On September 30, 2010 an incredibly important article for children with neuroblastoma was finally published in the New England Journal of Medicine. It was published nearly a year and a half after the safety monitoring committee determined that the study met the criteria for early stopping of the randomization, on the basis of the superiority of immunotherapy over standard therapy with regard to event-free survival for children with high risk neuroblastoma. I have anticipated the publishing of this article with greater fervor than many others as Sydney (yes, our very own super hero) was part of this study. She was a data point back in 2004.
Yes, a long time in coming for such an important piece of information.
The bottom-line of this article is the fact that a phase 3 trial (the gold standard for pediatric cancer research) showed that immunotherapy with ch14.18, GM-CSF, and interleukin-2 was associated with a significantly improved outcome as compared with standard therapy in patients
with high-risk neuroblastoma. More specifically, it showed a 2 year 20% increase in event free survival (66±5% vs. 46±5% at 2 years, P = 0.01) for children that received immunotherapy with this combination of drugs. It is also important to mention that it also showed an 11% increase in 2 year overall survival (86±4% vs. 75±5% at 2 years, P = 0.02 without adjustment for interim analyses) although this latter statistic had yet to meet the criteria to be considered statistically significant at the time of study stoppage.
I could literally write for weeks about interesting findings in this article. In my mind, it was literally chock full. As expected, it delivered on a thorough discussion of survival as it related to treatment both with and without immunotherapy, but it also included some incredibly interesting information on prognostic factors. As one would anticipate it also delivered on a thorough discussion regarding the toxicity of immunotherapy. Ironically this differed significantly from my perception, even given the 100s of kiddos that I have seen endure immunotherapy. It was funny in that I felt that the reported number of side effects were far less than I had anticipated and, yet, the description of those side effects was far worse than I expected. This all warrants further discussion and much more time and space than I can encompass in one blog entry. For that reason, over the next few weeks, I will plan on discussing several of these items in greater detail in their very own dedicated entries.
In the meantime, this is one of those articles that are worth the read. It should solidify your opinion that antibody therapy is the right way to go, it should give you some interesting insight into prognosis, and it should prepare you for what you need to watch out for when your child is undergoing antibody therapy.
This is proven purpose.