Integrated modeling of biomarkers, survival and safety in clinical oncology drug development DOI Creative Commons
Han Liu, Eman Ibrahim, Maddalena Centanni

et al.

Advanced Drug Delivery Reviews, Journal Year: 2024, Volume and Issue: unknown, P. 115476 - 115476

Published: Nov. 1, 2024

Model-based approaches, including population pharmacokinetic-pharmacodynamic modeling, have become an essential component in the clinical phases of oncology drug development. Over past two decades, models evolved to describe temporal dynamics biomarkers and tumor size, treatment-related adverse events, their links survival. Integrated models, defined here as that incorporate at least pharmacodynamic/ outcome variables, are applied answer development questions through simulations, e.g. support exploration alternative dosing strategies study designs subgroups patients or other indications. It is expected these pharmacometric approaches will be expanded regulatory authorities place further emphasis on early individualized dosage optimization inclusive patient-focused strategies. This review provides overview integrated literature, examples considerations need made when applying advanced outlook expansion model-informed anticancer drugs.

Language: Английский

Population Pharmacokinetic and Exposure–Response Analyses for Ponatinib in the Phase 3 PhALLCON Study DOI Creative Commons
Michael J. Hanley, Thomas R. Larson, Paul M. Diderichsen

et al.

Clinical and Translational Science, Journal Year: 2025, Volume and Issue: 18(3)

Published: March 1, 2025

ABSTRACT In March 2024, ponatinib received accelerated FDA approval for the treatment of newly diagnosed Philadelphia chromosome‐positive acute lymphoblastic leukemia (Ph + ALL) in combination with chemotherapy based on Phase 3 PhALLCON study (NCT03589326), which demonstrated a higher rate minimal residual disease (MRD)‐negative complete remission (CR) at end induction (EOI) (34.4%) versus imatinib (16.7%; p = 0.002). Patients (30 mg QD reduction to 15 upon achievement MRD‐negative CR EOI) or (600 QD) combined 20 cycles reduced‐intensity (induction: cycles; consolidation: 6 and maintenance: 11 cycles). Ponatinib pharmacokinetics (PK) were similar patients previous population PK analysis. Bayesian re‐estimation previously developed model adequately described data. Exposure–efficacy analyses did not identify significant relationship between exposure probability EOI ( 0.619), suggesting consistent efficacy benefit across exposures. was predictor arterial occlusive events, venous thromboembolic thrombocytopenia, lipase increase > 0.05). However, exposures associated hypertension 0.0340) alanine aminotransferase (ALT) 0.0034). Dose from 30 predicted decrease odds experiencing by 37.7% ALT 44.2%. Collectively, exposure–response support favorable benefit–risk profile approved dosage reduced EOI), chemotherapy, frontline Ph ALL.

Language: Английский

Citations

0

Optimizing Brentuximab Vedotin Dosing in Pediatric Patients with Advanced Hodgkin Lymphoma: A Population Pharmacokinetic and Exposure‐Response Analysis DOI Creative Commons
Xiaofei Zhou, Diane R. Mould, Lia Gore

et al.

Clinical Pharmacology & Therapeutics, Journal Year: 2025, Volume and Issue: unknown

Published: March 17, 2025

Pediatric patients with advanced‐stage newly diagnosed Hodgkin lymphoma (HL) were treated brentuximab vedotin (BV) combined adriamycin, vinblastine, and dacarbazine (A + AVD). Weight‐based BV dosing is employed in adult patients, while both body weight‐ surface area (BSA)‐based are used pediatric patients. Data from two studies for a population pharmacokinetics (PK) analysis. Study 1 was phase I/II dose‐escalation study which relapsed or refractory systemic anaplastic large‐cell HL received single‐agent weight‐based 1.4–1.8 mg/kg every 3 weeks. 2 tested BSA‐based 48 mg/m weeks AVD advanced‐stage, HL. Sources of PK variability quantified using nonlinear mixed‐effects modeling. The relationships between antibody‐drug conjugate (ADC) payload monomethyl auristatin E (MMAE) exposures progression‐free survival (PFS) incidence adverse events analyzed by Cox proportional hazards logistic regression, respectively. Population models ADC MMAE developed data 95 BSA identified as significant covariate the clearance MMAE. resulted similar across age groups (< 12, 12–16, > 16 years). A increase ( P < 0.05) febrile neutropenia related to increasing exposure No apparent relationship PFS. analyses support combination

Language: Английский

Citations

0

A Systematic Evaluation of the Dosing Regimens for Approved Targeted Therapies and Immune Checkpoint Inhibitors in Metastatic Renal Cell Carcinoma From a Project OPTIMUS Perspective DOI Creative Commons
Zhiyuan Tan, Swantje Völler, Aymara Sancho‐Araiz

et al.

The Journal of Clinical Pharmacology, Journal Year: 2025, Volume and Issue: unknown

Published: May 2, 2025

Abstract Targeted therapies and immune checkpoint inhibitors (ICIs) have significantly improved survival outcomes in metastatic renal cell carcinoma (mRCC) but are often associated with high rates of adverse events, leading to dose reductions or treatment discontinuation. The FDA's recent initiative, Project OPTIMUS, emphasizes the importance optimizing dosing regimens oncology clinical development, moves beyond conventional maximum tolerated approach. In this study, we aimed review redefine approved strategies for targeted ICIs mRCC from OPTIMUS perspective, including pazopanib, axitinib, cabozantinib, sunitinib, everolimus, nivolumab. A comprehensive summary FDA pharmacology reviews studies performed routine practice was conducted, alongside model‐informed simulations pharmacokinetic profiles alternative regimens. Results demonstrated that actual doses were 46.1% 86% lower than dosages, up 75% patients requiring adjustments. Model‐informed suggested most therapies, a 14%–50% reduction maintained comparable efficacy while improving tolerability. For nivolumab, confirmed adequate drug exposure flat regimens, without an increase effects. conclusion, identified optimized could improve tolerability maintaining mRCC. We suggest these should be considered use optimal range included labels support pharmacokinetically guided individualization practice.

Language: Английский

Citations

0

Integrated modeling of biomarkers, survival and safety in clinical oncology drug development DOI Creative Commons
Han Liu, Eman Ibrahim, Maddalena Centanni

et al.

Advanced Drug Delivery Reviews, Journal Year: 2024, Volume and Issue: unknown, P. 115476 - 115476

Published: Nov. 1, 2024

Model-based approaches, including population pharmacokinetic-pharmacodynamic modeling, have become an essential component in the clinical phases of oncology drug development. Over past two decades, models evolved to describe temporal dynamics biomarkers and tumor size, treatment-related adverse events, their links survival. Integrated models, defined here as that incorporate at least pharmacodynamic/ outcome variables, are applied answer development questions through simulations, e.g. support exploration alternative dosing strategies study designs subgroups patients or other indications. It is expected these pharmacometric approaches will be expanded regulatory authorities place further emphasis on early individualized dosage optimization inclusive patient-focused strategies. This review provides overview integrated literature, examples considerations need made when applying advanced outlook expansion model-informed anticancer drugs.

Language: Английский

Citations

0