Patient Benefit Index Research Articles

Improved Quality of Life in Patients with Psoriasis Receiving Apremilast: Real-World Data from the Netherlands.

Psoriasis is a chronic inflammatory condition that can significantly impact the quality of life (QoL), regardless of the level of skin involvement. Apremilast is indicated for the treatment of moderate to severe psoriasis. Real-world data regarding the impact of apremilast on patient-reported outcomes in clinical practice in the Netherlands is lacking. The prospective, multicenter observational Apremilast in Real-Life Psoriasis Treatment (APRIL) study enrolled patients ≥ 18years old with moderate to severe plaque psoriasis receiving apremilast in clinical practice in the Netherlands. Patients were followed-up for 12 months, with assessments scheduled at 6 and 12 months. The primary outcome was Dermatology Life Quality Index (DLQI) response (score ≤ 5 or ≥ 5-point improvement from baseline) at 6 months. Secondary patient-reported outcomes included EQ-5D and skin-specific parameters; exploratory outcomes were Patient Benefit Index (PBI) and Work Productivity and Activity Impairment (WPAI). Of the 155 patients enrolled (February 2016-June 2019), 153 received apremilast; 69 (45%) and 39 (26%) continued treatment at 6 and 12months, respectively. Psoriasis in special areas was common (scalp, 65%; nail, 51%; palmoplantar, 27%). Most patients (92%) had received prior systemic antipsoriatic therapies. Of the 151 patients with a baseline DLQI value, 56 (37%) achieved DLQI response at 6months. Mean (standard deviation) PBI scores were 3.5 (1.2) and 3.8 (1.1) at 6 and 12months, respectively. Improvements in DLQI, EQ-5D, and WPAI scores and disease signs and symptoms, including itch and special areas, were observed at 6 and 12months. Adverse events were consistent with the known safety profile. In the Netherlands, patients with moderate to severe psoriasis receiving apremilast for up to 12months reported improved disease-related QoL, skin involvement, and patient-reported outcomes. These data add to the growing body of evidence demonstrating apremilast is an effective treatment for psoriasis, itch, and special areas (scalp and palms). ClinicalTrials.gov, NCT02652494.

Goals and benefits in topical treatment for psoriasis: development and pilot validation of a patient-reported outcomes tool, the Patient Benefit Index for Topical Treatment (PBI-TOP).

Topical agents are an essential component of psoriasis therapy. To develop a new version of the patient-reported Patient Benefit Index assessing the importance and achievement of treatment goals in topical psoriasis therapy in adult patients (PBI-TOP). Through semi-structured interviews, focus groups and free-text questionnaires, patients reported their needs in topical treatment. Based on qualitative content analysis, items were developed by a consensus group and were refined in cognitive debriefing interviews. A pilot validation assessed the PBI-TOP and convergent criteria [Dermatology Life Quality Index (DLQI); Psoriasis Area and Severity Index (PASI); affected Body Surface Area (BSA)]. Thirty patients (26-72 years, mean 47; 60% male) reported various treatment goals relating to the themes 'effectiveness' and 'characteristics of the preparation'. Twenty patients took part in cognitive debriefings (22-84 years, mean 50.6, 50% male). There were 154 patients who participated in the pilot validation (18-85 years, mean 46.9, 63.6% male). An importance-weighted total score on overall effectiveness and three subscales based on exploratory factor analysis were defined: effectiveness on symptoms, effectiveness on quality of life (QoL), and characteristics of the preparation. All scores showed excellent internal consistency (α > 0.9). The global effectiveness score correlated significantly with DLQI (r = -0.41), PASI (r = -0.32) and BSA (r = -0.22). The effectiveness subscales (symptoms; QoL) correlated significantly with DLQI (r = -0.41; -0.32) and PASI (r = -0.27; -0.33). The score on characteristics of the preparation correlated significantly with the DLQI (r = -0.34). The PBI-TOP showed good feasibility and favourable psychometric characteristics in this pilot validation.

A value-based healthcare model for initiating and switching psoriasis therapies-Results from the prospective multicentre IMPROVE study.

