A retrospective study of clinical features and outcome in patients with refractory or recurrent hepatoblastoma: A single institution experience

Hou, Jen‐Yin; Yeh, Ting‐Chi; Huang, Ting‐Huan; Sheu, Jin‐Chuan; Liu, Hsi‐Che

doi:10.1016/j.pedneo.2021.03.018

Cited by 7 publications

(4 citation statements)

References 37 publications

(30 reference statements)

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“…[19][20][21][22] Despite significant advancements in treatment approaches, R/R hepatoblastoma still has a poor prognosis. 9,23,24 In this study, autologous HSCT was performed in 6 children with R/R hepatoblastoma after achieving CR with chemotherapy, and the OS rate was 100%. One patient relapsed, maintained CR status, and was treated with salvage chemotherapy.…”

Section: Discussionmentioning

confidence: 99%

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Successful Treatment of Refractory or Relapsed Hepatoblastoma With Autologous Hematopoietic Stem Cell Transplantation in Children

Kim,

Choi,

Hong

et al. 2024

Journal of Pediatric Hematology/Oncology

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Background: The standard-risk hepatoblastoma has a good prognosis in children; however, refractory or relapsed (R/R) hepatoblastoma has a poor prognosis and high mortality rate. This study aimed to demonstrate the efficacy of high-dose chemotherapy and autologous hematopoietic stem cell transplantation (HSCT) rescue in pediatric patients with R/R hepatoblastoma. Methods: We retrospectively analyzed 6 pediatric patients with R/R hepatoblastoma who underwent autologous HSCT. The MEC conditioning regimen was used for all patients, comprising melphalan 140 mg/m2/day intravenously (IV) on day 7 and 70 mg/m2 on day 6, etoposide 200 mg/m2 IV on days 5 to 8, and carboplatin 400 mg/m2 IV on days 5 to 8. One patient received a TopoThioCarbo regimen, comprising topotecan 2 mg/m2/day IV on days 4 to 8, thiotepa 300 mg/m2/day IV on days 6 to 8, and carboplatin 500 mg/m2/day IV on days 3 to 5, as the conditioning regimen for the first transplantation. This was followed by salvage chemotherapy for relapse, and the second transplantation was performed using MEC as the conditioning regimen. Results: We report the retrospective results of 6 patients with a median age of 1.8 (range 0.4 to 10.2) years who had R/R hepatoblastoma and underwent autologous HSCT. The median follow-up period was 58 (range 28 to 113) months after diagnosis. The median stage at diagnosis was 2.0 (range 2 to 4). Two patients had lung metastases during diagnosis. The median initial alpha-fetoprotein level was 292,888 (range 28,831 to 2,406,942) ng/mL, and the median number of chemotherapy lines before autologous HSCT was 3.5 (range 2 to 7). The disease status before HSCT was complete remission (CR) for all patients. The engraftment rate was 100%. No treatment-related mortality was reported. The 3-year event-free survival and overall survival rates were 83.3% and 100%, respectively. One patient relapsed after the second HSCT and achieved CR after salvage chemotherapy. Conclusion: This study suggests autologous HSCT as an effective treatment in pediatric patients with R/R hepatoblastoma. Nevertheless, future large-scale prospective studies are warranted.

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Section: Discussionmentioning

confidence: 99%

“…The CCG-8881A regimen, which we employed as first-line chemotherapy, is based on cisplatin and has been used at our institution since the 1990s 19–22 . Despite significant advancements in treatment approaches, R/R hepatoblastoma still has a poor prognosis 9,23,24 …”

Section: Discussionmentioning

confidence: 99%

Successful Treatment of Refractory or Relapsed Hepatoblastoma With Autologous Hematopoietic Stem Cell Transplantation in Children

Kim,

Choi,

Hong

et al. 2024

Journal of Pediatric Hematology/Oncology

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“…In addition, there are only a small number of articles providing insights into this area. The majority of these are case reports or single-center studies (e.g., [7]). The three most recent, larger studies of note in this area are based on results from the International Childhood Liver Tumor Strategy Group (SIOPEL) ( [6]: 59 relapse patients) as noted above, and from the Children's Oncology Group (COG) ( [8]: 71 patients, relapse + progression; [9]: 30 patients, relapse + progression).…”

