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Children's National News

  1. 1. Summer 2008 Children’s National News An Electronic Newsletter for Physicians Features Children’s Intestinal Rehabilitiation Program- Children’s Intestinal Rehabiliation The new multidisciplinary Intestinal Program Rehabilitation (IR) Program at Children’s- Children’s National Heart Institute: National provides individualized, Growing Volumes and Improving comprehensive medical, dietary, and Outcomes surgical treatment for often critically-ill children. The medical treatment focuses on aggressive dietary management with very precise control of metabolic balance Research: News from CRI and prompt and effective treatment of complications. Aggressive use of specialized enteral feeding - Children’s Neuropsychologist Leads Research and programs by the experienced medical team helps to maintain Development of Youth Sports Concussion Programs nutrition and hydration, which are important factors in long-term- Results of New Clinical Trial Demonstrate How survival. Another important component of the program is on-going Personalized Medicine May Drastically Change the parent education and support. [Continue reading about Children’s Treatment of Genetic Disorders Instestinal Rehabilitation Program]- Recently Published Journals and Abstracts Children’s National Heart Institute: Growing Volumes and Improving Highlights Outcomes As Children’s National Heart Institute- New Web Site URL (CNHI) celebrates its second year, many- Children’s Launches Neurofibromatosis Institute exciting changes have been made to the- Children’s Brain Tumor Institute program, allowing Children’s National to make a difference in the lives of patients- Children’s Annual Report locally, nationally, and internationally. Last year, Children’s team operated on more infants, children, and adults with congenital heart disease than ever in the history of Children’s National Medical Center. We are able to have impressive survival statistics for our Subscribe Now patients because of advances in non-invasive diagnosis, cardiac intensive care, interventional cardiac catheterization, and cardiac Subscribe Now surgery. [Continue reading about Children’s National Heart Institute]Features | Research | HighlightsChildren’s Home | Contact the Editor
  2. 2. Back to TopChildren’s Intestinal Rehabilitiation ProgramIntestinal failure is the inability Children’s Intestinal Rehabilitation Program is a part of theof the small bowel to absorb joint program of Liver and Small Bowel Transplantationadequate amounts of nutrients, developed between Children’s National and Georgetownfluids, and electrolytes. The University. Although the highly specialized Program is newcauses of intestinal failure in at Children’s, team members have extensive experience inchildren are usually grouped managing these challenging patients.into three major categories:short bowel syndrome (SBS), Clarivet Torres, MD, director of the Intestinal Rehabilitationneuromuscular diseases involving Program at Children’s National is a nationally recognizedthe gastrointestinal tract (such as leader in this field, based on work at the University oftotal aganglionosis, long-segment Nebraska. She is a long-time colleague of Stuart Kaufman,hirschprung disease, or chronic Clarivet Torres, MD MD, who is medical director of Georgetown’s Pediatric Liverintestinal pseudo-obstruction), and Intestinal Transplantation Program. Other members ofand congenital diseases of the Children’s team include Anthony Sandler, MD, division chief,intestinal epithelium (microvillus inclusion disease). The most General and Thoracic Surgery; Parvathi Mohan, MD, acommon cause of SBS, especially in premature infants, is nationally recognized expert in liver and bowel disease; andnecrotizing enterocolitis. Other etiologies of SBS in children Pat Zavosky, RN, who has more than 15 years of experienceinclude gastroschisis, intestinal atresia, and mid-gut volvulus. managing critically ill children with bowel disorders. The team also includes a dedicated staff of nurses, nutritionists, andRegardless of the etiology, children with intestinal failure social workers.require very complex medical care. This care includesproviding appropriate nutrition for normal growth, Until recently, the outcome for many children with intestinalmaintenance of optimal fluid and electrolyte balance, failure was very poor. For example, previous studies indicatedmanagement of potential complications, and provision of the that children with less than 40 cm of small bowel length andspecialized care for the subset of patients who will require no ileocecal valve (ICV) or children with less than 15 cm ofsmall bowel and/or liver transplantation. The goal of intestinal small bowel with an intact ileocecal valve were unlikely torehabilitation is to optimize bowel function through the use be able to be weaned from parenteral nutrition. In addition,of multiple therapies and to eventually wean patients with children with direct bilirubins more than 2.5 mg/dL were moreintestinal failure from parenteral nutrition. likely to die.The new multidisciplinary Intestinal Rehabilitation (IR) Although it is challenging to successfully treat children withProgram at Children’s National provides individualized, intestinal failure, newer data provide hope for the attainmentcomprehensive medical, dietary, and surgical treatment for of more successful outcomes. Dr. Torres recently publishedoften critically-ill children. The medical treatment focuses on a summary of her experience from the past six years. Duringaggressive dietary management with very precise control this period, 74 parenteral nutrition dependent intestinal failureof metabolic balance and prompt and effective treatment of patients were evaluated. The majority of the patients hadcomplications. Aggressive use of specialized enteral feeding only jejunum with a median intestinal length of 38 cm (rangeprograms by the experienced medical team helps to maintain 8 cm to 150 cm). The median daily caloric requirement bynutrition and hydration, which are important factors in long- parenteral nutrition at the time of evaluation was 100 percentterm survival. Another important component of the program is (range 55 to 100 percent). Fifty-one of the 73 patients hadon-going parent education and support. cholestatic liver disease: 12 with cirrhosis, 27 with bridgingChildren’s National News Back to Top Summer 2008, pg. 2
  3. 3. Back to TopChildren’s Intestinal Rehabilitiation Program (continued)fibrosis, and 12 with portal fibrosis. Forty-seven patients had The initiation of the Intestinal Rehabilitation Program at64 different intestinal surgical repairs, including 15 bianchi Children’s National provides children with intestinal failureprocedures and 17 step enteroplasties. Forty-one of the 51 the chance to receive comprehensive medical and surgicalwith direct bilirubin greater than 2.5 mg/dL normalized the care, giving them the chance for improved long-termserum bilirubin with treatment. survival, including weaning from parenteral nutrition and avoidance of the need for transplantation and long-termTen patients required transplantation; two are listed for immunosuppression. Early referral of patients with intestinaltransplant and seven patients died. Of the remaining 55 IRP failure prior to the development of advanced liver disease ispatients, 39 have been weaned from TPN, including 25 with recommended.severe liver disease marked by varying degrees of fibrosisor cirrhosis. Seventeen additional patients are in the process For inquiries about patients with intestinal failure or earlyof weaning from parenteral nutrition, and have decreased referral of patients, contact the Division oftheir median parenteral nutrition requirements from 100 Gastroenterology, Hepatology, and Nutrition atpercent to 40 percent. Several laboratory measurements 202-476-3032 and ask for Pat Zavosky.also improved in these 55 patients. The mean direct bilirubindropped from 6.3 mg/dL to 0.4 mg/dL (p = <0.0001), themean albumin increased from 3.0 to 3.7 (p = <0.0001), andthe mean platelet count increased from 200,000 to 300,000(p = 0.0007). One of the most clinically important outcomeswas the improvement in growth. The surviving non-transplantpatients have had statistically significant improvements intheir mean weight Z scores and height z-scores. The survivalrate of the patients in the Intestinal Rehabilitation Programstudy was 90 percent.These data are dramatically better than those publishedin previous medical literature and provide real hope for animproved future for children with intestinal failure, includingthose with chronic liver disease. More aggressive intestinalrehabilitation can be very effective in improving the outcomesfor patients with intestinal failure, including those who haverisk factors previously identified for poor prognosis in termsof parenteral nutrition weaning or survival. Even the patientswho subsequently required transplantation improved theirgeneral state of health while in the program and were betterable to tolerate surgery and immunosuppression. None of theIntestinal Rehabilitation Program patients on the transplantwaiting list in Dr. Torres’ study have died or were transplantedin critical condition.Children’s National News Back to Top Summer 2008, pg. 3
