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.