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) receptor, 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 develop a consensus-based Core Outcome Set (COS) for PAP for use in future clinical trials and studies.
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
- Patient/ support group representatives
- Researchers
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Interview
- Literature review
Phase 1 (Completed & Published): A scoping review following PRISMA-ScR guidelines was conducted to identify outcomes in PAP research. This phase has been completed and published.
Phase 2 (Ongoing): Semi-structured interviews with PAP patients to identify patient-important outcomes. Thematic analysis will be applied.
Phase 3 (Planned): A multi-stakeholder Delphi survey (minimum 2 rounds) using a 9-point Likert scale to rate outcome importance.
Phase 4 (Planned): A consensus meeting to finalize the Core Outcome Set through anonymous voting.