Many times when families see me, they ask what therapies they should try for their child. Unfortunately, there is no absolutely prescribed therapy or set of therapies for any child on the autism spectrum. Wouldn’t it be great if an easy roadmap to therapy existed in the dizzying world of therapy for children with autism? Wouldn’t it be great if the answers for how to treat a child (or adult) with autism were as easy as using an antibiotic for strep throat? Unfortunately children with autism spectrum disorders are so varied and their symptoms and problems are so diverse that choosing a single or many therapies is daunting.
Many of my colleagues at the Seattle Children’s Autism Center have written blog posts on a variety of approaches, such as Applied Behavioral Analysis and Social Skills Therapy. As a Developmental Pediatrician, a medical doctor, I’m asked another set of questions. What sort of “biomedical therapies” should a parent choose? The term “biomedical” is widely used, but often not clearly defined. Usually what parents mean is a treatment that involves drugs, or diets, or supplements. The presumption in these therapies is that, like treating strep throat, there is a substance, something one swallows, or applies to the body, that can modify the symptoms or improve function. There are numerous such therapies already suggested and new ones likely to appear in the future. How can one choose? What guidelines can a parent think about to help them decide?
I’ve thought about this topic quite often and here is how I look at this question. In this post I don’t intend to give my opinion on each and every therapy. There are too many and such decisions should be based on each individual person who is affected by autism. Nevertheless I think there are some specific issues one should be considered. Here are the ones I think most important:
- Placebo effect
Risk– Every therapy has potential risk. Penicillin is a life saving therapy that is remarkable because is it only chemically interacts with bacterial cell walls, not any portion of human cells. Nevertheless it has risks, as some people can be fatally allergic to this group of medications. Special diets like the popular gluten/casein free diet usually have little risk except such diets, improperly administered, can have dietary deficiencies, especially protein.
Benefit–Risk must always be balanced against benefit. Appendectomy is a treatment for appendicitis. It has defined risks and benefits and almost always (never “always” in medicine) its benefits outweigh its risks (unless the person taking out your appendix doesn’t know how to do it). Benefits may be assessed by research studies that show that this treatment, compared to other treatments, shows beneficial outcomes a certain percent of time. Nevertheless, if there is no research, or if the reported benefits are not compared to some sort of control group, (e.g. another therapy or no therapy) then the benefit is quite hard to judge. Sometimes benefit is described in terms of personal story. This “testimonial” evidence should be viewed cautiously, as it is highly subjective. Remember also that benefits can be large or quite modest. When trying to decide on a new therapy, try to find out from realistic sources how much benefit this might be for your child.
Evidence– Evidence is tightly linked to risk and benefit. Evidence is one way to quantify risk and benefit. “Evidence–based care” is an increasingly heard buzzword in health care these days. And it deserves to be. For long periods in health care history, decisions on therapies were based either on how you were taught or personal experience. These strategies often proved to not provide the best outcome, so over time more rigorous standards for grading evidence studies have evolved.
A study that reported 15 cases of strep throat being successfully treated by “Latest-and-greatest-cillin” wouldn’t impress me unless the study told me how “Latest-and-greatest-cillin” compared to plain old cheap penicillin. Comparison groups, trials blinded to the study group to prevent bias, and large studies always have greater evidence validity than case series which are at best another form of testimonial. If possible see if there is an evidence-base to support the risk and benefits of a therapy. You’ll unfortunately be sad to find out there is often little evidence for many of the treatments promoted for treatment of autism.
Plausibility– I can remember once someone told me that if I put a razor underneath a perfect pyramid the blade would get sharper. I recently told that story to a very knowledgeable physician colleague. She said “really?” Now come on, does this sound in any way plausible? Frankly, it did to me the first time I heard it. I wonder if we all instinctively want to believe in strange and unusual ideas.
In the early days of the increase in autism, there were a string of very bizarre ideas for treatment on the Internet. One in particular caught my eye. It was called the “ADAM” technology. “ADAM” stands for “Aphysical Dimensional Access Manager”. The inventor, dressed in a white lab coat, is pictured sitting in front of a computer screen with some odd looking machinery nearby. The idea was that after you paid a certain amount of $$ to this firm, they would treat your child over the internet by having her sit in front of the computer and her emotional and cognitive state would be felt by the “operator” through the “ether”. Per their literature “…you will receive communication through your sub-conscious mind because a dimensional rift in the mind is created. Through this, heavy duty power information of an enlightened body is passed to your mind causing physical improvement to your minds psychic ability and body.” Hummmm!!
Plausible? I think not. But someone might. Plausibility is slippery. Something is mysterious about pyramids, right? Nevertheless in hard light of “reality”, some ideas sound reasonable, “plausible”, and others not. When thimerosal was first proposed as a cause of autism the Institute of Medicine said that the biologic explanation of a mercury substance causing autism was mildly plausible. Nevertheless, plausibility was tested with reality, as numerous studies showed no connection between thimerosal and autism. In fact, it is notable that since thimerosal was removed from pediatric immunizations (with very uncommon exception) since 2000, the prevalence of autism continues to rise. How could less thimerosal cause more autism? Plausibility always needs to be tempered with evidence if it can be found.
Placebo– We humans have a need to have things work for us. I think at heart, we all are optimists and our hopes sometime interfere with our logic. The placebo effect is an excellent example of this. What has been true with multiple treatment trials with behavioral medications such as Ritalin, Prozac and others is that about 20-30% of people improve on a placebo (non-active sham medicine) versus the active drug being studied. How can we explain these findings? The placebo effect, the tendency to improve when we think we are being treated with something powerful, is a strong human behavior. Given that placebo effect is quite strong in behavioral treatments, a therapy needs to be tested against a non-active control in order to really understand if it is effective.
The best studies of medications to treat behavioral disorders such as stimulants (Ritalin and others) for ADHD and Risperdal for severe autism symptoms have been done in such a placebo-controlled fashion. That is, effects of those medicines are measured against a placebo substance to control for the tendency we have to see a benefit when we take something. Ideally all treatments should be studied with a control group that doesn’t receive the therapy to minimize the placebo effect.
So where does this leave a confused parent or even a confused developmental pediatrician? My advice is to try to do research on whatever therapy you are looking at. See if benefits and risks are defined. See if there is evidence to measure benefit and risk. Think for yourself if the therapy is plausible. Remember that all treatments have placebo effects, which can be quite strong.
In many cases there are no measures of risk, benefit, evidence, plausibility and placebo effect. In that case consider the following:
- -If a treatment has low risk, possible benefit and is somewhat plausible, then it might be worth trying.
- -Remember there are “costs” to everything we do. Vitamins may be inexpensive, but taking multiple supplements may take enough time and energy, as well as $$$, to limit doing something else.
- -Test therapies periodically to see if they are really doing anything.
- -Ask questions and beware of testimonials. Staff at the Seattle Children’s Autism Center and other facilities are happy to try to answer these questions.
- -Try new things even if unproven, if by doing so you feel they have no risk and you can “afford” them.
- -Never stop trying to help your children be the best they can be. We won’t stop trying as well.