US Cancer Patients Research Articles

Updated estimates of eligibility for and response to genome-targeted oncology drugs among US cancer patients, 2006-2020

Prior studies have evaluated the percentage of cancer patients with advanced or metastatic cancer who are eligible for and respond to genome-targeted therapy, but since that publication, the number of Food and Drug Administration (FDA) approvals for drugs targeting genetic indications has grown rapidly. We sought to update the estimates of both eligibility for and response to genome-targeted and genome-informed therapies in US cancer patients for FDA-approved drugs to reflect estimates as of 2020. We used mortality data from the American Cancer Society to estimate eligibility for these drugs, based on prevalence statistics from the published literature. We then multiplied eligibility by the response rate in the FDA label to generate an estimate for the percentage of US cancer patients who respond. For genome-targeted therapy, we estimate that the eligibility increased from 5.13% in 2006 to 13.60% in 2020. For genome-targeted therapy, we estimate that the response increased from 2.73% in 2006 to 7.04% in2020. The percentage of US cancer patients who are eligible for and respond to genome-targeted therapy has increased over time. Most of the increase in eligibility for genome-targeted therapies was seen after 2018, whereas most of the increase in response was seen before2018.

The financial coping strategies of US cancer patients and survivors.

Coping behaviors may play a mediating role in producing the negative health outcomes observed in financially burdened cancer patients and survivors. Exploratory factor and latent class analysis of survey data. A total of 510 people completed the survey, ages ranged from 25 to over 75 [over half greater than 55 years old (57.8%)]. Most respondents identified as female (64.7%), white (70.8%), or African American (18.6%). A four-factor model of financial coping was revealed: care-altering, lifestyle-altering, self-advocacy, and financial help-seeking. Respondents grouped into three financial coping classes: low burden/low coping (n = 212), high self-advocacy (n = 143), and high burden/high coping (n = 155). African American respondents were at far greater odds than white respondents of being in the high burden/high coping class (OR = 5.82, 95% CI 3.01-6.64) or the self-advocacy class (OR = 1.99, 95% CI 1.19-2.80) than the low burden/low coping class. Compared to respondents aged 65 years and older, those 35-44 were more likely in the high burden/high coping class (OR = 12.27, 95% CI 7.03-19.87) and the high self-advocacy class (OR = 7.08, 95% CI 5.89-8.28) than the low burden/low coping class. One-third of respondents were in the high burden/high coping class. Age and race/ethnicity were significantly associated with class membership. Some coping strategies may compromise health and well-being. Program and policy interventions that reduce the odds that patients will use strategies that undermine treatment outcomes and increase patient use of protective strategies are needed.

Initial experience of patients treated in a real-world clinical setting with bevacizumab-awwb: The first FDA-approved biosimilar to bevacizumab.

81 Background: Bevacizumab-awwb is the first anti-cancer biosimilar and the first biosimilar to bevacizumab approved by the FDA. It was launched in the United States on July 19, 2019. Bevacizumab-awwb approval was based on a phase 3 study in a pre-determined sensitive population of non-squamous non-small cell lung cancer (NSCLC) patients. This study investigated initial use of bevacizumab-awwb, across its approved indications for advanced cancer management, in real-world US community and academic oncology practices. Methods: This retrospective observational study described demographic and clinical characteristics of patients who received bevacizumab-awwb using structured patient-level data from the nationwide de-identified Oncology Services Comprehensive Electronic Records (OSCER), a Flatiron Health EHR-derived database. The database is longitudinal, demographically and geographically diverse, and includes more than 2.2 million US cancer patients from >280 cancer clinics. This analysis included adult patients who received ≥1 bevacizumab-awwb infusion between July 30, 2019 and April 30, 2020 and focused on describing initial biosimilar uptake in cancer patient populations beyond NSCLC. Results: First use of bevacizumab-awwb was in mCRC and occurred within 10 days of product launch. All bevacizumab-awwb approved cancer indications were represented among 2,422 patients treated with bevacizumab-awwb. Initial use of bevacizumab-awwb across indications: mCRC (68%), NSCLC (14%), brain cancer (11%), cervical cancer (5%), metastatic renal cell carcinoma (1%). Among 1657 mCRC patients treated with bevacizumab-awwb, 55% were male with a median age of 65 years (y). Most patients were white (60%), with ECOG scores of 0/1 (84%), and median body weight was 78 kg. Among mCRC patients, 59% had received the reference product before receiving the biosimilar. Among patients with other tumor types, 42-50% had prior bevacizumab exposure. Among mCRC patients with prior exposure to bevacizumab, 67% received bevacizumab-awwb within 28 days of last infusion of reference product (Table). Demographic and clinical characteristics were comparable between patients with and without prior bevacizumab use. Conclusions: Early evidence suggests physicians are comfortable initiating or transitioning patients to the first FDA-approved biosimilar to bevacizumab, with little evidence that demographic characteristics differ between patients with and without previous treatment with bevacizumab. Future analyses will determine whether these patterns persist across all indications. [Table: see text]

Fine Particulate Matter Air Pollution and Mortality Risk Among US Cancer Patients and Survivors.

