論文

国際誌
2016年11月

Efficacy and Safety of Long-Term Sirolimus Therapy for Asian Patients with Lymphangioleiomyomatosis.

Annals of the American Thoracic Society
  • Toshinori Takada
  • Ayako Mikami
  • Nobutaka Kitamura
  • Kuniaki Seyama
  • Yoshikazu Inoue
  • Katsura Nagai
  • Masaru Suzuki
  • Hiroshi Moriyama
  • Keiichi Akasaka
  • Ryushi Tazawa
  • Toyohiro Hirai
  • Michiaki Mishima
  • Mie Hayashida
  • Masaki Hirose
  • Chikatoshi Sugimoto
  • Toru Arai
  • Noboru Hattori
  • Kentaro Watanabe
  • Tsutomu Tamada
  • Hirohisa Yoshizawa
  • Kohei Akazawa
  • Takahiro Tanaka
  • Keita Yagi
  • Lisa R Young
  • Francis X McCormack
  • Koh Nakata
  • 全て表示

13
11
開始ページ
1912
終了ページ
1922
記述言語
英語
掲載種別
研究論文(学術雑誌)

RATIONALE: Sirolimus has been shown in a randomized, controlled clinical trial to stabilize lung function in patients with lymphangioleiomyomatosis (LAM) treated for a 12-month time period; however the pretreatment decline in lung function after the drug was discontinued indicated that continued exposure is required to suppress disease progression. OBJECTIVES: To elucidate the durability and tolerability of long-term sirolimus treatment in Asian patients with LAM. METHODS: We conducted a single-arm, open-label, investigator-initiated safety and efficacy study of sirolimus in 63 women with LAM at 9 sites in Japan. Subjects received sirolimus for 2 years at doses adjusted to maintain a trough blood level of 5-15 ng/ml. MEASUREMENTS AND MAIN RESULTS: Fifty-two subjects (82.5%) completed the trial with mean drug compliance of more than 80% overall during the study. The number of adverse events was greatest during the initial 6 months of therapy, but they continued to occur with declining frequency throughout the 2-year study period. Of the 1,549 adverse events, 27 were classified as serious, including reversible sirolimus pneumonitis in 3 patients. New hypercholesterolemia occurred in 30 patients (48%); microcytosis in 10 patients; loss of body weight in 33 patients; and increase in blood pressure that required treatment in 5 patients. FEV1, FVC, and quality-of-life parameters were stable in the overall study cohort during the study period, but baseline to 2-year improvements in lung function occurred in the subset of patients with a prior history of chylothorax. CONCLUSIONS: Although long-term sirolimus treatment of Asian patients with LAM was associated with a large number of adverse events, including three episodes of pneumonitis, most patients completed the 2-year course of medication with good drug compliance and stable quality of life and lung function.

リンク情報
PubMed
https://www.ncbi.nlm.nih.gov/pubmed/27513278
ID情報
  • eISSN : 2325-6621
  • PubMed ID : 27513278

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