Commentary
The Canadian Pediatric Surgery Network Congenital Diaphragmatic Hernia Evidence Review Project: Developing national guidelines for care Pramod S Puligandla MD MSc1, Erik D Skarsgard MD2 PS Puligandla, ED Skarsgard. The Canadian Pediatric Surgery Network Congenital Diaphragmatic Hernia Evidence Review Project: Developing national guidelines for care. Paediatr Child Health 2016;21(4):183-186. The Canadian Pediatric Surgery Network (CAPSNet) has been collecting population-based data regarding congenital diaphragmatic hernia (CDH) across its 17 perinatal sites since 2005. With >500 infants registered to date, CAPSNet has addressed many critical knowledge gaps pertaining to CDH care. Most importantly, it has identified variability in both CDH practice and outcome across Canada. Using the successful Evidence-based Practice for Improving Quality (EPIQ) method, CAPSNet is undertaking a national, multidisciplinary effort to standardize best practices for CDH, from prenatal diagnosis to hospital discharge, based on the best available evidence. The present article outlines the value of clinical research networks and the process CAPSNet will undertake to produce national consensus guidelines for CDH care. Key Words: Best practice guidelines; Canadian Pediatric Surgery Network;
Le projet d’analyse des données probantes sur les hernies diaphragmatiques congénitales du Réseau canadien de chirurgie pédiatrique : l’élaboration de lignes directrices nationales sur les soins Le Réseau canadien de chirurgie pédiatrique recueille des données en population sur la hernie diaphragmatique congénitale (HDC) dans 17 sites périnatals depuis 2005. Puisque plus de 500 nourrissons y sont inscrits jusqu’à présent, le Réseau a corrigé de nombreuses lacunes liées aux soins de la HDC. Qui plus est, il a décelé la variabilité dans la pratique et les résultats de la HDC au Canada. Selon la méthode EPIC (un acronyme anglais qui signifie pratique fondée sur des données probantes pour améliorer la qualité), le Réseau déploie des efforts nationaux et multidisciplinaires pour normaliser les pratiques exemplaires en matière de soins de la HDC, du diagnostic prénatal au congé de l’hôpital, d’après les meilleures données probantes. Le présent article souligne la valeur des réseaux de recherche clinique et le processus que le Réseau entreprendra pour produire des lignes directrices consensuelles nationales sur les soins de la HDC.
Clinical research networks; Congenital diaphragmatic hernia; EPIQ
I
n North America, structural birth defects complicate 3% to 6% of pregnancies leading to live birth, and are second only to prematurity as a cause of infant mortality (1). Of the structural birth defects associated with significant mortality, congenital diaphragmatic hernia (CDH) is among one of the more common anomalies, occurring at a frequency of one per 2200 live births (2). Although the anatomical consequences of CDH (hole in the diaphragm of the fetus, resulting in herniation of abdominal viscera into the chest) are quite simple, the degree of physiological derangement caused by the resulting pulmonary hypoplasia and pulmonary hypertension in newborns with CDH is difficult to predict. This limits our ability to accurately counsel expectant parents on what treatment measures may be required and what outcomes to expect. CDH babies and their families require complex, interdisciplinary care from a variety of paediatric subspecialties (including neonatology, cardiology, surgery and anesthesia), nursing and allied health services. Their neonatal intensive care unit (NICU) stays are arduous, resource intensive and prolonged, and may include advanced modes of cardiorespiratory support such as extracorporeal membrane oxygenation. In fact, a pregnancy with a known diagnosis of CDH triggers a cascade of resource consumption across perinatal health services, including advanced prenatal diagnosis with fetal imaging and genetic testing, as well as planned delivery in obstetrical units linked to tertiary/quaternary
NICUs where postnatal care is provided. As recently as 25 years ago, overall survival rates for CDH were approximately 50%. Since then, survival rates have risen to 75% to 80%, primarily through improvements in newborn care (3). However, the consequence of survival of the sickest babies has transferred much of the health care burden from the acute hospitalization to health services provided by community paediatricians and family physicians, subspecialty paediatricians and, in some centres, children’s hospital-based multispecialty CDH clinics. Long term follow-up of CDH reveals patterns of survival that resemble childhood chronic disease, with the observation of significant disability in somatic growth, pulmonary health and neuromotor development that lasts through childhood into young adulthood (4,5). As an illustration, of the 90 CDH infants born each year across 17 perinatal centres in Canada (3): • 22 babies will die in the NICU despite multispecialty ‘state of the art’ neonatal intensive care. • 68 babies will survive to hospital NICU discharge, of which: ○ 55 will have active, ongoing cardiorespiratory, feeding and/or motor problems that require specialized follow-up and treatment; ○ 30 will have detectable psychomotor or neurodevelopmental disability by one year of age. • 48 babies will have detectable disability by three years of age (6).