Little is known about the therapeutic benefits of a value-based healthcare model compared to a traditional activity-based incentive model in psoriasis (PsO). This prospective non-interventional study evaluated an outcome-based, patient-centred management model for patients with PsO. In total, 49 patients with a Psoriasis Area and Severity Index (PASI) ≥3 who were starting or switching between treatments were included. Patients were assessed at baseline, 3 and 9 months. The patient benefit index (PBI) was calculated using predefined questionnaires. An expected PBI was calculated and adjusted for risk factors known to complicate treatment, that is overweight and smoking. The model remunerated the department on whether the observed PBI exceeded the expected PBI to incentivize over-performance. In total, 40 patients (80%) completed all three visits; 32.7% were smokers and 73.5% were overweight. Mean PASI at baseline was 11.5 (SD 9.1); PASI improved significantly from baseline through 3 months: mean reduction, 8.0 (SD 9.2), p < 0.001 and was maintained until 9 months: mean further reduction, 0.1 (SD 3.3), p = 0.893. The mean PBI was 2.5 (SD 1.3) and 2.8 (SD 1.1) at 3 and 9 months, respectively. A PBI ≥1 was achieved by 87.8% at 3 and 95.1% at 9 months. Overall, the department was remunerated a mean 2721.1 DKK (SD 4472.8) per patient. In subgroup analysis, the department was remunerated a mean of, respectively, 2428.6 (SD 5089.5), 2636.6 (SD 4471.3) and 3196.5 (SD 4497.1) DKK for patients with none, 1 or 2 risk factors, that is smoking or/and overweight. The model evaluated herein is the first value-based model to calculate remuneration from patient reported outcomes and showed to successfully predict the expected PBI and remunerate treatment based on whether the expected treatment goal was met or exceeded. This can be utilized in the patient-centred management of PsO.

Tildrakizumab improves high burden skin symptoms, impaired sleep and quality of life of moderate-to-severe plaque psoriasis patients in conditions close to clinical practice.

Tildrakizumab (TIL) is an interleukin (IL)-23p19 inhibitor for the treatment of moderate-to-severe plaque psoriasis with long-term efficacy and safety demonstrated in Phase III trials. Studies conducted in conditions closer to clinical practice are needed. The TRIBUTE study (open-label, Phase IV) assessed the efficacy and impact on health-related quality of life (HRQoL) of TIL 100 mg in adult moderate-to-severe psoriasis patients (naïve to IL-23/Th17 pathway inhibitors) in conditions similar to clinical practice. Key efficacy measure was Psoriasis Area Severity Index (PASI). HRQoL was evaluated using the Dermatology Life Quality Index (DLQI) and Skindex-16. Additional patient-reported outcomes included Pain-, Pruritus- and Scaling-Numerical Rating Scale (NRS), Medical Outcome Study (MOS)-Sleep, Work Productivity and Activity Impairment (WPAI), Patient Benefit Index (PBI) and Treatment Satisfaction Questionnaire for Medication (TSQM). One hundred and seventy-seven patients were enrolled (six patients did not complete the study). After 24 weeks, the proportion of patients achieving PASI scores ≤ 3, PASI 75, PASI 90 and DLQI 0/1 was 88.4%, 92.5%, 74.0% and 70.4%, respectively. Skindex-16 overall score improved (mean absolute change from baseline, MACB [95%CI]: -53.3 [-58.1, -48.5]). Significant benefits (MACB [95%CI]) were found on pruritus-, pain- and scaling-NRS scores (-5.7 [-6.1, -5.2], -3.5 [-4.1, -3.0] and -5.7 [-6.2, -5.2], respectively), MOS-Sleep (-10.4 [-13.3, -7.4] Sleep problems Index II) and WPAI (-36.4 [-42.6, -30.2] activity impairment, -28.2 [-34.7, -21.7] productivity loss, -27.0 [-32.9, -21.1] presenteeism and -6.8 [-12.1, -1.5] absenteeism). 82.7% of patients reported PBI ≥ 3 and the mean (SD) global TSQM score was high (80.5 [18.5]). Only one serious treatment-emergent adverse event was reported (not-related to TIL). TIL 100 mg treatment after 24 weeks in conditions close to real clinical practice showed a quick and high improvement in psoriasis signs and HRQoL. Patient reported improvements in sleep outcomes and work productivity, relevant benefits and high treatment satisfaction. The safety profile was favourable and consistent with Phase III trials.

Development of a Specific Variant of Patient Benefit Index (PBI) Assessing Patient Needs, Goals and Benefits in Rosacea Treatment.

