Partnering With Parents to Change Measurement and Reporting of Preterm Birth Outcomes

My children were born at 25 weeks’ gestational age in 2009, and 1 of my daughters died. The birth of an extremely preterm child can plunge parents into a world of fear and uncertainty. Many parents are concerned with their child’s long-term outcomes, as well as the impact that having a child born extremely preterm will have on the life of their family. After my children were born, searching for information about the “outcomes of extreme prematurity” returned extremely pessimistic and deficit-based research, loaded with statistics and terms like neurodevelopmental and sensory disability, behavioral disorders, and cognitive impairment.

There is a long tradition of evaluating outcomes, especially neurodevelopment, in children born at the threshold of viability. Children born extremely preterm are assessed in neonatal follow-up programs for clinical care as well as data collection for bench marking, quality improvement, and information for management and family counseling. The choice of what information to collect and report during neonatal follow-up was based on feasibility, validity, availability, cost, and what was deemed to be important by doctors and scientists, without parental input. Parents are integral to the care of their child, yet in 2016, there was little available published information about parents’ perspectives regarding which outcomes are meaningful to them, so we sought to better understand this gap. Subsequently, rigorous studies have identified core outcome sets for neonatal research where families and key stakeholders contributed to selecting outcomes identified in systematic reviews.^1,2  Parents and patients ranked outcomes differently from other stakeholders, especially doctors.¹ 

We present our research experience integrating parents, clinicians, and researchers to identify the selection and reporting of meaningful outcomes after preterm birth, how we shared the knowledge gained, and changed practices.

The Canadian Neonatal Follow-Up Network (CNFUN) created a national standardized follow-up visit with data collection at 18 to 24 months corrected age for children born extremely preterm at less than 29 weeks’ gestation.³  During this visit, we have assessed neurodevelopment and reported outcomes similar to what others have done: cognitive, motor and language abilities, cerebral palsy, and hearing and visual impairments. This information is used to classify children according to their level of neurodevelopmental impairment (NDI). I was aware of the limitations of current outcome measures and definitions, yet still felt the need to use conventionally accepted outcomes to obtain funding and get academic recognition. A catalyst for change was a parent at a conference who courageously challenged the outcomes presented by CNFUN.

The opportunity arose to apply for funding through the Canadian Institutes for Health Research strategy for patient-oriented research. Principles of patient-oriented research are (1) patients are meaningful partners engaged in the continuum of research, (2) the research aims to improve patient outcomes, improve healthcare systems, and practices; and (3) research involves a multidisciplinary team.⁴  Patients also include family caregivers, relatives, and members of the public. CNFUN had the infrastructure to conduct research, the Canadian Premature Babies Foundation (CPBF)⁵  had interested, committed parents, and many clinicians wanted to participate. We chose to engage families in rethinking what outcomes were meaningful after the birth of an extremely premature baby. We planned a series of studies to: (1) capture the voices of a large number of diverse parents, (2) examine the perspectives of parents in more detail, and (3) address potential biases and involve parents in all aspects of research: methodologies, recruiting participants, analyzing verbatims from qualitative data, interpreting results, and revising manuscripts.

Our Parent-Centered Evidence-Based Care for Premature Graduates study (Parents’ Voice studies) obtained funding from the Canadian Institutes for Health Research strategy for patient-oriented research program as part of the Child Health Initiatives Limiting Disability - Brain Research Improving Growth and Health Trajectories (CHILD - BRIGHT) network in 2017.

In our first study, we engaged over 1000 Canadian parents with a child born extremely preterm attending a CNFUN clinic visit.⁶  They were told that their opinion was important and were asked whether they felt their child was developing normally or had a mild, moderate, or severe developmental impairment. There was only fair agreement between parents and the CNFUN classification; families usually viewed their child as less impaired.⁶ 

To evaluate how families agreed with the medical definitions of impairment used in CNFUN and how they perceived the severity of these impairment in children, we created clinical scenarios of children with 10 different developmental challenges categorized as severe.⁷  When the coronavirus disease 2019 pandemic hindered recruiting family members at in person visits, CPBF and other parent groups helped by distributing the survey on their social media platforms. We learned from over 800 stakeholders that most did not perceive the scenarios as severe.⁷  For example, needing cochlear implants for a hearing impairment was considered severe by 15%. The scenario considered by the largest proportion (56%) of participants as being severe was the combination of cerebral palsy and language delay.

