bluuebunny/crossref_metadata_2025 huggingface dataset:
Insights from C-CAT repository: A novel approach to identifying appropriate patient populations for anticancer drug development using the clinicogenomic nationwide database in Japan.
Authors: Kitadai Rui , Koyama Takafumi , Okuma Yusuke , Shibata Taro , Kohno Takashi
Published: June 2024

11159

Background: The integration of Comprehensive Genomic Profiling (CGP) with real-world data (RWD) provides crucial insights for anticancer drug development to achieve precision oncology, therefore, the current global trend to construct as the national projects, yet its clinical impact remains underexplored. The therapeutic efficacy of each line of anti-cancer drug in various cancer types and the relationship between genetic alterations and the effect of anticancer drugs is needed to guide the direction of future drug development promptly and precisely. Methods: A comprehensive analysis was conducted on data from Japan's Center for Cancer Genomics and Advanced Therapeutics (C-CAT) repository between June 2019 and December 2023. Five genomic profiling assays for CGP testing were reimbursed by nation-wide insurance in Japan. Utilizing the clinicogenomic repository of C-CAT, registered data of therapeutic efficacy of anticancer drug including cytotoxic agents, molecular-targeted agents, and immune checkpoint inhibitors were evaluated in association with genetic alterations. Data on anticancer drug of patients (pts) with recurrent or metastatic disease were included in the analysis. Results: The C-CAT registry data encompassed 60,256 pts across 32 tumor organ sites, classified ontology by the OncoTree over 4.5 years. Of these patients, 30,234 were male, and 30,017 were female, with 5 classified as unknown. In terms of age, 31,919 pts were < 65. The most registered origins were colorectal (n=10,110), pancreas (n=8,069), biliary tract (n=5,030), breast (n=3,744), esophagus/stomach (n=3,734), prostate (n=3,701), ovarian/fallopian tube (n=3,521), lung (n=3,427), and soft tissue (n=2,568). TP53mutations (58.3%, n=35,102) were most prevalent, followed by KRAS (25.8%, n=15,521), APC (19.9%, n=11,919), NOTCH3 (14.1%, n=8,502), and PIK3CA (13.0%, n=7,861). The frequency of BRCA1/2mutation was 4.1% (n=2,479). Overall response rate (ORR) for the first-, second-, third-, and fourth-lines treatment were 35.7%, 23.3%, 19.4%, and 19.2%, respectively. Pts treated with cisplatin or carboplatin in the first-line treatment, who had TP53 mutations, showed a better ORR than those without (44.0% vs. 38.9%, p < 0.001). Similarly, pts with BRCA1/2mutation showed a better ORR than those without (53.9% vs. 41.4%, p < 0.001) in the first-line treatment. Conclusions: This study was the first to report the relationship between genetic alterations and the therapeutic effect of anticancer drugs in each line. This approach based on big data analysis potentially accelerates drug development in the appropriate patient population by identifying significant associations between specific genetic mutations and improved outcomes with certain chemotherapy regimens in various cancers.
Census tract–level social determinants of health and variability in stage at diagnosis of bladder, breast, and non-small cell lung cancers.
Authors: Kaplan henry G , Scannell Bryan Molly , Hu Xiaohan , Mohammed Hina , Izano Monika A. , Brown Thomas D. , Simon George R. , Karam Jose A. , Berry Anna B.
Published: June 2024

11170

Background: Social determinants of health (SDOH) can identify underserved populations, inform the patient journey, and identify opportunities to improve outcomes. In cancer, SDOH may influence stage at diagnosis through exposure to risk factors for aggressive cancer and decreased access to care. However, privacy restrictions can limit research with SDOH in real world datasets, which often only know a patient’s broad location. Methods: Geocoding was undertaken on addresses of patients diagnosed between 1/1/17 and 9/30/22 with bladder (n=6078), breast (n=28,958), and non-small cell lung cancer (NSCLC, n=14,957) in the Syapse Learning Health Network of U.S. community health systems. Location was linked to five indicators of SDOH at the census tract level: the Social Vulnerability Index (SVI); percent (%) of tract spending >30% of income on housing; % of tract with broadband internet; county designation as a primary care shortage area; and rural urban commuting area (RUCA). Nested multivariable ordinal logistic regression models estimated independent associations between demographic, clinical, and SDOH factors with stage at initial cancer diagnosis. The statistical significance of including SDOH variables was assessed using a likelihood ratio test (LRT) comparing the models before and after including SDOH. Results: After successful geocoding and linkage to census tracts, there were marginal differences in the distribution of the five SDOH across stages at diagnosis within the three tumor types. In multivariable models adjusted for year of diagnosis, age, sex, race, ethnicity, smoking, and primary payor, among the tested SDOH measures, county designated as primary care shortage (proportional odds ratios (OR): 1.31, 95% confidence interval (95% CI): 1.05-1.64), and non-metropolitan area (0.69, 0.50-0.94) were statistically significantly associated with stage at bladder cancer diagnosis. SVI (1.52, 1.31-1.76) was also statistically significantly associated and directly proportional with stage at breast cancer diagnosis. Among patients with NSCLC, broadband internet access (1.01, 1.00-1.01), primary care shortage (1.15, 1.03-1.27) and non-metropolitan area (0.85, 0.74-0.99) were statistically associated with stage at diagnosis. In all three tumors, LRTs found that models with SDOH were statistically significantly better at predicting stage at diagnosis than those without (p <0.001). Conclusions: Using granular, census-tract geospatial resolution for geocoding of SDOH, we find that patients who live in greater vulnerability are diagnosed at later stages than those who live in areas of less vulnerability, suggesting that consideration of SDOH variables is important in assessing stage at diagnosis and identify patients with an unmet need for outreach and screening.
Primary localized malignant PEComa treated with surgery: A report of a large series of patients treated in a referral institution.
Authors: Sanfilippo Roberta , Miceli Rosalba , Fabbroni Chiara , Fiore Marco , Colombo Chiara , Radaelli Stefano , Callegaro Dario , Morosi Carlo , Fumagalli Elena , Collini Paola , Casali Paolo Giovanni , Gronchi Alessandro
Published: June 2024

