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Published on: February 6, 2019
Treatment of young children with CNS-primitive neuroectodermal tumors/pineoblastomas in the prospective multicenter
Carsten Friedrich1, André O von Bueren, Katja von Hoff
1Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Martinistr. 52, D-20246 Hamburg, Germany.
Background:
Especially in young children, primitive neuroectodermal tumors of the central nervous system (CNS-PNET) and pineoblastomas are associated with an unfavorable outcome, and only a few prospective trials have been conducted thus far.
Methods:
From January 2001 through January 2005, 17 eligible children aged <4 years with CNS-PNET not otherwise specified (n = 8), ependymoblastoma (n = 1), or pineoblastoma (n = 8) confirmed by central review were prospectively treated in the trial HIT 2000. In nonmetastatic disease (n = 11), up to 5 postoperative cycles of HIT-SKK systemic multiagent chemotherapy (8 months duration), followed by craniospinal radiotherapy (CSI), were given. In metastatic disease (M1-M3, n = 6), treatment consisted of a shorter induction chemotherapy (2-3 months) with carboplatin and etoposide, followed by tandem high-dose chemotherapy (HDCT) in case of good response to induction. During induction and HDCT, patients received intraventricular methotrexate. CSI was applied to all patients with poor response to induction or residual disease after HDCT and was optional for patients with residual disease before HDCT.
Results:
Five-year event-free survival and overall survival rates ± standard error for all eligible patients were 24% ± 10% and 40% ± 12%, respectively (median follow-up of survivors: 8.3 years). Only one patient with nonmetastatic disease remained free of relapse/progressive disease during induction. Three of 6 patients with metastatic disease responded to induction and received tandem-HDCT, followed by preventive CSI, and remain in continuous complete remission.
Conclusions:
Short intensive induction chemotherapy followed by tandem-HDCT in young children with CNS-PNET/pineoblastomas seems to be superior to the prolonged and less intensive induction regimen.
Insights
Short, intensive chemotherapy followed by high-dose chemotherapy improved outcomes for young children with central nervous system primitive neuroectodermal tumors (CNS-PNET) and pineoblastomas, offering better survival rates.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Clinical Trial Research
Background:
- Central nervous system primitive neuroectodermal tumors (CNS-PNET) and pineoblastomas in young children have poor prognoses.
- Limited prospective trials exist for these aggressive pediatric brain tumors.
Purpose of the Study:
- To evaluate a novel treatment regimen for young children (<4 years) diagnosed with CNS-PNET or pineoblastoma.
- To compare the efficacy of intensive induction chemotherapy followed by tandem high-dose chemotherapy (HDCT) versus traditional prolonged chemotherapy.
Main Methods:
- Prospective trial (HIT 2000) involving 17 children with CNS-PNET, ependymoblastoma, or pineoblastoma.
- Treatment included multiagent chemotherapy, craniospinal radiotherapy (CSI), and intraventricular methotrexate, with variations for metastatic disease and response to induction.
- Metastatic patients received shorter induction chemotherapy with carboplatin/etoposide, potentially followed by tandem HDCT.
Main Results:
- Five-year event-free survival was 24% ± 10%, and overall survival was 40% ± 12%.
- Only one non-metastatic patient remained relapse-free during induction.
- Three of six metastatic patients achieved continuous complete remission after induction chemotherapy, tandem HDCT, and CSI.
Conclusions:
- An intensive induction chemotherapy regimen followed by tandem HDCT appears more effective than prolonged, less intensive regimens for pediatric CNS-PNET/pineoblastoma.
- This approach may offer improved survival outcomes for young children with these challenging brain tumors.
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