@article{oai:niigata-u.repo.nii.ac.jp:00009510, author = {中田, 光}, issue = {11}, journal = {新潟医学会雑誌, 新潟医学会雑誌}, month = {Nov}, note = {リンパ脈管筋腫症(Lymphangioleiomyomatosis ; LAM)は若年女性が罹り, 予後不良の難病である. ラパマイシン標的蛋白質(mTOR)阻害薬のシロリムスが有効であることが, 医師主導の国際的な多施設共同臨床試験(Multicenter International LAM Efficacy of Sirolimus Trial ; MILES試験)で検証され, 2011年3月16日のThe New England Journal of Medicineに報告された. 生命科学医療センターと第二内科呼吸器グループは, 厚生労働科学研究費「臨床試験推進研究事業」費を得て, 近畿中央胸部疾患センターとともに同試験に参加し, 試験の成功に貢献した. 参加までの経緯と結果について述べたい., Lymphangioleiomyomatosis (LAM) is an uncommon lung disease that affects mostly female. LAM has an insidious onset, and is typically slowly progressive. Until recently, there was no proven therapies for LAM except for lung transplantation. To assess the effect of sirolimus, a mTOR inhibitor, on biological and clinical markers of lung function and to assess the safety, a trial was designed as a multi-center international, phase III, randomized, double-blind, placebo-controlled intention to treat (MILES trial). Respiratory disease group in The Second Department of Medicine and Bioscience Medical Research Center, Niigata University Hospital participated in the trial with Kinki Chuo Chest Medical Center and enrolled total 24 patients in Japan. On one year of oral sirolimus, patients with LAM and moderate respiratory impairment had significant but modest benefits on lung function and measures of quality of life or functional performance. The safety profile of the drug was acceptable and consistent with the known toxicities. Thus, Japanese research group contributed to the success of MILES trial.}, pages = {573--577}, title = {稀少肺難病との闘い : リンパ脈管筋腫症の国際共同臨床試験}, volume = {126}, year = {2012} }