A latent class analysis of factors influencing preferences for infant respiratory syncytial virus (RSV) preventives among pregnant people in the United States.

A maternal vaccine and long-acting monoclonal antibody (mAb) were recently approved to protect infants against respiratory syncytial virus (RSV). We identified subgroups of pregnant people with different preferences for RSV preventives and respondent characteristics associated with subgroup membership. An online survey, including a discrete choice experiment (DCE), was conducted among US pregnant people. RSV preventive attributes included effectiveness, duration of protection during RSV season, injection recipient/timing, preventive type (vaccine or mAb), and type of visit required to receive injection. In DCE choice tasks, pregnant people selected between two hypothetical preventive profiles with varying attribute-levels and a no-preventive option. Logistic regression, including latent class analysis (LCA), was used to analyze the data. Of 992 pregnant people (mean age: 30.0 years), 60.3% were expecting their second/later birth. LCA identified three preference subgroups: 'Effectiveness' (preventive choice mostly driven by increases in effectiveness; 51.4% class membership probability), 'Season' (preventive choice mostly driven by improvement in duration of protection during the RSV season; 39.2% class membership probability), and 'No Preventive' (frequently chose no-preventive option; 9.4% class membership probability). 'Effectiveness' and 'Season' preferred maternal vaccine over mAb; mAb was preferred by 'No Preventive.' Perceiving RSV as serious for infants, higher health literacy, and lower household income were associated with 'Effectiveness.' Perceiving RSV as serious for pregnant people was associated with 'Season.' Perceiving RSV to not be serious for pregnant people and not being employed were associated with 'No Preventive.' Subgroups of pregnant people vary in preferences for RSV preventives. Most pregnant people preferred a maternal vaccine, although some may be more willing to accept alternative preventive options.

Healthcare Providers' and Pregnant People's Preferences for a Preventive to Protect Infants from Serious Illness Due to Respiratory Syncytial Virus.

We assessed the impact of respiratory syncytial virus (RSV) preventive characteristics on the intentions of pregnant people and healthcare providers (HCPs) to protect infants with a maternal vaccine or monoclonal antibodies (mAbs). Pregnant people and HCPs who treated pregnant people and/or infants were recruited via convenience sample from a general research panel to complete a cross-sectional, web-based survey, including a discrete choice experiment (DCE) wherein respondents chose between hypothetical RSV preventive profiles varying on five attributes (effectiveness, preventive type [maternal vaccine vs. mAb], injection recipient/timing, type of medical visit required to receive the injection, and duration of protection during RSV season) and a no-preventive option. A best-worst scaling (BWS) exercise was included to explore the impact of additional attributes on preventive preferences. Data were collected between October and November 2022. Attribute-level preference weights and relative importance (RI) were estimated. Overall, 992 pregnant people and 310 HCPs participated. A preventive (vs. none) was chosen 89.2% (pregnant people) and 96.0% (HCPs) of the time (DCE). Effectiveness was most important to preventive choice for pregnant people (RI = 48.0%) and HCPs (RI = 41.7%); all else equal, pregnant people (RI = 5.5%) and HCPs (RI = 7.2%) preferred the maternal vaccine over mAbs, although preventive type had limited influence on choice. Longer protection, protection starting at birth or the beginning of RSV season, and use for both pre-term and full-term babies were ranked highest in importance (BWS). Pregnant people and HCPs strongly preferred a preventive to protect infants against RSV (vs. none), underscoring the need to incorporate RSV preventives into routine care.

Development of the multi-attribute Adolescent Health Utility Measure (AHUM).

OBJECTIVE: Obtain utilities (preferences) for a generalizable set of health states experienced by older children and adolescents who receive therapy for chronic health conditions. METHODS: 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). RESULTS: 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. DISCUSSION: The AHUM health state classification system may be used in future research to enable calculation of quality-adjust life expectancy for applicable health conditions.

Population preference values for treatment outcomes in chronic lymphocytic leukaemia: a cross-sectional utility study.

