POWER OF STORIES TO HIGHLIGHT THE COMPLEX ISSUES SURROUNDING BRONCHOPULMONARY DYSPLASIA
Wakako Minamoto Eklund, DNP, NNP-BC
No neonatal nurse can spend even a month in the neonatal intensive care unit (NICU) without caring for infants who are at risk for developing bronchopulmonary dysplasia (BPD). The risk of BPD in the early 1990s may be different from that of today; however, neonatal nurses understand the devastating course that extremely premature infants may take and the excruciating adjustments for families who take an infant home with pulmonary fragility. This brief provides a current understanding of BPD and how BPD incidence and concept of BPD have changed overtime. Caregiving trends for clinicians, researchers, as well as regulatory bodies are provided to increase understanding about what BPD is, what is required to advance the outcomes related to BPD, and what patients' experiences are with BPD infants today.
THE POWER OF STORIES
Three powerful stories told by mothers of former premature infants who survived NICU with lung disease recently appeared in the Journal of Pediatrics.1 The 3 mothers, Deb Discenza, Jennifer Degl, and Keira Sorrells, who are all members of the Preemie Parent Alliance,2 which is a dynamic network of mostly former NICU parents, are the courageous storytellers. The 3 mothers describe their raw experience of living with BPD with passionate words that demonstrate the deepest of love for their sick children who require complex daily care, some who were tragically overcome by the complications. The stories touch our human souls in a way to propel us to a desire for a deeper understanding of the needs and to recognize families' challenges with the warmest empathy during the NICU journey.
THE STORIES COMPLEMENT THE STUDY RESULTS
The stories were published to add tangible dimensions to a study also published in the same issue of the Journal of Pediatrics by Brady and colleagues,3 titled “Living With Severe Bronchopulmonary Dysplasia—Parental Views of Their Child's Quality of Life.” Brady and colleagues report the findings from a study of infants born before 32 weeks' gestational age (GA) with BPD who survived beyond 18 to 36 months corrected age. The researchers studied the parental views of the children's health-related quality of life (QOL), specifically physical and psychosocial QOL, and the relationship with the neonatal morbidities associated with BPD. The 3 mothers' stories add additional layers to the Brady and colleagues study results regarding the children's QOL and how BPD had impacted every area of their family life. Both of these articles are available at https://www.jpeds.com/article/S0022-3476(18)31718-9/fulltext and https://www.jpeds.com/article/S0022-3476(18)31412-4/fulltext. Both physical and psychosocial QOL scores were significantly lower for parents with children suffering from BPD than for the term or other preterm infants. Although we do not often continue to care for these infants and their families beyond NICU discharge, knowing the results of this study may allow deeper compassion and empathy in preparing families during the NICU journey.
HISTORY OF BPD
Some NICU caregivers today do not know the era before high-flow nasal cannula or continuous positive airway pressure devices were developed; however, some of the readers have been caring for babies since the time before surfactant was available. BPD was first described as pulmonary disease of premature infants who required the use of oxygen at 28 days of life by Northway and colleagues4 in 1967. However, the participants in this study were not the extremely low birth-weight infants (ELBWs) who weigh less than 1000 g at birth seen in the NICUs today. The BPD description by Northway and colleagues was mostly of premature infants older than 32 weeks' GA. Since the advent of artificial surfactant in the 1980s,5 infants who would have otherwise not survived without surfactant have survived, thereby increasing the incidence of infants who develop pulmonary morbidities. One study published in 1992 reported more than trifold increase in the incidence of BPD from 1980 (10.6%) to 1990 (32.9%) among infants born weighing less than 1500 g with a concurrent decrease in neonatal deaths from 26.4% to 15.9% during the same period.6 In the new era of advanced fertility treatments and refined neonatal care with limits of viability being pushed back, survival of significantly premature infants at risk for BPD became a more frequent occurrence.7 It is of note that the physiology and etiology of BPD described by Northway and colleagues and what we see in ELBW infants who are born at much earlier gestations are not identical, due primarily to the stages of lung development that are interrupted by much earlier gestation. Increased survival is a welcome news and, in fact, it reflects an amazing advance in neonatal care; however, survival with significant morbidities and its negative impact on individual families cannot be underestimated as seen in the mothers' stories noted earlier.1
BPD indeed saw a decline. In 2010, a large nationwide population study8 that examined 9,542,032 neonatal admissions found an annual decrease of 4.3% in BPD from 1993 to 2006 (P= .002). Yet, another study9 of 9575 infants (22-28 weeks' GA) by the National Institutes of Health–Neonatal Research Network published in the same year did not report an overall decrease in BPD incidence. However, when analyzed according to severity, incidence of severe BPD declined from 2003 to 2007.9 BPD is the most common complication of premature births, with known predictors being decreasing GA and receiving mechanical ventilation on day of life 7.10,11 Many changes in obstetric and neonatal practices occurred over the years, changing the face of respiratory care with increasing noninvasive respiratory management approaches with general decreases in more invasive ventilation.7 With the known predictor of BPD as a decreasing GA or an invasive respiratory care needs early in life, the best method to decrease BPD, therefore, is to prevent preterm births. However, BPD has not disappeared in 2019, and BPD still overpowers infants today, as witnessed by all neonatal care providers. Effort to reduce BPD is continuing globally in 2019, 52 years after Northway and colleagues4 first described BPD.12 BPD is a challenge not only for the NICU professionals and families involved but also for clinical researchers, the industry partners, and regulatory bodies that are committed to developing new therapies to improve BPD outcomes.