11582

Background: Perivascular Epithelioid Cell Tumors (PEComa) are mesenchymal neoplasms with a characteristic myomelanocytic immunophenotype. These tumors exhibit a broad anatomic distribution, and aggressive clinical behavior has been correlated with histological features such as tumor size and increasing mitotic activity. PEComas arising from the kidney are referred to as angiomyolipomas and usually lack metastatic potential, whereas epithelioid angiomyolipomas do have metastatic potential. Until now, only case reports or a limited number of patients have been documented in the literature, resulting in a lack of data regarding their clinical behavior. In this study, we present the outcomes of a large series of patients (pts) with primary localized malignant PEComa. Methods: This is a retrospective study involving all consecutive pts of any age affected by primary and localized malignant Pecoma surgically treated with curative intent at our institution from January 2000 to December 2023. Eligible pts had a pathologically confirmed diagnosis of malignant Pecoma. We present the outcome results in terms of Disease-Free Survival (that includes Local Recurrence (LR), Distant Metastases (DM), or death as first events) and Overall Survival (OS). Due to the low number of events only univariable analyses were performed. Results: 48 patients (19/48 M; 29/48 F) were identified. Median age at surgery was 50 yrs (range 18-80). Primary site was extremity and trunk in 19/48 pts, retroperitoneum in 13/48 pts and other in 16/48; median size of tumor was 11 cm (range 1-30 cm). At a m-FU of 57 mos (IQR:22-116), 8 pts died. Five- and 10-yr OS was 81.6% [95% Confidence Interval (CI): 70.1-94.9%)] and 77.3% (CI: 64.2-93.0%), respectively. One patient developed LR as first event, 9 developed DM and 3 died without any LR and DM; 3 and 1 patients developed DM as 2nd and 3rd event, respectively. Five- and 10-yr DFS was 69.4% [CI: 56.3-85.6%] and 65.1% (CI: 50.9-83.1%). Predictors of higher risk of DFS were age (>64 yrs old) and site of origin [p=0.5; HR trunk and arms: 1.2 (CI: 0.38-3.81) vs. retroperitoneum and 3.49 (CI: 0.43-28.37) vs other]. Conclusions: In this series PEComas have a 30% risk of recurrence at 5 years. While age and the site of origin are associated with the natural history of the disease, better pathologic criteria are needed to stratify their risk.
In-hospital outcomes and mortality of drug-induced pneumonitis and radiation pneumonitis in patients with thoracic cancer.
Authors: Lee Yi , Aldecoa Kim Abbegail Tan , Shirai Keisuke
Published: June 2024

12042

Background: Pneumonitis is one of the challenging adverse events in thoracic oncology patients undergoing cancer therapy. The cancer treatments, including targeted agents, systemic chemotherapy, immune-checkpoint inhibitors, and radiation therapy have recently advanced, however, the data on incidence of drug-induced and radiation pneumonitis (DP and RP) and their association with in-hospital outcomes is limited. Methods: Data from the National Inpatient Sample Database from 2016 to 2019 were analyzed. Adult patients with primary lung cancer were included and categorized into three groups: (1). Patients without pneumonitis, (2). Patients with DP, (3). Patients with RP. Outcomes included in-hospital mortality, intensive care unit (ICU) admission, and hospital length of stay (LOS). Data were extracted using ICD-10 codes. Results: Our analysis included 1,631,940 hospitalized primary lung cancer patients (based on a weighted sample). Among all, 4,515 patients had DP, and 16,905 patients had RP. Overall, 69.3% had smoking history, however, no significant difference between DP and RP. There was a higher prevalence of chronic obstructive pulmonary disease (COPD) in RP than DP (66.5% vs 53.2%; p<0.05) (Table). The in-hospital mortality rate was highest in DP compared to other groups. Coarsened exact matching method was used to balance covariates between non-pneumonitis and pneumonitis patients (DP and RP). Multivariate logistic regression showed that pneumonitis patients had higher odds of intubation (OR: 1.57; 95% CI: 1.24-1.99; p<0.05) and mortality (OR: 1.48; 95% CI: 1.28-1.71; p<0.05) than non-pneumonitis. Pneumonitis patients also had 23% longer length of stay than non-pneumonitis patients. We then compared the outcomes between DP and RP using the same matching algorithm, however, there was no significant difference in the outcomes between the two groups. Over the study years, the trend for DP significantly increased, whereas the RP trend was stable. Conclusions: Hospitalized pneumonitis patients had relatively poor clinical outcomes compared to patients without pneumonitis. The trend of hospitalized drug-induced pneumonitis is increasingly common. [Table: see text]
Real-world quality analysis of palliative care utilization in patients with brain metastases at three academic institutes.
Authors: Singh Rohit , Thomas Alissa A. , Kopp Emily , Amidon Ryan , Novicoff Wendy , Schuetz Samantha R , Bovi Joe A , Sarai Guneet , Iqbal Ausia N , Fadul Camilo E.
Published: June 2024

