静脈内増強治療と重度のα1抗トリプシン欠乏症における肺密度 (RAPID):ランダム化,ダブルブラインド,プラセボ対照試験
Kenneth R Chapman1, Jonathan G W Burdon2, Eeva Piitulainen3
1Asthma and Airway Centre, University Health Network, Toronto Western Hospital, and Division of Respiratory Medicine, Department of Medicine, University of Toronto, Toronto, ON, Canada.
Lancet (London, England)
|June 1, 2015
まとめ
アルファ1プロテインアース阻害剤 (A1PI) アグメンテーション治療は,アルファ1アンチトリプシン欠乏症の患者でエムフィゼマの進行を遅らせます. 全肺容量におけるCT肺密度に基づくこの発見は,肺組織を保存するためのA1PI療法を支持する.
科学分野:
- 肺内科 肺内科 肺内科
- 放射線学 放射線学
- 薬理学 薬理学とは
背景:
- アルファ1抗トリプシン欠乏症 (AATD) は,肺エムフィゼマにつながる遺伝疾患です.
- A1PI増強の有効性を立証するランダム化,プラセボ対照試験は不足しています.
- CTで測定された肺密度は,AATD肺縮症の進行に対する敏感なマーカーです.
研究 の 目的:
- AATDにおけるアルファ1プロテインアース阻害剤 (A1PI) アグメンテーション療法の有効性を評価する.
- 主なアウトカム指標としてCTで評価された肺密度を活用する.
- 厳格な,ランダム化,プラセボ対照試験 (RAPID研究) を実施する.
主な方法:
- 多センター,ダブルブラインド,ランダム化,プラセボ対照試験で,重度のAATD患者180人が参加しました.
- 患者は24ヶ月間,静脈内でのA1PI (60mg/kg/週) またはプラセボを投与された.
- 主要エンドポイント:全肺容量 (TLC) と機能残留容量 (FRC) のCT肺密度の変化を組み合わせて,別々に測定する.
主要な成果:
- A1PI治療は,TLCとFRC測定を組み合わせると,肺密度損失を有意に変化させなかった (p=0.06).
- しかし,A1PIは,TLC単独での肺密度低下率を著しく低下させました (差異は年間0.74g/L,p=0.03).
- 副作用はA1PIとプラセボ群の間で類似しており,A1PIはよく耐えた.
結論:
- TLC単独でのCT肺密度測定は,A1PIアグメンテーション療法がAATDにおける肺縮症の進行を遅らせることを示しています.
- この発見は,AATDに関連したエンフィゼマにおける肺パレンキマの保存のためのA1PI増強の検討を支持する.
- FRCでの肺密度のみまたは組み合わせられた測定では,有意な差異は示されませんでした.
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