bluuebunny/crossref_metadata_2025_split huggingface dataset:
Real-world clinical characteristics and treatment patterns among people with hepatocellular carcinoma in the United States (US) treated with resection or ablation.
Authors: Parikh Neehar , Underwood Dex L. , Hu Jenny , Stirnadel-Farrant Heide A. , Kebede Nehemiah , Patel Ravi , Garcia-Reyes Kirema 
Published: January 2024

436 Background: Treatment guidelines for early-stage hepatocellular carcinoma (eHCC) include therapies with curative intent comprising three primary strategies: liver transplantation, surgical resection, or ablation (for small lesions). However, many people experience HCC recurrence, with recurrence rates of up to approximately 80% within 5 years after resection. Furthermore, contemporary insights into the eHCC treatment landscape and post-recurrence treatment patterns in the real-world setting are lacking. This study was designed to assess demographic and clinical characteristics and treatment patterns for people with HCC who received resection or ablation as a proxy for defining early-stage disease. Methods: This was a retrospective observational cohort study of adults with HCC treated with resection or ablation in the US, conducted using linked electronic health records and healthcare claims from Optum’s de-identified Market Clarity Data. Index date was the date of the first observed resection or ablation between July 2016 and March 2021. Baseline characteristics were assessed in the 6-month period prior to index, and patients (pts) were followed until end of continuous enrolment in a medical and pharmacy plan, death, or data cut-off (September 2022), whichever occurred first. Demographic and clinical characteristics and treatment patterns, including subsequent treatments, were described. Results: A total of 649 pts met the study criteria and were included in the analyses. The median duration of follow-up was 23.0 months (range: 0.03–74.55 months). The mean age at index was 64.1 years (median 64.0 years), with a majority male gender (72.9%) and White race (65.5%). A total of 385 pts (59.3%) underwent ablation only, 242 pts (37.3%) underwent resection only, and 22 pts (3.4%) underwent both procedures at index. Transarterial embolization alone was received by 57 (8.8%) pts and was the most common embolization procedure received during follow-up, followed by transarterial chemoembolization alone in 46 (7.1%) pts and transarterial radioembolization in 46 (7.1%) pts. A total of 88 (13.6%) pts received systemic therapies for HCC (tyrosine kinase inhibitors, programmed cell death protein-1/programmed cell death-ligand 1/cytotoxic T-lymphocyte-associated antigen 4 inhibitors, or angiogenesis inhibitors) during follow-up. Conclusions: In this real-world cohort, ablation was the most received curative intent therapy for HCC. Among treatments received during follow-up, locoregional therapies were used most often, followed by systemic therapies. Although limited by its retrospective, observational nature, short duration of follow-up, and availability of data in the database, the results of this analysis provide a contemporary landscape of early-stage treatments for HCC in the real-world setting.
Incidence patterns of colorectal cancer in Norway 1993-2022 by age, site, and morphology.
