By Shreeya Nanda
Research presented at the American Society of Clinical Oncology annual conference suggests that consolidation therapy with tandem compared with single myeloablative autologous stem cell transplant (ASCT) can improve outcomes in paediatric patients with high-risk neuroblastoma.
However, Julie Park (Seattle Children's Hospital and University of Washington School of Medicine, USA), who presented the findings in Chicago, Illinois, USA, on behalf of the Children's Oncology Group, added the caveat that by the nature of the trial design this benefit is limited to "patients who survive induction without disease progression or severe induction-related toxicity".
In this phase III trial, patients with a new diagnosis of high-risk neuroblastoma initially received six cycles of induction chemotherapy. Subsequently, those without disease progression and with sufficient peripheral blood stem cell collection were randomly assigned to one of two treatment arms. One group received consolidation with one round of ASCT alongside carboplatin, etoposide plus melphalan (CEM) chemotherapy. And the other was given two rounds of ASCT, where the first round was preceded by cyclophosphamide plus thiotepa treatment and the second by a dose-modified version of the CEM regimen.
Three-year event-free survival (EFS) rates were significantly higher for the 176 children in the tandem ASCT treatment arm than for the 179 in the single ASCT arm, at 61.4% and 48.4%, respectively.
Overall survival (OS) was comparable between groups; however, Park pointed out that the trial was not powered to detect an OS difference.
Participants in both arms were eligible to enrol in trials evaluating post-consolidation anti-GD2 directed immunotherapy, and approximately 70% of enrolled patients did so. When just these patients were considered, both EFS and OS were significantly improved in the tandem ASCT (n=120) relative to the single ASCT (n=126) arm, with 3-year rates of 73.7% versus 56.0% and 83.7% versus 74.4%, respectively.
Park reported that tandem ASCT did not significantly increase the toxicity or regimen-related mortality. The most common grade 3 or 4 adverse events - namely, infections, mucosal and hepatic toxicities, including sinusoidal obstructive disorder - occurred at a similar rate in both groups. This was also the case for mortality rates, which were comparable at 1.2% and 4.1%, respectively.
The presenter concluded that tandem ASCT consolidation improves outcomes in this patient population with a poor prognosis, with even patients who subsequently receive immunotherapy benefiting from tandem ASCT.
"This finding will change the way we treat children with high-risk neuroblastoma in North America, which still claims many young lives and is in urgent need of better treatments", she told the press.
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