Methodological standards for using the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE) in cancer clinical trials

Basch, Ethan; Thanarajasingam, Gita; Dueck, Amylou C.

doi:10.1177/17407745221093922

Cited by 5 publications

(4 citation statements)

References 10 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…While a tailored tool cannot exhaustively capture all symptoms impacting tolerability, tools such as the full PRO-CTCAE already exist for this purpose and could be applied alongside our tailored survey at critical timepoints in a trial to balance completeness with efficiency. If there is a reasonable suspicion that an experimental therapy will have an AE of interest that is not covered by our tailored list, or that certain disease-related symptoms might be present at baseline, then additional specific terms could be added to our questionnaire, in keeping with recommendations for defining AE surveillance and for the use of PRO-CTCAE in phase I trial design [ 32 , 35 ]. Further, to ensure additional symptoms impacting tolerability are not overlooked, the option of including structured or unstructured free text reporting with characterization of severity/interference/frequency/amount attributes and symptom mapping could be considered [ 36 ].…”

Section: Discussionmentioning

confidence: 99%

A tailored phase I-specific patient-reported outcome (PRO) survey to capture the patient experience of symptomatic adverse events

et al. 2023

View full text Add to dashboard Cite

Background Patient perspectives are fundamental to defining tolerability of investigational anti-neoplastic therapies in clinical trials. Phase I trials present a unique challenge in designing tools for efficiently collecting patient-reported outcomes (PROs) given the difficulty of anticipating adverse events of relevance. However, phase I trials also offer an opportunity for investigators to optimize drug dosing based on tolerability for future larger-scale trials and in eventual clinical practice. Existing tools for comprehensively capturing PROs are generally cumbersome and are not routinely used in phase I trials. Methods Here, we describe the creation of a tailored survey based on the National Cancer Institute’s PRO-CTCAE for collecting patients’ perspectives on symptomatic adverse events in phase I trials in oncology. Results We describe our stepwise approach to condensing the original 78-symptom library into a modified 30 term core list of symptoms which can be efficiently applied. We further show that our tailored survey aligns with phase I trialists’ perspectives on symptoms of relevance. Conclusions This tailored survey represents the first PRO tool developed specifically for assessing tolerability in the phase I oncology population. We provide recommendations for future work aimed at integrating this survey into clinical practice.

show abstract

Section: Discussionmentioning

confidence: 99%

A tailored phase I-specific patient-reported outcome (PRO) survey to capture the patient experience of symptomatic adverse events

et al. 2023

View full text Add to dashboard Cite

show abstract

“…Tissue and blood biomarkers were obtained, and correlations with clinical outcomes (OS and PFS) were assessed. Safety and toxic effects were evaluated as indicated by intensity and incidence of adverse events, and graded according to Common Terminology Criteria for Adverse Events, version 4.03 . All serious adverse events that occurred following the patient’s written consent and through 100 days of treatment discontinuation were reported by the sites and to BMS Worldwide Safety.…”

Section: Methodsmentioning

confidence: 99%

Intensity-Modulated Reirradiation Therapy With Nivolumab in Recurrent or Second Primary Head and Neck Squamous Cell Carcinoma