BackgroundExposure to fine particulate matter (PM2.5) air pollution has been linked to increased risk of mortality, especially cardiopulmonary and lung cancer mortality. It is unknown if cancer patients and survivors are especially vulnerable to PM2.5 air pollution exposure. This study evaluates PM2.5 exposure and risk for cancer and cardiopulmonary mortality in cohorts of US cancer patients and survivors.MethodsA primary cohort of 5 591 168 of cancer patients and a 5-year survivor cohort of 2 318 068 was constructed using Surveillance, Epidemiology, and End Results Program data from 2000 to 2016, linked with county-level estimates of long-term average concentrations of PM2.5. Cox proportional hazards models were used to estimate PM2.5-mortality hazard ratios controlling for age-sex-race combinations and individual and county-level covariables.ResultsOf those who died, 26% died of noncancer causes, mostly from cardiopulmonary disease. Minimal PM2.5-mortality associations were observed for all-cause mortality (hazard ratio [HR] = 1.01, 95% confidence interval [CI] = 1.00 to 1.03) per 10 µg/m3 increase in PM2.5. Substantial adverse PM2.5-mortality associations were observed for cardiovascular (HR = 1.32, 95% CI = 1.26 to 1.39), chronic obstructive pulmonary disease (HR = 1.10, 95% CI = 1.01 to 1.20), influenza and pneumonia (HR = 1.55, 95% CI = 1.33 to 1.80), and cardiopulmonary mortality combined (HR = 1.25, 95% CI = 1.21 to 1.30). PM2.5-cardiopulmonary mortality hazard ratio was higher for cancer patients who received chemotherapy or radiation treatments.ConclusionsAir pollution is adversely associated with cardiopulmonary mortality for cancer patients and survivors, especially those who received chemotherapy or radiation treatment. Given ubiquitous and involuntary air pollution exposures and large numbers of cancer patients and survivors, these results are of substantial clinical and public health importance.

Clinical and treatment characteristics of patients treated with the first therapeutic oncology biosimilars bevacizumab-awwb and trastuzumab-anns in the US.

Background:In July 2019, bevacizumab-awwb and trastuzumab-anns were marketed in the USA as the first therapeutic oncology biosimilars. We aimed to investigate the initial real-world use of bevacizumab-awwb and trastuzumab-anns for cancer management in US oncology practices.Methods:A retrospective, observational analysis of data from US cancer patients (⩾18 years of age) was carried out to describe the use of bevacizumab-awwb and trastuzumab-anns during the first 12 months following their market entry, using structured data from the Flatiron Health electronic health record-derived database.Results:A total of 2952 and 2997 patients with recorded use of bevacizumab-awwb and trastuzumab-anns, respectively, were included in the analysis. The first use of bevacizumab-awwb and trastuzumab-anns was in a patient with metastatic colorectal cancer (mCRC) within 10 days of market availability and in a patient with early stage breast cancer (eBC) within 4 days, respectively. The use of these biosimilars was observed across all approved cancer indications; 68% of bevacizumab-awwb users were those diagnosed with mCRC and 72% of trastuzumab-anns users were those diagnosed with eBC. Approximately half the patients were previously exposed to reference product (RP) prior to initiation of bevacizumab-awwb or trastuzumab-anns. Among pre-exposed patients, the majority received the biosimilars [bevacizumab-awwb (63–85%) or trastuzumab-anns (75–81%)] within 28 days of the last infusion of the RP. For both biosimilars, no major differences were observed in patient characteristics between RP-naïve and pre-exposed patients.Conclusion:Initial evidence from the first 12 months following market entry suggests rapid clinical adoption of bevacizumab-awwb and trastuzumab-anns across all approved tumor types. Usage of these two biosimilars was observed in both RP-naïve patients and patients who were previously treated with RP, with no distinctive differences in patient characteristics between the two groups.A video abstract is available for this article as part of the Kanjintionline supplemental material.