Evaluation of patient-reported outcomes including health-related quality of life (HRQoL) and perceived benefits from treatment has become a fundamental component of medical decision-making. Standardized evaluation of treatment benefits in rosacea based on patient preferences is still lacking. Development and validation of an instrument for recording patient-defined benefits in rosacea therapy based on the Patient Benefit Index (PBI) methodology. In an open survey of n = 50 patients, potential benefits of therapy from the patient's perspective were examined. The generated item pool was combined with pre-existing PBI items for other skin conditions and reviewed by an expert panel of dermatologists, psychologists and patients. Items were condensed to n = 25 and converted into a Likert-scaled questionnaire. The validity and feasibility of the resulting Patient Benefit Index for rosacea (PBI-RO) were tested on individuals with rosacea recruited from a German rosacea patient organization. N = 446 patients with rosacea completed the PBI-RO. The internal consistencies measured by Cronbach's alpha were high (Patient Needs Questionnaire [PNQ] 0.94). Mean PBI-RO was 1.9 ± 1.2 (scale from 0 = no benefit to 4 = maximum benefit), 23.5% of the patients experienced a PBI-RO < 1 (no clinically relevant benefit). The PBI-RO correlated with HRQoL, health state, current extent of rosacea lesions and treatment satisfaction. The highest correlation was found between PBI-RO and satisfaction with previous treatment (r = -0.59, p < 0.001); correlation with the extent of rosacea lesions was low (r = 0.16, p < 0.001). The PBI-RO shows satisfying internal consistency and construct validity. It offers the option of a patient-weighted evaluation of the therapeutic benefit of rosacea therapy and may add to more stringent goal orientation in therapy.

Apremilast treatment of plaque psoriasis (PsO) after systemic therapy in Spanish clinical practice: Data from the APPROPRIATE study

AbstractBackgroundDespite the proven efficacy and safety of apremilast to treat plaque psoriasis (PsO), data regarding its real‐world use and patient‐perceived benefits are limited.ObjectivesDescribe apremilast use, persistence and tolerability, and its patient‐perceived benefits and effectiveness in patients with PsO in Spanish clinical practice.MethodsObservational, prospective, multicenter study including patients with moderate‐to‐severe PsO initiating apremilast 3 months (±4 weeks) before enrolment, after at least one conventional systemic therapy and no biologics. Prospective data were collected 3 (at enrolment), 6 and 12 months (±4 weeks) after apremilast initiation. Primary outcome was a Patient Benefit Index (PBI) score ≥1 (minimum clinically relevant benefit) at 6 months.ResultsOf 153 patients enroled, 119 were included in the analysis; mean (standard deviation [SD]) age, 52.8 (15.2) years. At apremilast initiation, mean (SD) Psoriasis Area and Severity Index (PASI) and Dermatology Life Quality Index (DLQI) were 8.3 (4.9) and 10.5 (6.8), respectively. Most patients (81%) had comorbidities; PsO manifestations included scalp (47% of patients), palmoplantar (26%), nails (24%) and genitals (11%). Over three‐quarters (86%) of patients were continuing apremilast at Month 6, with most (91%) achieving a PBI ≥ 1% and 43% achieving a PBI ≥ 3; two‐thirds (68%) were continuing apremilast at Month 12, with 91% and 42% achieving a PBI ≥ 1 and ≥3, respectively. Mean (SD) pruritus scores decreased from 54.5 (32.1) at apremilast initiation to 23.4 (27.0) at Month 12; 57%, 69% and 69% of patients achieved a DLQI 0‒1, PASI &lt; 3 and BSA &lt; 3, respectively. Adverse events were consistent with the known safety profile.ConclusionsPatients initiating apremilast after conventional systemic therapy in Spanish clinical practice had moderate PsO at bothersome/visible locations and impaired quality of life. Most patients remained on apremilast for 12 months, with improved patient‐reported outcomes and skin involvement, and almost all patients reported some clinical benefit. Apremilast was well tolerated without new safety signals.

The Needs of Patients with Psoriasis and Benefits of Apremilast in French Clinical Practice: Results from the Observational REALIZE Study.

