Validity of event-free survival as a surrogate endpoint in haematological malignancy: Review of the literature and health technology assessments

Assouline, Sarit; Wiesinger, Adriana; Spooner, Clare; Jovanović, Jelena; Schlueter, Max

doi:10.1016/j.critrevonc.2022.103711

Cited by 4 publications

(1 citation statement)

References 55 publications

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“…Surrogacy of EFS for OS has been demonstrated in multiple other tumor types, including gastroesophageal cancer, breast cancer, and hematologic malignancies. [35][36][37][38] Despite the lack of proven surrogacy, EFS can provide useful information regarding treatment tolerability and toxicity which is not encapsulated by OS. Given its ease of measurement and earlier maturity compared to OS, we advocate for EFS' use in neoadjuvant clinical trials.…”

Section: Introductionmentioning

confidence: 99%

What is the ideal endpoint in early-stage immunotherapy neoadjuvant trials in lung cancer?

Cameron,

Hines,

Torri

et al. 2023

Ther Adv Med Oncol

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Numerous clinical trials investigating neoadjuvant immune checkpoint inhibitors (ICI) have been performed over the last 5 years. As the number of neoadjuvant trials increases, attention must be paid to identifying informative trial endpoints. Complete pathologic response has been shown to be an appropriate surrogate endpoint for clinical outcomes, such as event-free survival or overall survival, in breast cancer and bladder cancer, but it is less established for non-small-cell lung cancer (NSCLC). The simultaneous advances reported with adjuvant ICI make the optimal strategy for early-stage disease debatable. Considering the long time required to conduct trials, it is important to identify optimal endpoints and discover surrogate endpoints for survival that can help guide ongoing clinical research. Endpoints can be grouped into two categories: medical and surgical. Medical endpoints are measures of survival and drug activity; surgical endpoints describe the feasibility of neoadjuvant approaches at a surgical level as well as perioperative attrition and complications. There are also several exploratory endpoints, including circulating tumor DNA clearance and radiomics. In this review, we outline the advantages and disadvantages of commonly reported endpoints for clinical trials of neoadjuvant regimens in NSCLC.

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Section: Introductionmentioning

confidence: 99%

What is the ideal endpoint in early-stage immunotherapy neoadjuvant trials in lung cancer?

Cameron,

Hines,

Torri

et al. 2023

Ther Adv Med Oncol

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Time‐to‐Event Endpoints in Imaging Biomarker Studies

Chen,

Wang

2024

Magnetic Resonance Imaging

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Time‐to‐event endpoints are widely used as measures of patients' well‐being and indicators of prognosis. In imaging‐based biomarker studies, there are increasingly more studies that focus on examining imaging biomarkers' prognostic or predictive utilities on those endpoints, whether in a trial or an observational study setting. In this educational review article, we briefly introduce some basic concepts of time‐to‐event endpoints and point out potential pitfalls in the context of imaging biomarker research in hope of improving radiologists' understanding of related subjects. Besides, we have included some review and discussions on the benefits of using time‐to‐event endpoints and considerations on selecting overall survival or progression‐free survival for primary analysis.Level of Evidence5Technical EfficacyStage 3

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Population‐level 5‐year event‐free survival for children with cancer in Australia

Youlden,

Baade,

Gottardo

et al. 2024

Pediatric Blood & Cancer

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BackgroundEvent‐free survival (EFS) considers other adverse events in addition to mortality. It therefore provides a more complete understanding of the effectiveness and consequences of treatment than standard survival measures, but is rarely reported at the population level for childhood cancer.ProcedureOur study cohort (n = 7067) was obtained from the Australian Childhood Cancer Registry, including children aged under 15 diagnosed with cancer between 2006 and 2015, with follow‐up potentially available to 31 December 2020. The events of interest were relapse following remission, progressive disease, diagnosis of a second primary cancer or death from any cause. Five‐year EFS and all‐cause observed survival were both calculated, stratified by type of childhood cancer, remoteness of residence and stage at diagnosis. Differences in EFS were assessed using multivariable flexible parametric models.ResultsApproximately one quarter of patients (n = 1605 of 7067, 23%) experienced at least one of the events of interest within 5 years of diagnosis. Relapse was twice as common for children with metastatic/advanced disease (22%) versus children with localised/limited cancers (11%). Overall 5‐year EFS was 75.0% (95% confidence interval [CI]: 73.9%–76.0%), compared to 85.8% observed survival (95% CI: 85.0%–86.6%). Patients with other gliomas had the lowest EFS (35.4%, 95% CI: 27.8%–43.1%). EFS was significantly lower among children with acute myeloid leukaemia in outer regional/remote areas compared to major cities (adjusted hazard ratio [HR] = 1.90, 95% CI: 1.20–3.00).ConclusionsReporting EFS at a population level provides further insight on a wider range of impacts apart from mortality alone, contributing towards efforts to improve the management and outcomes of childhood cancer.

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Validity of event-free survival as a surrogate endpoint in haematological malignancy: Review of the literature and health technology assessments

Cited by 4 publications

References 55 publications

What is the ideal endpoint in early-stage immunotherapy neoadjuvant trials in lung cancer?

What is the ideal endpoint in early-stage immunotherapy neoadjuvant trials in lung cancer?

Time‐to‐Event Endpoints in Imaging Biomarker Studies

Population‐level 5‐year event‐free survival for children with cancer in Australia

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