Continuum of Care of individuals born preterm

Federico Bianco PhD

NPMF 2024
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CONFLICT OF INTEREST

I’m a Chiesi employee

I am not a medical doctor.

I have a degree in veterinary medicine and a PhD in

biotechnology

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Continuum of Care of individuals born preterm

From premature birth to an asthma or COPD diagnosis,

what’s the journey of these individuals?
What can be done to improve this journey?
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Table of Contents

Prematurity as a predisposing risk factor of adult chronic

respiratory conditions

Patient journey from a clinician perspective: the Pre-Tell

survey

Patient journey from a patient

perspective: the EMPOWER study

Final remarks

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Implications of Childhood Lung Deficits into Adulthood

• The normal lung function trajectory from

birth to death has three phases: a growth
phase (from birth to early adulthood), a
plateau phase (that lasts for a few years),
and a decline phase resulting from
physiological lung ageing.
• COPD is characterized by an accelerated
decline of lung function, “lung ageing”, which
has been traditionally associated with
smoking.
• Notably, About 30% of patients with COPD
worldwide have never smoked.
• Therefore, the decline can also be due to
other factors, including lung injury and
remodelling.
• About 40% of lung function deficits at 6–7
years of age are already present at birth.

5 Agustí & Faner Lancet Respir Med 2019

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FEV1 in BPD Survivors

• Baraldi and Filippone (2007) reviewed 18 studies reported since 1990 in which BPD survivors at age 6 to 19 years were compared with a
reference population born at term.
• Except in two studies, the FEV 1 of BPD survivors was significantly lower than that of the reference population (80% of predicted FEV1 is the
lower limit of normal).
• Only 4/18 studies were conducted in the post-surfactant era (labelled with an *). Therefore, the graph better reflects the outcome of children
who most probably had old BPD.
• BPD survivors may have variable airflow limitation from the first years of life, with little evidence of catch-up. In many BPD survivors, FEV1 will
not reach the maximal value in early adulthood, and the declining phase will start from a substantially reduced maximal value.

6 Baraldi & Filippone NEJM 2007

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Follow-up of Former Preterm Infants (1/2)

• Quitting smoking and decreasing exposure to air pollutants (including cigarette smoke) during childhood
and puberty, close follow-up of former preterms, and promoting physical activity and a healthy diet must
be encouraged to prevent abnormal growth and reduce lung function decline1,2.

• Day-care attendance should be considered. The first winter of very young infants with severe BPD is
clearly of greatest concern2.

• In cases of moderate-severe BPD or frequent re-exacerbations, Bonadies et al. recommend follow-up visits
with pediatric pulmonologists, who can help general pediatricians assess patients’ respiratory symptoms
and monitor the benefits of any therapies, preferably measuring lung function2.

• Given the immature and less competent immune system of infants born preterm, their immunization
schedules should not be delayed1,2.

• Beyond 5 years of age, spirometry (and maybe IOS) is an easy and non-invasive tool, so it is useful in the
longitudinal follow-up of school-age children born preterm, especially if with BPD2.

1. Agustí & Faner Lancet Respir Med 2019

2. Bonadies et al. Eur J Pediatr 2022

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Follow-up of Former Preterm Infants (2/2)

• The long-term medical complications of premature

birth are not limited to the lungs. Epidemiological
studies have linked preterm birth with a higher risk
of hypertension, ischemic heart disease, diabetes,
lipid disorders, heart failure and Kidney failure,
among others1,2,3.
• Therefore, a comprehensive approach to the
former preterms is strongly recommended, which
implies a large multi-specialistic team coordinated
by the general pediatrician and including
pneumologists, cardiologists, neurologists,
nephrologists, endocrinologists, radiologists, etc3.

Crump et al. Early Hum Dev 2020

1. Crump et al. Early Hum Dev 2020

2. Agustí & Faner Lancet Respir Med 2019
3. Bonadies et al. Eur J Pediatr 2022
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Summing up

• Irrespective of their gestational age, preterm-born individuals show, on average, a lower

lung function compared with term-born infants.
• Airflow limitation and the incidence of respiratory morbidity through childhood into
adulthood generally increase with decreasing the gestational age at birth.
• The most significant lung function deficits and the higher risk of developing COPD are
seen in extremely preterm-born individuals who were diagnosed with BPD.
• Although lung function decline can be followed with spirometry, an unmet need is to
distinguish, early in life, those preterm-born individuals who have the highest risk of
developing COPD.
• In this regard, one of the main research priorities is the risk stratification of preterm-
born individuals, which may eventually allow identifying the most vulnerable subgroups,
enabling their close follow-up and the development of targeted treatments.