BACKGROUND: 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. METHODS: This was a cross-sectional study of 89 members of the general population in the UK (England and Scotland). Using standard gamble, each participant valued four health states describing response status, six describing treatment-related toxicities based on Common Toxicity Criteria, and two describing line of treatment. The health states incorporated standardized descriptions of treatment response (symptoms have "improved," "stabilized," or "gotten worse"), swollen glands, impact on daily activities, fatigue, appetite, and night sweats. Utility estimates ranged from 0.0, reflecting dead, to 1.0, reflecting full health. RESULTS: Complete response (CR) was the most preferred health state (mean utility, 0.91), followed by partial response (PR), 0.84; no change (NC), 0.78; and progressive disease (PD), 0.68. Among the toxicity states, grade I/II nausea and nausea/vomiting had the smallest utility decrements (both were -0.05), and grade III/IV pneumonia had the greatest decrement (-0.20). The utility decrements obtained for toxicity states can be subtracted from utilities for CR, PR, NC, and PD, as appropriate. The utilities for second- and third-line treatments, which are attempted when symptoms worsen, were 0.71 and 0.65, respectively. No significant differences in utilities were observed by age, sex, or knowledge/experience with leukaemia. CONCLUSIONS: This study reports UK population utilities for a universal set of CLL health states that incorporate intended treatment response and unintended toxicities. These utilities can be applied in future cost-effectiveness analyses of CLL treatment.

Review of the economic and quality-of-life burden of cervical human papillomavirus disease.

OBJECTIVE: The purpose of this study was to conduct a systematic literature review on the economic burden and health-related quality-of-life impact of cervical human papillomavirus disease. STUDY DESIGN: A systematic review of cost-of-illness studies and health-related quality-of-life studies was conducted. PubMed, Embase, and PsycINFO databases were searched with the use of predefined terms. RESULTS: Nine economic and 24 quality-of-life studies were identified. The annual health care costs of human papillomavirus-related conditions in the United States range from 2.25-4.6 billion dollars (2005 US dollars). The burden of human papillomavirus is second only to human immunodeficiency virus among sexually transmitted diseases. Health-related quality-of-life areas that are impacted substantially by human papillomavirus include emotional, social, and sexual functioning. CONCLUSION: The economic and quality-of-life burden of cervical human papillomavirus disease is significant and highlights the need for treatment and prevention options for this condition.

Psychometric evaluation of the Osteoporosis Patient Treatment Satisfaction Questionnaire (OPSAT-Q), a novel measure to assess satisfaction with bisphosphonate treatment in postmenopausal women.

BACKGROUND: The Osteoporosis Patient Satisfaction Questionnaire (OPSAT-Q) is a new measure of patient satisfaction with bisphosphonate treatment for osteoporosis. The objective of this study was to evaluate the psychometric characteristics of the OPSAT-Q. METHODS: The OPSAT-Q contains 16 items in four subscales: Convenience, Confidence with Daily Activities, Side Effects, and Overall Satisfaction. All four subscale scores and an overall composite satisfaction score (CSS) can be computed. The OPSAT-Q, Osteoporosis Targeted Quality of Life (OPTQoL), and sociodemographic/clinical questionnaires, including 3 global items on convenience, functioning and side effects, were self-administered to women with osteoporosis or osteopenia recruited from four US clinics. Analyses included item and scale performance, internal consistency reliability, reproducibility, and construct validity. Reproducibility was measured using the intraclass correlation coefficient (ICC) via a follow-up questionnaire completed by participants 2 weeks post baseline. RESULTS: 104 women with a mean age of 65.1 years participated. The majority were Caucasian (64.4%), living with someone (74%), and not currently employed (58.7%). 73% had osteoporosis and 27% had osteopenia. 80% were taking weekly bisphosphonates and 18% were taking daily medication (2% missing data). On a scale of 0-100, individual patient subscale scores ranged from 17 to 100 and CSS scores ranged from 44 to 100. All scores showed acceptable internal consistency reliability (Cronbach's alpha > 0.70) (range 0.72 to 0.89). Reproducibility ranged from 0.62 (Daily Activities) to 0.79 (Side Effects) for the subscales; reproducibility for the CSS was 0.81. Significant correlations were found between the OPSAT-Q subscales and conceptually similar global measures (p < 0.001). CONCLUSION: The findings from this study confirm the validity and reliability of the OPSAT-Q and support the proposed composition of four subscales and a composite score. They also support the use of the OPSAT-Q to examine the impact of bisphosphonate dosing frequency on patient satisfaction.

