Recently I had the pleasure of presenting alongside both Deb Discenza of Preemie World and Keira Sorrells (President of The Preemie Parent Alliance) at a special workshop for the FDA (Federal Drug Association) and Duke-Margolis Center for Health Policy at Duke University on Bronchopulmonary dysplasia in premature babies. We were honored to have been asked to sit at the table and contribute our preemie parent perspective to this very important conversation on chronic lung disease and bronchopulmonary dysplasia in premature babies.
The three of us shared our personal stories of pain and loss caused by premature birth and respiratory illness. We had a lot to say, but before I get to that, let me share the background on why we were invited to this table.
Preterm neonates are often at risk of ongoing pulmonary morbidity, particularly due to developmental immaturity of the lungs at birth as well as damage caused by mechanical ventilation and long-term use of supplemental oxygen. One measure of pulmonary morbidity is captured by a diagnosis of bronchopulmonary dysplasia (BPD), which has been typically defined as the need for oxygen, with or without positive pressure respiratory support, at 36 weeks post-menstrual age (PMA) or “corrected” age. In clinical trials designed to improve pulmonary and respiratory outcomes, the most commonly used endpoint has been BPD. However, there is no consensus on whether BPD is a clinically meaningful endpoint to predict pulmonary and respiratory outcomes at infancy, childhood, and beyond. Therefore, it can be challenging to encourage the development of products in this space because there is uncertainty over whether BPD is an optimal efficacy endpoint for regulatory submissions.
Efforts are underway to support the development of validated efficacy endpoints, that incorporate longer-term improvement or changes in pulmonary outcomes and are more data-driven. However, there are outstanding questions about how best to support the development of such endpoints.
Our expert workshop had the goal of providing stakeholders the opportunity to explore and discuss the work that has been done to date, highlight gaps and limitations in existing data and research, and identify a path forward to develop validated endpoints.
Keira started our presentation and shared her story of delivering premature triplets at 25 weeks 5 days. Although all of her triplets suffered lung complications, her daughter Zoe lost her battle with Bronchopulmonary dysplasia at 14 months old.
Next I shared my story of how my daughter was born at 23 weeks gestation and spent 121 days in the NICU. Joy was discharged on oxygen, a nebulizer, and a diagnosis of both Bronchopulmonary dysplasia and reflux, and a myriad of other health complications from her premature birth. I slept in her room for almost a year because I was afraid she would stop breathing or aspirate in the middle of the night. She almost lost her life again due to respiratory complications at 8 months old and a few other times after that- each requiring weeks in the PICU. She still takes inhaled steroids twice a day to keep her lung inflammation down. Although at 6 years old she is getting better at compensating when she is infected with a respiratory virus, she still required frequent breathing support, meds and her colds last for weeks.
Deb shared how her daughter, now a teenager, always struggled with respiratory illness and how it would take weeks for her to overcome any common cold or lung infection. SHe also shared how many parents of premature babies reach out to her and ask for support via Preemie World- and many of those questions involve respiratory illness and durable medical equipment for preterm infants.
It was amazing to speak in front of a room of so many distinguished physicians and health policy makers. What was very striking was that as each of the three of us spoke, almost every person in the room nodded their head and took notes. This is very telling. They wanted to hear what we had to say. They wanted to know what it was like to have a child who struggled with a chronic lung disease caused by a premature birth. They wanted OUR opinion on what a clinical meaningful endpoint would be, when working towards better treatments for chronic lung disease in premature babies. We had a lot to say and they listened to all of it and then asked a lot of insightful questions when we were done.
As it turns out, we just may be writing an article for the prestigious Journal of Pediatrics, as a result of what we shared!
I want to share a few different comments that parents shared with us, before we made this presentation. All three of us reached out to our respective communities and asked a very simple question:
What is biggest challenge with having a child with lung issues?
We got hundreds of responses (also very telling!) and most of them were very similar. Here is a summary:
- Constant rehospitalizations
- Respiratory illnesses that last FOREVER
- The amount of medical equipment and breathing support needed after discharge
- How cumbersome and difficult it is to leave the house with a baby on oxygen, especially if you have other children
- Not enough information given on RSV and other illnesses that could potentially put my baby back in the hospital or even take their life
- Constant worry of illness when someone comes in contact with your baby
- Being afraid of going to the doctors office because of germs
- The post-traumatic stress responses that resurface when your baby sneezes or coughs
- The hours and hours of using the nebulizer when your child is ill.
- Not sleeping when your baby has a cold because you are afraid that they will stop breathing
These are things that us preemie parents worry about. I could have made that list myself.
Imagine if new medications and therapies were developed that could prevent our tiny babies from suffering and could prevent us preemie parents from carrying these fears with us every day?
That’s what the workshop was about and although our day did not end with solutions, I believe that many important conversations will begin and this will lead to progress.
Don’t forget that November is National Prematurity Awareness Month and November 17th is World Prematurity Day!
Keep checking back to Speaking for Moms & Babies to see what information and resources we’re sharing to help spread awareness 🙂