Health-related quality of life burden in scleroderma patients treated with two different intravenous iloprost regimens.

Systemic sclerosis (SSc)-related Raynaud's phenomenon (RP) and digital ulcers (DU) can impair health-related quality of life (HRQoL). The aim of our study was to estimate HRQoL in SSc patients treated with two different intravenous (IV) iloprost (ILO) regimens and in patients not treated with IV ILO. 96 consecutive SSc patients were enrolled in a pragmatic, prospective and non-randomized study, and divided into 3 groups: not requiring therapy with IV ILO (N=52), IV ILO once monthly (N=24) or IV ILO for 5 consecutive days every 3 months (N=20). Patients were followed up for three months. We assessed HRQoL using the generic preference-based questionnaire EQ-5D-5L. We conducted multiple regression analyses to estimate, in each treatment group, the mean general health (GH) and the mean utility index of the EQ-5D-5L, adjusting for possible confounders. The mean adjusted utility index and GH score, after three months' follow-up, were not different in the three groups: IV ILO was able to make patients requiring IV ILO similar to those not requiring it. Moreover, there was no difference in this model between the two ILO regimens (1 day monthly vs 5 consecutive days every 3 months). The two different IV ILO regimens (the most appropriate regimen was decided according to patients' characteristics and needs) were able to stabilize HRQoL in RP secondary to SSc non-adequately controlled by oral therapy.

Immune tolerance induction in patients with haemophilia a and inhibitors: effectiveness and cost analysis in an European Cohort (The ITER Study).

INTRODUCTION: Although immune tolerance induction (ITI) is considered the first choice treatment to eradicate inhibitors in haemophilia A patients, little is known about outcomes determinants and cost magnitude. AIM AND METHODS: A retrospective, multicentre study was conducted to assess the relationship between ITI outcome, clinical and treatment characteristics and cost of ITI treatment in haemophilia A patients. Data from 12 months before inhibitor diagnosis to 12 months after ITI completion were collected. Treatment cost was calculated in the third-party perspective and expressed as mean € per patient-month. Cox regression models were used to identify predictors of better outcome and the time taken to achieve tolerance. RESULTS: Seventy-one patients, aged 0.4-41 years (median: 3.8 years) at ITI start, were enrolled. Undetectable inhibitor was achieved in 84.5% of patients and inhibitor eradication with normal factor VIII (FVIII) pharmacokinetics in 74.2%. Median time to successful tolerance was 10.7 months (range 2.0-90.0 months). Peak inhibitor level on ITI was a significant predictor of ITI success. Breakthrough bleeding event incidence during ITI was associated with time to success. The mean cost of treatment for the time period between inhibitor diagnosis and ITI start was €3188 per patient-month (92.1% for bypassing agents), and €60 078 during ITI (76.8% for FVIII use in ITI). CONCLUSION: Immune tolerance induction in this patient cohort was successful in 84.5% of patients with a mean cost of €60 000 per patient-month. This high cost is dwarfed by comparison with the prospect of lifelong care of an inhibitor patient, in addition to gains in life expectancy and health-related quality of life.

Clinical epidemiology of hand eczema in patients accessing dermatological reference centres: results from Italy.

BACKGROUND: Data on the epidemiological impact and clinical characteristics of chronic hand eczema in Southern Europe are lacking. OBJECTIVES: To estimate the prevalence of chronic hand eczema in its different stages of severity and refractoriness to standard therapy in patients accessing Italian dermatological reference centres, and to evaluate sociodemographic and clinical factors associated with each stage. METHODS: A cross-sectional multicentre study was conducted. Adult patients with hand eczema, consecutively accessing 14 centres over a 6-month period, were enrolled. Patients were classified according to disease duration, severity and response to standard therapy with potent topical corticosteroids. Logistical regression was performed to investigate the relationship between sociodemographic and clinical data with different stages of eczema. RESULTS: The total number of participants was 981. Hand eczema was chronic in 83·5% of patients; 21·3% had severe eczema, with 62·0% of these patients refractory to standard therapy. Food processing and related work, the health professions, craft and related trade works (building, plumbing, electrical), hairdressing/beauty and handicraft work were most frequently associated with chronic hand eczema. Severe chronic hand eczema was more likely to be seen in men, older patients and those with less education. Severe and refractory hand eczema was also more likely among the unemployed and patients with allergic rhinitis and/or atopic dermatitis. CONCLUSIONS: Chronic hand eczema is frequent among patients with hand eczema accessing dermatology centres. Many patients were severe and refractory to standard therapy. The appropriate identification of hand eczema is the first step in implementing effective and efficient treatments.

