Management and Long-Term Outcomes of Persistent Tachypnea of Infancy/Neuroendocrine Cell Hyperplasia of Infancy: A European Multicenter Retrospective Study.

BACKGROUND

Persistent tachypnea of infancy (PTI), also known as neuroendocrine cell hyperplasia of infancy (NEHI), represents 1 of the most common childhood interstitial lung diseases. Despite its frequency, standardized management protocol is lacking, and long-term outcome data remain limited.

RESEARCH QUESTION: What treatment is used for patients with PTI/NEHI, how does clinical management vary across European countries, and what are the long-term outcomes in affected patients?

STUDY DESIGN AND

METHODS

This was a European multicenter, retrospective, observational study. Clinical characteristics, therapeutic interventions, and long-term follow-up data were collected and analyzed. Treatment strategies were compared among countries that contributed at least 10 patients.

RESULTS

A total of 378 children (63.5% male [240 of 378]) from 73 centers across 17 countries were enrolled, with a median age at diagnosis of 9 months (interquartile range [IQR], 6-13 months). Therapeutic interventions included oxygen supplementation (75.9% [287 of 378]); inhaled bronchodilators, inhaled glucocorticoids, or both (62.4% [236 of 378]); systemic glucocorticoids (37.0% [140 of 378]); and nutritional support (33.8% [128 of 378]). Of the children who received oxygen therapy, 53.6% (154 of 287) were reported to have been weaned off, with a median age at weaning of 24 months (IQR, 16-36 months). Marked variability in treatment practices was observed across participating countries (P < .05). Longitudinal data were available for 48.9% of patients (185 of 378) with a median follow-up of 19 months (IQR, 16-57 months). The proportion of symptomatic children declined over time, with the most marked improvement observed at 4 years of age. Resolution of imaging and pulmonary function abnormalities also was reported; however, a subset of patients continued to demonstrate persistent hypoxemia, crackles, and exercise intolerance, as well as abnormal imaging and pulmonary function test findings into adolescence.

INTERPRETATION: Our results show that significant differences in treatment strategies for PTI/NEHI were observed across European countries, highlighting the need for evidence-based guidelines. Although long-term prognosis generally is favorable, residual symptoms remain in some patients, warranting continued follow-up.