Development of a conceptual model to illustrate the impact of multiple myeloma and its treatment on health-related quality of life.

PURPOSE: Little qualitative research exploring the impact of multiple myeloma (MM) and its treatment on the health-related quality of life (HRQL) of patients has been published. This study aimed to explore the burden of MM symptoms and treatment and the impact of these on HRQL. A model was developed to illustrate key concepts and their interrelationships. METHODS: Patients with MM were recruited to this cross-sectional, qualitative study through a patient panel and at two clinical sites in the USA. An interview discussion guide was developed using a review of published literature and interviews with experienced MM clinicians. In-depth, semistructured telephone interviews with MM patients were conducted to explore their experiences of the disease and its treatment. Data were analyzed using a thematic analysis approach. RESULTS: Twenty MM patients at various stages of treatment participated in open-ended, semistructured interviews. Patients reported both current and previous MM symptoms; most had experienced fatigue and pain. Other commonly reported symptoms were fractures, anemia, neuropathy, aches, and infections. MM treatment was found to have a negative impact on patients' HRQL; treatment-related adverse events included fatigue, neuropathy, insomnia, and gastrointestinal symptoms. MM treatment placed a substantial psychological and physical burden on patients, disrupting social activities, decreasing independence, and impacting on relationships. A model was developed to illustrate the relationship between these concepts. CONCLUSION: The conceptual model developed in this study illustrates the many aspects of MM and its treatment and how they can have a negative impact on patients' HRQL.

Patient-reported fatigue and its impact on patients with systemic lupus erythematosus.

Fatigue is a hallmark symptom of systemic lupus erythematosus (SLE), often associated with flares, side effects of treatment, and extensive organ damage and may have a significant impact on health-related quality of life (HrQoL). To date, the experience of fatigue in patients with SLE is underexplored. This study explored the experience of fatigue in patients with SLE and its impact on their lives through qualitative interviews. This cross-sectional qualitative study was conducted with 22 adult patients with SLE, recruited from two clinical sites in the United States. In-person semi-structured interviews were conducted and thematic analysis was performed focusing on the experience of fatigue in SLE. Results indicated that 21 out of 22 patients experienced fatigue due to SLE. Patients reported that fatigue was variable in nature in terms of both severity and frequency. Fatigue was described as having an impact on multiple aspects of a patient's life: emotions, cognition, work, activities of daily living, leisure activities, social activities, and family activities. Understanding how patients with SLE describe the symptom of fatigue and how it impacts their lives is the key to better understanding how to measure fatigue in clinical studies evaluating new treatments for SLE.

Development of a conceptual model of health-related quality of life for systemic lupus erythematosus from the patient's perspective.

AIMS: The aim of this study was to evaluate the impact of systemic lupus erythematosus (SLE) on the lives of patients in order to inform the development of a conceptual model. METHODS: Twenty-two participants with SLE (defined as meeting four of the 11 ACR criteria) were recruited for this study. Semi-structured, in-person interviews were conducted with each participant, exploring the symptoms experienced and the impact on the patient's life. Thematic analysis of interview transcripts was conducted in ATLAS.ti software to identify areas of impact and explore the interrelationships between concepts to inform the development of a conceptual model. RESULTS: Almost all participants were female (95%); the sample was diverse in terms of age (mean age of 45.5 years; age range of 20-60 years), ethnicity (59% black/African American) and disease duration. Commonly reported symptoms were pain, fatigue/tiredness and skin problems. Qualitative analysis revealed seven themes relating to the impact of SLE symptoms on patient's Health Related Quality of Life (HRQL): emotions, social, family and leisure activities, daily activities, cognition, appearance, employment activities and independence. The interrelationships between symptoms, impacts and symptom triggers are illustrated in a conceptual model. CONCLUSIONS: The conceptual model illustrates the wide-reaching impact of SLE symptoms on a patient's HRQL, and the potential broad impact of a treatment that improves SLE symptoms.

Can the functional assessment of multiple sclerosis adapt to changing needs? A psychometric validation in patients with clinically isolated syndrome and early relapsing-remitting multiple sclerosis.