  4. 4. Back to TopChildren’s National Heart Institute:Growing Volumes and Improving OutcomesAs Children’s National Cardiac surgery has seen similar growth since the arrival ofHeart Institute (CNHI) Richard A. Jonas, MD, in 2005 (Figure 2). Not only have thecelebrates its second number of cases increased by more than 100 percent, theyear, many exciting complexity of cases has increased significantly. Compared tochanges have been made previous years where the mix of closed cases to open casesto the program, allowing was equal, now more than 60 percent of Children’s operativeChildren’s National to make procedures involve cardio-pulmonary bypass (open).a difference in the lives of Additionally,patients locally, nationally, nearly 15 percentand internationally. Last of patients areyear, Children’s team from sites outsideoperated on more infants, our region,children, and adults with Children’s National Heart Institute traveling to Co-Directors Richard Jonas, MD,congenital heart disease Washington from and Gerard Martin, MD.than ever in the history across America orof Children’s National Medical Center. And because of from around theadvances in non-invasive diagnosis, cardiac intensive care, world.interventional cardiac catheterization, and cardiac surgery, weare able to have impressive survival statistics for our patients. Cardiac Intensive Care Unit (CICU) Children’s National has the only pediatric-focused CICUIncreases in Patient Volume in the Washington, DC, region and it is directed by JohnThe increase in patient volumes has been most impressive in Berger, MD, who is board certified in both pediatricthe areas of interventional cardiac catheterization and cardiac cardiology and critical care medicine. The CICU providessurgery. Since the arrival of Michael C. Slack, MD, in 2001 highly focused subspecialty care for a distinct population ofand the building of a dedicated catheterization suite in 2003, patients recovering from heart surgery and critical cardiacthere has been an 100 percent increase in procedures, with disease. The unit, a state-of-the art 15-bed unit, provides76 percent being therapeutic procedures (Figure 1). optimal family-centered care and is staffed by nurses, doctors, nutritionists, pharmacists, respiratory therapists,In 2006, social workers, and child life specialists specially trained forChildren’s the care of congenital heart disease.Nationalperformed Cardiac Outcomesmore than 500 The cardiac surgery program contributes to the Societycatheterization of Thoracic Surgery Database and compares Children’sprocedures. risk adjusted results with centers from around the country.Children’s Risk adjustment is done by the Risk-Adjustment-in-Joshua Kanter, MD, is a second interventional cardiologist Congenital-Heart-Surgery-1 method (RACHS-1), whichand Jonathan Kaltman, MD, is a second electrophysiologist assigns a prediction of survival based upon the disease(joining Jeffrey Moak, MD), allowing us to grow this program and procedure to be performed. Overall, survival for cardiacfurther. surgery procedures performed at Children’s National hasChildren’s National News Back to Top Summer 2008, pg. 4
  5. 5. Back to TopChildren’s National Heart Institute:Growing Volumes and Improving Outcomes (continued)increased to 98 percent with open survival of 96.8 percentand closed survival of 98.5 percent. The results (based uponindividual lesions with n of at least 10) are shown in Figure 3.Partnerships with other divisions in the hospital also improvepatient outcomes. Collaborations between critical care andcardiology enabled the development of a CAT Team (ClinicalAssessment Triage Team), which resulted in a 76 percentreduction in code blue on the cardiology unit. The teamachieved a “180 days since last code” performance recordlast year.The Future of CNHICNHI has accomplished much during the past two years, butmore is expected in the upcoming year. Under the directionof anesthesiologist Richard Kaplan, MD, cardiac anesthesiais expanding and providing superb care to patients requiringcardiac surgery, cardiac catheterization, and non-cardiacprocedures. Richard J. Levy, MD, recently joined Children’sNational as associate chief of Anesthesiology and CardiacAnesthesia.David Wessel, MD, joined Children’s National in July 2006as executive director of the Center for Hospital-BasedSpecialties. Dr. Wessel is the world’s foremost expert in Figure 3. Results are not risk-adjusted and include low-birth-cardiac intensive care and is expected to further develop weight infants weighing less than 2 kg. Procedures with ten orDr. Berger’s team, as well as increase the number of clinical more cases performed are included. The data are submitted annually to the Society of Thoracic Surgeons’ Congenital Hearttrials offered at Children’s. Surgery National Database. *** There were five mortalities among the 43 cases of patent ductus arteriosus ligation, all due to complications of prematurity not related to the surgical procedure. These have been excluded from mortality statistics in accordance with the guidelines of the Society of Thoracic Surgeons.Children’s National News Back to Top Summer 2008, pg. 5