1Divisions
of Pediatric General and Thoracic Surgery, Montreal Children’s Hospital, Montreal, Quebec; 2Divisions of Pediatric General and Thoracic Surgery, British Columbia Children’s Hospital, Vancouver, British Columbia Correspondence: Dr Pramod S Puligandla, The Montreal Children’s Hospital of the McGill University Health Centre, Divisions of Pediatric General and Thoracic Surgery, 1001 Decarie Boulevard, Room B04.2318, Montreal, Quebec H4A 3J1. Telephone 514-412-4438, fax 514-412-4289, e-mail [email protected] Accepted for publication January 25, 2016
Paediatr Child Health Vol 21 No 4 May 2016
©2016 Pulsus Group Inc. All rights reserved
183
Commentary
1. Victoria General Hospital, Victoria, British Columbia 2. BC Children’s Hospital, Vancouver, British Columbia 3. Alberta Children’s Hospital, Calgary, Alberta 4. University of Alberta Hospital, Edmonton, Alberta 5. Royal University Hospital, Saskatoon, Saskatchewan 6. Winnipeg Health Sciences Centre, Winnipeg, Manitoba 7. London Health Sciences Centre, London, Ontario 8. McMaster Children’s Hospital, Hamilton, Ontario 9. The Hospital for Sick Children, Toronto, Ontario 10. Kingston General Hospital, Kingston, Ontario 11. Children’s Hospital of Eastern Ontario, Ottawa, Ontario 12. Montreal Children’s Hospital, Montreal, Quebec 13. Hôpital Ste-Justine, Montreal, Quebec 14. Centre Hospitalier Universitaire Sherbrooke, Quebec
de
Sherbrooke,
15. Centre Hospitalier de L’Universitaire Laval, Quebec City, Quebec 16. IWK Health Centre, Halifax, Nova Scotia 17. Janeway Children’s Health and Rehabilitation Centre, St John’s Newfoundland & Labrador
Figure 1) Canadian Pediatric Surgery Network sitemap for participating centres
CDH treatment consumes a disproportionate share of health care resources compared with other conditions affecting term infants who require complex, multidisciplinary neonatal intensive care. For example, the cost of care for CDH infants in one Canadian hospital was four times greater than that of a gestational age- and illness severity-matched neonatal cohort. Furthermore, the extrapolated annual cost of hospital care for CDH from birth to hospital discharge in Canada approaches $10 million (7). Similarly, in the United States, the annual birth hospitalization cost of CDH care is estimated to be $250 million (8). Presently, there are no estimates of the health care costs associated with CDH survival beyond hospital discharge. Given the rates and severity of survival morbidity, and the inevitable impact on patient and family quality of life, it is certain that the direct and indirect economic impact on the patient, the family and society are substantial and likely exceed those of most birth defects. Thus, CDH presents an exceptional opportunity for care improvement leading to optimized outcome(s) that will benefit not only the child and family, but also our health system.
Barriers to Birth Defect Outcome Improvement
Outcome improvements for rare birth defects, such as CDH, face several challenges. First, given the rarity of the condition, the number of cases treated by individual hospitals is small and, therefore, many hospitals are limited in their ability to acquire experience and expertise. Second, the available literature from which evidence informing ‘best practices’ can be drawn is often from small, retrospective single-centre studies. To achieve sufficient numbers of cases, these observational studies are conducted over many years and, therefore, do not always reflect contemporary care, which severely limits their relevance to our current patients. Finally, many of these studies lack standardized definitions and risk adjustment, which limits the broad application of their findings. In fact, the lack of validated risk-adjustment tools and standardized variable (risk, treatment and outcome) definitions for CDH make 184
comparisons of therapeutic approaches and outcomes between individual studies difficult – if not impossible – to interpret.
