Science and Health Policy最新文献

{"title":"Abstract 2629: Young onset colorectal cancer patients, survivors and caregivers: self-report clinical, psychosocial, financial and quality of life outcomes","authors":"K. Newcomer, R. Yarden, Laura H. Porter","doi":"10.1158/1538-7445.AM2021-2629","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2629","url":null,"abstract":"Background: Colorectal cancer is the third-most commonly diagnosed cancer and the second-leading cause of cancer death in men and women combined in the United States. Young-onset colorectal cancer refers to individuals diagnosed under the age of 50. In recent years, the incidence has increased by 2.2% annually in individuals younger than 50 years, and 1% in individuals 50-64, in contrast to a 3.3% decrease in adults 65 years and older. Young-onset (YO) CRC patients and caregivers face unique clinical challenges including fear and stress around the disruption of family and career developmental tasks and goals suggesting a need for additional psychosocial and financial support. Methods: A cross-sectional study, conducted in the form of an online survey, was launched to better understand the experiences of YO-CRC patients and caregivers. YO-CRC patients and survivors (N=885) and caregivers (N=204) completed an online questionnaire that was based on established instruments including PROMIS, EORTC-QOL-30, and EORTC-CR-29. The final survey instrument and study plan were reviewed and approved by the Aspire Inc. Institutional Review Board. Results: Nearly 75% of patients/survivors shared that they have been concerned about their mental health and 64% responded that they have needed help for their depression. Further, 67% of caregivers surveyed responded that they were also concerned about their own mental health and 68% responded that they needed help with their depression. Emotional exhaustion was reported by 77% of caregivers, whether they were providing round-the-clock care or caregiving from a distance. Emotional exhaustion was more pronounced in the patient/survivor cohort, with 95% indicating that emotional exhaustion impacted their lives. As a result of psychosocial distress, 71% of caregivers and 29% of patients/survivors indicated that they had withdrawn from other people. YO-CRC diagnosis changes what the patient/survivor can contribute to the family, both physically and emotionally, resulting in the caregiver having a change in their previous responsibilities. Of our respondents, 48% of caregivers indicated that their role in childcare changed; in addition, changes occurred in household responsibilities (77%), sexual/intimacy (51%), work (59%), and financial responsibility (42%). Conclusions: These survey results indicate a need for the YO-CRC community to have access to resources to address unique issues. The physical and emotional stress on patients, survivors and caregivers is frequently not discussed which indicates a knowledge gap, not only in the lay population but also within the medical community. The Alliance is dedicated to working with all stakeholders, including policymakers, to address the unmet needs among caregivers, aiming to improve quality of life outcomes for caregivers in conjunction with their patients. Citation Format: Kimberly Lynn Newcomer, Ronit Yarden, Laura Porter. Young onset colorectal cancer patients, survivors an","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"89 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"84409898","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2628: Racial differences in the impact of socioeconomic status on cancer-specific survival in multiple myeloma","authors":"Huiwen Deng, A. Zolekar, Hsiao-Ching Huang, M. Smart, C. Hubbard, B. Chiu, P. Patel, K. Sweiss, G. Calip","doi":"10.1158/1538-7445.AM2021-2628","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2628","url":null,"abstract":"Background: Multiple myeloma (MM) incidence and outcomes differ across racial/ethnic groups in the United States. Interactions between socioeconomic status (SES) with ethnicity in MM incidence and survival outcomes are not well understood. Our objective was to evaluate disparities in cancer-specific survival of patients diagnosed with MM by race/ethnicity. Methods: We conducted a population-based retrospective cohort study of patients ages 20+ years diagnosed with MM between 2000 and 2015 using Surveillance, Epidemiology and End Results, Census Tract-level SES and Rurality Database. SES was defined using the National Cancer Institute9s time-dependent composite score developed by Yost et al. (2001). Yost index quintiles were where the 1st and 5th quintiles representing the lowest and highest SES categories respectively. Cumulative incidence functions were used to analyze cancer-specific survival across strata racial/ethnic and SES and equality of functions was determined using Gray9s test. Subdistribution hazard ratios (SHR) and 95% confidence intervals (CI) were calculated using the Fine and Gray regression models adjusted for age, sex, year of diagnosis, marital status, insurance status, and treatment with chemotherapy. Race-specific risk estimates were stratified by age ( Results: