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Statistical considerations in long-term efficacy evaluation of anti-cancer therapies.

ABSTRACT: Anti-cancer therapy has been a significant focus of research. Developing and marketing various types and mechanisms of anti-cancer therapies benefit a variety of patients significantly. The long-term benefit to patients in evaluating the risk-benefit ratio of anti-cancer therapy has become a significant concern. This paper discusses the evaluation of long-term efficacy within the estimand framework and summarizes the various strategies for addressing potential intercurrent events. Non-proportional hazards of survival data may arise with novel anti-cancer therapies, leading to potential bias in conventional evaluation methods. This paper reviews statistical methods for addressing this issue, including novel endpoints, hypothesis testing, and efficacy estimation methods. We also discuss the influences of treatment switching. Although advanced methods have been developed to address the non-proportional hazard, they still have limitations that require continued collaborative efforts to resolve issues.

SUBMITTER: Li R

PROVIDER: S-EPMC10579585 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature

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Statistical considerations in long-term efficacy evaluation of anti-cancer therapies.

Li Ruobing R Zhang Jingyi J Wang Jingzhao J Wang Jun J

Frontiers in pharmacology 20231003

Anti-cancer therapy has been a significant focus of research. Developing and marketing various types and mechanisms of anti-cancer therapies benefit a variety of patients significantly. The long-term benefit to patients in evaluating the risk-benefit ratio of anti-cancer therapy has become a significant concern. This paper discusses the evaluation of long-term efficacy within the estimand framework and summarizes the various strategies for addressing potential intercurrent events. Non-proportion ...[more]

PMID: 37854717

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Project description:BackgroundAntithrombotic therapy was commonly applied in treating Kawasaki disease (KD) children; however, the effects and mechanisms of different plans were not fully elucidated. In this study, we aimed to evaluate different antithrombotic drugs.MethodsEighty-two children diagnosed with KD in Hebei Children's Hospital from January 2017 to January 2020 were recruited. For cohort study, KD children were divided into a series of groups according to whether they were complicated with coronary artery lesions (CAL), drug therapy plan, and the presence of liver damage. The thromboelastogram (TEG) indexes [clotting time (R), clot formation time (K), clot formation angle (α), maximum amplitude of the clot (MA), arachidonic acid (AA), and adenosine diphosphate (ADP)] were employed to evaluate the relationship between disease state and drug treatment efficacy. Meanwhile, children were divided into different therapy groups according to their degree of CAL, the treatment efficiency was evaluated by TEG indexes, and the bleeding ratio was recorded. In addition, the warfarin metabolic gene was detected to explain the changes of coagulation parameters in children treated with warfarin.ResultsThe R value and coagulation index (CoI) were significantly lower (P<0.05) and MA value was significantly higher (P<0.05) in CAL group than those in non-coronary artery lesion (NCAL) group. There were significant individual differences in platelet inhibition between aspirin and dipyridamole groups. The AA% in aspirin group was 64% [95% confidence interval (CI): 49% to 74.3%] and the ADP% was 28.5% (95% CI: 26.2% to 37.2%) in dipyridamole group, and were significantly between groups. Warfarin and aspirin had a synergistic effect in anticoagulation. Warfarin metabolic gene detection was shown to be helpful for adjusting warfarin treatment dose, and shortening the initial attainment time of standard international normalized ratio (INR) index. The R value was significantly higher (P<0.05) in the liver injury group than that in the control group.ConclusionsKD Children complicated with CAL presented a hypercoagulable state, and the CAL was predictable with the R value. Warfarin metabolic gene detection and TEG can effectively evaluate and guide short- and long-term antithrombotic therapy in KD children.

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Statistical considerations in long-term efficacy evaluation of anti-cancer therapies.

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Statistical considerations in long-term efficacy evaluation of anti-cancer therapies.

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