Kathleen M. Beusterien

Background: Therapies for invasive breast cancer may be associated with an incremental survival advantage that should be weighed against the risk of toxicities when making treatment decisions. The objective of this study was to elicit patient preferences for a comprehensive profile of attributes associated with chemotherapies for breast cancer. Methods: This was a cross-sectional study of 121 patients with stage I-IV breast cancer who completed an internet-based conjoint survey that assessed the following attributes: ten grade III/IV toxicities, survival advantage, and administration regimen. Literature and expert input were used to identify descriptions for each attribute and respective levels (eg, different risks of toxicities). Subjects rated the attribute levels on a series of scales and indicated preferences in pair-wise comparisons of two hypothetical treatments differing in attribute levels. Ordinary least-squares regression was used to calculate utilities (preference weights) for each attribute level. Results: Of the twelve attributes, survival was the most important; specifically, a survival advantage of 3 months versus no survival advantage was most influential in the perceived value of chemotherapy. Among toxicities, the differences in the risks of neutropenia with hospitalization, diarrhea, nausea, and fatigue had the most impact on preferences; the risk differences of myalgia, stomatitis, and hand-foot syndrome had the least. In general, a more convenient administration regimen was less important than a 13% chance or more of severe toxicities, but more important than a 10%–12% chance of severe toxicities. Conclusion: Breast cancer patients place high value on small incremental survival advantages associated with treatment despite the risk of serious toxicities.

Hereditary angioedema (HAE) is a rare but serious disease marked by swelling attacks in the extremities, face, trunk, airway, or abdominal areas that can be spontaneous or the result of trauma and other triggers. It can be life-threatening due to the risk of asphyxiation. While there have been major advancements in our understanding of the immunogenetics of HAE, there are significant gaps in the literature regarding understanding of the humanistic and economic impact of the disease, particularly in Europe. The purpose of the HAE Burden of Illness Study-Europe (HAE-BOIS-Europe), the development and methodology of which is described here, is to better understand the management and impact of HAE from the patient perspective in Europe.

Objective: Given high rates of comorbidity, lack of awareness and global acceptance, and varying guidelines for its management, adult ADHD may be an especially difficult condition to diagnose and treat. The objective of this review was to explore and characterize similarities and differences among comorbidities associated with adult ADHD. Method: A review of the literature over the past 10 years was performed using Ovid. Results: A myriad of comorbid conditions such as impulse-control/personality, anxiety, mood, substance use, learning, and sleep disorders overlap with adult ADHD. Furthermore, a number of such conditions have symptoms that can mimic those of ADHD, including hyperactivity, impulsivity, inattention, and disruption of circadian rhythms, adding to the complexity of recognition and diagnosis of ADHD in adults. Extensive research shows that adults with ADHD appear to benefit from treatment with stimulant medications in similar ways as children, including significant improvements on driving performance. However, fear surrounding the abuse of stimulants is an important issue. Nevertheless, evidence suggests that children with ADHD who are treated with stimulant medication are less likely to develop a substance use disorder in adolescence and adulthood. Conclusion: There are a wide range of comorbidities with adult ADHD with many having overlapping symptoms. The benefits observed with ADHD treatment, however, emphasize the importance of recognition and treatment of adult ADHD.

Objective: To explore the literature focusing on cultural influences in the diagnosis of adult ADHD and respective societal burden. Method: A review of the literature over the past 10 years was performed using OVID. Results: Although numerous articles focused on diagnosis and burden of adult ADHD, few focused on cultural factors influencing diagnosis. Like other mental health disorders, cultural and social perspectives contribute to our understanding of adult ADHD and may play a significant role in the diagnosis and varying acceptance of the condition. Moreover, adults with ADHD may underestimate the impact of ADHD symptoms, and in many cases have learned to compensate for ADHD related impairments by choosing lifestyles that help compensate for symptoms. Some adults with ADHD may appear to function well, however they may expend excessive amounts of energy to overcome impairments; and they may be distressed by ongoing symptoms such as restlessness, mood instability and low self-esteem. Research shows that ADHD can be detrimental to many areas of life including work, daily activities, social and family relationships and psychological and physical well-being. Patient-reported impairments in productivity due to poor time management, procrastination, and distractibility can translate into significant indirect costs and decreased quality of life. ADHD in adults is also associated with increased accidents, medical resource utilization, antisocial behaviour and drug alcohol abuse. Conclusion: The substantial societal burden of adult ADHD highlights the importance of providing a better understanding of the factors that contribute to accurate diagnosis and of improving the low recognition of the disorder in many world regions.