Real-world data on the needs of patients with psoriasis and patient-perceived benefits of apremilast are limited. We report such data from France. The multicenter, observational REALIZE study was conducted in real-life clinical practice in France and enrolled patients with moderate-to-severe plaque psoriasis who had initiated apremilast per French reimbursement criteria in the 4weeks preceding enrollment (September 2018-June 2020). Physician assessments and patient-reported outcomes (PROs) were collected at enrollment, 6months, and 12months. PROs included the Patient Benefit Index for skin diseases (PBI-S), Dermatology Life Quality Index (DLQI), and 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9). The primary outcome was PBI-S ≥ 1 (minimum clinically relevant benefit) at month 6. Of 379 enrolled patients who received ≥ 1 dose of apremilast, most [n = 270 (71.2%)] remained on apremilast at 6months and over half [n = 200 (52.8%)] persisted at 12months. Patients reported the following treatment goals as most important (≥ 70% reported goal as "very important" in the Patient Needs Questionnaire): get better skin quickly, regain disease control, be healed of skin alterations, and have confidence in the therapy. Most patients persisting on apremilast achieved a PBI-S ≥ 1 at months6 and 12 (91.6% and 93.8%, respectively). Mean (SD) DLQI decreased from 11.75 (6.69) at enrollment to 5.17 (5.35) and 4.18 (4.39) at months6 and 12, respectively. Most patients (72.3%) reported moderate-to-severe pruritus at enrollment and no/mild pruritus at months6 and 12 (78.8% and 85.9%, respectively). Mean (SD) TSQM-9 Global Satisfaction scores were 68.4 (23.3) and 71.7 (21.5) at months6 and 12, respectively. Apremilast was well tolerated; no new safety signals were identified. REALIZE provides insights regarding the needs of patients with psoriasis and the patient-perceived benefits of apremilast. Patients who persisted on apremilast reported improvements in quality of life, high treatment satisfaction, and clinically relevant benefits. NCT03757013.

Impact of daily use of emollient ‘plus’ on corticosteroid consumption in patients with atopic dermatitis: An open, randomized controlled study

Emollients are the baseline treatment for mild or moderate atopic dermatitis (AD) to improve the epidermal barrier and provide anti-irritant and anti-pruritic effects. Emollient 'plus' can influence the skin microbiome of atopic eczema patients. To evaluate the benefits of using Emollient 'plus' to reduce corticosteroid consumption. In an open, single-centre, randomized, controlled study, patients with mild to moderate AD (Severity scoring of AD [SCORAD] score 20-30) were randomized 1:1 to apply the Emollient 'plus' twice daily for 28 days or to continue with their usual classical emollient (Control group). In addition, each patient received topical corticosteroids to use when necessary and according to the dermatologist's prescription. Assessments included SCORAD, PO-SCORAD, local SCORAD, quality of life questionnaires, and tolerability. A total of 119 patients were included in the PP population with a mean age of 26.50 ± 17.5 years old (min-max 3-71 years). Between baseline and day 28, the mean amount of corticosteroid used was lower for the Emollient 'plus' versus Control group (6.03 vs. 9.16 g; p=0.041) and corticosteroid was applied on fewer days (37.5% vs. 46.9% of days; p=0.0256) with fewer applications per day (0.55 vs. 0.71 applications per day; p=0.0203). Similar improvements were observed in both groups for SCORAD, PO-SCORAD, local SCORAD, skin sensation score, AD burden scale, patient benefit index >1, as well as subject and investigator efficacy and tolerability questionnaire assessments. Between baseline and day 28, there was significant corticosteroid-sparing in the Emollient 'plus' group compared to the Control group in quantity, number of applications per day and number of days of use, whilst efficacy was maintained with no significant differences between the two groups for all clinical evaluations, as well as for tolerability.

Cost-effectiveness and Quality of Specialized and Routine Care in a German Cohort of Patients with Chronic Pruritus

Chronic pruritus is a prevalent interdisciplinary symptom with a strong influence on health-related quality of life. Patients need extensive diagnostics and long-term treatment. This retrospective and prospective cohort study compared routine and university-based specialized care in terms of cost-effectiveness and patient benefit. Direct medical and non-medical costs and patient-reported outcomes (PRO; pruritus intensity, quality of life, treatment needs and benefits) were assessed. Data analyses were conducted using descriptive methods and non-parametric statistical tests. A total of 300 adult patients (54.3% female) participated in the study. Six months after the treatment start in a specialized German pruritus care unit, the total costs were significantly reduced (mean total costs 686 € vs 433 € per patient per half year (total cohort); p < 0.001; mean out-of-pocket costs 198 € vs 124 € per half year (total cohort), p < 0.001). Pruritus intensity (numerical rating scale 5.3 vs 3.7, p < 0.001), quality of life (Dermatology Life Quality Index 8.9 vs 5.7, p < 0.001) and patient benefit (Patient Benefit Index Pruritus 1.2 vs 2.1, p < 0.001) improved significantly (total cohort). The results of this study show, that treatment of chronic pruritus patients in a specialized itch centre leads to an improvement in patient benefit and reduces the economic burden at the same time.

