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POD-BIN: A PROBABILITY OF DECISION BAYESIAN INTERVAL DESIGN FOR TIME-TO-EVENT DOSE-FINDING TRIALS WITH MULTIPLE TOXICITY GRADES

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Annals of Applied Statistics 2025年第1期19卷 147-168页

作者： Liu, Meizi Lin, Ji Mi, Gu Lorenzato, Christelle Chen, Xun Ji, Yuan Statistical and Quantitative Sciences Takeda Japan Department of Biostatistics and Programming Sanofi France Department of Public Health Sciences University of Chicago United States

We introduce a Bayesian framework centered on the “probability of decision” for designing dose-finding trials. The proposed PoD-BIN design evaluates the posterior predictive probabilities of up-and-down decisions. In PoDBIN, multiple grades of toxicity, categorized as mild toxicity (MT) and doselimiting toxicity (DLT), are simultaneously modeled, with the primary outcome being time-to-toxicity for both MT and DLT. This approach allows the enrollment of new patients while previously enrolled patients are still being monitored for toxicity, potentially reducing the trial duration. The Bayesian decision rules in PoD-BIN employ the probability of decisions to balance the trade-off between accelerating the trial and the risk of exposing patients to excessively toxic doses. Through numerical examples, we illustrate the trade-off between speed and safety of PoD-BIN and compare it with existing designs. PoD-BIN demonstrates the ability to control the frequency of risky decisions while simultaneously shortening trial duration in simulations. © Institute of Mathematical Statistics, 2025.

关键词： and phrases. Dose limiting toxicity equivalence interval latent probit regression moderate toxicities phase I trial time to toxicity

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Dimension reduction with expectation of conditional difference

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statistical Theory and Related Fields 2023年第3期7卷 188-201页

作者： Wenhui Sheng Qingcong Yuan Department of Mathematical and Statistical Sciences Marquette UniversityMilwaukeeWIUSA Biostatistics and Programming SanofiUSBridgewaterNJUSA

In this article,we introduce a flexible model-free approach to sufficient dimension reduction analysis using the expectation of conditional difference *** any strict conditions,such as linearity condition or constant covariance condition,the method estimates the central subspace exhaustively and efficiently under linear or nonlinear relationships between response and *** method is especially meaningful when the response is *** also studiedthe√n-consistency and asymptotic normality of the *** efficacy of our method is demonstrated through both simulations and a real data analysis.

关键词： Central subspace expectation of conditionaldifference measure sufficientdimension reduction

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On some "disadvantages" of the population approach

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AAPS Journal 2005年第2期7卷 E374-E382页

作者： Nedelman, Jerry R. Biostatistics and Statistical Programming Novartis Pharmaceuticals East Hanover NJ 07936 One Health Plaza United States

In a seminal article on population pharmacokinetic modeling, researchers demonstrated how means and variances of pharmacokinetic parameters for a patient population could be inferred from sparse data collected under conditions of routine patient care. But they also identified 4 potential concerns about their methodology: unobserved confounding variables may bias the inferences;conditions under which data are collected may lead to inaccuracies of reporting or recording;correlations among important predictor variables may reduce statistical efficiency;and costs cannot be controlled by principles of study design. Experiences are reviewed that relate to these potential disadvantages. A method is presented for diagnosing the possible presence of confounding. A model is constructed and applied that captures the influences of data inaccuracies. An example of selecting from among correlated covariates is summarized. Finally, a methodology for optimal study design is reviewed and applied to an example. Copyright ©2003. All Rights Reserved.

关键词： Confounding Errors in variables Noncompliance Optimal design Variable selection

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On multi-armed bandit designs for dose-finding clinical trials

The Journal of Machine Learning Research

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The Journal of Machine Learning Research 2021年第1期22卷 686-723页

作者： Maryam Aziz Emilie Kaufmann Marie-Karelle Riviere Spotify NYC Univ. Lille CNRS Inria Centrale Lille UMR 9189 CRIStAL Lille France Statistical Methodology Group Biostatistics and Programming department Sanofi R&D Chilly-Mazarin France

We study the problem of finding the optimal dosage in early stage clinical trials through the multiarmed bandit lens. We advocate the use of the Thompson Sampling principle, a flexible algorithm that can accommodate different types of monotonicity assumptions on the toxicity and efficacy of the doses. For the simplest version of Thompson Sampling, based on a uniform prior distribution for each dose, we provide finite-time upper bounds on the number of sub-optimal dose selections, which is unprecedented for dose-finding algorithms. Through a large simulation study, we then show that variants of Thompson Sampling based on more sophisticated prior distributions outperform state-of-the-art dose identification algorithms in different types of dose-finding studies that occur in phase I or phase I/II trials.

