TY - JOUR

T1 - Diffuse alveolar haemorrhage in children

T2 - an international multicentre study

AU - Ring, Astrid Madsen

AU - Schwerk, Nicolaus

AU - Kiper, Nural

AU - Aslan, Ayse Tana

AU - Aurora, Paul

AU - Ayats, Roser

AU - Azevedo, Ines

AU - Bandeira, Teresa

AU - Carlens, Julia

AU - Castillo-Corullon, Silvia

AU - Cobanoglu, Nazan

AU - Elnazir, Basil

AU - Emiralioğlu, Nagehan

AU - Eyuboglu, Tugba Sismanlar

AU - Fayon, Michael

AU - Gursoy, Tugba Ramaslı

AU - Hogg, Claire

AU - Kötz, Karsten

AU - Karadag, Bülent

AU - Látalová, Vendula

AU - Krenke, Katarzyna

AU - Lange, Joanna

AU - Manali, Effrosyni D.

AU - Osona, Borja

AU - Papiris, Spyros

AU - Proesmann, Marijke

AU - Reix, Philippe

AU - Roditis, Lea

AU - Rubak, Sune

AU - Rumman, Nisreen

AU - Snijders, Deborah

AU - Stehling, Florian

AU - Weiss, Laurence

AU - Yalcın, Ebru

AU - Zirek, Fazilcan

AU - Bush, Andrew

AU - Clement, Annick

AU - Griese, Matthias

AU - Buchvald, Frederik Fouirnaies

AU - Nathan, Nadia

AU - Nielsen, Kim Gjerum

N1 - Publisher Copyright: © The authors 2023.

PY - 2023

Y1 - 2023

N2 - Background Paediatric diffuse alveolar haemorrhage (DAH) is a rare heterogeneous condition with limited knowledge on clinical presentation, treatment and outcome. Methods A retrospective, descriptive multicentre follow-up study initiated from the European network for translational research in children’s and adult interstitial lung disease (Cost Action CA16125) and chILD-EU CRC (the European Research Collaboration for Children’s Interstitial Lung Disease). Inclusion criteria were DAH of any cause diagnosed before the age of 18 years. Results Data of 124 patients from 26 centres (15 counties) were submitted, of whom 117 patients fulfilled the inclusion criteria. Diagnoses were idiopathic pulmonary haemosiderosis (n=35), DAH associated with autoimmune features (n=20), systemic and collagen disorders (n=18), immuno-allergic conditions (n=10), other childhood interstitial lung diseases (chILD) (n=5), autoinflammatory diseases (n=3), DAH secondary to other conditions (n=21) and nonspecified DAH (n=5). Median (IQR) age at onset was 5 (2.0–12.9) years. Most frequent clinical presentations were anaemia (87%), haemoptysis (42%), dyspnoea (35%) and cough (32%). Respiratory symptoms were absent in 23%. The most frequent medical treatment was systemic corticosteroids (93%), hydroxychloroquine (35%) and azathioprine (27%). Overall mortality was 13%. Long-term data demonstrated persistent abnormal radiology and a limited improvement in lung function. Conclusions Paediatric DAH is highly heterogeneous regarding underlying causes and clinical presentation. The high mortality rate and number of patients with ongoing treatment years after onset of disease underline that DAH is a severe and often chronic condition. This large international study paves the way for further prospective clinical trials that will in the long term allow evidence-based treatment and follow-up recommendations to be determined.

AB - Background Paediatric diffuse alveolar haemorrhage (DAH) is a rare heterogeneous condition with limited knowledge on clinical presentation, treatment and outcome. Methods A retrospective, descriptive multicentre follow-up study initiated from the European network for translational research in children’s and adult interstitial lung disease (Cost Action CA16125) and chILD-EU CRC (the European Research Collaboration for Children’s Interstitial Lung Disease). Inclusion criteria were DAH of any cause diagnosed before the age of 18 years. Results Data of 124 patients from 26 centres (15 counties) were submitted, of whom 117 patients fulfilled the inclusion criteria. Diagnoses were idiopathic pulmonary haemosiderosis (n=35), DAH associated with autoimmune features (n=20), systemic and collagen disorders (n=18), immuno-allergic conditions (n=10), other childhood interstitial lung diseases (chILD) (n=5), autoinflammatory diseases (n=3), DAH secondary to other conditions (n=21) and nonspecified DAH (n=5). Median (IQR) age at onset was 5 (2.0–12.9) years. Most frequent clinical presentations were anaemia (87%), haemoptysis (42%), dyspnoea (35%) and cough (32%). Respiratory symptoms were absent in 23%. The most frequent medical treatment was systemic corticosteroids (93%), hydroxychloroquine (35%) and azathioprine (27%). Overall mortality was 13%. Long-term data demonstrated persistent abnormal radiology and a limited improvement in lung function. Conclusions Paediatric DAH is highly heterogeneous regarding underlying causes and clinical presentation. The high mortality rate and number of patients with ongoing treatment years after onset of disease underline that DAH is a severe and often chronic condition. This large international study paves the way for further prospective clinical trials that will in the long term allow evidence-based treatment and follow-up recommendations to be determined.

U2 - 10.1183/23120541.00733-2022

DO - 10.1183/23120541.00733-2022

M3 - Journal article

C2 - 37101741

AN - SCOPUS:85159188757

VL - 9

JO - ERJ Open Research

JF - ERJ Open Research

SN - 2312-0541

IS - 2

M1 - 00733-2022

ER -