Outcomes of very preterm infants: what matters to parents?

Neonatologists can be said to have invented “Outcomes Research”, the systematic evaluation of patients after an acute-care event. It is a field of research which has now been extended from early infancy into the adult period and provided enormous insights into what happens to babies who are born very preterm. When the field was developing there were concerns about how our patients would fare when their brain development had been interrupted by preterm delivery, often accompanied by serious illness, and sometimes major complications, surgery, and brain injuries seen on CT scan or ultrasound. As a result much work has concentrated on neurological examinations and developmental screening tests, partly to evaluate and describe outcomes, but also to ensure that chlidren received any interventions they might need with minimal delay.

The major question that we need to ask now is: what outcomes are most important to parents? One serious problem with our outcome research had been the attempt to categorize infants into “normal” and “abnormal”. This has been driven by the appropriate concern that our NICUs should be aiming for not just improved survival, but for improved “intact survival”. That is a term that I have heard many times, but it requires some thought; what do we mean by intact? And who decides what is an acceptable outcome?

I have written many times on this blog about the problems that arise when we decide that a Bayley score at 18 to 24 months of age below 2 SD below the mean is an adverse outcome, whereas a score of 1.9 SD below the mean is a good outcome, even when the child has a serious behaviour disorder. This arbitrary and artificial dichotomising of the whole of child development skews the literature, and has impacts on how we care for babies.

What we measure matters also because we use the results of outcome studies in our counselling of parents regarding the institution and continuing of active NICU care. (Weiss EM, Kukora S, Barrington KJ. Use of composite NICU research outcomes for goals of care counselling creates ethical challenges. Acta Paediatr. 2021) Data which are collected in order to describe outcomes are then used to determine whether babies should be allowed to die without active intervention.

In this post I will not be concentrating much on how the artificial dichotomising of child development skews research design, even though that is a major problem. Because it is easier to design studies with a dichotomous composite, where death and “NDI” are given equal weight, and the answer to whether a treatment is better than another is supposed to them be yes or no, based on the impact on that composite outcome.

In everyday clinical practice we (as health care professionals) really want to know whether treatment A decreases death compared to treatment B, and if not, what are the relative impacts on developmental progress, pulmonary development, neurologic impairment, etc. But what do parents want to know? I think we can be sure that parents would want to know if one treatment compared to another affected mortality, but beyond that, what outcomes are really important to parents? What are their relative importance? What kind of adverse outcome would be enough to outweigh an improvement in survival?

Personally, I would be surprised if a group of parents cared whether their babies’ Bayley scores were more likely to be above or below 70.

A group of collaborators from my hospital have just published an article reporting a study (which is part of the Parent Voices Project) where parents were asked their opinions about the progress of their extremely preterm infants. Jaworski M, et al. Parental perspective on important health outcomes of extremely preterm infants. Arch Dis Child Fetal Neonatal Ed. 2021:fetalneonatal-2021-322711. This was a group of parents with 213 children aged from 18 months to 7 years of age, less than 29 weeks gestational age. Children are routinely followed until 36 months corrected age, and then some are discharged if they are doing well with normal developmental progress and the parents have no behavioural concerns, which means that the older children in the cohort tend to be those where there are some issues that need following. Overall there were 55% of parents who had some concerns about their infants developmental progress, including an 18% who had concerns about behavioural and emotional issues. Parents of the 53 infants identified as having “severe NDI” (defined as a motor, language, or cognitive composite score <70 on the Bayley3, or cerebral palsy with a GMFCS of 3,4, or 5, or needing amplification or being visually impaired in both eyes), were only slightly more likely to have concerns about the development of their infant than the 82 without “NDI”. Growth, feeding and respiratory concerns were similarly represented among the subgroups. Mothers and Fathers had similar concerns and there were no substantial differences by gestational age group.

Of the issues that matter to parents, the quotes suggest that it is really the child’s functioning that they worry about (‘I wish she would express herself more clearly’), whereas scores on standardised tests were never mentioned! Of course, if those scores help to identify infants who would benefit from intervention, then they may be of value to the individual. If they, overall, reflect the function of the group then they could be a reasonable way of summarizing outcomes. But behavioural/emotional development is very rarely mentioned in outcome studies, despite the major importance to many parents, nor are feeding and growth commonly reported.

This project says to me that we should systematically describe and report behavioural, emotional, feeding and growth outcomes in our follow-up studies, and in the follow-up of our RCTs in extremely preterm infants. Those things concern parents just as much as developmental delay, and have impacts on the families similar to other things that we describe routinely.

As a parent myself of an extremely preterm infant, perhaps I can allow myself, for this first post of the new year, to give my opinion of what outcomes matter. My daughter indeed had her standardized tests, administered by a friend and colleague in the McGill University follow up system (Dr Elise Couture). I was rather uninterested in the results of those tests, I don’t even remember if she “passed” or not. I knew she was progressing, was learning new skills, and had behavioural issues that were not out of the ordinary. She had feeding issues that were more disruptive than any problems with cognitive development, and when she got to school had some issues with her learning style, of an executive function type. With the dedication of her mother, in particular, she was able to progress through school, and is very soon to graduate from high school, at which she works extremely hard. For me the most important features of follow-up were to know whether there were any specific therapies that could help to make her life easier: Did she need physio? Would speech therapy help? How could we best assist her learning pattern? Her ENT problems and pulmonary development were also of major interest, and fortunately she didn’t have any major hearing or vision problems needing intervention, other than spectacles. I try to imagine what my attitude would have been if she had turned out to have a Bayley MDI (she did the version 2) score <70. I can’t imagine Annie or I would have treated her any differently, and the idea that having a low Bayley score would have been the same outcome as her being dead, I find offensive.

