在注册试验与一项真实世界研究之间对继发性急性髓系白血病中CPX-351进行匹配调整间接比较。
Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study.
作者信息
Fianchi Luana, Piciocchi Alfonso, Guolo Fabio, Marchesi Francesco, Marsili Giovanni, Cattaneo Chiara, Gottardi Michele, Restuccia Francesco, Candoni Anna, Ortu La Barbera Elettra, Fazzi Rita, Pasciolla Crescenza, Finizio Olimpia, Fracchiolla Nicola, Delia Mario, Lessi Federica, Dargenio Michela, Bonuomo Valentina, Del Principe Maria Ilaria, Zappasodi Patrizia, Picardi Marco, Basilico Claudia, Piedimonte Monica, Minetto Paola, Chiusolo Patrizia, Prezioso Lucia, Buquicchio Caterina, Melillo Lorella, Zama Daniele, Farina Francesca, Mancini Valentina, Rondoni Michela, Busca Alessandro, Pagano Livio
机构信息
Dipartimento Di Diagnostica Per Immagini, Fondazione Policlinico Universitario A. Gemelli, IRCCS- Università Cattolica del Sacro Cuore, Radioterapia Oncologica Ed Ematologia, Rome, Italy.
Unità Di Biostatistica Fondazione Gimema, Rome, Italy.
出版信息
Ann Hematol. 2025 May;104(5):2731-2736. doi: 10.1007/s00277-025-06381-3. Epub 2025 May 24.
A real-life study on CPX-351 and the standard arm ('7 + 3') of the CPX-351 registrative trial in adults with secondary Acute Myeloid Leukemia were compared by an unanchored Matching-adjusted indirect comparison (MAIC), in order to evaluate the efficacy and toxicity of CPX-351. Results of this study are important to confirm the role of CPX-351 in significantly improving survival and remission rates compared with '7 + 3' with a good safety profile in AML patients with high-risk features, a target group traditionally with a very poor prognosis. Moreover, this pilot analysis underlines the potentiality of the statistical method to compare studies with strong differences.
通过非锚定匹配调整间接比较（MAIC）对CPX-351与CPX-351在成人继发性急性髓系白血病注册试验的标准组（“7+3”）进行了一项真实研究，以评估CPX-351的疗效和毒性。本研究结果对于证实CPX-351在显著提高生存率和缓解率方面的作用非常重要，与“7+3”相比，CPX-351在具有高危特征的急性髓系白血病患者中具有良好的安全性，而这一目标群体传统上预后非常差。此外，这项初步分析强调了该统计方法在比较差异较大的研究方面的潜力。
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