Inhaled GM-CSF for Pulmonary Alveolar Proteinosis
Author(s) -
Ryushi Tazawa,
Takahiro Ueda,
Mitsuhiro Abe,
Koichiro Tatsumi,
Ryosuke Eda,
Shotaro Kondoh,
Konosuke Morimoto,
Takeshi Tanaka,
Etsuro Yamaguchi,
Ayumu Takahashi,
Miku Oda,
Haruyuki Ishii,
Shinyu Izumi,
Haruhito Sugiyama,
Atsushi Nakagawa,
Keisuke Tomii,
Masaru Suzuki,
Satoshi Konno,
Shinya Ohkouchi,
Naoki Tode,
Tomohiro Handa,
Toyohiro Hirai,
Yoshikazu Inoue,
Toru Arai,
Katsuaki Asakawa,
Takuro Sakagami,
Atsushi Hashimoto,
Takahiro Tanaka,
Toshinori Takada,
Ayako Mikami,
Nobutaka Kitamura,
Koh Nakata
Publication year - 2019
Publication title -
new england journal of medicine
Language(s) - Uncategorized
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1816216
Subject(s) - pulmonary alveolar proteinosis , medicine , granulocyte macrophage colony stimulating factor , pulmonary surfactant , alveolar macrophage , granulocyte , autoantibody , immunology , pulmonary alveolus , pathology , recombinant dna , inhalation , respiratory disease , lung , macrophage , cytokine , antibody , biology , in vitro , anesthesia , biochemistry , gene
Pulmonary alveolar proteinosis is a disease characterized by abnormal accumulation of surfactant in the alveoli. Most cases are autoimmune and are associated with an autoantibody against granulocyte-macrophage colony-stimulating factor (GM-CSF) that prevents clearing of pulmonary surfactant by alveolar macrophages. An open-label, phase 2 study showed some therapeutic efficacy of inhaled recombinant human GM-CSF in patients with severe pulmonary alveolar proteinosis; however, the efficacy in patients with mild-to-moderate disease remains unclear.
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