Apremilast Use in Severe Psoriasis: Real-World Data from Central and Eastern Europe.

The broad and sustained efficacy of apremilast for psoriasis has been demonstrated in randomized and real-world observational studies. Data from Central and Eastern Europe (CEE) are lacking. Moreover, apremilast use in this region is limited by country-specific reimbursement criteria. This is the first study to report data on the real-world use of apremilast in the region. APPRECIATE (NCT02740218) was an observational, retrospective, cross-sectional study assessing psoriasis patients 6 (± 1) months after apremilast treatment initiation. The study aimed to describe the characteristics of patients with psoriasis receiving apremilast, estimate treatment outcomes, including Psoriasis Area Severity Index (PASI), Body Surface Area (BSA), and Dermatology Life Quality Index (DLQI), and assess dermatologists' and patients' perspectives on treatment using questionnaires including the Patient Benefit Index (PBI). Adverse event reports were taken from the medical records. Fifty patients (Croatia: 25; Czech Republic: 20; Slovenia: 5) were enrolled. In patients continuing apremilast at 6 (± 1) months, mean (± SD) PASI score was reduced from 16.2 ± 8.7 points at treatment initiation to 3.1 ± 5.2 at 6 (± 1) months; BSA from 11.9% ± 10.3% to 0.8% ± 0.9%; DLQI from 13.7 ± 7.4 points to 1.6 ± 3.2. PASI 75 was reached by 81% of patients. Physicians reported that the overall treatment success fulfilled their expectations in more than two thirds of patients (68%). At least three-quarters of patients reported apremilast had a quite or very high benefit on the needs they identified as being most important. Apremilast was well tolerated; no serious or fatal adverse events were identified. Apremilast was effective in reducing skin involvement and improving quality of life in CEE patients having severe disease. Treatment satisfaction among physicians and patients was very high. These data add to the growing body of evidence showing consistent effectiveness of apremilast across the continuum of psoriasis disease severity and manifestations. ClinicalTrials.gov identifier, NCT02740218.

Patient-reported well-being in value-based care using tildrakizumab in a real-world setting: protocol of a multinational, phase IV, 1-cohort prospective observational study (the POSITIVE study)

IntroductionPsoriasis is a chronic inflammatory skin disease that negatively impacts the quality of life of patients and their families. However, the most commonly used decision-making tools in psoriasis, Psoriasis Area...

Introduction of a Specific Dermatological Rehabilitation Programme for Patients with Chronic Pruritus: A Pilot Study.

Chronic pruritus is a common symptom, associated with several severe medical conditions, great psychological burden, and reduced quality of life. It also poses socio-economic challenges concerning patients' work loss and healthcare costs. In Germany, medical rehabilitation programmes represent an integral part of the medical care of patients with chronic inflammatory skin diseases. However, such programmes play only a rudimentary role in the treatment of other dermatological diseases, such as dermatological oncology, genetic skin diseases, and chronic pruritus. Therefore, a specific antipruritic dermatological rehabilitation programme was developed in cooperation between the Department of Dermatology of the Medical Rehabilitation Center Bad Bentheim and the Center for Chronic Pruritus of the University Hospital of Muenster, Germany. This prospective study compared short-term patient-reported outcomes (n = 121) at the beginning and end of the rehabilitation programme. The majority of subjects had chronic pruritus on primary diseased, inflamed skin. Significant improvements in pruritus intensity (p ≤ 0.001), quality of life (p ≤ 0.001), anxiety symptoms (p ≤ 0.001) and depression (p ≤ 0.001), as well as an overall patient-relevant benefit (Patient Benefit Index 2.6 ± 1.06) and treatment-related patients' satisfaction, were shown. This suggests that implementation of this standardized rehabilitation programme for treatment of patients with chronic pruritus was successful.

Psychological (co)morbidity in patients with psoriasis: the impact of pruritus and anogenital involvement on symptoms of depression and anxiety and on body dysmorphic concerns – a cross-sectional study

ObjectivesWhile stress plays a paramount role on the onset/exacerbation of psoriasis, via overactivation of the hypothalamic–pituitary–adrenal axis and increased release of pro-inflammatory cytokines, cutaneous inflammatory response induces, in turn, anxiety/depression...