BACKGROUND: The Functional Assessment of Multiple Sclerosis (FAMS) is widely used in clinical trial programmes; however, it was developed before the rise in trials targeted at early stage multiple sclerosis (MS) and clinically isolated syndrome (CIS). OBJECTIVE: The aim of this study was to assess the psychometric properties of the FAMS within two clinically distinct populations, CIS and early relapsing-remitting MS (RRMS), and discern the appropriateness of the FAMS within these populations. METHODS: Secondary analysis was conducted on FAMS data from two clinical trials assessing interferon beta-1b in early RRMS and CIS. The statistical analysis assessed the scale acceptability, reliability, validity and responsiveness of the FAMS. Item response theory (IRT) was also conducted on the early RRMS sample in order to assess how well the FAMS discriminated amongst individuals with less severe MS. RESULTS: Results from both trials demonstrated an improvement in the FAMS psychometric properties with increased baseline disease severity. However, high ceiling effects were evident amongst less severe patients, and there was an overall lack of responsiveness to improvement and poor construct validity. IRT also demonstrated its lack of discrimination/sensitivity in early RRMS. CONCLUSIONS: In trials involving patients with early stage RRMS and CIS, modifications to the FAMS based on a qualitative assessment of its content validity in these populations would be required in order to potentially improve the FAMS psychometric properties and sensitivity.

A utility valuation study assessing the impact of postprandial glucose control on quality of life of individuals with type 1 or type 2 diabetes.

BACKGROUND: Consideration of health-related quality of life (HRQOL) in diabetes has been associated with long-term and short-term complications such as hypoglycaemia, but not with short-term glucose control. This study aimed to collect health utilities related to different degrees of poorly controlled postprandial glucose (PPG) and its impact on HRQOL in the UK and in Sweden. METHODS: Three health state descriptions were developed based on literature reviews and interviews with people with diabetes and healthcare professionals, characterising mild, moderate and severe impact of postprandial hyperglycaemic symptoms on HRQOL. Time Trade-Off (TTO) interviews with a 10-year trade-off period were conducted with samples of the UK general public and of Swedish people with diabetes. Mean TTO-derived health state values were expressed on a scale from 0 (death) to 1 (full health). RESULTS: One hundred fifty participants from the general population were interviewed in the UK (57% female, mean age 35 years) and 150 participants with diabetes in Sweden (64% female, mean age 51 years, 42% type 1 and 58% type 2 diabetes). The mean TTO-derived health state values were for the UK and Swedish participants: mild impact of poorly PPG control (0.89/0.76); moderate (0.75/0.71); severe (0.56/0.58). CONCLUSIONS: Glucose lowering treatments associated with improved control over PPG levels could have important benefits to people with type 1 and type 2 diabetes since findings suggest that increasing severity in postprandial hyperglycaemic symptoms is perceived as having significant negative impact on HRQOL of individuals with type 1 or type 2 diabetes.

A discrete choice experiment to determine patient preferences for injection devices in multiple sclerosis.

OBJECTIVE: Relapsing-remitting multiple sclerosis (MS) is usually managed with disease modifying drugs (DMDs), most commonly administered via self-injection. The aim of this study was to estimate the influence that different treatment-related attributes have for MS patients on their choice of MS DMD device. By establishing the relative importance of these characteristics for patients it should be possible to better understand the acceptability of a given device and to optimize the development of future devices. METHODS: A discrete choice experiment (DCE) survey was developed on the basis of a review of published literature. Attributes identified for inclusion in the survey were: ease of use; comfort of use; presence of additional functions, needle visibility; practicality and efficacy. Choice sets were presented as pairs of hypothetical treatments based upon a fractional factorial design. One-hundred device-using MS patients completed the survey online. Analysis was conducted using a mixed-logit approach. RESULTS: Analysis of the DCE data revealed that all attributes significantly predicted treatment choice. Efficacy exhibited the largest effect on treatment selection and this provided context for understanding the magnitude of impact for the other attributes. Reducing the discomfort associated with device use and eliminating the necessity for assembly or drug reconstitution were highly valued. The addition of reminder and time-stamping functions, improved needlestick injury prevention, and reduction in device size were secondary concerns but still deemed desirable. CONCLUSION: Efficacy is of primary importance to MS patients, but characteristics of drug delivery devices can play an important role in treatment decision-making. Not all device characteristics could be included, and results are based upon 100 participants only. Findings suggest there is significant potential value in developing self-injection devices that are not only efficacious but also convenient and comfortable to use. Reducing barriers to adherence could potentially translate into improved treatment outcomes for patients with MS.

Elicitation of health state utilities in neuroendocrine tumours.