Antipsychotic drug use among nursing home residents taking rivastigmine.

OBJECTIVE: To evaluate whether or not rivastigmine use is associated with a decrease in the initiation of antipsychotic drug therapy among nursing home residents in the United States. METHODS: A retrospective analysis was performed using Minimum Data Set data and physician order files for newly admitted residents of 452 US nursing facilities from 2000 through 2002. The rivastigmine group included those who were diagnosed with Alzheimers disease (AD) and began rivastigmine treatment within 30 days of diagnosis. Patients were required to be on treatment for a minimum of 30 days. The control group included those who were diagnosed with AD, but did not receive a cholinesterase inhibitor. All subjects were antipsychotic drug-naive within 30 days of baseline (initiation of rivastigmine or initial AD diagnosis). A Cox proportional hazards model was used to estimate predicted risk of antipsychotic drug use. RESULTS: This study included 845 patients in the rivastigmine group and 517 patients in the control group. The rivastigmine group had fewer female patients, was younger, and had more verbal distress, sleep issues, sadness, loss of interest, and behavioral symptoms at baseline compared with the control group (P < .01). Overall initiation of antipsychotics was lower in the rivastigmine group (8.6%) compared with the control group (17.0%). Patients in the control group were almost 2 times more likely (relative risk = 1.86; P < .001) to take antipsychotics compared with patients taking rivastigmine, after adjusting for demographic covariates and mental health conditions or behavioral symptoms at baseline. Patients with baseline mental health conditions or behavioral symptoms were more likely to start antipsychotics than those without such conditions (P < .001). CONCLUSIONS: Study results suggest that nursing home residents with Alzheimers disease treated with rivastigmine have a reduced risk of initiating therapy with an antipsychotic drug compared with residents who do not receive cholinesterase inhibitor treatment.

Impact of rivastigmine use on the risk of nursing home placement in a US sample.

INTRODUCTION: Although numerous studies have evaluated predictors of nursing home placement (NHP), few have focused on the effects of cholinesterase inhibitor (ChEI) use on NHP. The objective of this study was to compare the risk of NHP between rivastigmine patients versus no-ChEI patients (control group), and secondly, between rivastigmine versus donepezil patients. METHODS: A retrospective analysis of a large US medical claims database was performed. Eligible subjects were identified from those who had continuous medical coverage from 1 April 2000 to 30 June 2002. Rivastigmine and donepezil subjects were new users, defined as having received no ChEI treatment during the initial 6 months of the study. Control subjects were diagnosed with Alzheimer's disease (AD) at some point after the initial 6-month period. All subjects were followed from baseline (initiation of ChEI therapy or initial AD diagnosis) to the date of NHP or 30 June 2002, whichever occurred first. RESULTS: In the rivastigmine (n=1181), donepezil (n=3864), and control (n=517) groups, 3.7%, 4.4% and 11.0% of subjects, respectively, had an NHP (p <0.001 for rivastigmine versus control). A Cox proportional hazard model, controlling for age, gender, comorbidities and behavioural disorders, showed that the control subjects were almost 3-fold more likely to have NHP than rivastigmine subjects (hazard ratio [HR]=2.71; 95% CI 1.82, 4.03). The difference in the risk of NHP was not significant between the rivastigmine and donepezil groups (HR=1.23; 95% CI 0.89, 1.71). DISCUSSION: This study demonstrated that rivastigmine decreased the risk of NHP in a large insured population.

Gastrointestinal adverse events in a general population sample of nursing home residents taking cholinesterase inhibitors.

