Cacciavillano WD et al. (2004) Phase II study of high-dose cyclophosphamide in relapsing and/or resistant hepatoblastoma in children: a study from the SIOPEL group. Eur J Cancer 40: 2274–2279

There is currently no consensus regarding the treatment of children with resistant or relapsing hepatoblastoma. Partly because of the rarity of the condition, few chemotherapeutic agents have been rigorously tested in this setting. Until now, the efficacy of cyclophosphamide (CPM) as a single agent has not been assessed. Cacciavillano and colleagues from the SIOPEL group have recently published the results of their phase II trial, which was designed to evaluate the response to high-dose CPM in children with hepatoblastoma.

The international study included 18 children with refractory or relapsing hepatoblastoma, all of whom had received first-line chemotherapy according to the SIOPEL 2 or 3 protocols. One patient was excluded from the study after one course of CPM (disease stabilized); the remainder received between one and four courses of the drug and were then evaluated.

Only one patient responded to CPM, achieving complete response after three courses. Despite further treatment with carboplatin and doxorubicin, this child relapsed again and subsequently died. A second patient achieved stable disease but died during hepatic surgery. The remaining 15 patients all experienced disease progression and died.

Given the low response rate and severe side effects of CPM, the authors conclude that the drug is not effective as a single agent in this setting.