Comparative Efficacy and Safety of Peficitinib Versus Tofacitinib and Baricitinib for Treatment of Rheumatoid Arthritis: A Systematic Review and Network Meta-Analysis.

INTRODUCTION: Peficitinib, a Janus kinase (JAK) inhibitor, is approved for clinical use in Japan, Korea, and Taiwan, but head-to-head comparisons versus other JAK inhibitors are lacking. We indirectly compared peficitinib, tofacitinib, and baricitinib for rheumatoid arthritis treatment. METHODS: We searched MEDLINE, Embase, the Cochrane Central Register of Controlled Trials, ClinicalTrials.gov, and congress archives up until February 12, 2019, for randomized controlled trials of peficitinib, tofacitinib, and baricitinib. Efficacy (American College of Rheumatology responses, disease activity scores, modified total Sharp score, Simplified Disease Activity Index [SDAI]) and safety outcomes were compared using a Bayesian network meta-analysis. The Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) consensus was followed for reporting results. A network meta-regression assessed the impact on outcomes of proportions of patients receiving concomitant methotrexate or of Asian ethnicity. RESULTS: The network meta-analysis included 21 randomized controlled trials. At 12 weeks, all evaluable efficacy outcomes were comparable or improved with peficitinib 150 mg and 100 mg once daily, versus baricitinib 2 and 4 mg once daily and tofacitinib 5 mg twice daily. At 24 weeks, efficacy outcomes were comparable or improved for each peficitinib dose versus baricitinib and tofacitinib. Risk of adverse events and serious adverse events at 12 weeks were similar with peficitinib 100 and 150 mg versus baricitinib and tofacitinib. The proportion of patients receiving concomitant methotrexate had no effect on any outcome analyzed, but Asian ethnicity had a positive effect on multiple efficacy outcomes. CONCLUSIONS: Peficitinib had comparable efficacy versus tofacitinib and baricitinib for reduction in disease activity as measured by SDAI, and for reduction in progression of joint damage as measured radiographically. No notable differences in safety outcomes were observed. Further studies are required to better characterize the impact of ethnicity on the efficacy of JAK inhibitors.

Social Preferences for Orphan Drugs: A Discrete Choice Experiment Among the French General Population.

Objectives: While several authors have suggested using a multi-criteria approach for orphan drug assessment and proposed lists of determinants of orphan drug value, studies on social preferences regarding these determinants remain limited. The current study aimed to identify preferences of the French general population regarding attributes characterizing the value of orphan drugs in a discrete choice experiment. Methods: The list of attributes was formed based on a literature search and was refined through expert interviews, a focus group, and a pilot study. The final list included nine attributes: disease-associated disability, disease-associated mortality, number of patients, availability of alternative treatments, treatment impact on disease disability, treatment impact on mortality, treatment safety, uncertainty around therapeutic effect, and annual treatment cost per patient. Members of the General Public were presented with 12 choice sets containing two drug profiles described according to the attributes and an option to fund neither of these treatments. The questionnaire was disseminated online. A conditional logit model with random effects was used to estimate the weight of each attribute. Results: A total of 958 persons participated in the study (48.7% male, mean age: 47.5 years). All attributes except for disease-associated disability had a statistically significant influence on the choices made by participants. The attribute with the highest weight was treatment impact on mortality (p < 0.0001), followed by uncertainty around therapeutic effect (p < 0.0001). The direction of results was generally consistent with intuition: patients preferred a drug with a larger impact on mortality, a larger impact on disability, with mild or no adverse events, with less uncertainty. Although patients appeared to prefer drugs with a lower budget impact, the relationship between patient preferences and costs was more complex. Conclusions: Preferences of the general public between orphan drugs are mostly driven by the impact on mortality and the degree of certainty regarding the available evidence.

Family caregiving in dementia and its impact on quality of life and economic burden in Japan-web based survey.

