Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2024 Jun;71(6):e30976.
doi: 10.1002/pbc.30976. Epub 2024 Apr 5.

A nationwide phase II study of delayed local treatment for children with high-risk neuroblastoma: The Japan Children's Cancer Group Neuroblastoma Committee Trial JN-H-11

Akihiro Yoneda  1   2   3 , Hiroyuki Shichino  1   4 , Tomoro Hishiki  1   5 , Kimikazu Matsumoto  1   6 , Miki Ohira  1   7 , Takehiko Kamijo  1   7 , Tatsuo Kuroda  1   8 , Toshinori Soejima  1   9 , Atsuko Nakazawa  1   10 , Tetsuya Takimoto  1   11 , Isao Yokota  1   12 , Satoshi Teramukai  1   13 , Hideto Takahashi  1   14 , Takashi Fukushima  1   15 , Junichi Hara  1   16 , Michio Kaneko  1   17 , Hitoshi Ikeda  1   18 , Tatsuro Tajiri  1   19 , Hideo Mugishima  1   20 , Akira Nakagawara  1   21
Affiliations
Clinical Trial

A nationwide phase II study of delayed local treatment for children with high-risk neuroblastoma: The Japan Children's Cancer Group Neuroblastoma Committee Trial JN-H-11

Akihiro Yoneda et al. Pediatr Blood Cancer. 2024 Jun.

Abstract

Purpose: Survival rates of patients with high-risk neuroblastoma are unacceptable. A time-intensified treatment strategy with delayed local treatment to control systemic diseases has been developed in Japan. We conducted a nationwide, prospective, single-arm clinical trial with delayed local treatment. This study evaluated the safety and efficacy of delayed surgery to increase treatment intensity.

Patients and methods: Seventy-five patients with high-risk neuroblastoma were enrolled in this study between May 2011 and September 2015. Delayed local treatment consisted of five courses of induction chemotherapy (cisplatin, pirarubicin, vincristine, and cyclophosphamide) and myeloablative high-dose chemotherapy (melphalan, etoposide, and carboplatin), followed by local tumor extirpation with surgery and irradiation. The primary endpoint was progression-free survival (PFS). The secondary endpoints were overall survival (OS), response rate, adverse events, and surgical complications.

Results: Seventy-five patients were enrolled, and 64 were evaluable (stage 3, n = 8; stage 4, n = 56). The estimated 3-year PFS and OS rates (95% confidence interval [CI]) were 44.4% [31.8%-56.3%] and 80.7% [68.5%-88.5%], resspectively. The response rate of INRC after completion of the treatment protocol was 66% (42/64; 95% CI: 53%-77%; 23 CR [complete response], 10 VGPR [very good partial response], and nine PR [partial response]). None of the patients died during the protocol treatment or within 30 days of completion. Grade 4 adverse effects, excluding hematological adverse effects, occurred in 48% of patients [31/64; 95% CI: 36%-61%]. Major Surgical complications were observed in 25% of patients [13/51; 95% CI: 14%-40%].

Conclusion: This study indicates that delayed local treatment is feasible and shows promising efficacy, suggesting that this treatment should be considered further in a comparative study of high-risk neuroblastoma.

Keywords: clinical trial; delayed local treatment; high risk; multidisciplinary treatment; neuroblastoma.

PubMed Disclaimer

References

REFERENCES

    1. Park JR, Kreissman SG, London WB, et al. Effect of tandem autologous stem cell transplant vs single transplant on event‐free survival in patients with high‐risk neuroblastoma: a randomized clinical trial. JAMA. 2019;322(8):746‐755.
    1. Ikeda H, Iehara T, Tsuchida Y, et al. Experience with International Neuroblastoma Staging System and Pathology Classification. Br J Cancer. 2002;86(7):1110‐1116.
    1. Kaneko M, Tsuchida Y, Mugishima H, et al. Intensified chemotherapy increases the survival rates in patients with stage 4 neuroblastoma with MYCN amplification. J Pediatr Hematol Oncol. 2002;24(8):613‐621.
    1. Kawa K, Ohnuma N, Kaneko M, et al. Long‐term survivors of advanced neuroblastoma with MYCN amplification: a report of 19 patients surviving disease‐free for more than 66 months. J Clin Oncol. 1999;17(10):3216‐3220.
    1. Ohnuma N, Takahashi H, Kaneko M, et al. Treatment combined with bone marrow transplantation for advanced neuroblastoma: an analysis of patients who were pretreated intensively with the protocol of the Study Group of Japan. Med Pediatr Oncol. 1995;24(3):181‐187.

Publication types

MeSH terms