Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.
- Conditions
- Pulmonary Alveolar Proteinosis
- Interventions
- Procedure: Whole Lung Lavage(WLL)
- Registration Number
- NCT01983657
- Lead Sponsor
- Shanghai Pulmonary Hospital, Shanghai, China
- 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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 40
- Diagnosed PAP patients
- Aged 17-80
- Signed informed consent
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description D1 rhGM-CSF 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. D2 rhGM-CSF 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). D3 rhGM-CSF 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). D4 rhGM-CSF 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). D4 Whole Lung Lavage(WLL) 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).
- Primary Outcome Measures
Name Time Method Improvements in double pulmonary diffuse lesions （Chest CT score ） 6 months
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (1)
Shanghai Pulmonary Hospital
🇨🇳Shanghai, China