OBJECTIVE: To assess the occurrence of gastrointestinal (GI) adverse events in a national sample of nursing home residents taking donepezil, galantamine, or rivastigmine. DESIGN: A retrospective analysis of the Minimum Data Set (MDS) was performed. SETTING: Four hundred-fifty-two nursing facilities across the United States. PARTICIPANTS/INTERVENTIONS: During 2000, 2001, and 2002, eligible residents began donepezil, galantamine, or rivastigmine monotherapy. Among these treatment groups, 5,846, 750, and 1,672 residents, respectively, were included in the analysis. All MDS assessments were analyzed if completed. Analyses were conducted at baseline and within one year of initiating therapy. MAIN OUTCOME MEASURES: Occurrence of vomiting, stomach pain, diarrhea, weight loss, and insomnia. RESULTS: Overall, GI adverse events ranged from 2% to 9% and were comparable among the groups. Residents taking galantamine were more likely to experience diarrhea relative to those taking rivastigmine or donepezil (8.9% versus 6.8% versus 6.4%, respectively; P = 0.035), and less likely to lose weight relative to these groups (15.6% versus 20.0% versus 20.3%, respectively; P = 0.01). GI adverse events were less frequent during the maintenance phase compared with the initial drug-titration phase. CONCLUSION: GI events generally occurred with similar frequency in a national sample of nursing home residents taking donepezil, galantamine, or rivastigmine in the usual clinical practice setting.

A survey of children's preferences for influenza vaccine attributes.

BACKGROUND: While annual influenza vaccination is recommended by the CDC for children 6 months and older, vaccination rates remain suboptimal. For healthy, US children 2 years of age and older, influenza vaccine is available as an intramuscular injection (TIV) or an intranasal spray (LAIV), respectively. Little is known about children's experiences and preferences for influenza vaccine attributes. OBJECTIVE: To examine preferences for influenza vaccine attributes and their relative importance among children. METHODS: A quantitative web-survey was administered to children aged 8-12 years sampled from a standing online panel representative of the US population. Children were stratified by age, gender and parent's influenza vaccination behavior. The survey included questions to ascertain children's preferences for influenza vaccine attributes, including efficacy, chance of common side effects, and mode of administration. It included conjoint (trade-off) questions in which children traded-off different attributes in their choice between two influenza vaccines with differing features. We also surveyed children's comprehension of and ability to complete the conjoint questions. RESULTS: 544 children completed the survey (response rate 37%). Children most frequently selected efficacy as the most important vaccine attribute followed by mode of administration (45% and 31%, respectively). When asked for their preference to receive influenza vaccine as a "shot" or a "nose spray", the majority (69%) preferred the nose spray. An evaluation of children's ability to complete the conjoint survey demonstrated that 85% of the sample was able to complete the conjoint tasks. Analysis of the conjoint responses demonstrated that mode of administration and efficacy had the greatest impact on preferences, with a relative importance of 40.5% and 30.6%, respectively. In a direct comparison of vaccine profiles representing the efficacy, side effects, and other characteristics of LAIV and TIV, 79% of children preferred the LAIV-like profile. CONCLUSION: Children in the sample had consistent opinions regarding influenza vaccine attributes and consider vaccine efficacy and mode of administration to be important. Children can be informed participants in influenza prevention and can be included in discussions regarding influenza vaccination.

Parent preferences for pediatric influenza vaccine attributes.

Influenza vaccine is available as an intramuscular injection or an intranasal spray for eligible children. This study was conducted to examine parents' preferences for influenza vaccine attributes and the attributes' relative importance regarding the vaccination of their children. A quantitative Web survey was administered to 500 parents of children aged 2 to 12 years. The survey included general preference questions and conjoint (trade-off) questions. Parents most frequently selected efficacy, risk of temporary side effects, and physician recommendation as important vaccine attributes from a provided list (92%, 75%, and 59%, respectively). For attributes selected as important, parents rated the importance of the attribute; the highest mean importance ratings were given to efficacy, presence of mercury-containing preservative, and physician recommendation.The highest relative importance ratings in the conjoint section were given to efficacy and presence of mercury-containing preservative. Parental education on influenza vaccine efficacy and safety may help to improve pediatric vaccination rates.

Patient preferences for diabetic retinopathy health States.