CHALLENGES RELATED TO BPD RESEARCH
Since the development of surfactant and antenatal steroids, no newer therapies have made a significant impact on BPD.13 Efforts are still being made globally to enhance treatment options; however, BPD research is faced with significant issues due to lack of consensus related to the definitions14 as well as endpoints12 to build robust clinical studies. Numerous attempts have been made to define BPD that correlates with the actual pulmonary morbidities later in life,14 which is referred to by “chronic pulmonary insufficiency of prematurity.”12 Uniform agreement in defining BPD is needed to better understand which infants must have BPD diagnosis at the end of NICU journey (to observe any reduction later as a result of any research intervention). A simple example demonstrating the confusion related to the definition is described by Steinhorn et al.12 Some infants on 4 L/min high-flow nasal cannula at 36 weeks' corrected age are actually not viewed as having BPD due to no need for supplemental O2, while infants receiving 0.1 L/min at 100% O2 would be classified as having BPD.
Consensus is also necessary to consider endpoints, or the time at which various outcomes are measured in any studies. The endpoints must be meaningful not only to the researchers but also to the families. Some endpoints are described as short term and some are long term.12 If the endpoint is too far out, the research studies may risk losing the enrolled patients and the cost of the follow-up may increase. Long-term endpoints, however, may better capture the meaningful outcome to patients and families. Short-term endpoints may be highly valuable in certain types of interventions that are being studied.
Without well-thought-out definitions and endpoints, inaccurate data may be obtained, leading to inaccurate conclusions. New therapy that may have been truly effective and safe may be found to be ineffective due to the suboptimal endpoints or definitions that skew the analysis and results. The challenge in enrolling a sufficient number of neonates in any single study emphasizes the value of meta-analyses that offer more generalizable results. Ununified definitions or the endpoints among the studies make this process highly difficult.
Numerous Food and Drug Administration (FDA) members, other regulatory members from abroad, global researchers, clinicians, and industry partners who are committed to promote respiratory innovation gathered in Washington, District of Columbia, in October 2018 at the Duke-FDA Workshop on Endpoint Development for Preterm Neonates With Pulmonary Morbidities and discussed the urgent need to redefine important elements needed to promote further global research to decrease pulmonary morbidities. One of the high points of the discussion included the recommendation that BPD defined as “supplemental oxygen use at 36-week postmenstrual age” be changed to possibly 40 week since the current BPD definition is not known to be truly predictive of later pulmonary morbidities.15
Every NICU nurse contributes as a member of NICU team to improve both the short- and long-term pulmonary outcomes of the premature infants we take care of, and care of infants with BPD is inescapable. The National Association of Neonatal Nurses (NANN) also attended this meeting to represent the nursing voice in this matter and to support the voice of the parents. The 3 mothers who published their stories in the Journal of Pediatrics1 were also in attendance and shared their experiences to educate FDA members and other researchers about what is meaningful and what their life is like to live with BPD. Take a deep breath while you take care of the babies in your NICU. Reading the articles discussed earlier will provide some insight into the lives of the babies we take care of through the eyes of their dedicated mothers who are our partners during their NICU journey.
1. Degl J, Discenza D, Sorrells K. Remembering the power of stories in pediatric research. J Pediatr. 2019;207:14–17.
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15. Hines D, Modi N, Lee SK, et al Scoping review shows wide variation in the definitions of bronchopulmonary dysplasia in preterm infants and calls for a consensus. Acta Paediatr. 2017;106:366–374.