The Role of Patient Registries/Clinical Research Networks
Clinical research networks (CRNs) are groups of clinicians and researchers with a specific clinical research focus and an overall goal to improve clinical outcome for their condition of interest. CRNs may be based on specific disease entities, research populations, treatment modalities, specialty groups or research methodology. CRNs should engage several stakeholders including hospital leaders and decision makers, professional specialty societies, policy makers and, especially, patients. Of fundamental importance to a CRN is a patient registry: a database with standardized definitions that collects observational patient-level data regarding risk factors, treatment details and outcomes of interest. Most importantly, data collection within the network is carefully selected and strategically oriented toward its end users. The value of registry data for the observational study of a rare condition, such as CDH, is the circumvention of small case numbers and the ability to conduct large scale, risk-adjusted studies that are the ‘next best’ alternative to randomized controlled trials. Canada has been a global leader in the use of CRNs to improve perinatal care. The Canadian Neonatal Network (CNN), founded by Dr Shoo Lee, maintains a standardized NICU database and provides a unique opportunity for researchers to participate in clinical, epidemiological outcomes, health services, health policy and informatics research aimed at improving the efficacy and efficiency of neonatal care. Building on the successes of CNN, the Canadian Pediatric Surgery Network (CAPSNet), funded by the Canadian Institutes of Health Research since 2006, is a multidisciplinary group of Canadian investigators whose research efforts focus on CDH and gastroschisis. The network comprises 17 perinatal centres across Canada (Figure 1). CAPSNet maintains an integrated network and shared data platform with CNN and a third perinatal CRN, the Canadian Neonatal Follow-up Network. Paediatr Child Health Vol 21 No 4 May 2016
Commentary
Table 1 Canadian Pediatric Surgery Network (CAPSNet) contributions toward national standards for congenital diaphragmatic hernia (CDH) care in Canada Outcome prediction
Risk adjustment
Practice variation
Outcome variation
Prenatal ultrasound measurements (9)
Termination rates (13)
Prenatal
Ultrasound indices (13) Delivery location (14)
Postnatal
Ventilatory protocols
Validation of SNAP-II (17) Ventilatory modes
Blood gas targets (16)
Mortality models (18)
Gestational timing of delivery (14,15)
Use of pulmonary vasodilators ECMO
Survival complications (19,20) Disability at discharge Long-term disability
Surgical
Side of defect (21)
Need for patch repair (22)
Timing and type of surgery
Institutional case volume (23)
Current CAPSNet contributions are indicated by references in parentheses. Bolded items represent topics to be covered in the CAPSNet CDH Evidence-based Practice for Improving Quality (EPIQ) review project. ECMO Extracorporeal membrane oxygenation; SNAP-II Score for neonatal acute physiology II
CAPSNet Knowledge Synthesis
Since its inception, CAPSNet has been able to address key areas of knowledge deficit, with >30 peer-reviewed articles and 50 presentations at national and international meetings. Contributions to knowledge deficit have created an evidence base for practice change in the prenatal, neonatal and surgical domains of care (Table 1).
Bridging the Gap between Knowledge Synthesis and Practice Change
The term ‘knowledge mobilization’ captures the active process that is required after knowledge synthesis, in which evidence is used to drive and maintain best practices. Conditions for which there is variation in both the treatment provided and the outcomes observed stand to benefit the most from practice standardization. CAPSNet has previously shown substantial practice variation in all care domains and in the outcomes of CDH infants across Canada (9). In fact, mortality for CDH across Canada varies from 40% to 100%. These findings underscore the need to develop evidence-based guidelines for CDH.
What is Evidence-based Practice for Improving Quality?
Evidence-based Practice for Improving Quality (EPIQ) is a quality improvement method that uses published literature and quantitative institutional data to inform practice change. EPIQ enables collaborative, targeted intervention based on outcome variation identified from an analysis of institutional data. The advantages of the EPIQ method include its ability to address multifactorial interventions that may be applicable not only to local hospital settings but also more broadly to similar patient populations. An important advantage of EPIQ, compared with randomized control trials, is its ability to target individual behaviour and institutional culture. Furthermore, EPIQ can address both clinical and process outcomes.
