Unmet needs in uncomplicated urinary tract infection in the United States and Germany: a physician survey.

BACKGROUND: Uncomplicated urinary tract infections (uUTIs/acute cystitis) are among the most common infections in women worldwide. There are differences in uUTI treatment guidelines between countries and understanding the needs of physicians in diverse healthcare systems is important for developing new treatments. We performed a survey of physicians in the United States (US) and Germany to understand their perceptions of, and management approaches to uUTI. METHODS: This was a cross-sectional online survey of physicians in the US and Germany who were actively treating patients with uUTI (≥ 10 patients/month). Physicians were recruited via a specialist panel and the survey was piloted with 2 physicians (1 US, 1 Germany) prior to study commencement. Data were analyzed with descriptive statistics. RESULTS: A total of 300 physicians were surveyed (n = 200 US, n = 100 Germany). Across countries and specialties, physicians estimated 16-43% of patients did not receive complete relief from initial therapy and 33-37% had recurrent infections. Urine culture and susceptibility testing was more common in the US and among urologists. The most commonly selected first-line therapy was trimethoprim-sulfamethoxazole in the US (76%) and fosfomycin in Germany (61%). Ciprofloxacin was the most selected following multiple treatment failures (51% US, 45% Germany). Overall, 35% of US and 45% of German physicians agreed with the statement "I feel there is a good selection of treatment options" and ≥ 50% felt that current treatments provided good symptom relief. More than 90% of physicians included symptom relief amongst their top 3 treatment goals. The overall impact of symptoms on patients' lives was rated "a great deal" by 51% of US and 38% of German physicians, increasing with each treatment failure. Most physicians (> 80%) agreed that antimicrobial resistance (AMR) is serious, but fewer (56% US, 46% Germany) had a high level of confidence in their knowledge of AMR. CONCLUSIONS: Treatment goals for uUTI were similar in the US and Germany, although with nuances to disease management approaches. Physicians recognized that treatment failures have a significant impact on patients' lives and that AMR is a serious problem, though many did not have confidence in their own knowledge of AMR.

Caregivers of patients with malignant pleural mesothelioma: who provides care, what care do they provide and what burden do they experience?

PURPOSE: There are limited data on the impact of caregiving for patients with malignant pleural mesothelioma (MPM) on the caregiver. We aimed to identify the demographic characteristics of these caregivers, the caregiving activities they perform and how caregiving burden impacts their work productivity and overall activity. METHODS: This cross-sectional study collected data from caregivers of patients with MPM across France, Italy, Spain and the United Kingdom January-June 2019. Caregiver demographics, daily caregiving tasks and the impact of caregiving on physical health was collected via questionnaire. The Zarit Burden Interview (ZBI) was used to assess caregiver burden and the Work Productivity and Activity Impairment questionnaire (WPAI) assessed impairment at work and during daily activities. Analyses were descriptive. RESULTS: Overall, 291 caregivers provided data. Caregivers were mostly female (83%), living with the patient (82%) and their partner/spouse (71%). Caregivers provided over five hours of daily emotional/physical support to patients. ZBI scores indicated 74% of caregivers were at risk of developing depression. Employed caregivers had missed 12% of work in the past seven days, with considerable presenteeism (25%) and overall work impairment (33%) observed. Overall, the mean activity impairment was 40%. CONCLUSION: Caregivers provide essential care for those with MPM. We show caregiving for patients with MPM involves a range of burdensome tasks that impact caregivers' emotional health and work reflected in ZBI and WPAI scores. Innovations in the management of MPM must account for how caregivers may be impacted and can be supported to carry out this important role.

Quantifying The Burden of Persistent Musculoskeletal Pain in Employees at Rolls-Royce in The United Kingdom: A Real-World Cross-Sectional Survey.