Authors: Ystgaard Mari Fjørtoft , Myklebust Tor Åge , Smeby Jørgen , Larsen Inger Kristin , Guren Tormod Kyrre , Kure Elin H. , Tveit Kjell Magne , Glimelius Bengt , Guren Marianne G. , Hamfjord Julian 
Published: January 2024

44 Background: The incidence of colorectal cancer (CRC) has decreased or stabilized in most high-income countries, but an alarming increase is observed in younger adults. Norway has high CRC incidence, which increased until the past two decades, then stabilized. A national screening program started in 2022; hence, this study is on a screening-naïve population. We aim to describe up-to-date incidence patterns of CRC, with emphasis on age-specific differences in tumor site and morphology. Methods: We extracted data on all CRC cases diagnosed 1993 to 2022 from the Cancer Registry of Norway, with completeness estimated to be >99%. Data was stratified by age groups (20-49, 50-74, and ≥75), sex, primary tumor location and morphology. Age-adjusted incidence rates were calculated using Stata 17 and analyzed using Joinpoint regression to provide annual percentage change (APC) and average annual percentage change (AAPC) of incidence rates. Results: There were 107 524 cases eligible for analysis. In the age group ≥75, overall incidence increased (AAPC 0.8), mainly caused by right-sided colon cancer (RCC), which increased until 2016 (APC 2.5), followed by a slight decrease (APC -0.9). The age group 50-74 had stable incidence (AAPC -0.2). Incidence in the age group 20-49 increased (AAPC 1.3), caused by left-sided colon cancer (LCC) and rectal cancer (RC). When stratifying on morphology, incidence of mucinous carcinomas was stable (AAPC -0.6), approx. 10% of all CRC cases. Neuroendocrine neoplasms (NEN) increased in all age groups (AAPC 4.2), accounting for only 1.4% of all CRC cases; but contributed somewhat to the overall incidence in age group 20-49, in particular RC where 8.8% of cases were NEN. In the age group 20-49, adenocarcinomas had an AAPC of 1.4, mucinous carcinomas -0.8, and NEN 4.1. Conclusions: We found an increasing incidence of CRC in the youngest (<50) and elderly (≥75). LCC and RC incidence increased in the youngest age group, still increasing up until 2022. In contrast, the increase in the elderly was mostly due to RCC. Different incidence patterns of primary tumor locations in younger and older age groups may imply differences in etiology and prognosis, different biological features and possibly therapeutic implications. [Table: see text]
Disparities in surgical outcomes and survival based on insurance status among patients with fibrolamellar hepatocellular carcinoma: A national perspective.
Authors: Czaplicki Alec , Bine Christopher Reese , Lee Aron , Nielsen Eric , Silberstein Peter T. 
Published: January 2024

440 Background: Fibrolamellar hepatocellular carcinoma (FL-HCC), encompassing 1%-5% of all liver cancers, is a rare, malignant cancer that typically affects patients less than 40 years old. FL-HCC usually affects individuals with healthy livers and initially presents asymptomatically. Prior studies describe the importance of surgery on survival, with a 5-year survival of about 44%-68% with surgery versus 2%-17% without surgery. Disparities in outcomes regarding surgical factors and survival between private insurance, Medicare, Medicaid, and no insurance remain unknown. This study aims to determine differential surgical factors and survival of patients with FL-HCC based on insurance status. Methods: The National Cancer Database was used to identify patients diagnosed with FL-HCC from 2004-2019 using the histology code 8171 as assigned by the Commission on Cancer Accreditation program. Kaplan-Meier, Chi-Square, ANOVA, and Cox Proportional Hazards tests were performed. Data was analyzed using SPSS version 29 and statistical significance was set at α=0.05. Results: Of the 436 patients included, 241 (55%) had private insurance and 96 (22%) had Medicare. Patients with private insurance had a longer median survival (49 months) than patients with Medicare (18 months; p<.001) and Medicaid (26 months; p<.004);no significant survival difference existed between Medicare, Medicaid, and no insurance (39 months) ( p’s≥.063). After controlling for age, income, tumor size, adjuvant therapy, receiving surgery, and surgical margins of primary site, private insurance was associated with an independent decrease in hazard (HR=.899; p=.004).Patients with private insurance received more surgery (68%) than those with Medicaid (50%), no insurance (48%), and Medicare (32%) ( p’s<.001); grade did not differ amongst insurance types ( p=.069).Furthermore, patients with private insurance had more surgical margins of the primary tumor site without residual tumor (57%) compared to those with Medicaid (40%), no insurance (38%), and Medicare (23%) ( p’s<.001).While patients with Medicare presented with greater co-morbidities, age at diagnosis (67), tumor size (476 mm), positive regional lymph nodes (90), and days between diagnosis and treatment (45), patients with private insurance presented at a younger age (31) with fewer co-morbidities, smaller sized tumors (366 mm), and fewer days between diagnosis and treatment (26) ( p’s<.018). Conclusions: Since patients with private insurance received the most surgery, best surgical margins, and displayed the highest overall survival, while patients with Medicare were diagnosed at the highest age with the largest tumor size and most positive regional lymph nodes, future research should explore ways to alleviate the disparity in factors affecting surgical resections and early diagnoses among insurance statuses.