Overall, 58,095 MM patients were included in our analysis among whom 63.0% were non-Hispanic White, 19.5% were Black, 0.3% were American Indian/Alaskan Native, 5.8% were Asian/Pacific Islander and 11.4% were Hispanic. Compared to White MM patients (median age 69 years), Black (64 years), American Indian/Alaskan Native (64 years), Asian/Pacific Islander (67 years) and Hispanic (64 years) patients were younger on average. A higher proportion of Black (42.8%) and Hispanic (27.9%) MM patients were in the lowest SES quintile compared to White (10.6%), American Indian/Alaskan Native (15.2%), and Asian/Pacific Islander (8.9%) MM patients. Cumulative incidence functions for cancer-specific survival were significantly different across SES quintiles (P Conclusion: Low SES level is independently associated with poor MM-specific survival. However, the impacts of SES on MM-specific survival differ by race/ethnicity and age with the greatest increased risk observed in younger Black, Hispanic, and White patients. Citation Format: Huiwen Deng, Ashwini Zolekar, Hsiao-Ching Huang, Mary H. Smart, Colin C. Hubbard, Brian C. Chiu, Pritesh R. Patel, Karen Sweiss, Gregory S. Calip. Racial differences in the impact of socioeconomic status on cancer-specific survival in multiple myeloma [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;81(13_Suppl):Abstract nr 2628.","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"4 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"87509533","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2625: 3Din vitroprostate cancer PDX resource for studying cancer health disparities","authors":"Peter D A Shepherd, Andrei Bonteanu, Stanley E Hooker, K. Bircsak, D. Iyer, D. Dexter, D. Harrington, R. Kittles, N. Navone","doi":"10.1158/1538-7445.AM2021-2625","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2625","url":null,"abstract":"Prostate cancer (PCa) incidence and mortality is nearly twice that in Black men than any other race (Non-Hispanic White, Asian/Pacific Islander, American Indian/Alaska Native, Hispanic (any race)). Characterization of the underlying biological factors that contribute to this cancer health disparity (CHD) is required to close the gap and improve patient outcome for Black men. In vitro PCa research tools which reflect the racial/ethnic diversity of this patient population are urgently needed, as most PCa cell lines are of Non-Hispanic White-origin. PCa patient-derived xenografts (PDXs) retain many of the characteristics of patient tumors and can be isolated from specific racial/ethnic groups to address this concern. Rodent PDX models provide a valuable resource for studying human cancer, however recent trends in reducing animal usage have instituted a search for alternative methods for studying PDXs that also maintain their complexity. While 2D in vitro culture of PCa PDXs has been largely unsuccessful, recent evidence suggests 3D in vitro culture of PCa PDXs may enable better long-term culture of the tumor cells. Here, we present a racially/ethnically diverse PCa PDX library of specimens (Black, Non-Hispanic White, Hispanic) developed at MD Anderson Cancer Center (the MDA PCa PDX series) compatible with 3D in vitro culture. In order to confirm self-reported race/ethnicity, each PCa PDX was characterized by whole-exome sequencing and compared to reference populations for a genetic ancestry estimation. For 3D in vitro culture, we utilized a high throughput microfluidic culture platform with 96 chips, the MIMETAS OrganoPlate® 2-lane. This platform suits both 3D tissue/cell model development and throughput needs required for drug discovery. MDA-PCa PDX cell clusters were suspended in hyaluronic acid-based hydrogel solutions, seeded into the OrganoPlate, and cultured under continuous perfusion. Genetic ancestry estimation studies revealed that patient-reported race/ethnicity often aligned with the same racial/ethnic population genetic signatures. For example, two self-reported Black PDXs were primarily of West African origin (74.6-84.6%). When cultured in 3D, PCa PDX cultures were stable and viable for at least 7 days, as determined by high content fluorescence imagining coupled with cell viability dyes. By immunofluorescent staining, PCa PDX cultures exhibited appropriate expression of phenotypic prostate-specific antigen (PSA) and androgen receptor (AR) which was maintained over the life of the culture. Studies are ongoing to screen a panel of chemotherapy drugs and determine the predictivity of the platform. This well characterized racially/ethnically diverse PCa PDX library, together with the 3D in vitro platform and methods is a valuable resource for evaluating population-based tumor response. Citation Format: Peter Shepherd, Andrei Bonteanu, Stanley Hooker, Kristin Bircsak, Divya Iyer, Dwayne Dexter, Daniel A. Harrington, Rick Kittles, Nora M. Na","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"41 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"77662794","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2627: Socioeconomic status and utilization of major surgical procedures in the United States, Canada, and Australia","authors":"Hilary