OBJECTIVE: We aimed to investigate the burden of persistent musculoskeletal (MSK) pain in Rolls-Royce UK employees. METHODS: Employees with ( n = 298) and without ( n = 329) persistent MSK pain completed a cross-sectional survey. Weighted regression analyses were conducted to compare sickness absence, work ability, workplace accommodations/adaptations, and emotional well-being between these cohorts, controlling for confounders. RESULTS: Persistent MSK pain (particularly back pain) had a significant impact on physical work ability and was associated with increased sickness absence due to pain. Many employees (56%) had not disclosed their condition to their managers. Of these, 30% felt uncomfortable doing so, and 19% of employees reported insufficient support at work for their pain. CONCLUSIONS: These findings highlight the importance of creating a workplace culture that encourages the disclosure of work-relevant pain, enabling organizations to consider improved, tailored support for employees.

Real-world Treatment Patterns and Clinical Outcomes Associated With Palbociclib Combination Therapy: A Multinational, Pooled Analysis From the Ibrance Real World Insights Study.

PURPOSE: 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. PATIENTS AND METHODS: 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. FINDINGS: 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. IMPLICATIONS: 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.

Treatment and disease management patterns for bacillus Calmette-Guérin unresponsive nonmuscle invasive bladder cancer in North America, Europe and Asia: A real-world data analysis.

Background: This study examined real-world treatment and management of bacillus Calmette-Guérin (BCG)-unresponsive patients across 3 continents, including patients unable or unwilling to undergo cystectomy. Materials and methods: Physicians actively involved in managing patients with nonmuscle invasive bladder cancer completed online case report forms for their 5 consecutive patients from the broad BCG-unresponsive population and a further 5 consecutive BCG-unresponsive patients who did not undergo cystectomy (in Japan, physicians provided a total of 5 patients across both cohorts). Results: Most patients had received 1 (37%) or 2 (24%) maintenance courses of BCG. Five or more maintenance BCG courses were received by patients in Japan (59%) and China (31%), while in Germany 76% of patients received only 1 course. Most patients became BCG-unresponsive during their first (44%) or second (22%) treatment course; in Germany, 77% became BCG-unresponsive during their first treatment course. Most countries did not provide another course of BCG after a patient first became unresponsive, whereas unresponsive patients in Japan and China were most likely to be retreated with BCG. "Untreated - on watch and wait" was the main treatment/management approach received post-BCG treatment for 42% or more of patients in most countries except China (39%) and the United States (36%). "Following treatment guidelines" was consistently the top reason for post-BCG treatment selection across all treatment options. Conclusions: This study confirmed the global unmet need for patients with nonmuscle invasive bladder cancer, and found that many patients experienced periods of no treatment after not responding to BCG therapy.

Malignant pleural mesothelioma: treatment patterns and humanistic burden of disease in Europe.

BACKGROUND: Malignant pleural mesothelioma (MPM) is an aggressive and rare tumour with poor prognosis. Most patients are diagnosed with advanced disease and there is a paucity of data on the humanistic burden of MPM in terms of impact on health-related quality of life (HRQoL) and activity. This study examined real-world treatment patterns and humanistic disease burden of MPM in Europe. METHODS: Physicians abstracted demographic/clinical characteristics and treatment data from MPM-patient medical records; MPM patients self-completed a questionnaire including symptoms, 3-level-EQ-5D questionnaire and Visual Analogue Scale (VAS), Lung Cancer Symptom Scale for Mesothelioma (LCSS-Meso), and Work Productivity and Activity Impairment (WPAI) questionnaire. RESULTS: Physicians (n = 171) abstracted data of 1390 patients; 767/1390 patients self-completed questionnaires. Patients were elderly with advanced, unresectable MPM. Treatment patterns followed guidelines with most (81%) patients receiving platinum+antifolate chemotherapy at first line (1 L). Maintenance treatment use was high (51.1%) despite no recommended maintenance therapies. Symptom burden was high and health states and HRQoL were poor at 1; declining further with progression. Overall mean (SD): LCSS-Average Symptom Burden Index score was 48.8 (19.3; n = 758); EQ-5D Utility Index score was 0.510 (0.349; n = 763); EQ-5D VAS score was 54.2 (20.3;n = 766); LCSS-3-Item Global Index score was 143.2 (64.5; n = 762); LCSS-normal activities score was 51.9 (24.6;n = 765); WPAI-activity impairment was 56.0% (23.2%; n = 737). CONCLUSION: The humanistic burden of MPM is high, despite treatments being prescribed as per available guidance. Treatments that delay progression and provide palliation of symptoms are most likely to improve/maintain HRQoL.

