Pulmonary alveolar proteinosis (PAP) is a rare lung disease of characterized by the filling of alveoli with proteins, phospholipids, and some other flocculated material. It can be caused by hereditary dysfunction of the granulocyte macrophage colony-stimulating factor receptor (GM-CSF), autoantibodies against GM-CSF, or other diseases leading to alveolar macrophage (AM) dysfunction. There is no consensus on which outcomes should be assessed in studies of patients with pulmonary alveolar proteinosis. Therefore, it is very necessary and urgent to establish a Core outcome set (COS) for pulmonary alveolar proteinosis to enhance the efficiency and overall quality of clinical research.
Relevant COS: Pulmonary Alveolar Proteinosis is a rare disease and there is currently no relevant COS.
To provide information for the development of a COS for PAP by reviewing the published literature to summarize the outcomes reported in pulmonary alveolar proteinosis and how they are measured.
ContributorsJin Xiong, M.B.B.S.
College of Pediatrics, Chongqing Medical University, Chongqing, China.
Ruobing Lei, M.Sc.
Chevidence Lab of Child & Adolescent Health, Children’s Hospital of Chongqing Medical University, Chongqing, China.
Zhengxiu Luo, M.D
Department of Respiratory Medicine, Children’s Hospital of Chongqing Medical University, Chongqing, China
Yaolong Chen, M.D
Chevidence Lab of Child & Adolescent Health, Children’s Hospital of Chongqing Medical University, Chongqing, China
Disease Category: Lungs & airways
Disease Name: Pulmonary Alveolar Proteinosis
Age Range: 0 - 100
Sex: Either
Nature of Intervention: Any
- None
- Systematic review of outcomes measured in trials
- Systematic review
A systematic review was conducted according to PRISMA-ScR. We searched the electronic databases MEDLINE, Embase, Cochrane, and Web of science for studies published from inception to April 2024 using the keywords "Pulmonary Alveolar Proteinosis" and "lung Alveolar Proteinosis". Registered, unpublished trials in Clinical trial.gov and ICTRP were also included.