Philosophy(PhD) / This thesis presents a randomised controlled trial of the Lidcombe Program of Early Stuttering Intervention. The Lidcombe Program was developed for the treatment of stuttering in preschool-age children. The effectiveness of the Lidcombe Program was compared to a control group in a parallel group randomised controlled trial with blinded outcome assessment. A number of supplementary studies were conducted in support of the trial; two literature reviews, two retrospective file audits and a statistical simulation study. A review of randomised studies of treatments for stuttering showed that there have been 27 such studies published in English language journals. Of these only one was devoted to a treatment for early stuttering and that was the Lidcombe Program. The randomised study showed that 3 months of this treatment was associated with a lower level of stuttering compared to a control group who received no treatment. However, with a sample size of 23, this study lacked power and the children did not receive a full course of treatment. Despite these limitations, this study provided evidence that a medium to large effect size could be anticipated in an adequately powered and properly conducted randomised controlled trial. The second review was of sample size and power in stuttering research studies that had been published in two speech pathology journals; the Journal of Speech, Language and Hearing Research (Vol 39, No. 1 to Vol 40, No, 4) and the Journal of Fluency Disorders (Vol 21, No. 1 to Vol 22, No, 3). Results suggested that the majority (73%) of the 26 studies reviewed were insufficiently powered to detect even large effects. However it was acknowledged that it is very difficult to recruit even moderate sample sizes of people who stutter. It was concluded that one way to help improve this situation is collaboration of multiple research centres or, in the case of a randomised controlled trial, inclusion of multiple recruitment sites in one study. This strategy was adopted in the randomised controlled trial reported in this thesis. Two retrospective file audit studies of children treated with the Lidcombe Program were conducted in Australia and Britain. One purpose of these file audits was to obtain information relevant to the design and conduct of the randomised controlled trial. Data from the case reports on more than 300 children from the two sites were included in a meta-analysis. Results showed that a median of 11 weekly clinic sessions were required for children to attain the criteria for low levels of stuttering for completion of Stage 1 of the Lidcombe Program. Approximately 90% of children had achieved those criteria within 6 months of beginning treatment and almost all children had achieved them within 1 year. In addition two variables were found to be associated with longer treatment duration: more severe pre-treatment stuttering and shorter times from onset of stuttering to the start of treatment. The latter was apparent in the meta-analysis but not for the individual cohorts. As a result of these findings, pre-treatment stuttering severity was stratified along with other relevant variables in the randomised controlled trial and follow up for participants was a minimum of 9 months. A simulation study was conducted prior to analysis of data from the primary outcome measure of the randomised controlled trial: percentage of syllables stuttered (%SS). The distribution of %SS scores is positively skewed. Nonetheless, simulation showed t-test to be an appropriate analysis for this primary outcome measure. There were two treatment sites for the randomised controlled trial: the University of Canterbury (Christchurch, New Zealand) and the Stuttering Treatment and Research Trust (Auckland, New Zealand). A total of 54 preschool-age children were recruited: 29 to the Lidcombe Program and 25 to the control group. Half the proposed sample size was achieved due to slower than anticipated recruitment. This occurred because, as the trial progressed, treatment with the Lidcombe Program became common knowledge among parents in New Zealand and they became increasingly reluctant to agree to have their child randomised to the trial. Analysis with t-test showed a highly statistically significant difference (p = 0.003) at 9-months post-randomisation. The mean percentage of syllables stuttered (%SS) at 9-months post-randomisation was 1.5 (SD = 1.4) for the Lidcombe Program group compared to 3.9 (SD = 3.5) for the control group, resulting in a treatment effect of 2.3 %SS (95% confidence interval: 0.8-3.9). This treatment effect was more than double the minimum clinically worthwhile difference specified in the trial protocol. These results show that the Lidcombe Program is significantly more effective than natural recovery for reducing stuttering levels in preschool children. The Lidcombe Program is the first early stuttering treatment to be shown to be more effective than natural recovery in a randomised controlled trial.

Reflexology is one of the top six complementary therapies used in the UK. Reflexologists claim that massage to specific points of the feet increases blood supply to referred or 'mapped' organs in the body. Empirical evidence to validate this claim is scarce. This three-phase RCT measured changes in haemodynamic parameters in subjects receiving reflexology treatment applied to specific areas of the foot which are thought to correspond to the heart (intervention) compared with reflexology applied to other areas on the foot which are not (control).

