Most patients reported positively or neutrally of having served as controls in the trials within cohorts design

Verweij, Maaike E; Gal, Roxanne; Burbach, J.P.M.; Young‐Afat, Danny A.; Velden, Joanne M. van der; Graaf, Rieke van der; May, Anne M; Relton, Clare; Intven, Martijn; Verkooijen, Helena M.

doi:10.1016/j.jclinepi.2022.04.015

Cited by 9 publications

(8 citation statements)

References 25 publications

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“…The fact that participants randomized for the control arm are not specifically informed of their participation in a specific trial raised some concerns amongst physicians. However, a recent study has shown that patients themselves did not experience this as a problem [63].…”

Section: Discussionmentioning

confidence: 99%

A nationwide randomized controlled trial on additional treatment for isolated local pancreatic cancer recurrence using stereotactic body radiation therapy (ARCADE)

Goor

Daamen

Besselink

et al. 2022

Trials

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Background Disease recurrence is the main cause of mortality after resection of pancreatic ductal adenocarcinoma (PDAC). In 20–30% of resected patients, isolated local PDAC recurrence occurs. Retrospective studies have suggested that stereotactic body radiation therapy (SBRT) might lead to improved local control in these patients, potentially having a beneficial effect on both survival and quality of life. The “nationwide randomized controlled trial on additional treatment for isolated local pancreatic cancer recurrence using stereotactic body radiation therapy” (ARCADE) will investigate the value of SBRT in addition to standard of care in patients with isolated local PDAC recurrence compared to standard of care alone, regarding both survival and quality of life outcomes. Methods The ARCADE trial is nested within a prospective cohort (Dutch Pancreatic Cancer Project; PACAP) according to the ‘Trials within Cohorts’ design. All PACAP participants with isolated local PDAC recurrence after primary resection who provided informed consent for being randomized in future studies are eligible. Patients will be randomized for local therapy (5 fractions of 8 Gy SBRT) in addition to standard of care or standard of care alone. In total, 174 patients will be included. The main study endpoint is survival after recurrence. The most important secondary endpoint is quality of life. Discussion It is hypothesized that additional SBRT, compared to standard of care alone, improves survival and quality of life in patients with isolated local recurrence after PDAC resection. Trial registration ClinicalTrials.gov registration NCT04881487. Registered on May 11, 2021.

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Section: Discussionmentioning

confidence: 99%

A nationwide randomized controlled trial on additional treatment for isolated local pancreatic cancer recurrence using stereotactic body radiation therapy (ARCADE)

Goor

Daamen

Besselink

et al. 2022

Trials

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“… 65 66 Cancer patients participating in ongoing TwiCs 65 and a separate study were surveyed between two to six years after cohort entry. 66 The approach was found to be generally well accepted by patients with only 2% of patients in the trial usual care control group stating that they would feel negative about not being selected for interventions while their data are being used as a comparator. Both evaluations support the use of the TwiCs design with the staged-informed consent procedure.…”

Section: Discussionmentioning

confidence: 99%

Randomised trials conducted using cohorts: a scoping review

Nickolls,

Relton,

Hemkens

et al. 2024

BMJ Open

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IntroductionCohort studies generate and collect longitudinal data for a variety of research purposes. Randomised controlled trials (RCTs) increasingly use cohort studies as data infrastructures to help identify and recruit trial participants and assess outcomes.ObjectiveTo examine the extent, range and nature of research using cohorts for RCTs and describe the varied definitions and conceptual boundaries for RCTs using cohorts.DesignScoping review.Data sourcesSearches were undertaken in January 2021 in MEDLINE (Ovid) and EBM Reviews—Cochrane Methodology Registry (Final issue, third Quarter 2012).Eligibility criteriaReports published between January 2007 and December 2021 of (a) cohorts used or planned to be used, to conduct RCTs, or (b) RCTs which use cohorts to recruit participants and/or collect trial outcomes, or (c) methodological studies discussing the use of cohorts for RCTs.Data extraction and synthesisData were extracted on the condition being studied, age group, setting, country/continent, intervention(s) and comparators planned or received, unit of randomisation, timing of randomisation, approach to informed consent, study design and terminology.ResultsA total of 175 full-text articles were assessed for eligibility. We identified 61 protocols, 9 descriptions of stand-alone cohorts intended to be used for future RCTs, 39 RCTs using cohorts and 34 methodological papers.The use and scope of this approach is growing. The thematics of study are far-ranging, including population health, oncology, mental and behavioural disorders, and musculoskeletal conditions.Authors reported that this approach can lead to more efficient recruitment, more representative samples, and lessen disappointment bias and crossovers.ConclusionThis review outlines the development of cohorts to conduct RCTs including the range of use and innovative changes and adaptations. Inconsistencies in the use of terminology and concepts are highlighted. Guidance now needs to be developed to support the design and reporting of RCTs conducted using cohorts.