Pang, K. Chalmers, B. Landon, A. Elshaug, J. Matelski, V. Ling, M. Krzyzanowska, G. Kulkarni, B. Erickson, P. Cram","doi":"10.1158/1538-7445.AM2021-2627","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2627","url":null,"abstract":"Purpose: This study aimed to compare the utilization rates of three organ resection surgeries, predominantly indicated for the treatment of cancer, in the US, Canada, and Australia, and compare rates between residents of lower- and higher-income neighborhoods. Methods: We used population-based administrative data to identify all adults aged ≥18 years hospitalized for pancreatectomy (PX), radical prostatectomy (RP) and nephrectomy (NX) between 2011-2016 (New York, USA), 2011-2018 (Ontario, Canada), and 2013-2018 (New South Wales, Australia). We linked each patient9s zip-code of residence to 2016 census data to ascertain neighborhood income. We compared utilization rates for each procedure in each jurisdiction in aggregate and by neighborhood income quintile. Primary outcomes were: 1) each jurisdictions9 per-capita overall, age-, and sex-standardized utilization rates for each procedure; and 2) utilization rates amongst residents of lower- and higher-income neighborhoods. Results: Sociodemographics were similar across jurisdictions; patients in New South Wales were older for all procedures. New York hospitals were significantly likelier to perform each of the three procedures compared to Ontario and New South Wales (all P Conclusions: Utilization rates of PX, RP, and NX were significantly higher in New York and New South Wales than in Ontario. Rich-poor surgical utilization differences were significantly larger in New York and New South Wales and significantly smaller in Ontario. These findings suggest that income-based disparities are larger in US and Australian jurisdictions than those Canadian, and highlight the possible trade-offs of equity and access in cancer care of different countries. Citation Format: Hilary Pang, Kelsey Chalmers, Bruce Landon, Adam Elshaug, John Matelski, Vicki Ling, Monika K. Krzyzanowska, Girish Kulkarni, Bradley A. Erickson, Peter Cram. Socioeconomic status and utilization of major surgical procedures in the United States, Canada, and Australia [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;81(13_Suppl):Abstract nr 2627.","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"34 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"76524797","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2632: Fostering engagement in biobanking and research through the NCI Cancer Moonshot Biobank patient and provider engagement website","authors":"Esmeralda Casas-Silva, L. Agrawal, H. Ellis, Veena Gopalakrishnan, P. Guan, M. Jensen, Natalie Madero, S. McDermott, J. McLean, Abhi Rao, James Suh, J. Wanyiri, C. Weil, Mickey Williams, Helen M. Moore","doi":"10.1158/1538-7445.AM2021-2632","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2632","url":null,"abstract":"The National Cancer Institute9s (NCI) Cancer Moonshot Biobank (the Biobank) aims to accelerate cancer research on treatment resistance and sensitivity through collection of longitudinal biospecimens and health data donated by participants diagnosed with late-stage cancer and receiving standard of care therapy at participating NCI Community Oncology Research Program (NCORP) sites. Participant and provider engagement are a central tenet of the Biobank. We therefore created an innovative website to interface directly with participants and providers and to meet our engagement goals of returning value to stakeholders, operating with transparency, maintaining bi-directional communication, and enrolling a diverse population that represents the U.S. including racial/ethnic minorities, rural populations and others that are historically underrepresented in clinical research. The Biobank engagement website has several features designed to provide return of value to stakeholders. This includes clear information about the program, transparency about how biospecimens and data will be used in research, and the ability to access and download documents such as clinical biomarker tests and signed consent forms. The website also includes project updates, participant and provider resources, and educational material. The Cancer Moonshot Biobank Participant and Provider Engagement website serves as an important interface between the Biobank and its stakeholders. Its ultimate aim is to facilitate project engagement and return value to participants and providers. Citation Format: Esmeralda Casas-Silva, Lokesh Agrawal, Helena J. Ellis, Veena Gopalakrishnan, Ping Guan, Mark Jensen, Natalie Madero, Sean McDermott, Jeffrey McLean, Abhi Rao, James Suh, Jane Wanyiri, Carol J. Weil, Mickey Williams, Helen M. Moore. Fostering engagement in biobanking and research through the NCI Cancer Moonshot Biobank patient and provider engagement website [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;81(13_Suppl):Abstract nr 2632.","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"17 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"86899603","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2633: Advancing