Quality of life measurement in vitiligo. Position statement of the European Academy of Dermatology and Venereology Task Force on Quality of Life and Patient Oriented Outcomes with external experts.

Members of the European Academy of Dermatology and Venereology (EADV) Task Force on Quality of Life (QoL) and Patient Oriented Outcomes reviewed the instruments available for health-related (HR) QoL assessment in vitiligo and together with external vitiligo experts (including representatives of the EADV Vitiligo Task Force) have made practical recommendations concerning the assessment of QoL in vitiligo patients. The Dermatology Life Quality Index (DLQI) was the most frequently used HRQoL instrument, making comparison of results between different countries possible. Several vitiligo-specific instruments were identified. The vitiligo Impact Scale (VIS) is an extensively validated vitiligo-specific HRQoL instrument with proposed minimal important change and clinical interpretation for VIS-22 scores. VIS-22 was developed for use in India, where there are some specific cultural beliefs concerning vitiligo. The EADV Task Force on QoL and Patient Oriented Outcomes recommends use of the DLQI and the Children's Dermatology Life Quality Index (CDLQI) as dermatology-specific instruments in vitiligo. There is a strong need for a valid (including cross-cultural validation) vitiligo-specific instrument that can be either a new instrument or the improvement of existing instruments. This validation must include the proof of responsiveness.

Validation of the German version of the Family Reported Outcome Measure (FROM-16) to assess the impact of disease on the partner or family member.

BACKGROUND: The Family Reported Outcome Measure (FROM-16) assesses the impact of a patient's chronic illness on the quality of life (QoL) of the patient's partner or family members. The aim of the study was to translate, explore the structure of and validate the FROM-16. METHODS: The questionnaire was translated from English into German (forward, backward, four independent translators). Six interviews with family members were conducted to confirm the questionnaire for linguistic, conceptual, semantic and experiential equivalence and its practicability. The final German translation was tested for internal consistency, reproducibility and test validity. Criterion validity was tested by correlating the scores of the FROM-16 and the Global Health Scale (GHS). Principal component analysis, factor analysis, and confirmatory factor analysis was used to assess the questionnaire's structure and its domains. Reliability and reproducibility were tested computing the intraclass correlation coefficient (ICC) using one sample t-test for testing the hypothesis that the difference between the scores was not different from zero. RESULTS: Overall, 83 family members (61% female, median age: 61 years) completed the questionnaire at two different times (mean interval: 22 days). Internal consistency was good for the FROM-16 scores (Cronbach's α for total score = 0.86). In those with stable GHS, the ICC for the total score was 0.87 and the difference was not different from zero (p = 0.262) indicating reproducible results. A bi-factor model with a general factor including all items, and two sub-factors comprising the items from the original 2-factor construct had the best fit. CONCLUSIONS: The German FROM-16 has good reliability, test validity and practicability. It can be considered as an appropriate and generic tool to measure QoL of a patient's partner or family member. Due to the presence of several cross-loadings we do not recommend the reporting of the scores of the two domains proposed for the original version of FROM-16 when using the German version. Thus, in reporting the results emphasis should be put on the total score. TRIAL REGISTRATION: Retrospectively registered: DRKS00021070.

Exploring the EQ-5D Dimension of Pain/Discomfort in Dermatology Outpatients from a Multicentre Study in 13 European Countries.

Pain and discomfort are important symptoms in dermatology. The aim of this cross-sectional, multicentre study was to describe the prevalence of pain/discomfort and its associations in patients with several dermatological conditions across 13 European countries. The outcome was the prevalence of pain/discomfort according to a question of the EQ-5D questionnaire. Data collected from November 2011 to February 2013 were complete for 3,509 consecutive outpatients. Moderate or extreme pain/discomfort was reported by 55.5% of patients and 31.5% of controls with no skin conditions. The highest proportions were reported by patients with hidradenitis suppurativa (92.9%), leg ulcer (81.4%), prurigo (80%) and lichen planus (75.6%). Pain/discomfort was associated with older age, low educational level, clinical severity, flare on scalp or hands, itch, depression, anxiety, low quality of life, and thoughts of suicide. It is important to enquire specifically about pain/discomfort during clinical consultations and to address it when planning a patient's care.

