Kisho

Kisho is an information platform, not a medical provider. Nothing on this site constitutes medical advice, diagnosis, or treatment recommendations. All content is aggregated from publicly available sources (including ClinicalTrials.gov, PubMed, FDA.gov, and Orphanet) and is provided for informational purposes only. Clinical trial eligibility, treatment decisions, and any health-related actions should always be discussed with a qualified healthcare professional. Kisho does not endorse any specific therapy, organization, or clinical trial. Terms of use · Privacy policy

autoimmune pulmonary alveolar proteinosis (APAP) - Clinical Profile, Genes & Research | Kisho | Kisho

Features include always present findings: Anti-granulocyte-macrophage colony stimulating factor antibody positivity, Low blood oxygen levels (hypoxemia), and Restrictive ventilatory defect; and sometimes findings: Lung abscess and Brain abscess. 16 total HPO annotations.

Organ System	Phenotype Count	Example Features
Lungs and breathing	6	Lung abscess, Dyspnea, Low blood oxygen levels (hypoxemia)
Brain and nerves	1	Brain abscess
Lab test results	1	Anti-granulocyte-macrophage colony stimulating factor antibody positivity
Heart and blood vessels	1	Chest pain
Blood and immune system	1	Recurrent respiratory infections

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for autoimmune pulmonary alveolar proteinosis. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for autoimmune pulmonary alveolar proteinosis, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for autoimmune pulmonary alveolar proteinosis. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
sargramostim	sargramostim	Partner Therapeutics, Inc.	2018	—	Designated

Gene therapy approaches for autoimmune pulmonary alveolar proteinosis have been reported in the published literature.

Clinical Trials

5 trials found

Explore Trials

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

5 clinical trials registered, 1 recruiting. Interventions under study include drug therapy. Pipeline includes 2 PHASE3, 3 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT04544293](https://clinicaltrials.gov/study/NCT04544293)	Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)	PHASE3	Savara Inc.	ACTIVE_NOT_RECRUITING
[NCT06989333](https://clinicaltrials.gov/study/NCT06989333)	Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis	PHASE2	Jiuwu Bai	NOT_YET_RECRUITING
[NCT06111846](https://clinicaltrials.gov/study/NCT06111846)	Study of Human Bone Marrow Mesenchymal Stem Cells in APAP	PHASE2	Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.	ACTIVE_NOT_RECRUITING
[NCT06431776](https://clinicaltrials.gov/study/NCT06431776)	Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).	PHASE3	Savara Inc.	RECRUITING
[NCT07890649](https://clinicaltrials.gov/study/NCT07890649)	A Study of hBMMSC Injection in Patients With aPAP	PHASE2	Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.	NOT_YET_RECRUITING

150 publications have been identified in PubMed for autoimmune pulmonary alveolar proteinosis. Research spans Case Report / Case Series (42%), Review / Meta-Analysis (20%), and Epidemiology / Natural History (12%).

Research Type	Count	% of Total
Patient case studies	63	42%
Research summaries	30	20%
Disease patterns and progression	18	12%
Testing and diagnosis research	12	8%
Clinical study results	10	7%
Laboratory research	8

Recent Publications

Lian L (2026). [PMID: 41671176](https://pubmed.ncbi.nlm.nih.gov/41671176/). *Proceedings of the National Academy of Sciences of the United States of America*. [Case Report / Case Series]
Song X (2026). [PMID: 42119776](https://pubmed.ncbi.nlm.nih.gov/42119776/). *Chest*. [Epidemiology / Natural History]
Fujishiro E (2026). [PMID: 42250564](https://pubmed.ncbi.nlm.nih.gov/42250564/). *Respir Investig*. [Epidemiology / Natural History]
Dang M (2026). [PMID: 42045620](https://pubmed.ncbi.nlm.nih.gov/42045620/). *Lung*. [Review / Meta-Analysis]
Naeem U (2026). [PMID: 41742124](https://pubmed.ncbi.nlm.nih.gov/41742124/). *BMC pulmonary medicine*. [Review / Meta-Analysis]
Fujita S (2026). [PMID: 41649888](https://pubmed.ncbi.nlm.nih.gov/41649888/). *Immunol Med*. [Case Report / Case Series]
Sharma S (2026). [PMID: 41835822](https://pubmed.ncbi.nlm.nih.gov/41835822/). *Indian J Thorac Cardiovasc Surg*. [Case Report / Case Series]
Carrington JM (2026). [PMID: 29493933](https://pubmed.ncbi.nlm.nih.gov/29493933/). *Unknown Journal*. [Review / Meta-Analysis]
Hou SX (2026). [PMID: 42236457](https://pubmed.ncbi.nlm.nih.gov/42236457/). *Zhonghua Jie He He Hu Xi Za Zhi*. [Case Report / Case Series]
Sugino K (2026). [PMID: 41815743](https://pubmed.ncbi.nlm.nih.gov/41815743/). *Respirol Case Rep*. [Epidemiology / Natural History]

Kisho

autoimmune pulmonary alveolar proteinosis

Overview

Clinical Presentation

Explore clinical features

Diagnosis

Treatment

Orphan Drug Designations

Clinical Trials

Prognosis

Research Landscape

Recent Publications

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

OMIM

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Latest News & Developments

Secondary pulmonary alveolar proteinosis mimicking progression of interstitial lung disease during treatment for adult-onset still's disease: An autopsy case report.

Molgramostim improves exercise distance and duration in autoimmune pulmonary alveolar proteinosis: exploratory IMPALA-2 trial data.

Pulmonary alveolar proteinosis associated with ruxolitinib.

Savara’s Early Access Program

autoimmune pulmonary alveolar proteinosis

Overview

Clinical Presentation

Explore clinical features

Diagnosis

Treatment

Orphan Drug Designations

Clinical Trials

Prognosis

Research Landscape

Recent Publications

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

OMIM

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Latest News & Developments

Secondary pulmonary alveolar proteinosis mimicking progression of interstitial lung disease during treatment for adult-onset still's disease: An autopsy case report.

Molgramostim improves exercise distance and duration in autoimmune pulmonary alveolar proteinosis: exploratory IMPALA-2 trial data.

Pulmonary alveolar proteinosis associated with ruxolitinib.

Savara’s Early Access Program