Real-world (RW) characteristics, treatment patterns, and outcomes of patients with cholangiocarcinoma (CCA) treated with pemigatinib.
Authors: Saverno Kim , Brown-Bickerstaff Cherrishe , Kotomale Angele , Rodriguez Michael A. , Feinberg Bruce A. , Gordon Sarah , Ren Haobo , Braunstein Evan Mark , Kim Richard D. 
Published: January 2024

444 Background: Pemigatinib demonstrated efficacy in fibroblast growth factor receptor (FGFR)–altered CCA in the FIGHT-202 trial; however, limited RW evidence exists on treatment patterns and outcomes in patients (pts) with CCA treated with pemigatinib. This study assessed characteristics, FGFR2 testing patterns, treatment patterns, and outcomes of pts treated with pemigatinib for locally advanced or metastatic CCA in the United States as part of routine clinical care. Methods: This retrospective, multisite cohort study included pts with a diagnosis of unresectable locally advanced or metastatic CCA who were initially prescribed pemigatinib on or after 04/17/2020. Eligible pts were ≥18 years at pemigatinib prescription and had ≥4 months (mo) of follow-up (unless <4 mo due to death). Pts who received pemigatinib as part of a clinical trial or those who received systemic therapy for another primary malignancy (other than cancer of unknown primary or hepatic cancer) were ineligible. Participating physicians from Cardinal Health’s Oncology Provider Extended Network—approximately 72% from community oncology practices—abstracted data from the medical records of eligible patients into electronic case report forms. Results were summarized using descriptive statistics. Results: Data from 120 pts (49% male; 55% White; 19% Hispanic; median age at pemigatinib prescription, 65 years) were collected from 18 physicians/practices. Testing for FGFR2 alterations was completed in 93% (n=111) of pts; of those, all but one patient (result unknown) tested positive, and 95% were tested using next-generation sequencing. At time of pemi prescription, 90% of pts had metastatic disease. Across all pts, 94% and 6% were prescribed pemigatinib as 2nd and 3rd line of therapy, respectively. The most common starting dosage was 13.5 mg daily for 14 days of 21-day cycle (87.5% of pts). Among the 60 pts who discontinued pemi during the 6.5-month median study follow-up period, 68% discontinued due to disease progression. Median RW progression-free survival (rwPFS) from date of pemigatinib initiation was 7.4 (95% CI: 6.4–8.6) mo. Among the 116 patients with tumor response data available, RW overall response rate (rwORR) was 61% (95% CI: 52% - 71%). Conclusions: This study complements the FIGHT-202 clinical trial by assessing the use of pemigatinib among a diverse population of patients with CCA under RW conditions. Secondarily, the rwORR and rwPFS from this study support the clinical benefit of pemigatinib demonstrated in FIGHT-202.
Comparing first-line (1L) atezolizumab plus bevacizumab (A+B) to lenvatinib (L) or sorafenib (S) in patients with unresectable hepatocellular carcinoma (uHCC): Findings from the National Veteran Health Administration (VHA) database.
Authors: Kaplan David Edward , Tan Amie , Xiang Cheryl , Mu Fan , Ogale Sarika , Hernandez Sairy , Li Jiayang , Lin Yilu , Shi Lizheng , Singal Amit G. 
