Advanced or metastatic biliary tract cancer in Japan: a study using the Japan Medical Data Center payer claims database.

Aim: Biliary tract cancers are aggressive, with poor prognosis. This study describes clinical characteristics, treatment patterns and healthcare resource utilization in patients with metastatic biliary tract cancer in Japan. Materials & methods: This cohort-based study collected data from the Japan Medical Data Center claims database (2014-2018). Results: A total of 325 patients were included; 65.2% were male and the mean age was 59.2 years. A 47.6% had an Elixhauser Comorbidity Index score ≥5. Most frequent regimens were gemcitabine + cisplatin (52.9%) for first-line therapy and tegafur + gimeracil + oteracil for second-line therapy (48.6%) and third-line therapy (27.2%). Approximately 77% of patients had ≥1 hospital admission, with a median length of 57 days. Conclusion: This study provides insights on the characteristics and burden of metastatic biliary tract cancer in Japan, highlighting high disease burden in a younger population.

Real-world outcomes among patients with advanced or metastatic biliary tract cancers initiating second-line treatment.

BACKGROUND: Limited data are available regarding second-line (2 L) treatment for advanced or metastatic biliary tract cancers (BTC) in the US real-world setting. This study explores the rapidly evolving and growing treatment landscape in the 2 L setting for advanced or metastatic BTC with a large cohort of patients treated in a community oncology setting. METHODS: Adult patients with BTC initiating 2 L treatment after a platinum-containing first-line between 1/1/10- and 6/30/19 were identified from the US Oncology Network electronic healthcare record database and followed through 12/31/19. Baseline patient and treatment characteristics were analyzed descriptively, including overall response rate (ORR) in the real-world clinical setting. Kaplan-Meier methods were used to measure duration of response, progression-free survival (PFS), and overall survival (OS). RESULTS: The overall population (N = 160) included 74 patients (46.3%) with intrahepatic cholangiocarcinoma, 41 (25.6%) with extrahepatic cholangiocarcinoma, and 45 (28.1%) with gallbladder cancer. Thirty unique 2 L regimens were recorded for the study population, with folinic acid, fluorouracil and oxaliplatin (FOLFOX, 34.4%) and capecitabine monotherapy (20.0%) being the most common. ORR was 7.5% (95% CI, 3.9%-12.7%). From 2 L initiation, median PFS was 2.8 months (95% CI, 2.4-3.3 months), and median OS was 5.2 months (95% CI, 4.2-6.7 months). CONCLUSION: Results from this study provide real-world evidence that although patients treated in the community oncology setting receive a wide variety of 2 L treatments, the regimens are consistent with those recommended by guidelines. Although responses are observed with 2 L treatment, duration is brief and associated with poor OS in patients with advanced or metastatic disease.

Second-line treatments in advanced biliary tract cancer: systematic literature review of efficacy, effectiveness and safety.

Background: A systematic review was conducted to understand clinical, economic and health-related quality-of-life outcomes in second-line biliary tract cancer. Materials & methods: The review followed established recommendations. The feasibility of network meta-analysis revealed limited networks, thus synthesis was limited to a summary of reported ranges, percentiles and medians. Results: The review included 62 trials and observational studies highly variable with respect to key baseline characteristics. Commonly evaluated second-line treatments included fluoropyrimidine-, gemcitabine- and S-1-based regimens. Across active treatment arms, median overall survival ranged from 3.5 to 15.0 months (median: 6.9), median progression-free survival from 1.4 to 6.5 months (median: 2.9) and objective response from 0 to 36.4%. Outcomes were similar between study types, with a few notable outliers. Treatment-related/emergent adverse events were infrequently reported; no studies reported economic or health-related quality-of-life outcomes. Conclusions: Biliary tract cancer is a difficult-to-treat disease with poor prognosis. Despite evolving treatment landscapes, more recent studies did not show clinical outcome improvement, highlighting an unmet need among advanced/metastatic patients.