Childhood obesity is associated with serious physiological and psychological consequences including type 2 diabetes, higher rates of depression and low self-esteem. With the population of overweight and obese youth increasing, appropriate interventions are needed that speak to the issue of motivation to maintain adherence to healthy behavior changes. The current investigation was a randomized controlled trial examining the efficacy of Motivational Interviewing (MI) as an intervention for promoting self-efficacy and weight-loss in a sample of overweight and obese youth. Participants (N = 42) ages 10-18, were randomly assigned to a control (social skills training) or treatment (MI) group. Both groups received individual therapy (~30 minutes/month) in addition to usual care of diet/exercise counseling. Pre and post (at 6 months follow-up) variables included measures of self-efficacy and anthropometrics. Results indicated that while significant between-group differences were not found, individuals in the MI group attended more sessions. Overall, participants in both groups showed significant increases in self-efficacy and a trend of decreased BMI z-scores. Though health benefits from participation in individual therapy may have been accrued, specific benefits attributable to MI were limited. Findings from the current study suggest that more than one type of counseling intervention (i.e., MI and social skills training) may be beneficial when providing integrative treatment for obese youth. / Canadian Institutes of Health and Research (CIHR)

Accidental injury is a common experience for children (ABS, 2004-2005), yet the debilitating psychological effects of the trauma event often go undetected and thus not treated. Whilst for many the accident will result in some distress, many children will naturally recover and move on (Bryant, 2004). In spite of this, 10-18% of children will continue to experience persistent chronic stress symptoms for up to 2 years post accident (Le Brocque, Hendrikz, & Kenardy, 2009). Unfortunately, little psychological assistance is available or offered to children that may assist in their psychological recovery and even prevent the significant and debilitating effects of psychological distress. Therefore, the aim of this research was to develop and investigate an intervention for children following accidental injury. This was carried out via three stages and presented as three sections within the thesis. Section 1 aimed to develop a theory driven and evidenced-based intervention. Firstly, a meta-analysis was conducted to identify potential risk factors of significance that predict post-traumatic stress symptoms. This analysis resulted in pre-trauma psychopathology, threat to life, gender (female) and parental distress being relatively strong and consistent predictors. This information helps inform potential screening tools as well as directly guidinge the development of the intervention for this thesis. Secondly, this section conducted a comprehensive literature review of relevant approaches and interventions to help guide the development of the intervention. This review drew on models of traumatic stress development such as the Resilience Framework (Kumpfer, 1999), and Paediatric Medical Traumatic Stress (Kazak et al., 2006) as well as preventative frameworks such as the Paediatric Psychosocial Preventative Health Model (Kazak, 2006) that resulted in a Universal approach being argued as the most appropriate. Following this, a review was conducted of relevant universal preventative interventions currently available or recommended following trauma including, Psychological Debriefing (PD), Psychological First Aid (PFA) and information provision. Overall it was concluded that an information provision intervention that drew on strategies used within PD and PFA (such as normalisation and coping strategies) was the most appropriate and practical approach to offering psychological assistance to children and their parents following accidental injury. Finally, this section culminated in a chapter outlining the design, delivery and content of the intervention. The intervention developed aimed to normalise reactions and enhance coping in children following the accident whilst providing parents with accurate information regarding trauma reactions and how they could assist their child’s emotional recovery. Of importance was the decision to place the child’s intervention on the internet. It was argued that this would offer a more child friendly medium and provide greater and easier access for families. Section 2 encompassed the evaluation of the intervention developed. Children (7-16years) and their parents were recruited from the Royal Children’s Hospital and their trauma reactions were assessed over a 6 month period, with three assessment time points; Time 1 (baseline, within 1-2 weeks of the accident), Time 2 (4-6 weeks) and Time 3 (6 months). Children and their parents were randomised into the intervention group following Time 1. Analyses revealed that children within the intervention group reported significantly decreased anxiety, in comparison to a worsening of symptoms for children in the control group. No significant group differences were noted for the parents. Exploratory analysis conducted on high risk children (those with high initial distress) revealed that those in the control group exhibited a significant worsening of anxiety and social phobias over the 6 months. This significant increase in symptoms was not noted for children who had received the intervention. Furthermore, exploratory analyses revealed that children who initially reported high symptoms were the ones most likely to find the intervention helpful and effective. This result was exhibited in the qualitative responses from the parents and children. Overall, the intervention showed promise and encouraging results in its ability to aid child recovery. Furthermore, the results indicated that high risk children may benefit over and above all those recruited suggested the possible appropriateness for a stepped care approach. Section 3 was conducted in light of the results of Section 2, suggesting that children with high initial distress may benefit more greatly from the intervention compared to those with low initial distress. Therefore, this section examined a number of risk factors identified via the meta-analysis conducted in Section 1, to examine whether they would moderate a child’s and parent’s trauma symptoms following treatment compared with no treatment. Thus, this section investigated whether the intervention would be best delivered universally or targeted at children and parents who exhibited certain risk factors. Results indicated that when the child risk factors of initial child distress, attribution of self-blame and parental psychopathology were present, children in the control group had significantly higher trauma symptoms in comparison to those in the intervention. When these factors were not present, there were no significant group differences. For parents, their initial post-accident depression, anxiety and stress levels moderated treatment; in that when it was not present (low) there were no group differences, however when the risk factors was present (high) those in the control group exhibited significantly higher mood disturbances at 6 months. The findings of this section resulted in the conclusion that there are a number of risk factors that impact or influence treatment, thus suggesting that the intervention could be best utilised when targeted at those presenting with risk factors. Therefore, this research developed, investigated and optimised a universal preventative intervention for children and their parents following accidental injury. The limitations and implications for this research are discussed, including debate regarding a universal versus a targeted approach to intervention delivery.

