Khanna Reena, Son Surim, Zou Guangyong, Roshanov Pavel S
Department of Medicine, Division of Gastroenterology, Western University, London, Ontario, Canada.
Department of Epidemiology and Biostatistics, Western University, London, Ontario, Canada.
Inflamm Bowel Dis. 2025 Aug 1;31(8):2097-2105. doi: 10.1093/ibd/izae281.
Many registrational trials in Crohn's disease assess treatment efficacy with the 2-item Patient-Reported Outcome (PRO2), while the Harvey-Bradshaw Index (HBI) is prominent in pragmatic trials and clinical practice. The translation between PRO2 and HBI has not been established.
Data from a Phase 3 trial of vedolizumab in Crohn's disease were used to determine the Pearson correlation between PRO2 and HBI. Linear regression was used to fit equations that estimate between indices; 95% prediction intervals were determined for HBI scores corresponding to PRO2 thresholds for disease activity. Internal validation of the conversion equations was performed using the bootstrap methods.
PRO2 and HBI were highly correlated at baseline (r = 0.75 95% confidence interval (CI) 0.73-0.78; P < .001), induction (r = 0.87; 95% CI, 0.85-0.88; P < .001), and maintenance (r = 0.88; 95% CI, 0.85-0.90; P < .001). PRO2 and HBI change scores were moderately correlated (r = 0.72; 95% CI 0.69-0.75; P < .001) in induction and more strongly correlated during maintenance (r = 0.81; 95% CI 0.78- 0.84; P < .001). Regression equations for conversion of PRO2 to HBI from all cohorts (induction, maintenance, randomized, open-label) support an approximate conversion where HBI = 0.5 PRO2. As expected from the imperfect correlation between scores, the prediction intervals were generally wide. No evidence of overfitting was seen in bootstrap internal validation.
PRO2 and HBI correlate strongly and conversion between them is possible. These findings facilitate the practical application of trial results and clinical guidelines.
许多克罗恩病注册试验使用2项患者报告结局（PRO2）评估治疗效果，而哈维-布拉德肖指数（HBI）在务实试验和临床实践中较为突出。PRO2与HBI之间的转换关系尚未确立。
使用维多珠单抗治疗克罗恩病3期试验的数据来确定PRO2与HBI之间的皮尔逊相关性。采用线性回归拟合估计两个指标之间关系的方程；针对疾病活动的PRO2阈值对应的HBI分数确定95%预测区间。使用自助法对转换方程进行内部验证。
在基线期（r = 0.75，95%置信区间（CI）0.73 - 0.78；P <.001）、诱导期（r = 0.87；95% CI，0.85 - 0.88；P <.001）和维持期（r = 0.88；95% CI，0.85 - 0.90；P <.001），PRO2与HBI高度相关。在诱导期，PRO2和HBI变化分数中度相关（r = 0.72；95% CI 0.69 - 0.75；P <.001），在维持期相关性更强（r = 0.81；95% CI 0.78 - 0.84；P <.001）。所有队列（诱导期、维持期、随机分组、开放标签）中PRO2转换为HBI的回归方程支持近似转换，即HBI = 0.5 PRO2。正如分数之间不完全相关所预期的那样，预测区间通常较宽。自助法内部验证未发现过拟合证据。
PRO2与HBI密切相关，二者之间可以进行转换。这些发现有助于试验结果和临床指南的实际应用。
