Results of the JRS-I LRA0401 and LRB0402 Japan Rhabdomyosarcoma Study Group trials for low-risk embryonal rhabdomyosarcoma

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发表日期：

2024年8月23日

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Hajime Hosoi,
Mitsuru Miyachi,
Satoshi Teramukai,
Satomi Sakabayashi,
Kunihiko Tsuchiya,
Yasumichi Kuwahara,
Rie Onodera,
Kotone Matsuyama,
Isao Yokota,
Hiroshi Hojo,
Hajime Okita,
Jun-Ichi Hata,
Minori Hamasaki,
Masazumi Tsuneyoshi,
Yoshinao Oda,
Atsuko Nakazawa,
Miho Kato,
Tetsuya Takimoto,
Keizo Horibe,
Jun-Ichi Hara,
Sachiyo Suita,
Ryoji Hanada,
Hidekazu Masaki,
Miwako Nozaki,
Hitoshi Ikeda,
Seiji Kishimoto,
Michio Kaneko,
Akira Kawai,
Yasuhide Morikawa

摘要

Background

Failure-free survival (FFS) rates of low-risk patients with rhabdomyosarcoma improved in Intergroup Rhabdomyosarcoma Study IV after the escalation of cyclophosphamide total dose to 26.4 g/m². However, this dose may increase the risk of adverse events, including infertility, in some patients. The JRS-I LRA0401 and LRB0402 protocols aimed to reduce the cyclophosphamide dose to 9.6 g/m² and 17.6 g/m², respectively, without decreasing the FFS rates.

Methods

Subgroup-A patients received eight cycles (24 weeks) of vincristine, actinomycin D, and 1.2 g/m²/cycle cyclophosphamide. Subgroup-B patients received eight cycles (24 weeks) of vincristine, actinomycin D, and 2.2 g/m²/cycle cyclophosphamide, followed by six cycles (24 weeks) of vincristine and actinomycin D. Group II/III patients in both subgroups received radiotherapy.

Results

In subgroup A (n = 12), the 3-year FFS rate was 83% (95% confidence interval [CI], 48–96), and the 3-year overall survival (OS) rate was 100%. Only one isolated local recurrence was observed (8.3%). There were no unexpected grade-4 toxicities and no deaths. In subgroup B (n = 16), the 3-year FFS and OS rates were 88% (95% CI, 59–97) and 94% (95% CI, 63–99), respectively. There were no unexpected grade 4 toxicities and no deaths.

Conclusions

Shorter duration therapy using vincristine, actinomycin D, and lower dose cyclophosphamide with or without radiotherapy for patients with low-risk subgroup A rhabdomyosarcoma (JRS-I LRA0401 protocol) and moderate reduction of cyclophosphamide dose for patients with low-risk subgroup B rhabdomyosarcoma (JRS-I LRB0402 protocol) did not compromise FFS.