Abstract
Original language | English |
---|---|
Journal | Blood Cancer J. |
Volume | 10 |
Issue number | 10 |
DOIs | |
Publication status | Published - 2020 |
Keywords
- cytarabine plus daunorubicin
- adult
- aged
- allogeneic stem cell transplantation
- alopecia
- Article
- cancer incidence
- cancer mortality
- cancer patient
- cancer survival
- chromosome analysis
- clinical evaluation
- comorbidity
- compassionate use
- drug efficacy
- drug screening
- female
- follow up
- gene mutation
- human
- infection
- major clinical study
- male
- mortality rate
- mucosa inflammation
- overall survival
- Pneumocystis pneumonia
- pneumonia
- prediction
- pyrexia idiopathica
- rash
- risk assessment
- secondary acute myeloid leukemia
- sepsis
- systemic mycosis
- treatment duration
- treatment response
Fingerprint
Dive into the research topics of '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: Blood Cancer Journal'. Together they form a unique fingerprint.Cite this
- APA
- Standard
- Harvard
- Vancouver
- Author
- BIBTEX
- RIS
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 : Blood Cancer Journal. / Guolo, F.; Fianchi, L.; Minetto, P.; Clavio, M.; Gottardi, M.; Galimberti, S.; Rizzuto, G.; Rondoni, M.; Bertani, G.; Dargenio, M.; Bilio, A.; Scappini, B.; Zappasodi, P.; Scattolin, A.M.; Grimaldi, F.; Pietrantuono, G.; Musto, P.; Cerrano, M.; D’Ardia, S.; Audisio, E.; Cignetti, A.; Pasciolla, C.; Pavesi, F.; Candoni, A.; Gurreri, C.; Morselli, M.; Alati, C.; Fracchiolla, N.; Rossi, G.; Caizzi, M.; Carnevale-Schianca, F.; Tafuri, A.; Ferrara, F.; Pagano, L.; Lemoli, R.M.
In: Blood Cancer J., Vol. 10, No. 10, 2020.Research output: Contribution to journal › Article › peer-review
}
TY - JOUR
T1 - 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
T2 - Blood Cancer Journal
AU - Guolo, F.
AU - Fianchi, L.
AU - Minetto, P.
AU - Clavio, M.
AU - Gottardi, M.
AU - Galimberti, S.
AU - Rizzuto, G.
AU - Rondoni, M.
AU - Bertani, G.
AU - Dargenio, M.
AU - Bilio, A.
AU - Scappini, B.
AU - Zappasodi, P.
AU - Scattolin, A.M.
AU - Grimaldi, F.
AU - Pietrantuono, G.
AU - Musto, P.
AU - Cerrano, M.
AU - D’Ardia, S.
AU - Audisio, E.
AU - Cignetti, A.
AU - Pasciolla, C.
AU - Pavesi, F.
AU - Candoni, A.
AU - Gurreri, C.
AU - Morselli, M.
AU - Alati, C.
AU - Fracchiolla, N.
AU - Rossi, G.
AU - Caizzi, M.
AU - Carnevale-Schianca, F.
AU - Tafuri, A.
AU - Ferrara, F.
AU - Pagano, L.
AU - Lemoli, R.M.
