Pulmonary Alveolar Proteinosis
Conditions
Keywords
Pulmonary, Pulmonary Alveolar Proteinosis, PAP
Brief summary
The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP.
Detailed description
The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP. During the observation, study visits will occur at the end of each month. During the 1-year follow-up period which is lasting 6 months after the treatment, all participants will be required to check the various efficacy indicators.
Interventions
DRUGrhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
PROCEDUREWhole Lung Lavage(WLL)
using double lumen endotracheal tube (DLT) to selectively lavage one lung
Sponsors
Shanghai Pulmonary Hospital, Shanghai, China
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
17 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosed PAP patients * Aged 17-80 * Signed informed consent
Exclusion criteria
* Secondary PAP * Received whole lung lavage therapy within 4 weeks before enrollment * Received previous GM-CSF therapy within 6 months before enrollment * WBC≥12,000/ul * fever≥38℃ * Severe edema, severe liver, kidney, lung and cardiovascular disease. * Pregnant，planning to get pregnant or nursing * Inability to express the subjective discomfort * Serious drug allergy history, E.coli preparation or rhGM-CSF serious allergy history
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Improvements in double pulmonary diffuse lesions （Chest CT score ） | 6 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinical symptoms observation: shod of breath, cough (according to each score standard） | 6 months | — |
| Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change | 6 months | — |
| Improvements in pulmonary function | 6 months | Pulmonary function tests include residual volume/total lung capacity(RV/TLC), forced vital capacity(FVC), forced expiratory volume in one second/forced vital capacity(FEV1/FVC), diffusing capacity of carbon monoxide(DLCO). |
| Improvements in arterial blood gas, including alveolar-arterial oxygen difference(A-aDO2), partial pressure of oxygen(PaO2), partial pressure of carbon dioxide in artery(PaCO2), arterial oxygen saturation(SaO2). | 6 months | — |
Countries
China
Contacts
Primary ContactHuiping Li, Dr
Outcome results
None listed