Respiratory Treatment Burden and Meaningful Change: Interviews with Caregivers of Extremely Premature Infants to Inform A Phase 2B Clinical Trial Endpoint

Introduction Extremely premature (EP) infants (born 23 to <28 weeks’ gestational age) who continue to experience respiratory complications postdischarge from the neonatal intensive care unit (NICU) may utilize multiple medical resources, including emergency room (ER) visits, inpatient hospital admissions, home respiratory technology support (RTS), and respiratory medications. This study explored post-NICU discharge respiratory burden and perceptions of meaningful change among caregivers of EP infants in the United States and Europe to inform the primary endpoint of a Phase 2b study (NCT03253263).

Materials and Methods Adult primary caregivers of EP infants in the United States, Northern Ireland, and Germany were recruited through patient advocacy organizations and interviewed by phone. Caregivers were included if their infant (3–14 months corrected age), post-NICU discharge, experienced ≥1 of: ER visit/rehospitalization due to respiratory diagnosis, RTS (e.g., supplemental oxygen, breathing/heart rate monitor, and tracheostomy), respiratory medications (e.g., nebulizer, steroids, and diuretics). Interviews explored caregiver experiences with infants with respiratory issues, associated treatment burden, and meaningful change in reducing burden of treatment modalities. Sociodemographic data were summarized using descriptive statistics, and qualitative analysis of the interview data was performed.

Results A total of 40 caregivers (95% females; mean ± standard deviation age: 31.7 ± 5.0 years) of EP infants (65% females) were interviewed. Respiratory morbidities reported by caregivers post-NICU discharge included difficulty/changes in breathing (82.5%), bronchopulmonary dysplasia (60%), respiratory infections (60%), and apnea (32.5%). Infants with respiratory morbidities experienced medication use (92.5%), RTS (82.5%), hospitalizations (37.5%), and ER visits (35%). [Table A003] lists the top three self-reported negative impacts of each of these. Caregivers considered supplemental oxygen as the most burdensome treatment. They most wanted to avoid RTS (tracheostomy and home ventilator use), hospitalizations, and ER visits. Reduced need for oxygen, less frequent administration of medications, and reduced hospitalizations were considered the most meaningful treatment changes. This study found that all respiratory treatment modalities explored (RTS, ER visits, hospitalizations, and medication use) carried a negative impact and were burdensome.

Conclusion Reductions in the use of these treatment modalities would be a meaningful benefit to patients and their caregivers. The small sample size from three countries, limits the generalizability of our results.

Table A003

Impacts, n (%)	Total (n = 40)
Respiratory medications	n = 37
Difficulty administering	14 (37.8)
Requires planning/scheduling/remembering	8 (21.6)
Stress of missing a dose	7 (18.9)
Home respiratory technology support	n = 33
Effects on infant’s mobility/development	17 (51.5)
Sleep	13 (39.4)
Hassle/annoying	13 (39.4)
Hospitalization	n = 15
Caring for other children	4 (26.7)
Frustrating	2 (13.3)
Missed work	2 (13.3)
Emergency room visits	n = 14
Fear/worry	7 (50.0)
Sleep	3 (21.4)
Impacts other children/family	2 (14.3)

Conflict of Interest

None declared.