childhood cancer research through young investigator and advocate collaboration","authors":"Amber K Weiner, G. Lindberg, M. Moll, Antonia Palmer, Kevin M Reidy, S. Diskin, C. Mackall, J. Maris, P. Sullivan","doi":"10.1158/1538-7445.AM2021-2633","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2633","url":null,"abstract":"","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"10 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"88590584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2634: French health utilities for patients with glioblastoma using TTFields","authors":"G. Chavez, C. Proescholdt","doi":"10.1158/1538-7445.AM2021-2634","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2634","url":null,"abstract":"Background: There is little real-world data available on Quality of Life (QoL) and health state utilities for patients with glioblastoma (GBM). The only standard utilities available were elicited from healthy panel members of the UK National Health System (Garside et al. 2007) rather than from actual patients with GBM. There are no utilities whatsoever for patients with GBM using Tumor Treating Fields (TTFields) therapy. We sought to provide the first health state utilities directly estimated from patients with GBM using TTFields. Methods: We issued the EuroQol 5-Dimension (EQ-5D) survey to nearly 3,000 patients with GBM in the US and Europe who use TTFields treatment. Patient responses were completely anonymous and there was no other patient selection criteria. The EQ-5D is a validated and widely used tool for the evaluation of health related QoL and the estimation of health state utilities. We used a EuroQol-endorsed statistical model (Andrade et al. 2020) to estimate health state utilities from EQ-5D responses. This gives French tariffs i.e. utility values for application in French health economic evaluation. We sent an additional survey to collect data on age, disease progression status, time since diagnosis, treatment details, and other information. Results: We report interim analysis on 906 survey responses, representing a response rate of 32%. This was notably higher than our target response rate of 20%. 853 of these EQ-5D responses were sufficiently complete to estimate utilities and 754 had complete information on age, progression status, and time from diagnosis. The overall sample median/mean utility estimates were .913/.836. The median/mean utility estimates for patients who had not experienced disease progression were .929/.882, while those for patients who had experienced disease progression were .855/.748. Of the patients who were non-progressed, 112 or 20.7% reported no problems on their EQ-5D survey, resulting in a utility estimate of 1, the highest possible value, indicating very good or fully satisfactory QoL. Of those patients whose disease had progressed, 22 or 9% similarly reported no problems. For non-progressed patients, older age was significantly negatively correlated with utility estimates, while for progressed patients this correlation was not significant. More notably, among patients who had experienced disease progression, longer time from diagnosis had a highly significant, positive effect on utility estimates. Conclusion: High median utility estimates for both progressed and non-progressed patients, along with the percentage of patients reporting no problems, indicates that many patients with GBM using TTFields can expect a good Quality of Life. Additionally, in contrast to prevailing expectations and modeling assumptions, interim results indicate that utility and QoL of patients with GBM who have experienced disease progression is likely to increase with time. Citation Format: Gordon V. Chavez, Christina Proeschold","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"18 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73227285","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2631: Moving beyond population averages: A patient-centered research agenda advancing personalized medicine","authors":"C. Bens, David L. Davenport","doi":"10.1158/1538-7445.AM2021-2631","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2631","url":null,"abstract":"Personalized medicine presents opportunities to understand how differences in an individual9s biology affect their health so that prevention strategies and treatments are guided to those patients who will benefit. However, there are fundamental gaps in awareness and access that impact the speed at which personalized medicine products and services are integrated into health care. Through a two-year Engagement Award from the Patient-Centered Outcomes Research Institute (PCORI), the Personalized Medicine Coalition (PMC) convened more than 120 patients and other health care stakeholders with interest and expertise in various disease areas, including various cancers, to develop a research agenda advancing personalized medicine built on principles defined by patients. The research agenda defines 45 research questions for improving the delivery of personalized medicine. The project consisted of a series of four web forums to identify patient priorities, an online collaboration platform to brainstorm related research