Other and All-Cause Mortality among Men Diagnosed with Prostate Cancer in the PLCO Trial

Men with prostate cancer have high cause-specific survival, and most deaths are from other causes. This study aimed to investigate other and all-cause mortality in a large cancer screening cohort. From the PLCO (Prostate, Lung, Colorectal and Ovarian) Cancer Screening Trial cohort, we selected men diagnosed with prostate cancer from 1994-2014. We examined other and all-cause survival by prostate cancer risk level, defined as the D'Amico categories for localized disease (low, intermediate and high risk) plus nonlocalized disease. We developed 3 Cox proportional hazards models to assess the relationship between risk level and survival. Model I controlled for age, race, study arm and diagnosis year. Model II additionally controlled for other demographic and medical history factors. Model III additionally controlled for initial treatment. Of 76,672 men in PLCO and 10,859 prostate cancer cases, 9,248 (85.2%) had known prostate cancer risk level (mean±SD age 70.4±6.2 years). Median followup time from diagnosis was 10.8 years (IQR 6.8-15.0). Of 3,318 deaths 81% were from other causes. Compared to the low risk group, other-cause mortality HRs were 1.13 (95% CI 1.04-1.23), 1.35 (95% CI 1.21-1.50) and 1.63 (95% CI 1.35-1.97) for intermediate risk, high risk and advanced disease, respectively, in model II. Model III HRs were similar to model II except for advanced disease, where the HR decreased to 1.35. Other-cause survival was greater in lower vs higher risk disease, even after controlling for lifestyle characteristics and comorbidities. Further research is needed to identify factors contributing to this higher other-cause mortality to help mitigate the risk.

The association between Medicaid insurance, biomarker testing, and outcomes in patients with advanced non-small cell lung cancer (aNSCLC).

89 Background: Prior work suggests that patients (pts) with Medicaid experience lower quality cancer care and worse outcomes than pts with private insurance. It is unclear whether these disparities persist in the era of personalized medicine. We therefore examined the relation between Medicaid (vs private) insurance and receipt of biomarker testing, targeted therapy, and overall survival in a contemporary cohort of pts with aNSCLC. Methods: We conducted a retrospective study of pts with aNSCLC, diagnosed Jan 2011 through Sep 2019 using the nationwide US Flatiron Health EHR-derived de-identified databases comprising data from over 280 (largely community based) cancer clinics (~800 sites of care) and representing more than 2.4 million US cancer patients available for analysis. Eligible pts were 18-64 years old, had either Medicaid or Commercial insurance at the time of advanced diagnosis, and had a visit ≤120 days after advanced diagnosis. We assessed evidence of receipt of biomarker testing (ALK, EGFR, BRAF, ROS1, PDL1) according to year of diagnosis. The likelihood of testing, biomarker-driven therapy (immunotherapy (CIT) or tyrosine kinase inhibitor (TKI) treatment and mortality were compared by insurance type using Cox regression adjusted for baseline characteristics. Results: The sample included 6,145 commercially insured and 865 Medicaid pts. Compared with commercially insured pts, Medicaid beneficiaries were more likely to be Black (20% vs. 9.3%); P < 0.01) and less likely to have evidence of testing with any biomarkers (57% vs. 71%; P < 0.01). In the adjusted analysis, Medicaid pts were less likely to have evidence of testing (HR [95%CI]: 0.81 [0.74, 0.89], p < 0.01), any 1L treatment (HR [95%CI]: 0.72 [0.66, 0.79], p < 0.01) and 1L biomarker-driven therapy (HR [95%CI]: 0.70 [0.58, 0.84], p < 0.01). A higher risk of death was observed in Medicaid vs. commercially insured pts (HR [95%CI]: 1.23 [1.13, 1.34], p < 0.01) which was partially ameliorated after adjusting for testing and treatment (HR [95% CI: 1.15 [1.05, 1.25], p < 0.01). Among Medicaid pts, those who had no biomarker tests had a 27% higher risk of mortality compared to those who had at least one test (HR [95% CI: 1.27 [1.06, 1.53], p < 0.01). Conclusions: Medicaid pts with aNSCLC had a 23% higher risk of mortality than commercially insured pts. The increased risk of mortality was partially mediated by disparities in testing and treatment, and improved access to these resources may improve outcomes for Medicaid beneficiaries.

Abstract 5772: Validation analysis of a composite real-world mortality endpoint for US cancer patients

