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Long-term outcomes in five patients with autoimmune pulmonary alveolar proteinosis treated with molgramostim
Celia Montaño1, Elisabeth Bendstrup2, Ida Rønnov-Jessen2
1Interstitial Lung Diseases Unit, Bellvitge University Hospital, Bellvitge Biomedical Research Institute (IDIBELL), CIBERES, Barcelona, Spain.
Abstract:
Autoimmune pulmonary alveolar proteinosis (aPAP), which accounts for >90% of all cases of PAP, is a rare lung disease mediated by granulocyte-macrophage colony-stimulating factor (GM-CSF) autoantibodies that block GM-CSF signalling, leading to reduced surfactant clearance causing abnormal accumulation of alveolar surfactant and impaired gas exchange [1-3]. The current standard of care for aPAP is whole-lung lavage (WLL), which is invasive, resource intensive, carries procedural risk, does not address the underlying cause of disease and often must be repeated regularly [4]. Hence, there is a therapeutical need to address the underlying pathophysiology of the disease. Studies have explored inhaled GM-CSF augmentation as a primary treatment for aPAP [5-12]. In this real-world case series, we present the beneficial long-term effects of molgramostim inhalation solution, an investigational, recombinant GM-CSF, in five aPAP patients with therapeutic disease challenges.
Insights
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare lung disease. Inhaled molgramostim, a granulocyte-macrophage colony-stimulating factor (GM-CSF), shows promising long-term benefits for aPAP patients.
Area of Science:
- Pulmonology
- Immunology
- Rare Diseases
Background:
- Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare lung disease caused by GM-CSF autoantibodies blocking GM-CSF signaling.
- This blockage impairs surfactant clearance, leading to alveolar surfactant accumulation and reduced gas exchange.
- Current treatment, whole-lung lavage (WLL), is invasive and does not address the root cause.
Purpose of the Study:
- To investigate the long-term efficacy of inhaled molgramostim in patients with autoimmune pulmonary alveolar proteinosis (aPAP).
- To explore an alternative therapeutic approach for aPAP, addressing the underlying pathophysiology.
Main Methods:
- A real-world case series involving five aPAP patients.
- Treatment administered was inhaled molgramostim solution, an investigational recombinant GM-CSF.
- Focus on patients with challenging therapeutic histories.
Main Results:
- The study observed beneficial long-term effects of inhaled molgramostim in the treated aPAP patients.
- Molgramostim inhalation demonstrated positive outcomes in managing this rare lung disease.
Conclusions:
- Inhaled molgramostim may offer a viable therapeutic option for autoimmune pulmonary alveolar proteinosis (aPAP).
- This approach targets the underlying disease mechanism, providing a potential alternative to invasive treatments like WLL.
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