The CDH EPIQ project
Following the success of the CNN EPIQ program, which led to dramatic outcome improvements in bronchopulmonary dysplasia and nosocomial infections within NICUs across Canada (10), CAPSNet has created multidisciplinary workgroups to tackle the controversies related to specific aspects of CDH care. With representatives from the disciplines of paediatric surgery, neonatology, paediatric intensive care, paediatric anesthesia and maternal-fetal medicine, the workgroups seek to develop a library for evidencebased best practices in CDH that cover the trajectory of care for Paediatr Child Health Vol 21 No 4 May 2016
most babies with CDH from birth to surgical repair to discharge. Specific areas of care include perinatal management, ventilation strategies, pulmonary hypertension management, the type, timing and indications for surgical repair, and indications for surfactant use, as well as surveillance protocols for disability and ethical considerations regarding palliation. The first step of the CAPSNet CDH EPIQ Project will be the dissemination of the best-practice guidelines that our working groups have assembled. While other consensus treatment guidelines for CDH are available in the literature (11), our assessment of the literature uses the Grading of Recommendations Assessment, Development, and Evaluation (GRADE), as described by Guyatt et al (12). While GRADE does not completely remove personal judgment, it does provide guideline developers with a comprehensive and transparent framework for developing recommendations regardless of whether the quality of evidence is high or low (12). Moreover, it takes into consideration all patient-important outcomes as part of guideline development.
References
1. Hobbs CA, Chowdhury S, Cleves MA, et al. Genetic epidemiology and nonsyndromic structural birth defects: From candidate genes to epigenetics. JAMA Pediatr 2014;168:371-7. 2. Colvin J, Bower C, Dickinson JE, Sokol J. Outcomes of congenital diaphragmatic hernia: A population-based study in Western Australia. Pediatrics 2005;116:e356-63. 3. CAPSNet Annual Report 2014. www.capsnet.org. (Accessed September 11, 2015). 4. Peetsold MG, Heij HA, Kneepkens CM, Nagelkerke AF, Huisman J, Gemke RJ. The long-term follow-up of patients with a congenital diaphragmatic hernia: A broad spectrum of morbidity. Pediatr Surg Int 2009;25:1-17. 5. Chiu PP, Ijsselstijn H. Morbidity and long-term follow-up in CDH patients. Eur J Pediatr Surg 2012;22:384-92. 6. Tracy S, Estroff J, Valim C, Friedman S, Chen C. Abnormal neuroimaging and neurodevelopmental findings in a cohort of antenatally diagnosed congenital diaphragmatic hernia survivors. J Pediatr Surg 2010;45:958-65. 7. Lam JC, Claydon J, Mitton CR, Skarsgard ED. A risk-adjusted study of outcome and resource utilization for congenital diaphragmatic hernia. J Pediatr Surg 2006;41:883-7. 8. Raval MV, Wang X, Reynolds M, Fischer AC. Costs of congenital diaphragmatic hernia repair in the United States-extracorporeal membrane oxygenation foots the bill. J Pediatr Surg 2011;46:617-24. 9. Baird R, Eeson G, Safavi A, Puligandla P, Laberge JM, Skarsgard ED. Institutional practice and outcome variation in the management of congenital diaphragmatic hernia and gastroschisis in Canada: A report from the Canadian Pediatric Surgery Network. J Pediatr Surg 2011;46:801-7.
