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Clinical Trial
. 2020 Oct 6;10(10):96.
doi: 10.1038/s41408-020-00361-8.

CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use program

Fabio Guolo  1   2 , Luana Fianchi  3 , Paola Minetto  4   5 , Marino Clavio  4   5 , Michele Gottardi  6 , Sara Galimberti  7 , Giuliana Rizzuto  8 , Michela Rondoni  9 , Giambattista Bertani  10 , Michela Dargenio  11 , Atto Bilio  12 , Barbara Scappini  13 , Patrizia Zappasodi  14 , Anna Maria Scattolin  15 , Francesco Grimaldi  16 , Giuseppe Pietrantuono  17 , Pellegrino Musto  17   18 , Marco Cerrano  19 , Stefano D'Ardia  20 , Ernesta Audisio  21 , Alessandro Cignetti  22 , Crescenza Pasciolla  23 , Francesca Pavesi  24 , Anna Candoni  25 , Carmela Gurreri  26 , Monica Morselli  27 , Caterina Alati  28 , Nicola Fracchiolla  29 , Giovanni Rossi  30 , Manuela Caizzi  31 , Fabrizio Carnevale-Schianca  32 , Agostino Tafuri  33 , Giuseppe Rossi  34 , Felicetto Ferrara  35 , Livio Pagano  3 , Roberto Massimo Lemoli  4   5
Affiliations
Clinical Trial

CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use program

Fabio Guolo et al. Blood Cancer J. .

Abstract

Secondary acute myeloid leukemia (sAML) poorly responds to conventional treatments and allogeneic stem cell transplantation (HSCT). We evaluated toxicity and efficacy of CPX-351 in 71 elderly patients (median age 66 years) with sAML enrolled in the Italian Named (Compassionate) Use Program. Sixty days treatment-related mortality was 7% (5/71). The response rate at the end of treatment was: CR/CRi in 50/71 patients (70.4%), PR in 6/71 (8.5%), and NR in 10/71 (19.7%). After a median follow-up of 11 months relapse was observed in 10/50 patients (20%) and 12 months cumulative incidence of relapse (CIR) was 23.6%. Median duration of response was not reached. In competing risk analysis, CIR was reduced when HSCT was performed in first CR (12 months CIR of 5% and 37.4%, respectively, for patients receiving (=20) or not (=30) HSCT, p = 0.012). Twelve-months OS was 68.6% (median not reached). In landmark analysis, HSCT in CR1 was the only significant predictor of longer survival (12 months OS of 100 and 70.5%, for patients undergoing or not HSCT in CR1, respectively, p = 0.011). In conclusion, we extend to a real-life setting, the notion that CPX is an effective regimen for high risk AML patients and may improve the results of HSCT.

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Conflict of interest statement

The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1. Treatment overwiev.
Patients enrollment and treatment in Compassionate Use Program.
Fig. 2
Fig. 2. Relapse Risk in responding patients according to transplantation.
Cumulative risk of relapse in patients achieving complete remission (CR), receiving or not hematopoietic stem cell transplantation (HSCT).
Fig. 3
Fig. 3. Overall Survival.
Overall Survival in the whole cohort from the time of enrollment in Compassionate Use Program.
Fig. 4
Fig. 4. Overall Survival in responding patients according to transplantation.
Landmark Overall Survival analysis in patients alive and in complete remission (CR) at day 90, receiving or not hematopoietic stem cell transplantation (HSCT).
See this image and copyright information in PMC

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