TY - JOUR T1 - Autoimmune PAP (aPAP) in children JF - ERJ Open Research JO - erjor DO - 10.1183/23120541.00701-2021 SP - 00701-2021 AU - Matthias Griese AU - Panagiota Panagiotou AU - Effrosyni D. Manali AU - Mirjam Stahl AU - Nicolaus Schwerk AU - Vanessa Costa AU - Konstantinos Douros AU - Maria Kallieri AU - Ruth Maria Urbantat AU - Horst von Bernuth AU - Lykourgos Kolilekas AU - Lurdes Morais AU - Ana Ramos AU - Kerstin Landwehr AU - Katrin Knoflach AU - Florian Gothe AU - Karl Reiter AU - Vassiliki Papaevangelou AU - Athanasios G. Kaditis AU - Christina Kanaka-Gantenbein AU - Spyros A. Papiris Y1 - 2022/01/01 UR - http://openres.ersjournals.com/content/early/2022/01/27/23120541.00701-2021.abstract N2 - In childhood, a multitude of causes leads to pulmonary alveolar proteinosis (PAP), an excessive surfactant accumulation in the alveolar space limiting gas exchange. Autoantibodies against GM-CSF causing autoimmune PAP, the principle etiology in adults, are rare.In this first series on autoimmune PAP we detail the presentation and management issues of four children.Whereas three children presented insidiously with progressive dyspnea, one was acutely sick with suspected pneumonia. During management, one patient was hospitalised with COVID-19, non-invasively ventilated, and recovered. All treatment modalities known from adults including whole lung lavages, augmentation of GM-CSF by inhaled GM-CSF, removal of neutralising antibody by plasmapheresis and interruption of antibody production by Rituximab were considered, however not all options were available at all sites. Inhaled GM-CSF appeared a non-invasive 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 authorisation of novel treatments for autoimmune PAP competent authorities should grant an inclusion of adolescents into trials in adults.FootnotesThis manuscript has recently been accepted for publication in the ERJ Open Research. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJOR online. Please open or download the PDF to view this article.Conflict of interest: Dr. Griese reports receiving grants or contracts received from Böhringer Ingelheim paid to their institution. Participation on a Data Safety Monitoring Board or Advisory Board for Böhringer Ingelheim, personal fees received. Unpaid Head chILD-EU. All disclosures made outside the submitted work.Conflict of interest: Dr. MANALI reports receiving grants or contracts, paid to the institution, from HOFFMANN LA ROCHE, BOEHRINGER INGELHEIM and SAVARA. Personal payments received for lectures, presentations, speakers bureaus, manuscript writing or educational events received from HOFFMANN LA ROCHE and BOEHRINGER INGELHEIM. Support for attending meetings and/or travel paid to the institution received from HOFFMANN LA ROCHE and BOEHRINGER INGELHEIM. All disclosures made outside the submitted work.Conflict of interest: Dr. PAPIRIS reports receiving grants or contracts paid to the institution from HOFFMANN LA ROCHE, BOEHRINGER INGELHEIM and SAVARA. Personal payments received from HOFFMANN LA ROCHE and BOEHRINGER INGELHEIM for attending meetings and/or travel. All disclosures made outside the submitted work.Conflict of interest: Dr. von Bernuth reports receiving payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from CSL Behring and Octapharma. Participation on an Advisory Board for Takeda. Associate member for the Standing committee on vaccinations. All disclosures made outside the submitted work.Conflict of interest: Dr. Panagiotou has nothing to discloseConflict of interest: Dr. Stahl has nothing to discloseConflict of interest: Dr. Schwerk has nothing to discloseConflict of interest: Dr. Costa has nothing to discloseConflict of interest: Dr. Douros has nothing to discloseConflict of interest: Dr. Kallieri has nothing to discloseConflict of interest: Dr. Urbantat has nothing to discloseConflict of interest: Dr. Kolilekas has nothing to discloseConflict of interest: Dr. Morais has nothing to discloseConflict of interest: Dr. Ramos has nothing to discloseConflict of interest: Dr. Landwehr has nothing to discloseConflict of interest: Dr. Knoflach has nothing to discloseConflict of interest: Dr. Gothe has nothing to discloseConflict of interest: Dr. Reiter has nothing to discloseConflict of interest: Dr. Papaevangelou has nothing to discloseConflict of interest: Dr. Kaditis has nothing to discloseConflict of interest: Dr. Kanaka-Gantenbein has nothing to disclose ER -