The Neurofibromatosis Program at the Gill Center is the largest pediatric neurofibromatosis (NF) program in North Texas and the only regional program affiliated with the Children’s Tumor Foundation.
Under the leadership of UT Southwestern assistant professor of pediatrics and neurological surgery Laura Klesse, M.D., Ph.D., the number of active patients in the program has doubled and continues to grow. The Neurofibromatosis Program at Children’s Health currently treats patients with all types of neurofibromatosis – the more common neurofibromatosis type 1 as well as the less common neurofibromatosis type 2 and schwannomatosis. The growth of our program reflects the effectiveness of our comprehensive approach to patient care and our ability to leverage clinical trials to the benefit of patients.
NF is the most common cancer predisposition syndrome in children. Managing this chronic disease year in and year out requires continuity of care with a focus on screening and access to subspecialists. To that end, the NF Program holds two clinics a week to accommodate annual or more frequent offce visits for all patients. These visits allow us to track patient health and serve as the coordination point for regular screenings – ophthalmological and neuropsychology evaluations are common, for example.
Once a month, complex NF cases are brought before a comprehensive board of subspecialists to develop and coordinate medical treatment plans. The board includes not only medical subspecialists, such as neurologists, neuropsychologists and ophthalmologists, but also school services specialists and surgical subspecialists. Given this breadth of expertise, all NF-related issues receive the best possible treatment.
Patients in the Gill Center’s Neurofibromatosis Program have access to the country’s leading NF research. Our program was one of five nationally to participate in a clinical trial to determine the effectiveness of GLEEVEC (imatinib mesylate) to treat plexiform neurofibromas – disfiguring, painful congenital lesions associated with NF1 that can result in substantial morbidity and early mortality. The trial, which closed in 2012, concluded that the drug demonstrated potential to inhibit plexiform neurofibromatosis in the head, neck and pelvis.
Our Neurofibromatosis Program was also selected as a site for a clinical trial to determine whether Lovastatin™ significantly improved visual spatial learning and/or sustained attention in children with NF1 (NCT00853580: A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1). We were one of only 10 NF programs to participate in the study and the only one in the Southwest. Currently, two institutional studies are underway. Led by Alice Ann Holland, M.D., one clinical trial assesses the prevalence and possible health correlation of bullying in our NF patient population. The second study is a collaborative effort between Animesh Tandon, M.D., pediatric cardiologist and Dr. Klesse to study the prevalence of heart disease in NF patients.