Individuals born with primary immune deficiency diseases (PIDD) have a dysfunctional immune system, and many are treated by lifelong injections of immunoglobulin therapy. Studies have shown that these patients have low health-related quality of life (HRQOL) and well-being (WB) and that these outcomes might be improved by the availability of therapy innovated according to preferences for fewer needle sticks or a shorter infusion time. Regulators at the U.S. Food and Drug Administration (FDA) have approved therapies innovated per these preferences. However, there is limited data demonstrating how these innovations impact HRQOL and WB. Using the biopsychosocial model, the purpose of this cross sectional quantitative study was to evaluate whether patients with PIDD using therapies innovated for fewer needle sticks or a shorter infusion time had a higher mean HRQOL and WB compared to those who were not. The study included 153 patients who completed the Patient Reported Outcomes Measurement Information System (PROMIS)-29 survey. The dependent variables were HRQOL and WB measured by PROMIS-29, and the independent variables were the medical product innovations. Independent samples t tests results showed mean PROMIS-29 scores were not statistically different (p > .05). This suggests patients were optimized according to their treatment preference. A subgroup of patients who had taken the PROMIS-29 survey more than once concurrent with switching to a therapy aligned with patient preferences showed improved HRQOL and WB. These findings have implications for positive social change in that seeking the patient's voice to inform medical product innovation and FDA regulatory decision-making has potential to improve biopsychosocial outcomes.

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Contemporary conceptual models of health emphasize the importance of patient-based outcomes and recognize the complexity involved in their assessment. Various health conditions, personal, social, and environmental factors, are all thought to contribute to individual’s quality of life. However, the impact of orthodontic treatment on Oral Health-related Quality of Life (OH-QOL) outcomes in children has not yet been systematically studied. Hence, this research was planned to assess the effect orthodontic treatment has on pediatric OH-QOL outcomes. Further, the important moderational role of children’s psychological assets on OH-QOL reports is explored. Following completion of a preliminary study to confirm the psychometric properties of the Child Perception Questionnaire (CPQl1-14), the current two-phase study was undertaken. This consisted of a cross-sectional study examining the relationship among Self-Esteem (SE), malocclusion, and OH-QOL, and a longitudinal study examining the influence of orthodontics and children’s Psychological Wellbeing (PWB) on OH-QOL reports. This PhD dissertation is presented in the “Publishable Style”. The journals which hold the copyrights for the papers published from this thesis have given permission for the reproduction of the text and figures for this dissertation. The preliminary data confirmed that the CPQ11-14 is sensitive to change when used with children receiving orthodontic treatment. Our cross-sectional findings indicated that the impact of malocclusion on OH-QOL is substantial in children with low SE and identified SE as a salient determinant of OH-QOL in children seeking orthodontic treatment. Longitudinal data, on the other hand, detected significant improvement of OH-QOL outcomes after orthodontic treatment. As postulated, these improvements were most evident for the social and emotional domains of OH-QOL. However, covariate analysis emphasized the important role psychological factors play in moderating OH-QOL reports, as children with better PWB were more likely to report better OH-QOL regardless of their orthodontic treatment status. These results substantiate the validity of contemporary models of patient-based outcomes linking biological, personal, social, and environmental factors. Researchers and clinicians are encouraged to adopt this forward thinking approach when dealing with children with oro-facial conditions. Further studies with larger samples and longer follow-ups would be of value to expand on these findings.

Contemporary conceptual models of health emphasize the importance of patient-based outcomes and recognize the complexity involved in their assessment. Various health conditions, personal, social, and environmental factors, are all thought to contribute to individual’s quality of life. However, the impact of orthodontic treatment on Oral Health-related Quality of Life (OH-QOL) outcomes in children has not yet been systematically studied. Hence, this research was planned to assess the effect orthodontic treatment has on pediatric OH-QOL outcomes. Further, the important moderational role of children’s psychological assets on OH-QOL reports is explored. Following completion of a preliminary study to confirm the psychometric properties of the Child Perception Questionnaire (CPQl1-14), the current two-phase study was undertaken. This consisted of a cross-sectional study examining the relationship among Self-Esteem (SE), malocclusion, and OH-QOL, and a longitudinal study examining the influence of orthodontics and children’s Psychological Wellbeing (PWB) on OH-QOL reports. This PhD dissertation is presented in the “Publishable Style”. The journals which hold the copyrights for the papers published from this thesis have given permission for the reproduction of the text and figures for this dissertation. The preliminary data confirmed that the CPQ11-14 is sensitive to change when used with children receiving orthodontic treatment. Our cross-sectional findings indicated that the impact of malocclusion on OH-QOL is substantial in children with low SE and identified SE as a salient determinant of OH-QOL in children seeking orthodontic treatment. Longitudinal data, on the other hand, detected significant improvement of OH-QOL outcomes after orthodontic treatment. As postulated, these improvements were most evident for the social and emotional domains of OH-QOL. However, covariate analysis emphasized the important role psychological factors play in moderating OH-QOL reports, as children with better PWB were more likely to report better OH-QOL regardless of their orthodontic treatment status. These results substantiate the validity of contemporary models of patient-based outcomes linking biological, personal, social, and environmental factors. Researchers and clinicians are encouraged to adopt this forward thinking approach when dealing with children with oro-facial conditions. Further studies with larger samples and longer follow-ups would be of value to expand on these findings.