Abstract Objective: In oncology research, mortality, as a variable, and overall survival (OS), as an efficacy endpoint, are critical. As real-world data gains use in supporting regulatory decisions, assessing the validity of mortality data is important. Mortality data derived solely from electronic health records (EHRs) has known gaps. We developed a mortality variable based on multiple sources of death data and benchmarked it against the National Death Index (NDI) data as a gold standard within 4 cancer types (Curtis et al, 2018). We refreshed and expanded validity assessment to 18 cancer types. Methods: Patients diagnosed with at least 1 of 18 cancer types between 1/1/2011 and 12/31/2017 were included from the Flatiron Health EHR-derived de-identified US database. To develop a composite mortality variable, we amalgamated multiple sources of real-world death data, linking patient-level structured (EHR, commercial, Social Security Death Index) and unstructured data curated via technology-enabled abstraction. We calculated validity metrics (sensitivity, specificity, ±15-day accuracy) by benchmarking against the NDI data in each cancer type, as a composite variable, as well as by individual and combinations of death data sources. OS was estimated using the Kaplan-Meier method. Results: There were 160,436 patients included in the study cohort. In the 18 cancer types, sensitivity ranged from 83.9 - 91.5% (17 out of 18 had sensitivity ≥85%), specificity ranged from 93.5 - 99.7%, and ±15-day accuracy ranged from 95.6 - 97.6%, compared to the NDI (Table). Median OS estimates for the composite mortality variable when compared to that from the NDI ranged from 3 - 13% greater across all cancer types. Cancer Type Note: some patients could have >1 cancer typeNSensitivity (%)Specificity (%)+/- 15-day Accuracy (%)Solid TumorBreast Cancer (Early)166983.9 (77.4, 90.3)99.7 (99.5, 100)96.3 (92.7, 99.9)Breast Cancer (Metastatic)1647388.3 (87.6, 88.9)97.7 (97.4, 98.0)96.9 (96.6, 97.3)Colorectal Cancer (Metastatic)1723286.4 (85.7, 87.1)97.1 (96.8, 97.5)96.3 (95.9, 96.8)Gastro-Esophageal Cancer (Advanced)716988.5 (87.6, 89.4)94.3 (93.3, 95.3)97.1 (96.6, 97.6)Head and Neck Cancer (Advanced)527189.3 (88.2, 90.3)95.9 (95.0, 96.8)96.9 (96.3, 97.5)Hepatocellular Carcinoma278485.0 (83.3, 86.7)95.7 (94.5, 96.9)95.6 (94.5, 96.6)Malignant Pleural Mesothelioma170091.0 (89.4, 92.6)95.6 (93.7, 97.4)97.6 (96.7, 98.5)Melanoma (Advanced)703189.5 (88.4, 90.6)98.7 (98.4, 99.1)97.4 (96.8, 98)Non-small Cell Lung Cancer (Advanced)4507090.4 (90.1, 90.7)95.1 (94.8, 95.5)97.2 (97.0, 97.4)Ovarian Cancer496486.8 (85.2, 88.3)98.7 (98.3, 99.1)96.8 (95.9, 97.6)Pancreatic Cancer (Metastatic)545890.2 (89.3, 91.1)93.5 (92.2, 94.8)97.2 (96.7, 97.7)Prostate Cancer (Metastatic)849589.0 (88.0, 90)98.7 (98.4, 99.0)97.1 (96.5, 97.7)Renal Cell Carcinoma (Metastatic)577088.5 (87.4, 89.6)97.7 (97.1, 98.3)97.2 (96.6, 97.8)Small Cell Lung Cancer472490.4 (89.4, 91.4)95.8 (94.7, 96.8)97.5 (97.0, 98.1)Urothelial Cancer (Advanced)629390.2 (89.3, 91.1)96.6 (95.8, 97.4)97.5 (97.0, 98.0)Liquid TumorChronic Lymphocytic Leukemia903591.5 (90.4, 92.7)99.3 (99.1, 99.5)96.8 (96.1, 97.6)Diffuse Large B-cell Lymphoma434489.1 (87.4, 90.8)99 (98.7, 99.4)96.1 (95.0, 97.3)Multiple Myeloma780388.5 (87.3, 89.7)99.0 (98.8, 99.3)97.0 (96.3, 97.7) Conclusions: We observed high sensitivity, specificity, and date accuracy of our composite mortality variable across all cancer types. Future research will investigate the validity of our mortality variable stratified by demographic and clinical characteristics to understand performance in subpopulations. Citation Format: Qianyi Zhang, Anala Gossai, Shirley Monroe, Nathan C. Nussbaum, Christina M. Parrinello. Validation analysis of a composite real-world mortality endpoint for US cancer patients [abstract]. In: Proceedings of the Annual Meeting of the American Association for Cancer Research 2020; 2020 Apr 27-28 and Jun 22-24. Philadelphia (PA): AACR; Cancer Res 2020;80(16 Suppl):Abstract nr 5772.

A cost analysis of managing cancer-related pain among hospitalized US cancer patients.