Presenting signs and patient co-variables in Gaucher disease: outcome of the Gaucher Earlier Diagnosis Consensus (GED-C) Delphi initiative.

BACKGROUND: Gaucher disease (GD) presents with a range of signs and symptoms. Physicians can fail to recognise the early stages of GD owing to a lack of disease awareness, which can lead to significant diagnostic delays and sometimes irreversible but avoidable morbidities. AIM: The Gaucher Earlier Diagnosis Consensus (GED-C) initiative aimed to identify signs and co-variables considered most indicative of early type 1 and type 3 GD, to help non-specialists identify 'at-risk' patients who may benefit from diagnostic testing. METHODS: An anonymous, three-round Delphi consensus process was deployed among a global panel of 22 specialists in GD (median experience 17.5 years, collectively managing almost 3000 patients). The rounds entailed data gathering, then importance ranking and establishment of consensus, using 5-point Likert scales and scoring thresholds defined a priori. RESULTS: For type 1 disease, seven major signs (splenomegaly, thrombocytopenia, bone-related manifestations, anaemia, hyperferritinaemia, hepatomegaly and gammopathy) and two major co-variables (family history of GD and Ashkenazi-Jewish ancestry) were identified. For type 3 disease, nine major signs (splenomegaly, oculomotor disturbances, thrombocytopenia, epilepsy, anaemia, hepatomegaly, bone pain, motor disturbances and kyphosis) and one major co-variable (family history of GD) were identified. Lack of disease awareness, overlooking mild early signs and failure to consider GD as a diagnostic differential were considered major barriers to early diagnosis. CONCLUSION: The signs and co-variables identified in the GED-C initiative as potentially indicative of early GD will help to guide non-specialists and raise their index of suspicion in identifying patients potentially suitable for diagnostic testing for GD.

The burden of disease in pyruvate kinase deficiency: Patients' perception of the impact on health-related quality of life.

OBJECTIVES: This study explored how signs and symptoms of pyruvate kinase (PK) deficiency, a rare hemolytic anemia caused by mutations in the PKLR gene, impacts patients' health-related quality of life (HRQoL). METHODS: Interviews with 21 adults with PK deficiency in the United States, Netherlands, and Germany were conducted. Participants were asked to describe signs, symptoms, and impacts of the disease on their daily lives. Interviews were transcribed and analyzed using qualitative analysis methods. RESULTS: The most common signs and symptoms reported were yellow eyes (n = 19), tiredness (n = 18), yellow skin (n = 17), fatigue (n = 15), low energy (n = 13), and shortness of breath (n = 13). Furthermore, signs and symptoms of PK deficiency negatively impact the ability to perform physical activities, appearance, social activities, emotional states, activities of daily living, leisure activities, work and/or school, sleep, and cognitive states of those living with PK deficiency. A conceptual model is presented that further demonstrates the profound impact that signs and symptoms of PK deficiency have on dimensions of patients' HRQoL. CONCLUSIONS: This is the first study that provides patient perspective on the burden of living with PK deficiency and lays the foundation for future studies to examine the effect of pharmacologic interventions on overall HRQoL for patients living with PK deficiency.

The Role of Therapy in Impairing Quality of Life in Dermatological Patients: A Multinational Study.

