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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| D1 | Experimental | Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). If the treatment is effective, participants may be entered into low-dose group(D1)(rhGM-CSF administration 1.25 ug/kg/d, qd, sc, for 2 months，then rhGM-CSF administration 1.25 ug/kg/d, qod, sc, for 3 months), when the chest CT absorption≥25% , and /or the PaO2 elevated by 5 mm Hg. |
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| D2 | Experimental | Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). When the treatment is ineffective, and anti-GM-CSF antibody titers level ≥1:1000，the dose will be increased to 2.5 ug/kg/d. After 2 months, if the clinical response was optimal, that dose is continued for 3 months, and defined as group 2(D2). |
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| D3 | Experimental | Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). When the treatment is ineffective, and anti-GM-CSF antibody titers level ≥1:1000，the dose will be increased to 2.5 ug/kg/d. After 2 months, if the clinical response was not optimal, the patients will receive whole lung lavage(WLL), who are defined as group 3(D3). |
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| D4 | Active Comparator | Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). When the treatment is ineffective, and anti-GM-CSF antibody titers level <1:1000，the patients will receive whole lung lavage(WLL), then give rhGM-CSF administration (1.25 ug/kg/d) for 3 months, which belong to group4(D4). |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rhGM-CSF | Drug | GM-CSF will be given subcutaneously according to the rule in different groups. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Improvements in double pulmonary diffuse lesions （Chest CT score ） | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Huiping Li, Dr | Contact | 86-13817389991 | liw2013@126.com |
| Name | Affiliation | Role |
|---|---|---|
| Huiping Li, Dr | Shanghai Pulmonary Hospital, Shanghai, China | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Shanghai Pulmonary Hospital | Recruiting | Shanghai | 200433 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31898493 | Derived | Zhang F, Weng D, Su Y, Yin C, Shen L, Zhang Y, Zhou Y, Li Q, Hu Y, Li H. Therapeutic effect of subcutaneous injection of low dose recombinant human granulocyte-macrophage colony-stimulating factor on pulmonary alveolar proteinosis. Respir Res. 2020 Jan 2;21(1):1. doi: 10.1186/s12931-019-1261-1. |
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| ID | Term |
|---|---|
| D011649 | Pulmonary Alveolar Proteinosis |
| ID | Term |
|---|---|
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C082856 | regramostim |
| D016178 | Granulocyte-Macrophage Colony-Stimulating Factor |
| D003115 | Colony-Stimulating Factors |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
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|
| Whole Lung Lavage(WLL) | Procedure | using double lumen endotracheal tube (DLT) to selectively lavage one lung |
|
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| Improvements in pulmonary function |
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). |
| 6 months |
| 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 |
| D016207 |
| Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |