[Clinical efficacy of CCG7942/POG9354 protocol in treatment of peripheral primitive neuroectodermal tumor in...

To investigate the clinical manifestations, diagnosis, and treatment of peripheral primitive neuroectodermal tumor (pPNET) in children and the survival of patients treated with the CCG7942/POG9354 protocol.

A retrospective analysis was performed on the clinical data of 10 patients with pPNET admitted from October 2008 to October 2013. Of the 10 patients, 3 had metastasis, while others had no metastasis. The 7 patients without metastasis were treated with the Children's Cancer Study Group 7942 (CCG7942) protocol, and the other 3 patients with metastasis with the Pediatric Oncology Group 9354 (POG9354) protocol. The therapeutic response and chemotherapy-related toxicities were evaluated by WHO criteria and Common Terminology Criteria for Adverse Events (version 4.0).

In the 7 patients treated with the CCG7942 protocol, 4 achieved a complete remission (CR), 1 had stable disease, 2 developed progressive disease (PD), and 2 had recurrence. In the 3 patients treated with the POG9354 protocol, 1 achieved a CR, 2 developed PD, 2 had recurrence, and 2 died. For the 7 patients without metastasis, the survival time was 5-60 months, and the event-free survival rate was 71%. For the 3 patients with metastasis, the survival time was 13-25 months, and the event-free survival rate was 33%. All patients developed grade 4 bone marrow suppression, and other grade 1-2 toxicities, including gastrointestinal reactions, liver function impairment, and renal function impairment, were also seen.

CCG7942 protocol is effective and safe for children with non-metastatic pPNET. However, POG9354 protocol has unsatisfactory efficacy in children with metastatic pPNET, so further studies are needed to improve the therapy for this disease.