A systematic review of published literature was undertaken to understand the clinical, economic and health-related quality-of-life impact of second-line biliary tract cancer (BTC). A total of 62 relevant studies were identified. The patient populations included across these studies were highly variable with respect to key patient characteristics (i.e., age, sex, physical functioning and tumor type). Commonly evaluated treatments included fluoropyrimidine-, gemcitabine- and S-1-based regimens. Reported values for key outcomes varied substantially, somewhat explained by a few outlier studies. Median overall survival ranged from 3.5 to 15.0 months, median progression-free survival from 1.4 to 6.5 months and objective response from 0 to 36.4%. Treatment-related/emergent adverse events were infrequently reported; no studies reported economic or health-related quality-of-life outcomes. The results demonstrate that BTC is a difficult-to-treat disease with poor prognosis. Despite evolving treatment landscapes, more recent studies did not show clinical outcome improvement, highlighting an unmet need among advanced/metastatic second-line BTC patients.

A Real-World Claims Analysis of Costs and Patterns of Care in Treated Patients with Glioblastoma Multiforme in the United States.

BACKGROUND: Patients with glioblastoma multiforme (GBM) have a poor prognosis and high likelihood of recurrence. Routine care for incident cases in the United States involves surgical resection, followed by radiation therapy (RT) with concurrent and adjuvant temozolomide. Real-world data reporting the treatments and health care burden associated with GBM are limited. OBJECTIVE: To assess patterns of care, health care resource utilization (HCRU), and costs associated with treatment of GBM in the United States. METHODS: This study is a retrospective claims database analysis. Adult patients with a GBM diagnosis (index date) between January 1, 2010, and June 30, 2016, who had undergone brain surgery within 90 days of the index date, had received temozolomide and/or RT up to 90 days after index date, and had at least 6 months of continuous enrollment before the index date, were identified. Patients were excluded if they had (a) another primary cancer within 6 months pre-index, (b) secondary brain metastases, or (c) received temozolomide and/or RT pre-index. Baseline characteristics, treatments, HCRU, and costs were reported. First-line therapy began upon first receipt of RT and/or temozolomide after index date; second-line therapy began when a new drug was added > 28 days after initiation of first-line therapy or when there was a treatment gap > 90 days. Treatment regimens, duration of treatment (corrected group prognosis method), HCRU, and costs were reported descriptively in the 0- to 6-month and 7- to 12-month periods following initiation of first-line and second-line therapy. RESULTS: Baseline characteristics were comparable between patients receiving temozolomide and/or RT. Patients receiving RT without chemotherapy tended to be older, be retired, and have more baseline comorbidities. Of the 4,071 patients receiving first-line therapy for GBM, most (73.0%) received temozolomide + RT; 24.4% received RT; and 2.5% received temozolomide monotherapy. Of those receiving first-line therapy, 1,283 (31.5%) patients subsequently received second-line therapy: 39.4% received bevacizumab monotherapy; 28.9% received bevacizumab combination therapy (temozolomide, 45.2% of patients; irinotecan, 24.3%; and temozolomide + lomustine, 15.4%); 15.5% received temozolomide monotherapy; and 13.7% received other systemic cancer therapies. The proportion of patients with hospitalizations increased from 2.9% (4-6 months pre-index) to 20.8% in the 3 months before the index date (likely due to diagnostic procedures) and 28.1% in the first 6 months after index (likely due to surgery) and then decreased to 13.3% in the 7- to 12-month period after index. Mean total per-patient costs at 6 and 12 months were $117,325 and $162,550 (first line) and $126,128 and $243,833 (second line). Costs in all time periods were largely driven by costs of RT/systemic cancer therapy. CONCLUSIONS: Most patients with newly diagnosed GBM received treatment according to recommendations. However, relatively few patients received second-line therapy, and the HCRU burden and costs associated with both lines of therapy were substantial. Novel therapies for GBM are required to improve treatment options and outcomes in these patients. DISCLOSURES: This study was funded by Bristol-Myers Squibb (Princeton Pike, NJ). Neither honoraria nor payments were provided for authorship. Norden received consultancy fees relating to this study from Bristol-Myers Squibb. Dastani, Korytowsky, Le, Singh, and You are employees of Bristol-Myers Squibb. Dastani and Korytowsky are shareholders of Bristol-Myers Squibb. Bobiak was an employee of Bristol-Myers Squibb at the time of this study. Preliminary data from this study were previously presented at the International Society for Pharmacoeconomics and Outcomes Research 22nd Annual International Meeting in Boston, MA, May 20-24, 2017.

Variation in Definitive Therapy for Localized Non-Small Cell Lung Cancer Among National Comprehensive Cancer Network Institutions.

PURPOSE: This study determined practice patterns in the staging and treatment of patients with stage I non-small cell lung cancer (NSCLC) among National Comprehensive Cancer Network (NCCN) member institutions. Secondary aims were to determine trends in the use of definitive therapy, predictors of treatment type, and acute adverse events associated with primary modalities of treatment. METHODS AND MATERIALS: Data from the National Comprehensive Cancer Network Oncology Outcomes Database from 2007 to 2011 for US patients with stage I NSCLC were used. Main outcome measures included patterns of care, predictors of treatment, acute morbidity, and acute mortality. RESULTS: Seventy-nine percent of patients received surgery, 16% received definitive radiation therapy (RT), and 3% were not treated. Seventy-four percent of the RT patients received stereotactic body RT (SBRT), and the remainder received nonstereotactic RT (NSRT). Among participating NCCN member institutions, the number of surgeries-to-RT course ratios varied between 1.6 and 34.7 (P<.01), and the SBRT-to-NSRT ratio varied between 0 and 13 (P=.01). Significant variations were also observed in staging practices, with brain imaging 0.33 (0.25-0.43) times as likely and mediastinoscopy 31.26 (21.84-44.76) times more likely for surgical patients than for RT patients. Toxicity rates for surgical and for SBRT patients were similar, although the rates were double for NSRT patients. CONCLUSIONS: The variations in treatment observed among NCCN institutions reflects the lack of level I evidence directing the use of surgery or SBRT for stage I NSCLC. In this setting, research of patient and physician preferences may help to guide future decision making.

Bone metastases and skeletal-related events from neuroendocrine tumors.

Neuroendocrine tumors (NETs) metastasize to bone; however, a multi-institution evaluation of the natural history and complications of bone metastases across multiple NET subtypes has not, to our knowledge, previously been conducted. At two tertiary academic centers, we identified patients with bone metastases from databases of patients with a diagnosis of NET between 2004 and 2008. Detection of bone metastases, occurrence of skeletal-related events (SREs), and interventions were analyzed using summary statistics and categorical methods. Time-to-event data were assessed using Kaplan-Meier estimates and log-rank tests. Between 2004 and 2008, 82 out of 691 NET patients (12%) were reported to have bone metastases. Of the 82 patients with bone metastases, 55% were men and their median age was 49. Bone metastases occurred in 25% of pheochromocytomas and paragangliomas, 20% of high-grade neuroendocrine carcinomas, 9% of carcinoid tumors, and 8% of pancreatic NETs. At time of detection of bone metastases, 60% reported symptoms, including pain; 10% developed cord compression, 9% suffered a pathological fracture, and 4% developed hypercalcemia. Occurrence of SREs did not differ significantly with regard to tumor histology. Of patients with bone metastases, 67 (82%) received at least one form of bone-directed treatment, 50% received radiation, 45% received a bisphosphonate, 18% underwent surgery, 11% received (131)I-MIBG, 5% received denosumab, and 46% were treated with more than one treatment modality. Bone metastases occur in a substantial number of patients diagnosed with NETs. Patients are often symptomatic and many develop SREs. Given the recent therapeutic advances and increasing life expectancy of patients with NETs, development of guidelines for surveillance and clinical care of bone metastases from NETs is needed.

