TY - JOUR
T1 - Long-term Oncological and Functional Outcomes of Chemoradiotherapy Followed by Organ-Sparing Transanal Endoscopic Microsurgery for Distal Rectal Cancer
T2 - The CARTS Study
AU - Stijns, Rutger C. H.
AU - de Graaf, Eelco J. R.
AU - Punt, Cornelis J. A.
AU - Nagtegaal, Iris D.
AU - Nuyttens, Joost J. M. E.
AU - van Meerten, Esther
AU - Tanis, Pieter J.
AU - de Hingh, Ignace H. J. T.
AU - van der Schelling, George P.
AU - Acherman, Yair
AU - Leijtens, Jeroen W. A.
AU - Bremers, Andreas J. A.
AU - Beets, Geerard L.
AU - Hoff, Christiaan
AU - Verhoef, Cornelis
AU - Marijnen, Corrie A. M.
AU - de Wilt, Johannes H. W.
AU - Bokkerink, Guus M. J.
AU - Cats, Annemieke
AU - Doornebosch, Pascal G.
AU - Dwarkasing, Roy S.
AU - Rutten, Heidi
AU - ten Tije, Albert J.
AU - Tromp, Mike Stephen
AU - Schoon, Erik
AU - Verseveld, Mareille
AU - Buijsen, Jeroen
AU - CARTS Study Group
N1 - Publisher Copyright:
© 2018 American Medical Association. All rights reserved.
PY - 2019/1
Y1 - 2019/1
N2 - IMPORTANCE Treatment of rectal cancer is shifting toward organ preservation aiming to reduce surgery-related morbidity. Short-term outcomes of organ-preserving strategies are promising, but long-term outcomes are scarce in the literature.OBJECTIVE To explore long-term oncological outcomes and health-related quality of life (HRQL) in patients with cT1-3NOMO rectal cancer who underwent neoadjuvant chemoradiotherapy (CRT) followed by transanal endoscopic microsurgery (TEM).DESIGN, SETTING, AND PARTICIPANTS In this multicenter phase II feasibility study, patients with cT1-3NOMO rectal cancer admitted to referral centers for rectal cancer throughout the Netherlands between February 2011 and September 2012 were prospectively included. These patients were to be treated with neoadjuvant CRT followed by TEM in case of good response. An intensive follow-up scheme was used to detect local recurrences and/or distant metastases. Data from validated HRQL questionnaires and low anterior resection syndrome questionnaires were collected. Data were analyzed from February 2011 to April 2017.MAIN OUTCOMES AND MEASURES The primary study outcome of the studywas the number of ypT0-1 specimens by performing TEM. Secondary outcome parameters were locoregional recurrences and HRQL.RESULTS Of the 55 included patients, 30 (55%) were male, and the mean (SD) age was 64 (39-82) years. Patients were followed up for a median (interquartile range) period of 53 (39-57) months. Two patients (4%) died during CRT, 1 (2%) stopped CRT, and 1 (2%) was lost to follow-up. Following CRT, 47 patients (85%) underwent TEM, of whom 35 (74%) were successfully treated with local excision alone. Total mesorectal excision was performed in 16 patients (4 with inadequate responses, 8 with completion after TEM, and 4 with salvage for local recurrence). The actuarial 5-year local recurrence rate was 7.7%, with 5-year disease-free and overall survival rates of 81.6% and 82.8%, respectively. Health-related quality of life during follow-up was equal to baseline, with improved emotional well-being in patients treated with local excision (mean score at baseline, 72.0; 95% CI, 67.1-80.1; mean score at follow-up, 86.9; 95% CI, 79.2-94.7; P =.001). Major, minor, and no low anterior resection syndrome was experienced in 50%, 28%, and 22%, respectively, of patients with successful organ preservation.CONCLUSIONS AND RELEVANCE In early-stage rectal cancer (cT1-3N0M0), CRT enables organ preservation with additional TEM surgery in approximately two-thirds of patients with good long-term oncological outcome and HRQL. This multimodality treatment triggers a certain degree of bowel dysfunction, and one-third of patients still undergo radical surgery and are overtreated by CRT.
AB - IMPORTANCE Treatment of rectal cancer is shifting toward organ preservation aiming to reduce surgery-related morbidity. Short-term outcomes of organ-preserving strategies are promising, but long-term outcomes are scarce in the literature.OBJECTIVE To explore long-term oncological outcomes and health-related quality of life (HRQL) in patients with cT1-3NOMO rectal cancer who underwent neoadjuvant chemoradiotherapy (CRT) followed by transanal endoscopic microsurgery (TEM).DESIGN, SETTING, AND PARTICIPANTS In this multicenter phase II feasibility study, patients with cT1-3NOMO rectal cancer admitted to referral centers for rectal cancer throughout the Netherlands between February 2011 and September 2012 were prospectively included. These patients were to be treated with neoadjuvant CRT followed by TEM in case of good response. An intensive follow-up scheme was used to detect local recurrences and/or distant metastases. Data from validated HRQL questionnaires and low anterior resection syndrome questionnaires were collected. Data were analyzed from February 2011 to April 2017.MAIN OUTCOMES AND MEASURES The primary study outcome of the studywas the number of ypT0-1 specimens by performing TEM. Secondary outcome parameters were locoregional recurrences and HRQL.RESULTS Of the 55 included patients, 30 (55%) were male, and the mean (SD) age was 64 (39-82) years. Patients were followed up for a median (interquartile range) period of 53 (39-57) months. Two patients (4%) died during CRT, 1 (2%) stopped CRT, and 1 (2%) was lost to follow-up. Following CRT, 47 patients (85%) underwent TEM, of whom 35 (74%) were successfully treated with local excision alone. Total mesorectal excision was performed in 16 patients (4 with inadequate responses, 8 with completion after TEM, and 4 with salvage for local recurrence). The actuarial 5-year local recurrence rate was 7.7%, with 5-year disease-free and overall survival rates of 81.6% and 82.8%, respectively. Health-related quality of life during follow-up was equal to baseline, with improved emotional well-being in patients treated with local excision (mean score at baseline, 72.0; 95% CI, 67.1-80.1; mean score at follow-up, 86.9; 95% CI, 79.2-94.7; P =.001). Major, minor, and no low anterior resection syndrome was experienced in 50%, 28%, and 22%, respectively, of patients with successful organ preservation.CONCLUSIONS AND RELEVANCE In early-stage rectal cancer (cT1-3N0M0), CRT enables organ preservation with additional TEM surgery in approximately two-thirds of patients with good long-term oncological outcome and HRQL. This multimodality treatment triggers a certain degree of bowel dysfunction, and one-third of patients still undergo radical surgery and are overtreated by CRT.
KW - QUALITY-OF-LIFE
KW - TOTAL MESORECTAL EXCISION
KW - ANTERIOR RESECTION SYNDROME
KW - LOCAL EXCISION
KW - POSTOPERATIVE CHEMORADIOTHERAPY
KW - NEOADJUVANT CHEMORADIATION
KW - COLORECTAL-CANCER
KW - CLINICAL-TRIALS
KW - OPEN-LABEL
KW - MULTICENTER
U2 - 10.1001/jamasurg.2018.3752
DO - 10.1001/jamasurg.2018.3752
M3 - Article
C2 - 30304338
SN - 2168-6254
VL - 154
SP - 47
EP - 54
JO - JAMA Surgery
JF - JAMA Surgery
IS - 1
ER -