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

  1. Luana Fianchi
  2. Alfonso Piciocchi
  3. Fabio Guolo
  4. Francesco Marchesi
  5. Giovanni Marsili
  6. Chiara Cattaneo
  7. Michele Gottardi
  8. Francesco Restuccia
  9. Anna Candoni
  10. Elettra Ortu La Barbera
  11. Rita Fazzi
  12. Crescenza Pasciolla
  13. Olimpia Finizio
  14. Nicola Fracchiolla
  15. Mario Delia
  16. Federica Lessi
  17. Michela Dargenio
  18. Valentina Bonuomo
  19. Maria Ilaria Del Principe
  20. Patrizia Zappasodi
  21. Marco Picardi
  22. Claudia Basilico
  23. Monica Piedimonte
  24. Paola Minetto
  25. Patrizia Chiusolo
  26. Lucia Prezioso
  27. Caterina Buquicchio
  28. Lorella Melillo
  29. Daniele Zama
  30. Francesca Farina
  31. Valentina Mancini
  32. Michela Rondoni
  33. Alessandro Busca
  34. Livio Pagano
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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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  1. Version published to 10.21203/rs.3.rs-5915486/v1 on Research Square