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.

Background. Therapy for relapsed or refractory (r/r) T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) in children is challenging, and new treatment methods are needed. Previous studies have shown a promising response to the addition of nelarabine to chemotherapy for r/r T-ALL and T-LBL. Methods. We retrospectively analyzed the therapy of nelarabine in combination with etoposide, cyclophosphamide, and intrathecal therapy in eight pediatric patients with r/r T-ALL and T-LBL. The treatment regimen consisted of five consecutive days each of nelarabine (650mg/m 2/dose) and etoposide (100mg/m 2/dose)/cyclophosphamide (440mg/m 2/dose) separated by at least three days. Results. Five patients had T-ALL, and three patients had T-LBL. Of all patients, five achieved complete response, and the other three achieved partial response. All the patients underwent hematopoietic stem cell transplantation (HSCT) after two cycles of the treatment, except for one case with one course. Three patients who had previously received HSCT were treated with reduced-intensity conditioning regimens, including fludarabine, melphalan, and nelarabine; one of whom is still alive over five years after the second HSCT. Grade 2 neuropathy occurred in one patient, and other severe toxicities commonly associated with nelarabine were not observed during nelarabine-containing salvage therapy. With a median follow-up of 900 days for survivors, the 2-year overall survival and event-free survival rates were 60.0% and 36.5%, respectively. Conclusion. The addition of nelarabine to reinduction chemotherapy was useful for HSCT in remission and did not lead to excessive toxicity. In addition, a conditioning regimen including nelarabine appeared to be effective in previous HSCT patients.