Background: Dementia has become a growing health-care problem in the rapidly ageing Japanese population. This study assesses the impact of dementia on quality of life, economic burden, and productivity loss. Objective: The objective of this study was to assess the impact of dementia on the Quality of Life (QoL), economic burden, and productivity loss among families living with dementia. Methods: An online survey was conducted among families who lived with relatives with dementia. Demographic data and information about health condition and costs of long-term care and treatment were collected. Participants were asked to answer the EuroQol (EQ-5D-5L) questionnaire, Zarit Burden Interview (ZARIT-8), and Work Productivity and Activity Impairment Questionnaire (WPAI). Multivariate analyses were conducted to assess factors associated with burden by families living with dementia. Results: Six hundred and thirty-five participants completed the survey. Of these participants, 50.5% were primary caregivers. Overall, 78.7% of dementia patients suffered from Alzheimer, and 43.9% needed long-term care. Compared to non-primary caregivers, primary caregivers had lower health utility scores (0.896 vs 0.873; p = 0.02), higher burden of caregiving (ZARIT-8: 21.1 vs 24.5; p < 0.0001), and higher overall work impairment (40.2% vs 20.8%; p < 0.0001), absenteeism (15.3% vs 5.7%; p < 0.0001), and presenteeism-related impairment (33.2% vs 17.3%; p < 0.0001). Conclusion: Families living with dementia caring for a person with dementia experience increased burden. Health policies related to dementia need to be considered not only for patients, but also for their families living with dementia to improve their QoL.

Cost-effectiveness of rivaroxaban versus warfarin for stroke prevention in non-valvular atrial fibrillation in the Japanese healthcare setting.

Aims: This article aimed to examine the cost-effectiveness of rivaroxaban in comparison to warfarin for stroke prevention in Japanese patients with non-valvular atrial fibrillation (NVAF), from a public healthcare payer's perspective.Materials and methods: Baseline event risks were obtained from the J-ROCKET AF trial and the treatment effect data were taken from a network meta-analysis. The other model inputs were extracted from the literature and official Japanese sources. The outcomes included the number of ischaemic strokes, myocardial infarctions, systemic embolisms and bleedings avoided, life-years, quality-adjusted life-years (QALYs), incremental costs and incremental cost-effectiveness ratio (ICER). The scenario analysis considered treatment effect data from the same network meta-analysis.Results: In comparison with warfarin, rivaroxaban was estimated to avoid 0.284 ischaemic strokes per patient, to increase the number of QALYs by 0.535 per patient and to decrease the total costs by ¥118,892 (€1,011.11) per patient (1 JPY = 0.00850638 EUR; XE.com, 7 October 2019). Consequently, rivaroxaban treatment was found to be dominant compared to warfarin. In the scenario analysis, the ICER of rivaroxaban versus warfarin was ¥2,873,499 (€24,446.42) per QALY.Limitations: The various sources of data used resulted in the heterogeneity of the cost-effectiveness analysis results. Although, rivaroxaban was cost-effective in the majority of cases.Conclusion: Rivaroxaban is cost-effective against warfarin for stroke prevention in Japanese patients with NVAF, giving the payer WTP of 5,000,000 JPY.

Impact of Patient-Surgeon Relationship on Patient's Return to Work.

