Autoimmune Pulmonary Alveolar Proteinosis
Conditions
Interventions
None listed
Sponsors
Kumamoto University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1)Patients with autoimmune pulmonary alveolar proteinosis enrolled in the PAGE II specified clinical research* (cohort A) 2)Patients with disease severity III or higher who newly start GM-CSF inhalation therapy after GM-CSF is launched (Cohort B) 3)Patients with disease severity II or higher who newly start GM-CSF inhalation therapy after GM-CSF is launched (Cohort C) *PAGE II Specified Clinical Study of Sargramostim for Autoimmune Pulmonary Alveolar Proteinosis in 2022-2024.
Exclusion criteria
Exclusion criteria: (1) Patients diagnosed with secondary pulmonary alveolar proteinosis or hereditary pulmonary alveolar proteinosis (ii) Patients with severe symptoms due to heart or blood vessel disease such as congestive heart failure or angina pectoris (iii) Patients diagnosed with cancer within the past 5 years (excluding those whose condition has stabilized and who are in the follow-up period) (iv) Patients with bronchial asthma, pulmonary infection (including pulmonary tuberculosis), or other pulmonary diseases (including pulmonary tuberculosis). However, patients with non-tuberculous mycobacterial infection, pulmonary aspergillosis, or pulmonary nocardiosis who are under treatment or untreated and whose symptoms are stable may participate at the discretion of their physician), pulmonary fibrosis, interstitial pneumonia, bronchiectasis or other respiratory diseases that are expected to make it difficult to evaluate this study (v) Patients treated with whole lung lavage, repeated area lavage therapy, or rituximab within 1 month of enrollment (Cohort B only) (vi) Patients who received GM-CSF inhalation therapy within 1 month of enrollment (Cohort B only) (vii) Other patients deemed inappropriate by the physician in charge.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Remission rate after GM-CSF inhalation therapy Definition of remission=AaDO2 more than 10 torr from baseline and no additional treatment (whole lung lavage or GM-CSF inhalation) | — |
Secondary
| Measure | Time frame |
|---|---|
| Search for predictors of treatment response (responder/non-responder) Trends in %DLco Changes in CT imaging score Changes in mMRC transition of oxygen requirement, and number of patients who were able to wean off LTOT Trends in QOL score Changes in anti-GM-CSF antibodies Changes in serum biomarkers | — |
Countries
Japan
Contacts
Public ContactChieko Yoshida
Faculty of Life Sciences, Kumamoto University Department of Respiratory Medicine
Outcome results
None listed