Quality of patient‐reported outcomes in oncology clinical trials using immune checkpoint inhibitors: A systematic review

Malone, Eoghan Ruadh; Barua, Reeta; Meti, Nicholas; Li, Xuan; Fazelzad, Rouhi; Hansen, Aaron R.

doi:10.1002/cam4.4086

Cited by 5 publications

(4 citation statements)

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“…However, adverse effects were the cause of 20% of patients stopping treatment, the most common being rash, fatigue, or weight loss. Several studies have shown that the impact of adverse effects reported by participants in clinical trials is understated for both molecular and immunotherapies [15][16][17][18][19]. A systematic review by Peron and colleagues showed that 90% of 325 randomized controlled trials (RCTs) did not adhere to the Consolidated Standards of Reporting Trials (CONSORT) guidelines in the reporting methodology of adverse event collection [16].…”

Section: Discussionmentioning

confidence: 99%

Adjuvant Immunotherapy in Curative Intent Esophageal Cancer Resection Patients: Real-World Experience within an Integrated Health System

Kwak,

Banks,

Hung

et al. 2023

Cancers

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Background: Adjuvant immunotherapy has been shown in clinical trials to prolong the survival of patients with esophageal cancer. We report our initial experience with immunotherapy within an integrated health system. Methods: A retrospective cohort study was performed reviewing patients undergoing minimally invasive esophagectomy at our institution between 2017 and 2021. The immunotherapy cohort was assessed for completion of treatment, adverse effects, and disease progression, with emphasis on patients who received surgery in 2021 and their eligibility to receive nivolumab. Results: There were 39 patients who received immunotherapy and 137 patients who did not. In logistic regression, immunotherapy was not found to have a statistically significant impact on 1-year overall survival after adjusting for age and receipt of adjuvant chemoradiation. Only seven patients out of 39 who received immunotherapy successfully completed treatment (18%), with the majority failing therapy due to disease progression or side effects. Of the 17 patients eligible for nivolumab, 13 patients received it (76.4%), and three patients completed a full course of treatment. Conclusions: Despite promising findings of adjuvant immunotherapy improving the survival of patients with esophageal cancer, real-life practice varies greatly from clinical trials. We found that the majority of patients were unable to complete immunotherapy regimens with no improvement in overall 1-year survival.

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Section: Discussionmentioning

confidence: 99%

Adjuvant Immunotherapy in Curative Intent Esophageal Cancer Resection Patients: Real-World Experience within an Integrated Health System

Kwak,

Banks,

Hung

et al. 2023

Cancers

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“…We herein report the results for the subset of patients with high-grade serous ovarian carcinoma (HGSOC) histology. We additionally included patientreported outcome (PRO) assessments, which have been poorly explored in immunotherapy clinical trials, [10][11][12] to better understand patient experience and quality of life (QOL) throughout this trial.…”

Section: Introductionmentioning

confidence: 99%

Randomized phase 2 trial of tremelimumab and durvalumab in combination versus sequentially in recurrent platinum‐resistant ovarian cancer

Hinchcliff,

Knisely,

Adjei

et al. 2023

Cancer

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BackgroundSingle‐agent immune checkpoint inhibitors (ICIs) have demonstrated limited responses in recurrent ovarian cancer; however, 30%–40% of patients achieve stable disease. The primary objective was to estimate progression‐free survival (PFS) after sequential versus combination cytotoxic T‐lymphocyte antigen 4 and programmed death ligand 1 ICIs in patients with platinum‐resistant high‐grade serous ovarian cancer (HGSOC).MethodsPatients were randomized to a sequential arm (tremelimumab followed by durvalumab on progression) or a combination arm (tremelimumab plus durvalumab, followed by durvalumab) via a Bayesian adaptive design that made it more likely for patients to be randomized to the more effective arm. The primary end point was immune‐related PFS (irPFS).ResultsSixty‐one subjects were randomized to sequential (n = 38) or combination therapy (n = 23). Thirteen patients (34.2%) in the sequential arm received durvalumab. There was no difference in PFS in the sequential arm (1.84 months; 95% CI, 1.77–2.17 months) compared with the combination arm (1.87 months; 95% CI, 1.77–2.43 months) (p = .402). In the sequential arm, no responses were observed, although 12 patients (31.6%) demonstrated stable disease. In the combination arm, two patients (8.7%) had partial response, whereas one patient (4.4%) had stable disease. Adverse events were consistent with those previously reported for ICIs. Patient‐reported outcomes were similar in both arms.ConclusionsThere was no difference in irPFS for combination tremelimumab plus durvalumab compared to tremelimumab alone (administered as part of a sequential treatment strategy) in a heavily pretreated population of patients with platinum‐resistant HGSOC. Response rates were comparable to prior reports, although the combination regimen did not add significant benefit, as has been previously described.

