Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia.
Christina RautenbergFriedrich StölzelChristoph RölligMatthias StelljesVerena GaidzikMichael LausekerOliver KriegeMareike VerbeekJulia Marie UnglaubFelicitas TholStefan W KrauseMathias HänelCharlotte NeuerburgVladan VucinicChristian-Friedrich JehnJulia SevermannMaxi WassLars FranseckyJens ChemnitzUdo HoltickKerstin Schäfer-EckartJosephine SchröderSabrina KrausWilliam KrügerUlrich KaiserSebastian SchollKathrin KochLea HenningGuido KobbeRainer HaasNael AlakelMaximilian-Alexander RöhnertKatja SockelMaher HanounUwe PlatzbeckerTobias A W HolderriedAnke MorgnerMichael HeuserTim SauerKatharina S GötzeEva Wagner-DrouetKonstanze DöhnerHartmut DöhnerChristoph SchliemannJohannes ScheteligMartin BornhäuserUlrich GermingThomas SchroederJan Moritz MiddekePublished in: Blood cancer journal (2021)
To investigate the efficacy and toxicities of CPX-351 outside a clinical trial, we analyzed 188 patients (median age 65 years, range 26-80) treated for therapy-related acute myeloid leukemia (t-AML, 29%) or AML with myelodysplasia-related changes (AML-MRC, 70%). Eighty-six percent received one, 14% two induction cycles, and 10% received consolidation (representing 22% of patients with CR/CRi) with CPX-351. Following induction, CR/CRi rate was 47% including 64% of patients with available information achieving measurable residual disease (MRD) negativity (<10-3) as measured by flow cytometry. After a median follow-up of 9.3 months, median overall survival (OS) was 21 months and 1-year OS rate 64%. In multivariate analysis, complex karyotype predicted lower response (p = 0.0001), while pretreatment with hypomethylating agents (p = 0.02) and adverse European LeukemiaNet 2017 genetic risk (p < 0.0001) were associated with lower OS. Allogeneic hematopoietic cell transplantation (allo-HCT) was performed in 116 patients (62%) resulting in promising outcome (median survival not reached, 1-year OS 73%), especially in MRD-negative patients (p = 0.048). With 69% of patients developing grade III/IV non-hematologic toxicity following induction and a day 30-mortality of 8% the safety profile was consistent with previous findings. These real-world data confirm CPX-351 as efficient treatment for these high-risk AML patients facilitating allo-HCT in many patients with promising outcome after transplantation.
Keyphrases
- acute myeloid leukemia
- end stage renal disease
- ejection fraction
- chronic kidney disease
- newly diagnosed
- clinical trial
- randomized controlled trial
- prognostic factors
- healthcare
- electronic health record
- machine learning
- allogeneic hematopoietic stem cell transplantation
- cell proliferation
- social media
- coronary artery disease
- patient reported outcomes
- emergency department
- cardiovascular events
- mesenchymal stem cells
- stem cell transplantation
- big data
- data analysis
- cell cycle arrest
- adverse drug