N1 - Export Date: 4 February 2021 Correspondence Address: Guolo, F.; IRCCS Ospedale Policlinico San MartinoItaly; email: fabio.guolo21@gmail.com Chemicals/CAS: cytarabine plus daunorubicin, 1256639-86-7 Tradenames: cpx 351 References: Arber, D.A., The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia (2016) Blood, 127, pp. 2391-2405. , COI: 1:CAS:528:DC%2BC2sXjs1agu7g%3D; Lichtman, M.A., A historical perspective on the development of the cytarabine (7days) and daunorubicin (3days) treatment regimen for acute myelogenous leukemia: 2013 the 40th anniversary of 7+3 (2013) Blood Cells Mol. Dis., 50, pp. 119-130. , COI: 1:CAS:528:DC%2BC38Xhs1Kjtr7E; Dohner, H., Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel (2017) Blood, 129, pp. 424-447; Miesner, M., Multilineage dysplasia (MLD) in acute myeloid leukemia (AML) correlates with MDS-related cytogenetic abnormalities and a prior history of MDS or MDS/MPN but has no independent prognostic relevance: a comparison of 408 cases classified as “AML not otherwise specified”(AML-NOS) or “AML with myelodysplasia-related changes” (AML-MRC) (2010) Blood, 116, pp. 2742-2751. , COI: 1:CAS:528:DC%2BC3cXhsVSktb3O; Granfeldt Ostgard, L.S., Epidemiology and clinical significance of secondary and therapy-related acute myeloid leukemia: a national population-based cohort study (2015) J. Clin. Oncol., 33, pp. 3641-3649; Leith, C.P., Acute myeloid leukemia in the elderly: assessment of multidrug resistance (MDR1) and cytogenetics distinguishes biologic subgroups with remarkably distinct responses to standard chemotherapy. A Southwest Oncology Group study (1997) Blood, 89, pp. 3323-3329. , COI: 1:CAS:528:DyaK2sXivVymsro%3D; Grimwade, D., Refinement of cytogenetic classification in acute myeloid leukemia: determination of prognostic significance of rare recurring chromosomal abnormalities among 5876 younger adult patients treated in the United Kingdom Medical Research Council trials (2010) Blood, 116, pp. 354-365. , COI: 1:CAS:528:DC%2BC3cXhtVejt7vO; Fianchi, L., Characteristics and outcome of therapy-related myeloid neoplasms: Report from the Italian network on secondary leukemias (2015) Am. J. Hematol., 90, pp. E80-E85. , COI: 1:CAS:528:DC%2BC2MXntFSnsL8%3D; Rai, K.R., Treatment of acute myelocytic leukemia: a study by cancer and leukemia group B (1981) Blood, 58, pp. 1203-1212. , COI: 1:STN:280:DyaL38%2FmvFCntg%3D%3D; Yates, J.W., Cytosine arabinoside (NSC-63878) and daunorubicin (NSC-83142) therapy in acute nonlymphocytic leukemia (1973) Cancer Chemother. Rep., 57, pp. 485-488. , COI: 1:STN:280:DyaE2c%2FltlSltg%3D%3D, PID: 4586956; Fianchi, L., Therapy-related myeloid neoplasms: clinical perspectives (2018) Onco Targets Ther., 11, pp. 5909-5915. , COI: 1:CAS:528:DC%2BC1MXitVOis77L; Stone, R.M., Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation (2017) N. Engl. J. Med., 377, pp. 454-464. , COI: 1:CAS:528:DC%2BC2sXhtlyksrbK; Hills, R.K., Addition of gemtuzumab ozogamicin to induction chemotherapy in adult patients with acute myeloid leukaemia: a meta-analysis of individual patient data from randomized controlled trials (2014) Lancet Oncol., 15, pp. 986-996. , COI: 1:CAS:528:DC%2BC2cXhtFWnu7vO; Lim, W.S., Leukemia-selective uptake and cytotoxicity of CPX-351, a synergistic fixed-ratio cytarabine: daunorubicin formulation, in bone marrow xenografts (2010) Leuk. Res., 34, pp. 1214-1223. , COI: 1:CAS:528:DC%2BC3cXps1Wksr4%3D; Kim, H.P., Liposomal encapsulation of a synergistic molar ratio of cytarabine and daunorubicin enhances selective toxicity for acute myeloid leukemia progenitors as compared to analogous normal hematopoietic cells (2011) Exp. Hematol., 39, pp. 741-750. , COI: 1:CAS:528:DC%2BC3MXptlWmsLo%3D; Lancet, J.E., Phase 2 trial of CPX-351, a fixed 5:1 molar ratio of cytarabine/daunorubicin, vs cytarabine/daunorubicin in older adults with untreated AML (2014) Blood, 123, pp. 3239-3246. , COI: 1:CAS:528:DC%2BC2cXptlSjs7Y%3D; Lancet, J.E., CPX-351 (cytarabine and daunorubicin) liposome for injection versus conventional cytarabine plus daunorubicin in older patients with newly diagnosed secondary acute myeloid leukemia (2018) J. Clin. Oncol., 36, pp. 2684-2692. , COI: 1:CAS:528:DC%2BC1MXivVSrsrY%3D; Roboz, G.J., Final safety