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Successful treatment of infants with localized neuroblastoma based on their MYCN status

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Abstract

Background

The aim of this study was to evaluate the effectiveness of post-surgical chemotherapy for infants with localized neuroblastoma without MYCN amplification (MNA), and determine whether risk classification using MNA is reasonable.

Methods

Four hundred and fourteen eligible patients were registered between 1998 and 2004. Resectable patients in stage 1 and 2A/2B were treated by surgical resection only. Unresectable patients in stage 3 without MNA received either 6 cycles of regimen A or 3 cycles of regimen A plus 3 cycles of regimen C2; regimen A consisted of low doses of cyclophosphamide and vincristine and regimen C consisted of cyclophosphamide, vincristine and pirarubicin before surgical resection. The resectable and unresectable patients were randomly selected to receive post-surgical chemotherapy. The patients with MNA received intensive chemotherapy regimen D2, consisting of cyclophosphamide, vincristine, pirarubicin and cisplatin, and some of them received high-dose chemotherapy with stem cell transplantation.

Results

The 5-year event-free survival (5-EFS) rates of stage 1 and 2A/2B patients without MNA were 97.2 and 89.0% respectively (p = 0.02). A total of 31 patients in stage 3 without MNA received post-surgical chemotherapy, and 30 patients did not. The 5-EFS rates of these two groups (96.0 and 96.2%, respectively) were not significantly different (p = 0.869). The 5-EFS rate for localized patients with MNA (n = 6) was 50.0%, and that of patients without MNA was 95.0% (p < 0.001).

Conclusion

Post-surgical chemotherapy was therefore unnecessary for localized patients without MNA. This treatment strategy using MNA is considered to be appropriate in infants.

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Acknowledgments

The authors gratefully thank the many pediatric oncologists and pediatric surgeons in Japan who provided clinical data for this study. We especially wish to express our gratitude to the late Dr. Takafumi Matsumura who contributed to the establishment and the management of this study. This study was supported in part by grants for Scientific Research from the Ministry of Health, Labor, and Welfare of the Government of Japan and the Children’s Cancer Association of Japan.

Conflict of interest

All authors have no conflict of interest.

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Correspondence to Tomoko Iehara.

Appendix: Participating institutions and main investigators

Appendix: Participating institutions and main investigators

Kyushu University, Fukuoka (Suita S, Tajiri T); Kyoto Prefectural University of Medicine, Kyoto (Matsumura T); Tokyo Metropolitan Hachioji Children’s Hospital, Tokyo (Nisina T); Kagoshima University, Kagoshima (Kawano Y, Ijichi O); Tokyo Metropolitan Kiyose Children’s Hospital, Tokyo (Hayahi A, Kamagata S); Gunma Children’s Medical Center, Gunma (Tsuchida Y, Kuroiwa M); University of Tsukuba, Tsukuba (Kaneko M); Fukushima Medical University, Fukushima (Kikuta A); University of Yamagata, Yamagata (Kanazawa C); St. Marianna University of Medicine, Kawasaki (Shimada A, Okubo M); National Sapporo Hospital, Hokkaido (Hatae Y, Naito H); Hiroshima University, Hiroshima (Hiyama E, Nishimura S); Kurume University School of Medicine, Kurume (Tsuru T); Osaka University, Osaka (Fukuzawa M, Kusafuka T, Yoneda M); Nihon University School of Medicine, Tokyo (Mugishima H); National Hospital Organization, Kure Medical Center, Hiroshima (Tanaka T); Shiga University of Medicine, Shiga (Oota S).

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Iehara, T., Hamazaki, M., Tajiri, T. et al. Successful treatment of infants with localized neuroblastoma based on their MYCN status. Int J Clin Oncol 18, 389–395 (2013). https://doi.org/10.1007/s10147-012-0391-y

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  • DOI: https://doi.org/10.1007/s10147-012-0391-y

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