Real-world treatment patterns and clinical outcomes in patients receiving palbociclib combinations for HR+/HER2- advanced/metastatic breast cancer in Japan: Results from the IRIS study.

BACKGROUND: The incidence of breast cancer is rising in Japan, particularly in postmenopausal women. The CDK 4/6 inhibitor palbociclib has demonstrated efficacy in clinical studies in patients with hormone receptor-positive (HR+), human epidermal growth factor 2 (HER2)-negative advanced/metastatic breast cancer (ABC/MBC). The Ibrance Real World Insights (IRIS) study (NCT03159195) collected real-world data for palbociclib-treated patients in several countries including Japan, where such data are currently scarce. METHODS: IRIS was a retrospective chart review study of patients with confirmed HR+/HER2- ABC/MBC receiving palbociclib according to approved indications in real-world clinical practice. In Japan, physicians each abstracted data from patient medical records for up to eight sequential patients treated with palbociclib plus an aromatase inhibitor (P+AI) or fulvestrant (P+F). Outcomes included progression-free rates (PFRs) and survival rates (SRs). RESULTS: Fifty-eight physicians abstracted data for 170 patients receiving palbociclib in the first (64.1%) or second or later line (35.9%), in combination with AI (51.2%) or fulvestrant (48.8%). Median follow-up was 10.4 months. Most patients were initiated on palbociclib 125 mg/d (P+AI, 63.2%; P+F, 78.3%). PFRs at 12 and 24 months were 76.2% and 52.6%, respectively, for P+AI and 71.6% and 65.6%, respectively for P+F. PFRs at 12 and 24 months were 85.4% and 66.5%, respectively, for first-line palbociclib combinations and 56.4% and 50.7%, respectively, for second- or later-line palbociclib combinations. CONCLUSIONS: In this analysis of the Japanese IRIS cohort, outcomes in terms of PFRs and SRs appear to be better with first- versus second or later-line palbociclib, regardless of the endocrine partner.

Multi-country clinical practice patterns, including use of biomarkers, among physicians' treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC).

BACKGROUND: Intravesical bacillus Calmette-Guérin (BCG) fails in a considerable proportion of non-muscle invasive bladder cancer (NMIBC) patients despite treatment per recommended protocol. This real-world study aimed to understand the current patterns of treatment and disease management for the broad BCG-unresponsive NMIBC patient population, alongside collecting sufficient data on patients who do not undergo cystectomy. METHODS: This was a multicenter, retrospective survey of physicians treating BCG-unresponsive NMIBC patients. Data were collected in eight countries - France, Germany, Spain, Italy, United Kingdom, United States, China, and Japan - between January and May 2019. The study consisted of a short online physician survey and a retrospective chart review of eligible BCG-unresponsive NMIBC patients. Physicians abstracted chart data for the last 10 (five patients in Japan) eligible BCG-unresponsive NMIBC patients meeting the inclusion criteria, and the data were analysed for all countries combined using descriptive statistics. Country-specific analyses were also carried out, as appropriate. RESULTS: Overall, 508 physicians participated in the study. Almost one-quarter (22.9%) of physicians' current NMIBC patient caseload was BCG-unresponsive, whereby BCG therapy was no longer considered an option. Half of physicians (49.4%) did not regularly use biomarker tests in their practice, with particularly few physicians undertaking biomarker testing in Spain and Japan. Biomarker testing varied considerably, with the proportions of physicians selecting 'none' ranging from 11.4% in China to 70.3% in Japan. Physicians reported transurethral resection of the bladder tumor (TURBT) and BCG as the most common current treatments received by their patients. Chemotherapy and anti-PD-L1 treatment options were considered impactful new therapies by 94.7% and 90.0% of physicians surveyed in this study, respectively. CONCLUSIONS: The most common treatments received by patients in this study were TURBT and BCG. Emerging new treatments are driven by exploring biomarkers, but in real-world clinical practice only half of physicians or fewer regularly tested their NMIBC patients for biomarkers; PD-1/PD-L1 was the most common biomarker test used. Most physicians reported that, in addition to chemotherapy, anti-PD-L1 was an impactful new therapy.