Skin disease and its therapy affect health-related quality of life (HRQoL). The aim of this study was to measure the burden caused by dermatological therapy in 3,846 patients from 13 European countries. Adult outpatients completed questionnaires, including the Dermatology Life Quality Index (DLQI), which has a therapy impact question. Therapy issues were reported by a majority of patients with atopic dermatitis (63.4%), psoriasis (60.7%), prurigo (54.4%), hidradenitis suppurativa (54.3%) and blistering conditions (53%). The largest reduction in HRQoL attributable to therapy, as a percentage of total DLQI, adjusted for confounders, was seen in blistering conditions (10.7%), allergic/drug reactions (10.2%), psoriasis (9.9%), vasculitis/immunological ulcers (8.8%), atopic dermatitis (8.7%), and venous leg ulcers (8.5%). In skin cancer, although it had less impact on HRQoL, the reduction due to therapy was 6.8%. Treatment for skin disease contributes considerably to reducing HRQoL: the burden of dermatological treatment should be considered when planning therapy and designing new dermatological therapies.

Determinants of Psychosocial Health in Psoriatic Patients: A Multi-national Study.

There are limited data on the differences in the impact of psoriasis between various countries with respect to quality of life (QoL). The aim of this study was to explore the psychosocial health of patients with psoriasis in different European countries. A total of 682 patients were recruited in 13 European countries. All patients completed a questionnaire regarding socio-demographic information, negative life events, suicidal ideation and satisfaction with their dermatologist. Depression and anxiety were assessed with the Hospital Anxiety and Depression Scale (HADS), and QoL with the Dermatology Life Quality Index (DLQI) and EuroQoL (EQ-5D). The lowest anxiety and depression scoring was noted in patients from Denmark, the lowest level of impairment in QoL in subjects from Spain, and the highest level of impairment in QoL in patients from Italy. The most relevant parameters influencing patients' well-being were severity of pruritus and satisfaction with their dermatologist. The level of anxiety and depression symptoms correlated significantly with suicidal ideation.

Attachment Styles of Dermatological Patients in Europe: A Multi-centre Study in 13 Countries.

Attachment styles of dermatological outpatients and satisfaction with their dermatologists were investigated within the framework of a multicentre study conducted in 13 European countries, organized by the European Society for Dermatology and Psychiatry. Attachment style was assessed with the Adult Attachment Scale. Patient satisfaction with the dermatologist was assessed with an 11-degree scale. A total of 3,635 adult outpatients and 1,359 controls participated in the study. Dermatological outpatients were less able to depend on others, were less comfortable with closeness and intimacy, and experienced similar rates of anxiety in relationships as did the controls. Participants who had secure attachment styles reported stressful life events during the last 6 months significantly less often than those who had insecure attachment styles. Patients with secure attachment styles tended to be more satisfied with their dermatologist than did insecure patients. These results suggest that secure attachment of dermatological outpatients may be a protective factor in the management of stress.

Impairment of Sexual Life in 3,485 Dermatological Outpatients From a Multicentre Study in 13 European Countries.

Skin conditions may have a strong impact on patients' sexual life, and thus influence personal relationships. Sexual issues are difficult to discuss directly in clinical practice, and a mediated instrument may be useful to capture such information. In this study item 9 of the Dermatology Life Quality Index was used to collect information on sexual impact of several skin conditions in 13 European countries. Among 3,485 patients, 23.1% reported sexual problems. The impairment was particularly high in patients with hidradenitis suppurativa, prurigo, blistering disorders, psoriasis, urticaria, eczema, infections of the skin, or pruritus. Sexual impact was strongly associated with depression, anxiety, and suicidal ideation. It was generally more frequent in younger patients and was positively correlated with clinical severity and itch. It is important to address the issue of sexual well-being in the evaluation of patients with skin conditions, since it is often linked to anxiety, depression, and even suicidal ideation.

Equivalence of electronic and paper-based patient-reported outcome measures.

