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Background The Communicating Needs and Features of IBD Experiences (CONFIDE) study aimed to evaluate the experience and impact of ulcerative colitis (UC) symptoms on patients’ lives and elucidate gaps in communication between patients and health care professionals (HCPs). Methods Online, quantitative, cross-sectional surveys of patients with moderate-to-severe UC and HCPs responsible for making prescribing decisions were conducted in the United States (US) and Europe. UC disease severity was defined by treatment, steroid use, and/or hospitalization history. Results Surveys were completed by 200 US and 556 European patients and 200 US and 503 European HCPs. The most common UC symptoms experienced in the preceding month were diarrhea, bowel urgency, and increased stool frequency. Many patients (45.0% of US patients, 37.0% of European patients) reported wearing diapers/pads/protection at least once a week in the past 3 months due to fear/anticipation of fecal urge incontinence. The top reasons for declining participation in social events, work/school, and sports/exercise were due to bowel urgency and fear of fecal urge incontinence. HCPs ranked diarrhea, blood in stool, and increased stool frequency as the most common symptoms. While over half HCPs ranked bowel urgency as a top symptom affecting patients’ lives, less than a quarter ranked it in the top 3 most impactful on treatment decisions. Conclusions Similar disparities exist between patient and HCP perceptions in the United States and Europe on the experience and impact of UC symptoms. Bowel urgency has a substantial and similar impact on US and European patients, is underappreciated by HCPs, and should be addressed during routine appointments.

Palbociclib was the first cyclin-dependent kinase 4/6 inhibitor approved by the US Food and Drug Administration for use in combination with aromatase inhibitors (AIs) as initial endocrine-based therapy or with fulvestrant in postmenopausal women who previously received endocrine therapy based on data from randomized clinical trials. Real-world studies examining the effectiveness of palbociclib in large, diverse patient populations in routine clinical practice were needed. Ibrance Real World Insights (IRIS) was a retrospective medical record review study of women with confirmed hormone receptor–positive, HER2-negative advanced/metastatic breast cancer treated with palbociclib plus an AI or with palbociclib plus fulvestrant according to approved indications. Participating physicians reviewed medical records of up to 16 sequentially presenting patients, collecting demographic and clinical data. Outcomes included objective response rates, progression-free rates, and survival rates overall and in patients stratified according to age, race and ethnicity, Eastern Cooperative Oncology Group (ECOG) performance status (PS), disease-free interval, visceral disease, liver metastases, bone-only metastases, and previous lines of therapy. Data were abstracted by 417 physicians for 2954 patients in 13 countries; 1415 patients (47.9%) were ≥65 years of age, 369 patients (12.5%) had an ECOG PS ≥2 at initiation, and 835 patients (28.3%) were races other than White. The 12-month progression-free rate was 88% for palbociclib plus an AI and 79% for palbociclib plus fulvestrant; the 12-month survival rate was 96% in both groups. The objective response rates were 80% for palbociclib plus an AI and 75% for palbociclib plus fulvestrant. Palbociclib was similarly effective in most subgroups examined. Data from IRIS provide in-depth, real-world evidence for the use of palbociclib in a range of breast cancer populations in multiple countries. These data support the findings of the randomized PALOMA-2 and PALOMA-3 studies.

Moderate-to-severe ulcerative colitis (UC) and Crohn's disease (CD) are associated with substantial quality of life and economic burdens (Kawalec, 2016). The Communicating Needs and Features of IBD Experiences (CONFIDE) study aims to further the understanding of the experience and impact of symptoms on patients' lives and elucidate any gaps in communication between healthcare professionals (HCPs) and patients with moderate-to-severe UC and CD in the United States (US), Europe, and Japan. These data focus on US patients with UC and US HCPs. An online, quantitative, cross-sectional survey was conducted with HCPs (n=200) and patients with moderate-to-severe UC (n=200) in the US between May (HCPs) and July (patients) 2021. Moderate-to-severe UC was defined using criteria based on previous treatment experience, steroid use and/or hospitalization. The HCP survey included physicians (89%) and non-physician HCPs (11%) who are responsible for making prescribing decisions. Data collected included perspectives on the experience and impact of symptoms in patients with moderate-to-severe UC. The top three symptoms currently (past month) and ever suffered by patients (mean age: 40.4, 61.5% male) were diarrhea (62.5% and 74.0%, respectively), bowel urgency (47.0% and 61.5%) and increased stool frequency (38.5% and 57.5%). Blood in stool was reported by 27.0% of patients as currently suffering, and 51.0% ever. According to HCPs (78.0% male), the top three symptoms reported by patients were diarrhea (73.5% ranked in top 3), blood in stool (69.0%), and increased stool frequency (37.5%). Bowel urgency was recorded in the top 3 patient-reported symptoms by 24.0% of HCPs. Patients self-rated their disease-severity as 10.5% (n = 21) mild UC, 71.0% (n = 142) moderate UC, 17.5% (n = 35) severe UC, and 1.0% (n = 2) patients did not know. Bowel urgency was more frequently reported in patients with severe disease (62.9%, n = 22) when compared with those with mild-to-moderate disease (42.9%, n = 70). Among the overall patient population, 76.5% (n = 153) were receiving advanced therapies (biologic or novel oral therapy). Bowel urgency was currently experienced by 46.4% of these patients. Only 38.2% of patients felt completely comfortable reporting bowel urgency to their HCP. Of patients not comfortable reporting bowel urgency, 62.2% (n = 23) reported they felt embarrassed talking about it. Among HCPs, 75.5% (n = 151) reported they proactively discussed bowel urgency at routine appointments. Those HCPs who reported that they do not proactively discuss bowel urgency (24.5%, n = 49) cited the main reason as they expect the patient to bring it up (46.9%, n = 23). Bowel urgency is the second-most commonly reported symptom by patients with moderate-to-severe UC but is not among the HCP-perceived top three most reported symptoms. A substantial proportion of patients with moderate-to-severe UC receiving advanced therapies continue to report bowel urgency. A communication gap between patients and HCPs was identified and highlights the under appreciation of bowel urgency as an important symptom impacting patients' daily life.

