Academic Journal
Inhaled GM-CSF for Pulmonary Alveolar Proteinosis
| العنوان: | Inhaled GM-CSF for Pulmonary Alveolar Proteinosis |
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| المؤلفون: | TAZAWA, Ryushi, UEDA, Takahiro, ABE, Mitsuhiro, TATSUMI, Koichiro, EDA, Ryosuke, KONDOH, Shotaro, MORIMOTO, Konosuke, TANAKA, Takeshi, YAMAGUCHI, Etsuro, TAKAHASHI, Ayumu, ODA, Miku, ISHII, Haruyuki, IZUMI, Shinyu, SUGIYAMA, Haruhito, NAKAGAWA, Atsushi, TOMII, Keisuke, SUZUKI, Masaru, KONNO, Satoshi, OHKOUCHI, Shinya, TODE, Naoki, HANDA, Tomohiro, HIRAI, Toyohiro, INOUE, Yoshikazu, ARAI, Toru, ASAKAWA, Katsuaki, SAKAGAMI, Takuro, HASHIMOTO, Atsushi, TANAKA, Takahiro, TAKADA, Toshinori, MIKAMI, Ayako, KITAMURA, Nobutaka, NAKATA, Koh, 田澤, 立之, 安部, 光洋, 巽, 浩一郎, 江田, 良輔, 森本, 浩之輔, 田中, 健之, 山口, 悦郎, 高橋, 歩, 石井, 晴之, 泉, 信有, 杉山, 温人, 中川, 淳, 富井, 啓介, 鈴木, 雅, 今野, 哲, 大河内, 眞也, 東出, 直樹, 半田, 知宏, 平井, 豊博, 井上, 義一, 新井, 徹, 坂上, 拓郎, 田中, 崇裕, 高田, 俊範, 三上, 礼子, 北村, 信隆, 中田, 光 |
| بيانات النشر: | Massachusetts Medical Society |
| سنة النشر: | 2019 |
| المجموعة: | CURATOR - Chiba University Repository for Access to Outcomes from Research / 千葉大学学術成果リポジトリ |
| الوصف: | [ABSTRACT] BACKGROUND: 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. METHODS: We conducted a double-blind, placebo-controlled trial of daily inhaled recombinant human GM-CSF (sargramostim), at a dose of 125 μg twice daily for 7 days, every other week for 24 weeks, or placebo in 64 patients with autoimmune pulmonary alveolar proteinosis who had a partial pressure of arterial oxygen (Pao2) while breathing ambient air of less than 70 mm Hg (or <75 mm Hg in symptomatic patients). Patients with severe pulmonary alveolar proteinosis (Pao2 <50 mm Hg) were excluded to avoid possible exacerbation of the disease in patients who were assigned to receive placebo. The primary end point was the change in the alveolar-arterial oxygen gradient between baseline and week 25. RESULTS: The change in the mean (±SD) alveolar-arterial oxygen gradient was significantly better in the GM-CSF group (33 patients) than in the placebo group (30 patients) (mean change from baseline, -4.50±9.03 mm Hg vs. 0.17±10.50 mm Hg; P = 0.02). The change between baseline and week 25 in the density of the lung field on computed tomography was also better in the GM-CSF group (between-group difference, -36.08 Hounsfield units; 95% confidence interval, -61.58 to -6.99, calculated with the use of the Mann?Whitney U test and the Hodges?Lehmann estimate of confidence intervals for pseudo-medians). Serious adverse events developed in 6 patients in the GM-CSF group and in 3 patients in the placebo group. CONCLUSIONS: In this randomized, controlled trial, ... |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| تدمد: | 15334406 |
| Relation: | isVersionOf:https://doi.org/10.1056/NEJMoa1816216; pmid:info:pmid/31483963; jtitle:The New England Journal of Medicine; volume:381; spage:923; epage:932; https://opac.ll.chiba-u.jp/da/curator/106366/; https://opac.ll.chiba-u.jp/da/curator/106366/NEJMoa1816216.pdf |
| الاتاحة: | https://opac.ll.chiba-u.jp/da/curator/106366/ https://opac.ll.chiba-u.jp/da/curator/106366/NEJMoa1816216.pdf |
| Rights: | (c) 2019 Massachusetts Medical Society. |
| رقم الانضمام: | edsbas.A3EFEB28 |
| قاعدة البيانات: | BASE |
| تدمد: | 15334406 |
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