Generalized pustular psoriasis (GPP) is a rare disease characterized by episodic worsening (flares). Knowledge of the burden of GPP and the experience of affected individuals is limited. To conduct a survey of people living with GPP to understand how they experience GPP flares, which therapies they have received and are receiving, and how GPP impacts their activities of daily living. The online survey consisted of 43 questions answered by individuals recruited from an opt-in market research database. The research team performed a targeted outreach to identify individuals with GPP. The survey included screening questions to determine if potential participants qualified for inclusion. Eligible individuals were US residents aged ≥ 18 years who self-reported that they had been diagnosed with GPP. Respondents provided consent to participate and received compensation (fair market value) for their time. Between August 4 and 14, 2020, 66 people living with GPP in the USA were surveyed. Most participants were female, aged 40–59 years, had been diagnosed ≥ 1 year previously, and had experienced ≥ 2 flares in the past year. A substantial proportion of respondents had symptoms for years, had consulted multiple healthcare professionals, and experienced misdiagnoses before receiving a diagnosis of GPP. Emotional stress was the most common cause of flares and many respondents reported a fear of flares. Respondents defined flares by the presence of itching, an increase in the size of the affected area, more crusts or pustules, and fatigue. A change in mood was the most burdensome symptom. Most respondents were receiving topical corticosteroids and only approximately one-third felt their condition was well controlled. GPP had an impact on activities of daily living even in the absence of flares and many respondents felt that their physician did not understand the level of emotional, psychological, or physical pain caused by GPP. GPP imposes a substantial emotional burden on patients, with wide-ranging impacts on activities of daily living beyond the physical discomfort of skin lesions.

Background: Generalized pustular psoriasis (GPP) is a rare, severe neutrophilic skin disease with significant unmet clinical need. Historically, GPP has not been well characterized; however, the advent of the International Classification of Diseases, 10th revision, has made it possible to more accurately characterize patients with GPP. Objective: To describe the characteristics and estimate the burden of disease in patients with GPP compared with those with plaque psoriasis. Methods: A retrospective study was conducted using a US administrative claims database, Optum® Clinformatics® Data Mart, between October 1, 2015, and March 31, 2019. Patients with at least 1 inpatient or 2 outpatient diagnosis codes for GPP (L40.1) or psoriasis vulgaris (L40.0) were included. The main outcome measures included the percentage of individuals with comorbidities, medication use, and healthcare resource utilization (HCRU), which were compared between patients with GPP and those with plaque psoriasis and a general population comparator control cohort. Results: Overall, 1,669 patients with GPP were identified at baseline; most patients were female (67.6%). Patients with GPP had more comorbidities than those with plaque psoriasis, specifically metabolic disorders: hyperlipidemia and type 2 diabetes. Medication use for patients with GPP differed from those with plaque psoriasis—patients with GPP required more frequent use of antihypertensives and antibiotics than those with plaque psoriasis. Patients with GPP also had higher HCRU than those with plaque psoriasis. Conclusion: Although this study has limitations, it shows that patients with GPP have a high burden of illness that differs from patients with plaque psoriasis.

Background: Generalized pustular psoriasis (GPP) is a rare, severe neutrophilic skin disease with high unmet clinical need. The introduction of a GPP-specific International Classification of Diseases, 10th Revision (ICD-10), code has made it possible to generate a more accurate GPP patient profile. Objectives: To describe the characteristics and compare the patient profile and burden of disease of patients with GPP with patients with plaque psoriasis. Methods: A retrospective study was conducted using a US administrative claims database, the IBM® MarketScan® Research Database. The study took place between October 1, 2015, and September 30, 2018. Patients with at least 1 inpatient or 2 outpatient L40.1 (GPP) or L40.0 (psoriasis vulgaris) diagnostic codes were included for analysis. Outcome measures included descriptions of comorbidities, medication use, and healthcare resource utilization (HCRU) among GPP, plaque psoriasis, and general population (matched to those with GPP) cohorts. Results: Patients with GPP had more baseline comorbidities than those with plaque psoriasis and the matched cohort, including psoriatic arthritis (20.6% vs 6.4% and <0.1%) and hyperlipidemia (20.4% vs 16.3% and 11.8%). Patients with GPP also had greater medication use and higher HCRU than those with plaque psoriasis and the matched cohort. Conclusion: Patients with GPP generally experience more comorbidities, with higher HCRU, than patients with plaque psoriasis. Although the large dataset permitted identification of GPP patients with longitudinal follow-up, the lack of a validation algorithm for GPP is a limitation and a potential area for future research.

