(From Pediatric Directions, Issue 38)
The statement provides an extensive inventory of diagnostic and interventional techniques that are now considered as options for pediatric patients, noting that catheterization procedures carry a degree of risk for patients. The statement also covers several hybrid procedures that use traditional surgical techniques in combination with catheterization for treating conditions, such as hypoplastic left heart syndrome and stent implantation among others.
“There are numerous conditions that are best served by interventional catheterization procedures,” said Dr. Feltes. The statement is key to cardiologists who treat pediatric defects, because there are few other sources of such information. “By virtue of the relatively small number of children and adolescents with congenital heart disease, it is difficult to design clinical trials. Ideally, you need thousands of patients to compare one treatment versus another. Only one child in 100 is born with heart disease, so it is very unlikely that one center will have more than one patient to do a side-by-side comparison,” Feltes said. Click for more information about Dr. Feltes and The Heart Center.
Co-authors: Emile Bacha, M.D., chair; Robert H. Beekman III, M.D.; John P. Cheatham, M.D.; Jeffrey A. Feinstein, M.D., M.P.H.; Antoinette S. Gomes, M.D.; Ziyad M. Hijazi, M.D., M.P.H.; Frank F. Ing, M.D.; Michael de Moor, MB.BCh.; W. Robert Morrow, M.D.; Charles E. Mullins, M.D.; Kathryn A. Taubert, Ph.D. and Evan M. Zahn, M.D. Author disclosures are on the manuscript.
Published in the November 2010 issue of the Annals of Neurology, it is part of multi-phase, first-ever successful gene therapy trial in muscular dystrophy, showing sustained expression of a transferred gene to muscle. The findings provide proof of principle that gene replacement to restore missing proteins in muscular dystrophy is a viable approach to therapy for this devastating disease. The follow up to this line of attack is gene replacement through the circulation, and Dr. Mendell and his team of researchers have been awarded an NIH grant of $1,041,296 to continue their work.
GERD is a frequent consideration in infants at risk of the life-threatening events of chronic lung disease and dysphagia. Yet, the definition of GERD in neonates and infants and its treatment remains controversial. Acid suppressive medications and prokinetics are commonly prescribed to alter gastric acidity and improve gastrointestinal motility in neonates, yet such therapies can have harmful side effects.
According to Dr. Jadcherla, “It’s difficult to distinguish whether symptoms of reflux are part of a neonate’s normal functioning or if they are disease-based. As such, there are no definite standards to aid in the management of reflux among neonates in the intensive care unit, nor clarity regarding symptom recognition, nor standards to aid in the diagnosis of GERD.”
Dr. Jadcherla says that establishing objective evidence of gastroesophageal reflux events and the relationship of symptoms with the physical or chemical composition of the refluxate is essential to characterize whether patients have a disease that needs treatment. To help characterize reflux events, Nationwide Children’s investigators evaluated 30 neonates in Nationwide Children’s NICU who were suspected to have GERD. Using pH-impedance studies, the team determined refluxate presence, composition, distribution and clearing time. Nurses also documented whether the neonates showed any respiratory symptoms (coughing, gagging, grunting), sensory symptoms (irritability, grimacing, crying) or physical movement, such as stretching of their limbs, during the identified reflux events.
“It is clear from this study that symptoms can occur with acid, non-acid, gas, liquid or mixed events,” said Dr. Jadcherla. “So, treatment strategies based on modifying gastric acidity alone can be ineffective as acid is not the lone provoking agent.” Dr. Jadcherla says it is also clear why the management of GERD based on symptoms only is controversial, as there are reflux-type symptoms in the absence of reflux.
“Overall, this study helps clarify the definition of GERD: gastroesophageal reflux with increased frequency of respiratory, sensory or movement symptoms,” said Dr. Jadcherla.
CHCA has implemented RACE for Results in order to recognize the medical institutions that demonstrate clinical, financial or operational improvement. The annual competition is only open to CHCA Owner Hospitals that demonstrate these responsibilities for at least six months. Winners are selected by a panel of hospital peers and international health care experts.
“We are honored to receive such a great award and commend our Neonatal Services leadership and staff for their efforts in reducing length of stay for our most delicate patients,” said Rich Brilli, MD, FAAP, FCCM, chief medical officer at Nationwide Children’s Hospital. “Reducing length of stay is just one of the many ways Nationwide Children’s is working to ensure the best outcomes and experience for every child we treat.”