The Childhood Adenotonsillectomy Trial (CHAT funded by the National Institutes of Health) was a multi-institutional, collaborative clinical research study designed to determine the effect of adenotonsillectomy surgery on obstructive sleep apnea syndrome (OSAS) in children. OSAS can cause health problems including poor growth, high blood pressure, diabetes, and behavioral and learning difficulties. Although adenotonsillectomy is the usual treatment for children with OSAS, it was not previously known with any certainty whether OSAS symptoms significantly improve after surgery or if earlier surgery would be more beneficial than watchful waiting with supportive care prior to surgery.
Raouf S. Amin, MD is one of the principal investigators for the CHAT study. Over 450 children with OSAS, five through nine years of age were randomly assigned to early adenotonsillectomy or watchful waiting and assessed for polysomnographic, cognitive, behavioral, and health outcomes. The findings of the study recently were reported in the New England Journal of Medicine paper, “A randomized trial of adenotonsillectomy for childhood sleep apnea”. It was determined that adenotonsillectomy reduces symptoms and improves secondary outcomes of behavior, quality of life, and polysomnographic measures, thus providing evidence of the beneficial effects of early surgical intervention for children with OSAS.
Drug Discovery in CF
Cystic fibrosis (CF) is a disorder affecting over 30,000 patients in the US and > 70,000 worldwide. Mutations in the CF gene lead to defects in the CFTR protein, which works as a regulator of salt and water movement throughout the body. CF patients develop problems with mucus in the lungs and abnormal food absorption that lead to lung infections, poor growth, and death in early adulthood. Although improvements have been made in CF patient care for several decades, all previous therapies have targeted downstream symptoms of CF rather than early steps in the disease cascade.
John P. Clancy, MD, is the director of the Cincinnati Children’s Cystic Fibrosis Center. Over the past 18 months, dramatic changes have occurred in CF care, starting with the FDA approval of kalydeco for the treatment of CF caused by the G551D CFTR mutation (found in about 5% of CF patients). Kalydeco is a 'CFTR modulator', which is a class of drugs that targets the root cause of CF on a mutation by mutation basis. Currently, Phase II and Phase III studies are underway testing kalydeco in combination with other drugs ('correctors' of the F508del CFTR mutation) that target the most-common CF-causing mutation. Also, studies are underway examining kalydeco as a monotherapy in new CF populations beyond those with the G551D CFTR mutation. It is hoped that within a few years, essentially all CF patients will be treated with medications that target the root cause of CF and restore activity to disease-causing CFTR mutations. Cincinnati Children’s is playing a critical role in these studies, and enthusiasm for these breakthrough therapies is at an all-time high in the global CF care and research communities.
Summer Research Fellowship Training Program
Our division was one of only two pediatric pulmonary centers in the United States to receive the National Institutes of Health Ruth L. Kirschstein National Research Service Award (NRSA) in 2012 for a Short-Term Institutional Training Grant program for Clinician Scientists in Pediatric Respiratory Medicine. The program provides funding for research training opportunities in basic or clinical research areas in pediatric pulmonary and sleep medicine for medical students in the summer between the first and second years of medical school.
William D. Hardie, MD is the director of this training program. Recruitment efforts for the second year resulted in an increase in applicants from 18 in 2012 from six institutions to 36 in 2013 from 17 institutions, including three from countries outside of the United States. The eight applicants accepted into the 2013 program were matched with mentors for research projects in a variety of pediatric respiratory research areas, including asthma, cystic fibrosis, lung cancer, and sleep apnea. The strengths of our research programs, combined with an enthusiastic faculty and a well-organized summer program, continue to provide an excellent opportunity to attract physician trainees at a critical stage in their careers and increase the likelihood they will want to further pursue research as physician-scientists.