PURPOSE. To develop standardized descriptions of health states that characterize vision-specific functional impacts of diabetic retinopathy (DR) according to levels of visual acuity and contrast sensitivity and to elicit preferences for these health states from persons with DR and assign weighted values to them. METHODS. Vision-specific descriptions of health states were developed based on a literature review and patient and physician interviews. The content was based on items from the National Eye Institute Visual Functioning Questionnaire (VFQ) and reflected functional impacts experienced by DR patients. Values were assigned to the range of health states, anchored by the extremes full vision and death, by using the time-tradeoff method in a sample of 98 Canadian DR patients from three clinical centers. RESULTS. The mean age of the sample was 60.4 years, and 56% were men. Mean preferences decreased from 0.98 (better-eye logMAR [Snellen equivalent] acuity, > or =20/40; worse-eye Snellen equivalent, > or =20/200) to 0.67 (Snellen equivalent visual acuity, < or =20/200, contrast sensitivity, < or =21 letters bilaterally). Preferences decreased with increasing severity of functional deficits and did not vary significantly by sex, age, VFQ quartile, or better- or worse-eye acuity. CONCLUSIONS. This is the first study that has been conducted to estimate preferences for standardized DR-specific health states, accounting for visual acuity and contrast sensitivity in both eyes. The results showed that the development and progression of DR are associated with substantial declines in preferences. In addition to the progressively greater impact from declining ETDRS visual acuity and contrast sensitivity, preference weights declined with increasing bilateral disparity. These preference values are useful for comparing the cost effectiveness of ophthalmic treatments.

Parents' decision-making regarding vaccinating their children against influenza: A web-based survey.

BACKGROUND: Despite the recommendation from the Centers for Disease Control and Prevention that children between the ages of 6 months and 18 years be vaccinated against influenza annually, vaccination rates remain suboptimal. OBJECTIVES: This study was conducted to explore factors that influence parents' decisions regarding influenza vaccination for children aged 2 to 12 years, to quantify the relative importance of these factors, to identify an appropriate theoretical model for illustrating the relationships among these factors, and to characterize parents by their likelihood of vaccinating their children against influenza. METHODS: A quantitative Web-based survey was administered to a sample of parents from an online panel representative of the US population. Parents were stratified based on self-reported rates of their personal influenza vaccination (every year, sometimes, or never) and the age of their child (2-4 years or 5-12 years). The results were examined by parents' likelihood of vaccinating their child in the next year (high, medium, or low). Participants were asked to rank their agreement with statements representing various beliefs and perceptions about influenza and influenza vaccine on a scale from 1 = strongly agree to 5 = strongly disagree. Parents who indicated that they vaccinate their child every year were asked to select the drivers of their decision to vaccinate; parents who indicated that they never vaccinate their child were asked to select the barriers affecting their decision not to vaccinate; and parents who responded that they sometimes vaccinate their child were asked to select both the drivers and barriers affecting their decision. Participants were then asked to rank the importance of each driver or barrier on a scale from 1 = a little important to 5 = extremely important. Mean agreement ratings were calculated for parents' beliefs and perceptions about influenza and influenza vaccine and were compared across likelihood subgroups. Mean importance ratings of the drivers and barriers to vaccination were also calculated and compared across likelihood subgroups. RESULTS: The survey sample consisted of 500 parents; their mean (SD) age was 37.4 (6.82) years, 57.2% were female, and 78.2% were non-Hispanic white. Among those who reported that they vaccinated their child against influenza every year or sometimes, the major drivers of vaccination were prevention of influenza (95.1%), a doctor's recommendation (89.5%), and the desire to reduce influenza symptoms (83.3%). Among those who reported sometimes or never vaccinating their child against influenza, barriers to vaccination were more variable. The most common barriers were low perceived risk of influenza (46.0%), the perception that the vaccine caused influenza (44.0%), and side effects caused by the vaccine (36.6%). Distinct differences were found in beliefs and perceptions of influenza and influenza vaccine according to respondents' likelihood of vaccination. A high likelihood of vaccination was associated with a greater perceived threat of influenza and less concern about the efficacy and safety of the vaccine. Convenience was an important factor among parents with a medium likelihood of vaccination. The Health Belief Model was identified as an appropriate theoretical framework for illustrating the factors influencing parents' decision-making about influenza vaccination. CONCLUSIONS: Prevention of influenza, reduction of influenza symptoms, and doctor recommendation were the main drivers of parents' decision to vaccinate their child against influenza. Barriers to vaccination were more variable and primarily included the risk of adverse effects and the perceived low risk of influenza. Increasing parents' awareness of the threat of influenza and the efficacy and safety of the vaccine, as well as improving the convenience of getting vaccinated, may help improve rates of pediatric influenza vaccination.

