Successful treatment of young childhood standard-risk hepatoblastoma with cisplatin monotherapy using a central review system

被引：2

作者：

Saeki, Isamu ^{[1
]}

Ida, Kohmei ^{[2
]}

Kurihara, Sho ^{[1
]}

Watanabe, Kenichiro ^{[3
]}

Mori, Makiko ^{[4
]}

Hishiki, Tomoro ^{[5
]}

Yokoi, Akiko ^{[6
]}

Fujimura, Junya ^{[7
]}

Honda, Shohei ^{[8
]}

Nogami, Yuki ^{[9
]}

Iehara, Tomoko ^{[10
]}

Kazama, Takuro ^{[11
]}

Sekiguchi, Masahiro ^{[12
]}

Kitagawa, Norihiko ^{[13
]}

Matsumura, Risa ^{[14
]}

Nomura, Motonari ^{[15
]}

Yamada, Yohei ^{[16
]}

Hanaki, Ryo ^{[17
]}

Kaneda, Hide ^{[18
]}

Takama, Yuichi ^{[19
]}

Inoue, Takeshi ^{[20
]}

Tanaka, Yukichi ^{[21
]}

Miyazaki, Osamu ^{[22
]}

Nagase, Hiroki ^{[23
]}

Takimoto, Tetsuya ^{[24
]}

Yoshimura, Kenichi ^{[25
]}

Hiyama, Eiso ^{[1
,26
]}

机构：

[1] Hiroshima Univ Hosp, Dept Pediat Surg, 1-2-3 Kasumi Minami Ku, Hiroshima, Hiroshima 7348551, Japan

[2] Teikyo Univ, Dept Pediat, Mizonokuchi Hosp, Kawasaki, Kanagawa, Japan

[3] Shizuoka Childrens Hosp, Dept Hematol & Oncol, Shizuoka, Japan

[4] Saitama Childrens Med Ctr, Dept Hematol Oncol, Saitama, Japan

[5] Chiba Univ, Grad Sch Med, Dept Pediat Surg, Chiba, Japan

[6] Kobe Childrens Hosp, Dept Pediat Surg, Kobe, Japan

[7] Juntendo Univ, Sch Med, Dept Pediat, Tokyo, Japan

[8] Hokkaido Univ Hosp, Dept Gastroenterol Surg 1, Sapporo, Hokkaido, Japan

[9] Natl Canc Ctr, Dept Pediat Oncol, Tokyo, Japan

[10] Kyoto Prefectural Univ Med, Grad Sch Med Sci, Dept Pediat, Kyoto, Japan

[11] Tohoku Univ Hosp, Dept Pediat Surg, Sendai, Japan

[12] Natl Ctr Child Hlth & Dev, Childrens Canc Ctr, Tokyo, Japan

[13] Kanagawa Childrens Med Ctr, Childrens Canc Ctr, Dept Surg, Yokohama, Japan

[14] Hiroshima Univ Hosp, Dept Pediat, Hiroshima, Japan

[15] Osaka Univ, Dept Pediat Surg, Osaka, Japan

[16] Keio Univ, Sch Med, Dept Pediat Surg, Tokyo, Japan

[17] Mie Univ Hosp, Dept Pediat, Tsu, Japan

[18] Nihon Univ, Sch Med, Dept Pediat Surg, Tokyo, Japan

[19] Osaka City Gen Hosp, Dept Pediat Surg, Osaka, Japan

[20] Osaka City Gen Hosp, Dept Pathol, Osaka, Japan

[21] Kanagawa Childrens Med Ctr, Dept Pathol, Yokohama, Japan

[22] Natl Ctr Child Hlth & Dev, Dept Radiol, Tokyo, Japan

[23] Juntendo Univ, Grad Sch Med Diagnost & Therapeut Intractable Dis, Tokyo, Japan

[24] Natl Ctr Child Hlth & Dev, Dept Childhood Canc Data Management, Tokyo, Japan

[25] Hiroshima Univ Hosp, Med Ctr Translat & Clin Res, Hiroshima, Japan

[26] Hiroshima Univ, Nat Sci Ctr Basic Res & Dev, Hiroshima, Japan

来源：

PEDIATRIC BLOOD & CANCER | 2024年 / 71卷 / 11期

基金：

日本学术振兴会;

关键词：

central review; children; cisplatin; hepatoblastoma; toxicity; SODIUM THIOSULFATE; LIVER-TUMOR; CHILDREN;

D O I：

10.1002/pbc.31255

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Background The JPLT3-S (Japanese Study Group for Pediatric Liver Tumors-3) study, conducted cisplatin (CDDP) monotherapy for young children (<3 years old) with standard-risk hepatoblastoma (HB) using a central review system in Japan. In the previous JPLT2 study, cases with resectable tumors without any annotation factors in the PRETEXT (PRETreatment EXTent of disease) classification (standard-risk HB) showed favorable outcomes with treatment consisting of CDDP and pirarubicin, but showed toxicities and late complications. In the JPLT3-S trial, a less intense regimen consisting of CDDP alone was evaluated. Methods Patients who were less than 3 years of age and with PRETEXT I, II, or III HB without any annotation factors (e.g., E1, E1a, E2, E2a, H1, N1, P2, P2a, V3, and V3a) were eligible for inclusion in this study. In this trial, the central radiological and pathological features of all patients were reviewed. The primary outcome was the 3-year progression-free survival (PFS). Results A total of 38 patients (23 female) were included. The median patient age was 12 months (range: 2-34). Two patients discontinued treatment because of progressive disease, and five patients discontinued treatment for other reasons. The 3-year PFS rate was 93.9% (95% confidence interval [CI]: 86.4%-100%). All 38 patients survived (follow-up period 38-98 months), and the OS rate was 100% (CI: 100). Eighteen of the 38 patients (47.4%) experienced ototoxicity as a late complication. Conclusion CDDP monotherapy regimen is feasible in young patients with localized HB, as classified by a central review.

引用

页数：9

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