Appreciative Inquiry for quality improvement in primary care practices.

PURPOSE: To test the effect of an Appreciative Inquiry (AI) quality improvement strategy on clinical quality management and practice development outcomes. Appreciative inquiry enables the discovery of shared motivations, envisioning a transformed future, and learning around the implementation of a change process. METHODS: Thirty diverse primary care practices were randomly assigned to receive an AI-based intervention focused on a practice-chosen topic and on improving preventive service delivery (PSD) rates. Medical-record review assessed change in PSD rates. Ethnographic field notes and observational checklist analysis used editing and immersion/crystallization methods to identify factors affecting intervention implementation and practice development outcomes. RESULTS: The PSD rates did not change. Field note analysis suggested that the intervention elicited core motivations, facilitated development of a shared vision, defined change objectives, and fostered respectful interactions. Practices most likely to implement the intervention or develop new practice capacities exhibited 1 or more of the following: support from key leader(s), a sense of urgency for change, a mission focused on serving patients, health care system and practice flexibility, and a history of constructive practice change. CONCLUSIONS: An AI approach and enabling practice conditions can lead to intervention implementation and practice development by connecting individual and practice strengths and motivations to the change objective.

Measuring practice capacity for change: a tool for guiding quality improvement in primary care settings.

PURPOSE: Capacity for change, or the ability and willingness to undertake change, is an organizational characteristic with potential to foster quality management in health care. We report on the development and psychometric properties of a quantitative measure of capacity for change for use in primary care settings. METHODS: Following review of previous conceptual and empirical studies, we generated 117 items that assessed organizational structure, climate, and culture. Using information from direct observation and key informant interviews, a research team member rated these items for 15 primary care practices engaged in a quality improvement intervention. Distributional statistics, pairwise correlation analysis, Rasch modeling, and item content review guided item reduction and instrument finalization. Reliability and convergent validity were assessed. RESULTS: Ninety-two items were removed because of limited response distributions and redundancy or because of poor Rasch model fit. The final instrument comprising 25 items had excellent reliability (alpha = .94). A Rasch model-derived capacity for change score correlated well with an independently determined, qualitatively derived summary assessment of each practice's capacity for change (rhoS = 0.82), suggesting good convergent validity. CONCLUSION: We describe a new instrument for quantifying organizational capacity for change in primary care settings. The ability to quantify capacity for change may enable better recognition of practices likely to be successful in their change efforts and those first requiring capacity building prior to change interventions.

Opportunities for improved diabetes care among patients of safety net practices: a safety net providers' strategic alliance study.

OBJECTIVE: To identify barriers and opportunities for quality diabetes care in safety net practices. METHODS: In 3 federally qualified health centers and 1 free clinic, 19 primary care clinicians profiled patient and visit characteristics and quality of care measures for 181 consecutive visits by adult type 2 diabetic patients. Open-ended questions assessed patient and clinician perception of barriers to diabetes care and patient report of enabling factors. A multidisciplinary team identified themes from open-ended responses. Logistic regression analyses assessed the association of the identified barriers/enablers with 2 measures of quality care: glycosylated hemoglobin and prophylactic aspirin use. RESULTS: Ranked barriers noted by patients included adherence (40%), financial/insurance (23%), and psychosocial (13%) factors. Clinicians ranked systemic factors, including financial/ insurance (32%) and cultural/psychosocial (29%) factors, as important to adherence (29%) in determining quality diabetes care. Patients reported dietary and medical adherence (37%) and family/health care worker support (17%) as helpful factors. Among 175 patients with available data, glycosylated hemoglobin levels were associated with patient report of financial/insurance factors both as a barrier when visits and medications were unaffordable and as an opportunity when free or low-cost medications and services were provided. Patients' adherence with aspirin prophylaxis was strongly associated with African American race, prior prescription of aspirin and distribution of aspirin at the practice site (p<.001). CONCLUSIONS: Patients were less likely than clinicians to identify systemic and contextual factors contributing to poor diabetes care. From the front line's perspective, enabling patient self-management and systemic support is a target for improving diabetes care in safety net practices.