In 2 other more in depth studies,^8,9  we asked a total of 447 parents about the impact of the preterm birth on their lives; the well-being, health, and concerns about their child; their information needs and to identify relevant outcomes; and what should be communicated to parents. Parents reported that function was more important than diagnoses. They identified important outcomes that are not investigated as thoroughly as neurodevelopment, such as their child’s feeding, sleep, respiratory health, and behavior. Parents wanted a more balanced perspective and more optimism from doctors as well as more practical advice.

Parent partners can contribute to knowledge dissemination. Rebecca Pearce and/or Fabiana Bacchini, alone or accompanied by clinician-researchers in our group, presented our research results to a variety of audiences, including obstetricians, neonatologists and pediatricians, allied health care professionals, ethicists, and families. The parents lived experience made the presentations so much more meaningful. Results were also shared with a community of parents, such as CPBF preemie chat webinars.⁵  Parents and researchers, working together as equal partners, created blogs and infographics on the CPBF Web site.⁵ 

Our teams wanted our research to lead to real change in the ways that doctors collect and communicate the outcomes of prematurity. We used virtual meetings to share information, encourage discussion, and identify action plans (Supplemental Information). Research ethics board approval was obtained from the University of British Columbia Children’s and Women’s Research Ethics Board (H17-03490).

In the first step, results from the Parents’ Voice studies were shared with the parents, clinicians, and researchers who had been involved in the Parents’ Voice studies to identify themes where actionable items and consensus statement could emerge. A 3-hour virtual meeting was held in September 2021. Three parents and 9 clinicians and/or researchers participated. Results were presented by trainees and parent representatives. Ample time was allotted for discussion. Consensus statements that arose from the discussion were categorized from: (1) strong evidence from the Parents’ Voice studies; (2) good evidence from other sources; and (3) ideas for future consideration. Participants were asked via survey whether they (1) agreed or disagreed with the statement and (2) whether they thought the statement could be feasibly implemented in clinical practice. A priori, consensus was defined as at least 80% agreement. A total of 18 statements were generated (Table 1). Of the statements with consensus, the majority were considered feasible.

Participants agreed that the next step was to identify the best way to measure the outcomes in children 18 to 24 months of age that had been identified as important or meaningful from the Parents’ Voice studies. These include child well-being, quality of life or function, socioemotional and behavioral outcomes, respiratory, feeding, sleeping, and caregiver well-being and mental health.^8,9 

The planning committee created interdisciplinary clinician working groups for each of the 7 family important domains listed above. Each working group conducted a literature search of potential questionnaires or tools to evaluate the outcome domain and made recommendations. Results were presented to 22 parents, clinicians, and researchers at a virtual workshop in 2022. Family, clinician, and researcher perspectives were considered separately, and discussion encouraged. Final updates were shared, and workshop participants completed another survey. Recommended questionnaires and tools to measure the parent identified domains were identified and published in a scoping review of how to measure family, parent, and patient outcomes.¹⁰ 

Of the 14 actionable consensus statements, 10 have been or are in the process of being implemented throughout Canada. Unexpectedly, 5 consensus statements considered for future action are being implemented or became the focus of research grant applications (Table 1). Changes we have made include describing health states without any judgement values. For example, instead of saying “severe hearing impairment,” we will say “use of hearing aids or cochlear implants.” We avoid the composite outcome “death or NDI”: this was viewed as ethically problematic by families and as flawed by clinicians and researchers.

Our family partnership started with parents and clinicians listening to each other and identifying common goals. Applying for funding with parent partners required collaboration and team building. Obtaining funding provided the infrastructure, a timeline, and cemented the commitment to complete the projects. The Child Health Initiatives Limiting Disability - Brain Research Improving Growth and Health Trajectories network provided invaluable resources for parents and researchers new to patient-oriented research. We followed the principles of family engagement throughout our research. Over 2000 parents have participated in the Parents’ Voice studies and change is being implemented. As relationships and trust developed, the role of families with lived experience expanded. We had to be flexible and revised our protocols as conversations evolved. The core group of very dedicated parents created continuity and leadership. A much larger number of parents made meaningful contributions in specific aspects of the studies. Over time, as parents became more involved, the culture of parent engagement shifted. More parents were invited to conferences and were asked to speak or present posters. We learned that we had different strengths. For example, parents were more comfortable and knowledgeable about disseminating results via social media and infographics.