Generalized pustular psoriasis (GPP) is a rare and severe systemic, neutrophilic skin disease. To date, accurate clinical profiling of patients with GPP remains poorly understood. In this study, we present the characteristics and estimate the burden of disease in patients with GPP compared with those with plaque psoriasis, in Japan. This retrospective study was conducted using the Medical Data Vision database between January 1, 2015, and December 31, 2019. Patients with at least one confirmed inpatient or outpatient diagnostic code for GPP (L40.1) or psoriasis vulgaris (L40.0) were included for analysis. The main outcome measures included comparisons of the prevalence of comorbidities, medication use, and healthcare resource utilization between patients with GPP, patients with plaque psoriasis, and a general population-matched cohort. In total, 718 patients with GPP and 27,773 patients with plaque psoriasis were identified. Patients with GPP were more likely to be female than those with plaque psoriasis (51.6% vs. 38.7%). During the 12-month follow-up period, patients with GPP were more likely to experience comorbidities than those with plaque psoriasis, including psoriatic arthritis, other forms of psoriasis, osteoporosis, interstitial pneumonia, and peptic ulcer disease. Medication use also differed between those with GPP and those with plaque psoriasis: patients with GPP were more likely to be prescribed antibiotics and psychiatric medication. Patients with GPP were also more likely to require more healthcare resource utilization with longer hospitalizations than those with plaque psoriasis. Overall, in Japan, patients with GPP have a higher burden of illness than those with plaque psoriasis.

Introduction Generalized pustular psoriasis (GPP) is a rare, severe, and potentially life-threatening systemic and chronic autoinflammatory disease characterized by sterile, neutrophilic pustules. The standard of care for GPP varies by region, with limited information and experience of flares and their treatment. Our aim was to establish current unmet needs in GPP by better understanding the natural history of GPP, examining how dermatologists diagnose GPP and GPP flares, and establishing the range and adequacy of GPP treatment options currently prescribed by dermatologists. Methods Eligible dermatologists (N = 29) completed a 28-question structured survey, covering ten themes, ranging from GPP diagnostic criteria to GPP symptoms and treatment. Results All dermatologists stated that pustules were necessary to diagnose a GPP flare. The most frequently reported triggering factors for GPP were steroid withdrawal (64%), infection (58%), and stress (50%). Most dermatologists indicated that available treatment options for GPP flares were adequate "most" (79%) or "all" (14%) of the time. Despite this reported adequacy, 38% of dermatologists reported that it was at least "somewhat common" for a flare to require hospitalization. Furthermore, 72% of dermatologists indicated that treatments were too slow to control flares, and 66% indicated that treatments did not adequately prevent new flares at least "sometimes". Conclusion This survey suggests that there are key features of GPP flares, and could initiate discussion around forming consensus guidelines for diagnosis and management. While the results suggest that moderately effective therapies may exist, the need for GPP-specific treatments remains.

