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Sorafenib treatment in children with relapsed and refractory neuroblastoma: an experience of four cases

Keiko Okada1, Yoshiko Nakano1, Kai Yamasaki1

  • 1Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.

Cancer Medicine
|June 7, 2016
PubMed

Insights

Sorafenib showed temporary anti-tumor effects in children with advanced neuroblastoma but did not prevent disease progression. Further research may explore sorafenib for earlier-stage neuroblastoma treatment.

Area of Science:

  • Pediatric Oncology
  • Pharmacology

Background:

  • Metastatic neuroblastoma is an aggressive childhood cancer with a poor prognosis.
  • Sorafenib has demonstrated preclinical efficacy in neuroblastoma cell lines by reducing viability and inducing apoptosis.

Observation:

  • This study reports the compassionate use of sorafenib in four children with treatment-refractory metastatic neuroblastoma.
  • The treatment was administered without observed adverse effects.

Findings:

  • Sorafenib exhibited transient anti-tumor activity in all treated patients.
  • Despite initial stabilization, all patients experienced disease progression after a short period.

Implications:

  • Sorafenib demonstrated minimal efficacy in this cohort of advanced neuroblastoma patients.
  • The drug may hold potential for neuroblastoma treatment in earlier disease stages.
Keywords:
Metastatic neuroblastomapediatricsorafenib

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