RT Journal Article
SR Electronic
T1 Autoimmune PAP (aPAP) in children
JF ERJ Open Research
JO erjor
FD European Respiratory Society
SP 00701-2021
DO 10.1183/23120541.00701-2021
A1 Matthias Griese
A1 Panagiota Panagiotou
A1 Effrosyni D. Manali
A1 Mirjam Stahl
A1 Nicolaus Schwerk
A1 Vanessa Costa
A1 Konstantinos Douros
A1 Maria Kallieri
A1 Ruth Maria Urbantat
A1 Horst von Bernuth
A1 Lykourgos Kolilekas
A1 Lurdes Morais
A1 Ana Ramos
A1 Kerstin Landwehr
A1 Katrin Knoflach
A1 Florian Gothe
A1 Karl Reiter
A1 Vassiliki Papaevangelou
A1 Athanasios G. Kaditis
A1 Christina Kanaka-Gantenbein
A1 Spyros A. Papiris
YR 2022
UL http://openres.ersjournals.com/content/early/2022/01/27/23120541.00701-2021.abstract
AB 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