BACKGROUND: Upper-limb injuries and musculoskeletal disorders represent a major economic burden for both patients and society, largely due to limitations in returning to work. We hypothesized that a positive patient-surgeon relationship may facilitate patients' recovery and lead to a faster return to work. METHODS: This longitudinal observational study comprised 219 patients, from 8 French hand trauma centers, who were 18 to 55 years of age and were on sick leave from work because of an injury or musculoskeletal disorder of the upper limb. In addition to instruments measuring patients' functional scores and quality of life, the quality of the patient-surgeon relationship was assessed at enrollment using a specific questionnaire (Q-PASREL [Quality of PAtient-Surgeon RELationship]). Six months after enrollment, the return-to-work status was assessed. Logistic and Cox regression models were developed to identify predictors of return to work (yes/no) and the time off from work in days. RESULTS: Overall, 74% of the patients who returned to work within 6 months after enrollment had a high or medium-high Q-PASREL score, whereas 64% of the patients who were still on sick leave had a low or medium-low Q-PASREL score. The odds of patients with a low or medium-low Q-PASREL score returning to work were, respectively, 95% and 71% lower than the odds of patients with a high score doing so, with a percent difference of 56% (95% confidence interval [CI] = 40% to 71%) for low versus high (odds ratio [OR] = 0.05 [95% CI = 0.02 to 0.13]) and 25% (95% CI = 6% to 44%) for medium-low versus high (OR = 0.29 [95% CI = 0.11 to 0.76]). All Q-PASREL items and scores were significantly associated with return to work. CONCLUSIONS: Patients with a lower Q-PASREL score and more severe disability were less likely to return to work within 6 months and had a longer time off from work. Efforts to improve the quality of patient-surgeon relationships may minimize the duration of sick leaves and accelerate patient recovery. LEVEL OF EVIDENCE: Prognostic Level II. See Instructions for Authors for a complete description of levels of evidence.

Health-care resource use and current treatment of adult atopic dermatitis patients in Japan: A retrospective claims database analysis.

The real-world evidence on the profiles of patients suffering from atopic dermatitis (AD) in Japan is sparse. A retrospective claim database analysis was conducted to estimate the health-care resource use (HCRU) and current AD treatment. Data from October 2013 to September 2016 were extracted from the JMDC (Tokyo, Japan) claims database. HCRU was assessed by a comparison of AD patients and matched non-AD controls. A multivariate analysis was performed to estimate HCRU attributable to AD. AD patients (n = 39 893) have more claims of certain diagnoses such as rhinitis, viral and fungal infections, sleep disorders and conjunctivitis as well as higher HCRU (outpatient visits, prescriptions of AD-related and non-AD-related medications, phototherapy, laboratory tests) than matched non-AD controls (n = 39 893). Treatment pattern analysis included treatment-naive patients (n = 8478) and previously treated AD patients (n = 30 109). Approximately 20% of previously treated patients were on the continuous systemic treatment during 18-month follow up. Systemic corticosteroids were the most frequently used systemic treatments. Oral cyclosporin was less frequently used in both groups, but for the longest duration. Almost half of previously treated patients with oral cyclosporin continued treatment for more than 3 months. In conclusion, HCRU was higher in AD patients than non-AD controls, indicating a high burden of the disease imposed on AD patients. Continuous administration of systemic treatment, such as oral cyclosporin, systemic corticosteroids and phototherapy, observed in AD patients sheds light on the difficulties of managing AD in Japanese clinical practise.

Management and clinical practice of multiple face and scalp actinic keratosis in France.

Background: Actinic keratosis (AK) is characterized by the occurrence of thick and scaly skin areas caused by damage from ultraviolet radiation. The management of AK aims to reduce lesions and prevent their recurrence by regular monitoring. French guidelines, last updated in 2009, reflect European guidelines for the management of face and scalp AK. However, they do not address all current, available options. Objective: To assess the management of face and scalp AK in French clinical practice. Methods: A two-part online questionnaire comprising a survey among French dermatologists and an analysis of patient medical records was performed to describe AK patients treated with topical therapy, patients' profiles, and characteristics of the affected lesion areas. Results: Decisions for topical treatments for face and scalp AK made by dermatologists were mainly driven by the lesion size. According to the last 10 patients they have seen, dermatologists were prescribing physical therapy in 53% of the cases, a combination of topical and physical therapy in 27% and topical only in 20%. Patient records revealed the average surface area targeted for treatment was 139 ± 113cm2. Conclusions: Discrepancies between the guidelines on the treatment of face and scalp AK and clinical practice exist. Further research may help to standardize the treatment.

Estimation of the costs attributable to vitamin K antagonist treatment in patients with non-valvular atrial fibrillation from a French societal perspective.