12080

Background: Brain metastases (BMETs) are a devastating complication of cancer, occurring in up to 30-50% of patients with advanced solid tumors. Median survival after diagnosis of BMETs can be limited, with 3-6 months survival for patients with poorer performance status (Updated Recursive Partition analysis [U-RPA] classification class 2b and 3). Early integration of palliative care (PC) can improve a patient’s quality of life. Per guidelines, patients with advanced cancer should be referred to an interdisciplinary team, including PC, early in the disease. We aimed to assess the utilization and impact of PC in patients with BMETs at three academic centers. Methods: We obtained data from the electronic medical record (EMR) of all patients diagnosed with BMETS at the University of Virginia, the Medical College of Wisconsin, and the University of Vermont between 1 January 2017 and 31 December 2019. Inclusion criteria were age 18+ years, diagnosis of BMETs secondary to a solid tumor malignancy (excluding leptomeningeal metastases), and >3 months of follow-up treatment. Patients were included if considered at high risk for mortality, which was defined as KPS <70 (U-RPA Class 3) or KPS 70-80 and age > 65 (U-RPA Class 2b). Chi-squared and Fisher’s exact tests compared survival, hospice referral, emergency visits, readmissions, and advance directives (AD) on the file between the PC and non-PC groups. Results: In total, of the 309 patients that were assessed, 220 patients were class 2b, and 89 were class 3 per U-RPA classification. The mean age was 66 in both groups, and 167 (54%) were seen by PC. The median survival for patients seen by PC was 7.8 months compared to 8.3 months (p 0.056). There was no statistically significant difference in survival between the two groups. 80% of patients in the group seen by PC enrolled with hospice care compared to 58% in the non-PC group (p 0.0002). 80% of patients had an AD on file in the group seen by PC compared to 56% in the non-PC group (p <0.0001). In terms of ED visits or re-hospitalization, there was a trend for more ED visits/readmissions in the group seen by PC compared to the non-PC group (p 0.057). Conclusions: In this real-world quality analysis, we found that there is under-utilization of formal PC consultation in patients with BMETs. There was no difference in survival between groups, indicating PC is not synonymous with end-of-life or hospice care. PC was associated with more patients completing ADs and more patients using hospice. Health systems and policy initiatives promoting palliative care utilization earlier in the disease course may support patient values and preferences at the end of life. [Table: see text]
Retrospective analysis of peripheral neuropathy in blood cancer survivors evaluated for cellular therapy.
Authors: Hile Elizabeth S , Xu Chao , Feierabend Desirae R , Cha Abby , Neuhold Rachel , McIntire Abby , Phi Phuong , Nipp Ryan David , Asch Adam Steven , Selby George Basil
Published: June 2024

12087

Background: Blood cancer survivors (BCS) sustain peripheral neuromotor decline from toxic therapies but also aging, diabetes and cancer. Peripheral neuropathy (PN) threatens balance and quality of life, and pre-existing nerve deficits raise future PN risk. The evaluation for cellular therapies is an opportunity to identify functional deficits and optimize their management before additional toxic exposures or disease progression. PN is often diagnosed by paresthesias, but toe strength and sensory deficits also impact balance. We aim to quantify the prevalence of these 3 PN manifestations and walking difficulty in BCS evaluated at our center for possible cellular therapies and compare BCS with and without paresthesias. Methods: We retrospectively analyzed clinical data from 5 years of physical therapy (PT) evaluations during the medical assessment for cellular therapy. As count (%) or mean ± SD we described foot paresthesias [Functional Assessment of Cancer Therapy (FACT) Item NTX2], hallux extension weakness (Medical Research Council Grade ≤4 either side), hallux vibration deficit (biothesiometer ≥24.7 V), and reported trouble walking (FACT An6). We then dichotomized the cohort by NTX2 and compared groups with Chi-square, Fisher’s exact or Wilcoxon rank sum tests. Results: 598 (87.6%) of 683 BCS evaluated by PT had FACT data (79.1% White, 5.5% Latinos); 1 in 2 BCS reported paresthesias, 71.2% as moderate-severe. See table for group comparisons. Race (p=.654) & ethnicity (p=.608) were n.s. 506 BCS with FACT (84.6%) had both strength and sensory data; we found ≥ 1 hallux deficit in 396 (78.3%), more commonly with paresthesias (86.3% vs. 69.5%, p<.001). Conclusions: At cellular therapy evaluation, 1 in 2 BCS reported foot paresthesias, and 3 in 4 had strength or sensory hallux deficit. Regardless of the cause, these deficits could contribute to the trouble walking reported by 44%. BCS with paresthesias were older and had more myeloma, diabetes, chemotherapy, hallux deficits, and trouble walking. But trouble walking and hallux deficits were also common without paresthesias. Research is needed to quantify hallux-gait relationships in BCS, and to establish the highest-value rehabilitative approaches to improve strength and walking before further toxic exposures. [Table: see text]
Efficacy and safety of dinutuximab in the management of high-risk neuroblastoma: A systematic review and meta-analysis.
Authors: Khan Malik Waleed Zeb , Ahmad Muhammad , Bin Zahid Muhammad Maaz , Khan Salman , Shehzad Dawood , Ikram Jibran , Dhar Meekoo
Published: June 2024