185
Commentary 10. Lee SK, Aziz K, Singhal N, Cronin CM. The Evidence-based Practice for Improving Quality method has greater impact on improvement of outcomes than dissemination of practice change guidelines and quality improvement training in neonatal intensive care units. Paediatr Child Health 2015;20:e1-9. 11. Reiss I, Schaible T, van den Hout L, et al. Standardized postnatal management of infants with congenital diaphragmatic hernia in Europe: the CDH EURO Consortium consensus. Neonatology 2010;98:354-64. 12. Guyatt GH, Oxman AD, Schunemann HJ, Tugwell P, Knottnerus A. GRADE guidelines: A new series of articles in the Journal of Clinical Epidemiology. J Clin Epidemiol 2011;64:380-2. 13. Thomas SL, Baird R, Skarsgard E, Laberge J-M; CAPSNet. The factors associated with elective termination of pregnancy of fetuses with congenital diaphragmatic hernia. American Pediatric Surgical Congress, Fort Lauderdale, Florida, April 3 to May 3, 2015. 14. Safavi A, Lin Y, Skarsgard ED. Perinatal management of congenital diaphragmatic hernia: When and how should babies be delivered? Results from the Canadian Pediatric Surgery Network. J Pediatr Surg 2010;45:2334-9. 15. Mills JL, Lin Y, MacNab YC, Skarsgard ED. Does overnight birth influence treatment or outcome in congenital diaphragmatic hernia? Am J Perinatol 2010;27:91-5. 16. Brindle ME, Ma IW, Skarsgard ED. Impact of target blood gases on outcome in congenital diaphragmatic hernia (CDH). Eur J Pediatr Surg 2010;20:290-3.
186
17. Skarsgard ED, MacNab YC, Qiu Z, Little R, Lee SK. SNAP-II predicts mortality among infants with congenital diaphragmatic hernia. J Perinatol 2005;25:315-9. 18. Baird R, MacNab YC, Skarsgard ED. Mortality prediction in congenital diaphragmatic hernia. J Pediatr Surg 2008;43:783-7. 19. Mills J, Safavi A, Skarsgard ED. Chylothorax after congenital diaphragmatic hernia repair: A population-based study. J Pediatr Surg 2012;47:842-6. 20. Maxwell D, Baird R, Puligandla P. Abdominal wall closure in neonates after congenital diaphragmatic hernia repair. J Pediatr Surg 2013;48:930-4. 21. Beaumier CK, Beres AL, Puligandla PS, Skarsgard ED. Clinical characteristics and outcomes of patients with right congenital diaphragmatic hernia: A population-based study. J Pediatr Surg 2015;50:731-3. 22. Brindle ME, Brar M, Skarsgard ED. Patch repair is an independent predictor of morbidity and mortality in congenital diaphragmatic hernia. Pediatric surgery international 2011;27:969-74. 23. Grushka JR, Laberge JM, Puligandla P, Skarsgard ED. Effect of hospital case volume on outcome in congenital diaphragmatic hernia: The experience of the Canadian Pediatric Surgery Network. J Pediatr Surg 2009;44:873-6.
Paediatr Child Health Vol 21 No 4 May 2016
The Canadian Pediatric Surgery Network Congenital Diaphragmatic Hernia Evidence Review Project: Developing national guidelines for care Pramod S Puligandla MD MSc1, Erik D Skarsgard MD2 PS Puligandla, ED Skarsgard. The Canadian Pediatric Surgery Network Congenital Diaphragmatic Hernia Evidence Review Project: Developing national guidelines for care. Paediatr Child Health 2016;21(4):183-186. The Canadian Pediatric Surgery Network (CAPSNet) has been collecting population-based data regarding congenital diaphragmatic hernia (CDH) across its 17 perinatal sites since 2005. With >500 infants registered to date, CAPSNet has addressed many critical knowledge gaps pertaining to CDH care. Most importantly, it has identified variability in both CDH practice and outcome across Canada. Using the successful Evidence-based Practice for Improving Quality (EPIQ) method, CAPSNet is undertaking a national, multidisciplinary effort to standardize best practices for CDH, from prenatal diagnosis to hospital discharge, based on the best available evidence. The present article outlines the value of clinical research networks and the process CAPSNet will undertake to produce national consensus guidelines for CDH care. Key Words: Best practice guidelines; Canadian Pediatric Surgery Network;
Le projet d’analyse des données probantes sur les hernies diaphragmatiques congénitales du Réseau canadien de chirurgie pédiatrique : l’élaboration de lignes directrices nationales sur les soins Le Réseau canadien de chirurgie pédiatrique recueille des données en population sur la hernie diaphragmatique congénitale (HDC) dans 17 sites périnatals depuis 2005. Puisque plus de 500 nourrissons y sont inscrits jusqu’à présent, le Réseau a corrigé de nombreuses lacunes liées aux soins de la HDC. Qui plus est, il a décelé la variabilité dans la pratique et les résultats de la HDC au Canada. Selon la méthode EPIC (un acronyme anglais qui signifie pratique fondée sur des données probantes pour améliorer la qualité), le Réseau déploie des efforts nationaux et multidisciplinaires pour normaliser les pratiques exemplaires en matière de soins de la HDC, du diagnostic prénatal au congé de l’hôpital, d’après les meilleures données probantes. Le présent article souligne la valeur des réseaux de recherche clinique et le processus que le Réseau entreprendra pour produire des lignes directrices consensuelles nationales sur les soins de la HDC.
