Major achievements in pediatric research, often taken for granted, have been made possible with federal funding. A recent congressional briefing hosted by the AAP Committee on Pediatric Research highlighted discoveries from the last 40 years from the perspectives of researchers and families.
From passenger safety laws to the use of surfactant to rotavirus vaccines, pediatric research innovations over the last four decades have led to life-saving discoveries and policy changes that many take for granted.
Despite the importance of these advancements, researchers don’t always do a good job explaining the impact of their work and how it saves lives, said Tina Cheng, M.D., M.P.H., FAAP, chair of the AAP Committee on Pediatric Research (COPR).
That’s one reason the committee hosted a congressional briefing in December to present to lawmakers “7 Great Achievements in Pediatric Research” (see sidebar). Researchers and family members impacted by the research spoke to a standing-room-only crowd, focusing on innovations in the members’ lifetimes.
The topics covered immunizations, pediatric cancer, saving premature infants, preventing HIV transmission from mothers to babies, reducing sudden infant death syndrome (SIDS), increasing life expectancy for children with chronic diseases, and saving lives with car seats and seat belts. Forty years ago some of these discoveries may have seemed like science fiction.
To help select the topics, COPR surveyed its members along with the boards of the American Pediatric Society, Academic Pediatric Association, Society for Pediatric Research, Federation of Pediatric Organizations and Association of Medical School Pediatric Department Chairs. The groups, along with the Academy, sponsored the briefing.
“We chose the seven because we felt like they were recent successes and they were successes that would resonate with the public,” said Dr. Cheng, who moderated the briefing.
SPEAKING FROM THE HEART
Researchers shared their perspectives, but when family members and young people offered their stories about how research changed their lives, it was even more powerful, said neonatologist Scott Denne, M.D., FAAP, past chair of COPR.
Tokunbo Olaniyan, of Columbia, Md., a young woman whose late mother had sickle cell disease, talked about how grateful she was that her mother lived to be old enough to give birth. Forty years ago, sickle cell patients in the United States typically lived to only about 14 years.
College student Vikram Siberry, of Olney, Md., told how a seatbelt saved him during a car accident in high school that took the life of his friend who was behind the wheel.
Dr. Denne also shared how the introduction of surfactant to treat premature babies has affected his professional life.
“The difference is as night-and-day as any intervention has ever been,” he said.
“Before surfactant, our primary tools were the ventilator, and premature babies were born and immediately struggled to breathe,” he told the group. “The ventilator caused substantial damage — major ruptures of the lung — so you had to put in chest tubes. Babies needed to stay on ventilators for prolonged periods. Many babies who left the nursery had significant lung disease, and many babies simply didn’t survive.
“A daily event was babies dying … multiple chest tubes being placed … a whole host of rooms dedicated for babies who were going to be on ventilators for months. That was the reality before surfactant,” Dr. Denne said.
Today, many babies come off ventilators more quickly, lung damage is significantly less severe and survival rates have increased substantially, he noted.
SIDS was addressed by Marian Willinger, Ph.D., director of the research program in SIDS at the Eunice Kennedy Shriver National Institute for Child Health and Human Development. Dr. Willinger, consultant to the AAP Task Force on SIDS, coordinated much of the research efforts on the Back to Sleep campaign. Since 1994, the overall U.S. SIDS rate has declined by more than half as a result of babies being placed on their backs to sleep.
The briefing included graphs and charts on topics such as the progress made in life expectancy for patients with sickle cell anemia and cystic fibrosis. One chart showed the steep drop in perinatally acquired AIDS in the early 1990s with the introduction of an antiretroviral medication.
IMPORTANCE OF FUNDING
All of the stories were designed to help lawmakers and others understand the ongoing need for federal research funding.
“It’s very important for the general public to understand how impactful investing in research can and has been,” said Dr. Denne, who said funding should be maintained or better yet, increased.
Although the seven achievements will be no surprise to any pediatrician, Dr. Cheng said they all are taken for granted sometimes and continued investment is needed.
“All of these discoveries were the result of research funding innovation that led to decreased mortality, increased life expectancy, increased quality of life. There are more research discoveries to be made.”
7 Great Achievements in Pediatric Research in the Past 40 Years
Preventing disease with life-saving immunizations
Diseases like rotavirus and Haemophilus influenzae type b are now preventable due to vaccines.
Saving premature babies by helping them breathe
Deaths from respiratory distress syndrome have been reduced by two-thirds with the introduction of surfactant.
Reducing sudden infant death syndrome (SIDS) with Back to Sleep
SIDS has declined by half due to research and the Back to Sleep campaign.
Curing a common childhood cancer
More than 90% of children with acute lymphocytic leukemia now survive, compared with 57% in the 1970s.
Preventing human immunodeficiency virus (HIV) transmission from mother to baby
Twenty years ago, one in four mothers with HIV transmitted HIV to their babies. Now the transmission rate is less than 2% due to advances in medications given during pregnancy.
Increasing life expectancy for children with chronic diseases
Life expectancy of patients with sickle cell disease or cystic fibrosis has risen from 14 years to more than 40 years.
Saving lives with car seats and seat belts
Research leading to vehicle safety laws has significantly reduced pediatric motor vehicle deaths.
AAP评出40年儿科研究七大成就：1.疫苗接种预防疾病。2.表面活性剂助早产儿呼吸。3.仰卧降低婴儿猝死综合征。4.治愈一种儿童常见癌症（急淋白血病存活率从70年代的57%上升到90%）5.阻断HIV母婴传播（降低至<2%）6.提高慢性疾病儿童预期寿命。7.安全座椅和安全带救了很多孩子性命。
中文翻译：
本文地址：http://www.wjbb.com/know/1049
原文出处：http://aapnews.aappublications.org/content/36/5/11.full