e22000

Background: Neuroblastoma (NB) is a malignant tumor of the sympathetic nervous system that usually occurs in children below 5 years of age. High-risk neuroblastoma (HR-NB) has a poor prognosis despite a number of treatment strategies. Dinutuximab, an anti-GD2 monoclonal antibody, has been recently added to the standard of care due to its improved prognosis. We aimed to systematically assess the outcomes of HR-NB patients treated with dinutuximab and compare them with those on other treatment regimens. Methods: A comprehensive search strategy was used to search PubMed and the Cochrane Library for articles investigating the effect of dinutuximab on the outcomes of patients with HR-NB. Eligibility criteria included: 1) Diagnosis of HR-NB based on INRG and INSS staging and MYCN status 2) Dinutuximab as the primary agent used in the intervention group 3) Mean/median follow-up time greater than 6 months. Three investigators independently reviewed and extracted relevant articles. Any disagreements were addressed through consultation with other authors. The risk of bias assessment of the selected articles was conducted using the Cochrane Risk of Bias Tool for randomized controlled trials (RCTs) and the Newcastle-Ottawa scale for observational studies. Review Manager software was used to obtain and display the meta-analysis estimates in forest plots. Random effects models were used to calculate the mean difference and overall estimated effects for continuous variables. The primary outcomes were all-cause mortality and 5-year event-free survival (5-year-EFS). Results: Five studies, including two RCTs, two secondary analyses of clinical trials, and one retrospective cohort study, comprising 1,393 participants, were included in the analysis. 686 of the patients received dinutuximab, while the remaining 707 patients were assigned to other therapies as controls. Dinutuximab was associated with lower all-cause mortality as compared to control [pooled RR, 0.41; 95% CI, 0.22-0.75, P = 0.004, I2 = 31%]. 5-year-EFS was also greater for patients treated with dinutuximab [MD: 0.12; 95% CI, 0.09-0.16; P < 0.001, I2= 98%]. Conclusions: Our findings suggest that dinutuximab is linked to a significant reduction in overall mortality and a noteworthy improvement in the 5-Year-EFS for patients with HR-NB, when compared to other treatment options.
A fragment-based algorithm for identifying pan-cancer differential methylation markers from cfDNA.
Authors: He Xiaowen , Chen Zhiyu , Li Jian , Li Chunwei , Yang Lingjian , Li Jingjing , Cui Jian , Li Hefei , Liu Wen Xin , Hou Jingjing , Long Ying , Liu Zhichao , Wang Qiumin , Guo Shuang , Guo Qiang , Lan Ping
Published: June 2024

e22507

Background: Methylation-based sequencing has been developed as a promising approach for Multi-Cancer Early Detection (MCED). Conventionally, cancer-specific methylation markers were identified by comparing paired tumor and adjacent normal tissues using traditional algorithm DSS (Dispersion shrinkage for sequencing data). However, the clinical applicability is limited by the unsatisfactory sensitivity in early-stage disease. Hence, we included blood specific markers derived from paired tissue and blood samples and developed a novel fragment-based algorithm in cfDNA with a better signal to noise ratio (SNR) than DSS to improve the sensitivity of the assay. Methods: A total of 391 plasma samples and 433 tissue samples from 7 cancer types were subjected to whole genome bisulfite sequencing (WGBS) (Table). The proportion of stage I-III and stage IV was 3.5:1 among 7 cancer types. The average sequencing depth was 30X. For each bin, the proportion of fully methylated (M) / un-methylated (U) fragments, defined with methylation level ≥85% / ≤15%, was computed individually. One-sided Wilcoxon rank sum test was employed to test whether cancer cfDNA have significantly (FDR < 0.001) higher proportion of U/M fragments than control cfDNA. Results: To evaluate the performance of fragment-based algorithm in cfDNA, the false positive was assessed by resampling from healthy subjects. The healthy cfDNA were randomly assigned 20 times as pseudo case-control groups of 30 /170. The number of markers among healthy samples was recorded as NFalse Positive (NFP), while the number of markers between cancer and healthy samples was recorded as NPositive (NP). SNR was defined as (NP-NFP)/NFP. We calculated SNR using the fragment-based algorithm and achieved SNR of 135.2 for colorectal cancer, compared to SNR of 2.2 using DSS. The improved SNR was also reproduced in other cancer types. Concordance between plasma and tissue markers was 30.09%-95.92% (median 66.72%) among 7 cancer types, which was related to the proportion of ctDNA for each cancer type. Plasma markers which show weak signals in tissue are defined as blood-specific markers. These markers were enriched in immune-related pathways, suggesting a correlation to the immune response of cancer patients. Conclusions: We developed a novel fragment-based algorithm that achieves much higher SNR than DSS in cfDNA. We also identified blood-specific methylation markers which may improve the sensitivity of early cancer detection in clinical. [Table: see text]
Unintentional weight loss, body composition, and all-cause mortality among United States adults with cancer.
Authors: Lucido Thomas , Visaria Aayush , Bates Benjamin
Published: June 2024

