The two most common childhood liver cancers are hepatoblastoma (HB) and hepatocellular carcinoma (HCC). There is a third entity termed hepatocellular neoplasm not otherwise specified (HCN NOS) and felt to include both HB and HCC histologies or a hybrid histology. HB has an annual incidence of 0.8 per million children. The age-adjusted HCC incidence is about 0.4 per million children. There are no incidence data available for HCN NOS.
Newly diagnosed HB, HCC, or HCN NOS patients are usually treated within the framework of a clinical trial with well-defined treatment options which vary according to the extent of disease and anticipated prognosis of the individual case. Some patients will not be cured with upfront therapy and are considered to have treatment-refractory disease. Other patients may relapse after having been in remission. Patients with refractory or relapsed disease traditionally receive additional treatment which may include chemotherapy or additional surgical and/or interventional approaches aimed towards achieving local control. This “salvage” treatment is usually not specified in upfront trial protocols and is therefore largely individualized according to single institution opinion.
In some patients, the tumour may not be eradicated completely by first-line treatment. Patients in this circumstance are considered to have treatment-refractory disease and traditionally receive additional treatment including further chemotherapy and/or additional surgical or interventional approaches aimed towards achieving local control. This “salvage” treatment is usually not specified in upfront trial protocols and is therefore largely individualized, according to single institution opinion.
If a patient was deemed to be in remission, i.e. free of tumour at some point after initial treatment with curative intent, and later the disease returns, then the patient is considered to have relapsed disease. In such cases, patients are again treated on an individual basis according to clinician’s preference, since there are no standard trial protocols available for such patients. The treatment often needs to be adapted to the individual patient and selected in view of cumulative doses of chemotherapeutic agents and previous adverse effects. On occasion, children are enrolled on early-phase trials but these trials are often agent/pathway- as opposed to disease-specific (Trobaugh-Lotrario et al. PBC 2012).
Longterm outcome for patients with refractory or relapsed HB, HCC, and HCN-NOS is unsatisfactory. The international consortium currently conducting the PHITT trial plans to incorporate treatment guidelines and data acquisition for relapsed and refractory patients in a prospective fashion as part of a second generation protocol. To guide writing of this future proposal, the RELIVE consortium is proposing a retrospective collection of data regarding recently utilized treatment regimens as well as short- and longterm patient outcomes. Anecdotal information is insufficient; therefore, we propose to retrospectively collect cases in a central registry allowing for a systematic overview and statistical assessment of the various approaches used.