Patients' satisfaction, unmet needs, and treatment benefits in moderate to severe psoriasis in Greece: results from a cross-sectional survey.

Data regarding patients' satisfaction, individuals' therapeutic needs and their perception regarding the treatment benefit might provide useful insights for the unmet medical need from patients' perspective. To the best of our knowledge, no data are available in Greece investigating in depth moderate to severe psoriasis while there is a scarcity of studies in the international literature. The objective of this study was to capture patients' satisfaction and perception about treatment benefits in patients with moderate to severe psoriasis in Greece. A cross-sectional survey was conducted in March-April 2021. Eligible to participate were adults with moderate to severe psoriasis, members of the Greek patient association EPIDERMIA. A structured questionnaire including socio-demographic factors, history of disease, comorbidities, current treatment, severity self-assessment and impact in daily activities, treatment adherence, treatment satisfaction, and Patient Benefit Index (PBI), was developed. Univariate parametric and non-parametric tests along with generalized linear models were applied. A total of 314 adults with moderate to severe psoriasis responded. The 97.5% of the participants reported that they were under treatment, while 41.1% of them mentioned none or little/quite satisfaction by their current treatment, with the most frequently reported reason of dissatisfaction to be the "no satisfactory improvement since treatment onset." The most important therapeutic goals from patient perspective were to "be healed of all skin defects," and to "get better skin quickly." The therapeutic benefit for these goals was found to be moderate (55% and 67%, respectively). Multivariate analysis confirmed that treatment type and psoriasis severity were factors independently associated with treatment satisfaction, and PBI Score. The results of this survey reveal the unmet medical need for moderate to severe psoriasis from patient perspective, since 2/5 patients stated that they are not much or very much satisfied with their current treatment, and more than half patients stated that the most important therapeutic goals (i.e., clean skin, early onset) for them are not achieved.

Development and validation of a disease-specific quality of life questionnaire for patients with peripheral artery disease (QOLPAD)

BackgroundThe aim of this study was to develop and validate a short and feasible questionnaire to measure health-related quality of life (HRQoL) in patients with peripheral artery disease (PAD). The content of the new instrument is intended to correspond with the simultaneously developed instrument Patient Benefit Index for PAD (PBI-PAD), which evaluates treatment goals and benefits in this patient group.MethodsFifty patients stated their disease burden on free-text questionnaires, which was used by an interdisciplinary expert panel to develop 12 items for the new instrument, named Quality of Life questionnaire for patients with peripheral artery disease (QOLPAD). The validity of the instrument was tested in patients from Germany with PAD stages I to IV who completed the QOLPAD, EuroQol questionnaire (EQ-5D-3L; EuroQol visual analogue scale (EQ VAS)), and Vascular Quality of Life questionnaire (VascuQoL) before (baseline) and three months after (follow-up) treatment.ResultsOne hundred and three patients were included at baseline (mean age: 68.6 years; 68% male), among whom, 57 provided data at follow-up. Most patients (86.4%) rated the completion of QOLPAD as being easy. Internal consistency was satisfactory, with a Cronbach’s alpha of 0.74 (baseline) and 0.84 (follow-up). Convergent validity was indicated by significant correlations with the EQ-5D-3L (baseline: − 0.62; follow-up: − 0.81), EQ VAS (baseline: − 0.44, follow-up: − 0.79), VascuQoL global score (baseline: − 0.77; follow-up: − 0.87), global rating of impairment (baseline: 0.64; follow-up: 0.71), and PAD stage (baseline: 0.40; follow-up: 0.67). Sensitivity to change was confirmed by significant correlations of change in the QOLPAD with changes in convergent criteria; however, the high number of dropouts limits the generalizability of this finding.ConclusionThis study provided evidence that the QOLPAD is internally consistent and valid in patients receiving treatment for PAD in Germany.

Sharing Patient and Clinician Experiences of Moderate-to-Severe Psoriasis: A Nationwide Italian Survey and Expert Opinion to Explore Barriers Impacting upon Patient Wellbeing.

