<p>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.</p>

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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

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

摘要

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.