Correlates of baseline performance do not predict results of an intervention to improve preventive care.

BACKGROUND: Cross-sectional analyses of baseline performance often inform the development of interventions to improve care. An implicit assumption in these studies is that factors associated with better performance at baseline may also be useful in predicting change in performance over time. METHODS: We analyzed data collected from 1997-2002 at 77 practices in Northeast Ohio participating in an intervention to increase evidence-based preventive services delivery (PSD). Spearman's correlation coefficients and multivariable models assessed associations between practice-level characteristics (e.g., organizational structure, objectives, climate, and culture) and baseline PSD, and with final PSD controlling for baseline values. Patterns of associations for both outcomes were inspected for overlap. RESULTS: The mean PSD rate was 36.8% (+/-8.8%) at baseline. This measure increased by an average of 4.9% (+/-6.3%) by the end of the intervention. Of eight practice characteristics correlated with either baseline performance or change from baseline in PSD, only two were common to both: characteristics associated with baseline PSD did not predict final PSD in multivariable models. CONCLUSIONS: Correlates of baseline performance differ from those related to change in performance. Practice assessments that focus on factors associated with change may be more useful in developing and implementing interventions to improve care.

Possible unintended consequences of a focus on performance: insights over time from the research association of practices network.

BACKGROUND: New pay-for-performance initiatives and quality improvement efforts over the last decade have focused on evidenced-based process measures of quality of care. The effect on preventive service delivery (PSD) measures and patient satisfaction is not well described. The goals of this study were to describe PSD and patient satisfaction trends over the past decade. METHODS: We evaluated delivery of US Preventive Services Task Force (USPSTF)-recommended clinical preventive services and patient satisfaction in 3 studies conducted in the same health care market in 1995, 2000, and 2006, respectively, using previously validated measures. Trends in these 2 factors were compared using data from cross-sectional samples of 4454, 3462, and 1240 patients seen at 84, 80, and 31 participating primary care practices, respectively. RESULTS: In the 3 time periods, patients' rate of being up to date on USPSTF-recommended preventive services increased from 29% to 33% to 38%. Similar trends were observed in subscores for counseling, screening, and immunization services. However, patient satisfaction decreased to a clinically meaningful extent from 4.26 in 1995 to 3.93 in 2006 (range = 1-5). CONCLUSIONS: Quality improvement efforts focused on evidence-based recommendations appear to be working as intended, but with the possible unintended consequence of reduced patient satisfaction.

The association of how time is spent during outpatient visits and patient satisfaction: are there racial differences?

Both satisfaction with the physician and how time is spent in the patient-physician outpatient visit have been shown to differ between African-American and Caucasian patients. This study uses structural equation modeling to examine racial differences in the association between time use during the outpatient visit and patient satisfaction. This cross-sectional study employed direct observation of outpatient visits and surveys of 2,502 adult African-American and Caucasian outpatients visiting 138 primary care physicians in 84 family practices in Northeast Ohio. Patient satisfaction was measured using the Medical Outcome Study (MOS) nine-item Visit Rating Scale. Time use was assessed with the Davis Observation Code, which was used to classify every 20 seconds of a visit into 20 behavioral categories. No difference was found between African-American and Caucasian patients in the association between patient satisfaction with a physician and the time the physician spent chatting, planning treatment, providing health education, structuring the interaction, assessing health knowledge or answering patient questions. Patients were generally satisfied with their physicians, and no racial differences between Caucasians and African Americans were observed. Despite racial differences in how physicians spend time in the outpatient visit encounter, these differences are not associated with racial differences in patient satisfaction. Efforts to understand disparities in satisfaction should address areas other than how physicians allocate time in the physician-patient encounter.