Sorafenib treatment in children with relapsed and refractory neuroblastoma: an experience of four cases.
Metastatic neuroblastoma is an aggressive malignancy with a poor prognosis. Recent findings have shown that sorafenib decreases cell viability and increases apoptosis in human neuroblastoma cell lines. We report an experience of compassionate use of sorafenib in children with treatment-refractory neuroblastoma. Sorafenib showed transient anti-tumor activity in all four patients without adverse effects. However, progression was observed after a short stabilization phase. While sorafenib showed minimal anti-tumor activity in our patients, it might still be effective in patients with neuroblastoma in an earlier stage.
Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.
Keiko
K
Okada
Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.
Yoshiko
Y
Nakano
Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.
Kai
K
Yamasaki
Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.
Chika
C
Nitani
Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.
Hiroyuki
H
Fujisaki
Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan.
Junichi
J
Hara
D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use; D002675:Child, Preschool; D019008:Drug Resistance, Neoplasm; D017809:Fatal Outcome; D005260:Female; D006801:Humans; D008297:Male; D009447:Neuroblastoma / Q000175:diagnosis* / Q000188:drug therapy*; D009536:Niacinamide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D047428:Protein Kinase Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D012008:Recurrence; D019233:Retreatment; D000077157:Sorafenib; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D009536:Niacinamide; D000077157:Sorafenib
Metastatic neuroblastoma; pediatric; sorafenib