Published: January 2024

446 Background: The approval of A+B has reshaped the treatment paradigm for patients with uHCC, establishing a new standard of care for 1L uHCC treatment. The VHA is the largest independent healthcare delivery network and the largest provider of liver-related care in the US. We aimed to compare patient characteristics and clinical outcomes between veterans who received 1L A+B and those who received L or S for unresectable HCC. Methods: Using data from the VHA National Corporate Data Warehouse (1/1/2017 - 12/31/2022), we conducted a retrospective cohort study in patients diagnosed with uHCC who initiated A+B after 2020 or initiated L or S after 2018. Child-Pugh (CP) scores were calculated using a previously validated algorithm. Kaplan-Meier and multivariable Cox regression methods were applied to compare overall survival (OS) between the groups. Results: We identified 405 eligible patients who received A+B, 453 received L, and 1,016 received S. Compared with patients receiving L or S, those treated with A+B had a higher proportion of CP A5 (47% and 35% vs. 54%, respectively; both p<0.05), a similar proportion of A6 (30% and 32% vs. 31%, p=0.683 and 0.681, respectively), a lower proportion of B7 (15% and 20% vs. 9%, respectively; both p<0.05), and a greater comorbidity burden as measured by the mean Charlson Comorbidity Index (CCI) (5.8 [L or S] vs 6.6 [A+B], both p<0.05). Median OS (mOS) was 12.8 months (95% CI: 10.6, 17.1) in the A+B cohort, compared to 9.5 months [95% CI: 7.8, 11.4] in the L cohort, and 8.0 months [95% CI: 7.1, 8.6] in the S cohort. In subgroup analyses, longer mOS was observed with A+B than with L or S in CP A patients and those with ALBI grades 1-2A; similar mOS was observed among those with more severe liver dysfunction (Table). In multivariable analysis, A + B was associated with a 26% lower risk of death relative to L (HR=0.74, 95% CI: 0.62-0.88) and 30% lower risk of death compared to S (HR = 0.70, 95%CI 0.60-0.82), both p<0.001. Conclusions: In a real-world VHA experience with A+B among diverse patient groups, a clinically notable survival benefit was observed with A+B compared to L or S. Future evaluations of the safety of using A+B in patients with greater liver dysfunction, including CP B and cirrhosis, are needed to evaluate the expanded use of A+B. [Table: see text]
Zanidatamab (zani) in previously treated HER2+ biliary tract cancer (BTC): Impact on patient-reported pain outcomes in the phase 2b HERIZON-BTC-01 study.
Authors: Pant Shubham , Fan Jia , Oh Do-Youn , Choi Hye Jin , Kim Jin Won , Chang Heung-Moon , Bao Lequn , Sun Hui-Chuan , Macarulla Teresa , Xie Feng , Metges Jean-Philippe , Ying Jie-Er , Bridgewater John A , Tejani Mohamedtaki Abdulaziz , Chen Emerson Yu-sheng , Wasan Harpreet Singh , Ducreux Michel Pierre , Ma Jiafang , Garfin Phillip M. , Harding James J. 
Published: January 2024

450 Background: The HER2 bispecific antibody zani showed rapid, durable responses and a manageable safety profile in patients with HER2+ BTC in the pivotal HERIZON-BTC-01 study. Here we report pain outcomes and opioid use for patients in this trial. Methods: HERIZON-BTC-01 (NCT04466891), an ongoing, open-label, Phase 2b study, is assessing zani (20 mg/kg IV Q2W) in patients with HER2+ (gene amplification and immunohistochemistry 2+ or 3+ [Cohort 1] and 0 or 1+ [Cohort 2]), locally advanced, unresectable or metastatic BTC (gallbladder cancer, intra-/extra-hepatic cholangiocarcinoma) who received prior gemcitabine-containing treatment (tx). Exploratory endpoints assessed at baseline (BL) and every 8 weeks included the Brief Pain Inventory short form, the visual analog scale (VAS), and analgesic opioid use. Results: Of the 80 patients with HER2+ BTC in Cohort 1 who were assessed for primary efficacy (median age 64 yrs; 56% female), 1 (1%) had complete response (CR), 32 (40%) partial response (PR), 22 (28%) stable disease (SD), and 24 (30%) progressive disease (PD); 1 patient was not evaluable. Responders (CR or PR) generally reported improved pain scores and less pain interference with activities of daily living. Patients with PD generally had worsening pain scores and pain interference by time of best on tx score (BONT) (Table). Overall, patients who responded to zani reported improved quality of life (VAS scores) vs BL. Fewer patients with CR (0.0%; n=1) and PR (3.1%; n=32) increased opioid use overall post-BL vs patients with SD (9.1%; n=22) or PD (34.8%; n=23). Conclusions: Patients with HER2+ BTC who responded to zani reported less pain and pain interference compared with BL, further supporting the development of zani as a tx option for these patients, with the goal of improving patient outcomes. Clinical trial information: NCT04466891 . [Table: see text]
Real-world correlation of RFS and OS in early-stage hepatocellular carcinoma treated with curative intent: A retrospective analysis of SEER-Medicare data.
