{"title":"粒细胞-巨噬细胞集落刺激因子吸入治疗肺移植后复发性自身免疫性肺泡蛋白沉积症成功一例","authors":"Hiroshi Ishimoto , Noriho Sakamoto , Hirokazu Yura , Takahiro Takazono , Takashi Kido , Keitaro Matsumoto , Konosuke Morimoto , Tomoya Nishino , Koh Nakata , Hiroshi Mukae","doi":"10.1016/j.resinv.2024.12.001","DOIUrl":null,"url":null,"abstract":"<div><div>Sargramostim, a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) inhalation therapy, was recently approved for pharmaceutical use in Japan and shows promise as a treatment for autoimmune pulmonary alveolar proteinosis (APAP). For APAP patients with severe respiratory failure due to advanced lung fibrosis, lung transplantation is also a treatment option; however, APAP may recur after the procedure. Here, we report a case of successful sargramostim inhalation therapy for post-transplant APAP relapse in a patient who underwent living lung transplantation owing to severe fibrosis. Inhaled GM-CSF may be a useful treatment option for APAP recurrence in patients who have undergone lung transplantation.</div></div><div><h3>Clinical trial registration</h3><div>Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial II registered to Japan Registry of Clinical Trials (jRCTs031220127).</div></div>","PeriodicalId":20934,"journal":{"name":"Respiratory investigation","volume":"63 1","pages":"Pages 180-182"},"PeriodicalIF":2.4000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Successful granulocyte-macrophage colony-stimulating factor inhalation therapy for recurrent autoimmune pulmonary alveolar proteinosis after lung transplantation: A case report\",\"authors\":\"Hiroshi Ishimoto , Noriho Sakamoto , Hirokazu Yura , Takahiro Takazono , Takashi Kido , Keitaro Matsumoto , Konosuke Morimoto , Tomoya Nishino , Koh Nakata , Hiroshi Mukae\",\"doi\":\"10.1016/j.resinv.2024.12.001\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Sargramostim, a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) inhalation therapy, was recently approved for pharmaceutical use in Japan and shows promise as a treatment for autoimmune pulmonary alveolar proteinosis (APAP). For APAP patients with severe respiratory failure due to advanced lung fibrosis, lung transplantation is also a treatment option; however, APAP may recur after the procedure. Here, we report a case of successful sargramostim inhalation therapy for post-transplant APAP relapse in a patient who underwent living lung transplantation owing to severe fibrosis. Inhaled GM-CSF may be a useful treatment option for APAP recurrence in patients who have undergone lung transplantation.</div></div><div><h3>Clinical trial registration</h3><div>Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial II registered to Japan Registry of Clinical Trials (jRCTs031220127).</div></div>\",\"PeriodicalId\":20934,\"journal\":{\"name\":\"Respiratory investigation\",\"volume\":\"63 1\",\"pages\":\"Pages 180-182\"},\"PeriodicalIF\":2.4000,\"publicationDate\":\"2025-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Respiratory investigation\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2212534524001886\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"RESPIRATORY SYSTEM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Respiratory investigation","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2212534524001886","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
引用次数: 0
摘要
沙格列莫司汀是一种重组人粒细胞-巨噬细胞集落刺激因子(GM-CSF)吸入疗法,最近在日本获准用于制药,有望治疗自身免疫性肺泡蛋白沉着症(APAP)。对于因晚期肺纤维化导致严重呼吸衰竭的 APAP 患者,肺移植也是一种治疗选择;然而,APAP 可能会在术后复发。在此,我们报告了一例因严重肺纤维化而接受活体肺移植的患者移植后 APAP 复发的成功沙格列莫司汀吸入疗法。吸入GM-CSF可能是治疗肺移植患者APAP复发的有效方法。临床试验注册:肺泡蛋白病 GM-CSF 吸入疗效试验 II 已在日本临床试验注册中心注册(jRCTs031220127)。
Successful granulocyte-macrophage colony-stimulating factor inhalation therapy for recurrent autoimmune pulmonary alveolar proteinosis after lung transplantation: A case report
Sargramostim, a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) inhalation therapy, was recently approved for pharmaceutical use in Japan and shows promise as a treatment for autoimmune pulmonary alveolar proteinosis (APAP). For APAP patients with severe respiratory failure due to advanced lung fibrosis, lung transplantation is also a treatment option; however, APAP may recur after the procedure. Here, we report a case of successful sargramostim inhalation therapy for post-transplant APAP relapse in a patient who underwent living lung transplantation owing to severe fibrosis. Inhaled GM-CSF may be a useful treatment option for APAP recurrence in patients who have undergone lung transplantation.
Clinical trial registration
Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial II registered to Japan Registry of Clinical Trials (jRCTs031220127).