Preliminary report on a sargramostim inhalation study for moderate to severe autoimmune alveolar proteinosis
Koh Nakataa Tomohiro Handab Takuro Sakagamic Takuji Suzukid Yoshinori Taninoe Keiichi Akasakaf Satoru Itog Keisuke Tomiih Noriho Sakamotoi Ryosuke Edaj Hirokazu Kimurak Nobutaka Kitamuraa
aDivision of Pioneering Advanced Therapeutics, Niigata University Medical and Dental General Hospital
bDepartment of Respiratory Medicine, Kyoto University Graduate School of Medicine
cDepartment of Respiratory Medicine, Kumamoto University Hospital
dDepartment of Respirology, Graduate School of Medicine, Chiba University
eDepartment of Respiratory Medicine, Fukushima Medical University
fDepartment of Respiratory Medicine, Saitama Red Cross Hospital
gDepartment of Respiratory Medicine and Allergology, Aichi Medical University School of Medicine
hDepartment of Respiratory Medicine, Kobe City Medical Center General Hospital
iDepartment of Respiratory Medicine, Nagasaki University Hospital
jDepartment of Internal Medicine (Respiratory Medicine), Kurashiki Municipal Hospital
kDepartment of Respiratory Medicine, Hokkaido University Graduate School of Medicine
The purpose of this study was to assess the efficacy rate of inhaled sargramostim for moderate to severe autoimmune pulmonary alveolar proteinosis, and to explore the effect of increasing the dose in patients who did not improve. When 34 patients received 12 cycles of sargramostim 250 µg/day inhalation for 7 consecutive days every other week, 18 patients responded positively [Alveolar-arterial oxygen difference (A-aDO2) improved more than 10 mmHg from baseline]. Two patients withdrew. Among the remaining 14 patients, six patients responded positively after increasing the dose to 500 µg/day for 6 to 12 additional cycles. Additional inhalation at increased doses for patients who did not respond seems to be a treatment that should be attempted.
Autoimmune pulmonary alveolar proteinosis (APAP) Sargramostim Inhalation therapy Alveolar-arterial oxygen difference (A-aDO2) Anti-granulocyte-macrophage colony-stimulating factor (GM-CSF) autoantibody
Received 27 Mar 2024 / Accepted 29 Mar 2024
AJRS, 13(4): 140-147, 2024