BACKGROUND: Neuroendocrine tumours (NETs) are a rare form of neoplasm that can arise in most organs of the body and which share many common pathologic features. Although curative surgery can be conducted for patients with localised disease, once progression occurs and the disease becomes metastatic or un-resectable, treatment aims to extend life and maintain quality-of-life for as long as possible. The aim of the study was to elicit utilities for health state vignettes describing the burdens associated with receiving therapy for advanced NETs. METHODS: Health state vignettes were developed by reviewing published literature and conducting in-depth interviews with patients and clinical experts. These states described the burden associated with both stable and progressive disease, in addition to the experience of a number of serious toxicities commonly associated with treatments (grade III/IV diarrhoea, hand-foot syndrome, hyperglycaemia, nausea/vomiting, pneumonitis, rash, stomatitis, and thrombocytopenia). One hundred members of the UK general public valued the states using the time trade-off methodology to determine utility values. RESULTS: Stable disease had a utility value of 0.77 whilst disease progression was associated with a significant decline in health-related quality-of-life (HRQoL) and a value of 0.61. Toxicities experienced in the context of stable disease exhibited varying degrees of impact, with several being deemed as debilitating as disease progression (such as hand-foot syndrome [0.58] and stomatitis [0.56]). CONCLUSION: Although vignette studies have been criticised for the difficulty in establishing their validity, the collection of health utilities in rare populations is challenging. The findings from this study suggest that advanced NETs is associated with a considerable HRQoL burden, both as a direct result of the disease and the potential of experiencing a number of severe adverse events. These values could assist in future economic evaluation processes.

A blinded comparison of fluticasone propionate with budesonide via powder devices in adult patients with moderate-to-severe asthma: a clinical evaluation.

In Vitro and in vivo data have demonstrated that there are detectable differences between inhaled corticosteroids commonly used to treat asthma. However, controversy still remains as to whether these differences translate into clinical benefits. This 12-week, international, randomized, doubleblind, parallel-group study was undertaken to compare the efficacy and safety of fluticasone propionate (FP) 800 mug daily, administered as a powder via the Diskhaler((R)), and budesonide (BUD) 1600 mug daily, administered using the Turbuhaler((R)), in adult patients with moderate-tosevere asthma. A total of 518 patients participated in the study, 256 of whom received FP and 262 BUD. Assessment of mean morning peak expiratory flow (PEF) over the 12-week treatment period revealed a statistically significant difference in efficacy between FP 800 mug daily and BUD 1600 mug daily in favour of FP (p = 0.003), with an overall improvement of 20.9 l/min with FP compared with 12.4 l/min on BUD. Statistically significant differences in favour of FP were seen over the 12 weeks for mean evening PEF (p = 0.04), diurnal PEF variation (p = 0.03) and percentage predicted PEF (p = 0.003), as well as forced expiratory volume (p = 0.008), forced vital capacity (p = 0.02) and PEF (p = 0.005) measured at clinic visits. The median percentage of symptom-free nights increased over the 12-week study period in both treatment groups, with similar changes seen for the median percentage of days with symptom score < 2, rescue medication use and exacerbations of asthma. The incidence of adverse events was found to be comparable in the two treatment groups. The geometric mean ratios of serum cortisol levels were found to be 1.03 for FP, indicating no mean hypothalamic-pituitary-adrenal axis suppression from baseline, and 0.93 for BUD (p = 0.0002 compared with FP). In summary, FP 800 mug daily showed a greater efficacy/safety ratio in the treatment of moderate-to-severe asthma than BUD 1600 mug daily.

Tenoxicam and paracetamol-codeine combination after oral surgery: a prospective, randomized, double-blind, placebo-controlled study.

We studied 90 adults undergoing surgical removal of at least both lower third molar teeth as day cases under standardized general anaesthesia. Patients were allocated randomly (with stratification for surgeon) to receive tenoxicam 40 mg, tenoxicam 20 mg or placebo i.v. at induction of anaesthesia and orally (effervescent tablets) with food on each of the subsequent 2 days. Panadeine (paracetamol 500 mg-codeine 8 mg) was given before operation and was available as needed for pain thereafter, to a limit of two tablets every 4 h. Nefopam i.v. was also available. Efficacy variables and adverse reactions were assessed over 6 days. Over the 6-day period, patients who received tenoxicam reported less pain on rest (area under the curve; P < 0.05) and less disturbance in sleep (P < 0.01) even though they used fewer Panadeine tablets (P < 0.05). Differences between tenoxicam 40 mg and 20 mg were not significant. There was no significant difference in nefopam requirements or side effects, and no adverse event attributable to the study medication.