7079 Background: Pain is a common symptom of cancer, affecting patients' function and quality of life. It is also a common cause of hospitalization for cancer patients. The aim of this study was to evaluate the cost of in-hospital pain management among US cancer patients. Methods: A retrospective analysis of data from all US hospitals that contributed to the National Inpatient Sample for 2011-2015 was conducted. All cancer patients admitted for pain management were included in the analysis. Main outcomes were factors significantly associated with hospital length of stay, total charge per hospital stay, and in-hospital mortality. Weighted chi-square test was used for categorical covariates and univariate analysis was performed using a logistic model. Results: 122,776 patient discharges were identified. Mean age was 59.3 years and 52.3% were female. 65.9% stayed in the hospital for longer than 72 hours, with a median total hospital charge of $48,156. Conversely, the median total hospital charge for those spending less than 72 hours on admission was $15,966. Median total charge per hospital stay was similar among insured and uninsured/self-pay patients ($32,879 vs. $32,323; p=0.013), but higher in patients without metastatic disease ($33,315 vs. $29,369; p<0.001). It was also higher in those with the highest income quartile when compared with lowest income patients ($38,223 vs. $30,047; p<0.001). Co-morbid medical illnesses were more prevalent in those with longer hospital stay (15 vs. 12; p<0.001) and the overall in-hospital mortality rate was 8.2%. There was no significant difference in median total hospital charges between those who died in, or those discharged from the hospital ($33,746 vs. $32,795; p<0.001). On multivariate analyses, gender, race, insurance status, diagnosis of metastatic cancer, age, number of co-morbid medical illnesses, year of diagnosis, and median income were significant predictors of length of stay. Race, insurance payor, metastatic cancer, age, and number of co-morbid medical illnesses were significant predictors of total hospital charges, after adjusting for other covariates. Conclusions: In-patient pain management of cancer patients is associated with significant health care costs. Optimization of outpatient pain management strategies could significantly lower the cost of care for cancer.

A retrospective three-year analysis using real-world data on uptake of broad-based NextGen sequencing panels in community oncology practices.

e13668 Background: There are over 100 FDA approved targeted therapies across 15 cancer types, offering improved outcomes over existing therapies. However, many of these require genetic testing, for example, advanced non-small cell lung cancer (aNSCLC) patients have over 15 targeted therapies requiring a DNA-based test. Doing multiple tests can exhaust sample, while increasing cost and turn-around time. NGS panels, often with hundreds of genes, can address some of these issues. Here we asked across aNSCLC patients if the use of NGS panels has increased over the last 3 years in community oncology practices. Methods: The Integra Connect database, which includes electronic medical record (EMR) and claims data on over 1,000,000 US cancer patients, was queried across five community oncology practices to identify aNSCLC patients (stage IIIB or IV) treated between January 2017 and January 2020. Manual chart review abstracted tumor type, stage, treatment, and testing. Patients tested for all 7 NCCN recommended genes (EGFR, ALK, ROS1, BRAF, MET, RET, ERBB2) were grouped as “NGS Panel”, patients with less genes as “Single Gene/Small Panel”, and patients with no evidence of testing as “No Test”. A Chi-Square test was used to compare actionable results between patients with NGS panels versus small panels. Results: 1,007 aNSCLC patients were analyzed and showed a doubling of the use of broad-based NGS testing from 13% in 2017 to 26% in 2019 across over 100 oncologists (table). 23% of patients had actionable results when tested on broad-based panels versus 17% using single gene or small panels (p = .048). Targeted therapies were used in 17% of broad-based tested patients, versus 15% in patients tested for single genes or small panels. Conclusions: We see an uptake of broad-based NGS testing in community oncology, which can lead to more actionable results and better utilization of targeted therapies for those patients. However, this seems to be caused by providers shifting from small panels to large panels, rather than an overall increase in testing, as we do not see the percentage of untested patients decrease. [Table: see text]

Assessment of enrollment characteristics for Children's Oncology Group (COG) upfront therapeutic clinical trials 2004-2015.

BackgroundImprovements in pediatric cancer survival are attributed to cooperative clinical trials. Under-representation of specific demographic groups has been described in adult and pediatric cancer trials and poses a threat to the generalizability of results. An evaluation of data provided by the Children’s Oncology Group (COG) of upfront trial enrollment for US patients 0 to 29 years old between 2004 and 2015 was performed.MethodsUS cancer cases were estimated using incidence data and US population estimates from the Surveillance, Epidemiology, and End Results Program and compared to observed COG cases. Percent enrollment and standardized ratios of enrollment were calculated across demographic, disease, and socioeconomic groups. The COG website was utilized to quantify available trials and assess age eligibility.Results19.9% of estimated US cancer patients age 0 to 19 years enrolled on COG trials. Younger patients were more represented across diseases and races/ethnicities. Patients with hematologic malignancies were more represented compared to solid and central nervous system (CNS) tumors.ConclusionCOG trial enrollment rates are declining when compared to previously published data, potentially from challenges in pediatric drug development, difficulty designing feasible trials for highly curable diagnoses, and issues ensuring trial availability for the heterogeneous group of solid and CNS tumors. Though racial/ethnic groups and county-level socioeconomic factors were proportionally represented, under representation of the adolescent/young adult (AYA) population and younger patients with solid and CNS tumors remains a concern. Targeted efforts should focus on these subgroups and further research should evaluate AYA enrollment rates across all available trials.

