Inhaled molgramostim shows long-term benefits for aPAP patients in real-world case series
Inhaled molgramostim, a recombinant GM-CSF, shows promising long-term therapeutic benefits for patients with autoimmune pulmonary alveolar proteinosis (aPAP), according to a real-world case series.
Currently, the standard treatment for aPAP is whole-lung lavage, however, whole-lung lavage does not address the underlying cause of the disease. As a result, there is a need for therapeutic options that target the disease’s pathophysiology.
This real-world case series highlights the long-term benefits of molgramostim inhalation solution in 5 patients with aPAP.
- All patients had characteristic CT scan patterns, bronchoalveolar lavage results, and symptoms of PAP, along with a positive GM-CSF autoantibody test confirming the diagnosis of aPAP.
- The patients received inhaled molgramostim through a vibrating mesh nebulizer as part of a compassionate-use program coordinated by Savara Inc., in compliance with local regulations.
- High-resolution CT scans and clinical measures before and after treatment showed positive results.
- No serious adverse events were reported, and the treatment was well tolerated by all 5 patients.
None of the patients required whole-lung lavage after more than a year on the therapy, and 1 patient was able to avoid whole-lung lavage entirely after receiving molgramostim as a first-line treatment. Improvements in disease severity, gas exchange, and daily living activities were sustained over time.
However, the study’s retrospective nature limits the ability to draw definitive conclusions.
Reference
Montaño C, Bendstrup E, Rønnov-Jessen I, et al. Long-term outcomes in five patients with autoimmune pulmonary alveolar proteinosis treated with molgramostim inhalation solution. ERJ Open Res. 2025;11(1):00567-2024. doi: 10.1183/23120541.00567-2024. PMID: 39872386; PMCID: PMC11770771.