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Table of Contents

Prematurity as a predisposing risk factor of adult chronic ì

respiratory conditions

Patient journey from a clinician perspective: the Pre-Tell

survey

Patient journey from a patient

perspective: the EMPOWER study

Final remarks

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Continuum of Care of individuals born preterm

HCPs

Patients

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HCPs Insight gathering

Scientific board
•Alvar Agusti •Eugenio Baraldi
•Andrew Bush •Fulvio Braido
•Anne Greenough •Klaus Rabe
•Daiana Stolz •Leonardo Fabbri

Allergologists
Neonatologists Pulmonologists

Pediatricians

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April September
HCPs Insight gathering: PRE-TELL survey

Survey with a common part and specific sections

according to the HCP specialty

Four main macro topics:

1) awareness level of prematurity as a risk factor

2) Neonatal information: how they are shared and

collected

3) Any difference in approach if the patient was

born premature

4) Main gaps in the journey of these individuals

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HCPs Insight gathering: PRE-TELL survey

1002 healthcare providers completed the survey (n=282 neonatologists; n=183
paediatricians; n=247 respiratory consultants; and n=290 allergologists)

PRETERM INDIVIDUALS’ PATIENT JOURNEY

Based on the survey

results we have
identified main pain
points in the journey

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HCPs Insight gathering: Pre-Tell Advisory board

The medical community lacks
01 awareness on risk factors to develop The medical community lacks
respiratory dysfunction
01 awareness on risk factors to
There are no clear recommendations to do
develop respiratory dysfunction
02 regular respiratory check-ups for individuals
born preterm Continuous Medical Education
Awareness campaigns
There are no clear guidelines on when to Data generation and
03 refer premature patients to a pulmonary
dissemination
specialist

Once discharged from the care of

neonatologists and primary care pediatricians,
Preterm born individuals are
04 preterm individuals are not regularly followed
05 unaware of the risks of prematurity
by a doctor for their long-term health

Preterm born individuals are unaware of Parents and patients

05 the risks of prematurity for their long-
education and
term health
empowerment
The healthcare system organization does not
06 allow us to efficiently track the medical history of
15 such patients
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Table of Contents

Prematurity as a predisposing risk factor of adult chronic ì

respiratory conditions

Patient journey from a clinician perspective: the Pre-Tell

survey

Patient journey from a patient

perspective: the EMPOWER study

Final remarks

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Real-world Patient Research in Prematurity and respiratory diseases

Voice of adult individuals born prematurely and who have been diagnosed with
asthma and or COPD

❑ Awarness of their premature history

❑ Interaction with HCPs
❑ Main pain points in their journey
❑ QoL

18 yo

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Real-world Patient Research in Prematurity and respiratory diseases

Study design
▪ International cross-sectional study – Online questionnaire
STUDY BOARD ▪ Development of questionnaire for patient online self-administration
▪ Duration of questionnaire: LOI=20 min – screener + up to 35
questions including 3 open-ended questions

Lauren Ingledow Target Population

▪ Adult preterm patients who had asthma or COPD, living in the 4
european countries (France, Spain, Germany, United Kingdom) or
Juliette Lee in the US.
▪ Other Chronic respiratory disease not excluded
Giovanni Quaglia Sample Size
▪ Target : n=300 respondents (135 with asthma, 135 with COPD and
Josh Dalton 30 with asthma and COPD, homogeneously distributed in each
country)

Approval by an ethic committee (IRB)

Data collection estimation : 4 months

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Table of Contents

Prematurity as a predisposing risk factor of adult chronic

respiratory conditions

Patient journey from a clinician perspective: the Pre-Tell

survey

Patient journey from a patient

perspective: the EMPOWER study

Final remarks

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FINAL REMARKS
▪ Prematurity per se, not only BPD is a risk factor for asthma and COPD

▪ Much needs to be done to increase the awareness in particular among

pulmunologists

▪ More research is needed to understand if their phenotype responds differently

to different

▪ Parents need to be empowered to bring up the medical history of their children

with the different physicians that will visit their children

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Thank You