Generalized pustular psoriasis (GPP) is a rare, systemic disease characterized by recurrent flares of painful neutrophilic aseptic pustules. There is limited real-word evidence characterizing patients with GPP. Establishing the distinguishing characteristics of GPP relative to plaque psoriasis may help inform future treatment and improve patient outcomes. Adults with GPP (n = 60) or plaque psoriasis (n = 4894) identified from the North American-based Corrona Psoriasis Registry were included in descriptive analyses. The Corrona Psoriasis Registry is a prospective, multicenter registry for patients with psoriasis. Registry enrollment data were assessed as variables, including sociodemographics, disease characteristics, medications, and patient-reported outcomes. Though there were similarities between the populations, when assessed using median (p25, p75) visual analog scores (range 0-100), patients with GPP suffered from greater pain (20 [3, 62] vs 5 [0, 35]), fatigue (44 [15, 73] vs 20 [4, 50]), and itch (59 [10, 85] vs 22 [5, 70]) than patients with plaque psoriasis. Patients with GPP also reported more anxiety and depression (38% vs 26%, EQ-5D-3L) and had more treatment experience (15% received ≥2 previous systemic agents vs 7%). Greater symptom severity and impact on quality of life was apparent for patients with GPP vs those with plaque psoriasis. Patients with GPP had more treatment experience, suggesting that current treatment options do not adequately resolve disease. Further, the inclusion criteria of the Corrona Psoriasis Registry may have excluded acute GPP cases. Thus, these findings may underestimate the severity of GPP and its impact on patients’ lives, highlighting the need to develop GPP-specific treatments.

Palmoplantar pustulosis (PPP) is a subtype of psoriasis, localized to the palms and soles. Relatively little is known about the natural history of PPP. Given the paucity of available information, this descriptive study outlines the characteristics, clinical outcomes, and patient-reported outcomes (PROs) of patients with PPP versus those with plaque psoriasis. Enrollment data from the Corrona Psoriasis Registry (a prospective, multicenter registry for patients with psoriasis) were analyzed, comparing patients with PPP (n = 64) and plaque psoriasis (n = 4894). Data include sociodemographics, disease characteristics, PROs, and medication use. There are notable differences between patients with PPP and plaque psoriasis. Although patients with PPP had a smaller median (p25, p75) percentage body surface area (BSA) affected (3 [1, 5.5] vs 5 [1, 12]), when assessing median (p25, p75) visual analog scores (range 0-100), they had greater itch (40 [15, 76] vs 22 [5, 70]), higher fatigue (35 [10, 60] vs 20 [4, 50]), and more pain (35 [5, 63] vs 5 [0, 35]) than patients with plaque psoriasis. Importantly, median reported percentage of impairment while working (10 [0, 53] vs 0 [0, 15]) and in daily activities (30 [5, 50] vs 3 [0, 25]) was greater for patients with PPP. Despite affecting a smaller BSA than plaque psoriasis, the presence of pustules on the palms and soles among patients with PPP has a distinct impact on patient wellbeing, with greater itch, fatigue, and pain reported. These translate into a considerable impairment in both work and daily activities.

Little is known about the disease burden, health-care resource utilization (HCRU), or treatment of patients with generalized pustular psoriasis (GPP) in Japan. This retrospective cohort study used data from the Japanese Medical Data Center database to compare the demographics, comorbidities, and medication use of patients with GPP and plaque psoriasis and estimate their all-cause HCRU. The patient selection period was from January 1, 2015 to December 31, 2019, and patients must have had at least one confirmed inpatient claim or outpatient claim for GPP or plaque psoriasis. During the 12-month follow-up period, 110 patients with GPP and 20,254 patients with plaque psoriasis were identified. An age- and sex-matched (4:1) comparator control cohort, including members of the general population without a diagnosis of psoriasis (but allowing for a diagnosis of psoriatic arthritis), GPP, or palmoplantar pustulosis, was used. The most prevalent comorbidities in patients with GPP included allergic rhinoconjunctivitis, hypertension, and peptic ulcer disease. Patients with GPP were more likely to experience more comorbidities than those with plaque psoriasis, including asthma, chronic obstructive pulmonary disease, interstitial pneumonia, hyperuricemia and gout, tonsillitis, psoriatic arthritis, other psoriasis, and osteoporosis. Patients with GPP were more likely to be treated with a combination therapy than those with plaque psoriasis (65.5% vs 21.7%, respectively) and less likely to be treated with a topical medication alone (20.9% vs 50.8%). Patients with GPP had more outpatient visits than patients in the plaque psoriasis or matched control cohorts (mean [standard deviation], 14.8 [8.3] vs 11.0 [7.6] and 7.8 [7.2], respectively). They were also more likely to require inpatient hospitalization (24.5% vs 6.4% and 5.0%, respectively). Despite study limitations, patients with GPP in Japan were found to have a higher disease burden, including presence of comorbidities and medication use, than those with plaque psoriasis.

