Patients and methods: A retrospective analysis was conducted on the clinical data of children with relapsed or refractory YST and progressing conditions after receiving first- and second-line treatments who were undergoing S-TIC chemotherapy. The S-TIC regimen was as follows: oral sirolimus (1 mg/m²) on days 1-21, nab-paclitaxel (200 mg/m²) on day 1, ifosfamide (1200 mg/m²) on days 1-5, and carboplatin (500 mg/m²) on day 2. One cycle lasted for 3-4 weeks. Meanwhile, α-fetoprotein (AFP) was assessed every cycle and radiological assessment was performed every 2-3 cycles. The primary endpoint was the objective response rate (ORR) after four treatment cycles. Secondary endpoints included progression-free survival (PFS) rate, overall survival (OS), and safety profile.
Results: Twenty patients with multiple relapsed or refractory YST with a median age of 5 years received the S-TIC regimen. All patients received an average of 4.3 treatment cycles. Twelve (60%) patients achieved complete remission, six (30%) achieved partial remission, one (5%) achieved a stable disease, and one (5%) had a progressive disease after four treatment cycles. The ORR was 90%, the disease control rate was 95%, and the median follow-up time was 60 months. Four (20%) patients died of tumour progression, and the 5-year OS rate was 75%. Haematologic toxicity was the most common adverse reaction, with grades 3-4 neutropenia occurring in 95% of cases. Younger patients had a better OS than their older counterparts. Grades 3-4 toxicities and primarily neutropenia or thrombocytopenia were observed in 18 (95%) and 15 (75%) patients, respectively, and were considered acceptable for this patient population.
Conclusion: S-TIC treatment, with a very high ORR, may be an alternative regimen for children with multiple relapsed or refractory YST. Although the incidence of haematological adverse reactions is relatively high, it is controllable.
Keywords: germ cell tumours, relapsed or refractory yolk sac tumour, sirolimus, third-line regimens
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