Real-world treatment of patients with palbociclib for HR+/HER2-advanced/metastatic breast cancer: the Europe IRIS study.

Aim: To report the Europe Ibrance Real World Insights study findings. Methods: Physicians abstracted demographic/clinical characteristics, treatment and outcomes data for women with HR+/HER2- locally advanced breast cancer (ABC) or metastatic  breast cancer (MBC) receiving palbociclib + aromatase inhibitor (AI) or palbociclib + fulvestrant. Kaplan-Meier analysis estimated progression-free rates (PFRs) and survival rates (SRs). Results: 238 physicians abstracted data for 1723 patients. For patients (>90%) initiating at 125 mg/day, dose was reduced in 18.9% of palbociclib + AI and 12.3% of palbociclib + fulvestrant patients. At 12 months, PFR for palbociclib + AI was 88.1%, and SR was 97.3%; PFR for palbociclib + fulvestrant was 79.8%, and SR was 97.5%. Conclusion: Low dose-reduction rates and favorable PFRs and SRs suggest that palbociclib + AI/fulvestrant is well tolerated and effective for HR+/HER2- ABC/MBC in real-world clinical practice.

Lay abstract We describe findings of the Europe Ibrance Real World Insights study. Patients were women with a common type of breast cancer that had worsened but not spread or that had spread. Doctors collected medical information about the women and looked at their progress while on treatment. The treatment was either palbociclib + an aromatase inhibitor or palbociclib + fulvestrant. Two hundred thirty-eight doctors collected information on 1723 women. More than 90% of women started this treatment at a dose of 125 mg/day; the dose was reduced for fewer than 20% of women. At 12 months, more than 80% of women survived without their breast cancer worsening, and more than 97% of women survived. These good results suggest that this treatment is safe and effective for women with breast cancer that had worsened but not spread or that had spread.

Real-world study of the impact of recurrent/metastatic squamous cell carcinoma of the head and neck (R/M SCCHN) on quality of life and productivity in Europe.

BACKGROUND: Although current therapy for patients with early-stage squamous cell carcinoma of the head and neck (SCCHN) is potentially curative, the recurrence rate is high. Patients with recurrent or metastatic (R/M) SCCHN have a poor prognosis and substantial disease burden, including impaired health-related quality of life (HRQoL), productivity loss and indirect costs, such as need for caregiver support. The aim of this study was to characterize the impact of R/M SCCHN and its first-line treatment on patient and caregiver quality of life, daily activities and work productivity using real-world evidence from Europe. METHODS: This was a multicentre retrospective study of patients with R/M SCCHN in France, Germany, Italy, Spain and the United Kingdom incorporating patient and caregiver surveys, and a physician-reported medical chart review, conducted between January and May 2019. Patients aged 18 or over with a physician confirmed diagnosis R/M SCCHN completed four validated measures of disease activity and its impact on quality of life and work productivity, while caregivers also completed questionnaire to assess the burden of providing care. Physicians provided data for clinical characteristics, patient management, testing history and treatment patterns. RESULTS: A total of 195 medical/clinical oncologists provided data for 937, predominantly male (72%) patients, with almost half of patients aged over 65 years. The most frequently reported symptoms were fatigue (43%), weight loss (40%), pain (35%) and difficulty swallowing (32%). The EXTREME regimen was the most common first line therapy in over half of patients, who reported moderate or extreme pain/discomfort, and anxiety/depression, and problems with self-care resulting in a diminished health status compared with the general population. Only 14% were employed with high absenteeism or presenteeism, and over half of patients had a caregiver for whom the burden of care was substantial. CONCLUSION: Our results provide real-world insight into the multi-faceted burden associated with R/M SCCHN. The combination of poor HRQoL and the impairment in daily activities, social life and employment illustrates the wider impact of R/M SCCHN on patients and their caregivers, and highlights a need for novel 1 L treatment regimens to improve the humanistic and productivity burdens of this cancer.