Psychometric evaluation of the Satisfaction with Oral Anti-Diabetic Agent Scale (SOADAS).

In this study we developed and evaluated the psychometric properties of the Satisfaction with Oral Anti-Diabetic Agent Scale (SOADAS), the first treatment satisfaction instrument specific to oral anti-diabetic agents (OADs). Three focus groups and three rounds of cognitive debriefing interviews with type 2 diabetes patients using OADs informed the development of the six-item SOADAS. The measurement properties of the SOADAS were evaluated in a US cross-sectional study. Patients completed the SOADAS, Treatment Satisfaction for Medication Questionnaire (TSQM), the Short Form-12 (SF-12) Health Survey and a demographic questionnaire. The first half of the recruited patient population was asked to return at 7 days follow-up to complete the SOADAS again. An exploratory factor analysis on the SOADAS yielded a one-factor solution, indicating that the SOADAS was uni-dimensional. The SOADAS had high internal consistency reliability (Cronbach's alpha=0.86) and acceptable test-retest reliability (ICC=0.79). It was correlated most highly with the TSQM (r=0.71, p<0.001) and least with the mental summary component score of the SF-12 (r=0.24, p=0.014). Mean SOADAS scores were approximately two points higher for participants with lower HbA(1c) values (<7%) versus those with higher values, and for participants without versus with selected symptoms (p<0.05). The findings from this study suggest that the SOADAS is a valid and reliable measure of patient satisfaction with oral anti-diabetic medications. This questionnaire may be useful for assessing outcomes in future clinical studies of oral anti-diabetic medications.

Patient and caregiver perspective on pediatric eosinophilic esophagitis and newly developed symptom questionnaires*.

OBJECTIVE: Eosinophilic esophagitis (EE), a rare chronic inflammatory condition of the esophagus, is predominantly observed in children and is primarily manifested with feeding difficulties. To our knowledge, no self- or caregiver-reported questionnaires are available to assess pediatric EE symptoms and their impact as reported directly by children or their parents/caregivers. The objectives of this study were to characterize the symptoms and impact of EE among children as reported by patients and parents/caregivers and to assess the content validity of two newly developed pediatric eosinophilic esophagitis symptom questionnaires, one parent/caregiver-reported questionnaire for ages 2-7 years and one child-reported questionnaire for ages 8-17 years. The questionnaires were developed based on a review of the literature and clinical expert consultation. RESEARCH DESIGN AND METHODS: This cross-sectional study involving one-on-one interviews with patients and caregivers was conducted at an American Partnership for Eosinophilic Disorders conference. Parents of patients aged 2-7 years (n = 12) and patients aged 8-17 years (n = 16) were first asked about symptoms and their impact on everyday life, using open-ended questions. Participants then completed the appropriate symptom questionnaire and were asked to provide feedback on the relevance, comprehensiveness, and clarity of each item and other questionnaire issues (time to complete, length, format, etc.). All reported symptoms were enumerated, and the feedback on the symptom questionnaires was analyzed qualitatively. RESULTS: The majority of study participants were white (82%) and male (86%). The most frequently reported symptoms of 2-7-year olds were vomiting (92%), "reflux" (50%), dysphagia (25%), abdominal pain (25%), and trouble sleeping (25%). The 8-17-year group reported abdominal pain (56%), vomiting (31%), throat pain (25%), diarrhea (25%), and food getting stuck (25%). Symptoms and treatment were reported to have a major impact on daily life, particularly on school, after-school activities and social events, feeling frustrated regarding symptoms and treatment, and feeling "different". Overall, participants thought that the questionnaires were clear, relevant, and appropriate for symptom assessment. LIMITATION: This study was based on a small and convenient sample of participants attending an EE conference and hence may not be representative of the general EE patient population. CONCLUSIONS: EE is associated with a range of symptoms that vary in terms of the type, frequency and severity across and within patients. The results provide adequate support for the content validity of the self- and caregiver-reported versions of the symptom-specific questionnaires. Minor modifications were made based on the feedback obtained. A psychometric evaluation of the revised questionnaires is needed next to assess the construct validity, reliability, and responsiveness of the measures.

