Hepatoblastoma is the most frequent liver cancer affecting infants and children. While cure is possible in approximately 80% of patients, many children progress on initial therapy with refractory disease, having not achieved remission (refractory), or relapse after having achieved remission (relapse). No standards of care exist for such children, as relapsed/refractory hepatoblastoma (rrHBL) has not been formally studied to any significant extent through the international and national cooperative group mechanisms. This registry will fill this gap by prospectively collecting data on children with rrHBL, as well as biological material, to help advance treatment standards and effectiveness, as well as research into novel/better therapies.
Systemic Therapy and Published Outcomes for rrHBL:
There are only two publications describing experiences with relapsed hepatoblastoma in greater than several dozen affected children, coming from the SIOPEL1-3 studies in Europe (59 patients) and the INT0098 study in the United States (36 patients) respectively (1, 2). The SIOPEL study highlighted the need for combined treatment with chemotherapy in conjunction with surgical removal of the tumor and described an average time to relapse of 12 months from initial diagnosis, and three-year overall survival of 43% (1). However, patients who failed to achieve a complete remission with upfront therapy were excluded from that report and thus the survival data is likely an overestimate of true survival for relapsed and refractory hepatoblastoma patients treated on such studies. The INT0098 data demonstrates the role of doxorubicin for intensification of therapy at time of relapse in patients who have not previously received anthracycline therapy. Of the 173 children enrolled, 55 were found to have progression of disease or recurrence. 36 of these 55 children received cisplatin/5 fluorouracil/vincristine therapy when first diagnosed, and a doxorubicin-containing regimen was administered at time of relapse leading to cure in 31% when combined with surgery. Only 28% of this cohort overall were able to be rescued with chemotherapy and surgery (2). There is no general consensus in the literature or elsewhere for the best treatment strategies at time of relapse or refractory hepatoblastoma. The current registry aims to characterize all clinical aspects of rrHBL, with the primary goal being the advancement of knowledge enabling improved standardization and treatment outcomes for children with rrHBL and their families.
- Semeraro M, Branchereau S, Maibach R, Zsiros J, Casanova M, Brock P, Domerg C, Aronson DC, Zimmermann A, Laithier V, Childs M, Roebuck D, Perilongo G, Czauderna P, Brugieres L. Relapses in hepatoblastoma patients: clinical characteristics and outcome--experience of the International Childhood Liver Tumour Strategy Group (SIOPEL). Eur J Cancer. 2013 Mar;49(4):915-22.
- Malogolowkin MH, Katzenstein HM, Krailo M, Chen Z, Quinn JJ, Reynolds M, Ortega JA. Redefining the role of doxorubicin for the treatment of children with hepatoblastoma. J Clin Oncol. 2008 May 10;26(14):2379-83.