Philosophy(PhD) / This thesis presents a randomised controlled trial of the Lidcombe Program of Early Stuttering Intervention. The Lidcombe Program was developed for the treatment of stuttering in preschool-age children. The effectiveness of the Lidcombe Program was compared to a control group in a parallel group randomised controlled trial with blinded outcome assessment. A number of supplementary studies were conducted in support of the trial; two literature reviews, two retrospective file audits and a statistical simulation study. A review of randomised studies of treatments for stuttering showed that there have been 27 such studies published in English language journals. Of these only one was devoted to a treatment for early stuttering and that was the Lidcombe Program. The randomised study showed that 3 months of this treatment was associated with a lower level of stuttering compared to a control group who received no treatment. However, with a sample size of 23, this study lacked power and the children did not receive a full course of treatment. Despite these limitations, this study provided evidence that a medium to large effect size could be anticipated in an adequately powered and properly conducted randomised controlled trial. The second review was of sample size and power in stuttering research studies that had been published in two speech pathology journals; the Journal of Speech, Language and Hearing Research (Vol 39, No. 1 to Vol 40, No, 4) and the Journal of Fluency Disorders (Vol 21, No. 1 to Vol 22, No, 3). Results suggested that the majority (73%) of the 26 studies reviewed were insufficiently powered to detect even large effects. However it was acknowledged that it is very difficult to recruit even moderate sample sizes of people who stutter. It was concluded that one way to help improve this situation is collaboration of multiple research centres or, in the case of a randomised controlled trial, inclusion of multiple recruitment sites in one study. This strategy was adopted in the randomised controlled trial reported in this thesis. Two retrospective file audit studies of children treated with the Lidcombe Program were conducted in Australia and Britain. One purpose of these file audits was to obtain information relevant to the design and conduct of the randomised controlled trial. Data from the case reports on more than 300 children from the two sites were included in a meta-analysis. Results showed that a median of 11 weekly clinic sessions were required for children to attain the criteria for low levels of stuttering for completion of Stage 1 of the Lidcombe Program. Approximately 90% of children had achieved those criteria within 6 months of beginning treatment and almost all children had achieved them within 1 year. In addition two variables were found to be associated with longer treatment duration: more severe pre-treatment stuttering and shorter times from onset of stuttering to the start of treatment. The latter was apparent in the meta-analysis but not for the individual cohorts. As a result of these findings, pre-treatment stuttering severity was stratified along with other relevant variables in the randomised controlled trial and follow up for participants was a minimum of 9 months. A simulation study was conducted prior to analysis of data from the primary outcome measure of the randomised controlled trial: percentage of syllables stuttered (%SS). The distribution of %SS scores is positively skewed. Nonetheless, simulation showed t-test to be an appropriate analysis for this primary outcome measure. There were two treatment sites for the randomised controlled trial: the University of Canterbury (Christchurch, New Zealand) and the Stuttering Treatment and Research Trust (Auckland, New Zealand). A total of 54 preschool-age children were recruited: 29 to the Lidcombe Program and 25 to the control group. Half the proposed sample size was achieved due to slower than anticipated recruitment. This occurred because, as the trial progressed, treatment with the Lidcombe Program became common knowledge among parents in New Zealand and they became increasingly reluctant to agree to have their child randomised to the trial. Analysis with t-test showed a highly statistically significant difference (p = 0.003) at 9-months post-randomisation. The mean percentage of syllables stuttered (%SS) at 9-months post-randomisation was 1.5 (SD = 1.4) for the Lidcombe Program group compared to 3.9 (SD = 3.5) for the control group, resulting in a treatment effect of 2.3 %SS (95% confidence interval: 0.8-3.9). This treatment effect was more than double the minimum clinically worthwhile difference specified in the trial protocol. These results show that the Lidcombe Program is significantly more effective than natural recovery for reducing stuttering levels in preschool children. The Lidcombe Program is the first early stuttering treatment to be shown to be more effective than natural recovery in a randomised controlled trial.

