This study aimed to investigate the short-term efficacy and adverse reactions of irinotecan combined with first-line chemotherapeutics for the treatment of pediatric hepatoblastoma with pulmonary metastasis (HB-PM). Forty-one pediatric patients with HB-PM undergoing cisplatin + fluorouracil + vincristine + doxorubicin (C5VD) treatment with bad therapeutic effect or bad response were instead treated with two cycles of an irinotecan protocol (vincristine + irinotecan + cyclophosphamide + cisplatin). The changes in recent alpha-fetoprotein (AFP), efficacy and adverse reactions in these patients were statistically analyzed. Results showed that, the median level of AFP before chemotherapy was 56432 μg/L; however, it was significantly lower (749 μg/L) after two cycles of chemotherapy (rank sum test, P = 0.00). After two cycles of chemotherapy, three patients achieved a complete response and 32 patients achieved a partial response. The recent efficacy cases accounted for 85.36% of patients (35/41). The delayed diarrhea was the most common adverse reaction to irinotecan, with an incidence rate of 58.53% (24/41), which was improved after symptomatic treatment. In conclusion, the protocol of irinotecan combined with first-line chemotherapeutics can be used for the treatment of HB-PM that is not sensitive to the C5VD protocol, with good short-term curative effect and tolerable adverse reactions.
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