e22527

Background: While unintentional weight loss (UWL) is a common presenting symptom of malignancy, weight changes also occur regularly in patients with diagnosed cancer.UWL is often characterized by considerable muscle and fat loss and can lead to cachexia, which may increase risk of mortality. This nationally representative study aims to assess the association between UWL and mortality among adults with history of cancer and characterize differences in body composition. Methods: This was a retrospective cohort study of the National Health and Nutrition Examination Survey 1999-2018 linked to the 2019 National Death Index. We identified adults with self-reported history of cancer, excluding non-melanoma skin cancer. Weight change was determined as the difference between current weight and self-reported weight 1 year prior to interview. We categorized weight change into: UWL (unintentionally losing > 5% of weight), intention weight loss (IWL) (intentional loss > 5% weight), no weight change (NWC) (≤5% weight change), weight gain (WG) ( > 5% weight gain). We estimated risk of all-cause mortality using gender-stratified multivariable Cox regression, adjusting for demographics (age, race, income, education, insurance status), sociobehavioral factors (smoking, alcohol, nutrition, physical activity, self-reported health), comorbidities, and albumin. We additionally identified participants with cancer who had whole-body dual x-ray absorptiometry scans (2011-2018) and assessed body composition measurements including total lean mass, lean-to-fat ratio, subcutaneous fat mass, and visceral fat mass by weight change category using multivariable linear regression. Results: Among 3,153 participants (mean age 62 years, 55% women, 82% non-Hispanic White), the top four primary cancers were breast (21%), prostate (14%), melanoma (11%), and cervical (11%). Approximately 9% of men and 11% of women experienced UWL. Adults with UWL were more likely to have lower socioeconomic status, more functional limitations, and higher comorbidity burden. Across a median 7 years (IQR, 3-11y) follow-up, 22% of females and 34% of men died. Both men and women experiencing UWL had more than 2 times the risk of all-cause mortality compared to NWC (adjusted HR [95% CI]; men: 2.02 [1.47, 2.78]), women: 2.43 [1.78, 3.34]). Participants with UWL had less subcutaneous fat mass (-113g, 95% CI [-242, 16]) and visceral fat mass (-65g, 95% CI [-146, 16]) compared to NWC, though not statistically significance. Conclusions: UWL in patients with history of cancer is independently associated with more than 2 times the risk of all-cause mortality in both men and women. UWL was associated with proportional loss of both subcutaneous and visceral fat mass. Further longitudinal studies examining UWL’s impact on body composition changes and downstream outcomes by cancer type and stage are needed.
Does the most common gynecological cancer have an awareness blind spot? An Israeli preliminary study.
Authors: Levy Bar , Vaknin Zvi
Published: June 2024

e22540

Background: Our objectives were to assess the awareness of healthy women who present online to endometrial cancer (EC) symptoms and risk factors among the Israeli population. Methods: A survey regarding awareness of EC symptoms and risk factors was published in secure link on popular Israeli feminine social-media groups ( > 20,000 users), for accurate reach. The survey was conducted during May 2023 just before establishing June as Uterine Cancer Awareness Month by IGCS. 1161 healthy women completed the survey voluntarily and anonymously. Results: Thousand-fifty-six women met the survey’s inclusion criteria. About 90% graduated college, all graduated high-school. The respondents were predominantly non-orthodox Jewish. More than quarter had a healthcare background. About two-thirds visit gynecologist at least once a year and about three-quarters had a gynecological examination in the last year. Most respondents were aware of other gynecological cancers, mainly cervical and ovarian cancers. Only about a quarter of the survey population had some awareness of EC symptoms. Nearly half of study population had some idea about signs and symptoms of any gynecological cancer. Regarding EC, more than a quarter were unaware of vaginal bleeding as an EC symptom and about 90% were unaware of any EC risk factors. More than three-quarters were defined as unaware at all. Only about 1% were fully aware. A statistical trend was observed as women older than 45 were more aware than younger. If a symptom occurs, most declared they would visit a gynecologist and only about 8% would refer to their general-practitioner. Conclusions: In our study population, we observed an extremely-low awareness of the most common gynecological cancer in the industrial world (EC) which has easily diagnosed symptoms and risk factors. This striking findings, in a highly-educated and media-exposed group, are a wakeup-call for policymakers and regulators to take further action in order to achieve earlier diagnosis and curative prognosis in EC patients.
Progress Tracker Breast Cancer Registry: A Breast Cancer Canada initiative.
Authors: Leduc Shaniah , Gibson Amanda Williams , Dean Michelle Liane , Carson Kimberly , Howell Doris , Khan Omar Farooq
Published: June 2024

