Wednesday, May 6, 2009

Neuroblastoma Heads Up

I am sure this entry will make plenty of people pretty mad but, to be fair, it is a subject that drives me absolutely crazy. There are several parents out there in the neuroblastoma world that are touting new drug combinations for relapsed and refractory children. I am bothered by this because so many of these combinations have little support for efficacy in kids with neuroblastoma. None the less, because of the hype (and false hope), these therapies are being chosen over known treatments that we know have an effect in children with neuroblastoma. In many cases, these choices end up preventing kids from getting the very therapies that have the absolute best chance at getting kiddos into a sustained remission. There is a time and a place for "last ditch effort" testing but it is not for someone that is newly relapsed or even a child that still has options with some of the most tried and true relapsed and refractory therapies.

Somehow with all of this hype these new options have become leading relapse choices for parents. They claim fantastic responses but I am yet to see one who has had a response that would be considered "real" in the medical world. I am yet to see a response that is any better than standard relapse chemotherapy. Families are choosing these unproven combinations over therapies which we know have produced good results and in many cases have put kids back into remission. For example, I can't tell you how many kids I have seen achieve a second (or first) remission with MIBG therapy and / or antibodies - but, there have been many. These are all relapsed and refractory kiddos - the worst of the worst. Furthermore, I have also seen kids maintain years of remission after refractory or relapsed disease with all different kinds of low dose agents that are available and have proven track records. Most importantly, these are all drugs with a response history in kids with neuroblastoma. These trials have parents with children who can talk to you and tell you about their experience.

Maximize your options!
  • Choose therapies and options with the highest response rates before jumping into the unknown.
  • Be conscious of the toxicities - known and unknown.
  • Consider the impact and recovery of the marrow from every regimen. Sometimes a high dose therapy can be appropriate if it provides the possibility of sparing the marrow in the future.
  • Be critical of everything you hear. Verify responses. Make sure they are real.
  • Find the successes. They are out there.
I have no intention of telling you what to do but, as another parent, I ask you to look at these things logically. When you talk to other parents consider the successes that they had. Go to the source. Talk to the successes. Learn about the preclinical research and, where applicable, look at the response rates of other kids (make sure they are real - minor changes in HVA/VMA are not considered real responses in the medical world unless they are extremely large differences or confirmed by another method). When you look at combinations of drugs (even known ones like topotecan and cytoxan) that are adding other experimental drugs make sure their response rates are at least as good as the combination alone and not worse than the same combination with another drug.

Ask questions. Look everywhere.

Do it with purpose.

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