Excellent survival outcomes and high response rates were associated with a regimen of neoadjuvant chemotherapy with or without second-look surgery before cranial irradiation in children with newly diagnosed nongerminomatous germ cell tumors.
The regimen should be included as a backbone for further studies, according to results from a phase 2 trial released online ahead of print in the Journal of Clinical Oncology.
In this trial, patients were treated with induction chemotherapy, which consisted of six cycles of carboplatin/etoposide alternating with ifosfamide/etoposide. Those who demonstrated less than complete response were encouraged to undergo second-look surgery.
Those who did not demonstrate complete or partial response with or without second-look surgery were administered high-dose chemotherapy, which consisted of thiotepa and etoposide and autologous peripheral blood stem-cell rescue before craniospinal irradiation.
There were 102 patients in the study, treated between January 2004 and July 2008. The median age was 12 years; 76% were male; 53.9% had pineal region masses and 23.5% had suprasellar lesions.
Of those who received neoadjuvant therapy, 69% achieved complete or partial response; 5-year event-free survival was 84% ± 4% (SE); and overall survival was 93% ± 3%.
At a median follow-up of 5.1 years, 16 patients recurred or progressed, and seven died after relapse. None of the deaths were related to treatment.
RELATED: MicroRNA miR-371a-3p Levels a Promising Biomarker in Testicular Germ Cell Tumors
Ten patients experienced relapse at the site of primary disease and three at a distant site. One patient experienced relapse at both.
Progression was detected by marker increase in two patients. Increased serum alpha-fetoprotein was a negative prognostic value, whereas histologic subtype and increased beta-human chorionic gonadotropin were not signifinantly linked with negative outcomes.
- Goldman S, Bouffet E, Fisher PG, et al. Phase II trial assessing the ability of neoadjuvant chemotherapy with or without second-look surgery to eliminate measurable disease for nongerminomatous germ cell tumors: a Children’s Oncology Group study. J Clin Oncol. 2015. [epub ahead of print]. doi: 10.1200/JCO.2014.59.5132.