CNCF Neuroblastoma Conference Part 2

Finally, I have a chance to get back to the conference before it all slips away. Thankfully, most of it will be available on video online. That is, of course, if I ever get the raw video. In the meantime, I can only offer whatever stuck to the sides of the tube that extends between my two ears. The last time I talked about the conference it was all about the transplant discussion. As I mentioned, there was not much that was comparable from one treatment to another. That was a little disappointing for us as parents of children with neuroblastoma but I think we have to realize also that it is not on purpose. The very nature of many of these studies make it impossible to compare. These differences come from the types of patients on trial (chemo sensitive vs. chemo-resistant, NMYC amplified vs. non-NMYC amplified, and responders vs. non-responders), the type of measure (overall survival, progression free survival, event free survival, or response), the amount of time (1 year, 3 years, 5 years), and the type of trial(phase I, phase II, or phase III). There simply is no direct comparison. This is why we have to be careful when making a decision. The key is not always the trial that has the highest percentage. It is critical to understand the differences. A trial with a seemingly low survival percentage (20%) may very well be better than one with a 100% survival rate. I know, I know, it sounds counter intuitive. It sounds wrong. But, it isn't! That statement is right on the money. If you want to compare one treatment to another you have to understand these seemingly subtle differences. It isn't just about the numbers - in fact, it is far from it. And, we have not even begun to weigh in on the toxicities.

Now, last week I kept telling you that even amongst this confounding statistical mess there were some rules that we could clearly discern. I would like to get to one that I felt was important. This one actually came from one of my questions and although my question was leading I was totally surprised to see agreement on the subject. Frankly, I had always felt it personally but I was bit shocked to see it addressed and agreed upon so openly amongst the experts. It is one of those things that I have seen happen all to often at many hospitals across the country. Essentially, my question was: If you have a child with resistant disease to induction should you be considering a single autologous transplant (or even a tandem - which I neglected to mention). There was a resounding "No, probably not." from the panel. Essentially, you should be considering other options at this point. This is not to say that a transplant may not be the answer. But you must be looking outside the box as well. I think this makes a lot of sense and I have always been surprised by those committed to sending kids into transplant with disease. While I have personally seen a few go into transplant with minimal disease and come out with a complete response, the vast majority have not or have relapsed so quickly they had little to fight with. This is just a Dad's view on five years of watching. I use no scientific measure. Although it is not always a perfect outcome I have seen many respond to other therapies and I thought this was a key point to make and question to ask.

When your child still has disease at the end of induction there are still viable options. Believe me, there is a surprisingly large number of patients that do not respond to induction. These kiddos have been successfully treated with other modalities. As a parent (or oncologist) you have to understand the type of disease (solid tumor, bone marrow, or bone disease) and what types of treatments that have historically worked well with that type(s) of disease. It is also very important to take into consideration the health of the child. This may also lead you to make a very different decision. Regardless, I think we clearly saw at the conference that kids with marrow disease clearly responded to antibody therapy and although it was not addressed directly I know of several examples of kids that have also had bone lesions respond. Another therapy that seems to work well in these cases is MIBG (+/-) transplant. I have seen many kids respond, some with solid mass, some with bone disease, and some with marrow. I just think this is important to realize and to soak in. One of my favorite comments that really drove the point home for me was: "If you have given your child chemo for all of these months and he(she) hasn't responded, why would you think that more chemo would cure them?" Although this is a very generalized statement I think it really drives the point home. What we are doing is not working. Let's try something else.

I think another important point that was made during this particular discussion was related to toxicities. Dr. Yanik kept trying to give us some insight into the world of a transplanter. We, as parents, are always concerned about the toxicities that come from infection or organ failure. One of the organs that we rarely consider is the soil of our bone marrow. One of the toxicities that scare transplanters the most is damaging the soil that allows our stem cells to grow. Especially when we are considering multiple transplants or other highly toxic treatments. We run the risk of creating kids that are highly dependent on platelets for extended periods of time. We can do some serious damage. I think this is especially important to consider if you have a child that is unlikely to respond to these types of therapies. That is a significant risk of many of these transplant regimens and not something to take lightly. We must consider the relative risks and rewards for our child with every therapy. We have to be mindful of what has worked and what has not for children that are similar to our own.

Ask questions. Don't except the norm.

And, of course, I am just a Dad - NOT a medical professional. Ask your doctor. More importantly, ask an oncologist that specializes in neuroblastoma treatment.

You are your child's best advocate.

That is our purpose.