关键词： multi-armed bandits adaptive clinical trials phase I clinical trials phase I/II clinical trials Thompson sampling Bayesian methods

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Bayesian dose-regimen assessment in early phase oncology incorporating pharmacokinetics and pharmacodynamics

arXiv

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arXiv 2020年

作者： Gerard, Emma Zohar, Sarah Thai, Hoai-Thu Lorenzato, Christelle Riviere, Marie-Karelle Ursino, Moreno Centre de Recherche des Cordeliers Sorbonne Université Inserm Université de Paris ParisF-75006 France Oncology biostatistics Biostatistics and Programming department Sanofi R&D Vitry-sur-Seine France Statistical Methodology Group Biostatistics and Programming department Sanofi R&D Chilly-Mazarin France Translation Disease Modeling Digital and Data Science Sanofi R&D Chilly-Mazarin France F-CRIN PARTNERS platform AP-HP Université de Paris Paris France

Phase I dose-finding trials in oncology seek to find the maximum tolerated dose (MTD) of a drug under a specific schedule. Evaluating drug-schedules aims at improving treatment safety while maintaining efficacy. However, while we can reasonably assume that toxicity increases with the dose for cytotoxic drugs, the relationship between toxicity and multiple schedules remains elusive. We proposed a Bayesian dose-regimen assessment method (DRtox) using pharmacokinetics/pharmacodynamics (PK/PD) information to estimate the maximum tolerated dose-regimen (MTD-regimen), at the end of the dose-escalation stage of a trial to be recommended for the next phase. We modeled the binary toxicity via a PD endpoint and estimated the dose-regimen toxicity relationship through the integration of a dose-regimen PD model and a PD toxicity model. For the dose-regimen PD model, we considered nonlinear mixed-effects models, and for the PD toxicity model, we proposed the following two Bayesian approaches: a logistic model and a hierarchical model. We evaluated the operating characteristics of the DRtox through simulation studies under various scenarios. The results showed that our method outperforms traditional model-based designs demonstrating a higher percentage of correctly selecting the MTD-regimen. Moreover, the inclusion of PK/PD information in the DRtox helped provide more precise estimates for the entire dose-regimen toxicity curve;therefore the DRtox may recommend alternative untested regimens for expansion cohorts. The DRtox should be applied at the end of the dose-escalation stage of an ongoing trial for patients with relapsed or refractory acute myeloid leukemia (NCT03594955) once all toxicity and PK/PD data are collected. © 2020, CC-BY.

关键词： Toxicity

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Ex utero intrapartum therapy in infants with congenital diaphragmatic hernia:a propensity score matching analysis

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World Journal of Pediatric Surgery 2022年第4期5卷 267-274页

作者： Yunlong Zhao Ying Wang Chao Liu Yulin Jiang Yandong Wei Hua Meng Shan Jian Xiting Zhu Lijian Pei Xiaochen Bai Feng Feng Yan Lv Xiya Zhou Qingwei Qi Jingna Li Lishuang Ma Peking Union Medical College Graduate School BeijingChina Department of Neonatal Surgery Children’s Hospital of Capital Institute of PediatricsCapital Institute of PediatricsBeijingChina Department of Obstetrics Peking Union Medical College HospitalBeijingChina Department of Ultrasound Peking Union Medical College HospitalBeijingChina Department of Pediatrics Peking Union Medical College HospitalBeijingChina Department of Biostatistics and Statistical Programming Everest Clinical Research CorporationBeijingChina Department of Anesthesiology Peking Union Medical College HospitalBeijingChina Department of Pediatrics Beijing Obstetrics and Gynecology HospitalCapital Medical UniversityBeijingChina Department of Radiology Peking Union Medical College HospitalBeijingChina