Clinical research networks; Congenital diaphragmatic hernia; EPIQ
I
n North America, structural birth defects complicate 3% to 6% of pregnancies leading to live birth, and are second only to prematurity as a cause of infant mortality (1). Of the structural birth defects associated with significant mortality, congenital diaphragmatic hernia (CDH) is among one of the more common anomalies, occurring at a frequency of one per 2200 live births (2). Although the anatomical consequences of CDH (hole in the diaphragm of the fetus, resulting in herniation of abdominal viscera into the chest) are quite simple, the degree of physiological derangement caused by the resulting pulmonary hypoplasia and pulmonary hypertension in newborns with CDH is difficult to predict. This limits our ability to accurately counsel expectant parents on what treatment measures may be required and what outcomes to expect. CDH babies and their families require complex, interdisciplinary care from a variety of paediatric subspecialties (including neonatology, cardiology, surgery and anesthesia), nursing and allied health services. Their neonatal intensive care unit (NICU) stays are arduous, resource intensive and prolonged, and may include advanced modes of cardiorespiratory support such as extracorporeal membrane oxygenation. In fact, a pregnancy with a known diagnosis of CDH triggers a cascade of resource consumption across perinatal health services, including advanced prenatal diagnosis with fetal imaging and genetic testing, as well as planned delivery in obstetrical units linked to tertiary/quaternary
NICUs where postnatal care is provided. As recently as 25 years ago, overall survival rates for CDH were approximately 50%. Since then, survival rates have risen to 75% to 80%, primarily through improvements in newborn care (3). However, the consequence of survival of the sickest babies has transferred much of the health care burden from the acute hospitalization to health services provided by community paediatricians and family physicians, subspecialty paediatricians and, in some centres, children’s hospital-based multispecialty CDH clinics. Long term follow-up of CDH reveals patterns of survival that resemble childhood chronic disease, with the observation of significant disability in somatic growth, pulmonary health and neuromotor development that lasts through childhood into young adulthood (4,5). As an illustration, of the 90 CDH infants born each year across 17 perinatal centres in Canada (3): • 22 babies will die in the NICU despite multispecialty ‘state of the art’ neonatal intensive care. • 68 babies will survive to hospital NICU discharge, of which: ○ 55 will have active, ongoing cardiorespiratory, feeding and/or motor problems that require specialized follow-up and treatment; ○ 30 will have detectable psychomotor or neurodevelopmental disability by one year of age. • 48 babies will have detectable disability by three years of age (6).
