Successful Treatment of Autoimmune Pulmonary Alveolar Proteinosis in a Pediatric Patient.
BACKGROUND Pulmonary alveolar proteinosis (PAP) is a rare condition characterized by the intra-alveolar accumulation of surfactant-derived material, which impairs gas exchange and results in respiratory insufficiency. Two major subtypes of PAP are autoimmune and non-autoimmune PAP. The diagnosis relies on clinical presentation, ground glass opacities on CT scan, bronchoscopy with PAS stain of BAL fluid (BALF), lung biopsy with PAS-positive material in the alveoli, and the presence of anti GM-CSF antibodies in serum or BALF for an autoimmune subtype. The therapeutic approach to pediatric cases varies according to age and the general clinical state of the child; however, whole lung lavage (WLL) and inhaled or subcutaneous GM-CSF are generally first-line therapy. CASE REPORT We report a unique case of an autoimmune type of PAP in a 12-year-old boy, who underwent successful bilateral lung transplantation after inefficacious treatment with GM-CSF, and who developed post-transplant lymphoproliferative disease (PTLD) and was successfully treated with a chemotherapeutic protocol. CONCLUSIONS Although lung transplantation is a rarely used therapeutic approach for patients with an autoimmune subtype of PAP, in cases of inefficacious treatment with other modalities, lung transplantation should be considered.
Department of Pediatric Allergology and Pulmonology, Children's Hospital Srebrnjak, Zagreb, Croatia.
Mirjana
M
Trukalj
Department of Pediatric Allergology and Pulmonology, Children's Hospital Srebrnjak, Zagreb, Croatia.
Marija
M
Perica
Department of Pediatric Allergology and Pulmonology, Children's Hospital Srebrnjak, Zagreb, Croatia.
Željko
Ž
Ferenčić
Department of Pediatric Allergology and Pulmonology, Children's Hospital Srebrnjak, Zagreb, Croatia.
Damir
D
Erceg
Department of Pediatric Allergology and Pulmonology, Children's Hospital Srebrnjak, Zagreb, Croatia.
Marta
M
Navratil
Department of Pulomonology, University Hospital Zagreb, Zagreb, Croatia.
Gzim
G
Redžepi
Department of Pediatric Allergology and Pulmonology, Children's Hospital Srebrnjak, Zagreb, Croatia.
Boro
B
Nogalo
D001327:Autoimmune Diseases; D001706:Biopsy; D018893:Bronchoalveolar Lavage / Q000379:methods*; D001999:Bronchoscopy; D002675:Child, Preschool; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D008168:Lung / Q000000981:diagnostic imaging*; D008297:Male; D011649:Pulmonary Alveolar Proteinosis / Q000175:diagnosis / Q000276:immunology / Q000628:therapy*; D014057:Tomography, X-Ray Computed
D016178:Granulocyte-Macrophage Colony-Stimulating Factor