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New insights into pediatric idiopathic pulmonary hemosiderosis: the French RespiRare(®) cohort.

Taytard J, Nathan N, de Blic J, Fayon M, Epaud R, Deschildre A, Troussier F, Lubrano M, Chiron R, Reix P, Cros P, Mahloul M, Michon D, Clement A, Corvol H, French RespiRare® gro - Orphanet J Rare Dis (2013)

Bottom Line: In 13 cases, immunosuppressants were introduced due to corticoresistance and/or major side effects.One patient developed severe pulmonary fibrosis, and another with Down syndrome died as a result of severe pulmonary hemorrhage.The present cohort provides substantial information on clinical expression and outcomes of pediatric IPH.

View Article: PubMed Central - HTML - PubMed

Affiliation: Pediatric Pulmonary Department, AP-HP, Hôpital Trousseau, Université Pierre et Marie Curie-Paris6, Inserm U938, 26, avenue du Docteur Arnold-Netter, 75012 Paris, France. harriet.corvol@trs.aphp.fr.

ABSTRACT

Background: Idiopathic pulmonary hemosiderosis (IPH) is a rare cause of alveolar hemorrhage in children and its pathophysiology remains obscure. Classically, diagnosis is based on a triad including hemoptysis, diffuse parenchymal infiltrates on chest X-rays, and iron-deficiency anemia. We present the French pediatric cohort of IPH collected through the French Reference Center for Rare Lung Diseases (RespiRare®, http://www.respirare.fr).

Methods: Since 2008, a national network/web-linked RespiRare® database has been set up in 12 French pediatric respiratory centres. It is structured as a medical recording tool with extended disease-specific datasets containing clinical information relevant to all forms of rare lung diseases including IPH.

Results: We identified 25 reported cases of IPH in children from the database (20 females and 5 males). Among them, 5 presented with Down syndrome. Upon diagnosis, median age was 4.3 [0.8-14.0] yrs, and the main manifestations were: dyspnea (n = 17, 68%), anemia (n = 16, 64%), cough (n = 12, 48%), febrile pneumonia (n = 11, 44%) and hemoptysis (n = 11, 44%). Half of the patients demonstrated diffuse parenchymal infiltrates on chest imaging, and diagnosis was ascertained either by broncho-alveolar lavage indicating the presence of hemosiderin-laden macrophages (19/25 cases), or lung biopsy (6/25). In screened patients, initial auto-immune screening revealed positive antineutrophilic cytoplasmic antibodies (ANCA) (n = 6, 40%), antinuclear antibodies (ANA) (n = 5, 45%) and specific coeliac disease antibodies (n = 4, 28%). All the patients were initially treated by corticosteroids. In 13 cases, immunosuppressants were introduced due to corticoresistance and/or major side effects. Median length of follow-up was 5.5 yrs, with a satisfactory respiratory outcome in 23/25 patients. One patient developed severe pulmonary fibrosis, and another with Down syndrome died as a result of severe pulmonary hemorrhage.

Conclusion: The present cohort provides substantial information on clinical expression and outcomes of pediatric IPH. Analysis of potential contributors supports a role of auto-immunity in disease development and highlights the importance of genetic factors.

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Related in: MedlinePlus

Thoracic imaging. a shows ground-glass opacities on a chest X-ray in a 5 yrs old IPH patient. b shows on a chest CT-scan an interstitial pattern with micronodules and ground-glass opacities in a 5 yrs old IPH patient.
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Figure 1: Thoracic imaging. a shows ground-glass opacities on a chest X-ray in a 5 yrs old IPH patient. b shows on a chest CT-scan an interstitial pattern with micronodules and ground-glass opacities in a 5 yrs old IPH patient.

Mentions: Interstitial and/or alveolar interstitial patterns were visualized by chest X-rays for 13 patients and by chest CT-scans for 17 patients. The chest CT scans showed ground-glass opacities (12 patients), sub pleural cysts (6 patients), and, less frequently, micronodules (2 patients), thickened interlobular septa (2 patients), honeycomb patterns (1 patient), and traction bronchiectasis (1 patient). Images were often diffuse and bilateral. An illustration is given in Figure 1.