Real-World Palbociclib Use in HR+/HER2- Advanced Breast Cancer in Canada: The IRIS Study.

BACKGROUND: Palbociclib is a selective cyclin-dependent kinase (CDK) 4/6 inhibitor used in combination with aromatase inhibitors or fulvestrant for patients with hormone receptor-positive (HR+) human epidermal growth factor receptor 2 (HER2)-negative advanced/metastatic breast cancer (ABC/MBC). Palbociclib was the first CDK 4/6 inhibitor approved for HR+/HER2- ABC/MBC treatment in Canada in combination with letrozole (P+L) as an initial endocrine-based therapy (approved March 2016), or with fulvestrant (P+F) following disease progression after prior endocrine therapy (approved May 2017). The Ibrance Real World Insights (IRIS) study (NCT03159195) collected real-world outcomes data for palbociclib-treated patients in several countries, including Canada. METHODS: This retrospective chart review included women with HR+/HER2- ABC/MBC receiving P+L or P+F in Canada. Physicians reviewed medical records for up to 14 patients, abstracting demographic and clinical characteristics, treatment patterns, and clinical outcomes. Progression-free rates (PFRs) and survival rates (SRs) at 6, 12, 18, and 24 months were estimated via Kaplan-Meier analysis. RESULTS: Thirty-three physicians examined medical records for 247 patients (P+L, n = 214; P+F, n = 33). Median follow-up was 8.8 months for P+L and 7.0 months for P+F. Most patients were initiated on palbociclib 125 mg/d (P+L, 90.2%; P+F, 84.8%). Doses were reduced in 16.6% of P+L and 14.3% of P+F patients initiating palbociclib at 125 mg/d. The PFR for P+L was 90.3% at 12 months and 78.2% at 18 months; corresponding SRs were 95.6% and 93.0%. For P+F, 6-month PFR was 91.0%; 12-month SR was 100.0%. CONCLUSIONS: Dose reduction rates were low and PFR and SR were high in this Canadian real-world assessment of P+L and P+F treatments, suggesting that palbociclib combinations are well tolerated and effective.

Immune thrombocytopenia (ITP) World Impact Survey (iWISh): Patient and physician perceptions of diagnosis, signs and symptoms, and treatment.

Immune thrombocytopenia (ITP) is now well-known to reduce patients' health-related quality of life. However, data describing which signs and symptoms patients and physicians perceive as having the greatest impact are limited, as is understanding the full effects of ITP treatments. I-WISh (ITP World Impact Survey) was an exploratory, cross-sectional survey designed to establish the multifaceted impact of ITP, and its treatments, on patients' lives. It focused on perceptions of 1507 patients and 472 physicians from 13 countries regarding diagnostic pathway, frequency and severity of signs and symptoms, and treatment use. Twenty-two percent of patients experienced delayed diagnosis (caused by several factors), 73% of whom felt anxious as a result. Patients rated fatigue among the most frequent, severe symptom associated with ITP at diagnosis (58% most frequent; 73% most severe), although physicians assigned it lower priority (30%). Fatigue was one of the few symptoms persisting at survey completion (50% and 65%, respectively) and was the top symptom patients wanted resolved (46%). Participating physicians were experienced at treating ITP, thereby recognizing the need to limit corticosteroid use to newly-diagnosed or first-relapse patients and espoused increased use of thrombopoietin receptor agonists and anti-CD20 after relapse in patients with persistent/chronic disease. Patient and physicians were largely aligned on diagnosis, symptoms, and treatment use. I-WISh demonstrated that patients and physicians largely align on overall ITP symptom burden, with certain differences, for example, fatigue. Understanding the emotional and clinical toll of ITP on the patient will facilitate shared decision-management, setting and establishment of treatment goals and disease stage-appropriate treatment selection.

Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): Impact of ITP on health-related quality of life.

Immune thrombocytopenia (ITP) has a substantial, multifaceted impact on patients' health-related quality of life (HRQoL). Data describing which aspects of ITP physicians and patients perceive as having the greatest impact are limited. The ITP World Impact Survey (I-WISh) was a cross-sectional survey, including 1507 patients and 472 physicians, to establish the impact of ITP on HRQoL and productivity from patient and physician perspectives. Patients reported that ITP reduced their energy levels (85% of patients), capacity to exercise (77%), and limited their ability to perform daily tasks (75%). Eighty percent of physicians reported that ITP symptoms reduced patient HRQoL, with 66% reporting ITP-related fatigue substantially reduced patient HRQoL. Patients believed ITP had a substantial impact on emotional well-being (49%) and 63% worried their condition would worsen. Because of ITP, 49% of patients had already reduced, or seriously considered reducing their working hours, and 29% had considered terminating their employment. Thirty-six percent of patients employed at the time of the survey felt ITP decreased their work productivity, while 51% of patients with high/very high symptom burden reported that ITP affected their productivity. Note, I-WISh demonstrated substantive impact of ITP on patients' HRQoL both directly for patients and from the viewpoint of their physicians. Patients reported reduced energy levels, expressed fears their condition might worsen, and those who worked experienced reduced productivity. Physicians should be aware not only of platelet counts and bleeding but also the multi-dimensional impact of ITP on patients' lives as an integral component of disease management.

Real-World Treatment Patterns and Clinical Outcomes in Patients Receiving Palbociclib for Hormone Receptor-Positive, Human Epidermal Growth Factor Receptor 2-Negative Advanced or Metastatic Breast Cancer in Argentina: The IRIS Study.

PURPOSE: The selective cyclin-dependent kinase 4/6 inhibitor palbociclib was approved in Argentina in 2015 for postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer (ABC) or metastatic breast cancer (MBC) based on phase III study results. The Ibrance Real World Insights (IRIS) study aims to evaluate palbociclib in patients with HR-positive/HER2-negative ABC or MBC in the real-world setting in multiple countries globally. Here we report results from patients enrolled in the IRIS study in Argentina. PATIENTS AND METHODS: This retrospective medical chart review study included postmenopausal women with confirmed HR-positive/HER2-negative ABC or MBC who were treated with palbociclib plus letrozole as first-line endocrine-based therapy or with palbociclib plus fulvestrant in women with disease progression after endocrine therapy. Participating physicians reviewed medical records of up to six patients each, collecting demographic and clinical data. Outcomes included progression-free and overall survival rates. RESULTS: Records were extracted for 162 patients in Argentina (palbociclib plus letrozole, n = 105 [65%]; palbociclib plus fulvestrant, n = 57 [35%]). The 6-month progression-free survival rate was 94% for patients treated with palbociclib plus letrozole and 95% for patients treated with palbociclib plus fulvestrant; 85% and 80% of patients treated with palbociclib plus letrozole were progression free at 12 and 18 months, respectively. Six-month survival rates were 98% for palbociclib plus letrozole and 98% for palbociclib plus fulvestrant; 93% and 89% of patients treated with palbociclib plus letrozole were alive at 12 and 18 months, respectively. CONCLUSION: Results from this first real-world evaluation of clinical outcomes in Argentina suggest that palbociclib plus letrozole or fulvestrant delivers favorable effectiveness, as measured by progression-free and overall survival rates.

Cost burden associated with advanced non-small cell lung cancer in Europe and influence of disease stage.