On the other hand, evaluating, and finding a way to summarize, the developmental progress of our patients is worthwhile, having a more robust development, more similar to babies born at term would be a good thing. A more detailed description of outcomes, including average scores and the spread of scores, on standardized screening tests, would give a more useful picture of the outcomes of our patients than just the proportion of babies below various thresholds. More information about what outcomes matter to parents should help us to design follow up programs that respond to their needs.

About Keith Barrington

I am a neonatologist and clinical researcher at Sainte Justine University Health Center in Montréal
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5 Responses to Outcomes of very preterm infants: what matters to parents?

  1. Dear Doctor

    It is very interesting to take this look at the neurodevelopment of those who were born before the term. For me it is a daily challenge to respond to the needs of families. We keep looking for alternatives

    At the same time, I seek to converse with young doctors in training looking from the “achievements” of our patients and not from the “deficits”. For now we always read in the papers: “deterioration” in development and not “achievements” in development. Why will it be? How do you see this that I mention? Perhaps, from that change of perspective, we can approach the needs of families. Do not know

    Sorry for my English

    • Thanks for the comment, that I agree with wholeheartedly, we discuss and study the limitations of our patients more than their capacities, their disabilities rather than their abilities! It is understandable, given the history of follow up and our focus, which is quite appropriate in some ways, on finding problems that we can fix. But there is so little work on how to support and aid the development of our patients compared to describing how messed up they are. I think it is remarkable that despite passing 3 months of a period which is critical for cerebral growth and development, often interrupted by major complications, that most preterm babies do so well.

  2. Deb Discenza says:

    Excellent post Dr. Barrington. Agreed, we should talk about abilities 100% but also not to the neglect of realizing that there may be disabilities that should be diagnosed and not ignored by the medical/therapeutic communities.

    I say this as a parent of a 30-weeker now 18 years old.My daughter had a rollercoaster NICU stay and post-NICU it was like walking off a cliff into the generalist pediatrician’s office. Medical and state therapeutic limitations prevented my daughter from accessing Early Intervention at discharge but finally at 18 months of age when there were global delays. It really took my husband and I being vocal to get her the help she so obviously needed. Kindergarten – diagnosed with ASD because I was clear with her IEP team that something wasn’t right. 2nd Grade – ADHD because I made it clear we were talking about it. A feeding issue and program at 12 years old because my daughter was malnourished and I was able to focus on her needs only after helping my two parents with major health issues. And then 13.5 years old, she was diagnosed with Cerebral Palsy (spastic diplegia) and the comorbidity of Scoliosis after I had been talking about this for a decade and I was honestly tired and figured I was wrong. My daughter and I walked out of that appointment stunned.

    How could so many people not notice all this, especially in the Washington DC area that boasts a solid medical and therapeutic environment? It shouldn’t take a parent demanding help or vocalizing concerns several times in an appointment to be heard. We need to do better. And with that I am creating a data portal to give better outcomes information. We are asking NICU doctors to have a magical crystal ball to predict many years on how their child will do. There are simply guesses. I hope to do my part to increase that data, improve those outcomes and help families advocate with better information in their hands other than Google or WebMD. It is not about making a doctor mad or second-guess their expertise. It’s about partnering with the professionals and working together for the best outcome of the child. And I use our experience to help support families and provide them with skills toward advocacy because they are key in all of this. Advocacy is not outsmarting the doctor, it’s combining everyone’s expertise to help the child which is the professionals and the parents as well as the child him/herself as a young adult.

    Despite all of this? That 30 weeker child is now a young adult and is about to graduate high school and has taking college classes since she was 14. I gave her the gift of a mantra early on that “Challenges come with gifts and everyone has challenges so everyone has gifts.” Disabilities were a known and we have gotten her so much help over the years. But we also leveled the playing field for her with our mantra. She is an incredible person with stunning artistic abilities, a knowledge-base of a PHD and more. But the most important thing? She has compassion for those who struggle. She loves animals, the elderly and the disabled. With them, she is not a number of labels but quite simply a wonderful human being. We celebrate her and have done so since birth.
    With Gratitude,              

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    • Thanks for the comment, and for all you do for Preemie families. I agree we must not ignore the challenges that preterm born babies are more likely to have compared to babies born at term. The importance of early intervention should not be understated, which is why routine surveillance and evaluation of former preemies is so important. Cerebral Palsy (as one example) is much more common among former preterms, and a good follow up system should be able to detect motor problems at a stage where physiotherapy can lead to major improvements in long-term function. I am happy for you and your daughter that she is doing so well despite all the difficulties she has had to overcome.
      I don’t mean to imply that these problems are unimportant, not having CP is always going to be preferable to having CP, but a child with CP is worth exactly as much as one without. Motor development is also something that should not simply be dichotomised into normal (=good outcome) and abnormal (=bad outcome), the GMFCS system is a start in having a more nuanced description of motor outcomes.
      Outcome studies cannot just list all of the individual detailed outcomes of each individual, we need to be able to summarize those outcomes somehow, we just need to do it better.

  3. Deb Discenza says:

    Dr. Barrington, Exactly. It is a double-edged sword with many prematures. On one hand they are at risk for disability and on the other hand they can also be twice exceptional thereby challenging the public assumption that all disabled people are not abled in any way. It’s sad but still true these days. Educational professionals have started to see the light as well as employers. But in viewing a Washington Post article yesterday where a woman in a wheelchair after many challenges with potential workplaces finally got an offer for employment that could provide accommodations for her so she could do her best work. This is 2022 and ADA in the US happened in the 1990’s.

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