| Item | Content |
|---|
| Research project name |
Practical Research Project for Rare / Intractable Diseases |
| Study title |
Study on development of evidence directly connected with treatment of pulmonary alveolar proteinosis: diagnosis, treatment and management of severe intractable cases |
| Name of principal investigator |
Yoshikazu Inoue |
| Name of institution of the principal investigator |
Clinical Research Center, National Hospital Organization Kinki-Chuo Chest Medical Center |
| Target disease |
Pulmonary alveolar proteinosis |
| Registry name |
Establishment of basic database of pulmonary alveolar proteinosis |
| Purpose of registry |
Investigation into the number or the distribution of patients, Patient recruitment for clinical trials or other interventional studies
|
| Overview of registry |
The data of the PAP basic database will be collected for the patients who were diagnosed with pulmonary alveolar proteinosis based on measurement of serum anti-GM-CSF autoantibody once a year at Niigata University Life Science Center, Clinical Research Center, National Hospital Organization Kinki-Chuo Chest Medical Center, and Division of Respiratory Medicine & Allergology, Department of Internal Medicine, Aichi Medical University. The annual incidence will be clarified by the estimated number of all PAP patients in Japan. The number of the patients with extremely rare pulmonary disease PAP targeted for the rhGM-CSF inhalation therapy was estimated based on the PAP basic database. As a result, the number of patients was estimated in the sponsor-initiated study and the investigator-initiated rhGM-CSF inhalation trial, which enabled to conduct clinical trials in Japan.
|
| Research item |
Sex, Information about the current medical institutions of patients, Others (age, classification of pulmonary alveolar proteinosis, institution measuring anti-GM-CSF antibody, and institutional anonymized number)
|
| Contact |
Yoshikazu Inoue, Kinki-Chuo Chest Medical Center |
| E-mail address |
giichi●kch.hosp.go.jp |
| Registry URL |
|
* If the E-mail address is shown, replace "●" with "@", please.
