Real-World Experience With CPX-351 Treatment for Acute Myeloid Leukemia in England: An Analysis From the National Cancer Registration and Analysis Service.

BACKGROUND: CPX-351 demonstrated improved overall survival (OS) versus conventional 3 + 7 daunorubicin/cytarabine chemotherapy in a registrational phase III study in older patients with newly diagnosed, high-risk secondary acute myeloid leukemia (AML). This retrospective, population-based cohort study aimed to describe and compare the characteristics and survival outcomes of younger (<60 years) versus older (≥60 years) patients with AML treated with CPX-351 in England. PATIENTS AND METHODS: The study included adults aged ≥18 years diagnosed with AML in England between January 2013 and March 2022, and treated with CPX-351 in routine clinical practice (patients who received CPX-351 in a clinical trial were excluded). Patient records were sourced from the population-level cancer analysis system database available through the National Cancer Registration and Analysis Service. RESULTS: Of 353 included patients, 104 (29.5%) were <60 years. With a median follow-up of 10.9 months from diagnosis, the estimated median OS was 12.9 months overall, 17.3 months for adults <60 years and 11.7 months for those ≥60 years. All-cause mortality by Day 30 from diagnosis was 6% overall, 4% for adults <60 years and 6% for those ≥60 years. Hematopoietic cell transplantation (HCT) was received by 54% of adults <60 years and 38% of those ≥60 years after CPX-351, with median OS landmarked from the HCT date not yet reached for either age subgroup. CONCLUSION: This study provides real-world survival outcomes data suggesting that CPX-351 is an effective treatment for both younger (<60 years) and older (≥60 years) adults with AML.

Comparing Findings From a Friends of Cancer Research Exploratory Analysis of Real-World End Points With the Cancer Analysis System in England.

PURPOSE: This study compared real-world end points extracted from the Cancer Analysis System (CAS), a national cancer registry with linkage to national mortality and other health care databases in England, with those from diverse US oncology data sources, including electronic health care records, insurance claims, unstructured medical charts, or a combination, that participated in the Friends of Cancer Research Real-World Evidence Pilot Project 1.0. Consistency between data sets and between real-world overall survival (rwOS) was assessed in patients with immunotherapy-treated advanced non-small-cell lung cancer (aNSCLC). PATIENTS AND METHODS: Patients with aNSCLC, diagnosed between January 2013 and December 2017, who initiated treatment with approved programmed death ligand-1 (PD-[L]1) inhibitors until March 2018 were included. Real-world end points, including rwOS and real-world time to treatment discontinuation (rwTTD), were assessed using Kaplan-Meier analysis. A synthetic data set, Simulacrum, on the basis of conditional random sampling of the CAS data was used to develop and refine analysis scripts while protecting patient privacy. RESULTS: Characteristics (age, sex, and histology) of the 2,035 patients with immunotherapy-treated aNSCLC included in the CAS study were broadly comparable with US data sets. In CAS, a higher proportion (46.7%) of patients received a PD-(L)1 inhibitor in the first line than in US data sets (18%-30%). Median rwOS (11.4 months; 95% CI, 10.4 to 12.7) and rwTTD (4.9 months; 95% CI, 4.7 to 5.1) were within the range of US-based data sets (rwOS, 8.6-13.5 months; rwTTD, 3.2-7.0 months). CONCLUSION: The CAS findings were consistent with those from US-based oncology data sets. Such consistency is important for regulatory decision making. Differences observed between data sets may be explained by variation in health care settings, such as the timing of PD-(L)1 approval and reimbursement, and data capture.