A nationwide survey was conducted in adult patients with psoriasis (PsO) across Italy to obtain their real-world perspective of the impact of PsO on their wellbeing. Patients completed a 26-question survey (based on the patient benefit index; PBI, The Dermatology Life Quality Index; DLQI and the World Health Organization-five; WHO-5 wellbeing index) and workshop discussion sessions were undertaken by dermatologists to interpret results from the survey. 392 patients with PsO completed the survey. Analysis of results was restricted to patients who had moderate-to-severe plaque psoriasis (assessed by patients; n = 252; 64.3%). Dermatologists (n = 32) completed one question from the survey related to wellbeing and rated social, physical and mental domains as contributing to a similar extent, with comparable scores also observed by patients. For treatment, biologics yielded higher scores on average, whereas little difference was observed between topical and conventional systemic treatments. Only 23.8% of patients felt that their dermatologist was taking into consideration their wellbeing and 32.6% of the patients considered their therapy as inadequate in improving signs and symptoms of the disease. This survey identified key factors contributing to barriers impacting on patient wellbeing. Simple, but comprehensive questionnaires can provide important insight to patients’ needs that may significantly increase clinician awareness during visits leading to tailored treatment.

Development and Validation Features of the Patient Benefit Index for the Treatment of Allergic Rhinoconjunctivitis with Allergen Immunotherapy

PurposeAllergic rhinoconjunctivitis (ARC) is one of the most common diseases worldwide. Allergen immunotherapy (AIT) is the only causal treatment available so far. Due to health policy provisions, the assessment of treatment benefit from the patient’s perspective is of high relevance. To date, no instrument for assessing treatment needs and benefits of patients with ARC who receive AIT has been published. The aim of the study was to validate an instrument to assess the patient-relevant treatment benefit of patients with ARC who receive AIT.MethodsWe developed the Patient Benefit Index questionnaire for AIT (PBI-AIT), consisting of 33 items. Longitudinal data of patients with ARC were used to test feasibility, reliability and validity. The PBI was compared between the beginning of the study (t1) and the end of the study (t5).ResultsN = 279 patients with AIT completed the PBI-AIT at t1, n = 333 at t5; n = 226 at both timepoints. Mean number of missing values per patient was 0.7 in the Patient Needs Questionnaire (PNQ) at t1 and 1.2 in the Patient Benefit Questionnaire (PBQ) at t5. The internal consistencies measured by Cronbach’s alpha were 0.98 (PNQ) and 0.99 (PBQ). The mean PBI of the patients with AIT was significantly lower at t1 and improved at t5. The PBI-AIT correlated with all tested external criteria at t5. The correlation between PBI-AIT and satisfaction with previous treatment (r = −0.57, p < 0.001) was higher than the correlation between PBI-AIT and current disease severity (r = −0.26, p < 0.001).ConclusionThe results indicate feasibility, reliability, convergent and discriminant validity as well as sensitivity to change of the PBI-AIT.

Treatment Goals for Psoriasis as Measured by Patient Benefit Index: Results of a National Psoriasis Foundation Survey.

IntroductionThis cross-sectional survey was conducted with National Psoriasis Foundation (NPF) to capture treatment perspectives and expectations in patients with psoriasis (PsO) using Patient Needs Questionnaire (PNQ) of Patient Benefit Index (PBI).MethodsAdult participants with self-reported diagnosis of PsO responded to the PNQ portion of PBI by indicating how much they valued different treatment attributes. All the treatment goals were captured on a five-point Likert scale (0 = “Not important”, 4 = “Very important”). Treatment goals were obtained for overall population and subgroups based on severity of disease Patient Global Assessment (PGA), age, gender, and Dermatology Life Quality Index (DLQI) total score. All data were expressed as mean and standard deviation [SD].ResultsA total of 1200 participants completed the survey (mean age 51.5 years). Top treatment goal in the overall population was “to have confidence in the therapy” (3.46 [1.01]). Unique to the higher severity subgroup (PGA ≥ 3), “to find a clear diagnosis and therapy” was a top five goal and “to get better skin quickly” was for those with lesser severity (PGA < 3). “To be free of itching” (3.36 [0.99]) was the unique goal in the < 40 age group whereas it was “to get better skin quickly” (3.27 [1.12]) in the ≥ 40 group. In women and men, “to be free of itching” (3.38 [1.13]) and “to get better skin quickly” (3.20 [1.09]) were top five goals, respectively. Patients with ≥ 10 DLQI scores expressed higher treatment goal “to regain control of the disease” (3.66 [0.67]) compared to those with ≤ 10 DLQI scores who expressed “to have confidence in the therapy” (3.40 [1.11]) as the topmost treatment goal.ConclusionOur results suggest that in patients with PsO, treatment preferences can vary with different characteristics such as age, severity, and gender as measured by using PNQ. Further exploration of this data will help inform treatment decisions and optimize patient outcomes.Supplementary InformationThe online version contains supplementary material available at 10.1007/s12325-022-02124-2.