AIM: Electronic formats (ePROs) of paper-based patient-reported outcomes (PROs) should be validated before they can be reliably used. This review aimed to examine studies investigating measurement equivalence between ePROs and their paper originals to identify methodologies used and to determine the extent of such validation. METHODS: Three databases (OvidSP, Web of Science and PubMed) were searched using a set of keywords. Results were examined for compliance with inclusion criteria. Articles or abstracts that directly compared screen-based electronic versions of PROs with their validated paper-based originals, with regard to their measurement equivalence, were included. Publications were excluded if the only instruments reported were stand-alone visual analogue scales or interactive voice response formats. Papers published before 2007 were excluded, as a previous meta-analysis examined papers published before this time. RESULTS: Fifty-five studies investigating 79 instruments met the inclusion criteria. 53 % of the 79 instruments studied were condition specific. Several instruments, such as the SF-36, were reported in more than one publication. The most frequently reported formats for ePROs were Web-based versions. In 78 % of the publications, there was evidence of equivalence or comparability between the two formats as judged by study authors. Of the 30 publications that provided preference data, 87 % found that overall participants preferred the electronic format. CONCLUSIONS: When examining equivalence between paper and electronic versions of PROs, formats are usually judged by authors to be equivalent. Participants prefer electronic formats. This literature review gives encouragement to the further widespread development and use of ePROs.

The psychological burden of skin diseases: a cross-sectional multicenter study among dermatological out-patients in 13 European countries.

The contribution of psychological disorders to the burden of skin disease has been poorly explored, and this is a large-scale study to ascertain the association between depression, anxiety, and suicidal ideation with various dermatological diagnoses. This international multicenter observational cross-sectional study was conducted in 13 European countries. In each dermatology clinic, 250 consecutive adult out-patients were recruited to complete a questionnaire, reporting socio-demographic information, negative life events, and suicidal ideation; depression and anxiety were assessed with the Hospital Anxiety and Depression Scale. A clinical examination was performed. A control group was recruited among hospital employees. There were 4,994 participants--3,635 patients and 1,359 controls. Clinical depression was present in 10.1% patients (controls 4.3%, odds ratio (OR) 2.40 (1.67-3.47)). Clinical anxiety was present in 17.2% (controls 11.1%, OR 2.18 (1.68-2.82)). Suicidal ideation was reported by 12.7% of all patients (controls 8.3%, OR 1.94 (1.33-2.82)). For individual diagnoses, only patients with psoriasis had significant association with suicidal ideation. The association with depression and anxiety was highest for patients with psoriasis, atopic dermatitis, hand eczema, and leg ulcers. These results identify a major additional burden of skin disease and have important clinical implications.

Evaluation of the Gulf Cooperation Council Centralized Procedure: The Way Forward.

The aim of the study was to evaluate the Gulf Cooperation Council (GCC) centralized regulatory review process. Regulatory review times-including submission and application dates for new active substances (NASs) and existing active substances (EASs) using a standardized template for the period of 2006 to 2010-were collected directly from the GCC office located in Riyadh, Saudi Arabia. A total of 413 products (96 NASs and 317 EASs) were approved during the period, with an overall significant increase in the EASs ( P < .001). The median approval times increased from 107 calendar days in 2006 to 265 in 2010 ( P < .001). The lowest approval time was for EASs submitted by the Gulf companies (134 days) and the longest for NASs submitted by international companies (346 days) ( P < .001). These data were also analyzed according to therapeutic classes and dosage forms. The results also showed that the lowest number (n = 16) approved during the period was in 2010, and this was due to a major regulatory change implementing the International Conference on Harmonisation product stability guideline for the region. The findings indicate that the delay and the wide range in approval times could be reduced by utilizing a standard assessment template for product review and the implementation of a clock stop system for company responses to questions from the GCC central registration committee. Furthermore, using information technology tools would speed up the registration process rather than the manual exchange of product registration files between the executive office and the member states.

The development and validation of the major life changing decision profile (MLCDP).