questions, a virtual roundtable with patients and other stakeholders to finalize research questions, and an advisory committee consisting largely of patient representatives to guide the development of the research agenda. Patients, patient advocates, caregivers, patient advocacy organization representatives, health care professionals, researchers, and other stakeholders representing diverse backgrounds, disease areas, health needs, and with varying levels of experience with personalized medicine participated. Participants focused largely on their desire for research that could improve the quality and quantity of interactions between patients and providers of health care services, with an emphasis on the importance of education and access. Takeaways from these discussions led to the development of 45 research questions addressing concerns related to: 1. Patient-provider communication 2. Patient education 3. Caregiver, pediatric, and family considerations 4. Provider education, resources, and collaboration 5. Access, affordability, and utilization 6. Coverage and reimbursement 7. Clinical trials 8. New technologies and data management 9. Outcomes research. This research agenda should inform future studies that will provide patients, caregivers, and health care professionals with the information they need to make more informed health care decisions and, ultimately, improve the delivery of personalized medicine to patients in ways most meaningful to them. PCORI, public-private partnerships, private foundations, and other entities interested in ensuring that all patients receive personalized medicine should fund these studies. Citation Format: Cynthia A. Bens, David L. Davenport. Moving beyond population averages: A patient-centered research agenda advancing personalized medicine [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;8","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"39 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89661937","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2626: The effectiveness of healthcare professional training on the legal issues impacting individuals diagnosed with cancer to improve patient quality of life","authors":"Joanna Morales, Monica Bryant, M. Ward","doi":"10.1158/1538-7445.am2021-2626","DOIUrl":"https://doi.org/10.1158/1538-7445.am2021-2626","url":null,"abstract":"","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"55 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91160793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Abstract 2630: Meta-analysis of pazopanib and trabectedin in 2L+ metastatic synovial sarcoma (2L+ mSS)","authors":"C. Carroll, Nashita Patel, N. Gunsoy, D. Parks, H. Stirnadel-Farrant, S. Pokras","doi":"10.1158/1538-7445.AM2021-2630","DOIUrl":"https://doi.org/10.1158/1538-7445.AM2021-2630","url":null,"abstract":"Background: Pazopanib (P) and trabectedin (T) are approved targeted treatments for metastatic soft tissue sarcoma (mSTS). Our objective was to evaluate the efficacy (from clinical trials [CT]) and effectiveness (from real-world studies [RW]) of P and T in second line (2L+) mSS. Methods: This meta-analysis included all English language studies assessing P or T efficacy/effectiveness in patients with 2L+ mSS, 1999 onwards, identified by a systematic literature review. Outcomes were overall response rate (ORR) representing the proportion of patients who have achieved CR or PR during the study and median overall survival (mOS) in months (mos) from start of study medication. Proportions from studies were pooled using generalized linear mixed models to account for small sample sizes, zero events and random site effects. Medians were combined using the weighted median of the medians method with 95% confidence intervals from an inverted sign test. Results: 16 studies with 405 patients with 2L+ mSS met the inclusion criteria. Response was measured variably across studies (CT: RECIST v1.0, v1.1; RW: modified RECIST or clinician assessment). ORR estimates were higher in RW than CTs (Table). OS results were consistent across CTs and RW (Table). Conclusions: This is the first study to provide estimates for patients with mSS in the 2L+ setting, distinct from mSTS and pool outcomes of targeted therapies. RW estimates of ORR for both treatments were higher than those observed in trials likely due to variable definition of response and in underlying prognostic factors. mOS was consistent across both types of studies and was similar to mOS seen on placebo (10.7 mos in PALETTE study) suggesting a high unmet need in this population. Funding: GSK (213368). Citation Format: Charlotte Carroll, Nashita Patel, Necdet B. Gunsoy, Dan Parks, Heide A. Stirnadel-Farrant, Shibani Pokras. Meta-analysis of pazopanib and trabectedin in 2L+ metastatic synovial sarcoma (2L+ mSS) [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2021; 2021 Apr 10-15 and May 17-21. Philadelphia (PA): AACR; Cancer Res 2021;81(13_Suppl):Abstract nr 2630.","PeriodicalId":21579,"journal":{"name":"Science and Health Policy","volume":"2013 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"88174248","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}