e23018

Background: Significant gaps exist in the collection of real-world evidence and validated patient-reported outcome measures (PROMs) data from individuals with breast cancer. Variations in treatment and surveillance care across regional jurisdictions present knowledge gaps across diverse populations which remain difficult to study. The PROgress Tracker Breast Cancer Registry, a large, prospective Canadian PROMs cohort study aims to identify care gaps through the lived patient experience to inform patient-centered care and new research directions. Methods: PROgress Tracker is a real-world, longitudinal, non-interventional PROMs registry directed by Breast Cancer Canada, a registered non-profit patient-led organization, and data managed by University of Calgary’s POET program. Launched in October 2023, this registry aims to enroll up to 50,000 adult Canadian participants with a histologic diagnosis of in-situ or invasive breast cancer (stage 0 through IV) within 10 years. A volunteer-led peer-to-peer support model, based in regional and provincial hubs across Canada, facilitates participant recruitment, engagement, and retention. Individuals who self-identify for study inclusion provide informed consent, demographic and socioeconomic profile, and clinical information on their breast cancer diagnosis. Every three months (and up to 10 years) a bilingual digital platform to reduce barriers to access is used to provide all PROgress Tracker participants 8 validated PROMs to capture evolving measures of self-efficacy for wellbeing, symptoms and overall global functioning. Based on treatment, up to 10 additional PROMs to assess the immediate adverse effects and long-term impacts of treatment are collected. PROgress Tracker has the additional capacity to link to external ethics-approved studies to investigate clinical outcome and healthcare utilization metrics for participants consenting to additional studies. Results: In progress. Conclusions: PROgress Tracker represents Canada’s first national PROMs breast cancer registry. Its unique design and implementation by a patient-led research organization helps fill gaps in current knowledge through longitudinal follow up of patients prior to, during, and after cancer care, and from the time from diagnosis through active treatment, long-term survivorship, and/or palliative care. The PROgress Tracker Registry will comprehensively gather the validated real-world data information rarely collected in in the traditional clinical or trial-based research setting using a novel self-enroll design and digital process. Such information is crucial to support evidence-based decision making to inform patient-centered healthcare change. This allows comparison across Canadian jurisdictions, is scalable to other jurisdictions around the world and further explores the utility of electronic PROMs data capture within a diverse patient population.
Budget impact analysis of participating in sponsored clinical trials in cancer within the Colombian healthcare system: A real-world evidence approach.
Authors: Cardona Zorrilla Andres Felipe , Rojas Puentes Luis Leonardo , Robles Antonio , Badillo Carlos , Ceballos Jorge , Sanchez Natalia , Abello Virginia , Bonilla Gonzalez Carlos Eduardo E. , Mantilla William Armando , Zuluaga Jairo Andrés
Published: June 2024

e23031

Background: Clinical trials (CTs) are a key element in researching and developing new technologies for cancer. Assessing the economic impact of CTs and the potential savings for healthcare systems in Low- and Middle-Income Countries (LMICs) is vital to guide healthcare decision-making. This research aims to quantify the budget impact for the Colombian Health System of including hematology and oncology patients in sponsored clinical trials. Methods: This study uses real-world data (RWD) from CTIC, a high-complexity cancer center from Bogotá, Colombia. We followed the Budget Impact Analysis methodology defined by the local HTA agency using data for five tumors retrieved from an institutional data lake resource using clinical information and related costs. Tumors were selected based on their prevalence and economic burden. CTIC’s clinical experts validated the cost-generating events for each disease. The eligible population for phase 3 clinical trials was estimated from the literature and CTIC oncology units’ leaders’ opinions. Prevalent and incident population data were obtained from the Colombian High-Cost Account (CAC). Results: There are 7,703 potential patients to be included in CTs, representing a total cost of 217,544,023 for 2023 USD (1 USD = 4,325 COP). If at least 25% of patients with these tumors were included in CTs in 2023, the approximate cost would be 54,386,006 USD. Advanced prostate cancer represented the highest cost, given its prevalence and CT inclusion opportunity. Conclusions: With approximately USD 24 billion spent on healthcare (USD 5 billion in drugs), Colombia could save around 4% of its drug expenditure if all potential candidates were to be included in cancer CTs. Furthermore, assuming similar average costs per patient, inclusion rates, and population growth for 5-years, this could represent savings of around USD 1.1 billion. These savings would ease the reimbursement and access to innovation for Colombian patients, improving health outcomes and enhancing long-term financial sustainability. [Table: see text]
Discharge outcomes in COVID and non-COVID cohorts of hospitalized patients with cancer: A comparative analysis in the first year of the COVID-19 pandemic.
Authors: Sajith Bilja Kurian , Cesario Sandra K. , Fadol Anecita P. , Nates Joseph ,   D3CODE
Published: June 2024

e23063

Background: This research aimed to assess and compare the discharge outcomes in two cohorts of hospitalized oncology patients – those with COVID-19 and those without COVID-19 in the initial year of COVID pandemic. Methods: This study compared the length of hospital stay, 30-day readmission, in-hospital mortality, death within 30 days of admission, and death within 90 days of admission between the COVID cohort and the non-COVID cohort, based on prospectively collected hospitalization data from March 2020 to February 2021 at a prominent US cancer center. Results: The study included 4,225 oncology patients, with 551 having COVID-19. Aged on average around 61, both cohorts showed similar gender distribution and were predominantly White or Caucasian, with the most common cancers being hematological. Patients in the COVID cohort experienced a significantly extended hospital stay with a mean length of 12.2 days versus 8.27 days for the non-COVID cohort (p < 0.001). The median stay was also longer for the COVID cohort at 8 days [1, 103] compared to 5 days [1, 141] for the non-COVID cohort. The COVID cohort had a lower 30-day readmission rate post-discharge at 66.6% compared to 71.7% in the non-COVID cohort, with the difference being statistically significant (p = 0.015). Within the COVID cohort, a smaller proportion of patients were readmitted within 30 days of discharge (66.6%), as opposed to the non-COVID cohort (71.7%), with a p-value of 0.015. Although the COVID cohort experienced a higher rate of in-hospital mortality at 11.3% compared to 6.9% in the non-COVID cohort (p < 0.001), there were no significant differences in the rates of death within 30 days of admission (COVID cohort: 25.6% vs. non-COVID cohort: 27.6%, p = 0.349) or within 90 days of admission (COVID cohort: 28.9% vs. non-COVID cohort: 31.7%, p = 0.195) between the two cohorts. Conclusions: Oncology patients with COVID-19 faced longer hospital stays and higher in-hospital mortality rates than those without COVID-19. However, the COVID-19 cohort had a lower rate of readmission within 30 days after discharge. Despite the greater severity of hospitalization for COVID-19 patients, the mortality rates 30 and 90 days post-admission did not significantly differ from those without COVID-19. These findings underscore the need for intensified care and monitoring of hospitalized oncology patients with COVID-19, while also acknowledging the comparable longer-term survival between the cohorts.
In-hospital mortality patterns and risk factors in patients with cancer with COVID-19.
Authors: Didier Alexander James , Campbell Andrew , Nandwani Swamroop , Fahoury Alan , Arsene Camelia
Published: June 2024