Aims:We are developing a new measure of quality of life (QoL), the UK-PSC-QoL, for people with primary sclerosing cholangitis (PSC), a rare incurable disease of the bile ducts and liver which can considerably impact QoL.Method: Following initial issue generation and reduction, 83 issues in six domains were constructed as items.The resulting provisional UK-PSC-QoL was then pilot-tested with PwPSC in the UK.Consensus on how to reliably stage PSC is lacking, so we hypothesised eight categories relating to disease severity, including: co-morbid inflammatory bowel disease, awaiting liver transplant, recurrent PSC, and PSC-related cancers.Participants in all categories were recruited and completed the measure.All participants with more severe PSC, plus a sub-group with less severe disease, were interviewed to explore item comprehensibility, acceptability, relevance, and redundancy.We calculated mean scores and prevalence ratios, and examined response distributions for each item.Results: Sixty PwPSC completed the measure, and 25 were also interviewed.Most participants were male (57%), white British (82%), with median age 52 (range 23-75).The rarity of the condition made recruiting sufficient numbers of PwPSC challenging, particularly those more severely affected.Forty-four participants were recruited in the five less severe PSC categories, but only 16 were recruited in three categories: awaiting liver transplant (n = 5), recurrent PSC (n = 6), and with PSCrelated cancer (n = 5).Analysis of participants' perceptions of item comprehensibility and acceptability resulted in the re-phrasing of 22 items, mostly due to item ambiguity/clarity.Six items were deleted, and one new item added.Other findings were indeterminate owing to under-recruitment in some categories.Conclusions: Initial pilot-testing analysis resulted in deleting six items from the provisional UK-PSC-QoL, but recruitment challenges prevented definitive conclusions on other items.Future research, broadened internationally, would enable recruiting more people in under-represented groups, and thereby assist final decisions on item inclusion.

Background: Randomised controlled trial data support the use of mepolizumab (mepo) in severe eosinophilic asthma (SEA). Real-world evidence from patients with diverse clinical profiles reflecting routine medical care will enhance clinical decision making. Objective: Describe a novel real-world hybrid data capture approach in an ongoing mepo study. Methods: This is a single-arm prospective observational study, aiming to recruit ~1,050 patients with SEA who have been prescribed mepo 100mg subcutaneous for up to 2y. The primary objective is to compare clinically significant exacerbation rates (requiring corticosteroids/emergency room visit/hospitalisation) in the 12m pre- vs post-mepo use. Key secondary assessments include mepo usage patterns, asthma control status, work productivity, healthcare utilisation (HCU), and change in oral corticosteroid use. A key feature of this study is the integration of various data capture methods: (i) electronic case report forms (eCRFs) to capture primary and secondary outcomes; (ii) claims data and electronic health records (EHR) to capture HCU; (iii) a smartphone app to collect exploratory data such as the impact of corticosteroid reduction on sleep; (iv) a sub-cohort will provide data on changes in activity and sleep via a wearable activity monitor. Primary endpoint data from eCRFs will be compared with EHR/claims data when available to assess the feasibility of using the latter in future studies. Conclusions: This study’s novel hybrid data capture, utilising both established and patient-direct approaches will provide patients, physicians and payers with real-world insights into patient care, asthma management and effectiveness of mepo treatment. Funding: GSK (204710 study)