STAR-TREC：早中期直肠癌的应答适应性器官保留

Figure 1: STAR-TREC trial profile Data on allocation of treatment and the first treatment received. Further details of all treatments received by 6 months and salvage surgeries at 12 months are specified in the appendix (p 207). LCCRT-OP=long-course chemoradiotherapy-based organ preservation. NOM=non-operative management. SCRT-OP=short-course radiotherapy-based organ preservation. TME=total mesorectal excision.

Appendix 5: Response-adapted treatment based on a standardised, 2-step (chemo)radiotherapy response assessment The tumour was examined at baseline using endoscopy (A, D). The first response assessment at 11-13 weeks (after starting treatment) classified cases as non-responders (<50% reduction in diameter) for conversion to TME or responders (B) who progressed to a second assessment. The second tumour response assessment at 16- 20 weeks graded responders as clinical complete response (cCR) for ‘watch and wait’ (C), near complete response (nCR) (E), for transanal excision within four weeks (F) or unsatisfactory response for conversion to TME. cCR was defined as a flat white scar, with no evidence of mucosal tumour, mucosal ulceration or submucosal swelling. nCR was defined as a small area of residual mucosal irregularity, or a small flat mucosal ulcer, or an area of submucosal irregularity/ swelling, without evidence of interval tumour progression. Baseline examination and the first response assessment were supplemented by MRI. The second response assessment was endoscopic, and MRI was only performed if progression suspected. To facilitate accurate histopathological evaluation, TAE specimens were pinned out in the operating room prior to fixation (F). 173

Figure 2: Total mesorectal excision-free survival estimates at 12-months HR=hazard ratio. LCCRT-OP=long-course chemoradiotherapy-based organ preservation. SCRT-OP=short-course radiotherapy for organ preservation.

Data are n (%) events. LCCRT-OP=long-course chemoradiotherapy-based organ preservation. OP=organ preservation. SCRT-OP=short-course chemoradiotherapy-based organ preservation. TME=total mesorectal excision. *The number of adverse events is equal to the number of patients in all cases except for grade 3 events in the TME group, in which one patient had 2 grade 3 events. †One fever and sore throat event in the SCRT-OP group was an unclassified event. Table 2: Serious adverse events within 12 months in the modified intention-to-treat population

Figure 3: Patient-reported outcomes Data are mean values with 95% CIs shown in the shaded area. The colorectal core outcome set for the EORTC patient-reported outcomes are shown (A–L); LARS score (M); and EQ-5D visual analogue scale score (N). Where available, dashed horizontal lines represent published population normative values. For panels A–D, and I, these normal values represent the EORTC-QLQ-C30 general population normative data averaged over Denmark, the Netherlands, Sweden, and the UK’s country specific value.28 Values for Belgium were not available. For panel N, the EQ-5D visual analogue scale represents the EQ-5D population forms averaged over Belgium, Denmark, the Netherlands, Sweden, and the UK’s country specific values.29 No normative values are available for either the EORTC QLQ-CR29 (panels E–H, J–L) nor the LARS score (panel M). For panels A–E, G and H, a score of 0 represent very poor functioning in the domain, 30 poor function, 70 moderate function, and 100 very high function. For panels F and I–L, a score of 0 represents no problems in the domain, 30 mild problems, 70 moderate problems, and 100 severe problems. For panel M, 0–20 indicates no LARS, 21–29 minor LARS, and 30–42 indicates major LARS. For panel N, 0 indicates the best health state and 100 the best health state. LCCRT-OP=long-course chemoradiotherapy-based organ preservation. EORTC QLQ-CR29=European Organisation for Research and Treatment of Cancer Quality of Life Core 29-item questionnaire. EORTC QLQ-CR30=European Organisation for Research and Treatment of Cancer Quality of Life Core 30-item questionnaire. LARS=Low Anterior Resection Syndrome. SCRT-OP=short-course radiotherapy for organ preservation.

Figure 3: Patient-reported outcomes Data are mean values with 95% CIs shown in the shaded area. The colorectal core outcome set for the EORTC patient-reported outcomes are shown (A–L); LARS score (M); and EQ-5D visual analogue scale score (N). Where available, dashed horizontal lines represent published population normative values. For panels A–D, and I, these normal values represent the EORTC-QLQ-C30 general population normative data averaged over Denmark, the Netherlands, Sweden, and the UK’s country specific value.28 Values for Belgium were not available. For panel N, the EQ-5D visual analogue scale represents the EQ-5D population forms averaged over Belgium, Denmark, the Netherlands, Sweden, and the UK’s country specific values.29 No normative values are available for either the EORTC QLQ-CR29 (panels E–H, J–L) nor the LARS score (panel M). For panels A–E, G and H, a score of 0 represent very poor functioning in the domain, 30 poor function, 70 moderate function, and 100 very high function. For panels F and I–L, a score of 0 represents no problems in the domain, 30 mild problems, 70 moderate problems, and 100 severe problems. For panel M, 0–20 indicates no LARS, 21–29 minor LARS, and 30–42 indicates major LARS. For panel N, 0 indicates the best health state and 100 the best health state. LCCRT-OP=long-course chemoradiotherapy-based organ preservation. EORTC QLQ-CR29=European Organisation for Research and Treatment of Cancer Quality of Life Core 29-item questionnaire. EORTC QLQ-CR30=European Organisation for Research and Treatment of Cancer Quality of Life Core 30-item questionnaire. LARS=Low Anterior Resection Syndrome. SCRT-OP=short-course radiotherapy for organ preservation.