Improvements in itch and sleep following treatment with baricitinib in combination with topical corticosteroids are associated with better quality of life and productivity in adult patients with moderate-to-severe atopic dermatitis: a post hoc analysis from BREEZE-AD7

Treatment with baricitinib in combination with topical corticosteroids previously showed greater improvements in itch and sleep versus placebo in adults with moderate-to-severe AD. To assess whether improvements in itch and sleep translate to greater quality of life (QoL), productivity and treatment benefit in AD. In this post hoc analysis with data from BREEZE-AD7 (NCT03733301), itch and sleep improvements at Week 16 were defined by ≥4-point improvements in the Itch Numeric Rating Scale and ≥1.5 decreases in the number of night-time awakenings since baseline, respectively. Dermatology Life Quality Index, Work Productivity and Activity Impairment-AD and Patient Benefit Index (PBI) scores were compared in patients with and without improvements. Proportions were analysed using logistic regression with non-responder imputation. Changes from baseline were calculated using ANCOVA, with last observation carried forward. Least square mean PBI scores were assessed using ANOVA. More patients with itch improvement versus no itch improvement reported no impact of AD on QoL (28.4% vs. 6.0%). Daily activity impairment was lower in patients with itch improvement (-39.6% vs. -15.6%). A greater proportion of patients with sleep improvement versus no sleep improvement had no AD-related impact on QoL (24.1% vs. 1.5%). Patients with sleep improvement had less daily activity impairment (-35.0% vs. -18.5%). Patients with itch and sleep improvements experienced greater treatment benefit. Patients with AD who experienced clinically meaningful improvements in itch and sleep following treatment had significantly better QoL, productivity and treatment benefit. Addressing these symptoms is important to achieving meaningful and patient-relevant improvements in well-being.

Adaptation of the Patient Benefit Assessment Scale for Hospitalised Older Patients: development, reliability and validity of the P-BAS picture version

BackgroundThe Patient Benefit Assessment Scale for Hospitalised Older Patients (P-BAS HOP) is a tool developed to both identify the priorities of the individual patient and to measure the outcomes relevant to him/her, resulting in a Patient Benefit Index (PBI), indicating how much benefit the patient had experienced from the hospitalisation. The reliability and the validity of the P-BAS HOP appeared to be not yet satisfactory and therefore the aims of this study were to adapt the P-BAS HOP and transform it into a picture version, resulting in the P-BAS-P, and to evaluate its feasibility, reliability, validity, responsiveness and interpretability.MethodsProcess of instrument development and evaluation performed among hospitalised older patients including pilot tests using Three-Step Test-Interviews (TSTI), test-retest reliability on baseline and follow-up, comparing the PBI with Intraclass Correlation Coefficient (ICC), and hypothesis testing to evaluate the construct validity. Responsiveness of individual P-BAS-P scores and the PBI with two different weighing schemes were evaluated using anchor questions. Interpretability of the PBI was evaluated with the visual anchor-based minimal important change (MIC) distribution method and computation of smallest detectable change (SDC) based on ICC.ResultsFourteen hospitalised older patients participated in TSTIs at baseline and 13 at follow-up after discharge. After several adaptations, the P-BAS-P appeared feasible with good interviewer’s instructions. The pictures were considered relevant and helpful by the participants. Reliability was tested with 41 participants at baseline and 50 at follow-up. ICC between PBI1 and PBI2 of baseline test and retest was 0.76, respectively 0.73. At follow-up 0.86, respectively 0.85.For the construct validity, tested in 169 participants, hypotheses regarding importance of goals were confirmed. Regarding status of goals, only the follow-up status was confirmed, baseline and change were not. The responsiveness of the individual scores and PBI were weak, resulting in poor interpretability with many misclassifications. The SDC was larger than the MIC.ConclusionsThe P-BAS-P appeared to be a feasible instrument, but there were methodological barriers for the evaluation of the reliability, validity, and responsiveness. We therefore recommend further research into the P-BAS-P.