Transfusion medicine

The study find that the tandem autologous stem-cell transplant (ASCT) may provide better event-free survival (EFS) in patients with high-risk neuroblastoma, compared with single ASCT, a new clinical trial shows. Therefore “Although it was our hypothesis that tandem transplant would decrease risk for recurrence, we were surprised by the positive results, including the magnitude of the difference, and that tandem transplant improved EFS and overall survival (OS) even for those patients who received postcon solidation immunotherapy,” Dr. Julie R. Park from Seattle Children’s Hospital told Reuters Health by email.

Autologous stem-cell transplant

Patients with high risk neuroblastoma are typically treat with multiagent chemotherapy induction and surgical resection; consolidated high dose chemotherapy with ASCT, posttransplant radiotherapy; and postconsolidation treatment with biological agents and immunotherapy. Despite this intensive therapy; 50% to 60% of patients relapse and more than 90% of those who relapse die of the disease.  Dr. Park and colleagues from 142 Children’s Oncology Group centers in five countries investigated whether intensifying consolidation treatment with tandem transplant ;(two transplants six to 10 weeks apart) could improve EFS versus a single transplant in their study of 355 patients with high-risk neuroblastoma.

Among the 652 patients who had been eligible for randomization, the three-year EFS was 51.1%. The three-year EFS from the time of randomization was 54.9% for the 355 enrolled patients. The three-year EFS from the time of randomization was significantly higher in the tandem-transplant group than in the single-transplant group (61.6% vs. 48.4%, P=0.006); the team reports in the August 27 issue of JAMA. There were 17 deaths due to toxicity (seven during induction and 10 during consolidation therapy). The three-year OS from the time of randomization did not differ significantly between the tandem transplant group (74.1%); and the single transplant group (69.1%).

Eligible for randomization

Among patients who went on to receive isotretinoin plus anti-GD2 chimeric antibody and cytokines (immunotherapy) in two other trials; three-year ESS and OS from the time of initiating immunotherapy were significantly higher in the tandem transplant group (73.3% and 84.0%, respectively) than in the single transplant group ;(54.7% and 73.5%, respectively). “Patients who meet the criteria outlined in our article (adequate organ function; no evidence of progressive disease) should be consider for tandem transplant using the regimen we report;” Dr. Park said.

“Intensification of therapy beyond induction therapy is require to achieve the best state of minimal residual disease to enter postconsolidation immunotherapy.” Because “Therapy for high-risk neuroblastoma is intense and places patients at risk for late effects of therapy. While we are please with the continue improvement in survival for these patients; but it is paramount that we continue research into novel targeted therapies (molecular or immunological) that will improve efficacy and hopefully limit late effects.”