The cohort of those with early stuttering.

The term "cohort" refers to a group with a particular characteristic or characteristics who participate in research. There are many cohorts of interest in stuttering research: ones with different genetic histories, different stuttering severities, different psychological profiles, and so on. The cohort of interest in this paper is children younger than 6 years. In this paper I want to present a personal view of what I think science has done for this group.

Why science?

Because I want to talk about what science has done for this cohort, this does not mean that I necessarily think that science is the key to life. Science is only one way of doing things. After all, there are limits to what science will do for us. You won't, for example, find peace, love, or happiness in the output of science.

However, scientific treatment research can bring some benefits. One branch of scientific treatment development--clinical trials--allows scientific pursuit of efficacious treatments (treatments that show positive results), and when those efficacious treatments are found, clinical trials development enables them to be refined by comparing the new treatment to established best practice. In the field of stuttering research, clinical trials enable us researchers to search for ways to find some measure of lasting relief from stuttering. You might bliss in life such as peace, love, and happiness outside the domain of science, but you won't find a blissful treatment for early stuttering. I certainly would not entertain a treatment for stuttering that emerged from somewhere else than science. When a child and parent come for help to our clinic at the Australian Stuttering Research Centre (ASRC), I consider the responsibility too serious to do otherwise.

But can lasting, even blissful, relief from the effects of this disorder in young children be found in the clinical trials output of science? That question is my topic today, and I will give the best answer that I can at this time.

What is a clinical trial?

So first, what is a clinical trial of a stuttering treatment? Recently, my colleagues and myself (Onslow, et al., 2007) have suggested that the clinical trial is the smallest unit of output of stuttering treatment efficacy research that clinicians can usefully interpret. It is the only way for them to find out scientifically how good a treatment is in improving the lives of clients who stutter. We argue that, to be clinically useful, a clinical trial of a stuttering treatment should, among other things, evaluate an entire treatment, and in the case where speech observations are made, those observations should be made on speech beyond the clinic. Without improved speech beyond the clinic, a trial is just not convincing. I, for one, would not consider attempting to use a treatment to alleviate a child's stuttering without evidence that the treatment produced benefits beyond the clinic.

All evidence is not equal. Robey (2005) recently drew attention to how the traditional phases of clinical trial development might be applied to speech pathology treatments. Phase I clinical trials are preliminary investigations into the possibility of a new treatment, conducted on only a few volunteers. The number of participants in Phase I trials are quite few. Phase I trials may be prompted by laboratory evidence indicating a potentially worthwhile treatment. Or they can be prompted simply by a good idea that makes a lot of sense. The primary considerations in such trials are fundamental safety issues with the treatment, and whether the treatment works from the perspective of the client and the service provider. It is also important to determine whether people will comply with the treatment in these trials. It's no use if the treatment works but no- one will put up with it. You don't want the cure to be worse than the disorder, as, I'm sure you will know, has happened more than once during the history of stuttering treatments.

In the event that a Phase I trial shows promise, the researchers can move to a Phase II trial, which involves a few more subjects. The prime considerations during this stage of treatment development are to establish estimates of (1) how many participants who stutter will respond to the treatment, (2) how much the stuttering is reduced, and (3) the "dose" of treatment that is needed. Phase II trials data that are unconvincing on any or all of these points potentially indicate that treatment development should be stopped at this phase. In a worse case scenario, a treatment that produces a small treatment effect in few participants after a long period would be abandoned without question.

Phase III clinical trials evidence is the "gold standard" of evidence for a treatment. It is one or more randomized controlled trials. These trials commonly involve two groups, one receiving the experimental treatment and a control group or comparison group. This strategy is used to see whether a treatment is better than nothing, and how better than nothing it is, which is useful information. However, randomized controlled trials can also be used to see which of two or more treatments is superior.

The good news

The news is good, or at least the scientific news is good, about the treatment of preschool children who stutter. Using the Onslow et al. definition of a clinical trial, there have been 13 clinical trials of treatments for preschoolers. Interestingly, 12 of these have been for verbal response contingent stimulation procedures--treatments where adults say something when a child does or does not stutter. The first of these trials were what could be classified as Phase I trials in the 1970s (Martin et al. 1972; Reed & Godden, 1977), and the more recent are Phase II and Phase III trials. Examples of the latter two are Miller and Guitar (2007) and Jones et al. (2005). These are trials of the Lidcombe Program, which is a particular treatment model for verbal response contingent stimulation. Some of this clinical trials literature about verbal response contingent stimulation contains reasonably compelling follow-up periods. Phase III trials allow us to estimate what the effect size might be. For example, the odds ratio in the Jones et al. Phase III trial was 7.7, suggesting that at 9 months after treatment children who received the Lidcombe Program were 7.7 times more likely to be stuttering below 1.0 %SS during everyday conversations than children who did not receive the Lidcombe Program.

Incidentally, I mentioned 13 trials. What was the 12th? Well, Trajkovski et al. (2006) published a Phase I report of a child who received a treatment involving nonprogrammed instruction in syllable timed speech. Although the results for this single case were intriguing, I don't think we can make much of them at this stage.

Why only verbal response contingent stimulation trials?

