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Autologous peripheral blood stem‐cell transplantation with a double‐conditioning regimen for recurrent hepatoblastoma after liver transplantation – A valid therapeutic option or just too much?

The report by Niwa et al. (1) on ‘‘Successful autologous peripheral blood stem cell transplantation with a double-conditioning regimen for recurrent hepatoblastoma (HB) after liver transplantation’’ challenges the readers in many aspects. The authors describe a complex clinical history, of a four-year-old child who suffered from a relapsing HB after orthotopic liver transplantation (LTX), which was successfully brought to an apparent happy end. Because of this successful outcome one would be tempted to accept the appropriateness of all the clinical decisions made. However, caution is needed in interpreting these data, to understand which are the real ‘‘take-home messages.’’ The possibility that LTX recipients may also undergo high-dose chemotherapy (HD-CT) with autologous peripheral blood stem cell (PBSC) reconstitution is an important finding. The prompt hematologic engraftment this child had, with an almost normal bone marrow reconstitution within the first two wk after PBSC infusion, is unquestionably significant. HD-CT has been repetitively proven to represent a potential valid therapeutic option for treating high risk pediatric solid tumors (2). Thus, this report may be used to support the decision to use this therapeutic option in the unlikely event that after solid organ transplant, a patient develops a malignant condition that can be potentially treated with HD-CT and PBSC reconstitution. It is worth noting that this child, despite resumed treatment with tacrolimus on day 11, developed laboratory and histologic evidence of mild rejection. This finding highlights the need of properly modulating the immunosuppressive regimen even in face of aggressive myeloablative regimens. Other than that, no other real messages are confidentially apparent. More precisely, the role of double HD-CT with PBSC rescue for the treatment of relapsing HB cannot really be delineated. Patients with relapsing HB, particularly if receiving significant pretreatment, have a very poor salvage rate. Thus, one would be tempted to assume the important role of HD-CT on the successful outcome in this case. The justification for the use of HD-CT was appropriate; a past history of a chemosensitive tumor [despite what the authors say, the tumor was chemosensitive; the a-fetoprotein (AFP) dropped significantly in response to therapy and the fact that the tumor remained unresectable most likely was due to tumor location rather than a lack of response], an unfavorable prognosis, minimal residual disease, and lack of consolidated valid alternatives. These four elements are usually used to indicate such an approach of a patient affected by a solid tumor for HD-CT with PBSC rescue. However, by themselves, even in the face of a successful outcome, these elements do not prove the role of this therapy in salvaging relapsing HB. Among children affected by HB, long-term survivors after the appearance of an isolated lung lesion both at diagnosis or at the time of tumor recurrence, which is amenable to complete resection, have been reported by different authors regardless of specific treatment received afterwards. In the SIOPEL experience, Perilongo et al. Pediatr Transplantation 2009: 13: 148–149 2008 Wiley Periodicals, Inc.