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“…Women who accepted HBOT and provided additional informed consent were referred for HBOT, whereas women who declined received standard care. Following the TWICS concept, participants randomized to the control arm were not informed about the trial, and their UMBRELLA cohort data were used comparatively . For logistic reasons, participants were recruited and randomized in 4 batches between November 2019 and August 2022 …”

Section: Methodsmentioning

confidence: 99%

Hyperbaric Oxygen Therapy and Late Local Toxic Effects in Patients With Irradiated Breast Cancer

Mink van der Molen,

Batenburg,

Maarse

et al. 2024

JAMA Oncol

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ImportanceHyperbaric oxygen therapy (HBOT) is proposed as treatment for late local toxic effects after breast irradiation. Strong evidence of effectiveness is lacking.ObjectiveTo assess effectiveness of HBOT for late local toxic effects in women who received adjuvant radiotherapy for breast cancer.Design, Setting, and ParticipantsThis was a hospital-based, pragmatic, 2-arm, randomized clinical trial nested within the prospective UMBRELLA cohort following the trials within cohorts design in the Netherlands. Participants included 189 women with patient-reported moderate or severe breast, chest wall, and/or shoulder pain in combination with mild, moderate, or severe edema, fibrosis, or movement restriction 12 months or longer after breast irradiation. Data analysis was performed from May to September 2023.InterventionReceipt of 30 to 40 HBOT sessions over a period of 6 to 8 consecutive weeks.Main Outcomes and MeasuresBreast, chest wall, and/or shoulder pain 6 months postrandomization measured by the European Organization for Research and Treatment of Cancer QLQ-BR23 questionnaire. Secondary end points were patient-reported fibrosis, edema, movement restriction, and overall quality of life. Data were analyzed according to intention-to-treat (ITT) and complier average causal effect (CACE) principles.ResultsBetween November 2019 and August 2022, 125 women (median [range] age at randomization, 56 [37-85] years) with late local toxic effects were offered to undergo HBOT (intervention arm), and 61 women (median [range] age at randomization, 60 [36-80] years) were randomized to the control arm. Of those offered HBOT, 31 (25%) accepted and completed treatment. The most common reason for not accepting HBOT was high treatment intensity. In ITT, moderate or severe pain at follow-up was reported by 58 of 115 women (50%) in the intervention arm and 32 of 52 women (62%) in the control arm (odds ratio [OR], 0.63; 95% CI, 0.32-1.23; P = .18). In CACE, the proportion of women reporting moderate or severe pain at follow-up was 32% (10 of 31) among those completing HBOT and 75% (9.7 of 12.9) among control participants expected to complete HBOT if offered (adjusted OR, 0.34; 95% CI, 0.15-0.80; P = .01). In ITT, moderate or severe fibrosis was reported by 35 of 107 (33%) in the intervention arm and 25 of 49 (51%) in the control arm (OR, 0.36; 95% CI, 0.15-0.81; P = .02). There were no significant differences in breast edema, movement restriction, and quality of life between groups in ITT and CACE.Conclusions and RelevanceIn this randomized clinical trial, offering HBOT to women with late local toxic effects was not effective for reducing pain, but was effective for reducing fibrosis. In the subgroup of women who completed HBOT, a significant reduction in pain and fibrosis was observed. A smaller than anticipated proportion of women with late local toxic effects was prepared to undergo HBOT.Trial RegistrationClinicalTrials.gov Identifier: NCT04193722

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Most patients reported positively or neutrally of having served as controls in the trials within cohorts design

Cited by 9 publications

References 25 publications

A nationwide randomized controlled trial on additional treatment for isolated local pancreatic cancer recurrence using stereotactic body radiation therapy (ARCADE)

A nationwide randomized controlled trial on additional treatment for isolated local pancreatic cancer recurrence using stereotactic body radiation therapy (ARCADE)

Randomised trials conducted using cohorts: a scoping review

Hyperbaric Oxygen Therapy and Late Local Toxic Effects in Patients With Irradiated Breast Cancer

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