臨床試験における複合エンドポイントの再考:患者および試験参加者からの洞察
Joshua M Stolker1, John A Spertus2, David J Cohen2
1From Saint Louis University, St. Louis, MO (J.M.S., K.K.J.); University of Missouri-Kansas City, Kansas City (J.A.S., D.J.C., E.B., B.B.L., P.S.C.); and Saint Luke's Mid America Heart and Vascular Institute; Kansas City, MO (D.J.C., P.G.J., P.S.C.). jstolker@yahoo.com.
Circulation
|September 10, 2014
まとめ
患者と臨床試験参加者は,複合エンドポイントを異なる方法で評価します. 患者は心筋梗塞と脳卒中を優先し,試験参加者は死亡に焦点を当てているが,複合エンドポイントは真の好みを反映していないことを示している.
科学分野:
- 心血管医学は,心臓血管医学である.
- 臨床試験のデザイン
- 患者によって報告されたアウトカム
背景:
- 複合エンドポイントは,統計力を高めるために臨床試験で頻繁に使用されます.
- 複合エンドポイント内の個々のコンポーネントの相対的な重要性は,患者と研究者の間で大きく異なる可能性があります.
研究 の 目的:
- 心血管疾患の患者と臨床試験の参加者が,複合エンドポイントの個々の成分をどのように評価するかを調査し,比較する.
- 現在の複合エンドポイントの加重が,患者と臨床医の優先順位を正確に反映しているかどうかを判断する.
主な方法:
- 785人の心血管疾患の患者と164人の臨床試験の著者を対象に,5つの有害事象 (死亡,心筋梗塞,脳卒中,冠動脈再血管化,胸痛の入院) の知覚された重要性について調査を行った.
- 認知された重さを定量化するために,死亡に対して割り当てられた"支出重量"と計算されたエンドポイント比.
- 患者とトライアリストのグループ間の重み付けの違いを分析し,人口統計学的および臨床的要因を調整した.
主要な成果:
- 死亡 (重量5) と比較して,心筋梗塞 (比率1.12) と脳卒中 (比率1.08) に類似またはより大きな重量を与えられた患者.
- 臨床試験の参加者は,心筋梗塞 (比率0.63) と脳卒中 (比率0.53) よりも死亡 (重量8) を優先した.
- 両グループは,リバスカライゼーションと入院を,死亡よりも実質的に軽いものとみなしました.
結論:
- 複合エンドポイントの個々のコンポーネントに対する患者とトライアリストの好みには大きな相違がある.
- 現在の複合エンドポイントの方法論は,患者または試験参加者の値を適切に反映していない可能性があります.
- 将来の臨床試験の設計では,患者によって報告された結果の指標を組み込むことを検討し,複合エンドポイントと患者の優先順位をより良く調整する必要があります.
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