Use of health-related quality-of-life measurements to estimate individuals' health on screening.

OBJECTIVES: to investigate the utility of assessing Health-Related Quality of Life (HRQoL) in a large group of subjects participating in a screening program for aneurysm and the relationship between HRQoL, diagnosis of aneurysm and related risk factors. METHODS: subjects involved in this screening program were submitted the EQ-5D questionnaire to report their own HRQoL. HRQoL was reported also a second time by the subjects who were diagnosed with aneurysm during screening and who returned for a monitoring follow up visit a few months later. We evaluated compliance with HRQoL data collection and performed multiple regression analyses in order to investigate the possible relationship between demographic and clinical data with HRQoL. RESULTS: 1,633 subjects screened (6.1% diagnosed with aneurysm) and 125 subjects diagnosed with aneurysm and attending a follow-up visit reported their HRQoL. Completion of the EQ-5D questionnaire was well accepted by both physicians and subjects undergoing screening. HRQoL was not significantly different between the screening and followup visits, on adjusting for age and sex. At the screening visit, HRQoL was associated with ASA class, heart condition, BMI and respiratory diseases. No associations were found at the follow-up visit. CONCLUSION: Assessing HRQoL in screening programs is feasible and well accepted and add useful information on health of large numbers of subjects from general population. This could be considered as a routine approach to optimizing the informative role of screening programs in guiding other investigations or interventions.

Preferences of patients, their family caregivers and vascular surgeons in the choice of abdominal aortic aneurysms treatment options: the PREFER study.

OBJECTIVE: Factors influencing the choice between endovascular (endovascular aneurysm repair, EVAR) and open repair (OPEN) of abdominal aortic aneurysm (AAA) are of increasing interest. We quantified their importance among the different subjects involved in the treatment. METHODS: Pre- and postoperative patients (pts), their relatives and vascular surgeons completed questionnaires evaluating six treatment characteristics: anaesthesia; recovery time to basic everyday activities; risk of re-intervention at 5 years (RR); complexity of follow-up; risk of major complications; and additional cost of intervention (AC). Through a discrete choice experiment, hypothetical scenarios of treatment were obtained and the relative importance (RI) of each characteristic was determined through a conditional logistic regression model. RESULTS: A total of 160 pts, 102 relatives and 30 surgeons from nine centres completed the questionnaires. Major complications and re-intervention risk were the most important characteristics (RI = 56.0% and 27.2%, respectively) for all the respondent categories. Pts and their relatives considered very important also a possible out-of-pocket AC. Recovery time and type of anaesthesia were among the least important characteristics, including hospital additional cost for surgeons. The different categories of respondents showed different opinions towards different treatment characteristics depending also on possible previous treatment. CONCLUSION: Preferences for AAA treatment characteristics differ between groups of involved subjects. Understanding individuals' preferences could help in optimising treatment benefits.

[Health related quality of life assessment in patients with systemic sclerosis]. / La valutazione della qualità della vita nei pazienti con sclerodermia.

OBJECTIVE: Health-related quality of life (HRQoL) in patients with systemic sclerosis (SSc), a chronic disabling disease associated to physical and psychological impairment, is often left behind in clinical practice and research. This is due to the use of tools that are not complete or mainly designed for the physical condition only. We tested EQ-5D, a valid, simple and brief questionnaire for HRQoL that has never been validated in SSc. METHODS: Thirty-three consecutive SSc patients referring to our Rheumatology Department and undergoing treatment have been asked to fulfill EQ-5D together with HAQ. RESULTS: EQ-5D demonstrated good acceptability, feasibility and validity in patients affected by SSc. Conceptually equivalent domains of EQ-5D demonstrated a good correlation with HAQ correspondent domains. CONCLUSIONS: We suggest the use of EQ-5D in SSc patients as a HRQoL measure in clinical practice, as well as an out come parameter in randomized clinical trials and/or in pharmaco-economic evaluations.