Real-World Dosing Patterns and Outcomes of Patients With Metastatic Pancreatic Cancer Treated With a Liposomal Irinotecan Regimen in the United States.

Liposomal irinotecan (nal-IRI) is a topoisomerase inhibitor proven to improve survival in metastatic pancreatic cancer (mPC). This study describes real-world characteristics of patients treated with nal-IRI for mPC. Patients 18 years or older diagnosed with stage IV mPC and treated with nal-IRI were selected retrospectively from a deidentified electronic health record database of more than 2 million US cancer patients. Demographics, clinical and dosing characteristics, and treatment outcomes were collected. Of 257 total patients, 145 (57%) received nal-IRI as first- or second-line therapy. Median nal-IRI treatment duration was 51 days, longer when nal-IRI was used as first/second versus as third-line therapy or later (62 vs 44.5 days). Seventy patients (27.2%) experienced dose modification. Median time to treatment discontinuation was 2.3 versus 1.6 months for first-/second- versus third-line therapy or later, respectively. Median overall survival from nal-IRI initiation was 5.6 versus 4.1 months for first-/second- versus third-line therapy or later, respectively. Prior irinotecan treatment, baseline serum albumin less than 40 g/L, and baseline neutrophil-to-lymphocyte ratio greater than 5 were associated with reduced overall survival. This is the first large US study of real-world US mPC patients treated with nal-IRI. These results, comparable to the NAPOLI-1 trial, can help inform future studies and the efficacy of nal-IRI in mPC therapy.

Mobile Head Tracking for eCommerce and Beyond

Long term collections of human tissue or body fluids in biobanks combined with health-related data are important resources for medical research and the development of personal medicine in the future. At present, increasing number of clinical studies published new diagnostic, preventive options or treatment approaches of especially age related diseases such as diabetes, cancer or neurodegeneration. For some years, a rapid improvement in the mutual exchange of data and common quality assurance policies between biobanks can be observed especially in western countries. However, global web-based interfaces suitable to search for disease-related information and biomaterial are not yet in practice partly due to differences in national private policy laws and missing cooperative efforts between health care IT systems. In a new collaborative project between the Technical Hochschule of Brandenburg and the Faculty of Health Sciences, a joint Faculty of the Brandenburg University of Technology Cottbus-Senftenberg, the Brandenburg Medical School Theodor Fontane and the University of Potsdam, we try to fill this gap and review the technical and juristic requirements to accesses biomaterial and clinical data of US and European cancer patients ultimately leading to a concept for an international biobank network interface. Our aim is to provide instantly clinical data such as diagnostic and treatment approaches in real time that can be used by physicians and medical scientists from all over the world.

A population-based study of cardiovascular disease mortality risk in US cancer patients.

This observational study characterized cardiovascular disease (CVD) mortality risk for multiple cancer sites, with respect to the following: (i) continuous calendar year, (ii) age at diagnosis, and (iii) follow-up time after diagnosis. The Surveillance, Epidemiology, and End Results program was used to compare the US general population to 3 234 256 US cancer survivors (1973-2012). Standardized mortality ratios (SMRs) were calculated using coded cause of death from CVDs (heart disease, hypertension, cerebrovascular disease, atherosclerosis, and aortic aneurysm/dissection). Analyses were adjusted by age, race, and sex. Among 28 cancer types, 1 228 328 patients (38.0%) died from cancer and 365 689 patients (11.3%) died from CVDs. Among CVDs, 76.3% of deaths were due to heart disease. In eight cancer sites, CVD mortality risk surpassed index-cancer mortality risk in at least one calendar year. Cardiovascular disease mortality risk was highest in survivors diagnosed at <35 years of age. Further, CVD mortality risk is highest (SMR 3.93, 95% confidence interval 3.89-3.97) within the first year after cancer diagnosis, and CVD mortality risk remains elevated throughout follow-up compared to the general population. The majority of deaths from CVD occur in patients diagnosed with breast, prostate, or bladder cancer. We observed that from the point of cancer diagnosis forward into survivorship cancer patients (all sites) are at elevated risk of dying from CVDs compared to the general US population. In endometrial cancer, the first year after diagnosis poses a very high risk of dying from CVDs, supporting early involvement of cardiologists in such patients.

