Patient-reported outcomes and healthcare resource utilization in systemic lupus erythematosus: impact of disease activity.

BACKGROUND: Limited real-world data exists on clinical outcomes in systemic lupus erythematosus (SLE) patients by SLE Disease Activity Index 2000 (SLEDAI-2 K), hereafter, SLEDAI. We aimed to examine the association between SLEDAI score and clinical, patient-reported and economic outcomes in patients with SLE. METHODS: Rheumatologists from the United States of America and Europe provided real-world demographic, clinical, and healthcare resource utilization (HCRU) data for SLE patients. Patients provided self-reported outcome data, capturing their general health status using the EuroQol 5-dimension 3-level questionnaire (EQ-5D-3 L), health-related quality of life using the Functional Assessment of Chronic Illness Therapy (FACIT) and work productivity using the Work Productivity and Activity Impairment questionnaire (WPAI). Low disease activity was defined as SLEDAI score ≤ 4 and ≤ 7.5 mg/day glucocorticoids; patients not meeting these criteria were considered to have "higher" active disease. Data were compared between patients with low and higher disease activity. Logistic regression estimated a propensity score for SLE based on demographic and clinical characteristics. Propensity score matched analyses compared HCRU, patient-reported outcomes, income loss and treatment satisfaction in patients with low disease activity versus higher active disease. RESULTS: Data from 296 physicians reporting on 730 patients (46 low disease activity, 684 higher active disease), and from 377 patients' self-reported questionnaires (24 low disease activity, 353 higher active disease) were analyzed. Flaring in the previous 12 months was 2.6-fold more common among patients with higher versus low active disease. Equation 5D-3 L utility index was 0.79 and 0.88 and FACIT-Fatigue scores were 34.78 and 39.79 in low versus higher active disease patients, respectively, indicating better health and less fatigue, among patients with low versus higher active disease. Absenteeism, presenteeism, overall work impairment, and total activity impairment were 47.0-, 2.0-, 2.6- and 1.5-fold greater in patients with higher versus low disease activity. In the previous 12 months there were 28% more healthcare consultations and 3.4-fold more patients hospitalized in patients with higher versus low disease activity. CONCLUSION: Compared to SLE patients with higher active disease, patients with low disease activity experienced better health status, lower HCRU, less fatigue, and lower work productivity impairment, with work absenteeism being substantially lower in these patients.

Systemic Lupus Erythematosus Symptom Clusters and Their Association With Patient-Reported Outcomes and Treatment: Analysis of Real-World Data.

OBJECTIVE: To identify discrete clusters of systemic lupus erythematosus (SLE) patients based on symptoms and investigate differences across clusters. METHODS: Data were collected in the US and 5 European countries via the Adelphi Real World Lupus Disease Specific Programme, a cross-sectional survey. Rheumatologists provided data for 5 consecutively consulting adult patients with SLE, who were invited to participate. Identified SLE symptoms were reduced to factors based on commonly concurrent symptoms, using principal-component factor analysis. Factors were used as covariates in a latent-class cluster analysis to identify discrete patient clusters. Patient-reported outcomes and physician-reported data were compared across clusters. RESULTS: Among 1,376 patients, 87% were female and 74% were White. We identified 4 patient clusters (very mild, mild, moderate, and severe) based on 39 signs/symptoms. Physician-reported symptom burden, organ involvement, disease activity, and the number of flares increased with increasing cluster severity (P < 0.0001). Patient-reported impact (health status, fatigue, work productivity impairment, anxiety/depression, and emotional impact) increased with increasing cluster severity (P < 0.0001). Glucocorticoid and immunosuppressant use increased, and antimalarial use decreased, with increasing cluster severity. In all clusters, <20% of patients received biologics; >15% of patients not receiving biologics were considered eligible for treatment by their physician. The proportion of physicians and patients satisfied with treatment decreased with increasing cluster severity (P < 0.0001). CONCLUSION: Our large, international, real-world survey of SLE patients and physicians demonstrated strong associations between increased impairment, organ involvement, and humanistic burden in SLE, highlighting an unmet need for effective treatment options in patients with high disease activity.