  6. 6. Back to TopChildren’s Neuropsychologist Leads Research andDevelopment of Youth Sports Concussion ProgramsWorld-renowned traumatic brain injury expert, Gerard Gioia,PhD, division chief of Neuropsychology at Children’sNational Medical Center and director of Safe ConcussionOutcome, Recovery and Education (SCORE) Program,published a report on a successful concussion managementprogram based on his pioneering research in concussionmanagement in children. Most concussion managementprograms for youth sports lag behind collegiate andprofessional sports, despite the serious consequences ofbrain injury on a developing brain.Dr. Gioia’s research and “10 steps” to a successful Gerard Gioia, PhDconcussion management process are detailed in a recentedition of Brain Injury Professional (Vol. 4, Issue 4, pp14-15).“Implementing an effective sports concussion managementprogram is essential to safeguard young participants andreduce long term risks,” writes Dr. Gioia. “Management of thisserious injury must consider the various effects in the home,school, social, and sports environments.”Dr. Gioia’s work in effective management of mildtraumatic brain injury (TBI)/concussion in children iswell documented. His research has translated a model ofneurocognitive testing used in professional athletes for usein children who have suffered from a mild TBI. This work,funded by the Centers for Disease Control and Prevention(CDC), uses computer-based testing and standardizedparent/teacher reports of the student athlete’s neurocognitivefunctioning to better guide recovery after a mild TBI.For more information about Dr. Gioia’s work, see Children’smost recent Annual Report.Children’s National News Back to Top Summer 2008, pg. 6
  7. 7. Results of New Clinical Trial Demonstrate How Back to TopPersonalized Medicine May Drastically Changethe Treatment of Genetic Disorders One of the nation’s pre- eminent genetic researchers, Eric Hoffman, PhD, of Children’s Research Institute at Children’s National Medical Center, predicts that in relatively short order, medicine’s next innovation–individualized molecular therapies–will have the unprecedented ability to treat muscular dystrophies,Eric Hoffman, PhD and other disorders.In a recent edition of the New England Journal of Medicine,Dr. Hoffman posits that the results of a small clinicaltrial involving a new treatment for Duchenne musculardystrophy provides a proof-of-principle for personalizedmolecular medicine. Practical implementation of the ‘exon-skipping’ approach described in the co-published reportof vanDeutekom et al. will require advances in systemicadministration of large amounts of customized DNA-likedrugs, and proof that long-term delivery is not toxic. However,these advances are likely to come in short order, with theoversight and regulations of the FDA critical for appropriatelabeling and marketing of such personalized molecular targetdrugs.Though this particular treatment remains in its early stages,within the foreseeable future the now-standard Phase I,II, and III pathway to drug approvals may need to be re-evaluated.The study featured the New England Journal of Medicine,involves application of a nucleic acid drug called PRO051.It shows some success at restoring the expression of thespecific protein - dystrophin that is linked to healthy muscletissue. This approach was shown to reactivate dystrophinprotein production in small areas of muscle tissue at theinjection site of muscular dystrophy patients.Children’s National News Back to Top Summer 2008, pg. 7
  8. 8. Back to TopHighlightsNew Web Site URL Children’s Neurofibromatosis Institute was chosen as one of eight programs in the United States to participate inChildren’s National launched the newly designed the Neurofibromatosis Clinical Trials Consortium and Please note Children’s new Packer is group chair of the consortium. The Institute alsoURL and update your address books. participates in developing new therapeutic treatments for the neurocognitive aspects of neurofibromatosis, and assesses the agent, lovastatin, for children with neurofibromatosis and learning disabilities. The Institute provides extensive support for families and children, including the coordination of the largest neurofibromatosis camp on the East Coast for children between the ages of 7 and 16. Children’s Brain Tumor Institute Children’s new Brain Tumor Institute is a multidisciplinary,The new site has many features, including: internationally renowned collaboration that evaluates one out- An improved home page navigation and design of every 10 children in the United States diagnosed with a- Section for referring physicians, with access to information brain tumor. about insurance and referral resources and guidelines- A new section dedicated to Children’s nurses Children’s unique collaboration between the Divisions of- Information about traveling to Children’s from Oncology and Neurology means that patients see an outside the Washington, DC, region oncologist and a neurologist at every visit. In addition, the program includes neurosurgeons, neuropsychologists, radiologists, psychologists, endocrinologists, andChildren’s Launches pathologists.Neurofibromatosis Institute As one of the most active clinical research programs in theChildren’s new Daniel and Jennifer