutrition and growth in very low birth weight infants, researchers quantified the gains and losses of different nutrition delivery practices during the transition to enteral feeds. Their results were published in The Journal of Pediatrics.
Scientists at the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago and colleagues discovered how a type of RNA, called Evf2 enhancer RNA, regulates key genes during a critical stage in embryonic brain development.
Scientists identify a safe drug that for the first time could treat – and possibly reverse – the thickening of lung artery walls in pulmonary arterial hypertension;
clinical trial is expected in 2019
Symptoms of food-induced anaphylaxis in infants are much less severe than in toddlers and older children, according to a study from Ann & Robert H. Lurie Children’s Hospital of Chicago published in the Annals of Allergy, Asthma and Immunology. Anaphylaxis is defined as a reaction that involves multiple systems in the body or a presentation with significant cardiac or respiratory symptoms. While in older children an allergic reaction to food can be life-threatening, anaphylaxis in infants mostly manifests as hives and vomiting, the study found. With over 350 cases analyzed, including 47 infants, this is the largest study to date to describe food-induced anaphylaxis in infants under 1 year of age compared to other age groups.
Young adults who had parents incarcerated during childhood do not receive timely healthcare and have more unhealthy behaviors, Lurie Children’s researchers find
Researchers develop a simple and efficient method to induce human pluripotent stem cells to become blood, which could be key in future treatments for blood disorders, immune deficiencies and cancer
In 2001, when 30 children under 5 fell out of a window, Chicago was facing a public health crisis that was preventable. By 2016, window falls were cut in half after the 2002 launch of an educational campaign called “Stop the Falls” that urges families of young children to never open windows more than four inches and to use child-safety window stoppers or window guards. This data comes from a study from Ann & Robert H. Lurie Children’s Hospital of Chicago, which leads the campaign that was created by the Injury Free Coalition for Kids of Chicago.
As the first step toward predicting cerebral palsy in premature infants, scientists have identified a panel of microRNAs that are changed in babies who later develop abnormal muscle tone. MicroRNAs are small, noncoding RNA molecules that are important regulators of gene expression affecting developmental processes. Searching for microRNAs that could serve as early biomarkers – biological signs of disease – scientists for the first time have demonstrated that it is feasible to evaluate over 750 microRNAs using only one-half milliliter of blood collected from babies weighing less than 1500 grams (or under three pounds). Results were published in Physical Medicine and Rehabilitation – International.
One in five parents did not feel that their child with food allergy was safe while at school, according to results of a national survey published in BMC Pediatrics. While most of the 289 parents surveyed reported that their child’s school had implemented at least one food allergy policy, they felt that more could be done.
Scientists at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago have identified a gene called FoxM1 as a promising target for treatment of pulmonary hypertension, or high blood pressure in the lung arteries. Patients with this severe lung disease that damages the right side of the heart have a five-year survival rate of 50 percent. The study results, published in the American Journal of Respiratory and Critical Care Medicine, will drive development of new drugs to reverse a process called vascular remodeling, or thickening of lung artery walls – a key feature in pulmonary hypertension.
Survival of childhood cancers dramatically improved through national cooperative group research and care protocols; specialists call for the same model to improve outcomes of early life epilepsies.
Two-thirds of surveyed caregivers felt that their child’s food allergy affected their own daily lives very much or extremely, according to a report in the Annals of Allergy, Asthma and Immunology. Two-thirds of caregivers also expressed significant fear that their child would have an allergic reaction to food. Despite this fear, the majority of caregivers were eager to enroll their child in a clinical trial for immunotherapy, which involves giving the child a gradually increasing dose of the food allergen under close supervision in order to train the immune system to not react to that food. Only 8 percent of caregivers responded that they would not enroll their child in this type of clinical trial.
In a new approach to precision medicine research, scientists used bioinformatics tools to identify common features of genes associated with infantile spasms compared to other forms of early life epilepsy. Their analysis, published in PLOS ONE, reveals that infantile spasms are not only unique clinically, but also biologically. Focus on specific biological mechanisms underlying the genes that cause infantile spasms could help find new targets for treatment.
Nearly all school nurses participating in a national survey (96 percent) reported that staff at their school received training on handling severe allergic reactions to food. Over 80 percent asserted that their school had an emergency epinephrine auto-injector on hand to stop a potentially life-threatening allergic reaction. The study findings, published in the Annals of Allergy, Asthma and Immunology, also underscore the dire need for these policies, with over one-third of the school nurses reporting at least one severe allergic reaction to food at their school in the last academic year.