e23070

Background: The current body of knowledge regarding outcomes of patients with cancer and COVID-19 is conflicting, with some studies suggesting worsened mortality in those with both diseases This study seeks to shed light on the distinctive factors influencing in-hospital mortality in these two cohorts, providing crucial insights that can guide targeted interventions and enhance healthcare strategies for individuals facing the dual burden of cancer and COVID-19. Methods: We conducted a retrospective review of all patients hospitalized with COVID-19 at our institution between January 1, 2021 and April 1, 2023. Patients were stratified into a cohort of those with cancer and coexisting COVID-19 infection and those with solely COVID-19. The cancer and COVID-19 cohort included patients over the age of 18 who had received treatment for cancer within the last 6 months. Information regarding patient demographic characteristics and comorbidities, as well as in-hospital treatment characteristics and outcomes. We performed descriptive analysis and conducted Chi-squared and t-tests to assess for differences in baseline characteristics between cohorts, then developed Cox proportional hazards models to evaluate risk factors for in-hospital mortality. Results: 456 patients with cancer and COVID-19 were compared with 12,566 patients with only COVID-19. In-hospital mortality was 8.2% in the cancer cohort compared with 9.0% in the COVID-19 only cohort. Patients in the cancer cohort were more likely to be male (55.7% vs 48.2%, p = 0.004), older (71 vs 64 years, p < 0.001) with histories of heart failure (27% vs 21%, p = 0.002), chronic kidney disease (50% vs 39%, p < 0.001), COPD (35% vs 24%, p < 0.001), and anemia (57% vs 24%, p < 0.001). The cancer cohort experienced significantly longer length of stay (9 vs 7 days, p < 0.001). Requirement of mechanical ventilation and ICU stay were similar between groups. Multivariate Cox proportional hazards ratios demonstrated similar mortality between cohorts (HR = 0.863, p = 0.361), with age (HR = 1.041, p < 0.001) and history of acute myocardial infarction (HR = 1.275, p = 0.01) being significant predictors of death. Conclusions: Our study demonstrates the complex relationship between cancer, COVID-19, and in-hospital mortality. Despite differences in baseline characteristics between the cohorts, our findings reveal that, after adjusting for confounding factors, there is no significant disparity in mortality rates between patients concurrently battling cancer and COVID-19 and those solely afflicted by COVID-19. The identification of age and a history of acute myocardial infarction as significant predictors of mortality underscores the need for targeted interventions and vigilant management strategies for individuals facing the dual burden of cancer and COVID-19, ensuring equitable and effective healthcare outcomes in this complicated patient population.
Patient (pt) interview–based content validation of the Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0).
Authors: Cardellino Anna , Zhang Shiyuan , Phiri Kelesitse , Gelhorn Heather , Cutts Katelyn
Published: June 2024

e23106

Background: Pts with myelofibrosis (MF) often experience debilitating symptoms that negatively impact health-related quality of life and functioning. The MFSAF v4.0 is a 7-item questionnaire assessing the burden of MF symptoms, including fatigue, night sweats, pruritus, abdominal discomfort, pain under left ribs, early satiety, and bone pain. Each item is evaluated on an 11-point numeric rating scale (NRS) from 0 (absent) to 10 (worst imaginable). We sought to evaluate the content validity of the MFSAF v4.0 in Janus kinase inhibitor–experienced pts with MF. Methods: Qualitative interviews were conducted remotely by trained interviewers with 20 Janus kinase inhibitor–experienced and symptomatic (MFSAF Total Symptom Score [TSS] ≥10) pts with MF from the US (n = 5) and UK, Germany, Italy, Poland, and Spain (n = 3 each). The interview began with a concept elicitation section to allow spontaneous reporting of important concepts prior to content validation of the MFSAF v4.0 items and NRS as relevant, important, and understandable to pts. Results: Mean pt age was 59.4 years; 50% were male, 55% were diagnosed within the past year, 95% were on any treatment for MF, and 75% were anemic. On average, pts had 2.1 transfusions within the past 3 mo. Mean MFSAF v4.0 TSS was 28.6.The top spontaneously endorsed symptoms were tired (70%), bone/muscle pain (70%), fatigue (55%), night sweats (45%), and pruritis (45%), many of which are also items in the MFSAF v4.0. An additional 18 symptoms were spontaneously mentioned by pts prior to completion of the MFSAF v4.0. Many of these other symptoms were endorsed by ≤10% of pts (1 or 2 pts each), and 90% of pts felt that the measure represented their experience and did not recommend inclusion of additional symptoms after reviewing the measure. Overall, pts showed a clear understanding of the MFSAF v4.0 (Table). No more than 3 pts (15%) mentioned that a given item was not relevant to their individual experience. Pts consistently felt that it was easy to select an answer on the response scale and were able to differentiate response options. When describing meaningful change in terms of NRS, pts most often suggested that an improvement of 2 to 4 points would be meaningful. Conclusions: Based on the findings from this study, the MFSAF v4.0 is a content-valid measure for pts with MF and captures the most important concepts. All items are comprehensible and relevant, with the NRS endorsed as an appropriate response scale. [Table: see text]
Improving the multiple myeloma and clinical trial awareness among Los Angeles County adults.
Authors: Equils Ozlem , Bakaj Amonda , Bakaj Angela , Gonzalez Yenis , Hernandez Linda , Cornejo Jacqueline , Salazar Jimena , Alonso Janneth , Morales Abigail
Published: June 2024

