PROs vs clinician-reported adverse events in a large clinical trial: findings from the phase 3 POLARIX study

Diffuse large B-cell lymphoma (DLBCL) poses a challenge in hematology given its varied symptoms, and the complex interplay between disease and treatment effects on health-related quality of life (HRQoL). The phase 3 POLARIX study demonstrated superior progression-free survival and a similar safety profile with polatuzumab vedotin plus rituximab, cyclophosphamide, doxorubicin, and prednisone (Pola-R-CHP) vs R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone) in patients with previously untreated DLBCL. Here, we evaluate HRQoL through patient-reported outcome (PRO) instruments to fully characterize the patient experience in the POLARIX study. Changes from baseline in HRQoL, lymphoma symptoms, and gastrointestinal (GI) symptoms were assessed, as well as incidence and severity of common symptoms by PROs vs clinician-reported adverse events (AEs). Baseline characteristics of PRO-evaluable patients (N = 874) were consistent. Comparison between PROs and clinician-reported AEs revealed a notable discordance; patients generally reported a higher incidence of symptoms than clinicians, emphasizing the need for patient-centric tools to accurately capture the patient experience. Both treatments exhibited rapid and sustained improvements in HRQoL and lymphoma symptoms, with the most substantial improvements seen in global health status/QoL, lymphoma symptoms, fatigue, role, emotional, and social functioning. GI symptoms (diarrhea, constipation, nausea, and vomiting) were generally similar between treatment arms and returned to baseline levels after treatment completion. These HRQoL data underscore the complementarity of PROs, as an adjunct to clinician-reported AEs, in evaluating the efficacy and tolerability of new treatments, including Pola-R-CHP, which may represent a new benchmark for patient-reported HRQoL in previously untreated DLBCL. This trial was registered at www.clinicaltrials.gov as NCT03274492.

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Journal
Blood
Published
2025-09-25
DOI
https://doi.org/10.1182/blood.2025028848
Citations
1
Primary Topic
Lymphoma Diagnosis and Treatment
Type
article
Field-Weighted Citation Impact
1.47

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article

PROs vs clinician-reported adverse events in a large clinical trial: findings from the phase 3 POLARIX study

Hervé Tilly, Matthew Sugidono, Huilai Zhang, Jeff Porter Sharman et al.
1 citations
Blood
Lymphoma Diagnosis and Treatment
1.47
article

PROs vs clinician-reported adverse events in a large clinical trial: findings from the phase 3 POLARIX study

Hervé Tilly, Matthew Sugidono, Huilai Zhang, Jeff Porter Sharman, Carrie A. Thompson, Bei Hu, Amitkumar Mehta, Catherine Thiéblemont, Mark P. Hertzberg, Jonathan W. Friedberg, Neil Chua, Franck Morschhauser, Awadhesh Kumar Singh, Seung‐Tae Lee, Jamie Hirata, Olivier Casasnovas, Fiona Miall, Neha Mehta–Shah, P. M. Reagan, Maurizio Martelli, W. Kim, Christopher R. Flowers, Gilles Salles, Cheng‐Hong Tsai, Sunita D. Nasta, Marek Trněný, Tae Min Kim, Calvin Lee, Gustavo Fonseca, Mark Yan
article en
1 citations

Abstract

Diffuse large B-cell lymphoma (DLBCL) poses a challenge in hematology given its varied symptoms, and the complex interplay between disease and treatment effects on health-related quality of life (HRQoL). The phase 3 POLARIX study demonstrated superior progression-free survival and a similar safety profile with polatuzumab vedotin plus rituximab, cyclophosphamide, doxorubicin, and prednisone (Pola-R-CHP) vs R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone) in patients with previously untreated DLBCL. Here, we evaluate HRQoL through patient-reported outcome (PRO) instruments to fully characterize the patient experience in the POLARIX study. Changes from baseline in HRQoL, lymphoma symptoms, and gastrointestinal (GI) symptoms were assessed, as well as incidence and severity of common symptoms by PROs vs clinician-reported adverse events (AEs). Baseline characteristics of PRO-evaluable patients (N = 874) were consistent. Comparison between PROs and clinician-reported AEs revealed a notable discordance; patients generally reported a higher incidence of symptoms than clinicians, emphasizing the need for patient-centric tools to accurately capture the patient experience. Both treatments exhibited rapid and sustained improvements in HRQoL and lymphoma symptoms, with the most substantial improvements seen in global health status/QoL, lymphoma symptoms, fatigue, role, emotional, and social functioning. GI symptoms (diarrhea, constipation, nausea, and vomiting) were generally similar between treatment arms and returned to baseline levels after treatment completion. These HRQoL data underscore the complementarity of PROs, as an adjunct to clinician-reported AEs, in evaluating the efficacy and tolerability of new treatments, including Pola-R-CHP, which may represent a new benchmark for patient-reported HRQoL in previously untreated DLBCL. This trial was registered at www.clinicaltrials.gov as NCT03274492.

BloodVol. 147(3)
University of Maryland, Baltimore (US), Mayo Clinic (US), Memorial Sloan Kettering Cancer Center (US), The University of Texas MD Anderson Cancer Center (US), Washington University in St. Louis (US), Université Paris Cité (FR), Charles University (CZ), Université de Lille (FR), Hospital of the University of Pennsylvania (US), Pennsylvania Hospital (US), Saint Louis University Hospital (US), Samsung Medical Center (KR), University Hospitals of Leicester NHS Trust (GB), Tianjin Medical University Cancer Institute and Hospital (CN), Seoul National University Hospital (KR), Centre Hospitalier Universitaire de Lille (FR), University of Alabama at Birmingham (US), Kettering University (US), Roche (Canada) (CA), Assistance Publique – Hôpitaux de Paris (FR), Levine Cancer Institute (US), Sarah Cannon (US), Prince of Wales Hospital (AU), CHU Dijon Bourgogne (FR), Alberta Cancer Foundation (CA), Florida Cancer Specialists & Research Institute (US), National Taiwan University Hospital (TW), Hôpital Saint-Louis (FR), Willamette Valley Cancer Institute and Research Center (US), General University Hospital in Prague (CZ), University of Rochester (US), Université de Rouen Normandie (FR), Sungkyunkwan University (KR), Sapienza University of Rome (IT)
Amgen, AstraZeneca, F. Hoffmann-La Roche, Memorial Sloan-Kettering Cancer Center, Univerzita Karlova v Praze, University of Rochester, Regeneron Pharmaceuticals, Université de Lille, Yuhan, University of Alberta, Sungkyunkwan University, Daiichi-Sankyo, Tianjin Medical University, Genentech, Samsung, University of Texas MD Anderson Cancer Center, Wake Forest School of Medicine, lékařská fakulta Univerzity Karlovy
Openalex Percentile: Top 15%
Lymphoma Diagnosis and Treatment
1.47
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