abstract: Cancer survivors engaged in either six-week Internet-delivered mindfulness training or a usual-care control and were compared on the following outcome battery: The Hospital Anxiety and Depression Scale, the Profile of Mood States, the Pittsburgh Sleep Quality Index, and the Fatigue Symptom Inventory. Assessments were conducted before and after treatment and intervention compliance was monitored. Mindfulness treatments were delivered at a time and on a computer of the participants’ choosing. Multivariate analysis indicated that mindfulness training produced significant benefits on all measures (p < .05). Online mindfulness instruction represents a widely-accessible, cost-effective intervention for reducing psychological distress and its behavioral manifestations in cancer survivors, especially those who are unable to participate in in-person training. / Dissertation/Thesis / Doctoral Dissertation Counseling Psychology 2017

京都大学 / 新制・論文博士 / 博士(社会健康医学) / 乙第13422号 / 論社医博第16号 / 新制||社医||11(附属図書館) / 京都大学大学院医学研究科社会健康医学系専攻 / (主査)教授 佐藤 俊哉, 教授 藤渕 航, 教授 黒田 知宏 / 学位規則第4条第2項該当 / Doctor of Public Health / Kyoto University / DFAM

Importance: Evidence from randomized controlled trials (RCTs) is required to guide treatment of critically ill children. Unfortunately such evidence is not always available. Objectives: To describe the RCT research enterprise in pediatric critical care — the evidence and the process of creating it, along with problems and some solutions. Methods: To meet these objectives I undertook a series of 5 related studies. First a scoping review to describe the output of the research enterprise. Second, a social network analysis of coauthorship patterns to describe the community of researchers who produce this evidence. Third, a survey to investigate the importance of RCTs in clinicians’ decision-making. Fourth, a survey of trialists to identify barriers and facilitators of high quality RCTs. Fifth, a qualitative interview study to identify acceptable, feasible and effective strategies to improve the evidence available from RCTs in pediatric critical care. Results and conclusions: The number of RCTs in pediatric critical care is increasing but there is a preponderance of small, single-centred RCTs focusing on laboratory or physiological outcomes that are often stopped early because of feasibility problems or futility. The research community is highly fragmented and highly clustered. Experienced trialists identified approaches to improve the pediatric critical care research enterprise, including building a sense of community and ensuring key training and relevant practical experiences for new investigators. Because of the barriers that researchers face and their ethical obligation to undertake trials that are feasible and make a meaningful contribution to advancing the care of critically ill children, individuals and groups must take an active role in building a healthy research community. Only by changing how we function as a research community can we train the next generation of investigators and undertake the type of trials needed to improve the care of critically ill children. / Thesis / Doctor of Philosophy (PhD) / Evidence from randomized controlled trials (RCTs) is required to guide treatment of critically ill children. Unfortunately such evidence is not always available. My objectives in this thesis are to describe the RCT research enterprise in pediatric critical care — the evidence and the process of creating it, along with problems and some solutions. To meet these objectives I undertook a series of 5 related studies: to identify and describe the RCTs, describe how researchers collaborate, understand how clinicians use RCTs, identify barriers and facilitators of conducting high quality RCTs, and understand how we can improve the evidence available from RCTs in pediatric critical care. We found that the number of RCTs is increasing but there are opportunities to improve the methods, outcome measures, and quality of reporting. We identified strategies that researchers can adopt to facilitate the rigorous RCTs that are needed to improve the care of critically ill children.

