特发性肺间质纤维化合并 Hermansky-Pudlak 综合征 4 型患者应用不同抗纤维化药物治疗的疗效观察
Interstitial Pneumonia Secondary to Hermansky-Pudlak Syndrome Type 4 Treated with Different Antifibrotic Agents.
机构信息
Department of Allergy and Respiratory Medicine, National Hospital Organization Minami-Okayama Medical Center, Japan.
Department of Hematology, Oncology and Respiratory Medicine, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Japan.
出版信息
Intern Med. 2021 Mar 1;60(5):783-788. doi: 10.2169/internalmedicine.5493-20. Epub 2021 Jan 20.
Hermansky-Pudlak syndrome (HPS) is an autosomal recessive hereditary disease that may be complicated by progressive and potentially fatal interstitial pneumonia. We herein report a 64-year-old woman with interstitial pneumonia associated with HPS type 4 whom we treated with nintedanib after pirfenidone proved ineffective. To our knowledge, there have been no previous reports of nintedanib being used to treat a patient with HPS type 4. There is a need for clinical trials of antifibrotic agents, including nintedanib, pirfenidone, and new therapeutic agents with different mechanisms of action in these patients.
Hermansky-Pudlak 综合征（HPS）是一种常染色体隐性遗传疾病，可能并发进行性和潜在致命性间质性肺炎。我们在此报告一例 4 型 HPS 相关间质性肺炎患者，在吡非尼酮治疗无效后，我们使用尼达尼布进行治疗。据我们所知，之前尚无报道使用尼达尼布治疗 4 型 HPS 患者。这些患者需要进行抗纤维化药物的临床试验，包括尼达尼布、吡非尼酮和具有不同作用机制的新型治疗药物。
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