1Divisions
of Pediatric General and Thoracic Surgery, Montreal Children’s Hospital, Montreal, Quebec; 2Divisions of Pediatric General and Thoracic Surgery, British Columbia Children’s Hospital, Vancouver, British Columbia Correspondence: Dr Pramod S Puligandla, The Montreal Children’s Hospital of the McGill University Health Centre, Divisions of Pediatric General and Thoracic Surgery, 1001 Decarie Boulevard, Room B04.2318, Montreal, Quebec H4A 3J1. Telephone 514-412-4438, fax 514-412-4289, e-mail [email protected] Accepted for publication January 25, 2016
Paediatr Child Health Vol 21 No 4 May 2016
©2016 Pulsus Group Inc. All rights reserved
183
Commentary
1. Victoria General Hospital, Victoria, British Columbia 2. BC Children’s Hospital, Vancouver, British Columbia 3. Alberta Children’s Hospital, Calgary, Alberta 4. University of Alberta Hospital, Edmonton, Alberta 5. Royal University Hospital, Saskatoon, Saskatchewan 6. Winnipeg Health Sciences Centre, Winnipeg, Manitoba 7. London Health Sciences Centre, London, Ontario 8. McMaster Children’s Hospital, Hamilton, Ontario 9. The Hospital for Sick Children, Toronto, Ontario 10. Kingston General Hospital, Kingston, Ontario 11. Children’s Hospital of Eastern Ontario, Ottawa, Ontario 12. Montreal Children’s Hospital, Montreal, Quebec 13. Hôpital Ste-Justine, Montreal, Quebec 14. Centre Hospitalier Universitaire Sherbrooke, Quebec
de
Sherbrooke,
15. Centre Hospitalier de L’Universitaire Laval, Quebec City, Quebec 16. IWK Health Centre, Halifax, Nova Scotia 17. Janeway Children’s Health and Rehabilitation Centre, St John’s Newfoundland & Labrador
Figure 1) Canadian Pediatric Surgery Network sitemap for participating centres
CDH treatment consumes a disproportionate share of health care resources compared with other conditions affecting term infants who require complex, multidisciplinary neonatal intensive care. For example, the cost of care for CDH infants in one Canadian hospital was four times greater than that of a gestational age- and illness severity-matched neonatal cohort. Furthermore, the extrapolated annual cost of hospital care for CDH from birth to hospital discharge in Canada approaches $10 million (7). Similarly, in the United States, the annual birth hospitalization cost of CDH care is estimated to be $250 million (8). Presently, there are no estimates of the health care costs associated with CDH survival beyond hospital discharge. Given the rates and severity of survival morbidity, and the inevitable impact on patient and family quality of life, it is certain that the direct and indirect economic impact on the patient, the family and society are substantial and likely exceed those of most birth defects. Thus, CDH presents an exceptional opportunity for care improvement leading to optimized outcome(s) that will benefit not only the child and family, but also our health system.
Barriers to Birth Defect Outcome Improvement
Outcome improvements for rare birth defects, such as CDH, face several challenges. First, given the rarity of the condition, the number of cases treated by individual hospitals is small and, therefore, many hospitals are limited in their ability to acquire experience and expertise. Second, the available literature from which evidence informing ‘best practices’ can be drawn is often from small, retrospective single-centre studies. To achieve sufficient numbers of cases, these observational studies are conducted over many years and, therefore, do not always reflect contemporary care, which severely limits their relevance to our current patients. Finally, many of these studies lack standardized definitions and risk adjustment, which limits the broad application of their findings. In fact, the lack of validated risk-adjustment tools and standardized variable (risk, treatment and outcome) definitions for CDH make 184
comparisons of therapeutic approaches and outcomes between individual studies difficult – if not impossible – to interpret.
The Role of Patient Registries/Clinical Research Networks
Clinical research networks (CRNs) are groups of clinicians and researchers with a specific clinical research focus and an overall goal to improve clinical outcome for their condition of interest. CRNs may be based on specific disease entities, research populations, treatment modalities, specialty groups or research methodology. CRNs should engage several stakeholders including hospital leaders and decision makers, professional specialty societies, policy makers and, especially, patients. Of fundamental importance to a CRN is a patient registry: a database with standardized definitions that collects observational patient-level data regarding risk factors, treatment details and outcomes of interest. Most importantly, data collection within the network is carefully selected and strategically oriented toward its end users. The value of registry data for the observational study of a rare condition, such as CDH, is the circumvention of small case numbers and the ability to conduct large scale, risk-adjusted studies that are the ‘next best’ alternative to randomized controlled trials. Canada has been a global leader in the use of CRNs to improve perinatal care. The Canadian Neonatal Network (CNN), founded by Dr Shoo Lee, maintains a standardized NICU database and provides a unique opportunity for researchers to participate in clinical, epidemiological outcomes, health services, health policy and informatics research aimed at improving the efficacy and efficiency of neonatal care. Building on the successes of CNN, the Canadian Pediatric Surgery Network (CAPSNet), funded by the Canadian Institutes of Health Research since 2006, is a multidisciplinary group of Canadian investigators whose research efforts focus on CDH and gastroschisis. The network comprises 17 perinatal centres across Canada (Figure 1). CAPSNet maintains an integrated network and shared data platform with CNN and a third perinatal CRN, the Canadian Neonatal Follow-up Network. Paediatr Child Health Vol 21 No 4 May 2016
