Adjusting for covariates in evaluating markers for selecting treatment, with application to guiding chemotherapy for treating estrogen-receptor-positive, node-positive breast cancer

Janes, Holly; Brown, Marshall; Crager, Michael; Miller, Dave P.; Barlow, William E.

doi:10.1016/j.cct.2017.08.004

Cited by 6 publications

(5 citation statements)

References 46 publications

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“…We found that the RS category was independently associated with chemotherapy recommendation in pN1 patients in our study, which was in consistency with previous findings 22–24 . For low RS pN1 patients, 40.68% (24/59) were exempt from chemotherapy.…”

Section: Discussionsupporting

confidence: 93%

21-Gene Recurrence Score and Adjuvant Chemotherapy Decision for Breast Cancer Patients with Positive Lymph Nodes

Tong

Huang

et al. 2019

Sci Rep

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The 21-gene recurrence score (RS) assay is prognostic and predictive for hormone receptor (HR)+/HER2-/node- breast cancer (BC) patients. However, its clinical value in node + patients hasn’t been elucidated. HR+/HER2-/pN1 patients operated in Comprehensive Breast Health Center, Shanghai Ruijin Hospital from January 2014 to December 2018, with available RS results were retrospectively included. Clinico-pathological characteristics were compared. Adjuvant chemotherapy recommendations pre-/post- RS assay and actual usage were analyzed. A total of 303 patients were included, with 59, 178, 66 RS < 18, 18–30 and ≥ 31. Age (P < 0.001), comorbidity (P = 0.013), and RS category (P < 0.001) were independently associated with chemotherapy recommendation. Compared with low RS patients, those with intermediate (OR 6.58, 95% CI 2.37–18.31, P < 0.001) or high (OR 54.14, 95% CI 3.77–776.54, P = 0.003) RS were more likely to be recommended with chemotherapy. RS independently influence chemotherapy decision in postmenopausal population as well. Chemotherapy recommendation changed for 9.57% patients after RS assay. Patient adherence rate to chemotherapy recommendation was 94.72% (287/303). The 21-gene RS independently influenced chemotherapy recommendation in pN1 BC patients, which could provide additional information to guide chemotherapy decision with relatively good treatment adherence rate.

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Section: Discussionsupporting

confidence: 93%

21-Gene Recurrence Score and Adjuvant Chemotherapy Decision for Breast Cancer Patients with Positive Lymph Nodes

Tong

Huang

et al. 2019

Sci Rep

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“…It is common in clinical trials that only a subgroup of treated patients may benefit from an experimental therapy [ 54 – 57 ]. Identifying these subgroups would allow tailoring of treatment, avoiding costly or toxic treatment of individuals who will not benefit.…”

Section: Overview Of Innovative Methods For Immune-mediated Inflammatory Disease Trialsmentioning

confidence: 99%

Innovative trial approaches in immune-mediated inflammatory diseases: current use and future potential

et al. 2021

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Background Despite progress that has been made in the treatment of many immune-mediated inflammatory diseases (IMIDs), there remains a need for improved treatments. Randomised controlled trials (RCTs) provide the highest form of evidence on the effectiveness of a potential new treatment regimen, but they are extremely expensive and time consuming to conduct. Consequently, much focus has been given in recent years to innovative design and analysis methods that could improve the efficiency of RCTs. In this article, we review the current use and future potential of these methods within the context of IMID trials. Methods We provide a review of several innovative methods that would provide utility in IMID research. These include novel study designs (adaptive trials, Sequential Multi-Assignment Randomised Trials, basket, and umbrella trials) and data analysis methodologies (augmented analyses of composite responder endpoints, using high-dimensional biomarker information to stratify patients, and emulation of RCTs from routinely collected data). IMID trials are now well-placed to embrace innovative methods. For example, well-developed statistical frameworks for adaptive trial design are ready for implementation, whilst the growing availability of historical datasets makes the use of Bayesian methods particularly applicable. To assess whether and how these innovative methods have been used in practice, we conducted a review via PubMed of clinical trials pertaining to any of 51 IMIDs that were published between 2018 and 20 in five high impact factor clinical journals. Results Amongst 97 articles included in the review, 19 (19.6%) used an innovative design method, but most of these were relatively straightforward examples of innovative approaches. Only two (2.1%) reported the use of evidence from routinely collected data, cohorts, or biobanks. Eight (9.2%) collected high-dimensional data. Conclusions Application of innovative statistical methodology to IMID trials has the potential to greatly improve efficiency, to generalise and extrapolate trial results, and to further personalise treatment strategies. Currently, such methods are infrequently utilised in practice. New research is required to ensure that IMID trials can benefit from the most suitable methods.