The crippling financial toxicity of cancer in the United States

ABSTRACTThe financial cost of cancer treatment in the United States is astronomically high and is expected to rise. The economic burden of cancer care increasingly falls on the patients. Patients thus experience “financial toxicity” of cancer care that can have catastrophic consequences on health and quality of life. Here we examine the results reported by Gilligan et al. in their study of financial toxicity in US cancer patients over 50 years old. This study provided corroborating and compelling data about the financial toxicity experienced by cancer patients. Many questions remain, however, about the consequences of financial toxicity and the full reality of cancer care economics in America.

Assessment of enrollment characteristics for Children’s Oncology Group (COG) upfront therapeutic clinical trials 2004-2015.

6564 Background: Improvements in pediatric cancer survival are attributed to cooperative clinical trials. Under representation of specific demographic groups has been described in adult and pediatric cancer and poses a threat to the generalizability of trial results. A comprehensive evaluation of data provided by the Children’s Oncology Group (COG) of upfront trial enrollment for US patients 0 to 29 years old between 2004 and 2015 was performed to assess for disparities in participation. Methods: Estimates of cancer cases were calculated using the Surveillance, Epidemiology, and End Results registry and the US Census and compared to observed COG cases. Percent enrollment and Standardized Ratios of enrollment were calculated across various demographic, disease, and socioeconomic groups. The COG website was utilized to quantify available upfront trials during the study period and assess age eligibility criteria. Results: 21.3% of estimated US cancer patients age 0 to 19 years enrolled on COG trials. Younger patients were consistently more represented across disease types and race/ethnicities. Patients with hematologic malignancies were more represented compared to solid and central nervous system (CNS) tumors. Conclusions: COG clinical trial enrollment rates are declining, which may be due to challenges in pediatric drug development, difficulty designing feasible trials for highly curable diseases, and issues in ensuring trial availability for the heterogeneous group of solid and CNS tumors. Though racial/ethnic groups and county-level socioeconomic factors were proportionally represented, under representation of the adolescent/young adult (AYA) population and younger patients with solid and CNS tumors remain significant concerns. Targeted enrollment efforts should focus on the identified subgroups and further research should evaluate AYA enrollment across all available trials to provide continued treatment advances for all patients.

Metastasis pattern and prognosis of male breast cancer patients in US: a population-based study from SEER database.

Background:The aims of this study were to analyze the metastasis pattern and prognosis of male breast cancer (MBC) and compare it with female breast cancer (FBC), and to determine the independent factors affecting the prognosis of MBC patients.Methods:Metastatic MBC diagnosed in the Surveillance, Epidemiology and End results (SEER) database from 2010 to 2015 were selected. Chi-squared test was used to compare clinicopathological characteristics. Survival differences were compared by Kaplan–Meier analysis. Cox proportional hazard model was used to determine the prognostic factors affecting overall survival.Results:A total of 2754 MBC patients were identified, of which 196 had distant metastasis. Compared with nonmetastatic MBC, metastatic MBC patients had a higher proportion of <60 years old and grade III–IV, and were more likely to receive chemotherapy and radiotherapy, while the proportion of surgery, central portion of the breast, and Her2–/HR+ was lower. Compared with metastatic FBC, metastatic MBC patients had a higher proportion of ⩾60 years old, central portion of the breast, surgery, simultaneous bone and lung metastasis, while the proportion of Her2+/HR–, triple negative, liver metastasis only, and simultaneous bone and liver metastasis was lower. MBC patients with lung alone, bone alone, and simultaneous lung and bone metastasis had a higher hazard ratio (2.41; 3.06; 2.52; p < 0.0001) compared with nonmetastatic patients.Conclusions:Compared with nonmetastatic MBC patients, metastatic MBC patients had unique clinicopathological features, and were also different from metastatic FBC patients. However, there was no difference in prognosis between metastatic MBC and FBC patients. Distant metastasis was an independent risk factor for the prognosis of MBC patients.