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.

ObjectiveManagement of erosive oesophagitis (EE) remains suboptimal, with many patients experiencing incomplete healing, ongoing symptoms, and relapse despite proton pump inhibitor (PPI) treatment. The Study of Acid-Related Disorders investigated patient burden of individuals with EE in a real-world setting.DesignUS gastroenterologists (GIs) or family physicians (FPs)/general practitioners (GPs) treating patients with EE completed a physician survey and enrolled up to four patients with EE for a patient survey, with prespecified data extracted from medical records.Results102 GIs and 149 FPs/GPs completed the survey; data were available for 73 patients (mean age at diagnosis, 45.4 years). Omeprazole was healthcare professional (HCP)-preferred first-line treatment (60.8% GIs; 56.4% FPs/GPs), and pantoprazole preferred second line (29.4% and 32.9%, respectively). Price and insurance coverage (both 55.5% HCPs) and familiarity (47.9%) key drivers for omeprazole; insurance coverage (52.0%), price (50.0%), familiarity (48.0%), initial symptom relief (46.0%), and safety (44.0%) key drivers for pantoprazole. Only 49.3% patients took medication as instructed all the time; 56.8% independently increased medication frequency some of the time. Despite treatment, 57.5% patients experienced heartburn and 30.1% regurgitation; heartburn was the most bothersome symptom. 58.9% patients believed that their symptoms could be better controlled; only 28.3% HCPs were very satisfied with current treatment options. 83.6% patients wanted long-lasting treatment options. Fast symptom relief for patients was a top priority for 66.1% HCPs, while 56.6% would welcome alternatives to PPIs.ConclusionThis real-world multicentre study highlights the need for new, rapidly acting treatments in EE that reduce symptom burden, offer durable healing and provide symptom control.

Abstract Background Von Hippel-Lindau syndrome (VHL) is a rare hereditary neoplastic disorder caused by mutations in the VHL gene. Treatment options for patients are limited to multiple surgeries dispersed between regular scans, watchful waiting, and treatments that preserve organ function. Methods An international, cross-sectional survey comprising patients in the United States (USA), Canada (CA), the United Kingdom (UK), France (FR), and Germany (DE) was conducted. Patients were recruited via the VHL Alliance; data were collected between Dec 2021 and May 2022. For inclusion, patients must have renal cell carcinoma, pancreatic neuroendocrine tumors, and/or central nervous system hemangioblastoma. Results In all, 220 patients (68.2% female, median age 40.0, median disease duration 15.8 years) in the USA (n = 108), CA (n = 37), the UK (n = 21), FR (n = 3), and DE (n = 51) completed the study. In this sample, n = 205 (93.2%) patients had experienced surgery; n = 171 (77.7%) had experienced multiple surgeries (median number of surgeries, 4.0); 166 (n = 75.5%) patients recorded data on their most recent surgery. Of these, patients reported that their most recent surgery worsened (scored 1-3) their fatigue (51.8%, n = 86), mental health (51.2%, n = 85), and ability to go about daily life (45.2%, n = 75). Approximately, 47.3% (n = 104) of patients selected reducing the number of surgeries as a top treatment goal, whereas 73% (n = 161) of patients indicated they would prefer to take a pill which would possibly delay the time until surgery. Conclusion Surgery negatively impacts the lives of patients, leading to a worsening in their fatigue, mental health, and ability to go about daily life.