Autoimmune PAP (aPAP) in children

Griese, Matthias; Panagiotou, Panagiota; Manali, Effrosyni D.; Stahl, Mirjam; Schwerk, Nicolaus; Costa, Vanessa; Douros, Konstantinos; Kallieri, Maria; Urbantat, Ruth Maria; von Bernuth, Horst; Kolilekas, Lykourgos; Morais, Lurdes; Ramos, Ana; Landwehr, Kerstin; Knoflach, Katrin; Gothe, Florian; Reiter, Karl; Papaevangelou, Vassiliki; Kaditis, Athanasios G.; Kanaka-Gantenbein, Christina; Papiris, Spyros A. (2022): Autoimmune PAP (aPAP) in children. ERJ Open Research. pp. 1-6. ISSN 2312-0541

Abstract

In childhood, a multitude of causes lead to pulmonary alveolar proteinosis (PAP), an excessive surfactant accumulation in the alveolar space, limiting gas exchange. Autoantibodies against granulocyte–macrophage colony-stimulating factor (GM-CSF) causing autoimmune PAP, the principal aetiology in adults, are rare.

In this first case series on autoimmune PAP, we detail the presentation and management issues of four children.

Whereas three children presented insidiously with progressive dyspnoea, one was acutely sick with suspected pneumonia. During management, one patient was hospitalised with coronavirus disease 2019, noninvasively ventilated, and recovered. All treatment modalities known from adults including whole-lung lavage, augmentation of GM-CSF by inhaled GM-CSF, removal of neutralising antibody by plasmapheresis and interruption of antibody production using rituximab were considered; however, not all options were available at all sites. Inhaled GM-CSF appeared to be a noninvasive and comfortable therapeutic approach.

The management with best benefit-to-harm ratio in autoimmune PAP is unknown and specialised physicians must select the least invasive and most effective treatment. To collect this cohort in a rare condition became feasible as patients were submitted to an appropriate registry. To accelerate the authorisation of novel treatments for autoimmune PAP, competent authorities should grant an inclusion of adolescents into trials in adults.

• Doc-Type: Article (LMU Hospital)
• Organisational unit (Faculties): 07 Medicine > Medical Center of the University of Munich > Pediatric Clinic and Outpatient Clinic in the Dr. von Hauner Children‘s Hospital
• DFG subject classification of scientific disciplines: Life sciences
• DFG: Funded by the Deutsche Forschungsgemeinschaft (DFG, German Research Foundation) - 413551332
• DFG: Funded by the Deutsche Forschungsgemeinschaft (DFG, German Research Foundation) - 491502892