Real-world impact of flaring on patient-reported outcomes and healthcare resource utilisation in systemic lupus erythematosus.

OBJECTIVES: We investigated the association of SLE flares with patient-reported outcomes (PRO) and healthcare resource utilisation (HCRU) using real-world data. METHODS: Rheumatologists from the USA, France, Germany, Spain, Italy provided demographic, clinical, and HCRU data for patients with SLE, who provided PRO data. "Flaring" was defined as ≥1 rheumatologist-reported flare in the past 12 months. Demographic/clinical data were analysed descriptively, and findings compared statistically by flaring status. Logistic regression estimated a propensity score for flaring based on ethnicity, disease duration, and severity at diagnosis. Propensity score-matched flaring and non-flaring patients were compared for their HCRU, PROs, income loss and treatment satisfaction. RESULTS: Physicians (n=263) provided data for 1,278 patients (408 flaring/870 non-flaring); 729 patients (241 flaring/488 non-flaring) provided matched patient data. Patients had a mean 2.1 flares in the previous 12 months. Propensity score matched analyses indicated worse outcomes and greater HCRU in the past 12 months in flaring than non-flaring patients: EuroQoL 5D-3L Utility Index: 0.72 vs. 0.83; Functional Assessment of Chronic Illness Therapy-Fatigue scale: 30.06 vs. 36.48; Work Productivity and Activity Impairment Index: absenteeism 5.87% vs. 2.53% / presenteeism 33.44% vs. 19.16% / overall work impairment 35.98% vs. 20.66% / total activity impairment 42.47% vs. 30.23%; healthcare consultations (8.10 vs. 6.41), hospitalisations (24.26 vs. 7.63), emergency department visits (20.83 vs. 4.19), tests (46.59 vs. 38.90); current medications (2.76 vs. 2.19) (all p<0.001 except absenteeism, p=0.004). CONCLUSIONS: Similar flaring SLE patients had worse PROs and higher HCRU than non-flaring patients, underscoring the need for more effective strategies and treatments to alleviate or prevent flaring.

Burden of illness among subgroups of patients with primary Sjögren's syndrome and systemic involvement.

OBJECTIVES: To describe how patients with primary SS (pSS) and systemic organ involvement are classified and clustered in routine practice. METHODS: This multinational, cross-sectional survey of real-world quantitative data was conducted across Europe and the US. Rheumatologists who treated seven or more adult patients per month with pSS and current/past systemic manifestations undertook a survey before completing a patient record form capturing demographic, clinical and treatment information for their next six eligible patients. Patients with a completed patient record form were invited to complete a patient self-completion questionnaire capturing insights into their disease and treatment. Subgroups were defined by physicians' assessment of disease severity; clusters were derived based on key clinical characteristics using latent class analysis. RESULTS: Rheumatologists completed 316 physician surveys and 1879 patient record forms; 888 patients completed the patient self-completion questionnaire. pSS severity reflected organ involvement and symptomatology. Latent class analysis produced five clusters distinguished by the organ systems involved and the presence of pain and fatigue symptoms at the time of the survey. A minority of patients [n = 67 (4%)] were categorized with multiple organ involvement and the highest frequency of pain and fatigue. A total of 324 patients (17%) were categorized as 'low burden'. The remaining three clusters exhibited high frequencies of articular involvement but were distinguished by the extent of other organ system involvement. CONCLUSION: Cluster analysis using a real-world cohort of patients with pSS and systemic organ involvement highlights the heterogeneous presentation of patients with pSS and confirms the importance of pain and fatigue as well as organ involvement when determining disease burden.

Full, Partial, and Modified Permutations of the Mayo Score: Characterizing Clinical and Patient-Reported Outcomes in Ulcerative Colitis Patients.