We sought to facilitate parent participation in our data collection. We asked many parents in several different ways to make participation as easy as possible, explored the question of NDI severity from different angles, and delved into the topics of what outcomes to measure and how to describe outcomes. To our knowledge, the Parents’ Voice studies are the largest investigation that involved parents in all aspects, capturing opinions from over 1000 parents on categorization of NDI severity in their own child, over 800 stakeholders described their perspective of NDI using clinical scenarios, 199 parents described the importance of capturing positive outcomes as well as challenges, and 248 parents described the outcomes that were important to them. Consistent messages were heard. Most parents do not categorize children according to traditional medical definitions of NDI. The term “severe” is interpreted differently. Parents wish to hear about the positive aspects, as well as the challenges of children born preterm, and they identified other aspects of preterm children’s lives that should be included in discussions of outcomes of prematurity.

Partnering with families and patients is feasible, productive, and facilitates change. The process described in our study is generalizable to other countries and other patient populations and research groups. We highlighted how parents can disseminate crucial knowledge to medical and lay audiences. Implementing change can be hard. Working collaboratively with strong representation from a parent advocacy group, clinicians and a network of researchers, we succeeded at identifying outcomes deemed important by parents, generated suggestions for measuring these outcomes using valid scientific methods, and provided guidance to scientists and clinicians on the words to use to avoid stigma and to promote strength. Our strength is that we sought the opinions of thousands and engaged parents throughout the research journey. Furthermore, these studies have shown that bringing different and complementary perspectives can foster change in research and health care delivery that is beneficial to all.

Jehier Afifi, MBBCh, MSc: Department of Pediatrics, Dalhousie University and Division of Neonatal Perinatal Medicine, IWK Health, Halifax, NS, Canada

Audrey-Anne Milette, MD: Research Center, CHU Sainte-Justine, Montreal, QC, Canada

Claude Julie Bourque, PhD: Research Center, CHU Sainte-Justine, Department of Pediatrics, CHU Sainte-Justine, Department of Family Medicine and Emergency Medicine, Université de Montréal and Center for Applied Health Sciences Education, Faculty of Medicine, Université de Montréal, Montreal, QC, Canada

Paige Church, MD: Boston Children’s Hospital and Beth Israel Deaconess Medical Center, Boston, MA

Keith J Barrington, MD: Department of Pediatrics, CHU Sainte-Justine, Université de Montréal, Montreal, QC, Canada

Lindsay Colby, RN, MSN: Neonatal Follow-up Program, British Columbia Women’s Hospital, Vancouver, BC, Canada

Karel O’Brien, MBBCh, MSc: University of Toronto, Department of Paediatrics, Maternal-Infant Care Research Centre, Mount Sinai Hospital and Mount Sinai Hospital, Department of Paediatrics, Toronto, ON, Canada

Kamini Raghuram, MD, MSc: University of Toronto, Department of Paediatrics and Mount Sinai Hospital, Department of Paediatrics, Toronto, ON, Canada

M Florencia Ricci, MD, PhD: Department of Pediatrics and Child Health, University of Manitoba and Children’s Hospital Research Institute of Manitoba, Winnipeg, MA, Canada

Saroj Saigal, MBBS, MD: Department of Paediatrics, McMaster University and McMaster Children’s Hospital, Hamilton, ON, Canada

Judy Seesahai, MD, MSc: Department of Paediatrics, Schulich School of Medicine and Dentistry and Windsor Regional Hospital Metropolitan Campus, Department of Paediatrics, Windsor, ON, Canada

Prakesh Shah, MD, MSc, MBBS: University of Toronto, Department of Paediatrics, Maternal-Infant Care Research Centre, Mount Sinai Hospital and Mount Sinai Hospital, Department of Paediatrics, Toronto, ON, Canada

Marie-Noelle Simard, OT, PhD: Research Center, CHU Sainte-Justine and School of Rehabilitation, Faculty of Medicine, Université de Montréal, Montreal, QC, Canada

Karen Thomas, MD: Department of Paediatrics, McMaster University and McMaster Children’s Hospital, Hamilton, ON, Canada

Tom Wiebe: Patient and Family Engagement Advisor Committee, British Columbia Women’s Hospital Neonatal Program, Vancouver, BC, Canada

Jill G. Zwicker, PhD, OT: BC Children’s Hospital Research Institute, Department of Pediatrics, University of British Columbia and Department of Occupational Science & Occupational Therapy, University of British Columbia, Vancouver, BC, Canada

Dr Osman Ipsiroglu provided specialist content expertise

We thank the members of the Parents’ Voice Network who participated in data collection, contributed to generating ideas, and critically reviewed and revised the manuscript.

CNFUN

Canadian Neonatal Follow-Up Network

CPBF

Canadian Premature Babies Foundation

NDI

neurodevelopmental impairment