Authors: Mehta Neil , Desai Kaushal , Kamble Shital , Song Yan , Gao Emily , Malhotra Usha , Lax Angela , Sundaresan Sanjana , Downes Nathaniel , Zhang Adina , Siegel Abby B. 
Published: January 2024

451 Background: Curative intent therapy with resection or ablation for early-stage HCC is associated with high incidence of recurrence. Effective adjuvant therapy is needed to improve outcomes. While OS is the preferred endpoint for demonstrating efficacy of new adjuvant therapies, recurrence-free survival (RFS) may better reflect the benefits associated with adjuvant therapy due to being independent of subsequent therapies upon recurrence. Additionally, RFS may provide insights into patient (pt) outcomes while OS data are immature. We assessed the correlation between OS and RFS in pts with early-stage HCC after treatment with curative intent. Methods: This study used the SEER-Medicare database (2007–2017). RFS was defined as time from curative intent therapy, by local ablation or surgical resection, until death or disease recurrence (first of: new HCC treatment, liver transplant, metastatic disease). OS was defined as time from curative intent therapy until death. Kaplan-Meier analyses were used, censoring on end of Medicare Parts A/B/D continuous eligibility or end of data availability. RFS/OS were summarized overall and by type of prior local therapy and risk of recurrence. Pts with a solitary tumor with histologic grade 3 or 4, solitary tumor >3cm, or multiple tumors were classified as high to very high risk. Pts with a solitary tumor not histologic grade 3 or 4 or a solitary tumor ≤3 cm were classified as low to intermediate risk. Correlation between RFS and OS was assessed using the Kendall’s τ rank correlation. Results: 611 HCC pts met the inclusion criteria (mean follow-up 3.3 years; mean age 73.3 years). Average (median) primary tumor size at diagnosis was 4.8 (4) cm. 91.6% of pts had a solitary tumor at diagnosis. 66.1% of pts experienced death or recurrence (Table). Median [IQR] OS and RFS post-curative intent therapy were 4.6 [1.8, NR] and 1.8 [0.8, 6.3] years, respectively. OS and RFS were poorer for those in the ablation (median OS 3.2 years; RFS 1.2 years) and high to very high risk of recurrence group (median OS 4.3 years; RFS 1.4 years). Kendall’s τ rank correlation model in the overall group demonstrated a statistically significant positive correlation between RFS and OS (τ = 0.66; 95% CI: 0.60–0.70; P < 0.001). Conclusions: Based on real-world data, a significant positive correlation between RFS and OS supports RFS as an efficacy marker and appropriate endpoint for pts in adjuvant HCC setting when OS data are immature. [Table: see text]
First-line therapies in a Latino-rich cohort of US patients (pts) with hepatocellular carcinoma (HCC) and Child Pugh B (CPB) cirrhosis.
