Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world study
Introduction: Metronomic maintenance therapy (MMT) has significantly improved the survival of patients with high-risk rhabdomyosarcoma in clinical trials. However, there remains a lack of relevant data on its effectiveness in real-world situations.Methods: We retrospectively retrieved data of 459 pa...
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Frontiers Media S.A.
2023-05-01
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Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2023.1132219/full |
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author | Yingxia Lan Yingxia Lan Liuhong Wu Liuhong Wu Ye Hong Ye Hong Xiaofei Sun Xiaofei Sun Juan Wang Juan Wang Junting Huang Junting Huang Feifei Sun Feifei Sun Jia Zhu Jia Zhu Zijun Zhen Zijun Zhen Yizhuo Zhang Yizhuo Zhang Mengjia Song Mengjia Song Suying Lu Suying Lu |
author_facet | Yingxia Lan Yingxia Lan Liuhong Wu Liuhong Wu Ye Hong Ye Hong Xiaofei Sun Xiaofei Sun Juan Wang Juan Wang Junting Huang Junting Huang Feifei Sun Feifei Sun Jia Zhu Jia Zhu Zijun Zhen Zijun Zhen Yizhuo Zhang Yizhuo Zhang Mengjia Song Mengjia Song Suying Lu Suying Lu |
author_sort | Yingxia Lan |
collection | DOAJ |
description | Introduction: Metronomic maintenance therapy (MMT) has significantly improved the survival of patients with high-risk rhabdomyosarcoma in clinical trials. However, there remains a lack of relevant data on its effectiveness in real-world situations.Methods: We retrospectively retrieved data of 459 patients < 18 years of age diagnosed with rhabdomyosarcoma at Sun Yat-sen University Cancer Center from January 2011 to July 2020 from our database. The MMT regimen was oral vinorelbine 25–40 mg/m2 for twelve 4-week cycles on days 1, 8, and 15, and oral cyclophosphamide 25–50 mg/m2 daily for 48 consecutive weeks.Results: A total of 57 patients who underwent MMT were included in the analysis. The median follow-up time was 27.8 (range: 2.9–117.5) months. From MMT to the end of follow-up, the 3-year PFS and OS rates were 40.6% ± 6.8% and 58.3% ± 7.2%, respectively. The 3-year PFS was 43.6% ± 11.3% in patients who were initially diagnosed as low- and intermediate-risk but relapsed after comprehensive treatment (20/57), compared with 27.8% ± 10.4% in high-risk patients (20/57) and 52.8% ± 13.3% in intermediate-risk patients who did not relapse (17/57). The corresponding 3-year OS for these three groups was 65.8% ± 11.4%, 50.1% ± 12.9%, and 55.6% ± 13.6%, respectively.Conclusion: We present a novel study of MMT with oral vinorelbine and continuous low doses of cyclophosphamide in real-world pediatric patients with RMS. Our findings showed that the MMT strategy significantly improved patient outcomes and may be an effective treatment for high-risk and relapsed patients. |
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spelling | doaj.art-b2fb7830c2ec493496e812594b02cd9c2023-05-03T04:59:07ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122023-05-011410.3389/fphar.2023.11322191132219Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world studyYingxia Lan0Yingxia Lan1Liuhong Wu2Liuhong Wu3Ye Hong4Ye Hong5Xiaofei Sun6Xiaofei Sun7Juan Wang8Juan Wang9Junting Huang10Junting Huang11Feifei Sun12Feifei Sun13Jia Zhu14Jia Zhu15Zijun Zhen16Zijun Zhen17Yizhuo Zhang18Yizhuo Zhang19Mengjia Song20Mengjia Song21Suying Lu22Suying Lu23Sun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaSun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangzhou, ChinaDepartment of Pediatric Oncology, Sun Yat-sen University Cancer Center, Guangzhou, ChinaIntroduction: Metronomic maintenance therapy (MMT) has significantly improved the survival of patients with high-risk rhabdomyosarcoma in clinical trials. However, there remains a lack of relevant data on its effectiveness in real-world situations.Methods: We retrospectively retrieved data of 459 patients < 18 years of age diagnosed with rhabdomyosarcoma at Sun Yat-sen University Cancer Center from January 2011 to July 2020 from our database. The MMT regimen was oral vinorelbine 25–40 mg/m2 for twelve 4-week cycles on days 1, 8, and 15, and oral cyclophosphamide 25–50 mg/m2 daily for 48 consecutive weeks.Results: A total of 57 patients who underwent MMT were included in the analysis. The median follow-up time was 27.8 (range: 2.9–117.5) months. From MMT to the end of follow-up, the 3-year PFS and OS rates were 40.6% ± 6.8% and 58.3% ± 7.2%, respectively. The 3-year PFS was 43.6% ± 11.3% in patients who were initially diagnosed as low- and intermediate-risk but relapsed after comprehensive treatment (20/57), compared with 27.8% ± 10.4% in high-risk patients (20/57) and 52.8% ± 13.3% in intermediate-risk patients who did not relapse (17/57). The corresponding 3-year OS for these three groups was 65.8% ± 11.4%, 50.1% ± 12.9%, and 55.6% ± 13.6%, respectively.Conclusion: We present a novel study of MMT with oral vinorelbine and continuous low doses of cyclophosphamide in real-world pediatric patients with RMS. Our findings showed that the MMT strategy significantly improved patient outcomes and may be an effective treatment for high-risk and relapsed patients.https://www.frontiersin.org/articles/10.3389/fphar.2023.1132219/fullpediatricrhabdomyosarcomametronomic maintenance therapysoft tissue sarcomachemotherapy |
spellingShingle | Yingxia Lan Yingxia Lan Liuhong Wu Liuhong Wu Ye Hong Ye Hong Xiaofei Sun Xiaofei Sun Juan Wang Juan Wang Junting Huang Junting Huang Feifei Sun Feifei Sun Jia Zhu Jia Zhu Zijun Zhen Zijun Zhen Yizhuo Zhang Yizhuo Zhang Mengjia Song Mengjia Song Suying Lu Suying Lu Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world study Frontiers in Pharmacology pediatric rhabdomyosarcoma metronomic maintenance therapy soft tissue sarcoma chemotherapy |
title | Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world study |
title_full | Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world study |
title_fullStr | Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world study |
title_full_unstemmed | Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world study |
title_short | Oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma: a real-world study |
title_sort | oral vinorelbine and continuous low doses of cyclophosphamide in pediatric rhabdomyosarcoma a real world study |
topic | pediatric rhabdomyosarcoma metronomic maintenance therapy soft tissue sarcoma chemotherapy |
url | https://www.frontiersin.org/articles/10.3389/fphar.2023.1132219/full |
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