: 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.
Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study / 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. - In: ANNALS OF HEMATOLOGY. - ISSN 0939-5555. - (2025), pp. 1-6. [10.1007/s00277-025-06381-3]
Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study
Candoni, Anna;
2025
Abstract
: 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.
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