Authors: Alfaro Moses Andrew , Arora Sukeshi Patel 
Published: January 2024

453 Background: There are limited prospective clinical trials in patients with both HCC and CPB cirrhosis. As the prevalence of HCC and cirrhosis has been rising among Latinos in South Texas, it is crucial to examine the liver dysfunction within this demographic to understand the contributing factors to treatment patterns. Therefore, we conducted a retrospective analysis of 36 patients with both HCC and Child Pugh B (CPB) cirrhosis. Methods: A dataset of 65 patients with HCC and cirrhosis from 2009 – 2019 was used to identify 36 patients with CPB treated at a Latino-Rich NCI-designated Cancer Center. We further segmented patients of Child Pugh Class B by ALBI grades (1-3) to explore variations in demographics, liver dysfunction, and first-line therapies. Results: Median age overall of the cohort is 59 (50-71). Ethnicity: Latino White (83%), Non-Latino White (14%), Black (3%). Etiology: HCV (78 %) EtOH (50%), NASH (19%), and HBV (3%). ALBI Grade: I (6%), II (50%), III (44%). First line treatment in CPB: Sorafenib (86%), Nivolumab (8%), Lenvatinib (6%). Sorafenib was used across all ALBI grades, while Lenvatinib and Nivolumab were given only in ALBI II (Table). Conclusions: Sorafenib was more often given to patients with CPB cirrhosis, in the first line setting; however, more patients were treated with nivolumab or lenvatinib with ALBI II. Further investigation of survival based on ALBI levels and the trajectory of liver function over time in response to different treatment approaches is underway in a larger cohort who has received newer immunotherapy-based regimens. With more agents approved for HCC, prospective clinical trials are needed in CPB and HCC. [Table: see text]
Real-world use of pemigatinib (pemi) for cholangiocarcinoma (CCA) among racial and ethnic minorities in the United States.
Authors: Kim Richard D. , Brown-Bickerstaff Cherrishe , Kotomale Angele , Rodriguez Michael A. , Feinberg Bruce A. , Gordon Sarah , Savill Kristin M. Zimmerman , Ren Haobo , Blecker Michael , Saverno Kim 
Published: January 2024

461 Background: Pemi received accelerated approval by the FDA in 2020 for treatment of patients (pts) with unresectable locally advanced or metastatic CCA with a fibroblast growth factor receptor 2 ( FGFR2) fusion or other FGFR2 rearrangement. A recent real-world study (RWS; submitted to ASCO-GI 2024) assessing characteristics, FGFR2 testing patterns, treatment patterns, and outcomes of pts treated with pemi for locally advanced or metastatic CCA in the US as part of routine clinical care found substantial racial and ethnic diversity in pts treated with pemi. The purpose of this analysis is to describe the findings of the pemi RWS for CCA by race and ethnicity. Methods: This was a retrospective cohort study in which US-based participating physicians from Cardinal Health’s Oncology Provider Extended Network abstracted data from eligible pts’ medical records into electronic case report forms. Eligible pts were ≥18 years of age, initially prescribed pemi for unresectable locally advanced or metastatic CCA on or after 4/17/2020, and had ≥4 mo of follow-up (unless <4 mo due to death). Pts who did not receive pemi or had received pemi in a clinical trial or systemic therapy for another primary malignancy (except for cancer of unknown primary or hepatic cancer) were excluded. Results were summarized by race (White, Black/African American, “Other” [pts with mixed race or a race other than White, Black/African American, or Unknown]) and ethnicity (Non-Hispanic, Hispanic) using descriptive statistics. Results: 18 physicians abstracted data for 120 eligible pts. Median follow-up from initial pemi prescription was 6.5 mo, at which time 71 pts (59%) were alive and 60 (50%) were still receiving pemi. The racial makeup of the study population was 55% White, 21% Black, 18% Other, and 7% Unknown. Ethnic composition was 78% Non-Hispanic, 19% Hispanic, and 3% Unknown. Key results are presented in the Table. Conclusions: The diverse population in this RWS is reflective of the heterogeneous CCA pt population in the US. These real-world overall response rates support the clinical benefit of pemi across racial and ethnic groups and complement the results of the clinical trial. [Table: see text]