A Vasista1, M R Stockler2, T West3, N Wilcken4, B E Kiely5. 1. NHMRC Clinical Trials Centre, University of Sydney, NSW, Australia; Department of Medical Oncology, Westmead Hospital, Sydney, NSW, Australia. 2. NHMRC Clinical Trials Centre, University of Sydney, NSW, Australia; Sydney Medical School, University of Sydney, NSW, Australia. 3. Sydney Medical School, University of Sydney, NSW, Australia. 4. Sydney Medical School, University of Sydney, NSW, Australia; Department of Medical Oncology, Westmead Hospital, Sydney, NSW, Australia. 5. NHMRC Clinical Trials Centre, University of Sydney, NSW, Australia. Electronic address: belinda.kiely@ctc.usyd.edu.au.
Abstract
OBJECTIVES: To estimate worst-case, typical and best-case scenarios for survival as a communication aid for managing patients with HER2-positive metastatic breast cancer (MBC) starting HER2-targeted therapies. METHODS: We sought randomised trials of HER2-targeted therapies and recorded the following percentiles (representative scenarios) from each OS curve: 90th (worst-case), 75th (lower-typical), 50th (median), 25th (upper-typical) and 10th (best-case). We then tested whether we could estimate these percentiles for each OS curve by multiplying its median by four simple multiples: 0.25 (to derive the 90th percentile), 0.5 (75th), 2 (25th) and 3 (10th). Estimates were deemed accurate if within 0.75-1.33 times the actual value. RESULTS: We identified 15 trials with 4798 patients. For first-line, single-agent HER2-targeted therapy (15 treatment groups), the median (interquartile range [IQR]) for median OS was 33.3 months (29.1-38.4), and for each percentile was: 90th 9.5 months (7.7-11.0); 75th 19.2 months (16.4-20.8); and 25th 50.6 months (47.1-63.3). The 10th percentile was unavailable for all treatment groups. For first-line dual HER2-targeted therapy (1 treatment group), the median OS was 56.5 months. Simple multiples of the median OS accurately estimated the: 90th percentile in 79%; 75th percentile in 100%; and 25th percentile in 89% of OS curves. CONCLUSIONS: Surprisingly little is known of survival beyond the median for HER2-positive MBC. Longer trial follow-up is required to help clinicians estimate and explain the best-case scenario. Simple multiples of the median OS provide a reasonable framework for estimating then explaining survival times to patients.
OBJECTIVES: To estimate worst-case, typical and best-case scenarios for survival as a communication aid for managing patients with HER2-positive metastatic breast cancer (MBC) starting HER2-targeted therapies. METHODS: We sought randomised trials of HER2-targeted therapies and recorded the following percentiles (representative scenarios) from each OS curve: 90th (worst-case), 75th (lower-typical), 50th (median), 25th (upper-typical) and 10th (best-case). We then tested whether we could estimate these percentiles for each OS curve by multiplying its median by four simple multiples: 0.25 (to derive the 90th percentile), 0.5 (75th), 2 (25th) and 3 (10th). Estimates were deemed accurate if within 0.75-1.33 times the actual value. RESULTS: We identified 15 trials with 4798 patients. For first-line, single-agent HER2-targeted therapy (15 treatment groups), the median (interquartile range [IQR]) for median OS was 33.3 months (29.1-38.4), and for each percentile was: 90th 9.5 months (7.7-11.0); 75th 19.2 months (16.4-20.8); and 25th 50.6 months (47.1-63.3). The 10th percentile was unavailable for all treatment groups. For first-line dual HER2-targeted therapy (1 treatment group), the median OS was 56.5 months. Simple multiples of the median OS accurately estimated the: 90th percentile in 79%; 75th percentile in 100%; and 25th percentile in 89% of OS curves. CONCLUSIONS: Surprisingly little is known of survival beyond the median for HER2-positive MBC. Longer trial follow-up is required to help clinicians estimate and explain the best-case scenario. Simple multiples of the median OS provide a reasonable framework for estimating then explaining survival times to patients.
Authors: Connor J Kinslow; Andrew L A Garton; Ali I Rae; Logan P Marcus; Christopher M Adams; Guy M McKhann; Michael B Sisti; E Sander Connolly; Jeffrey N Bruce; Alfred I Neugut; Adam M Sonabend; Peter Canoll; Simon K Cheng; Tony J C Wang Journal: J Neurooncol Date: 2019-08-12 Impact factor: 4.130
Authors: Sharon H Nahm; Martin R Stockler; Andrew J Martin; Peter Grimison; Peter Fox; Rob Zielinski; Geoffrey At Hawson; Martin Hn Tattersall; Belinda E Kiely Journal: Support Care Cancer Date: 2022-06-15 Impact factor: 3.359