Patient-Reported Outcomes in Systemic Light-Chain Amyloidosis

Background: Patients with systemic light-chain (AL) amyloidosis have a high symptom burden which can negatively impact their health-related quality of life (HRQoL). However, there is a lack of data on patient-reported outcomes (PROs) in this population in the current era of effective plasma cell directed therapies. The primary objectives of our study were to assess baseline HRQoL using FACT-G (Functional Assessment of Cancer Therapy-General) and PROMIS-GH (Patient Reported Outcomes Measurement Information System-Global Health), evaluate the degree of correlation between FACT-G and PROMIS-GH, and compare HRQoL by hematologic response to first-line therapy in systemic AL amyloidosis.Method: This was an observational study using the Cleveland Clinic Knowledge Program database, which contains HRQoL data captured at outpatient visits. FACT-G was administered every 90 days since its implementation on September 2012. PROMIS-GH was implemented in October 2015, initially administered every 30 days and subsequently every 90 days since July 2017.Results: A total of 81 patients with systemic AL amyloidosis diagnosed between September 2012 and December 2017 and ≥1 HRQoL measurement were included in our analysis. The median age at diagnosis was 64 years. Cardiac involvement at diagnosis was present in 49% of patients. The most common induction therapy was Bortezomib-Cyclophosphamide-Dexamethasone (86%). Autologous stem cell transplantation was performed in 38% of patients.Data on HRQoL at baseline (≤2 months from diagnosis) using FACT-G was available for 43 patients. The mean FACT-G total score at baseline was 74 (standard deviation [S.D.] ±15). In comparison, the mean FACT-G total score for general US population is 80.1 (±18.1) and that of US cancer patients is 79.3 (±17.0) [Normative data from Pearman et al; Cancer. 2014]. Maximal HRQoL deficit was seen in the functional well-being (FWB) domain of FACT-G, with the mean score being >0.5 S.D. below that of the general population (Table 1). Data on HRQoL at baseline using PROMIS-GH was available for 18 patients. There was a significant deficit in global physical health (GPH) compared to the general population, with a mean T-score of 37.7 (±7.8) [Mean T-score in general US population being 50±10]. The mean T-score for global mental health (GMH) was 44.4 (±6.7).A total of 72 patients had 128 outpatient visits where FACT-G and PROMIS-GH were captured concurrently (range, 1-4 visits per patient). Using Cohen's criterion, GPH domain of PROMIS-GH had a large and statistically significant correlation with FACT-G total (r=0.66), physical well being (PWB) (r=0.77) and FWB (r=0.66) scores. PROMIS GMH domain also had a strong and statistically significant correlation with FACT-G total (r=0.73), PWB (r=0.60), emotional well-being (EWB) (r=0.64) and FWB (r=0.73) scores. The scatterplots for correlation between PROMIS-GH domains and FACT-G total score is shown in Figure I.At follow-up, a total of 50 patients had data on best hematologic response and HRQoL assessment. Patients achieving a complete response (CR) to first-line therapy had a significantly superior FACT-G score at all domains compared to those with less than CR or no response. Maximal benefit in complete responders was noted in the FWB domain of FACT-G (Mean score ≥2 S.D. higher than patients with less than CR or no response; P=0.002).Conclusion: Patients with AL amyloidosis have a worse HRQoL at diagnosis compared to US cancer and general adult population. Deficit in HRQoL was most prominent in the FWB domain of FACT-G and GPH domain of PROMIS-GH. Most domains of FACT-G and PROMIS-GH had strong and significant correlation. Patients achieving a CR to first-line therapy had significantly superior HRQoL at all FACT-G domains. Clinical trials in AL amyloidosis should include patient-reported HRQoL as a key endpoint and focus on the domains of physical health and functioning to assess meaningful benefit of novel therapies. Psychometric validation of FACT-G and PROMIS-GH in AL amyloidosis would be helpful in generating robust PRO data in future studies. DisclosuresMajhail:Anthem, Inc.: Consultancy; Incyte: Honoraria; Atara: Honoraria.

Patients v. Myriad or the GDPR Access Right v. the EU Database Right.

In 2016, four US cancer patients legally challenged Myriad by claiming full access to all genomic information produced in the course of Myriad's testing of their risks for a variety of cancers. Asserting that Myriad's refusal to provide them with this information violated the HIPAA Privacy Rule, the patients sought a determination of a right to access all their genetic information from testing laboratories. Such access would not only serve their own care, but also enable them to share their genetic data with the scientific community which they alleged Myriad failed to do. A similar case may be brought in Europe under the novel EU GDPR. Specifically, it would put the GDPR right of access to personal data against Myriad's database right under the EU Database Right Directive. The outcome of this case could impact the fate of personalized medicine, which depends on the one hand on patients' having control over their genetic data, and on the other hand on incentives for genetic testing companies to generate these data. We first address the issue of whether the GDPR applies to medical records. We then analyse how GDPR rights could play out in the context of clinical genetic testing and conclude that the GDPR access right stops short of granting unconditional access to all datagenerated in the process of testing, to the extent that its exercise would result in the violation of medical-professional norms, expose the testing company to potential liability, or compromise normal exploitation of the database of which the personal data form part.

Validation of a software-based clinical trial matching platform for oncology using comprehensive clinical information.

e18589Background: Despite the critical need for cancer patients to participate in clinical trials, only 3% of US cancer patients actually do so, and 40-60% of oncology clinical trials fail to recru...