Section: Introductionmentioning

confidence: 99%

Hepatoblastoma Relapse—Findings from the German HB99 Trial and the German Liver Tumor Registry

Maxwell,

Häberle,

Kappler

et al. 2024

Cancers

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Survival rates for HB patients have improved; however, outcomes for patients who relapse remain poor. A retrospective review of information gathered for the HB99 study and the German Liver Tumor Registry identified 25 relapse patients (6.9%, 25/362). The median time from initial diagnosis to first relapse was 13 months (range: 5–66 months). Two patients relapsed >36 months after initial diagnosis. A total of 68% (17/25) of relapses were metastatic, 24% local, and 8% combined. 67% of local relapses were alive at the last follow-up, in contrast to 53% of metastatic and 0% of combined relapses. At the last follow-up, 73% (8/11) of patients with lung relapses were still alive (0/4 with peritoneal, 1/2 with CNS involvement). A total of 20% of the patients had AFP-negative relapses, 64% of the relapse patients achieved a second complete remission, 69% were still in complete second remission at the last follow-up (median FU of 66 months), and 83% (5/6) of irinotecan-naïve patients who received relapse treatment including irinotecan were in second complete remission at the last follow-up. The 3-year overall survival/event-free survival from relapse was 63%/48% respectively. There is a good chance that HB patients will achieve a second remission despite a first relapse. However, patients who suffer further relapses tend to have a poorer prognosis.

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“…HB treatment entails surgical resection or liver transplantation combined with pre- and post-operative administration of cisplatin, carboplatin, and doxorubicin ( 6 ). This approach has improved the 5-year overall survival (OS) rate to greater than 80% ( 7 ), though patients with metastases or relapsed/refractory disease have significantly lower survival rates, emphasizing the need for novel treatment strategies ( 8 ).…”

Section: Introductionmentioning

confidence: 99%

SLC-0111, an inhibitor of carbonic anhydrase IX, attenuates hepatoblastoma cell viability and migration

et al. 2023

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BackgroundIn response to hypoxia, tumor cells undergo transcriptional reprogramming including upregulation of carbonic anhydrase (CA) IX, a metalloenzyme that maintains acid-base balance. CAIX overexpression has been shown to correlate with poor prognosis in various cancers, but the role of this CA isoform in hepatoblastoma (HB) has not been examined.MethodsWe surveyed the expression of CAIX in HB specimens and assessed the impact of SLC-0111, a CAIX inhibitor, on cultured HB cells in normoxic and hypoxic conditions.ResultsCAIX immunoreactivity was detected in 15 out of 21 archival pathology HB specimens. The CAIX-positive cells clustered in the middle of viable tumor tissue or next to necrotic areas. Tissue expression of CAIX mRNA was associated with metastasis and poor clinical outcome of HB. Hypoxia induced a striking upregulation of CAIX mRNA and protein in three HB cell models: the immortalized human HB cell line HUH6 and patient xenograft-derived lines HB-295 and HB-303. Administration of SLC-0111 abrogated the hypoxia-induced upregulation of CAIX and decreased HB cell viability, both in monolayer and spheroid cultures. In addition, SLC-0111 reduced HB cell motility in a wound healing assay. Transcriptomic changes triggered by SLC-0111 administration differed under normoxic vs. hypoxic conditions, although SLC-0111 elicited upregulation of several tumor suppressor genes under both conditions.ConclusionHypoxia induces CAIX expression in HB cells, and the CAIX inhibitor SLC-0111 has in vitro activity against these malignant cells.

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A retrospective study of clinical features and outcome in patients with refractory or recurrent hepatoblastoma: A single institution experience

Cited by 7 publications

References 37 publications

Successful Treatment of Refractory or Relapsed Hepatoblastoma With Autologous Hematopoietic Stem Cell Transplantation in Children

Successful Treatment of Refractory or Relapsed Hepatoblastoma With Autologous Hematopoietic Stem Cell Transplantation in Children

Hepatoblastoma Relapse—Findings from the German HB99 Trial and the German Liver Tumor Registry

SLC-0111, an inhibitor of carbonic anhydrase IX, attenuates hepatoblastoma cell viability and migration

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