BACKGROUND: Although evidence suggests that stage of disease may influence costs associated with non-small cell lung cancer (NSCLC), there remains a relative paucity of data on the financial burden incurred directly by patients and their informal caregivers as they progress through the disease course. As part of a large, cross-sectional study of the "real-world" humanistic and financial burden of advanced NSCLC in Europe, an analysis was conducted to quantify the cost burden of disease from a patient and caregiver perspective, and to evaluate how stage of disease impacts these costs. METHODS: Financial data were collected (May 2015-June 2016) during a multinational (France, Germany, and Italy) cross-sectional study of adults with advanced NSCLC (stage IIIB-IV) and their informal (unpaid) caregivers. Data were obtained via medical chart reviews and patient/caregiver self-completion forms. Costs were annualized and unadjusted or adjusted for government financial support. Statistical significance was assessed using Mann-Whitney U tests. RESULTS: One thousand thirty patients and 427 accompanying caregivers were recruited and asked to provide cost data. Mean total unadjusted direct and indirect out-of-pocket expenses were €5691 for patients and €4125 for caregivers; after adjusting for government financial support, values were €2644 and €3477. Mean wage losses were significantly higher for patients with stage IV vs IIIB NSCLC (€2282 vs €499; p = 0.0135) as were unadjusted direct out-of-pocket expenses (€4020 vs €1546; p = 0.0306). For caregivers, a similar but non-significant trend was observed. Mean total unadjusted direct and indirect out-of-pocket costs were numerically higher for stage IV vs IIIB NSCLC among patients (€5925 vs €3528) and caregivers (€4319 vs €2232); government financial support normalized patient costs, but they remained numerically higher for stage IV disease among caregivers. CONCLUSIONS: The financial burden of advanced NSCLC is considerable and appears to be influenced by stage of disease, with direct and indirect costs increasing as the disease progresses. Government financial support programmes appear to mitigate additional cost burdens among patients, but not among caregivers.

The humanistic burden of advanced non-small cell lung cancer (NSCLC) in Europe: a real-world survey linking patient clinical factors to patient and caregiver burden.

PURPOSE: Advanced non-small cell lung cancer (aNSCLC) impacts the lives of patients and their caregivers. This analysis examined the association between patient clinical characteristics and patient and caregiver humanistic burden. METHODS: Data for patients with aNSCLC and their informal caregivers in France, Germany and Italy, were collected between May 2015 and June 2016 via chart review and patient and caregiver surveys. Patients and caregivers completed validated instruments to evaluate their health state (EuroQol-5-dimensions-3-levels [EQ-5D-3L]), work and activity impairment (Work Productivity Activity Impairment [WPAI]) and health-related quality of life (HRQoL; European Organisation for Research and treatment of Cancer Quality of Life Questionnaire [EORTC QLQ-C30]). Caregivers also completed the Zarit Burden Interview (ZBI). Univariate and regression analyses were stratified by patient Eastern Cooperative Group Performance Status (ECOG-PS 0, 1, 2 or 3/4). RESULTS: In total, 1030 patients and 427 accompanying informal caregivers participated. Regression analyses indicated that patients reported lower EQ-5D-3L utility index, EQ-VAS and EORTC QLQ-C30 global health status and greater work and activity impairment with worsening ECOG-PS (all p < 0.05). Caregivers also reported greater activity impairment and higher ZBI scores with worsening ECOG-PS of the patient they were providing care for (all p < 0.05). CONCLUSIONS: As patients' functionality deteriorates as measured by the ECOG-PS, so do their outcomes related to health utility, work productivity, activity impairment and HRQoL. This deterioration is also reflected in increased caregiver burden and activity impairment. There is a need for interventions to maintain patients' physical function to relieve the humanistic burden of both patients and caregivers.

Treatment patterns and clinical outcomes among patients receiving palbociclib in combination with an aromatase inhibitor or fulvestrant for HR+/HER2-negative advanced/metastatic breast cancer in real-world settings in the US: Results from the IRIS study.