Background: Understanding ulcerative colitis disease activity assessed via the full, modified, or partial Mayo Score may help clinicians apply results from clinical trials to practice and facilitate interpretation of recent and older studies. Methods: Mayo Score variables were assessed in a cross-sectional study of 2608 ulcerative colitis patients. Results: Permutations of Mayo Scores were highly correlated, and models predicting the omitted variable from each permutation demonstrated significant agreement between predicted and observed values. Conclusions: Partial/modified Mayo Scores may be used to predict endoscopic and Physician's Global Assessment scores, and serve as proxies for the full Mayo Score in clinical practice/trials.

Exploring the real-world profile of refractory and non-refractory chronic idiopathic urticaria in the USA: clinical burden and healthcare resource use.

Introduction: Chronic idiopathic/spontaneous urticaria (CIU/CSU) is a debilitating skin condition that is burdensome for patients and healthcare providers. We aimed to describe clinical characteristics, consultation patterns and healthcare resource utilization in real-world US patients with refractory and non-refractory CIU/CSU. Methods: Data was collected from the Adelphi Real World 2015 Urticaria Disease Specific Programme. Physicians completed patient record forms (PRFs) for the next four patients consulting with non-refractory CIU/CSU and the next six with refractory CIU/CSU; patients were considered refractory if symptomatic and on treatment step ≥2. The same patients were asked to complete patient self-completion (PSC) forms describing how CIU/CSU affected them. Results: Seventeen physicians (15 allergists; 2 dermatologists) completed 184 PRFs (108 refractory CIU/CSU; 76 non-refractory CIU/CSU); 140 patients completed PSC forms (93 refractory CIU/CSU; 47 non-refractory CIU/CSU). Mean time from first consultation to diagnosis was 13.5 (SD 28.3) weeks; mean time from diagnosis to first treatment was 16.0 (SD 37.9) weeks. Patients with refractory CIU/CSU were more likely to initially consult primary care physicians than those with non-refractory CIU/CSU (51% and 28%, respectively). The most common symptoms were itching, sleep problems and anxiety/distress, affecting 75%, 23% and 18%, respectively. Patient-perceived disease severity was greater than physician-perceived severity (refractory CIU/CSU kappa 0.1512; non-refractory CIU/CSU 0.1590). Conclusions: Patients with CIU/CSU in this real-world study - particularly those with refractory CIU/CSU - were slow to receive specialist care and had substantial symptom burdens; patient-physician perception of disease severity was discordant. Earlier diagnosis of CIU/CSU may lead to timely use of CIU/CSU therapies.

Humanistic Burden of Refractory and Nonrefractory Chronic Idiopathic Urticaria: A Real-world Study in the United States.

PURPOSE: Chronic idiopathic urticaria (CIU) is a debilitating skin condition that can profoundly affect patients' quality of life. This study explored the impact of refractory and nonrefractory CIU on patients in the real-world setting in the United States. METHODS: Data were collected from the Adelphi Real World 2015 Urticaria Disease-Specific Programme. Physicians completed patient record forms (PRFs) for 4 consecutive patients consulting with nonrefractory CIU and 6 patients with refractory CIU. The PRF included information on patient characteristics, medication, and disease severity; physicians were asked about the impact of CIU on patients, and to rate their satisfaction with patients' treatment (scale, 1-7 [1 = extremely dissatisfied and 7 = extremely satisfied]). The same patients were asked to complete a patient self-completion form (PSC). This included questions regarding how CIU affected their everyday life and their satisfaction with and understanding of their condition and medications. The PSC included a number of patient-reported outcomes measures: the Dermatology Life Quality Index (scored from 0 to 30, with higher scores indicating greater impact), the Work Productivity and Activity Impairment (4 scores calculated [absenteeism, presenteeism, work productivity loss, and activity impairment], each scored from 0% to 100% after transformation, with higher scores indicating greater impairment), and the Jenkins Sleep Scale (assessed over 30 days; scored from 0 to 20, with higher scores indicating greater sleep disturbance). Completion of the PSC was voluntary. FINDINGS: Seventeen physicians completed a total of 184 PRFs (108 for patients with refractory CIU; 76 for those with nonrefractory CIU); 140 of these 184 patients completed a PSC form (93 with refractory CIU; 47 with nonrefractory CIU). Overall, 26% of the entire population (30% of patients with refractory CIU and 17% with nonrefractory CIU) reported that skin symptoms had a great effect on their lives (Dermatology Life Quality Index score ≥11). Sleep problems were common: mean Jenkins Sleep Scale scores were 7.8 overall (8.1 for patients with refractory CIU and 7.2 for those with nonrefractory CIU). Overall work impairment was 19% in the overall population, with similar values in refractory and nonrefractory patients (18% and 20%, respectively). Physician satisfaction with disease control was not high, with physicians reporting a mean score of 4.8 on a 7-point scale. Twenty-one percent of patients were extremely dissatisfied, very dissatisfied, or dissatisfied with their treatment, and 52% believed better control could be achieved. IMPLICATIONS: The humanistic burden of CIU is high. There is clearly a need for better management of CIU to improve outcomes for patients.

