Pulmonary Alveolar Proteinosis, Treatment
Conditions
Keywords
pulmonary alveolar proteinosis, granulocyte-macrophage colony-stimulating factor, whole lung lavage
Brief summary
The purpose of the study is to evaluate the effectiveness of the sequential therapy with whole lung Lavage (WLL)/inhaling granulocyte-macrophage colony stimulating factor, compared to WLL only, for adult patients with severe autoimmune pulmonary alveolar proteinosis in China over a two-year period.
Interventions
DRUGGM-CSF
After the patients were randomly divided into two groups, they will receive whole lung lavage (WLL), and then one of the two groups with continue the next step as follows: Induction period: The time of beginning is 1 week after whole lung lavage, aerosolized GM-CSF was given for 7 days (150ug bid), and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle, a total of 6 cycles (3 months) were known as the induction period. Maintenance period: maintenance period came up after the induction period. The dose of aerosolized GM-CSF was reduced to 150ug/d for three times a week, and then the durg was stopped for 7 days, the 2 weeks was designed as a cycle and maintenance period lasted for 9 months.
Sponsors
Dai Huaping
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Comfirmed diagnosis of autoimmune pulmonary alveolar proteinosis * Disease severity score (DSS) is 4-5
Exclusion criteria
* The acute respiratory infection * Heart failure (such as cardiogenic pulmonary edema) * The serious liver and kidney dysfunction (creatinine or ALT were equal to or more than 2 times of the upper limit of normal range); * Pregnancy; * The patients with hereditary and secondary factors (inhalation of dust, hematological diseases, autoimmune diseases, etc.); * DSS is 1-3; * The patient is allergic to the drugs that be used in our research; * The patients with poor compliance, or suffering from mental illness; * The patients have not signed informed consent; * They were treated with whole lung lavage or regular GM-CSF therapy (treatment for more than 2 weeks) within 3 months before the enrollment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to the first relapse (the date: day/month/year) | Up to 2 years | The standard of relapse: the condition of the disease after GM-CSF inhalation meeted the standard of effective treatment or the stability criteria, and then the condition deteriorated and meeted the deteriorate standard. The standard of effective treatment: oxygenation improvement (an increase in PaO2 of more than 10mmHg and / or a reduction in A-aDO2 more than 10mmHg). The stability criteria: an increase or a reduction in PaO2 of less than 10mmHg and / a reduction or an increase in A-aDO2 of less than 10mmHg. The deteriorate standard: deteriorate oxygenation (a reduction in PaO2 of more than 10mmHg and / or an increase in A-aDO2 of more than 10mmHg). |
Secondary
| Measure | Time frame |
|---|---|
| Relapse rate | Up to 2 years |
| FEV1 difference | Up to 2 years |
Countries
China
Contacts
Primary ContactHuaping Dai
Outcome results
None listed