Inhaled GM-CSF Shows Promise for Rare Lung Disease- NEJM
Now open: Certificate Course in Management of Covid-19 by Govt. Of Gujarat and PlexusMDKnow more...Now open: Certificate Course in Management of Covid-19 by Govt. Of Gujarat and PlexusMDKnow more...
Daily treatment with molgramostim, an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF), in patients with autoimmune pulmonary alveolar proteinosis (PAP) was associated with improvements in pulmonary gas transfer and functional health status, a randomized trial found.

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disease characterized by progressive surfactant accumulation and hypoxemia. It is caused by disruption of granulocyte–macrophage colony-stimulating factor (GM-CSF) signaling, which pulmonary alveolar macrophages require to clear surfactant.

In a double-blind, placebo-controlled, three-group trial, researchers randomly assigned patients with aPAP to receive the recombinant GM-CSF molgramostim (300 g once daily by inhalation), either continuously or intermittently (every other week), or matching placebo. The primary end point was the change from baseline in the alveolar–arterial difference in oxygen concentration at week 24 (A-aDo2).

Results:
-- In total, 138 patients underwent randomization; 46 were assigned to receive continuous molgramostim, 45 to receive intermittent molgramostim, and 47 to receive placebo.
-- Invalid A-aDo2 data for 4 patients who received nasal oxygen therapy during arterial blood gas measurement were replaced by means of imputation.
-- For the primary end point — the change from baseline in the A-aDo2 at week 24 — improvement was greater among patients receiving continuous molgramostim than among those receiving placebo.
-- Patients receiving continuous molgramostim also had greater improvement than those receiving placebo for secondary end points, including the change from baseline in the St. George’s Respiratory Questionnaire total score at week 24.
-- For multiple end points, improvement was greater with continuous molgramostim than with intermittent molgramostim.
-- The percentages of patients with adverse events and serious adverse events were similar in the three groups, except for the percentage of patients with chest pain, which was higher in the continuous-molgramostim group.

Conclusively, In patients with aPAP, daily administration of inhaled molgramostim resulted in greater improvements in pulmonary gas transfer and functional health status than placebo, with similar rates of adverse events.

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1913590
Like
Comment
Share