Objective Previous studies have shown that ex utero intrapartum therapy(EXIT)is safe and feasible for newborns with congenital diaphragmatic hernia(CDH).This study reports our experience with EXIT in fetuses with CDH in an attempt to explore the efficacy of EXIT on the survival rate of this *** A retrospective analysis of the clinical data of 116 children with CDH was *** children were assigned to EXIT and non-EXIT *** score matching(PSM)toward clinical data was performed,and the clinical characteristics and outcomes were *** survival at discharge as the main outcome,logistic regression analysis was carried out to explore the efficacy of EXIT on *** During the study period,30 of 116 children received *** PSM,the survival rates of the EXIT group and the non-EXIT group were 82.76%(24/29)and 48.28%(14/29),respectively(p=0.006).EXIT(OR=0.083,95%CI=0.013to 0.525,p=0.008),liver herniation(OR=16.955,95%CI=2.342 to 122.767,p=0.005),and gestational age at diagnosis(OR=0.662,95%CI=0.497 to 0.881,p=0.005)were independent mortality-related risk factors of all children with ***-nine of 116 children underwent *** PSM,the postoperative survival rates of the EXIT group and non-EXIT group were 84.6%(22/26)and 76.9%(20/26),respectively(p=0.754).Liver herniation(OR=10.451,95%CI=1.641 to 66.544,p=0.013)and gestational age at diagnosis(OR=0.736,95%CI=0.577 to 0.938,p=0.013)were independent mortality-related risk factors of children after *** EXIT can be performed safely for selected prenatally diagnosed CDH neonates with potentially better survival and does not cause more maternal complications compared with traditional cesarean section.

关键词： hernia diagnosis congenital

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2376. Incidence ofClostridioides difficileInfection Among United States Medicare Advantage Enrollees

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Open Forum Infectious Diseases 2019年第SUPPLEMENT_2期6卷 S819–S820页

作者： Heinrich, Kirstin Emir, Birol Ivanova, Jasmina Zhou, Jingying Yu, Holly Health Economics and Outcomes Research Pfizer Inc. Collegeville Pennsylvania Biostatistics Pfizer Inc. New York New York Real-World Data and Analytics Pfizer Inc. New York New York Statistical Programming Pfizer Inc. New York New York Outcome Research Heath Economics Pfizer Inc. Collegeville Pennsylvania

Background Clostridioides difficile infection (CDI) may be life-threatening, and individuals aged ≥ 65 years are at increased risk. CDI burden among Medicare fee-for-service enrollees and nursing home residents in the United States have been characterized previously. The present study aimed to describe the incidence of CDI among Medicare Advantage Enrollees (MAEs), who account for 34% of all Medicare beneficiaries with enrollment increasing annually since 2004. Methods De-identified claims data for this retrospective cohort study were collected from the Optum® Clinformatics® Data Mart and included MAEs aged ≥ 65 years with continuous enrollment for ≥ 1 year before January 1, 2016, followed through death or disenrollment. CDI incidence was defined using the International Classification of Diseases 9th Revision diagnosis code of 008.45 or 10th Revision code of A04.7 (other than admitting diagnosis) or by treatment with nontopical metronidazole, oral vancomycin, or fidaxomicin within 14 days of CDI test. Incident CDI cases were identified from January 1 to December 31, 2016, and required that no CDI occurred within the previous 60 days in 2016. Incidence in 2016 was calculated as CDI cases and CDI patients per 100,000 person-years (PY) of observation time. Results Of 2,542,341 MAEs analyzed, 15,201 patients (0.6%) experienced a total of 18,842 incident CDI episodes. Overall, incidence rates were 762.8 CDI cases and 616.5 CDI patients per 100,000 PY. Incidence increased with age (539.6, 847.3, and 1259.6 cases per 100,000 PY in patients aged 65‒74 years, 75‒84 years, and ≥ 85 years, respectively). Most episodes (50.9%) were community acquired; the remaining 37.7% and 11.4% of episodes were hospital acquired and indeterminate, respectively. CDI patients were more likely than non-CDI patients to be older (mean age, 78.3 vs. 76.1 years, P < 0.0001), be women (64.5% vs. 58.1%, P < 0.0001), or have comorbidities (mean Charlson comorbidity index score, 4.5 vs. 1.8, P < 0.0001

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Effects of a specialized oral nutritional supplement with dietary counseling on nutritional outcomes in community-dwelling older adults at risk of malnutrition: A randomized controlled trial

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Clinical Nutrition ESPEN 2024年 63卷 1318-1318页

作者： Muangpaisan, W. Wichansawakun, S. Huynh, D.T.T. Intalapaporn, S. Chalermsri, C. Thititagul, O. Chupisanyarote, K. Chuansangeam, M. Laiteerapong, A. Yalawar, M. Huang, C. Tey, S.L. Liu, Z. Faculty of Medicine Siriraj Hospital Mahidol University Bangkok Faculty of Medicine Thammasat University Pathum Thani Thailand Abbott Nutrition Research and Development Asia-Pacific Center Singapore Singapore Thammasat University Hospital Pathum Thani Abbott Nutrition Research and Development Bangkok Thailand Biostatistics and Statistical Programming Cognizant Technologies Solution Pvt. Ltd Bangalore India Abbott Nutrition Research and Development Shanghai China

