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Editorial |

Searching for Effective Treatment

Frederick P. Rivara, MD, MPH
Arch Pediatr Adolesc Med. 2011;165(12):1137-1138. doi:10.1001/archpediatrics.2011.194.
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Bronchiolitis is one of the most common causes of hospitalization for children during the first year of life.1 I often hear the statement, usually said in jest by hospital administrators, that if it were not for bronchiolitis, we would not need about 25% of the beds in our children's hospitals during the winter months.

In this issue of Archives, there is yet another article on the treatment of acute bronchiolitis, this time using helium-oxygen mixtures.2 It is a good article, with strong methods and positive results, and we are proud to publish it. However, the Archives of Pediatrics & Adolescent Medicine has published many such articles on the treatment of bronchiolitis and respiratory syncytial virus infections (eg, use of inhaled nitric oxide,3 3% hypertonic saline with epinephrine,4 and epinephrine vs albuterol5) and reviews on the diagnosis6 and treatment7 of this disorder. Usually when there are so many different treatments suggested for a disorder, none is entirely efficacious, which accounts for the frequent hospitalizations and sometimes long lengths of hospital stay for children with these infections. This is certainly the case for respiratory syncytial virus infections and bronchiolitis.

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Reply to Dr. Rivara's editorial: Search for Effective Treatment
Posted on December 13, 2011
In K. Kim, MD, MBA
University of Louisville
Conflict of Interest: None Declared
To the Editor-in-Chief: We are very grateful for the comments about our methods and manuscript by Dr. Rivara. In addition, we echo the insights expressed by his editorial. We agree that funding is, perhaps, the most significant barrier to national multi-center trials. Our preliminary cost estimate is ~$2.5 million for a helium-oxygen bronchiolitis study with a sample size similar to adult multi-center trials where there are several hundred patients per group. The $500,000 maximum for R01 funding may create a financial ceiling that operationally inhibits multi-center trials.
In the context of the present economic downturn and public budget battles, it would be hopeful and unlikely that increased funding would come from national or state government agencies. The pressing realities of unemployment and economic survival may make long term visions of national research potentially unrealistic in a near time horizon.
Alternatively, we could turn to new approaches to funding national multi-center trials. Private philanthropy has served to jump start innovation in multiple sectors. For example, in 1927, the $25,000 Orteig Prize (Present 2011 value, ~$323,000) helped Charles Lindbergh to create a business strategy for the first trans-Atlantic flight. Similarly, in 2010, the $10 million Ansari X prize for a reusable space orbiter was a financial catalyst for this achievement.
One possible solution is a $10 million award targeting multi-center trials. Perhaps an award every 2 years for five cycles could provide the framework for addressing pressing pediatric questions. Although the total $50 million investment may appear daunting in scope, the reality of similar sized funding ($15-21 million over 5-7 years) already exists for select principal investigators by our adult medicine and basic science colleagues.
Fortunately, in pediatric emergency medicine and pediatric critical care, we have the multi-center national research infrastructure to address these large questions with several national collaborations (PECARN: Pediatric Emergency Care Applied Research Network; PEMCRC: Pediatric Emergency Medicine Collaborative Research Center; PERC: Pediatric Emergency Research Canada; CPCCRN: Collaborative Pediatric Critical Care Research Network;) as well as several regional collaborations.
We are optimistic that ambitious creative pediatric researchers could utilize the $10 million in funding to address significant and important clinical questions that could only be answered though multi-center trials. These researchers could create outstanding value for this significant investment. One could even imagine funding 5-7 pediatric sub-specialties to create national infrastructure with one cycle of funding, if not already in place. Multiple private philanthropic foundations exist and one may have the opportunistic foresight to see that the time may be ripe to fund multi-center national pediatric trials.
Thank you.
In K. Kim, MD, MBA Kosair Children’s Hospital Associate Professor Division of Pediatric Emergency Medicine University of Louisville

Conflict of Interest: Disclosure: Dr. Kim is not a member of PECARN or PEMCRC. His division does participate in PEMCRC multi-center trials.
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