With this year’s severe flu season, one statistic is especially chilling. Each year, around 50 percent of all children under 5 years old who die from the flu were previously healthy, according to the Centers for Disease Control and Prevention (CDC). Adults who die from the flu, on the other hand, typically had a medical condition that increased their risk of mortality. A new study published in the Journal of Immunology offers new insights as to why healthy children are much more vulnerable. It also opens new opportunities for treatment.
Comparison of two of the most commonly prescribed drugs for infants with nonsyndromic epilepsy revealed that levetiracetam was more effective than phenobarbital, according a multicenter, observational study published in JAMA Pediatrics. After six months of single-drug treatment, 40 percent of infants who received levetiracetam met criteria for successful outcome – they did not require a second anti-epileptic drug to control their seizures and they became seizure-free within three months of starting treatment. Only 16 percent of infants treated with phenobarbital achieved the same outcome.
In a new international collaborative study between The Chinese University of Hong Kong and Ann & Robert H. Lurie Children’s Hospital of Chicago, researchers created a machine learning algorithm that uses brain scans to predict language ability in deaf children after they receive a cochlear implant. This study’s novel use of artificial intelligence to understand brain structure underlying language development has broad reaching implications for children with developmental challenges. It was published in the Proceedings of the National Academy of Sciences of the United States of America.
A study published in Pediatrics examined interdisciplinary provider perspectives on legal medical marijuana use in children with cancer. It found that 92 percent of providers were willing to help children with cancer access medical marijuana. However, providers who are legally eligible to certify for medical marijuana were less open to endorsing its use. While nearly a third of providers received one or more requests for medical marijuana, the lack of standards on formulations, dosing and potency was identified as the greatest barrier to recommending it. These findings reflect survey responses from 288 providers in Illinois, Massachusetts and Washington.
Ann & Robert H. Lurie Children’s Hospital of Chicago is one of the study sites in the open label Phase 3 study that showed safety and effectiveness of the cystic fibrosis drug Kalydeco (ivacaftor) in children ages 1 to 2 years. Based on these results, Vertex Pharmaceuticals Inc., (Nasdaq: VRTX) plans to submit applications for the drug in this age group to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Kalydeco is currently approved by the FDA for the treatment of cystic fibrosis in patients aged 2 years and older.
Findings published in Oncotarget offer new hope for children with highly aggressive brain tumors like atypical teratoid/rhabdoid tumor (AT/RT) and medulloblastoma. Previously, the authors of the study have shown that an experimental drug that inhibits polo-like kinase 4 (PLK4) stopped pediatric brain tumor growth in vitro. Now, they have demonstrated its success in an animal model – the drug shrank the tumor and increased survival. Importantly, the PLK4 inhibitor was able to cross the blood-brain barrier, which means that the drug can target cancer cells in the brain even when taken orally, avoiding the need for injection into the spinal fluid. In this new study, researchers also demonstrated that when they associate a PLK4 inhibitor with traditional chemotherapy drugs, they kill tumor cells with significantly lower drug doses.
A large multicenter study found that patients with cystic fibrosis (CF) – who tend to develop chronic lung infections – had more good and bad bacteria in their lungs compared to controls. The study used bronchoalveolar lavage, in which a bronchoscope is directed into the lung to directly collect lung secretions, and also found less diversity of bacteria in the lungs of CF patients who were older and had more lung disease or inflammation. Results, published in the European Respiratory Journal, provide a basis for future treatment studies.
Ann & Robert H. Lurie Children’s Hospital of Chicago is one of the few centers participating in ASPIRO, an international Phase 1/2 clinical trial of a gene therapy product called AT132 for X-linked myotubular myopathy – a rare disease characterized by severe muscle weakness, breathing difficulty and early death.
Post-operative fevers in children are rarely due to infection, yet they are often subjected to non-targeted testing. This conclusion has been widely recognized in adult patients undergoing surgery, but this is the first large-scale study to verify this finding in children.
More than half of the babies with infantile-onset spinal muscular atrophy (SMA) who were treated with nusinersen (Spinraza) gained motor milestones, compared to none of the babies in the control group. Infants treated with the drug also had 63 percent lower risk of death. These final results from the 13-month, international, randomized, multicenter, sham procedure-controlled, phase 3 trial called ENDEAR were published in the New England Journal of Medicine.
