Treatment of hepatoblastoma with high-dose chemotherapy and stem cell rescue

The pediatric blood and marrow transplant consortium experience and review of the literature

Erin E. Karski, Christopher C. Dvorak, Wing Leung, Weston Miller, Peter J. Shaw, Muna Qayed, Emmanuel Katsanis, James H. Feusner

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

BACKGROUND: Children with high-risk or relapsed hepatoblastoma continue to represent treatment challenges. Multiple case reports have documented the use of high-dose chemotherapy with stem cell rescue (HDC) for this population; however, the efficacy and appropriate use of HDC remains unclear. PROCEDURE: A literature search was performed to identify cases of hepatoblastoma that were treated with HDC. Additional patients were identified by a query through the Pediatric Blood and Marrow Transplant Consortium. All cases were categorized as undergoing HDC as part of their initial treatment or for relapsed disease. Overall survival (OS) and event-free survival (EFS) proportions were calculated for each group. Subgroup analyses were performed looking at the effects of remission status, initial stage, and relapse site. RESULTS: Forty-two patients were identified. Thirty-one patients received HDC as part of their initial treatment and 55% were long-term survivors with 48% event-free. Eleven received HDC for relapsed disease and 64% were long-term survivors, 36% without events. CONCLUSIONS: It is difficult to draw firm conclusions from a small number of nonrandomized patients who had different stages, treatments, and events before undergoing HDC. However, our calculated EFS and OS proportions are consistent with current data using multimodal therapy without HDC, suggesting that HDC (at least as currently delivered) for hepatoblastoma may not be beneficial.

Original languageEnglish (US)
Pages (from-to)362-368
Number of pages7
JournalJournal of Pediatric Hematology/Oncology
Volume36
Issue number5
DOIs
StatePublished - 2014

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Hepatoblastoma
Stem Cells
Bone Marrow
Pediatrics
Transplants
Drug Therapy
Disease-Free Survival
Survivors
Therapeutics
Survival
Recurrence
Population

Keywords

  • hepatoblastoma
  • high-dose chemotherapy
  • stem cell transplant

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Oncology
  • Hematology

Cite this

Treatment of hepatoblastoma with high-dose chemotherapy and stem cell rescue : The pediatric blood and marrow transplant consortium experience and review of the literature. / Karski, Erin E.; Dvorak, Christopher C.; Leung, Wing; Miller, Weston; Shaw, Peter J.; Qayed, Muna; Katsanis, Emmanuel; Feusner, James H.

In: Journal of Pediatric Hematology/Oncology, Vol. 36, No. 5, 2014, p. 362-368.

Research output: Contribution to journalArticle

Karski, Erin E. ; Dvorak, Christopher C. ; Leung, Wing ; Miller, Weston ; Shaw, Peter J. ; Qayed, Muna ; Katsanis, Emmanuel ; Feusner, James H. / Treatment of hepatoblastoma with high-dose chemotherapy and stem cell rescue : The pediatric blood and marrow transplant consortium experience and review of the literature. In: Journal of Pediatric Hematology/Oncology. 2014 ; Vol. 36, No. 5. pp. 362-368.
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abstract = "BACKGROUND: Children with high-risk or relapsed hepatoblastoma continue to represent treatment challenges. Multiple case reports have documented the use of high-dose chemotherapy with stem cell rescue (HDC) for this population; however, the efficacy and appropriate use of HDC remains unclear. PROCEDURE: A literature search was performed to identify cases of hepatoblastoma that were treated with HDC. Additional patients were identified by a query through the Pediatric Blood and Marrow Transplant Consortium. All cases were categorized as undergoing HDC as part of their initial treatment or for relapsed disease. Overall survival (OS) and event-free survival (EFS) proportions were calculated for each group. Subgroup analyses were performed looking at the effects of remission status, initial stage, and relapse site. RESULTS: Forty-two patients were identified. Thirty-one patients received HDC as part of their initial treatment and 55{\%} were long-term survivors with 48{\%} event-free. Eleven received HDC for relapsed disease and 64{\%} were long-term survivors, 36{\%} without events. CONCLUSIONS: It is difficult to draw firm conclusions from a small number of nonrandomized patients who had different stages, treatments, and events before undergoing HDC. However, our calculated EFS and OS proportions are consistent with current data using multimodal therapy without HDC, suggesting that HDC (at least as currently delivered) for hepatoblastoma may not be beneficial.",
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