Trial reportOrphanet journal of rare diseases2020
Inhaled granulocyte-macrophage colony stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis - a six month phase II randomized study with 24 months of follow-up.
Trial report in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02243228 (A Prospective Study of Inhaling Granulocyte-macrophage Colony Stimulating Factor in Adult Patients With Mild-to-moderate Autoimmune Pulmonary Alveolar Proteinosis in China), which is not on this map. Cited by 17 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Prospective Study of Inhaling Granulocyte-macrophage Colony Stimulating Factor in Adult Patients With Mild-to-moderate Autoimmune Pulmonary Alveolar Proteinosis in China: a Randomized Open-label Study
Who cites it
17 citing papers in PubMed, 3 syntheses or guidelines pooled it, 28 citations in OpenAlex.
- Pooled it
- The burden of autoimmune pulmonary alveolar proteinosis: a systematic review.European respiratory review : an official journal of the European Respiratory Society · 2026Pooled it
- Nebulised granulocyte-macrophage colony-stimulating factor (GM-CSF) in autoimmune pulmonary alveolar proteinosis: a systematic review and meta-analysis.European respiratory review : an official journal of the European Respiratory Society · 2023Pooled it
- Clinical characterization of 39 patients with autoimmune pulmonary alveolar proteinosis.The Journal of international medical research · 2026Article
- Lipid nanoparticle GM-CSF replacement for autoimmune pulmonary alveolar proteinosis.Proceedings of the National Academy of Sciences of the United States of America · 2026Article
- Outcomes and outcome measures in studies of pulmonary alveolar proteinosis: a scoping review.European respiratory review : an official journal of the European Respiratory Society · 2026Review
- Recombinant GM-CSF drug evaluation review.Immunotherapy · 2025Review
- Review
- Summary for clinicians: ERS guidelines on pulmonary alveolar proteinosis.Breathe (Sheffield, England) · 2025Review
- Phenotypic heterogeneity in mortality and prognosis of pulmonary alveolar proteinosis: a large-scale, global pooled analysis of individual-level data.Orphanet journal of rare diseases · 2025Article
- Long-term outcomes in five patients with autoimmune pulmonary alveolar proteinosis treated with molgramostim inhalation solution.ERJ open research · 2025Article
- Auto-Abs neutralizing type I IFNs in patients with severe Powassan, Usutu, or Ross River virus disease.The Journal of experimental medicine · 2024Article
- Effects of COVID-19 infection in patients with autoimmune pulmonary alveolar proteinosis: a single-center study.Orphanet journal of rare diseases · 2023Article
- Alveolar macrophages in pulmonary alveolar proteinosis: origin, function, and therapeutic strategies.Frontiers in immunology · 2023Review
- Autoimmune Pulmonary Alveolar Proteinosis.American journal of respiratory and critical care medicine · 2022Article
- Rituximab rescue therapy for autoimmune pulmonary alveolar proteinosis.Respiratory medicine case reports · 2022Article
- Update on Diagnosis and Treatment of Adult Pulmonary Alveolar Proteinosis.Therapeutics and clinical risk management · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTreatment of autoimmune pulmonary alveolar proteinosis (aPAP) by inhaled granulocyte-macrophage colony stimulating factor (GM-CSF) is considered safe and effective. Evidence of benefit from GM-CSG inhalation for mild to moderate aPAP patients is limited.
methodsIn this multicenter, randomized, open-labeled clinical trial, 36 aPAP patients with mild to moderate disease severity were randomized into either the GM-CSF treatment group or control group. Inhaled GM-CSF was prescribed for 6 months, and patients received follow-up for another 18 months without treatment. Physiological features of the patients were analyzed.
resultsThere were 36 patients (19 in the treatment group, 17 in the control group) included. There were no significant differences in the primary endpoints as measured by the change of alveolar arterial oxygen gradient (A-aDO
conclusionSix months of inhaled GM-CSF treatment had no effect on the alveolar-arterial oxygen gradient in patients with mild to moderate pulmonary alveolar proteinosis. There were changes in some clinical or laboratory measures, but no clinically important changes were noted at the end of study. (Clinical Trial Registry: NCT02243228, Registered on September 17, 2014, https://www.clinicaltrials.gov/ct2/show/NCT02243228?term=NCT02243228&draw=2&rank=1 ).
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.