Yes / Background: The increasing prevalence and associated cost of treating chronic obstructive pulmonary disease (COPD) is unsustainable, and focus is needed on self-management and prevention of hospital admissions. Telehealth monitoring of patients’ vital signs allows clinicians to prioritise their workload and enables patients to take more responsibility for their health. This paper reports the results of a pilot randomised controlled trial (RCT) of Telehealth-supported care within a community-based COPD supported-discharge service. Methods: A two-arm pragmatic pilot RCT was conducted comparing the standard service with a Telehealth-supported service and assessed the potential for progressing into a full RCT. The co-primary outcome measures were the proportion of COPD patients readmitted to hospital and changes in patients’ self-reported quality of life. The objectives were to assess the suitability of the methodology, produce a sample size calculation for a full RCT, and to give an indication of cost-effectiveness for both pathways. Results: Sixty three participants were recruited (n = 31 Standard; n = 32 Telehealth); 15 participants were excluded from analysis due to inadequate data completion or withdrawal from the Telehealth arm. Recruitment was slow with significant gaps in data collection, due predominantly to an unanticipated 60% reduction of staff capacity within the clinical team. The sample size calculation was guided by estimates of clinically important effects and COPD readmission rates derived from the literature. Descriptive analyses showed that the standard service group had a lower proportion of patients with hospital readmissions and a greater increase in self-reported quality of life compared to the Telehealth-supported group. Telehealth was cost-effective only if hospital admissions data were excluded. Conclusions: Slow recruitment rates and service reconfigurations prevented progression to a full RCT. Although there are advantages to conducting an RCT with data collection conducted by a frontline clinical team, in this case, challenges arose when resources within the team were reduced by external events. Gaps in data collection were resolved by recruiting a research nurse. This study reinforces previous findings regarding the difficulty of undertaking evaluation of complex interventions, and provides recommendations for the introduction and evaluation of complex interventions within clinical settings, such as prioritisation of research within the clinical remit.

Background: It is unclear whether antidepressants can prevent suicides or suicide attempts, particularly during longterm use. Methods: We carried out a comprehensive review of long-term studies of antidepressants (relapse prevention). Sources were obtained from 5 review articles and by searches of MEDLINE, PubMed Central and a hand search of bibliographies. We meta-analyzed placebo-controlled antidepressant RCTs of at least 3 months’ duration and calculated suicide and suicide attempt incidence rates, incidence rate ratios and Peto odds ratios (ORs). Results: Out of 807 studies screened 29 were included, covering 6,934 patients (5,529 patient-years). In total, 1.45 suicides and 2.76 suicide attempts per 1,000 patient-years were reported. Seven out of 8 suicides and 13 out of 14 suicide attempts occurred in antidepressant arms, resulting in incidence rate ratios of 5.03 (0.78–114.1; p = 0.102) for suicides and of 9.02 (1.58–193.6; p = 0.007) for suicide attempts. Peto ORs were 2.6 (0.6–11.2; nonsignificant) and 3.4 (1.1–11.0; p = 0.04), respectively. Dropouts due to unknown reasons were similar in the antidepressant and placebo arms (9.6 vs. 9.9%). The majority of suicides and suicide attempts originated from 1 study, accounting for a fifth of all patient-years in this meta-analysis. Leaving out this study resulted in a nonsignificant incidence rate ratio for suicide attempts of 3.83 (0.53–91.01). Conclusions: Therapists should be aware of the lack of proof from RCTs that antidepressants prevent suicides and suicide attempts. We cannot conclude with certainty whether antidepressants increase the risk for suicide or suicide attempts. Researchers must report all suicides and suicide attempts in RCTs.