Innovations in Pediatric Medicine International Conference brings together pediatrics experts

- 7 Nov 2008

By New York- Presbyterian Hospital/Columbia University Medical Center

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Dr. Alain Fischer of Descartes University Hospital Necker–Enfants Malades, Paris, France, will discuss gene therapy for inherited disorders based on research on the treatment of severe combined immunodeficiency. Introducing genes into bone marrow stem cells led to sustained correction of the disease for almost 10 years, providing evidence that the approach can be effective and could be used to treat other genetic diseases of blood cells. One challenge is the viral vector used to introduce the gene has been linked to cancer. In response, new vectors are being designed.
Dr. Gary Fleisher of the Children's Hospital Boston and Harvard Medical School will present a framework for planning for disaster management, highlighting unique pediatric aspects. He will discuss the conditions likely to be encountered by providers arriving in the first 24 to 72 hours and the skills necessary for success. In terms of response teams, Dr. Fleisher will describe the structure of PST-1 (Pediatric Specialty Team-1), the first team developed by the NDMS (National Disaster Medical System) dedicated to treating children in disasters. He will share lessons learned from Hurricane Marilyn on St. Thomas; the 2001 attack on the World Trade Center in New York; the 2003 earthquake in Bam, Iran; and Hurricane Katrina.
Dr. Margaret K. Hostetter of Yale–New Haven Children's Hospital will present on advances in the genetic origins of common childhood infections, focusing on newly discovered genes that are linked to early onset staphylococcal infection, recurrent pneumococcal infections, and rarer disorders such as Bruton's agammaglobulinemia, hyper IgE syndrome, chronic granulomatous disease and severe combined immunodeficiency.
Dr. Jennifer M. Puck of the University of California, San Francisco, will present evidence that early diagnosis of primary immunodeficiencies is critical for optimal treatment. The challenge is that these disorders are rare and hard detect until serious complications have developed; a life-threatening situation usually has to occur before a correct diagnosis was made. As a solution, Dr. Puck suggests that all newborns be screened for severe combined immunodeficiencies, with the goal of improving timely diagnosis and outcomes. Her laboratory has developed a screening test that can be done on the dried blood spots routinely used for screening for other serious conditions.