Saba,

Wong,

Nasti

et al. 2024

JAMA Oncol

View full text Add to dashboard Cite

ImportanceIntensity-modulated radiation therapy (IMRT) reirradiation of nonmetastatic recurrent or second primary head and neck squamous cell carcinoma (HNSCC) results in poor progression-free survival (PFS) and overall survival (OS).ObjectiveTo investigate the tolerability, PFS, OS, and patient-reported outcomes with nivolumab (approved standard of care for patients with HNSCC) during and after IMRT reirradiation.Design, Setting, and ParticipantsIn this multicenter nonrandomized phase 2 single-arm trial, the treatment outcomes of patients with recurrent or second primary HNSCC who satisfied recursive partitioning analysis class 1 and 2 definitions were evaluated. Between July 11, 2018, and August 12, 2021, 62 patients were consented and screened. Data were evaluated between June and December 2023.InterventionSixty- to 66-Gy IMRT in 30 to 33 daily fractions over 6 to 6.5 weeks with nivolumab, 240 mg, intravenously 2 weeks prior and every 2 weeks for 5 cycles during IMRT, then nivolumab, 480 mg, intravenously every 4 weeks for a total nivolumab duration of 52 weeks.Main Outcomes and MeasuresThe primary end point was PFS. Secondary end points included OS, incidence, and types of toxic effects, including long-term treatment-related toxic effects, patient-reported outcomes, and correlatives of tissue and blood biomarkers.ResultsA total of 62 patients were screened, and 51 were evaluable (median [range] age was 62 [56-67] years; 42 [82%] were male; 6 [12%] had p16+ disease; 38 [75%] had salvage surgery; and 36 [71%.] had neck dissection). With a median follow-up of 24.5 months (95% CI, 19.0-25.0), the estimated 1-year PFS was 61.7% (95% CI, 49.2%-77.4%), rejecting the null hypothesis of 1-year PFS rate of less than 43.8% with 1-arm log-rank test P = .002 within a 1-year timeframe. The most common treatment-related grade 3 or higher adverse event (6 [12%]) was lymphopenia with 2 patients (4%) and 1 patient each (2%) exhibiting colitis, diarrhea, myositis, nausea, mucositis, and myasthenia gravis. Functional Assessment of Cancer Therapy–General and Functional Assessment of Cancer Therapy–Head and Neck Questionnaire quality of life scores remained stable and consistent across all time points. A hypothesis-generating trend favoring worsening PFS and OS in patients with an increase in blood PD1+, KI67+, and CD4+ T cells was observed.Conclusions and RelevanceThis multicenter nonrandomized phase 2 trial of IMRT reirradiation therapy and nivolumab suggested a promising improvement in PFS over historical controls. The treatment was well tolerated and deserves further evaluation.Trial RegistrationClinicalTrials.gov Identifier: NCT03521570

show abstract

“…Therefore, algorithms are being developed to generate a single composite numerical grade for each PRO-CTCAE symptomatic adverse event based on the mapping of its individual item scores and to evaluate the composite grades to assure that their validity, reliability and sensitivity to change are comparable with individual item scores. At the present time, it is recommended to select specific PRO-CTCAE items for a given trial based on common and expected reactions [72].…”

Section: Other Instrumentsmentioning

confidence: 99%

Guidelines for the Use and Reporting of Patient-Reported Outcomes in Multiple Myeloma Clinical Trials

Laane,

Salek,

Oliva

et al. 2023

Cancers

View full text Add to dashboard Cite

In the era of personalized medicine there is an increasing need for the assessment of patient-reported outcomes (PROs) to become a standard of patient care. Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient’s own perspective. It is well established that active multiple myeloma (MM) can be characterized by a high burden of disease and treatment-related symptoms, with considerable worsening of quality of life (QoL). In general, and over the past decade, the focus has shifted to obtaining the most durable remissions with the best QoL as primary goals for MM treatment. Patients place considerable value on their QoL and communicating about QoL data prior to treatment decisions allows them to make informed treatment choices. Consequently, optimization of QoL of patients with MM is an important therapeutic goal and the incorporation of PROs into clinical trials has the potential of improving treatment outcomes. In this regard, guidance for the use and reporting of PROMs in MM in clinical trials is warranted. Under the auspices of the European Hematology Association, evidence-based guidelines for the use and reporting of PROs in patients with MM have been developed according to the EHA’s core Guidelines Development Methodology. This document provides general considerations for the choice of PROMs in MM clinical trials as well as a series of recommendations covering a selection of PROMs in MM clinical trials; the mode of administration; timing of assessments; strategies to minimize missing data; sample size calculation; reporting of results; and interpretation of results.

show abstract

Methodological standards for using the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE) in cancer clinical trials

Cited by 5 publications

References 10 publications

A tailored phase I-specific patient-reported outcome (PRO) survey to capture the patient experience of symptomatic adverse events

A tailored phase I-specific patient-reported outcome (PRO) survey to capture the patient experience of symptomatic adverse events

Intensity-Modulated Reirradiation Therapy With Nivolumab in Recurrent or Second Primary Head and Neck Squamous Cell Carcinoma

Guidelines for the Use and Reporting of Patient-Reported Outcomes in Multiple Myeloma Clinical Trials

Contact Info

Product

Resources

About