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“…5 Regulatory agencies have recognized the value and challenges of incorporating COAs in drug development, 7,8 and multiple initiatives and guidelines for trial design, analysis, and reporting advocate for the use of COAs across clinical research. [9][10][11][12] PRO use has been evaluated in general oncology trials and focused cancer trial subsets [13][14][15][16][17][18] but the comprehensive use of COAs in oncology trials across time and how it compares to the use of COAs in non-oncology clinical trials has not been evaluated. Moreover, trends in relation to landmark regulatory and advocacy events have not been assessed, and with few exceptions 19,20 previous evaluations relied on manual curation of the literature which is not feasible for periodic assessments.…”

Section: Introductionmentioning

confidence: 99%

“…PRO use has been evaluated in general oncology trials and focused cancer trial subsets 13 , 14 , 15 , 16 , 17 , 18 but the comprehensive use of COAs in oncology trials across time and how it compares to the use of COAs in non‐oncology clinical trials has not been evaluated. Moreover, trends in relation to landmark regulatory and advocacy events have not been assessed, and with few exceptions 19 , 20 previous evaluations relied on manual curation of the literature which is not feasible for periodic assessments.…”

Section: Introductionmentioning

confidence: 99%

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

et al. 2023

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BackgroundClinical outcome assessments (COAs) are key to patient‐centered evaluation of novel interventions and supportive care. COAs are particularly informative in oncology where a focus on how patients feel and function is paramount, but their incorporation in trial outcomes have lagged that of traditional survival and tumor responses. To understand the trends of COA use in oncology and the impact of landmark efforts to promote COA use, we computationally surveyed oncology clinical trials in ClinicalTrials.gov comparing them to the rest of the clinical research landscape.MethodsOncology trials were identified using medical subject heading neoplasm terms. Trials were searched for COA instrument names obtained from PROQOLID. Regression analyses assessed chronological and design‐related trends.ResultsEighteen percent of oncology interventional trials initiated 1985–2020 (N = 35,415) reported using one or more of 655 COA instruments. Eighty‐four percent of the COA‐using trials utilized patient‐reported outcomes, with other COA categories used in 4–27% of these trials. Likelihood of COA use increased with progressing trial phase (OR = 1.30, p < 0.001), randomization (OR = 2.32, p < 0.001), use of data monitoring committees (OR = 1.26, p < 0.001), study of non‐FDA‐regulated interventions (OR = 1.23, p = 0.001), and in supportive care versus treatment‐focused trials (OR = 2.94, p < 0.001). Twenty‐six percent of non‐oncology trials initiated 1985–2020 (N = 244,440) reported COA use; they had similar COA‐use predictive factors as oncology trials. COA use increased linearly over time (R = 0.98, p < 0.001), with significant increases following several individual regulatory events.ConclusionWhile COA use across clinical research has increased over time, there remains a need to further promote COA use particularly in early phase and treatment‐focused oncology trials.

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Quality of patient‐reported outcomes in oncology clinical trials using immune checkpoint inhibitors: A systematic review

Abstract: This is an open access article under the terms of the Creat ive Commo ns Attri bution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.

Cited by 5 publications

References 24 publications

Adjuvant Immunotherapy in Curative Intent Esophageal Cancer Resection Patients: Real-World Experience within an Integrated Health System

Adjuvant Immunotherapy in Curative Intent Esophageal Cancer Resection Patients: Real-World Experience within an Integrated Health System

Randomized phase 2 trial of tremelimumab and durvalumab in combination versus sequentially in recurrent platinum‐resistant ovarian cancer

Clinical outcome assessment trends in clinical trials—Contrasting oncology and non‐oncology trials

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