Preface

Children’s hospitals were first developed during the 19th century, typically as local altruistic efforts driven by women who sought to improve the health and welfare of children in their communities. Both in the United States and elsewhere, these were most often located in urban centers which developed in the course of the societal shift from an agrarian to a mercantile and industrial base. These were commonly focused on the care of poor children, and the effort was to provide a compassionate and protected environment, often beginning simply in a home. Quality of care was assessed only subjectively; indeed, “quality” and “outcomes assessment” as we use these terms now were not introduced until Codman’s work in the early 20th century. During the latter 20th century, specialization of facilities, design of systems of care, and training of specialized professionals fully committed to the care of ill children all transformed these early children’s hospitals. The effort became one of providing specific expertise to young patients regardless of socioeconomic status. It was, however, only in the last few years of the last century that focused and rigorous outcomes analysis of clinical care began to emerge as a discipline with regard to children’s care, and in particular children’s surgery. Even now, in the first decade of the 21st century, it is distressingly frequent that we are unable to answer seemingly simple questions related to the surgical care of children using definitive data rather than depending on anecdote, personal experience, or tradition. It is clear now that patients, families, other professionals, and various administrative managers and policy makers are actively seeking evermore robust information that relates to the outcomes for various types of care provided to sick or injured children. Furthermore, it is apparent that there is significant variation in the types and quality of care provided to similar patients across the country and around the world. The need to assess outcomes has never been greater in children’s surgical care.

The compilation of manuscripts in this issue of Seminars in Pediatric Surgery is designed to be illuminating and contemporary with regard to several important and common problems within the scope of modern pediatric surgery. The discussions are intended to be illustrative rather than encyclopedic, and to emphasize various analytical approaches. The authors have been pioneers in the use of various forms of data collection and analysis. It is apparent that many of the pathologic conditions that pediatric surgeons deal with are relatively uncommon. Therefore, most of the authors emphasize cooperative efforts and sharing of data. Cooperative work involving the study of children’s oncologic problems offered a notable early example of this approach. The sequential multicenter trials that defined the management of Wilms’ and other tumors have yielded substantial and progressive improvements in care over a period of decades. The resulting oncology protocols for current treatment have indeed become a defacto standard of care. The focus in these selected reviews is in areas where there are recent and important changes, with lessons to be learned. I believe these are not quite so well known as the classic oncology work.

To begin, Dr. Calkins provides a comprehensive and critical summary of specific pediatric outcomes tools. These include some of the most common and widely used tools which have been developed and validated for children with regard to evaluation of quality of life, functional outcomes, and cost effectiveness. Each of these areas of inquiry represents an opportunity for us in pediatric surgery to define outcomes for the surgical care that we provide, going well beyond the traditional surgical view of morbidity and mortality. The information available from these types of analyses increasingly drives public policy and patient/family decisions. In a time of increasing external scrutiny, it behooves every pediatric surgeon to be well informed with regard to this vocabulary and methodology. Professor Rintala reviews his personal and institutional experience with imperforate anus, using tools developed specifically to quantify and compare individuals following anorectal reconstructive surgery. Every pediatric surgery surgeon is aware that subjective and inconsistent assessment of outcomes after surgery to correct anorectal malformations has made it very difficult to interpret published outcomes comparing different procedures, institutions, and approaches. Professor Rintala offers a model of how this type of analysis can be approached objectively and using large databases, both retrospective and prospective. Dr. Kevin Lally addresses a number of problems and questions related to congenital diaphragmatic hernia, demonstrating the extraordinary value of a large voluntary registry, with investigators from across many participating institutions. Given the relative infrequency of this anomaly and the heterogenous patient population, the ability to collect a large cohort of patients, define appropriate risk adjustment, and standardize data reporting is key to being able to address certain questions. Dr. Larry Moss provides a review of recent clinical research regarding neonatal necrotizing enterocolitis (NEC). He has made a major personal contribution to this work, most recently as the principal investigator of a prospective randomized trial examining two different surgical management strategies: primary laparotomy versus peritoneal drainage for perforated NEC. Whereas that specific outcomes analysis is valuable and is detailed in his discussion, the work is perhaps most notable in that it serves as a model of how certain difficult clinical problems may benefit from rigorous, prospective analysis. The cost and complexity of a randomized clinical trial are not to be underestimated, but the lessons learned here are noteworthy, both with regard to NEC itself and with regard to clinical outcomes research. Dr. Stylianos summarizes certain aspects of pediatric trauma care, in particular solid visceral injury, using data drawn from very large, public, administrative databases, including both federal and state hospital discharge data sets. Although there are substantial limitations to the use of this type of administrative data, notably appropriate risk adjustment, it is clear that the sample size obtainable with this approach offers extraordinary potential in the analysis of individual patient care as well as for systems of care. Dr. Mark Stringer reviews the experience with the management of biliary atresia in the UK over the last two decades. He offers data contrasting the UK with national systems elsewhere, demonstrating that planned regionalization of highly specialized care for biliary atresia surgery can yield demonstrably superior clinical results. In the case of the UK, data-driven public policy decisions by the National Health Service led to regionalization of care for biliary atresia in three centers in the UK, to the exclusion of all other centers. Although this has been a point of controversy and concern in the professional community, there are lessons to be learned from these data that every pediatric surgeon should consider. Dr. Fred Ryckman offers insight into the pediatric liver transplantation experience, making extensive use of federally mandated and other public data sources. Transplantation is an area where payors and regulatory groups have systematically used these data for years to analyze results, to drive quality improvement, and indeed to mandate regionalization of services and care. Dr. Peter Dillon provides a glimpse of the future as he describes a new partnership between the American College of Surgeons National Surgical Quality Improvement Program and the pediatric surgery community. This (NSQIP) program was initially developed in Veterans Administration hospitals in the mid 1990s, but has been validated and transferred to academic and community hospitals subsequent. He describes the process and a children’s surgical tool which has been developed now to allow assessment of children’s surgical care in various institutions, with risk adjustment and meaningful comparison between institutions, using medically relevant data points rather than administrative data. Lastly, Dr. George Hoffman, a senior and thoughtful pediatric anesthesiologist, offers a perspective which I believe most children’s surgeons will find illuminating. He provides a state of the art, data driven analysis of the value of pediatric-specific anesthesia training and practice experience. It is consistent with a bias that most pediatric surgeons share, I believe, but it is an objective and rigorous examination of the data which support the conclusion that children with surgical problems are best served by anesthesia providers with specialized children’s training and experience.

To summarize, I believe that this compilation of discussions offers a thoughtful, rigorous, and contemporary look at evidence-based outcomes assessment as it relates to certain areas of pediatric surgical practice at the outset of the 21st century. I hope that practicing pediatric surgeons will find this of interest and of value in their work.