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7 Phase 3 Trial of an Oral CXCR4 Antagonist, Mavorixafor, for Treatment of Patients With WHIM Syndrome: Preliminary Results From Ongoing Open-Label Extension Period of Continuous Mavorixafor Treatment

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Clinical Immunology 2024年 262卷 109949-109949页

作者： Tarrant, Teresa Badolato, Raffaele Donadieu, Jean Dubuc, Susan Hu, Yanping Jiang, Honghua Li, Sunny Steiner, Deborah Yan, Tina Arbet-Engels, Christophe Associate Professor of Medicine Rheumatology and Immunology/Division of Rheumatology and Immunology Department of Medicine Duke University Durham NC USA Full Professor of Pediatrics Chair of Pediatrics ASST Spedali Civili Brescia/Department of Clinical and Experimental Sciences University of Brescia & ASST Spedali Civili Brescia Italy Pediatric Physician/CHU Paris Est - Hôpital d’Enfants Armand-Trousseau Paris France Senior Director Pharmacovigilance & Risk Management/X4 Pharmaceuticals Boston MA USA Director Clinical Research/X4 Pharmaceuticals Boston MA USA Senior Director Biostatistics/X4 Pharmaceuticals Boston MA USA Senior Director Statistical Programming/X4 Pharmaceuticals Boston MA USA Vice President Clinical Development/X4 Pharmaceuticals Boston MA USA Director Statistical Programming/X4 Pharmaceuticals Boston MA USA Chief Medical Officer/X4 Pharmaceuticals Boston MA USA

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POS1050 INCIDENCE RATES OF INTERSTITIAL LUNG DISEASE AMONG PATIENTS WITH RHEUMATOID ARTHRITIS TREATED WITH ABATACEPT: A POST HOC POOLED ANALYSIS FROM A COMPENDIUM OF CLINICAL TRIALS

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Annals of the Rheumatic Diseases 2023年 82卷 843-843页

作者： P. Dieudé J. Sparks A. Fischer L. Chen K. Lozenski S. Dahan M. Chaballa W. Little Paris Cité University Bichat Hospital APHP Department of Rheumatology Paris France Brigham and Women's Hospital Harvard Medical School Division of Rheumatology Inflammation and Immunity Boston United States of America Bristol Myers Squibb Lung Fibrosis Lawrenceville United States of America Syneos Health Biostatistics and Statistical Programming Richmond Canada Bristol Myers Squibb Rheumatology Pipeline and Early Assets Lawrenceville United States of America Bristol Myers Squibb Worldwide Medical Lawrenceville United States of America Bristol Myers Squibb US Medical Immunology and Fibrosis Lawrenceville United States of America Bristol Myers Squibb Worldwide Rheumatology Lawrenceville United States of America

Background Interstitial lung disease (ILD) is a recognized complication of RA. Prior studies have suggested stabilization or improvement of ILD in patients with RA (RA-ILD) treated with abatacept. [1,2] Few studies have evaluated the background incidence rate of RA-ILD. Objectives To determine the incidence rate of clinically significant ILD in a cohort of patients with RA receiving abatacept + MTX versus placebo + MTX from multiple clinical trials. Methods This retrospective analysis examined pooled safety data from ten phase 3 clinical trials of patients with RA treated with background MTX in combination with abatacept or placebo. The term ‘interstitial lung’ was used to identify incidences of ILD reported as AEs in the safety data. The exposure period for each patient was censored at first incidence of clinically significant ILD, 56 days after last study drug administration, or 1 day prior to commencement of another study drug, whichever occurred first. Poisson regression models were used to estimate crude incidence rates per 100 person-years for baseline risk factors within treatment groups, and to estimate incidence rate ratios for the placebo + MTX versus abatacept + MTX treatment groups. Disease activity parameters, DAS28 (CRP) and HAQ-DI, were estimated from baseline to the time of ILD diagnosis (as reported by AEs). Results In total, 3,708 patients (10,521 person-years) treated with abatacept + MTX and 999 patients (938 person-years) treated with placebo + MTX were included. Patients treated with placebo + MTX had a higher incidence rate of ILD per 100 person-years (95% CI) versus those treated with abatacept + MTX (0.43 [0.16–1.14] vs 0.10 [0.05–0.18], respectively; Figure 1). The incidence rate ratio of placebo + MTX versus abatacept + MTX treatment groups for the total population was 4.49 (95% CI, 1.23–13.42). For all subpopulations stratified by baseline risk factors (where ≥ 1 patient in each treatment group had an ILD event), incidence rate ratios wer

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