e23108

Background: Multiple Myeloma (MM) is the second most common bone marrow cancer in the United States. The prominent risk factors include Black race and male gender. The purpose of this study was to assess the awareness of MM and clinical trial access among a diverse community of people in the Los Angeles County, and to educate the participants. Methods: An IRB-approved Qualtrics survey was administered electronically to a group of 132 participants aged 18 years and older in Los Angeles County between May 3rd and July 15th of 2023. The survey investigated participants’ knowledge on MM risk factors and symptoms, as well as their understanding and attitudes towards resource and clinical trial access. Information on participant demographics was also collected. Statistical tests including chi-square analysis, descriptive statistics, filtering and sub-setting, and correlation analysis, were all utilized and conducted using the software, R-Studio. MiOra Health Educators provided face-to-face education on MM and clinical trials using CDC-based flyers. Results: Out of 132 respondents, 40 (30.3%) were male, 92 (69.7%) were Latinx, and 78 (59.0%) were a first-generation resident of the United States. 71 out of 92 females (77.4%) and 9 out of the 40 males (22.6%) reported attending their annual wellness visit. Prior to the educational intervention, only 6 out of the 132 participants (4.5%) reported being very knowledgeable about cancer and the way it works. Interestingly, 87 (65.9%) reported being aware of the familial risk of MM. 77 (58.1%) of the respondents reported not knowing what a clinical trial is and clinical trial awareness was found to be the main factor predicting the willingness to participate in one (p < 0.01). 108 (81.8%) of the respondents reported that hearing from past clinical trial participants may be useful, but 111 (84.1%) reported that healthcare professionals (including primary care doctors, nurses, medical practitioners, and oncologists) would be the most appropriate educators on clinical trials. Following the health education intervention, 89 (66.9%) of the respondents reported feeling more knowledgeable about clinical trials, and 93 (70.8%) reported feeling more willing to participate in one. Additionally, 87 (65.9%) reported feeling more knowledgeable about MM risk factors, symptoms, screening, and treatment. Conclusions: Although the risk for MM is higher among males, this group appears to significantly less likely than females to comply with their annual wellness visit, signalling a gender disparity in primary care resource utilization. Knowledge on clinical trials appears to influence participation and although primary care providers are the most trusted resources, community health educators and exposure to past clinical trial participants may help close these gaps in clinical trials.
Impact of COVID-19 on post-treatment breast cancer surveillance and outcomes.
Authors: Hahn Erin Elizabeth , Eng Sarah , Chen Aiyu , Haupt Eric C. , Goodall Shannon , Munoz-Plaza Corrine E. , Gould Michael K. , Nguyen Huong Q , Ganz Patricia A. , Shen Ernest
Published: June 2024

e23114

Background: Little is known about the impact of the COVID-19 pandemic on post-treatment surveillance for patients diagnosed with breast cancer. Disruptions in recommended cancer surveillance screening to detect progression or recurrence could impact long-term outcomes. In this study, we compared use of recommended surveillance screening in pre- and post-COVID time periods for patients diagnosed with breast cancer within a large integrated healthcare system, Kaiser Permanente Southern California (KPSC). We also evaluated detection of breast cancer recurrence or progression. Methods: We identified patients in post-treatment surveillance for early-stage breast cancer in two time periods, pre-COVID and post-COVID. Pre-COVID included patients diagnosed 01/01/2015-12/31/2016 with 2 years of follow-up time through 12/31/2019; post-COVID included patients diagnosed 06/01/2018-05/31/2020 with 2 years of follow-up time through 05/31/2023. Eligible patients were diagnosed with stage 0-II breast cancer and received curative surgery. We excluded patients with multiple cancers, and those who disenrolled from the health plan, died, or were referred to hospice within the 2-year surveillance period. Based on NCCN guidelines, we identified the proportion of patients who underwent annual breast imaging during each year of the surveillance period. A log Poisson model examined the association between imaging and COVID period; covariates included patient age, race/ethnicity, cancer stage, treatment, and comorbidity score. We identified cancer recurrence or progression using diagnostic, treatment, and imaging codes. Results: We identified 3,369 pre-COVID patients and 3,925 post-COVID patients; groups were similar (Table 1). A significantly smaller proportion of post-COVID patients received surveillance screening (first surveillance year: 85% in the pre-COVID group vs. 77% post-COVID, p = 0.001; second year: 83% vs. 80%, p = 0.01). Patients were 4% less likely to receive imaging post-COVID compared to pre-COVID (adjusted OR 0.96, 95% CI: 0.94-0.98). Similar percentages of patients had breast cancer recurrence or progression in the pre-COVID (2.7%) and post-COVID (2.5%) periods (p-value = 0.44). Conclusions: Fewer post-treatment breast cancer patients received recommended annual breast cancer screening for surveillance in the post-COVID period compared to pre-COVID, but breast cancer recurrence or progression was similar between groups. Given the less frequent surveillance in the post-COVID period, cancer recurrence and progression should be evaluated over 5- and 10-year periods as data become available. [Table: see text]