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Letter from Dr. Stephen I. Katz: The Promise of Pediatric Research
Letter From Dr. Stephen I. Katz: The Promise of Pediatric Research
In October 2015, the NIH announced the Environmental influences on Child Health Outcomes (ECHO) program, a major new initiative to improve understanding of environmental effects on the health and development of children. The agency also announced a change to its long-standing policy on the inclusion of children as participants in research involving human subjects. The new policy redefines the age of a child from 21 years old or younger to below 18 years to align the NIH definition with the common perception of the age of adulthood. In light of these important developments, this month’s letter focuses on new and ongoing pediatric research activities at NIAMS and the NIH.
The ECHO program, a new multi-year initiative that will begin in fiscal year 2016, represents an alternative approach to the former National Children’s Study that the NIH closed in 2014. As a precursor to ECHO, in September 2015, the NIH funded several programs to develop new tools and measures for investigating the effects of environmental exposures on children’s health and development. The NIAMS is leading one of the 2015 initiatives, called the Validation of Pediatric Patient-Reported Outcomes in Chronic Diseases (PEPR) Consortium, with participation from several other NIH components. PEPR expands the Patient Reported Outcomes Measurement Information System (PROMIS), an NIH Common Fund program, by validating PROMIS measures for children. The program will assess children with a variety of chronic diseases in clinical research and care settings to understand the effects of the environment (including socioeconomic factors) on symptoms and quality of life. Many of the conditions being studied by the Consortium, such as atopic dermatitis (eczema), lupus, and juvenile idiopathic arthritis, are relevant to the NIAMS mission.
PEPR is just one recent example of NIAMS-supported pediatric research. Since its inception, the Institute has made significant investments in intramural and extramural research on pediatric diseases, including rare genetic conditions. Several of these were discussed in my August 2014 Director’s letter. For example:
- Dr. Robert Colbert, Chief of the NIAMS Intramural Research Program’s (IRP) Pediatric Translational Research Branch, is studying the genes and molecular mechanisms involved in spondyloarthritis, including forms of the disease that occur in children, to pave the way for new treatments.
- Dr. Raphaela Goldbach-Mansky, Acting Chief of the Translational Autoinflammatory Disease Section, has advanced how we care for children with rare autoinflammatory diseases.
- Dr. Michael Ombrello, Head of the Translational Genetics and Genomics Unit, is using cutting-edge genomic and bioinformatics approaches to improve interventions for children affected by autoimmune and autoinflammatory diseases.
Within the extramural community, the NIAMS supports a broad portfolio of pediatric research on several diseases, including muscular dystrophy, a debilitating genetic disease that causes progressive muscle weakness. The NIAMS is one of four NIH Institutes that lead the Muscular Dystrophy Coordinating Committee, a group that coordinates activities relevant to the various forms of the disease across NIH and with other federal health programs and activities.
In addition, many groups are collaborating to facilitate pediatric research, which is particularly important for rare diseases. For example, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) , an organization of pediatric rheumatologists, researchers and research coordinators, was formed to find treatments for pediatric rheumatic diseases. The NIAMS has made awards to help CARRA develop its infrastructure, and has funded studies that leverage CARRA resources, such as the:
- Atherosclerosis Prevention in Pediatric Lupus Erythematosus (APPLE) trial, which tested the use of statins to prevent atherosclerosis in pediatric lupus patients;
- Trial of Early Aggressive Therapy (TREAT) in Juvenile Idiopathic Arthritis (JIA), which compared the effectiveness of an aggressive three-drug treatment regimen with a single drug in children with polyarticular JIA; and
- Rituximab in Myositis (RIM) trial, which assessed the efficacy of the drug rituximab to treat adult and pediatric patients with dermatomyositis.
These and other CARRA studies have provided critical information about the care of children with rheumatic diseases. More recently, CARRA has served as a model for the development of the Pediatric Dermatology Research Alliance (PeDRA) , a similar organization within the dermatology community that is focused on pediatric skin diseases.
The NIAMS has supported considerable research on genetic factors and molecular mechanisms involved in pediatric diseases within the Institute’s mission. New NIAMS and NIH initiatives to identify environmental drivers of disease will provide insights into etiology, and will offer exciting new opportunities to integrate genetic, biochemical, and environmental data to improve child health.
Stephen I. Katz, M.D., Ph.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases
National Institutes of Health