Commentary
Table 1 Canadian Pediatric Surgery Network (CAPSNet) contributions toward national standards for congenital diaphragmatic hernia (CDH) care in Canada Outcome prediction
Risk adjustment
Practice variation
Outcome variation
Prenatal ultrasound measurements (9)
Termination rates (13)
Prenatal
Ultrasound indices (13) Delivery location (14)
Postnatal
Ventilatory protocols
Validation of SNAP-II (17) Ventilatory modes
Blood gas targets (16)
Mortality models (18)
Gestational timing of delivery (14,15)
Use of pulmonary vasodilators ECMO
Survival complications (19,20) Disability at discharge Long-term disability
Surgical
Side of defect (21)
Need for patch repair (22)
Timing and type of surgery
Institutional case volume (23)
Current CAPSNet contributions are indicated by references in parentheses. Bolded items represent topics to be covered in the CAPSNet CDH Evidence-based Practice for Improving Quality (EPIQ) review project. ECMO Extracorporeal membrane oxygenation; SNAP-II Score for neonatal acute physiology II
CAPSNet Knowledge Synthesis
Since its inception, CAPSNet has been able to address key areas of knowledge deficit, with >30 peer-reviewed articles and 50 presentations at national and international meetings. Contributions to knowledge deficit have created an evidence base for practice change in the prenatal, neonatal and surgical domains of care (Table 1).
Bridging the Gap between Knowledge Synthesis and Practice Change
The term ‘knowledge mobilization’ captures the active process that is required after knowledge synthesis, in which evidence is used to drive and maintain best practices. Conditions for which there is variation in both the treatment provided and the outcomes observed stand to benefit the most from practice standardization. CAPSNet has previously shown substantial practice variation in all care domains and in the outcomes of CDH infants across Canada (9). In fact, mortality for CDH across Canada varies from 40% to 100%. These findings underscore the need to develop evidence-based guidelines for CDH.
What is Evidence-based Practice for Improving Quality?
Evidence-based Practice for Improving Quality (EPIQ) is a quality improvement method that uses published literature and quantitative institutional data to inform practice change. EPIQ enables collaborative, targeted intervention based on outcome variation identified from an analysis of institutional data. The advantages of the EPIQ method include its ability to address multifactorial interventions that may be applicable not only to local hospital settings but also more broadly to similar patient populations. An important advantage of EPIQ, compared with randomized control trials, is its ability to target individual behaviour and institutional culture. Furthermore, EPIQ can address both clinical and process outcomes.
The CDH EPIQ project
Following the success of the CNN EPIQ program, which led to dramatic outcome improvements in bronchopulmonary dysplasia and nosocomial infections within NICUs across Canada (10), CAPSNet has created multidisciplinary workgroups to tackle the controversies related to specific aspects of CDH care. With representatives from the disciplines of paediatric surgery, neonatology, paediatric intensive care, paediatric anesthesia and maternal-fetal medicine, the workgroups seek to develop a library for evidencebased best practices in CDH that cover the trajectory of care for Paediatr Child Health Vol 21 No 4 May 2016
most babies with CDH from birth to surgical repair to discharge. Specific areas of care include perinatal management, ventilation strategies, pulmonary hypertension management, the type, timing and indications for surgical repair, and indications for surfactant use, as well as surveillance protocols for disability and ethical considerations regarding palliation. The first step of the CAPSNet CDH EPIQ Project will be the dissemination of the best-practice guidelines that our working groups have assembled. While other consensus treatment guidelines for CDH are available in the literature (11), our assessment of the literature uses the Grading of Recommendations Assessment, Development, and Evaluation (GRADE), as described by Guyatt et al (12). While GRADE does not completely remove personal judgment, it does provide guideline developers with a comprehensive and transparent framework for developing recommendations regardless of whether the quality of evidence is high or low (12). Moreover, it takes into consideration all patient-important outcomes as part of guideline development.
References
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Paediatr Child Health Vol 21 No 4 May 2016