The first patient in a new Phase 1 gene therapy trial for pediatric brain tumors underwent a leading-edge procedure at Ann & Robert H. Lurie Children’s Hospital of Chicago. During surgery to remove the brain tumor, the patient was injected with an adenovirus, a common cold virus, at the tumor site. The virus was bioengineered not to cause illness but rather deliver a gene that produces human interleukin 12 (hIL-12), a powerful protein to jumpstart the immune system to kill remaining tumor cells. For the next 14 days, the patient is given a pill – veledimex – to activate the gene and control the immune response, so that the inflammation fights the tumor without overwhelming the rest of the body.
White children with viral diagnoses treated in pediatric emergency departments were up to twice as likely to receive antibiotics compared to minority children, according to a study published in Pediatrics. Although viral respiratory tract infections do not warrant antibiotic treatment, antibiotics were prescribed for these illnesses to 4.3 percent of white, 1.9 percent of black and 2.6 percent of Hispanic children.
Katherine Barsness, MD, MS, pediatric surgeon at Ann & Robert H. Lurie Children’s Hospital of Chicago and Associate Professor of Surgery and Medical Education, Northwestern University Feinberg School of Medicine has received a second funding award from the Patient-Centered Outcomes Research Institute (PCORI).
The $25,000 award, provided through PCORI’s “Pipeline to Proposal” program, will support a project that brings together patients and clinicians to discuss ways to improve the pediatric surgery patient experience.
A study published in JAMA Pediatrics supports the use of genetic testing, especially with sequencing, as first-line diagnostic method for young children with seizures. Specific genetic factors were found to be the cause of epilepsy in 40 percent of patients evaluated for first presentation with seizures. Genetic testing also yielded a diagnosis in 25 percent of children who had epilepsy with an otherwise unknown cause.
For the second year, Ann & Robert H. Lurie Children’s Hospital of Chicago has been named a Level I pediatric surgery center by the American College of Surgeons (ACS). In 2017 Lurie Children’s became the first children’s hospital in Illinois to earn this status and is currently one of only five in the country.
The Level I verification is awarded by a multi-organizational taskforce led by the ACS, the body responsible for setting the nation’s standards for quality of surgical care, practice and training.
Analysis of fatal and near-fatal physical abuse cases of children under 4 years of age revealed that psychosocial risk factors in the home, such as criminal history, were present in all cases. Two-thirds of the cases with prior medical records available (nine children) involved unexplained or atypical bruising – bruises on non-mobile infants, bruises on the ears, buttocks or eyes, and patterned bruises consistent with inflicted injury. All nine of these children suffered subsequent brain injury, resulting in four deaths. Findings were published in Child Abuse & Neglect.
Children with eosinophilic esophagitis (EoE) – a chronic inflammatory disease that injures the esophagus – who temporarily eliminated cow’s milk, wheat, egg and soy from their diet for eight weeks had their symptoms and esophageal swelling resolve, according to a study published in Clinical Gastroenterology and Hepatology. This elimination diet is less restrictive than the standard of care six-food elimination diet that is approved to treat EoE, a condition in which an abnormal immune response is triggered by certain foods, causing symptoms that range from difficulty swallowing to abdominal pain and vomiting. After remission, foods are reintroduced one by one until the food that triggers esophageal swelling and symptoms is identified and eliminated from the child’s diet. This is a lengthy process that involves multiple endoscopies to monitor the effect of reintroduced foods on the esophagus.
Aimen Shaaban, MD, a pediatric surgeon and leading expert in the area of fetal surgery, has joined Ann & Robert H. Lurie Children’s Hospital of Chicago as Director of The Chicago Institute for Fetal Health.
Heart health in children will be the focus of three closely synergistic research projects and an integrated multidisciplinary training program, that are newly funded by a $3.7 million four-year grant led by Bradley S. Marino, MD, MPP, MSCE, a pediatric cardiologist from Ann & Robert H. Lurie Children’s Hospital of Chicago and Professor of Pediatrics and Medical Social Sciences at Northwestern University Feinberg School of Medicine. As one of only four centers selected to participate in the American Heart Association’s Strategically Focused Children’s Research Network, research by Marino and colleagues will provide evidence for innovative policies, programs and practices to preserve cardiovascular health in childhood and beyond.
A study published in Molecular Cancer Research reveals that a tumor suppressor gene p16 is turned off by a histone mutation (H3.3K27M), which is found in up to 70 percent of childhood brain tumors called diffuse intrinsic pontine glioma (DIPG). This insight suggests that restoring p16 is a promising therapeutic strategy. The authors have demonstrated that this can be accomplished in vitro using a drug that is approved for treatment of adult leukemia and other cancers.