Background: Little is known about the costs associated with vitamin K antagonist (VKA) treatment in patients with non-valvular atrial fibrillation (NVAF) in France. Objectives: To evaluate monthly per-patient costs attributable to VKA treatment in NVAF patients from a French societal perspective. Study design: Retrospective data were obtained from 7 international normalised ratio (INR) monitoring centres in France. Patients older than 18 years of age with NVAF treated with VKA were recruited. Additional patient-level data assessing resource use corresponding with VKA treatment were collected via self-completed questionnaires. Unit costs applicable to 2015 were multiplied by resource use and summed to generate VKA treatment costs. Results: 363 patients were included; 53% were men. The majority of patients received fluindione (72%). The number of INR tests per patient per month was 1.69 (95% CI, 1.59-1.80). The monthly patient cost was €39.72 (€36.23-43.21) from the French societal perspective. Direct medical costs comprised 76% of overall costs, with drug costs representing 7.4% (€2.4); direct non-medical and indirect costs comprised 10% and 14% respectively. Conclusions: Costs associated with VKA treatment in NVAF cannot be estimated only with drug costs. When direct and indirect attributable costs associated with VKA treatment are considered, the VKA treatment costs are more substantial.

Burden of Anemia in Chronic Kidney Disease Patients in Japan: A Literature Review.

Anemia is a common complication for patients with chronic kidney disease (CKD) and is associated with cardiovascular comorbidities and reduced quality of life. The incidence of anemia increases as kidney function declines and affects approximately 32% of Japanese patients with stage 3-5 CKD. This review examined the current literature on anemia in CKD patients in Japan to provide an overview of the burden of anemia in CKD. Medline, Embase, and Igaku Chuo Zasshi databases were searched to identify relevant manuscripts and abstracts published from 2004 onward. The population included CKD patients with anemia, and the outcomes of interest were epidemiology, economic, humanistic, and treatment patterns. Observational studies, database analysis, and economic evaluation studies were included in the analysis. A total of 1151 references were identified, and 50 were eligible for final review. Economic burden was reported in most studies (n = 37) followed by treatment patterns (n = 26), and epidemiological (n = 25) and humanistic (n = 1) burdens. Prevalence of anemia varied largely (0-95%) based on the different definitions of anemia, and increased with CKD severity. Higher mortality was associated with erythropoiesis-stimulating agent (ESA) resistance and lower hemoglobin levels among patients treated with ESA. Drug dosage was the most reported economic burden (n = 33), followed by medical, and non-medical outcomes. Costs associated with anemia were considerable and depended on dialysis status and ESA treatment. Only one study reported data on quality of life, suggesting that further investigation on the humanistic burden of anemia in CKD is needed.

Key drivers for market penetration of biosimilars in Europe.

Background & Objectives: Potential drivers and barriers of biosimilar uptake were mainly analysed through qualitative approaches. The study objective was to conduct a quantitative analysis and identify drivers of biosimilar uptake of all available biosimilars in the European Union (EU). Methods: A three-step process was established to identify key drivers for the uptake of biosimilars in the top 10 EU member states (MS) pharmaceutical markets (Belgium, France, Germany, Greece, Hungary, Italy, Poland, Spain, Sweden, and the UK): (1) literature review to identify incentive policies in place to enhance biosimilars adoption; (2) assessment of biosimilar market dynamics based on database analysis; (3) regression model analysis on price using the following explicative variables: incentive policies; price difference between the biosimilar and the originator product; distribution channel; generic uptake and generic price cut; pharmaceutical expenditure per capita; and market competition. Results: At the study cut-off date, 20 biosimilars were available on the market. Incentive policies applied to biosimilars were found to be heterogeneous across countries, and uptakes of biosimilars were also very heterogeneous between different therapeutic classes and countries. Results from the model demonstrated that incentive policies and the date of first biosimilar market entry were correlated to biosimilar uptake. Pharmaceutical expenditure per capita and the highest generic uptake were inversely correlated with biosimilar uptake. Average generic price discount over originator and the number of biosimilars showed a trend toward statistical significance for correlation with biosimilar uptake, but did not reach the significance threshold. Biosimilar price discount over original biologic price, the number of analogues, and the distribution channel were not correlated with the biosimilar uptake. Conclusions: Understanding drivers of biosimilar uptake becomes a critical issue to inform policy decision-makers. This study showed that incentive policies to enhance uptake remain an important driver of biosimilar penetration, while biosimilar price discounts have no impact. Future research is warranted when the biosimilar market gains maturity.

