Sunday, October 26, 2008

Children & Clinical Studies.

A topic for which I feel extremely strongly is that of research pertaining to children's health. Luckily the NIH is beginning to come around and started a new children's health research initiative and website. They have a wonderful video on the main page that secretly made me get a little teary eyed.

We have neglected the safety and interests of children for long enough. It is time that we make sure that what we do as treatments is safe and effective. We have long assumed that just because it works in adults, it works in children, too. Evidence to the contrary is emerging at an astonishing rate.

From the website are the reasons why we MUST do research in children:
  • "Understand the differences in children as they grow and develop.
  • Identify the best dose of medicines to prevent harmful effects or under-treatment.
  • Produce chewables, liquids or tablets that are easier for children to take.
  • Find treatments for problems that occur only in children, like prematurity.
  • Find treatments for certain diseases or conditions that occur in both children and adults but which act differently in children and adults, like arthritis or heart disease.
  • Understand how medicines are used in and filtered out of the body in children of all ages.
  • Find treatments for new or existing diseases to improve the health of children in the future, like vaccine studies that were done years ago help children stay healthier today.
  • Treat our children like children, rather than little adults."


Mächtige Maus said...

Good deal that this is finally being addressed. It seems such an obvious thing. We've known almost all along how adults respond differently to treatments. To date, I've not found a single pain medication (save for morphine) that works on me as it is supposed to...which makes for a hard task when it comes to pain management.

If my body doesn't respond in the same manner as the 37 year-old sitting right next to me, why on earth would a child's body respond the same way.

So, Loup...the research is starting up. How easy do you think it is going to be to eventually learn to tailor treatment methods to an individual, child or adult?

Habladora said...

Intriguing post, and clearly an important topic. Parents will be relieved to know that more thought is being given to the ways in which children of different ages respond to treatments. As a layperson, I'm curious to know how ethical clinical trials can be done on children - adults can give their consent, but children can't be expected to understand the risks and benefits so it will be up to the parents. I guess this is why we've been slow to acknowledge that treatments approved for adults can't simply be approved for children as well. As a doctor, what are your thoughts, Loup?

Again, thanks for posting on this topic - I'm looking forward to seeing the video that made you bawl.

Another Anonymous Poster said...

MM -

Since Loup is out of town, I'll try my best to answer.

In the long run, personally tailored treatments will come down to genetic analysis. We'll eventually figure out how to correlate genotypes in each of us to drugs that do or do not work. Then it'll just be a matter of getting one's genotype and picking the drugs that work.

We already have a test like this for the blood thinner warfarin (coumadin). It's a test for the liver enzymes that metabolize the drug. Know the genotype, and you'll know an approximate starting dose and whether the person will break it down quicker or slower (and thus be more prone to have over- or under-thinned blood). The problem is, with the current technology, it takes a week or two to get the test results back. By that time, the person's usually already therapeutic on the drug. That and insurance won't cover it.

Anonymous said...

I have an initial negative response to clinical trials in children--we never want to experiment on the unwilling.

The problem is, when the treatments we do use in are untested in that population, it basically amounts to poorly controlled trial based on anecdotes, not evidence. Meaning, we are essentially using 'experimental' medicine on kids all the time because virtually none of it has been tested [on kids]. We need to run official trials to show that we are doing good and no harm, rather than treating this way because it's been done this way for the last 50 years.

With children, the decision does ultimately go towards the parents, although as doctors, we should never even give options that may be harmful.

Maus--I think that through clinical trials, we can find treatments that are beneficial to children. I think that funding will be the biggest obstacle, as children have poor lobbying power.

Mächtige Maus said...

Thanks gang...I knew you two would have something for me.

AAP, that is what I was going for. Where are we on the path to genotype analysis? I fear that society may not be all that fond of the concept, but I have to tell you that I feel it just makes sense. One fit for all is simply not feasible, be it child vs. adult, child vs. child, or adult vs. adult. I suppose the insurance aspect of it is even more problematic.

It's good to know that we are at least on that path though.

Not-So-Normal-Mom said...

Very interestng post! Thank you all for your insights.

As a mom, I feel that it is always important to keep up-to-date; however, that doesn't always mean that I have the best options available to me all the time.

I will never forget being a young Army wife stationed in Ft. Bliss. The Army hospital there was the worst military hospital at the time. I brought my 9 month old baby in to the ER with a very high fecer, and after a few hours there, the resident in the ER was ordering a test for Spinal Menangitis, which meant he was going to do a "spinal tap." I was horrified, and insisted on speaking to a pediatrician. About an hour later, a pediatrican came from another floor in the hospital, not looking too happy. She performed a simple test-asked me to stand behind him and call his name, which made him turn his head around to see me, and said "he doesn't have spinal menangitis" as she walked away.

I later found out that all the moms on my street had kids who had been given that test.

I know that, as parents, it is a fine line to walk between trusting the doctors and asking questions. We assume that the doc knows everything and we put the lives of our children in their hands. It's tricky.

Another Anonymous Poster said...


I'm sorry to say, but the resident was right. Until the age of about one year old, looking for a stiff neck (or "nuchal rigidity" in the medical parlance) is an iffy test at best. Babies that young just don't manifest the same symptoms that even older kids do. So just because your baby could turn his head didn't mean that he didn't have meningitis. I'm very glad that he didn't - you were really lucky. But the only way to really rule out meningitis is by doing an LP and analyzing/culturing the fluid.