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“…It is common in clinical trials that only a subgroup of treated patients may bene t from an experimental therapy (52)(53)(54)(55). Identifying these subgroups would allow tailoring of treatment, avoiding costly or toxic treatment of individuals who will not bene t. To identify such subgroups, predictive biomarkers are required.…”

Section: Use Of High-dimensional Data To Stratify Patients: Adaptive mentioning

confidence: 99%

“…What was the length of patient recruitment (in weeks)? 2 Median: 96 IQR: [55,120] Range: [16,296] What was the primary endpoint timepoint (in weeks)? 1 One article used a sequential multiple assignment randomised trial design with an interim futility assessment.…”

Section: Biomarker Adaptivementioning

confidence: 99%

Innovative Trial Approaches in Immune-Mediated Inflammatory Diseases: Current Use and Future Potential

Grayling

Bigirumurame

Cherlin

et al. 2020

Preprint

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Background: Despite progress that has been made in the treatment of many immune-mediated inflammatory diseases (IMIDs), there remains a need for improved treatments. Randomised controlled trials (RCTs) provide the highest form of evidence on the effectiveness of a potential new treatment regimen, but they are extremely expensive and time consuming to conduct. Consequently, much focus has been given in recent years to innovative design and analysis methods that could improve the efficiency of RCTs. In this article, we review the current use and future potential of these methods within the context of IMID trials.Methods: We provide a review of several innovative methods that would provide utility in IMID research. These include novel study designs (adaptive trials, Sequential Multi-Assignment Randomised Trials, basket, and umbrella trials) and data analysis methodologies (augmented analyses of composite responder endpoints, using high-dimensional biomarker information to stratify patients, and emulation of RCTs from routinely collected data). IMID trials are now well-placed to embrace innovative methods. For example, well-developed statistical frameworks for adaptive trial design are ready for implementation, whilst the growing availability of historical datasets makes the use of Bayesian methods particularly applicable.To assess whether and how these innovative methods have been used in practice, we conducted a review via PubMed of clinical trials pertaining to any of 51 IMIDs that were published between 2018-20 in five high impact factor clinical journals. Results: Amongst 97 articles included in the review, 19 (19.6%) used an innovative design method, but most of these were relatively straightforward examples of innovative approaches. Only two (2.1%) reported the use of evidence from routinely collected data, cohorts, or biobanks. Eight (9.2%) collected high-dimensional data. Conclusions: Application of innovative statistical methodology to IMID trials has the potential to greatly improve efficiency, to generalise and extrapolate trial results, and to further personalise treatment strategies. Currently, such methods are infrequently utilised in practice. New research is required to ensure that IMID trials can benefit from the most suitable methods.

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Adjusting for covariates in evaluating markers for selecting treatment, with application to guiding chemotherapy for treating estrogen-receptor-positive, node-positive breast cancer

Cited by 6 publications

References 46 publications

21-Gene Recurrence Score and Adjuvant Chemotherapy Decision for Breast Cancer Patients with Positive Lymph Nodes

21-Gene Recurrence Score and Adjuvant Chemotherapy Decision for Breast Cancer Patients with Positive Lymph Nodes

Innovative trial approaches in immune-mediated inflammatory diseases: current use and future potential

Innovative Trial Approaches in Immune-Mediated Inflammatory Diseases: Current Use and Future Potential

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