The quality of life of Spanish patients with Huntington's disease measured with H-QoL-I and EQ-5D.

BACKGROUND AND OBJECTIVE: Huntington's disease (HD) is an inherited neurodegenerative disorder that heavily affects the patient's motor, cognitive, and psychological functions. Yet, very few studies have measured the impact of this disease on the patient's health-related quality of life (HRQoL) with specific and validated instruments. The aim of this study was to explore the impact of HD on the HRQoL of Spanish HD patients using the self-reported, Huntington Quality of Life Instrument (H-QoL-I) and the generic instrument EuroQoL five dimensions (EQ-5D-3L) and thereafter compare the results obtained with the two instruments. METHODS: Fifty-five patients and an equal number of caregivers participated. Patient assessments included the questionnaires of the Huntington Self-Assessment Instrument's four parts: background information assessment, Huntington clinical self-reported instrument, disease-specific HRQoL assessment (H-QoL-I instrument) and Huntington resource utilisation interview, and the EQ-5D-3L questionnaire. Levels of disease severity were also determined based on the Unified Huntington's Disease Rating Scale that was completed by caregivers. Pearson's correlation tests were computed between H-QoL-I and EQ-5D-3L scores. RESULTS: The scores obtained with the H-QoL-I instrument showed that motor dimension was the most altered followed by the psychological dimension while the social dimension was the least altered. Increase of disease severity resulted in lower patient QoL. The usual activities and anxiety/depression were the most severely altered dimensions according to the EQ-5D-3L results. Mobility was also altered to a great extent while pain was the least altered dimension. All correlations between H-QoL-I and EQ-5D-3L scores were moderate to high and statistically significant (p<0.01) with the exception of the correlation between H-QoL-I socialising score and EQ-5D-3L anxiety score. The highest correlations were found between H-QoL-I motor score and three EQ-5D-3L scores: mobility, self-care, and usual activity. CONCLUSIONS: The quality of life of the Spanish HD patients included in this study was severely affected by HD as demonstrated by the results of the generic EQ-5D-3L and the specific H-QoL-I instruments, which showed considerable impact of the disease on the motor and psychological functions. The H-QoL-I instrument was able to discern psychological and motor functioning dimensions that were altered by the disease with more specificity and accuracy than the generic instrument.

Cost-effectiveness of droxidopa in patients with neurogenic orthostatic hypotension: post-hoc economic analysis of Phase 3 clinical trial data.

OBJECTIVE: Falls are associated with neurogenic orthostatic hypotension (nOH) and are an economic burden on the US healthcare system. Droxidopa is approved by the US FDA to treat symptomatic nOH. This study estimates the cost-effectiveness of droxidopa vs standard of care from a US payer perspective. METHODS: A Markov model was used to predict numbers of falls and treatment responses using data from a randomized, double-blind trial of patients with Parkinson's disease and nOH who received optimized droxidopa therapy or placebo for 8 weeks. The severity of falls, utility values, and injury-related costs were derived from published studies. Model outcomes included number of falls, number of quality-adjusted life-years (QALYs), and direct costs. Incremental cost-effectiveness ratios (ICERs) were calculated. Outcomes were extrapolated over 12 months. RESULTS: Patients receiving droxidopa had fewer falls compared with those receiving standard of care and gained 0.33 QALYs/patient. Estimated droxidopa costs were $30,112, with estimated cost savings resulting from fall avoidance of $14,574 over 12 months. Droxidopa was cost-effective vs standard of care, with ICERs of $47,001/QALY gained, $24,866 per avoided fall with moderate/major injury, and $1559 per avoided fall with no/minor injury. The main drivers were fall probabilities and fear of fall-related inputs. LIMITATIONS: A limitation of the current study is the reliance on falls data from a randomized controlled trial where the placebo group served as the proxy for standard of care. Data from a larger patient population, reflecting 'real-life' patient use and/or comparison with other agents used to treat nOH, would have been a useful complement, but these data were not available. CONCLUSION: Using Markov modeling, droxidopa appears to be a cost-effective option compared with standard of care in US clinical practice for the treatment of nOH.