New insights into pediatric idiopathic pulmonary hemosiderosis: the French RespiRare(®) cohort.

Taytard J, Nathan N, de Blic J, Fayon M, Epaud R, Deschildre A, Troussier F, Lubrano M, Chiron R, Reix P, Cros P, Mahloul M, Michon D, Clement A, Corvol H, French RespiRare® gro - Orphanet J Rare Dis (2013)

Thoracic imaging. a shows ground-glass opacities on a chest X-ray in a 5 yrs old IPH patient. b shows on a chest CT-scan an interstitial pattern with micronodules and ground-glass opacities in a 5 yrs old IPH patient.
© Copyright Policy - open-access
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC3852822&req=5

Figure 1: Thoracic imaging. a shows ground-glass opacities on a chest X-ray in a 5 yrs old IPH patient. b shows on a chest CT-scan an interstitial pattern with micronodules and ground-glass opacities in a 5 yrs old IPH patient.
Mentions: Interstitial and/or alveolar interstitial patterns were visualized by chest X-rays for 13 patients and by chest CT-scans for 17 patients. The chest CT scans showed ground-glass opacities (12 patients), sub pleural cysts (6 patients), and, less frequently, micronodules (2 patients), thickened interlobular septa (2 patients), honeycomb patterns (1 patient), and traction bronchiectasis (1 patient). Images were often diffuse and bilateral. An illustration is given in Figure 1.

Bottom Line: In 13 cases, immunosuppressants were introduced due to corticoresistance and/or major side effects.One patient developed severe pulmonary fibrosis, and another with Down syndrome died as a result of severe pulmonary hemorrhage.The present cohort provides substantial information on clinical expression and outcomes of pediatric IPH.

View Article: PubMed Central - HTML - PubMed

Affiliation: Pediatric Pulmonary Department, AP-HP, Hôpital Trousseau, Université Pierre et Marie Curie-Paris6, Inserm U938, 26, avenue du Docteur Arnold-Netter, 75012 Paris, France. harriet.corvol@trs.aphp.fr.

ABSTRACT

Background: Idiopathic pulmonary hemosiderosis (IPH) is a rare cause of alveolar hemorrhage in children and its pathophysiology remains obscure. Classically, diagnosis is based on a triad including hemoptysis, diffuse parenchymal infiltrates on chest X-rays, and iron-deficiency anemia. We present the French pediatric cohort of IPH collected through the French Reference Center for Rare Lung Diseases (RespiRare®, http://www.respirare.fr).

Methods: Since 2008, a national network/web-linked RespiRare® database has been set up in 12 French pediatric respiratory centres. It is structured as a medical recording tool with extended disease-specific datasets containing clinical information relevant to all forms of rare lung diseases including IPH.

Results: We identified 25 reported cases of IPH in children from the database (20 females and 5 males). Among them, 5 presented with Down syndrome. Upon diagnosis, median age was 4.3 [0.8-14.0] yrs, and the main manifestations were: dyspnea (n = 17, 68%), anemia (n = 16, 64%), cough (n = 12, 48%), febrile pneumonia (n = 11, 44%) and hemoptysis (n = 11, 44%). Half of the patients demonstrated diffuse parenchymal infiltrates on chest imaging, and diagnosis was ascertained either by broncho-alveolar lavage indicating the presence of hemosiderin-laden macrophages (19/25 cases), or lung biopsy (6/25). In screened patients, initial auto-immune screening revealed positive antineutrophilic cytoplasmic antibodies (ANCA) (n = 6, 40%), antinuclear antibodies (ANA) (n = 5, 45%) and specific coeliac disease antibodies (n = 4, 28%). All the patients were initially treated by corticosteroids. In 13 cases, immunosuppressants were introduced due to corticoresistance and/or major side effects. Median length of follow-up was 5.5 yrs, with a satisfactory respiratory outcome in 23/25 patients. One patient developed severe pulmonary fibrosis, and another with Down syndrome died as a result of severe pulmonary hemorrhage.

Conclusion: The present cohort provides substantial information on clinical expression and outcomes of pediatric IPH. Analysis of potential contributors supports a role of auto-immunity in disease development and highlights the importance of genetic factors.

Show MeSH
Related in: MedlinePlus