Gilbert country, the Institute delivers the most innovative and bestNeurofibromatosis Institute, one of the largest therapies possible, including those which delay, and at timescollaborations focused on neurofibromatosis in the world, eliminate, radiotherapy for infants and children; biologic-specializes in the diagnosis, evaluation, and treatment of based translational therapies; and high-dose chemotherapychildren and adults with the full range of conditions that relate with stem cell therapy support. Additionally Children’s brainto this disorder. tumor laboratories study the neurology of the tumor and evaluate the long-term effect of the tumor or its treatment onLed by Children’s neurologist, Roger Packer, MD, outcomes.Children’s Neurofibromatosis Institute is designated aNeurofibromatosis Center of Excellence by the National Children’s patients have access to leading edge treatmentsNeurofibromatosis Foundation. The Institute offers a through Children’s Oncology Group’s Phase I trials, Pediatricmultispecialty clinic with one central location for patients Brain Tumor Consortium Protocols, the National Cancerand families to meet with specialists in genetics, neurology, Institutes, and Neurofibromatosis Clinical Trials Consortiumand ophthalmology. From that clinic, patients and families Protocols.are referred as needed to specialists in oncology,neurosurgery, neuropsychology, and otolaryngology.Children’s National News Back to Top Summer 2008, pg. 8
  9. 9. Back to TopHighlights (continued)Children’s Annual ReportTransforming Children’s HealthOne Child at a TimeAt Children’s National MedicalCenter, our team of pediatricexperts is dedicated to transformingchildren’s health, not only in ourregion, but in the nation andthroughout the world. Our teamaddresses the needs of eachchild on an individual basis. Whatwe do through our mission ofCARE—world-class care, advocacy,research, and education—extendsfar beyond the walls of our hospital. In addition to treatingpatients, our clinicians are in the lab developing innovativetreatments, or testifying on the Hill, or teaching the nextgeneration of pediatric specialists.We invite you to read about the transformations occurringeveryday at Children’s National, and we hope to inspire youto join us as we improve the lives of children, one child at atime.Request a copy of Children’s Annual Report.Children’s National Opens NewInpatient TowerOn November 18,2007, Children’sNational MedicalCenter moved into itsnewest wing of thehospital—the EastInpatient Tower.Learn more about our new facility.Children’s National News Back to Top Summer 2008, pg. 9
  10. 10. Recently Published Journals and Abstracts Back to Top - Cross-sectional multicenter study of patients with - Sensitivity and specificity of decreased CSF urea cycle disorders in the United States. asialotransferrin for eIF2B-related disorder. Mol Genet Metab. 2008 June 16. Neurology. 2008 Jun 3;70(23):2226-32. PMID: 18562231 PMID: 18519871 - Exposure to oxygen and head growth in infants - Protein Expression of Platelet-Derived Growth Factor with bronchopulmonary dysplasia Receptor Correlates with Malignant Histology and Am J Perinatol. 2008 Apr;25(4):251-4 PTEN with Survival in Childhood Gliomas. PMID: 18548401 Clin Cancer Res. 2008 Jun 1;14(11):3386-94. PMID: 18519768 - A Descriptive Study of Noise in the Neonatal Intensive Care Unit Ambient Levels and Perceptions - Pulmonary eosinophilia requires interleukin-5, of Contributing Factors. eotaxin-1, and CD4+ T cells in mice immunized with Adv Neonatal Care. 2008 Jun;8(3):165-175. respiratory syncytial virus G glycoprotein. PMID: 18535422 J Leukoc Biol. 2008 Jun 2. PMID: 18519743 - Influence of Cytotoxic T Lymphocyte-associated Antigen 4 (CTLA4) Common Polymorphisms on - Relationship of temporal lobe volumes to Outcome in Treatment of Melanoma Patients With neuropsychological test performance in CTLA-4 Blockade. healthy children. J Immunother. 2008 July/August;31(6):586-590. Brain Cogn. 2008 May 28. PMID: 18528295 PMID: 18513844 - Received, understanding and satisfaction of National Health Insurance premium subsidy scheme by families of children with disabilities: A census study in Taipei City. Res Dev Disabil. 2008 Jun 3 PMID: 18524537 - A review of pediatric uveitis: Part I. Infectious causes and the masquerade syndromes. J Pediatr Ophthalmol Strabismus. 2008 May- Jun;45(3):140-9. PMID: 18524191Children’s National News StaffEdwin K. Zechman, Jr. Katherine D. JaucianPresident & CEO Manager, Interactive CommunicationsJacqueline D. BowensVice President & Chief Government Corinne Ahrensand External Affairs Officer Editor/WriterAndrew D. Hertzberg ContributersExecutive Director, Mina YoussefPublic Affairs Janiene Torch Victoria Hughes Children’s National Medical Center 111 Michigan Avenue, NW, Washington, DC 20010 | | 202-476-5000 Copyright © 2008, Children’s National Medical Center. All rights reserved.Children’s National News Summer 2008, pg. 10