Responder and health-related quality of life analyses in men with lower urinary tract symptoms treated with a fixed-dose combination of solifenacin and tamsulosin oral-controlled absorption system: results from the NEPTUNE study.

OBJECTIVE: To evaluate the effect of a fixed-dose combination (FDC) of solifenacin and an oral-controlled absorption system (OCAS™) formulation of tamsulosin (TOCAS) on health-related quality of life (HRQoL) in men with lower urinary tract symptoms associated with benign prostatic hyperplasia (LUTS/BPH). PATIENTS AND METHODS: Men with moderate-to-severe storage symptoms and voiding symptoms were treated for 12 weeks with a FDC of solifenacin 6 or 9 mg plus TOCAS (0.4 mg), TOCAS monotherapy (0.4 mg) or placebo in a randomised, double-blind study (NEPTUNE). The co-primary endpoints were Total Urgency Frequency Score (TUFS) and total International Prostate Symptom Score (IPSS). HRQoL was assessed by several secondary endpoints: IPSS QoL index, overactive bladder questionnaire (OAB-q), and Patient Global Impression (PGI) scale. The correlation between symptom improvement (TUFS) and HRQoL was assessed by Spearman rank correlation coefficients. Single and double responder analyses, using subjective and objective measures, were also performed. RESULTS: In the responder analyses, men treated with a FDC of solifenacin 6 mg plus TOCAS consistently had significantly improved outcomes compared with placebo (8/8 responder analyses performed) and TOCAS (6/8 responder analyses performed). There was a significant correlation (P < 0.001) between the reduction in TUFS and the improvement in HRQoL defined by IPSS QoL score, OAB-q symptom bother score, PGI overall bladder symptoms and PGI general health. CONCLUSIONS: In men with LUTS/BPH who have moderate-to-severe storage symptoms and voiding symptoms, the reduction in symptoms with a once-daily FDC of solifenacin and TOCAS was associated with consistent patient-relevant improvements in HRQoL compared with placebo and TOCAS monotherapy.

A chart review of management of ischemic stroke patients in Germany.

BACKGROUND: Ischemic stroke (IS) poses physical, emotional, and economic burdens on both patients and the healthcare system in Germany. However, the management of IS is not well described, especially after hospital discharge. In this study, we aim to describe the management of IS at onset, admission, and during follow-up. METHODS: German general practitioners (GPs) (n=40) extracted data on patient characteristics, hospitalizations, discharge, and ambulatory care from both GPs patient databases and hospital letters. Descriptive analyses were conducted. RESULTS: The sample included 185 patients with a mean age of 70 years [standard deviation (SD)=11.7]. Most patients (63%) contacted the Emergency Medical Services, while 36% contacted their GPs. The majority of patients were hospitalized within 1 h from onset, and the length of stay was on average 14 days. Half of the patients (50%) were admitted to the stroke unit, and 16% of patients received thrombolysis treatment with 2 h (SD=2.6) of time to treatment. Of the admitted patients, 32% were discharged to their homes, while the remaining patients were discharged to nursing homes (16.2%) and rehabilitation centers (47.6%). During the 12 months follow-up, 22% of patients were re-hospitalized and patients visited their GP (11.7 times), psychologist or psychiatrist (9.5 times), and neurologist (2.2 times). Death rate after stroke event was 13%. CONCLUSION: The rate of patients who received thrombolysis is lower than the optimal rate in Germany. More research is needed to determine the factors that could predict the utilization of thrombolysis treatment.