BACKGROUND: Chronic diseases may influence patients taking major life changing decisions (MLCDs) concerning for example education, career, relationships, having children and retirement. A validated measure is needed to evaluate the impact of chronic diseases on MLCDs, improving assessment of their life-long burden. The aims of this study were to develop a validated questionnaire, the "Major Life Changing Decision Profile" (MLCDP) and to evaluate its psychometric properties. METHODS: 50 interviews with dermatology patients and 258 questionnaires, completed by cardiology, rheumatology, nephrology, diabetes and respiratory disorder patients, were analysed for qualitative data using Nvivo8 software. Content validation was carried out by a panel of experts. The first version of the MLCDP was completed by 210 patients and an iterative process of multiple Exploratory Factor Analyses and item prevalence was used to guide item reduction. Face validity and practicability was assessed by patients. RESULTS: 48 MLCDs were selected from analysis of the transcripts and questionnaires for the first version of the MLCDP, and reduced to 45 by combination of similar themes. There was a high intraclass correlation coefficient (0.7) between the 13 members of the content validation panel. Four more items were deleted leaving a 41-item MLCDP that was completed by 210 patients. The most frequently recorded MLCDs were decisions to change eating habits (71.4%), to change smoking/drinking alcohol habits (58.5%) and not to travel or go for holidays abroad (50.9%).Factor analysis suggested item number reduction from 41 to 34, to 29, then 23 items. However after taking into account item prevalence data as well as factor analysis results, 32 items were retained. The 32-item MLCDP has five domains education (3 items), job/career (9), family/relationships (5), social (10) and physical (5). The MLCDP score is expressed as the absolute number of decisions that have been affected. CONCLUSIONS: The 32-item (5 domains) MLCDP has been developed as an easy to complete generic tool for use in clinical practice and for quality of life and epidemiological research. Further validation is required.

A review of the health-related quality of life and economic impact of Parkinson's disease.

Parkinson's disease (PD) is a chronic, neurodegenerative disease that places a substantial burden on patients, their families and carers, as well as on society as a whole. PD can severely affect the health-related quality of life (HR-QOL) of both patients and their carers and, as the disease progresses, HR-QOL deteriorates. This review aims to critically evaluate the literature on a number of important aspects that influence HR-QOL in relation to PD. Factors associated with a negative impact and ways to improve HR-QOL are highlighted, and tools for HR-QOL assessment reviewed. The economic impact of PD and related cost-effectiveness studies are also reviewed. Over the course of the disease, patients with PD experience changes in their HR-QOL that are affected by factors such as depression, motor complications, education and surgery. However, a lack of uniformity in the choice of HR-QOL tools used in studies makes comparison of results difficult. Research on motor fluctuations and dyskinesias has shown conflicting results, whereas it is clear from the available data that depression needs to be more clearly recognised and treated. Inequality in the numbers of men and women receiving surgery still needs to be addressed and, again, in this area there is a lack of uniformity with respect to assessment for surgery. Education programmes have been shown to be successful in improving HR-QOL, although more research is needed about how to introduce such programmes to all PD patients. In particular, there has been little detailed research into young-onset PD and juvenile patients to assess the true impact of the disease on their HR-QOL. The literature has also shown that PD can affect the HR-QOL of the carer, which may have a 'knock-on' effect for the patient. The HR-QOL of carers needs more attention because these individuals can significantly reduce the burden that would otherwise fall on the health services in terms of cost and care. Research shows that the economic costs of PD are high, particularly for patients in advanced stages of the disease and those with motor complications. Although carer burden is a major source of costs, this is not factored into cost-effectiveness analyses. Furthermore, because too few studies use quality-adjusted life years as their health outcome, particularly in studies of the costs of surgery, comparison of costs of treatments is difficult. The review highlights the need for HR-QOL tools such as the EuroQol-5D to be used together with disease-specific tools to provide the most comprehensive picture of the costs and impact of PD.A recent upsurge in published literature on PD resulting from increased interest in HR-QOL issues has led to an at times overwhelming amount of new information. The present review assembles the most important points relating to HR-QOL in PD raised in the literature, adds value to previously covered issues, and examines areas of HR-QOL in PD that have not previously been reviewed, such as education, carer burden and surgery, highlighting where more research is warranted.