HTA - 10/34/01: OPTIMA prelim: a randomised feasibility study of personalised care in the treatment of women with early breast cancer
|Project title||OPTIMA prelim: a randomised feasibility study of personalised care in the treatment of women with early breast cancer|
|Research type||Primary Research|
|Start date||May 2012|
|Link to journal publication|
|Chief Investigator||Dr Robert Stein|
|Co-investigators||Professor Janet Dunn (University of Warwick), Professor John Bartlett (Ontario Insitute for Cancer Research), Professor David Cameron (The University of Edinburgh), Dr Peter Canney, Professor Jenny Donovan (University of Bristol), Dr Helena Earl (University of Cambridge), Dr Adele Francis (University Hospitals Birmingham NHS Foundation Trust), Dr Peter Hall (University of Edinburgh), Dr Louise Hiller (University of Warwick), Dr Luke Hughes-Davies (Cambridge University Hospitals NHS Foundation Trust), Professor Christopher McCabe (University of Alberta), Dr Adrienne Morgan (Independent Cancer Patients' Voice), Professor Sarah Pinder (King's College London), Professor Christopher Poole (University Hospitals Coventry & Warwickshire NHS Trust), Dr Elena Provenzano (Cambridge University Hospital NHS Trust), Dr Daniel Rea (University of Birmingham), Professor Nigel Stallard (University of Warwick), Dr Andreas Makris (Mount Vernon Hospital)|
|Contractor||University College London|
|Plain English summary||Women with primary breast cancer are commonly treated with chemotherapy and with endocrine therapy in addition to surgery to reduce the risk of disease recurrence. Chemotherapy is usually reserved for patients at higher risk. It is clear however that many patients don t benefit from chemotherapy despite its side effects and costs. The OPTIMA trial will evaluate multi-parameter pathology tests that could offer improved identification of patients who might benefit from chemotherapy. Specifically it will examine whether these tests provide equivalent outcomes for patients, but with lower use of chemotherapy and lower total costs of care including the costs of the tests. OPTIMA prelim, the feasibility study for the main trial, will recruit 300 surgically-treated patients from about 25 UK hospitals. Patients will have hormone receptor positive HER2-negative tumours, which is the group where there is greatest doubt about the value of chemotherapy, and will have risk factors such as axillary node involvement. In the NHS they would routinely be offered chemotherapy followed by 5 years of endocrine therapy. Patients will be randomised to standard chemotherapy or to have an Oncotype DX test performed on tumour tissue with treatment (the same chemotherapy or not) assigned according to the result of the test. Oncotype DX is the best validated multi-parameter test. OPTIMA prelim has 3 objectives. (1) To establish whether randomisation to test-directed treatment is acceptable to patients and clinicians. Attitudes of patients and research staff will be studied using qualitative research techniques. (2) Alternative multi-parameter tests will be evaluated on all patients tumours and their performance compared to Oncotype DX to enable the best test(s) appropriate for NHS use to be evaluated in the main trial. (3) Obstacles to efficient and timely tumour sample collection and analysis will be analysed in detail with the aid of a sample tracking database. The results of OPTIMA prelim will influence aspects of the main study design such as choice of test but the 2 phases of the study will have the same basic structure.|
|Scientific summary||"Multi-parameter" prognostic tests such as Oncotype DX are increasingly used to identify women with ER +ve HER2 -ve early breast cancer treated with endocrine therapy who are unlikely to benefit meaningfully from chemotherapy. The supporting evidence for predictive testing is retrospective and is strongest for women without nodal involvement. More evidence is needed, especially for patients with node positive disease. There is early evidence that other multi-parameter tests in development also have a predictive utility. OPTIMA will assess the value of multi-parameter tests in the NHS. OPTIMA prelim, the feasibility phase, will recruit 300 patients (with a 200 patient bridging extension to the main study). Eligible patients will have ER +ve HER2 -ve tumours with involved nodes (pN1-2). Patients will be randomised to standard treatment with chemotherapy or to have "test-directed treatment" with either the same chemotherapy or no chemotherapy, assigned according to the result of an Oncotype DX test. All patients will receive endocrine therapy. OPTIMA prelim has 3 objectives. (1) To establish whether randomisation to test-directed treatment is acceptable to patients and clinicians. This will be done through qualitative research by recording and analysing consultations when the trial is discussed with potential participants and with in-depth interviews with OPTIMA researchers and participants. (2) Alternative multi-parameter tests will be evaluated on all patients tumours and their performance will be compared to Oncotype DX using statistical and cost-effectiveness analysis to select a candidate test(s) appropriate for NHS use to be evaluated in the main trial. (3) The success of the main trial depends on efficient tumour sample collection and analysis to avoid treatment delays. The obstacles to this will be analysed in detail using a sample tracking database. The main study is an efficacy trial of 2-3 arms. It will compare 5-year relapse free survival of test-directed vs. conventional treatment with a non-inferiority hypothesis.|
Protocol (PDF File - 1.0 MB)
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