Background: Knee osteoarthritis is a degenerative disease characterized by damaged joint tissues (e.g., cartilage) that leads to joint pain, and reduced mobility and quality of life. Various factors are involved in disease progression, including biomechanical, patient-reported outcome and mobility measures. This thesis provides important longitudinal data on the role of these factors in disease progression, and the trajectory of biomechanical factors in persons with knee osteoarthritis. Objectives: (1) Determine the extent to which changes over 2.5 years in knee cartilage thickness and volume in persons with knee osteoarthritis were predicted by the knee adduction and flexion moment peaks, and knee adduction moment impulse and loading frequency. (2) Determine the extent to which changes over 2 years in walking and stair-climbing mobility in women with knee osteoarthritis were predicted by quadriceps strength and power, pain and self-efficacy. (3) Estimate the relative and absolute test-retest reliabilities of biomechanical risk factors for knee osteoarthritis progression. Methods: Data were collected at 3-month intervals during a longitudinal (3-year), observational study of persons with clinical knee osteoarthritis (n=64). Magnetic resonance imaging of the study knee was acquired at the first and last assessments, and used to determine cartilage thickness and volume. Accelerometry and dynamometry data were acquired every 3 months, and used to determine knee loading frequency and knee muscle strength and power, respectively. Walking and stair-climbing mobility, as well as pain and self-efficacy data, were also collected every 3 months. Gait analyses were performed every 6 months, and used to calculate lower-extremity kinematics and kinetics. Results: (1) The knee adduction moment peak and impulse each interacted with body mass index to predict loss of medial tibial cartilage volume over 2.5 years. These interactions suggested that larger joint loads in those with a higher body mass index were associated with greater loss of cartilage volume. (2) In women, lower baseline self-efficacy predicted decreased walking and stair ascent performances over 2 years. Higher baseline pain intensity/frequency also predicted decreased walking performance. Quadriceps strength and power each interacted with self-efficacy to predict worsening stair ascent times. These interactions suggested that the impact of lesser quadriceps strength and power on worsening stair ascent performance was more important among women with lower self-efficacy. (3) Relative reliabilities were high for the knee adduction moment peak and impulse, quadriceps strength and power, and body mass index (i.e., intraclass correlation coefficients >0.80). Absolute reliabilities were high for quadriceps strength and body mass index (standard errors of measurement <15% of the mean). Data supported the use of interventions effective in reducing the knee adduction moment and body mass index, and increasing quadriceps strength, in persons with knee osteoarthritis. Conclusion: Findings from this thesis suggest that biomechanical factors play a modest independent role in the progression of knee osteoarthritis. However, in the presence of other circumstances (e.g., obesity, low self-efficacy, high pain intensity/frequency), biomechanical factors can vastly worsen the disease. Strategies aiming to curb structural progression and improve clinical outcomes in knee osteoarthritis should target biomechanical and clinical outcomes simultaneously. / Thesis / Doctor of Philosophy (PhD) / Knee osteoarthritis is a multifactorial disease whose progression involves worsening joint structure, symptoms, and mobility. Various factors are linked to the progression of this disease, including biomechanical, patient-reported outcome and mobility measures. This thesis provides important information on how these factors, separately and collectively, are involved in worsening disease over time, as well as benchmarks that are useful to clinicians and researchers in interpreting results from interventional or longitudinal research. First, we examined how different elements of knee loading were associated with changes in knee cartilage quantity over time in persons with knee osteoarthritis. Second, we examined how different elements of knee muscle capacity and patient-reported outcomes were related to changes in mobility over time in persons with knee osteoarthritis. Third, we examined the stability over time of various biomechanical risk factors for the progression of knee osteoarthritis. Novel results from this thesis showed that: (1) larger knee loads predicted cartilage loss over 2.5 years in obese individuals with knee osteoarthritis but not in persons of normal weight or overweight; (2) among women with knee osteoarthritis with lower self-efficacy (or confidence), lesser knee muscle capacity (strength, power) was an important predictor of declining stair-climbing performance over 2 years; and (3) clinical interventions that can positively alter knee biomechanics include weight loss, knee muscle strengthening, as well as specific knee surgery and alterations during walking to reduce knee loads. Interventions for knee osteoarthritis should target biomechanical and clinical outcomes simultaneously.