Abstract
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.
Footnotes
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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 disclose
Conflict of interest: Dr. Stahl has nothing to disclose
Conflict of interest: Dr. Schwerk has nothing to disclose
Conflict of interest: Dr. Costa has nothing to disclose
Conflict of interest: Dr. Douros has nothing to disclose
Conflict of interest: Dr. Kallieri has nothing to disclose
Conflict of interest: Dr. Urbantat has nothing to disclose
Conflict of interest: Dr. Kolilekas has nothing to disclose
Conflict of interest: Dr. Morais has nothing to disclose
Conflict of interest: Dr. Ramos has nothing to disclose
Conflict of interest: Dr. Landwehr has nothing to disclose
Conflict of interest: Dr. Knoflach has nothing to disclose
Conflict of interest: Dr. Gothe has nothing to disclose
Conflict of interest: Dr. Reiter has nothing to disclose
Conflict of interest: Dr. Papaevangelou has nothing to disclose
Conflict of interest: Dr. Kaditis has nothing to disclose
Conflict of interest: Dr. Kanaka-Gantenbein has nothing to disclose
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- Received December 14, 2021.
- Accepted January 19, 2022.
- Copyright ©The authors 2022
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