and efficacy results from the CPX-351 early access program for older patients with high-risk or secondary acute myeloid leukemia (2020) Leuk. Lymph., 61, pp. 1188-1194. , COI: 1:CAS:528:DC%2BB3cXktFCgsrc%3D; Cilloni, D., Real-time quantitative polymerase chain reaction detection of minimal residual disease by standardized WT1 assay to enhance risk stratification in acute myeloid leukemia: a European LeukemiaNet study (2009) J. Clin. Oncol., 27, pp. 5195-5201. , COI: 1:CAS:528:DC%2BD1MXhsFahsLvP; Delgado, J., Survival analysis in hematologic malignancies: recommendations for clinicians (2014) Haematologica, 99, pp. 1410-1420; Appelbaum, F.R., Age and acute myeloid leukemia (2006) Blood, 107, pp. 3481-3485. , COI: 1:CAS:528:DC%2BD28Xkt1GitL8%3D; Kayser, S., The impact of therapy-related acute myeloid leukemia (AML) on outcome in 2853 adult patients with newly diagnosed AML (2011) Blood, 117, pp. 2137-2145. , COI: 1:CAS:528:DC%2BC3MXjtVWksLc%3D; Bejar, R., Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation (2014) J. Clin. Oncol., 32, pp. 2691-2698; Feldman, E.J., First in-man study of CPX-351: a liposomal carrier containing cytarabine and daunorubicin in a fixed 5:1 molar ratio for the treatment of relapsed and refractory acute myeloid leukemia (2011) J. Clin. Oncol., 29, pp. 979-985. , COI: 1:CAS:528:DC%2BC3MXkslOhsLo%3D; Feldman, E.J., Pharmacokinetics of CPX-351; a nano-scale liposomal fixed molar ratio formulation of cytarabine: daunorubicin, in patients with advanced leukemia (2012) Leuk. Res., 36, pp. 1283-1289. , COI: 1:CAS:528:DC%2BC38XhtFahtL7J; Goldberg, A.D., TP53 mutations predict poorer responses to CPX-351 in acute myeloid leukemia (2018) Blood, 132, p. 1433; Chiche, E., CPX-351 induces deep response and suppress the impact of poor prognosis mutations (TP53, ASXL1, RUNX1 and EVI-1) defined by ELN 2017 in t-AML and MRC-AML: a report from a multicentric French cohort (2019) Blood, 134, p. 1355; Jabbour, E., Outcome of patients with myelodysplastic syndrome after failure of decitabine therapy (2010) Cancer, 116, pp. 3830-3834. , COI: 1:CAS:528:DC%2BC3cXhtFWgsLfE; Prébet, T., Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure (2011) J. Clin. Oncol., 29, pp. 3322-3327; Buckley, S.A., Minimal residual disease prior to allogeneic hematopoietic cell transplantation in acute myeloid leukemia: a meta-analysis (2017) Haematologica, 102, pp. 865-873. , COI: 1:CAS:528:DC%2BC1cXitVWlurnI; Guolo, F., Combining flow cytometry and WT1 assessment improves the prognostic value of pre-transplant minimal residual disease in acute myeloid leukemia (2017) Haematologica, 102, pp. e348-e351. , COI: 1:CAS:528:DC%2BC1cXitV2js7%2FE; Buccisano, F., Prognostic and therapeutic implications of minimal residual disease detection in acute myeloid leukemia (2012) Blood, 119, pp. 332-341. , COI: 1:CAS:528:DC%2BC38XhsVKmt74%3D; Di Nardo, C.D., Venetoclax combined with decitabine or azacitidine in treatment naive, elderly patients with acute myeloid leukemia (2019) Blood, 133, pp. 7-17; Talati, C., Genomic landscape impacts induction outcome with CPX-351 in patients with acute myeloid leukemia (2018) Blood, 132, p. 2741
PY - 2020
Y1 - 2020
N2 - 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. © 2020, The Author(s).
AB - 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. © 2020, The Author(s).
KW - cytarabine plus daunorubicin
KW - adult
KW - aged
KW - allogeneic stem cell transplantation
KW - alopecia
KW - Article
KW - cancer incidence
KW - cancer mortality
KW - cancer patient
KW - cancer survival
KW - chromosome analysis
KW - clinical evaluation
KW - comorbidity
KW - compassionate use
KW - drug efficacy
KW - drug screening
KW - female
KW - follow up
KW - gene mutation
KW - human
KW - infection
KW - major clinical study
KW - male
KW - mortality rate
KW - mucosa inflammation
KW - overall survival
KW - Pneumocystis pneumonia
KW - pneumonia
KW - prediction
KW - pyrexia idiopathica
KW - rash
KW - risk assessment
KW - secondary acute myeloid leukemia
KW - sepsis
KW - systemic mycosis
KW - treatment duration
KW - treatment response
U2 - 10.1038/s41408-020-00361-8
DO - 10.1038/s41408-020-00361-8
M3 - Article
VL - 10
JO - Blood Cancer J.
JF - Blood Cancer J.
SN - 2044-5385
IS - 10
ER -