Management and Long-Term Outcomes of Persistent Tachypnea of Infancy/Neuroendocrine Cell Hyperplasia of Infancy: A European Multicenter Retrospective Study.
Publisher DOI
PubMed ID
41687810
Abstract
Background
Persistent tachypnea of infancy (PTI), also known as neuroendocrine cell hyperplasia of infancy (NEHI), represents one 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, 240/378) from 73 centers across 17 countries were enrolled, with a median age at diagnosis of 9 months (IQR, 6-13 months). Therapeutic interventions included oxygen supplementation (75.9%, 287/378), inhaled bronchodilators and/or inhaled glucocorticoids (62.4%, 236/378), systemic glucocorticoids (37.0%, 140/378), and nutritional support (33.8%, 128/378). Of the children who received oxygen therapy, 53.6% (154/287) 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<0.05). Longitudinal data were available for 48.9% of patients (185/378) 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 was also reported; however, a subset of patients continued to demonstrate persistent hypoxemia, crackles, exercise intolerance, as well as abnormal imaging and pulmonary function into adolescence.Interpretation
Significant differences in treatment strategies for PTI/NEHI were observed across European countries, highlighting the need for evidence-based guidelines. While long-term prognosis is generally favorable, residual symptoms remain in some patients, warranting continued follow-up.
Persistent tachypnea of infancy (PTI), also known as neuroendocrine cell hyperplasia of infancy (NEHI), represents one 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, 240/378) from 73 centers across 17 countries were enrolled, with a median age at diagnosis of 9 months (IQR, 6-13 months). Therapeutic interventions included oxygen supplementation (75.9%, 287/378), inhaled bronchodilators and/or inhaled glucocorticoids (62.4%, 236/378), systemic glucocorticoids (37.0%, 140/378), and nutritional support (33.8%, 128/378). Of the children who received oxygen therapy, 53.6% (154/287) 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<0.05). Longitudinal data were available for 48.9% of patients (185/378) 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 was also reported; however, a subset of patients continued to demonstrate persistent hypoxemia, crackles, exercise intolerance, as well as abnormal imaging and pulmonary function into adolescence.Interpretation
Significant differences in treatment strategies for PTI/NEHI were observed across European countries, highlighting the need for evidence-based guidelines. While long-term prognosis is generally favorable, residual symptoms remain in some patients, warranting continued follow-up.
Date Issued
2026-06
Publication Type
Article
Subject(s)
Language(s)
en
Author(s)
Marczak, Honorata | |
Krenke, Katarzyna | |
Griese, Matthias | |
Peradzyńska, Joanna | |
Lange, Joanna | |
Kulus, Marek | |
Grochowska, Magdalena | |
Seidl, Elias | |
Dubus, Jean-Christophe | |
Rodler, Julia | |
Schwerk, Nicolaus | |
Carlens, Julia | |
Breuer, Oded | |
Hevroni, Avigdor | |
Castillo-Corullón, Silvia | |
Aldeco, Malena | |
Buchvald, Frederik Fouirnaies | |
Nielsen, Kim G | |
Mayell, Sarah | |
Torrent, Alba | |
Bravo-López, Maynor | |
Stehling, Florian | |
Zschocke, Anna | |
Hatziagorou, Elpis | |
Ayats, Roser | |
Aslan, Ayse Tana | |
Erdem, Ela | |
Proesmans, Marijke | |
Cunningham, Steve | |
Moriki, Dafni | |
Pekcan, Sevgi | |
Cobanoglu, Nazan | |
Naehrlich, Lutz | |
Lex, Christiane | |
Ullmann, Nicola | |
Baden, Winfried | |
Krikovszky, Dora | |
Gaboli, Mirella | |
Monge, Nuria Diez | |
Vivas, David Naranjo | |
Mørck Rubak, Sune Leisgaard | |
Willemse, Brigitte | |
Petrarca, Laura | |
Wiemers, Anna | |
Schramm, Dirk | |
Mueller, Christoph | |
Prenzel, Freerk | |
Koucky, Vaclav | |
López-Andreu, Juan A | |
Nathan, Nadia |
Additional Credits
Journal
Chest Journal
Publisher
Elsevier
ISSN
1931-3543
0012-3692
Access(Rights)
embargo