Parikh Ravi B, Ferrell William J, Girard Anthony, White Jenna, Fang Sophia, Bekelman Justin E, Schapira Marilyn M
Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Department of Medical Ethics and Health Policy, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Med Decis Making. 2025 Jul 4:272989X251349489. doi: 10.1177/0272989X251349489.
BackgroundMachine learning (ML) algorithms may improve the prognosis for serious illnesses such as cancer, identifying patients who may benefit from earlier palliative care (PC) or advance care planning (ACP). We evaluated the impact of various presentation strategies of a hypothetical ML algorithm on clinician prognostic accuracy and decision making.MethodsThis was a randomized clinical vignette survey study among medical oncologists who treat metastatic non-small-cell lung cancer (mNSCLC). Between March and June 2023, clinicians were shown 3 vignettes of patients presenting with mNSCLC. The vignettes varied by prognostic risk, as defined from the Lung Cancer Prognostic Index (LCPI). Clinicians estimated life expectancy in months and made recommendations about PC and ACP. Clinicians were then shown the same vignette with a hypothetical survival estimate from a black-box ML algorithm; clinicians were randomized to receive the ML prediction using absolute and/or reference-dependent prognostic estimates. The primary outcome was prognostic accuracy relative to the LCPI.ResultsAmong 51 clinicians with complete responses, the median years in practice was 7 (interquartile range 3.5-19), 14 (27.5%) were female, 23 (45.1%) practiced in a community oncology setting, and baseline accuracy was 54.9% (95% confidence interval [CI] 47.0-62.8) across all vignettes. ML presentation improved accuracy (mean change relative to baseline 20.9%, 95% CI 13.9-27.9, < 0.001). ML outputs using an absolute presentation strategy alone (mean change 27.4%, 95% 16.8-38.1, < 0.001) or with reference dependence (mean change 33.4%, 95% 23.9-42.8, < 0.001) improved accuracy, but reference dependence alone did not (mean change 2.0% [95% CI -11.1 to 15.0], = 0.77). ML presentation did not change the rates of recommending ACP nor PC referral (mean change 1.3% and 0.7%, respectively).LimitationsThe singular use case of prognosis in mNSCLC, low initial response rate.ConclusionsML-based assessments may improve prognostic accuracy but not result in changed decision making.ImplicationsML prognostic algorithms prioritizing explainability and absolute prognoses may have greater impact on clinician decision making.Trial Registration: CT.gov: NCT06463977HighlightsWhile machine learning (ML) algorithms may accurately predict mortality, the impact of prognostic ML on clinicians' prognostic accuracy and decision making and optimal presentation strategies for ML outputs are unclear.In this multicenter randomized survey study among vignettes of patients with advanced cancer, prognostic accuracy improved by 20.9% when clinicians reviewed vignettes with a hypothetical ML mortality risk prediction, with absolute risk presentation strategies resulting in greater accuracy gains than reference-dependent presentations alone.However, ML presentation did not change the rates of recommending advance care planning or palliative care referral (1.3% and 0.7%, respectively).ML-based prognostic assessments without explanations improve prognostic accuracy but do not change decisions around palliative care referral or advance care planning.
背景
机器学习(ML)算法可能会改善癌症等严重疾病的预后,识别出可能从早期姑息治疗(PC)或预立医疗计划(ACP)中获益的患者。我们评估了一种假设的ML算法的各种呈现策略对临床医生预后准确性和决策的影响。
方法
这是一项针对治疗转移性非小细胞肺癌(mNSCLC)的肿瘤内科医生的随机临床病例调查研究。在2023年3月至6月期间,向临床医生展示了3例mNSCLC患者的病例。这些病例根据肺癌预后指数(LCPI)定义的预后风险而有所不同。临床医生估计患者的预期寿命(以月为单位),并就PC和ACP提出建议。然后,向临床医生展示相同的病例,并给出一种假设的黑箱ML算法的生存估计;临床医生被随机分配接受使用绝对和/或参考依赖预后估计的ML预测。主要结局是相对于LCPI的预后准确性。
结果
在51名给出完整回复的临床医生中,从业年限的中位数为7年(四分位间距3.5 - 19年),14名(27.5%)为女性,23名(45.1%)在社区肿瘤环境中执业,所有病例的基线准确性为54.9%(95%置信区间[CI] 47.0 - 62.8)。ML呈现提高了准确性(相对于基线的平均变化为20.9%,95% CI 13.9 - 27.9,P < 0.001)。仅使用绝对呈现策略的ML输出(平均变化27.4%,95% CI 16.8 - 38.1,P < 0.001)或结合参考依赖的ML输出(平均变化33.4%,95% CI 23.9 - 42.8,P < 0.001)提高了准确性,但仅参考依赖策略未提高准确性(平均变化2.0% [95% CI -11.1至15.0],P = 0.77)。ML呈现并未改变推荐ACP或PC转诊的比例(平均变化分别为1.3%和0.7%)。
局限性
mNSCLC预后的单一用例,初始回复率较低。
结论
基于ML的评估可能会提高预后准确性,但不会导致决策改变。
启示
优先考虑可解释性和绝对预后的ML预后算法可能对临床医生的决策有更大影响。
CT.gov:NCT06463977
要点
虽然机器学习(ML)算法可能准确预测死亡率,但预后性ML对临床医生预后准确性和决策的影响以及ML输出的最佳呈现策略尚不清楚。
在这项针对晚期癌症患者病例的多中心随机调查研究中,当临床医生查看带有假设ML死亡风险预测的病例时,预后准确性提高了20.9%,绝对风险呈现策略比仅参考依赖呈现带来的准确性提升更大。
然而,ML呈现并未改变推荐预立医疗计划或姑息治疗转诊的比例(分别为1.3%和0.7%)。
无解释的基于ML的预后评估提高了预后准确性,但未改变围绕姑息治疗转诊或预立医疗计划的决策。