Shinji Ohno1, Hirofumi Mukai2, Kazutaka Narui3, Yasuo Hozumi4,5, Yasuo Miyoshi6, Hiroshi Yoshino7, Hiroyoshi Doihara8, Akihiko Suto9, Motoshi Tamura10, Takashi Morimoto11, Hisamitsu Zaha12, Takashi Chishima13, Reiki Nishimura14, Takashi Ishikawa15, Yukari Uemura16, Yasuo Ohashi17. 1. Breast Oncology Center, Cancer Institute Hospital, Tokyo, Japan. 2. Division of Breast and Medical Oncology, National Cancer Center Hospital East, 6-5-1, Kashiwanoha, Kashiwa, Japan. hrmukai@east.ncc.go.jp. 3. Breast and Thyroid Surgery, Yokohama City University Medical Center, Yokohama, Japan. 4. Department of Breast and Endocrine Surgery, University of Tsukuba Hospital, Tsukuba, Japan. 5. Department of Breast Surgery, Ibaraki Prefectural Central Hospital, Tsukuba, Japan. 6. Department of Breast and Endocrine Surgery, Hyogo College of Medicine, Nishinomiya, Japan. 7. Gastrointestinal Surgery and Breast and Endocrinological Surgery, Ishikawa Prefectural Central Hospital, Kanazawa, Japan. 8. Breast and Endocrine Surgery, Okayama University Hospital, Okayama, Japan. 9. Department of Breast Surgery, Yamato Municipal Hospital, Yamato, Japan. 10. Department of Surgery and Pathology, KKR Sapporo Medical Center, Sapporo, Japan. 11. Department of Breast Surgery, Yao Municipal Hospital, Yao, Japan. 12. Department of Breast Surgery, Nakagami Hospital, Okinawa, Japan. 13. Department of Breast Surgery, Yokohama Rosai Hospital, Yokohama, Japan. 14. Department of Breast Oncology, Kumamoto Shinto General Hospital, Kumamoto, Japan. 15. Department of Breast Surgery, Tokyo Medical University, Tokyo, Japan. 16. Biostatistics Division, Clinical Research Support Center, The University of Tokyo Hospital, Tokyo, Japan. 17. Department of Integrated Science and Engineering, Chuo University, Tokyo, Japan.
Abstract
PURPOSE: While some studies show improved outcomes in clinical trial participants as compared to non-participants, existence of such a trial effect has not been proved precisely. METHODS: This was a prospective cohort study to compare the prognoses for participants in the randomized controlled trial (SELECT BC) and non-participants. SELECT BC compared S-1 and taxane as first-line treatment for metastatic breast cancer. Non-participants were all patients who met the eligibility criteria of SELECT BC and who had been requested to participate in that trial by attending doctors and declined. The study aimed to compare the prognoses between participants and non-participants. The primary endpoint was median overall survival. RESULTS: The median OS in participants was significantly superior to that in non-participants with a statistically significant difference (36.8 months vs. 25.2 months. HR 1.48, p = 0.022). A similar result was obtained when only patients who received the same chemotherapy (S-1 or taxane) used in SELECT BC after declining participation were assumed as non-participants (36.8 months vs. 22.0 months. HR 2.03, p = 0.006). CONCLUSIONS: This study may suggest the existence of a trial effect, in which, for a given treatment, participation in a clinical trial is associated with a better outcome.
RCT Entities:
PURPOSE: While some studies show improved outcomes in clinical trial participants as compared to non-participants, existence of such a trial effect has not been proved precisely. METHODS: This was a prospective cohort study to compare the prognoses for participants in the randomized controlled trial (SELECT BC) and non-participants. SELECT BC compared S-1 and taxane as first-line treatment for metastatic breast cancer. Non-participants were all patients who met the eligibility criteria of SELECT BC and who had been requested to participate in that trial by attending doctors and declined. The study aimed to compare the prognoses between participants and non-participants. The primary endpoint was median overall survival. RESULTS: The median OS in participants was significantly superior to that in non-participants with a statistically significant difference (36.8 months vs. 25.2 months. HR 1.48, p = 0.022). A similar result was obtained when only patients who received the same chemotherapy (S-1 or taxane) used in SELECT BC after declining participation were assumed as non-participants (36.8 months vs. 22.0 months. HR 2.03, p = 0.006). CONCLUSIONS: This study may suggest the existence of a trial effect, in which, for a given treatment, participation in a clinical trial is associated with a better outcome.
Authors: Caroline Hamm; Dora Cavallo-Medved; Devinder Moudgil; Lee McGrath; John Huang; Yueyang Li; Tyler W Stratton; Tyler Robinson; Krista Naccarato; Stephen Sundquist; Janet Dancey Journal: Cancer Control Date: 2022 Jan-Dec Impact factor: 2.339