Successful treatment of young childhood standard-risk hepatoblastoma
with cisplatin monotherapy using central review system
Abstract
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) evaluated central review system in Japan. In the previous JPLT2
study, cases with resectable tumors without any annotation factors in
the PRETEXT classification (standard risk HB) showed favorable outcomes
by the therapies consisted of CDDP and pirarubicin, but showed
toxicities and late complications. In this JPLT3-S trial, less intensity
regimen consisted of CDDP alone were evaluated in the young children
(< 3 years old) with standard risk HB. Methods:
Patients who were less than three years of age, who had 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, we introduced central radiological and pathological reviews
of all patients. The primary outcome was 3-year progression-free
survival (PFS). Results: A total of 38 patients (23 female)
were included. The median patient age was 12 (range, 2-34) months. 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 to 100). All 38
patients survived (follow-up period 38-98 months), and the OS rate was
100% (confidence interval, 100). There were no cases with late
complication without ototoxicity. Conclusion: CDDP monotherapy
regimen is feasible in young patients with localized HB classified by
central review.