Caregiver preference for rivastigmine patch relative to capsules for treatment of probable Alzheimer's disease.

BACKGROUND: Family caregivers comprise a critical component in the care of Alzheimer's disease (AD) patients. Among their many tasks, caregivers are responsible for administering and managing medications. Effective interventions incorporate the needs of both the AD patient and the caregiver, and understanding treatment preferences may maximize intervention effectiveness. Transdermal patches may offer advantages over conventional oral formulations. METHODS: A 24-week randomized controlled trial compared the rivastigmine patch to the rivastigmine capsule and placebo in patients with probable AD. At baseline and Weeks 8 and 24, the AD Caregiver Preference Questionnaire (ADCPQ) was used to evaluate caregiver expectations, preferences and satisfaction with treatment. Double-dummy treatment blinding ensured that caregiver preference for the patch or capsule was not confounded by perceptions of efficacy or tolerability. Reasons for preference were also elicited. The analytic sample included caregivers who completed the ADCPQ at Weeks 8 and/or 24. RESULTS: One thousand and fifty-nine caregivers completed the ADCPQ. More than 70% of caregivers preferred the rivastigmine patch to the capsule. The patch was significantly preferred to the capsule with respect to ease of following the schedule and ease of use. Caregivers indicated greater satisfaction overall, greater satisfaction with administration, and less interference with daily life with the patch versus the capsule (all p

Understanding patient preferences for HIV medications using adaptive conjoint analysis: feasibility assessment.

OBJECTIVE: Choosing among HIV medications involve making trade-offs among various efficacy, convenience, resistance, and side-effect attributes. This study tested the feasibility of using adaptive conjoint analysis (ACA) to assess preferences (utilities) for HIV medication attributes. METHODS: HIV individuals were recruited through newspaper advertisements. Participants completed a computerized ACA survey that assessed 12 attributes, including side effects, regimen convenience, resistance, and efficacy. Literature on third-agent HIV drugs was used to identify percentage risk and severity level descriptions for each attribute. Based on the ACA-derived utilities, we assessed the relative importance of the attributes by averaging individually calculated importance and estimated the percentages that would prefer selected HIV medications over others. To check validity of the ACA utilities, the survey also had respondents choose among medications with different attribute profiles. RESULTS: The 35 respondents were primarily African Americans (94%) and unemployed (54%). Of these, 28 (80%) provided consistent responses and were analyzed. Of the 12 medication attributes evaluated, the risk of developing resistance, regimen convenience, and the risk of sleep disturbance had the greatest impact on preferences; each accounting for more than 8.5% of the variation in preferences. These were followed by risk of drug failure (8.2%), cholesterol elevation (7.1%), diarrhea (7.1%) and nausea (6.9%). The ACA utilities accurately predicted patients' actual medication choices 75% of the time. CONCLUSIONS: Adaptive conjoint analysis was successful in predicting HIV treatment preferences under different medication scenarios. Resistance, regimen convenience, and sleep disturbance would likely make the most difference in the perceived value of a third-agent HIV medication.

The cost-effectiveness of beta-radiation therapy for treatment of in-stent restenosis: an analysis at 290-day follow-up.

PURPOSE: We studied the cost-effectiveness of percutaneous coronary intervention (PCI) plus beta-radiation (Radiotherapy) vs. PCI alone for treating in-stent restenosis. METHODS AND MATERIALS: We analyzed medical resource data collected at US sites during the Intimal Hyperplasia Inhibition with beta In-stent Trial (INHIBIT) to compare the incremental costs between Radiotherapy and PCI alone for each repeat target lesion revascularization (TLR) avoided. Costs were assigned for cardiac-related medical services using the Medicare payer perspective. RESULTS: Radiotherapy increased initial inpatient costs by US$3360 (P<.001). In the Radiotherapy and PCI alone groups, the mean per-patient rates for TLR were 0.13 vs. 0.30 (P=.001), and mean per-patient total costs were US$19,286 vs. US$18,349 (P<.001), respectively. The incremental cost of Radiotherapy relative to PCI alone was US$5512 per TLR avoided, which compares favorably to the observed mean per-patient cost of treating restenosis (US$16,852). CONCLUSIONS: beta-radiation is a cost-effective adjunct to PCI in treating patients with in-stent restenosis.