Ischemic stroke management in West Scotland: a chart review.

BACKGROUND: Little information is available about the long-term management of ischemic stroke (IS) in West Scotland. In this study we aim to describe the management of IS at onset, admission, and during follow-up among patients who survived an IS event. METHODS: General practitioners (GPs) (n=20) were randomly selected to recruit IS patients and extract data about patient characteristics, hospitalizations, discharge, and ambulatory care from GP databases, hospital letters, and direct contact with patients and their relatives. Descriptive analyses were conducted. RESULTS: One hundred and one patients were included, with a mean age of 65.6±13.4. About half of the patients contacted their GPs at the time of onset (45.4%). Cardiovascular history was prevalent in 29.7% of cases, and 14% of all cases were recurrences. Of the patients, 89 (88%) were hospitalized with mean length of stay (LOS) 11.8 days. Treatment was administered on average within 12.9 hours of admission and 23.6% of the admitted patients received thrombolytic treatment. During the 1-year follow-up period, 33.6% of patients were rehospitalized and the mean LOS was 15.1±29.5 days. Further, patients on average sought nursing care (10.9%), physical therapy (45.5%), occupational therapy (27.7%), speech therapy (12.9%), and professional caregivers (12%). CONCLUSION: The health-care resource utilization of IS patients is a major driver of economic burden.

Further evidence of reliability and validity of the Huntington's disease quality of life battery for carers: Italian and French translations.

BACKGROUND: Existing research suggests that family caregivers of persons with Huntington's disease (HD) face a distinct series of problems, linked to the complex nature of the disease. Aubeeluck and Buchanan (Clin Genet, 71(5):434-445, 2007) developed and validated a disease-specific measure used to explore caregivers quality of life and assess the efficacy of therapeutic interventions. This current study builds on this research through the validation of French and Italian translations of the Huntington's disease quality of life battery for carers (HDQoL-C). METHOD: A total of 301 family carers completed the HDQoL-C. Participants were recruited through the "Euro-HDB" study which is measuring the burden in HD across Europe and the USA. RESULTS: Factor analysis demonstrated good internal consistency, reliability and congruent validity. Carers who cared for patients with less clinically severe symptoms reported significantly better QoL than carers of patients with more clinically severe symptoms. DISCUSSION: Findings indicate the HDQoL-C is multi-lingual, multi-cultural and easily applicable in other languages.

Validation of the first quality-of-life measurement for patients with Huntington's disease: the Huntington Quality of Life Instrument.

Health-related quality-of-life instruments are critical for assessing disease burdens. Generic tools allow comparison between diseases but do not discriminate between disease severities. Specific tools also tend to be more sensitive. No specific tool is available to assess quality of life in patients with Huntington's disease (HD). In the context of the European study on HD burden, a specific tool was created: the Huntington Quality of Life Instrument (H-QoL-I). The aim of this study was to optimize the content and validate the H-QoL-I. After a semistructured interview with patients, caregivers and HD specialists, we conducted a patient focus group. A self-reported questionnaire was then developed in French and Italian. A total of 252 patients were recruited to answer the questionnaire. Face, internal and external validities were examined using a variety of methods. The shortened H-QoL-I that resulted from the successive analyses comprises 11 items, which are divided into three dimensions: motor functioning (four items), psychology (four items) and socializing (three items). These three domains were identified as being essential to cover the full domain of the quality of life for patients affected by HD. The H-QoL-I showed an acceptable reliability (Cronbach's α>0.84). Factor analyses demonstrated satisfactory construct validity. Moreover, the item internal consistency and item discriminant validity criteria were fulfilled. No differential item functioning was detected. External validity supported the scale's robustness. These data support the validity of the H-QoL-I in patients with HD.