Virtually all of the clinical trials reported to date for our young cohort have been trials of verbal response contingent stimulation. I think that says a lot, but I am not sure what it says. Why is it that these treatments are virtually the only ones that have been evaluated in their entirety with speech measures made beyond the clinic? Fortunately, though, there are treatments in the pre-trial stage of development, and we can anticipate that clinical trials of other treatments are available soon. The preliminary report by Yaruss et al. (2006) is a good case in point.

How good is the news for the young cohort?

So, the news is good. There is compelling, replicated evidence that verbal response contingent stimulation is an efficacious treatment for preschoolers who stutter. But wait, there is more! Nature's treatment weighs in also. Natural recovery does not work as often as verbal response contingent stimulation, but it does work. Exactly how often is a discussion for another day, but when it does work it is certainly cheap. So, is that good news the end of it? Stuttering goes away and the problem is fixed? Is it not the case that there may be nonbehavioral features of stuttering during its first years? Surely we can't ignore information that peers of stuttering children recognize stuttered speech and evaluate it negatively in the preschool years (e.g., Ezrati-Vinacour et al., 2001). Surely we cant ignore information that preschoolers may express emotional distress at their stuttering (e.g., Bloodstein, 1995). And surely we cannot ignore negative attitudes to communication that may emerge at the end of the preschool years in stuttering children (e.g., De Nil & Brutten, 1991).

No, obviously we cannot ignore those findings as clinicians. It would be inappropriate not to include them in our clinical practices. But the issue is whether nonbehavioral problems go away when stuttering goes away in preschool children. Is that the case? My answer to that is that there are no scientific findings to suggest otherwise. Further, my answer is that clinical observations made by myself and colleagues at clinics here at the ASRC and the nearby Stuttering Unit in Bankstown Health Service have led us to form the view that there is no reason to believe otherwise.

Blissful relief?

Could science and we be wrong? Yes on both counts. Future research may show residual nonbehavioral problems some years later with stutter-free children who have been treated successfully during the preschool years. And we--my colleagues and myself who made observations of children who have been successfully treated--are not clinical psychologists and something could have been going on that we have been missing. But as I mentioned earlier, this is a personal view, and that is my story, and I am sticking to it. Has the Holy Grail been found for stuttering preschoolers. No, that would be going too far. Has blissful relief from the disorder been found? I would go as far as saying yes. When stuttering goes away during the preschool years after successful treatment--due to the work of a clinician or nature--then the experiences of children and parents are indeed blissful. And, if I may, one of the benefits of being a stuttering treatment researcher is to experience that bliss vicariously. I recall vividly talking to various children years after they participated in clinical trials, and being delighted that they could barely recall why they came for treatment. But this is about them ultimately, not about their parents, or me, or anyone else. Is their relief lasting? That is for science to say, and it will say something about that shortly (Jones et al., 2007). And finally, has science gone as far as it can in providing relief, even blissful relief, for children who stutter and their parents? Is verbal response contingent simulation the only contender for a serious treatment? Surely the answer is inconceivably not.

References

De Nil, L. F., & Brutten, G. J. (1991). Speech-associated attitudes of stuttering and nonstuttering children.[erratum appears in J Speech Hear Res 1991 Jun;34(3):703]. Journal of Speech & Hearing Research, 34, 60-66.

Ezrati-Vincour, R., Platzky, R., & Yairi, E. (2001). The young child's awareness of stuttering-like disfluency. Journal of Speech, Language, and Hearing Research, 44, 368-380.

Jones, M., Onslow, M., Packman, A., Williams, S., Ormond, T., Schwarz, I., et al. (2005). Randomised controlled trial of the Lidcombe programme of early stuttering intervention. British Medical Journal, 331, 659-661.

Jones, M., Hearne, A., Onslow, M., Ormond, T., Williams, S., Schwarz, I., & O'Brian, S. (2007) Extended follow up of a randomised controlled trial of the Lidcombe Program of Early Stuttering Intervention. Manuscript in preparation.

Martin, R. R., Kuhl, P., & Haroldson, S. (1972). An experimental treatment with two preschool stuttering children. Journal of Speech and Hearing Research, 15, 743-752.

Miller, B., & Guitar, B. (2007). Long term outcome of the Lidcombe Program of Early Stuttering intervention. Manuscript in preparation.

Onslow, M., Jones, M., O'Brian, S., & Menzies. (2007). Biostatistics for clinicians: Defining, identifying, and evaluating clinical trials of stuttering treatments. Manuscript in preparation.

Reed, C. and A. Godden (1977). "An experimental treatment using verbal punishment with two preschool stutterers." Journal of Fluency Disorders, 2, 225-233. Robey, R. R. (2005). An introduction to clinical trials. ASHA Leader, May 24, 6-7 & 22-23.

Trajkovski, N., Andrews, C., O'Brian, S., Onslow, M., & Packman, A. (2006). Treating stuttering in a preschool child with syllable timed speech: A case study. Behavior Change, 23, 270-277.

Yaruss, I. S., Coleman, C., & Hammer, D. (2006). Treating preschool children who stutter: Description and preliminary evaluation of a family-focused treatment approach. Language Speech and Hearing Services in Schools, 37, 118-132.

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