Congenital diaphragmatic hernia: The role of multi-institutional collaboration and patient registries in supporting best practice

https://doi.org/10.1053/j.sempedsurg.2017.04.004Get rights and content

Abstract

Among congenital malformations, congenital diaphragmatic hernia (CDH) is distinguished by its relatively low occurrence rate, need for resource intensive, integrated multidisciplinary care, and widespread variation in practice and outcome. Although randomized controlled trials (RCTs) are considered the gold standard for generating evidence, they are poorly suited to the study of a condition like CDH due to challenges in illness severity adjustment, unpredictability in clinical course and the impact limitations of studying a single intervention at a time. An alternative to RCTs for comparative effectiveness research for CDH is the patient registry, which aggregates multi-institutional condition-specific patient level data into a large CDH-specific database for the dual purposes of collaborative research and quality improvement across participating sites. This article discusses patient registries from the perspective of structure, data collection and management, and privacy protection that guide the use of registry data to support collaborative, multidisciplinary research. Two CDH-specific registries are described as illustrative examples of the “value proposition” of registries in improving the evidence basis for best practices for CDH.

Section snippets

What are patient registries?

A patient registry is a collection of standardized information about a group of patients who share a condition or experience that serves a predetermined scientific, clinical, or policy purpose.2 The use of the word “patient” signifies that the focus of the data is on health information and health care delivery. Other terms such as clinical registries, clinical data registries, disease registries, and outcomes registries are also used. A key attribute of patient registries is the encouragement

Registries and comparative effectiveness research

The gold standard for generating high-quality evidence to inform best practice is the randomized controlled trial (RCT). However, the challenges of large sample sizes, extreme costs, the need for highly controlled and often exclusionary enrollment criteria, and the lack of infrastructure and expertise required to conduct trials in the context of clinical care all limit the value of the RCT as the primary evidence source for many conditions. For a complex condition like CDH, which requires

The value proposition of patient registries for studying CDH

Congenital diaphragmatic hernia (CDH) is ideally suited to study using patient registries. It is a rare congenital malformation requiring complex care across multiple specialties within specialized children׳s hospitals. There is a recognized variation in illness severity that is usually diagnosed before birth, which allows caregivers and families some opportunity for prenatal planning. And while there have been significant improvements in survival from 50% to nearly 80% over the past 3 decades,

The Canadian Pediatric Surgery Network (CAPSNet)

CAPSNet was established in 2005 by a group of Canadian pediatric surgeons working collaboratively with counterparts in neonatology, maternal fetal medicine and medical genetics. At that time, there was an integrated national effort within the perinatal research community to build upon the success of the Canadian Neonatal Network (CNN) established 10 years earlier, which led to CAPSNet, the Canadian Perinatal Network (CPN-2006), Canadian Neonatal Followup Network (CNFUN-2008), and the Canadian

Congenital Diaphragmatic Hernia Study Group (CDHSG)

The Congenital Diaphragmatic Hernia Study Group (CDHSG) is a voluntary consortium of centers with an interest in treating infants with congenital diaphragmatic hernia (CDH). The CDHSG was established in 1991 at a meeting of ELSO (Extracorporeal Life Support Organization), where a group of pediatric surgeons, pediatric intensivists and neonatologists interested in CDH pledged “a willingness to work together and attempt to put aside previous biases and large egos to collectively address CDH

Future directions

Patient registries for CDH have much to offer as sources of high volume, high-quality patient level data that supports comparative effectiveness research and creates evidence that can be used in support of best practices. Registries also strengthen collaboration between institutions and clinical disciplines, and can provide opportunities for patient and family engagement leading to greater alignment and integration of efforts to improve care for this complex birth defect. Future opportunities

References (51)

  • K. Tsao et al.

    Congenital diaphragmatic hernia in the preterm infant

    Surgery

    (2010)
  • K.P. Lally et al.

    Corticosteroids for fetuses with congenital diaphragmatic hernia: can we show benefit?

    J Pediatr Surg

    (2006)
  • C.E. Colby et al.

    Surfactant replacement therapy on ECMO does not improve outcome in neonates with congenital diaphragmatic hernia

    J Pediatr Surg

    (2004)
  • K. Van Meurs

    Is surfactant therapy beneficial in the treatment of the term newborn infant with congenital diaphragmatic hernia?

    J Pediatr

    (2004)
  • L.E. Hollinger et al.

    A risk-stratified analysis of delayed congenital diaphragmatic hernia repair: does timing of operation matter?

    Surgery

    (2014)
  • K. Tsao et al.

    Minimally invasive repair of congenital diaphragmatic hernia

    J Pediatr Surg

    (2011)
  • R. Seetharamaiah et al.

    Factors associated with survival in infants with congenital diaphragmatic hernia requiring extracorporeal membrane oxygenation: a report from the Congenital Diaphragmatic Hernia Study Group

    J Pediatr Surg

    (2009)
  • R. Gliklich et al.

    Registries for Evaluating Patient Outcomes: A User׳s Guide

    (2010)
  • American College of Surgeons. SCR Training and Resources....
  • Rahm E, Do HH. Data Cleaning: Problems and Current...
  • US Department of Health and Human Services. The HIPPA privacy rule....
  • T.A. Workman

    Engaging Patients in Information Sharing and Data Collection: The Role of Patient-Powered Registries and Research Networks

    (2013)
  • O. Frobert et al.

    Thrombus aspiration during ST-segment elevation myocardial infarction

    N Engl J Med

    (2013)
  • M.S. Lauer et al.

    The randomized registry trial—the next disruptive technology in clinical research?

    N Engl J Med

    (2013)
  • Cited by (20)

    • Long term outcome of babies with pulmonary hypertension

      2022, Seminars in Fetal and Neonatal Medicine
      Citation Excerpt :

      Despite the advances in neonatal intensive care, survival rates for those requiring ECMO remain approximately 50–60%. The long term outcome of CDH is strongly influenced by comorbid factors (e.g. genetic disease or associated congenital cardiac lesions (Table 4), and is also closely related to ECMO and its complications [62,64,65]. Non-specific pulmonary sequelae can be seen later in childhood which demonstrated lower peak expiratory flow and a degree VQ mismatch [66].

    • Down Syndrome and Postoperative Complications in Children Undergoing Intestinal Operations

      2019, Journal of Pediatric Surgery
      Citation Excerpt :

      Those same opinions, especially in fields with high risk patients and low case volumes, like pediatric surgery, however, that have historically hampered the acceptance of change from such trials and analyses [40]. While this has been countered by enhanced utilization of large, national databases, a substantial amount of work is needed to continue progress at the institutional and multi-institutional collaborative level [41–43]. The use of the KID presents a few major limitations to this study.

    • Early Postnatal Ventricular Dysfunction Is Associated with Disease Severity in Patients with Congenital Diaphragmatic Hernia

      2018, Journal of Pediatrics
      Citation Excerpt :

      This in turn may lead to a vicious cycle of worsening cardiac function and pulmonary vasoconstriction. Further investigation of the relationship between early cardiac function and outcome in CDH in larger cohorts, such as CDH registries, is now warranted.36 In this study, early RV function did not correlate with clinical outcomes, in contrast to the findings of a previous investigation.29

    View all citing articles on Scopus
    View full text