The health-related quality-of-life impact of histamine dihydrochloride plus interleukin-2 compared with interleukin-2 alone in patients with metastatic melanoma.

The aim of this study was to compare the quality-of-life (QOL) effects of interleukin-2 (IL-2) alone with those of IL-2 plus histamine dihydrochloride in the setting of a multicenter, randomized trial for patients with metastatic melanoma. QOL data were collected from July 1997 to March 2000 during a phase III trial comparing subcutaneous histamine plus IL-2 and IL-2 alone. Prior to each treatment cycle, patients completed the 76-item Quality-of-Well-Being Scale-Self-Administered (QWB-SA) questionnaire, the Overall State of Health (OSH) item, and the General Health Perception (GHP) item. A longitudinal data analysis using the generalized estimating equations approach was performed to compare changes in QWB-SA scores over time between treatment groups, and predicted QWB-SA scores from the regression analysis were used to calculate quality-adjusted survival duration over the 12-month study period. QOL analyses were conducted for all randomized patients (intent-to-treat overall population, ITT-OA) and all patients who had liver metastases at randomization (ITT-LM population). In the ITT-OA population, differences in QWB-SA scores over time between the histamine plus IL-2 group (150 patients) and the IL-2 alone group (151 patients) were not significant (P=0.511, type III F test). In the ITT-LM population (53 histamine plus IL-2 patients and 73 IL-2 alone patients), changes in QWB-SA scores over time favored the histamine plus IL-2 group (P=0.018, type III F-test). In both the ITT-OA and ITT-LM populations, QWB-SA scores deteriorated more quickly over time in the IL-2 alone group than in the histamine plus IL-2 group, resulting in a significant difference in median quality-adjusted survival duration in favor of the histamine plus IL-2 group by 31.3 days in the ITT-OA population (P=0.007, Mann-Whitney U-test), and 50.2 days in the ITT-LM population (P=0.011). OSH and GHP scores did not differ between groups. The addition of subcutaneous histamine dihydrochloride to IL-2 treatment improved median quality-adjusted survival duration and did not adversely affect QOL in patients with malignant melanoma.

Low intraindividual variability of cyclosporin A exposure reduces chronic rejection incidence and health care costs.

The present study applied a receiver operating characteristic (ROC) analysis to assess the role of intraindividual variability of cyclosporin A (CsA) drug exposure in predisposing renal transplant recipients to the occurrence of chronic rejection, as well as to increased health care costs using a resource-based economic analysis. Two hundred and four adult renal transplant recipients were treated with tapering doses of prednisone (Pred) and with a concentration-controlled strategy that selected doses of the olive oil-based formulations of CsA (Sandimmune(R)) that achieved target concentrations based on serial pharmacokinetic profiles. The ROC analysis revealed an inflection point of plots of the coefficient of variation (%CV) of CsA exposure versus the risk of chronic rejection at >/=28.4% for the average concentration (C(av)), i.e., the dosing interval-corrected area under the concentration-time curves, and >/=36% for the trough concentration (C(0)). The incidence of chronic rejection over a period of 5 yr was 24% among the less variable (LV) versus 40% among the variable (V) cohort. The economic analysis revealed that the total mean facility and physician costs per patient were $48,789 versus $60,998, respectively (P < 0.01). The degree of variability displayed by any individual could only be predicted by serial measurements of CsA concentrations, and not by demographic features, laboratory determinations, clinical characteristics, individual or mean values of any observed CsA concentration, or other pharmacokinetic parameters calculated following a single drug exposure. Thus, strategies that reduce intrapatient variability of CsA exposure over time may lead to reductions in chronic allograft loss and in treatment costs.