Background and Importance Autoimmune pulmonary alveolar proteinosis (aPAP) is a disease caused by IgG antibodies against granulocyte-macrophage colony-stimulating factor (GM-CSF). The treatment of choice is bronchoalveolar lavage (BAL).
Aim and Objectives To present aPAP evolution during 24 months of treatment with inhaled GM-CSF.
Material and Methods A 37-year-old male diagnosed with aPAP in April 2014 has required 3 BAL (October 2014, February 2016 and March 2019). In the last BAL, he developed major complications that required admission to the intensive care unit. In October 2019, he presented a new worsening, so off-label treatment with sargramostim (250µg inhaled every 12 hours for 7 days every other week) was chosen.
The pharmacy service prepared a favorable report on off-label treatment and requested drug from regulatory agency. In the pharmaceutical care consultation, the administration technique, stability, dosage regimen, storage (2-8°C) were explained and doubts were resolved.
Results The clinical and functional evolution of the ventilatory parameters and the six-minute walk test are shown in the table 1.
The figure 1 shows the radiological evolution (chest X-ray) from the situation before third BAL (1), further worsening after 7 months after third BAL (2), improvement after 3 months of treatment with inhaled GM-CSF (3) and stability after 18 months of treatment (4).
After 24 months of treatment, the patient has not presented any adverse events and maintains an excellent response with significant improvement in gas exchange, which has allowed home oxygen therapy to be withdrawn.
Conclusion and Relevance In conclusion, our case supports that inhaled GM-CSF has been safe and effective in the treatment of aPAP and represents a therapeutic option after resistance or contraindication to BAL.
Conflict of Interest No conflict of interest
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