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Rapid decrease of serum alpha-fetoprotein and tumor volume predicts outcome in children with hepatoblastoma treated

Rosa Nguyen1, M Beth McCarville2, April Sykes3

  • 1Department of Oncology, St. Jude Children's Research Hospital, 262 Danny Thomas Place, Memphis, TN, 38105, USA.

International Journal of Clinical Oncology
|May 11, 2018
PubMed

Insights

An early, significant drop in serum alpha-fetoprotein (sAFP) levels and tumor volume during neoadjuvant chemotherapy may predict better survival for children with unresectable hepatoblastoma.

Area of Science:

  • Pediatric Oncology
  • Hepatobiliary Malignancies
  • Chemotherapy Response Assessment

Background:

  • Neoadjuvant chemotherapy is crucial for treating unresectable hepatoblastoma in children, aiming to facilitate surgical resection.
  • Serum alpha-fetoprotein (sAFP) levels typically decrease as tumors respond to treatment.

Purpose of the Study:

  • To investigate the correlation between early changes in sAFP levels and tumor volume and patient outcomes in pediatric unresectable hepatoblastoma.
  • To assess the predictive value of early treatment response on survival in this patient population.

Main Methods:

  • Retrospective analysis of 34 children with unresectable hepatoblastoma treated with neoadjuvant chemotherapy.
  • Correlation of sAFP level dynamics and tumor volume changes with event-free and overall survival.
  • Assessment of tumor response using RECIST 1.1 criteria.

Main Results:

  • A decrease of ≥90% in sAFP levels after two chemotherapy courses was associated with improved event-free survival (P=0.039) and overall survival (OS; P=0.045).
  • Significant reduction in average tumor volume (481 mL to 268 mL; P<0.001) during early treatment correlated with OS (P=0.029).
  • RECIST 1.1 response and relative changes in sAFP or tumor volume between course 2 and pre-surgery did not predict OS.

Conclusions:

  • Early decline in sAFP levels and tumor volume may serve as a predictive marker for survival in pediatric unresectable hepatoblastoma.
  • This finding could help identify patients who may require alternative therapies for improved outcomes.
  • Further validation in larger cohorts is needed before clinical implementation in prospective trials.
Abstract

Keywords:
HepatoblastomaPediatric oncologyTumor marker

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