Treatment Strategies for Children With Relapsed Pancreatoblastoma: A Literature Review

Abstract

Pancreatoblastoma (PB) is a tumor typically seen in childhood. Despite its rarity, there are some internationally agreed recommendations for its first-line treatment, but very little is known about the management of relapse. We reviewed the literature on the treatment and outcome of children with progressing/recurrent PB, and the role of high-dose chemotherapy (HD-CT) or liver transplantation in difficult cases. A first analysis concerned 15 patients: liver metastases were the most frequent cause of first-line treatment failure. Eight patients underwent surgery, only 3 were irradiated. Various second-line chemotherapy regimens were adopted, with evidence of response in 8 children. The most often-used combinations included etoposide, cyclophosphamide/ifosfamide, and cisplatin/carboplatin. Overall, 7 patients are alive with a median follow-up of 24 months (range, 3 to 88 mo). In a separate analysis, considering patients in first-line or second-line treatment, we found 5 of 6 patients alive after HD-CT and 3 of 3 after liver transplantation. Our review shows that the outcome for patients with recurrent PB is not always dismal, especially when surgery is possible. Different chemotherapy combinations can be used, and HD-CT or liver transplantation may be considered in selected cases.

International Society of Paediatric Surgical Oncology (IPSO) Surgical Practice Guidelines

“…The most often used combinations included etoposide, cyclophosphamide/ifosfamide and cisplatin/carboplatin. A review from Reggiani et al [ 37 ] showed that the outcome for patients with recurrent PBL was not always dismal, especially when surgery is possible.…”

Solid pancreatic masses in children: A review of current evidence and clinical challenges

“…Nonetheless, PBLs are rare with a recent systematic review identifying 81 pediatric cases since 1980 (5). PBLs are typically diagnosed in the first decade of life, with the median age being 4-5 years old (3,(6)(7)(8), although there are several cases reported in older children and adults (9)(10)(11)(12). Boys are more affected than girls.…”

“…The most effective and mainstay treatment of PBL is complete surgical resection. It is also the most important prognostic factor, with 5-year overall survival rates of >70% reported in patients without metastases who underwent R0 resection (6,18). In 81 pediatric cases of PBL, most patients underwent pancreaticoduodenectomy (PD; Whipple procedure) (44.1%), followed by spleen-preserving distal pancreatectomy (DP) (26.5%), central pancreatectomy (11.8%), tumor enucleation (11.8%), and DP with splenectomy (5.8%) (5).…”

“…Neoadjuvant chemotherapy may be required in cases of metastases or local invasion to allow for complete surgical resection. Often, after establishing a tissueconfirmed diagnosis of PBL, cisplatin and doxorubicin are administered for 4-6 cycles (6,8,18,30). Although substantial tumor regression has been reported in 50%-73% of patients undergoing neoadjuvant chemotherapy, the benefits of adjuvant chemotherapy are less clear and often utilized when surgery is incomplete (R1 or R2) or to prevent relapse (8,16,(27)(28)(29).…”

Pancreatoblastoma in children: EXPeRT/PARTNER diagnostic and therapeutic recommendations

“…In children with PBL relapse who received six cycles of PLADO chemotherapy in the first‐line treatment, ICE, VAC, or other regimens with or without anthracyclines might be therapeutic options 63 . The role of high‐dose chemotherapy with autologous peripheral blood stem cell rescue, RT and liver transplantation has not been established due to small number of patients treated with these modalities; however, these may be therapeutic strategies in selected cases (Level V; Grade C) 61,64 …”