Obtain utilities (preferences) for a generalizable set of health states experienced by older children and adolescents who receive therapy for chronic health conditions. A health state classification system, the Adolescent Health Utility Measure (AHUM), was developed based on generic health status measures and input from children with Hunter syndrome and their caregivers. The AHUM contains six dimensions with 4-7 severity levels: self-care, pain, mobility, strenuous activities, self-image, and health perceptions. Using the time trade off (TTO) approach, a UK population sample provided utilities for 62 of 16,800 AHUM states. A mixed effects model was used to estimate utilities for the AHUM states. The AHUM was applied to trial NCT00069641 of idursulfase for Hunter syndrome and its extension (NCT00630747). Observations (i.e., utilities) totaled 3,744 (12*312 participants), with between 43 to 60 for each health state except for the best and worst states which had 312 observations. The mean utilities for the best and worst AHUM states were 0.99 and 0.41, respectively. The random effects model was statistically significant (p < 0.0001; adjusted R2 = 0.361; RMSE = 0.194). When AHUM utilities were applied to the idursulfase trial, mean utilities in the idursulfase weekly and placebo groups improved +0.087 and +0.006, respectively, from baseline to week 53. In the extension, when all patients received idursulfase, the utilities in the treatment group remained stable and the placebo group improved +0.039. The AHUM health state classification system may be used in future research to enable calculation of quality-adjust life expectancy for applicable health conditions.

Given that treatments for chronic lymphocytic leukaemia (CLL) are palliative rather than curative, evaluating the patient-perceived impacts of therapy is critical. To date, no utility (preference) studies from the general public or patient perspective have been conducted in CLL. The objective of this study was to measure preferences for health states associated with CLL treatment.

Background: Methods: Results: Conclusions:

Research on HIV medication adherence has relied mainly on quantitative methods. The objective of this study was to explore factors associated with adherence from the HIV-infected patient's perspective. Six focus groups were convened with treatment-experienced HIV-positive individuals. The discussions focused on issues that make it easy or difficult to adhere to HIV regimens. Thirty-five patients participated in the focus groups, which were conducted in Washington, D.C., and Los Angeles. The mean age was 48; 66% were male; 63% were black; and 40% contracted HIV through heterosexual contact. Six major themes emerged from the data that influenced adherence to medication: regimen complexity/medication features (including number of pills), lifestyle fit, emotional impacts (including worry, anger, stress and anxiety), side effects, medication effectiveness, and communication (including information from friends, physicians, and published sources). The data informed a conceptual framework, illustrating the possible interactions among these themes that can potentially be used by clinicians when discussing HIV treatment options with patients. This is potentially one of the first focus group studies concentrating on HIV medication adherence. The findings highlight specific factors that should be considered when trying to improve adherence and may be helpful in clinical decision-making.

The objective is to assess patient preferences for attributes associated with third agent HIV medications, including fosamprenavir/ritonavir (FPVr), fosamprenavir (FPV), lopinavir/ritonavir (LPVr), atazanavir (ATZ), and efavirenz (EFV). Subjects with HIV were recruited in the US and Germany to complete a computerized adaptive conjoint survey that assessed 13 attributes, including moderate to severe side effects, regimen convenience, drug resistance and efficacy. Literature on the target third-agent HIV drugs was used to identify percentage risk and severity level descriptions for each attribute. The derived preference (utility) weights for each attribute level informed the calculation of relative importance estimates for each attribute and the desirability of combinations of attributes matching the respective target third agents. The analysis included 288 HIV-positive participants (US: 132; Germany: 156), 205 of whom were treatment-experienced and 83 of whom were treatment-naïve. Of the 13 medication attributes evaluated, developing drug resistance, the risk of lipodystrophy, the risk of gastronitestinal side effects (diarrhoea, nausea and vomiting) and regimen convenience had the greatest impact on preferences. The profile based on FPVr was most preferred. Differences in the risk of developing drug resistance, risk of lipodystrophy, risk of gastrointestinal side effects and regimen convenience would likely be most influential in the perceived relative value of a third-agent medication. Physicians may wish to consider these features, especially when discussing HIV treatment options with their patients.