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Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study

by Marsili, Giovanni , Del Principe, Maria Ilaria , Prezioso, Lucia , Restuccia, Francesco , Piciocchi, Alfonso , Basilico, Claudia , Candoni, Anna , Pagano, Livio , Fazzi, Rita , Minetto, Paola , Mancini, Valentina , Farina, Francesca , Chiusolo, Patrizia , Fianchi, Luana , Ortu La Barbera, Elettra , Melillo, Lorella , Picardi, Marco , Gottardi, Michele , Dargenio, Michela , Bonuomo, Valentina , Zappasodi, Patrizia , Lessi, Federica , Fracchiolla, Nicola , Marchesi, Francesco , Finizio, Olimpia , Buquicchio, Caterina , Guolo, Fabio , Delia, Mario , Pasciolla, Crescenza , Piedimonte, Monica , Busca, Alessandro , Rondoni, Michela , Cattaneo, Chiara , Zama, Daniele

in Adult / Age / Aged / Antineoplastic Combined Chemotherapy Protocols - adverse effects / Antineoplastic Combined Chemotherapy Protocols - therapeutic use / Cytarabine - administration & dosage / Cytarabine - adverse effects / Cytarabine - therapeutic use / Daunorubicin - administration & dosage / Daunorubicin - adverse effects / Daunorubicin - therapeutic use / Female / Hematology / Humans / Leukemia / Leukemia, Myeloid, Acute - drug therapy / Leukemia, Myeloid, Acute - mortality / Male / Medical prognosis / Medicine / Medicine & Public Health / Middle Aged / Mortality / Neoplasms, Second Primary - drug therapy / Neoplasms, Second Primary - mortality / Oncology / Patients / Pilot Projects / Registries / Remission (Medicine) / Survival Rate / Toxicity / Transplants & implants

2025

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Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study

2025

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2025

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Journal Article

Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study

Marsili, Giovanni,

Del Principe, Maria Ilaria,

Prezioso, Lucia,

Restuccia, Francesco,

Piciocchi, Alfonso,

Basilico, Claudia,

Candoni, Anna,

Pagano, Livio,

Fazzi, Rita,

Minetto, Paola,

Mancini, Valentina,

Farina, Francesca,

Chiusolo, Patrizia,

Fianchi, Luana,

Ortu La Barbera, Elettra,

Melillo, Lorella,

Picardi, Marco,

Gottardi, Michele,

Dargenio, Michela,

Bonuomo, Valentina,

Zappasodi, Patrizia,

Lessi, Federica,

Fracchiolla, Nicola,

Marchesi, Francesco,

Finizio, Olimpia,

Buquicchio, Caterina,

Guolo, Fabio,

Delia, Mario,

Pasciolla, Crescenza,

Piedimonte, Monica,

Busca, Alessandro,

Rondoni, Michela,

Cattaneo, Chiara,

Zama, Daniele

2025

Overview

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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Publisher

Springer Berlin Heidelberg,Springer Nature B.V

Subject

Adult

/ Age

/ Aged

/ Antineoplastic Combined Chemotherapy Protocols - adverse effects

/ Antineoplastic Combined Chemotherapy Protocols - therapeutic use

/ Cytarabine - administration & dosage

/ Cytarabine - adverse effects