Patients', physicians', and pharmacists' preferences towards coagulation factor concentrates to treat haemophilia with inhibitors: results from the COHIBA Study.

Despite modern highly efficacious technologies, there is still a lack of consensus on how to optimally treat haemophilia patients with inhibitors. The aim of the study was to evaluate preferences towards the characteristics of different coagulation factor concentrates for haemophilia inhibitors patients, from the perspective of patients or their caregivers, haematologists and pharmacists. A discrete choice study was conducted. Potential products were described with eight selected characteristics: perceived viral safety, risk of anamnestic response, possibility of undergoing major surgery, frequency of infusions in prophylaxis, number of infusions to stop bleeding, time to stop bleeding, time to pain recovery and cost. Participants received 16 pairs of potential products and chose from each pair the option they considered better. Data were analysed with a random-effects conditional logistic model. Totally 1614 observations were obtained from 37 patients/caregivers, 39 physicians and 25 pharmacists from Italy. Cost was the most important characteristic to every group. For patients/caregivers, the next most important factors were: risk of anamnestic response, possibility of undergoing major surgery and perceived viral safety. For physicians, the next most important characteristics were: risk of anamnestic response, number of infusions to stop bleeding and possibility of undergoing major surgery. For pharmacists, the next most important factors were: time to stop bleeding, time to pain recovery and possibility of undergoing major surgery. Decisions on treatments must take into account patients clinical needs; however, preferences can also play an important role in the choice and success of treatments. The results of this study could, therefore, help decision-makers to optimize the overall benefits of treatments.

Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors.

Inhibitors represent one major complication of haemophilia treatment, as they increase the risk of bleeding, physical disability and mortality. The Cost Of Care Inhibitors Study (COCIS) showed that modern strategies applied to manage patients with inhibitors adsorb high amounts of resources but provide satisfactory levels of Health-Related Quality-of-Life (HR-QoL). This paper focuses on determinants of HR-QoL in inhibitory patients. Fifty adult patients, enrolled by 11 Italian Haemophilia Centres, were clinically assessed and filled in two HR-QoL generic questionnaires: the EuroQol instrument (EQ-5D) and the Short Form-36 (SF-36). According to our results, bleeding frequency and inhibitor titres were not found associated with HR-QoL. Global HR-QoL, and in particular the physical component of wellbeing in these patients was found negatively associated with their orthopaedic condition: the EQ-5D Visual Analogue Scale (P<0.001) scores, the SF-36 domain 'physical functioning' and 'physical component summary' (P<0.01) scores were found significantly correlated with the orthopaedic joint score, even after adjusting for patients' age. These results were confirmed by those from the EQ-5D profile. To conclude, the COCIS study is the first study showing that HR-QoL in inhibitory patients is impaired by their orthopedic status, while other aspects do not seem to influence patients' global wellbeing. Our results suggest that while the management of this complication is satisfactory, the attention has now to be focused on the prevention of the orthopaedic problems in these patients, which nowadays constitute one of the most important aspects to be considered in the haemophilia care.

Differences between patients', physicians' and pharmacists' preferences for treatment products in haemophilia: a discrete choice experiment.

The provision of health care to patients with haemophilia through replacement of the deficient coagulation factor is the result of a complex interaction between patients, physicians and policy makers, each carrying their individual sets of preferences. Preferences of patients, physicians and pharmacists towards perceived viral safety, risk of inhibitor development, infusion frequency during prophylaxis, pharmaceutical dosage form, distribution modes and price were evaluated by conjoint analysis, using a discrete choice experiment. Overall 178 patients', 69 physicians and 58 pharmacists completed the study. Patients, physicians and pharmacists displayed preferences: (i) similar in direction and strength for risk of inhibitors and frequency of prophylaxis, (ii) similar in direction, but not in strength for perceived viral safety and price, with patients showing lower strength compared with physicians and pharmacists, and (iii) dissimilar in direction and/or strength for: (i) dosage form, which tested important only for pharmacists and (ii) distribution mode, which tested important for patients and physicians only. Our study provides evidence of the differences between different stakeholders in the preferences towards haemophilia replacement therapy, indicating that different opinions should be taken into account when planning optimal care.