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.
Background Cardiac tumours are of rare incidence and usually occur in the form of secondary tumours. Most metastatic tumours are melanomas, sarcomas, lung, and haematological malignancies. Neuroendocrine carcinomas (NECs) of the heart are extremely unusual. This case report demonstrates a solitary high-grade NEC of the heart with an individual therapy strategy and follow-up. Case summary A 50-year-old gentleman presented with a 2 days history of recurrent episodes of chest pain. Echocardiography, computed tomography, and magnetic resonance imaging revealed tumorous lesions of the ventricles and aortic valve with large circular pericardial effusion. Histopathology results of the biopsy revealed a poorly differentiated small cell tumour of the neuroendocrine type. Despite further investigations with multiple imaging modalities and laboratory, no primary was found. Chemotherapy was initiated but size progression of the tumour was detected. As no other tumorous lesions were detected and resection was not possible because of the tumour complexity, decision on heart transplantation was made. However, due to the necessary immunosuppression after the heart transplantation, multiple metastasis where discovered in the course of treatment. Discussion The presence of a NEC in the heart without evidence of any other metastasis or evidence of primary tumour in other organs is clinically unique. For this individual case, heart transplantation was the therapy of choice due to tumour progression under chemotherapy and lacking possibility of resection, as no other suspect lesion was found other than the ones found in the heart. However, the risk of exacerbation of undiscovered micrometastases under necessary immunosuppression following the heart transplantation should be considered.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
customersupport@researchsolutions.com
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.