Trial reportThe European respiratory journal2024
Inhaled recombinant GM-CSF reduces the need for whole lung lavage and improves gas exchange in autoimmune pulmonary alveolar proteinosis patients.
Trial report in The European respiratory journal, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 3 of them syntheses that pooled it.
What it found
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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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 3 syntheses or guidelines pooled it, 39 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
- Efficacy of recombinant human GM-CSF compared to placebo for autoimmune pulmonary alveolar proteinosis: a systematic review and meta-analysis.BMC pulmonary medicine · 2026Pooled it
- Developmental Progress and Future Potential for Inhaled Biologics in the Treatment of Respiratory Diseases.Drugs · 2026Review
- 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
- Pulmonary alveolar proteinosis andFrontiers in pediatrics · 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
- Targeting GM-CSF in Rheumatoid Arthritis: Advances in Cytokine-Directed Immunotherapy and Clinical Implications.Life (Basel, Switzerland) · 2025Review
- mGem: When immunity turns against itself-GM-CSF autoantibodies drive opportunistic infection risk.mBio · 2025Review
- Macrophages: sentinels, warriors, and healers.Human molecular genetics · 2025Review
- Recombinant GM-CSF drug evaluation review.Immunotherapy · 2025Review
- Review
- A New Era in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis.Respirology (Carlton, Vic.) · 2025Article
- Restitutio ad integrum: Rescuing the Alveolar Macrophage Function with HSCT in Pulmonary Alveolar Proteinosis Due to CSF2Rα Deficiency.Journal of clinical immunology · 2024Article
- Inhaled treatment for dyspnea in a rare childhood disease.Pediatric pulmonology · 2024Article
- Pulmonary hypertension during high-dose GM-CSF therapy of autoimmune pulmonary alveolar proteinosis.Pulmonary circulation · 2024Article
- Pathogenesis-driven treatment of primary pulmonary alveolar proteinosis.European respiratory review : an official journal of the European Respiratory Society · 2024Review
- A Comprehensive Outlook on Pulmonary Alveolar Proteinosis-A Review.International journal of molecular sciences · 2024Review
Corrections and comments
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Authors and funding
14 authors at 5 institutions in 3 countries.
Funding
Abstract
rationaleWhole lung lavage (WLL) is a widely accepted palliative treatment for autoimmune pulmonary alveolar proteinosis (aPAP) but does not correct myeloid cell dysfunction or reverse the pathological accumulation of surfactant. In contrast, inhaled recombinant granulocyte-macrophage colony-stimulating factor (rGM-CSF) is a promising pharmacological approach that restores alveolar macrophage functions including surfactant clearance. Here, we evaluate WLL followed by inhaled rGM-CSF (sargramostim) as therapy of aPAP.
methods18 patients with moderate-to-severe aPAP were enrolled, received baseline WLL, were randomised into either the rGM-CSF group (receiving inhaled sargramostim) or control group (no scheduled therapy) and followed for 30 months after the baseline WLL. Outcome measures included additional unscheduled "rescue" WLL for disease progression, assessment of arterial blood gases, pulmonary function, computed tomography, health status, biomarkers and adverse events. Patients requiring rescue WLL were considered to have failed their assigned intervention group.
resultsThe primary end-point of time to first rescue WLL was longer in rGM-CSF-treated patients than controls (30
conclusionsThis long-term, prospective, randomised trial demonstrated inhaled sargramostim following WLL reduced the requirement for WLL, improved lung function and was safe in aPAP patients. WLL plus inhaled sargramostim may be useful as combined therapy for aPAP.
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What OpenQuestion holds
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.