PURPOSE: Palbociclib is a selective cyclin-dependent kinase (CDK) 4/6 inhibitor approved for use in postmenopausal women with hormone receptor-positive, human epidermal growth factor 2-negative (HR+/HER2-) advanced/metastatic breast cancer (ABC/MBC). Palbociclib has proven benefits in phase III placebo-controlled studies; however, real-world outcome data are lacking. The Ibrance Real World Insights (IRIS) study evaluated palbociclib use in patients with HR+/HER2- ABC/MBC in the real-world setting in the US, Argentina, and Germany. Here we describe results for the US patient subgroup. PATIENTS AND METHODS: IRIS was a retrospective medical chart review study of patients with confirmed HR+/HER2- ABC/MBC who received palbociclib with either an aromatase inhibitor (AI) as initial endocrine-based therapy in postmenopausal women or fulvestrant-based therapy in women with disease progression following endocrine therapy. Physicians extracted data from patient medical records for ≤16 sequential patients each. Outcomes included progression-free and survival rates. RESULTS: Records were extracted for 652 patients: 360 (55.2%) treated with palbociclib + AI and 292 (44.8%) treated with palbociclib + fulvestrant. The 12-month progression-free rate was 84.1% for patients treated with palbociclib + AI and 79.8% for those treated with palbociclib + fulvestrant; 12-month survival rates were 95.1% for palbociclib + AI and 87.9% for palbociclib + fulvestrant. CONCLUSION: In this first real-world assessment of clinical outcomes in US patients with HR+/HER- ABC/MBC, treatment with palbociclib in combination with AI or fulvestrant demonstrated favorable effectiveness in terms of progression-free and survival rates. Ongoing studies are needed to deliver mature clinical outcome data beyond 12/24 months in the real-world setting.

Chart review across EU5 in MM post-ASCT patients.

AIM: To understand the current treatment patterns, clinical outcomes and healthcare resource utilization-associated costs for multiple myeloma patients, post autologous stem cell transplant (ASCT) across Europe. PATIENTS & METHODS: Medical records were used to abstract data for 337 multiple myeloma patients who had received ASCT. RESULTS: Following ASCT, 7% received maintenance therapy prior to progression. Lenalidomide was the most frequently prescribed maintenance, second- and third-line therapy. Monthly resource use was considerably lower in patients who received maintenance therapy (€638.14 vs €1001.74). Median time to progression was longer for patients who had received maintenance therapy. CONCLUSION: The study highlights the diversity in current treatment patterns post-ASCT. Results suggest patients who receive maintenance therapy have a prolonged remission period, and as a result their associated healthcare resource utilization is spread across the treatment pathway.

The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey.

Myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) are myeloproliferative neoplasms (MPNs) associated with high disease burden, reduced quality of life (QOL), and shortened survival. To assess how MPNs affect patients, we conducted a global MPN Landmark survey. This online survey of patients with MPNs and physicians was conducted in Australia, Canada, Germany, Japan, Italy, and the United Kingdom. The survey measured MPN-related symptoms and the impact of MPNs on QOL and the ability to work as well as disease-management strategies. Overall, 219 physicians and 699 patients (MF, n = 174; PV, n = 223; ET, n = 302) completed the survey; 90% of patients experienced MPN-related symptoms. The most frequent and severe symptom was fatigue. Most patients experienced a reduction in QOL, including those with low symptom burden or low-risk scores. A substantial proportion of patients reported impairment at work and in overall activity. Interestingly, physician feedback and blood counts were the most important indicators of treatment success among patients, with improvements in symptoms and QOL being less important. Regarding disease management, our study revealed a lack of alignment between physician and patient perceptions relating to communication and disease management, with patients often having different treatment goals than physicians. Overall, our study suggested that therapies that reduce symptom burden and improve QOL in patients with MPNs are crucial in minimizing disease impact on patient daily lives. Additionally, our findings showed a need for improved patient-physician communication, standardized monitoring of symptoms, and agreement on treatment goals.