Patient- and Physician-reported Satisfaction With Systemic Lupus Erythematosus Treatment in US Clinical Practice.

PURPOSE: This two-part study comprised two descriptive, cross-sectional surveys to evaluate treatment satisfaction among patients with systemic lupus erythematosus (SLE) and their physicians from US clinical practices. The Lupus Plus Project (LPP; part one) involved belimumab-containing regimens; the Disease Specific Program (DSP; part two) included all treatments and was designed to build on the body of evidence from part one. METHODS: The LPP recruited patients receiving belimumab, and comprised 2 paper questionnaires: a patient self-completion questionnaire (PSC) and a patient record form (PRF) completed by the physician. The DSP enrolled patients with SLE receiving any treatment and comprised four parts: a PSC, a PRF completed by the physician after patient consultation, face-to-face physician interviews, and a workload form completed by the physicians to indicate their total SLE patient workload. The key objective of this study was to assess physician and patient satisfaction with current treatment. FINDINGS: From the PSCs, data regarding patient-reported satisfaction with current treatment were available for 263 patients who were receiving belimumab combination therapy (LPP) and 250 patients who were receiving non-belimumab treatment (DSP). The majority of patients (belimumab, 86.3% [227/263]; non-belimumab, 78.4% [196/250]) responded positively (at least "somewhat satisfied") when asked about current treatment satisfaction, as did physicians (belimumab, 82.9% [311/375]; non-belimumab, 74.3% [326/439]). In multivariate analysis, factors most strongly associated with patient-reported satisfaction for patients receiving belimumab were patient-reported improvements in leisure activities since taking belimumab (odds ratio [OR] = 4.66), physician-reported improvements in fatigue (OR = 3.72), patient-reported improvements in general symptoms (OR = 3.02), and pain/achiness (OR = 2.71). Physician satisfaction was associated with clinical outcome such as improvements in pain/achiness (OR = 6.16), fatigue (OR = 3.76), and patient-reported satisfaction with treatment frequency (OR = 3.91). In patients receiving other SLE treatments, dosing frequency of current treatment (OR = 3.64) and a reduction in fatigue severity (OR = 3.61) were most strongly associated with patient-reported satisfaction; physician satisfaction was most strongly associated with a reduction in fatigue (OR = 6.22) and current remission status (OR = 6.05). IMPLICATIONS: When considering SLE treatment satisfaction patients tend to consider impact on daily functioning, whereas physicians take into account a wider range of clinical outcomes; however, both strongly consider improvements in fatigue. These surveys provide insights into treatment satisfaction among prescribers and patients with SLE. GSK-ClinicalStudyRegister.com identifiers: GSK study 202146 [HO 15-15509] and 205086 [HO 15-16709].