Pulmonary Alveolar Proteinosis
Conditions
Brief summary
This study aims to explore a new therapeutic approach: the feasibility, safety and preliminary efficacy of directly spraying GM-CSF into the airway through bronchoscopy for the treatment of aPAP.
Interventions
Bronchoscopy under general anesthesia or sedation; The predetermined dose of recombinant GM-CSF was dissolved in normal saline. The diseased lung segments were located through bronchoscopy and the liquid medicine was sprayed.
Sponsors
Jiuwu Bai
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Age: 18 to 60 years old; 2. A clear diagnosis of aPAP must meet at least one of the following diagnostic criteria: 1\) BALF appears milky white; Or cytological examination reveals a large amount of PAS-positive protein deposition; 2) HRCT shows typical paving stone-like changes; 3) Positive for serum GM-CSF antibody. 3. There are more than one of the following treatment indications: Symptoms such as progressive breathing difficulties, coughing, and shortness of breath after activity occur; 2) Without oxygen inhalation, PaO2 \> 65 mmHg 3) Pulmonary function DLCO accounts for % of the predicted value, ranging from 60% to 80%, including the critical value. 4\. No other PAP specific treatments (such as WLL, inhaled GM-CSF, biological agents, etc.) have been received recently (for more than 4 weeks). 5\. Agree to participate in this study and sign the informed consent form.
Exclusion criteria
1. Secondary PAP (such as secondary to blood diseases, etc.); 2. Patients with obvious pulmonary fibrosis, emphysema or irreversible lung function impairment; 3. Patients in the acute exacerbation stage; 4. Patients with other lung diseases (such as active pulmonary tuberculosis, bronchiectasis with purulent infection or other chronic infections; Have severe asthma, chronic bronchospasm, etc. 5. Have a history of allergy to GM-CSF antibodies or related drug components; 6. Patients who have participated in other clinical drug trials within the past three months; 7. Patients who have experienced severe complications related to bronchoscopy or are intolerant to bronchoscopy; 8. Concurrent with other serious cardiovascular and cerebrovascular diseases, hematological disorders, malignant tumors, etc. 9. Pregnant or lactating women.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Chest HRCT | 12 weeks |
Contacts
Primary ContactJiu-Wu Bai Doctor
Outcome results
None listed