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Aim: To understand US physicians' frontline (1L) treatment preferences/decision-making for stage III/IV classic Hodgkin lymphoma (cHL). Materials & methods: Medical oncologists and/or hematologists (≥2 years' practice experience) who treat adults with stage III/IV cHL were surveyed online (October–November 2020). Results: Participants (n = 301) most commonly considered trial efficacy/safety data and national guidelines when selecting 1L cHL treatments. Most physicians (91%) rated overall survival (OS) as the most essential attribute when selecting 1L treatment. Variability was seen among regimen selection for hypothetical newly diagnosed patients, with OS cited as the most common reason for regimen selection. Conclusion: While treatment selection varied based on patient characteristics, US physicians consistently cited OS as the top factor considered when selecting a 1L treatment for cHL. Classic Hodgkin lymphoma (cHL) is a type of cancer that grows in lymph nodes. The researchers created a survey to assess how doctors in the USA choose medicine to treat patients who are newly diagnosed with an advanced stage of cHL (stage 3 or 4 out of 4 stages). We surveyed 301 doctors who treat patients with cHL. When choosing a medicine to treat cHL, most doctors said they consider results from research studies, how well the medicine works, information on the medicine's safety and recommendations in official guidelines. Most doctors said that overall survival (how long the patient survives after being diagnosed with cHL) is the most important outcome they consider when choosing a medicine to treat cHL. During the survey, doctors saw four unique patient profiles. These profiles differed in age, disease stage (how far along the cHL is) and other illnesses the patient has. While medicine choice was different across profiles, overall survival was still the reason for choosing each individual patient's medicine. These survey results show that doctors in the USA highly consider overall survival when choosing medicine for patients newly diagnosed with an advanced stage of cHL. Survey of US physicians who treat patients with #cHL shows that while frontline treatment selection varies based on individual characteristics of those with stage III/IV cHL, the regimen's reported overall survival is the top consideration. [ABSTRACT FROM AUTHOR]

Summary: We explored patient front‐line treatment preferences in newly diagnosed stage III/IV classic Hodgkin lymphoma (cHL). The CONNECT patient survey, administered online from 30 December 2020 to 1 March 2021, examined preferences overall and by age at diagnosis in 182 adult patients diagnosed with stage III/IV cHL within the past 10 years in the United States. At diagnosis, patients' median age was 36 years; 66% of patients were younger (aged 16–41 years) and 34% older (aged 42–85 years). When asked about initial treatment goals, 74% of patients ranked cure as their first or second goal (86% younger vs. 52% older patients; p < 0.001). At diagnosis, 72% of patients preferred aggressive treatment, and 85% were willing to accept more short‐term risks in exchange for a better‐working therapy long term. For long‐term risks, younger versus older patients were significantly more concerned about second cancers (p < 0.001) and fertility issues (p = 0.007), whereas older patients were more concerned about lung damage (p = 0.028) and infections (p < 0.001). Most patients (94%) reported having a caregiver at some point, but 99% of these patients retained some control of treatment decisions. Collectively, these survey results highlight patient treatment preferences and differences in treatment goals and long‐term side effect concerns based on patient age. [ABSTRACT FROM AUTHOR]

Background Since Food and Drug Administration approval of brentuximab vedotin in combination with cyclophosphamide, doxorubicin, and prednisone (A + CHP) as initial therapy for previously untreated CD30-expressing peripheral T-cell lymphoma (PTCL), there has been limited research on real-world patient characteristics, treatment patterns, and clinical outcomes. Methods We retrospectively analyzed claims of patients with PTCL treated with frontline A + CHP or CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone) using the Symphony Health Solutions database. Adults with International Classification of Diseases-9/10 PTCL diagnosis codes who initiated A + CHP or CHOP between November 2018 and July 2021 were included. A 1:1 propensity score matching analysis was performed that adjusted for potential confounders between groups. Results A total of 1344 patients were included (A + CHP, n = 749; CHOP, n = 595). Before matching, 61% were men; median age at index was 62 (A + CHP) and 69 (CHOP) years. The most common A + CHP-treated PTCL subtypes were systemic anaplastic large cell lymphoma (sALCL; 51%), PTCL-not otherwise specified (NOS; 30%), and angioimmunoblastic T-cell lymphoma (AITL; 12%); the most common CHOP-treated subtypes were PTCL-NOS (51%) and AITL (19%). After matching, similar proportions of patients treated with A + CHP and CHOP received granulocyte colony-stimulating factor (89% vs. 86%, P = .3). Fewer patients treated with A + CHP received subsequent therapy than CHOP overall (20% vs. 30%, P  < .001) and specifically with the sALCL subtype (15% vs. 28%, P  = .025). Conclusions Characteristics and management of this real-world PTCL population who were older and had a higher comorbidity burden than that in the ECHELON-2 trial demonstrate the importance of retrospective studies when assessing the impact of new regimens on clinical practice. [ABSTRACT FROM AUTHOR]

PURPOSE: We surveyed oncologists who treat classic Hodgkin lymphoma (cHL) as part of the CONNECT study to understand the treatment decision-making process, including the impact of positron emission tomography/computed tomography (PET/CT) imaging. METHODS: US physicians self-identifying as oncologists, hematologists, or hematologists/oncologists with ≥2 years of practice experience who treated ≥1 adult with stage III/IV cHL in the frontline setting in the last year were surveyed (October 19-November 16, 2020). Physician demographics, guideline adherence, and PET/CT utilization, interpretation, and access barriers were assessed. RESULTS: In total, 301 physicians participated in the survey. Eighty-eight percent of physicians gave somewhat-to-significant consideration to NCCN guidelines. Most physicians (94%; n = 284) reported obtaining a PET/CT scan at diagnosis; of these physicians, 97% reported obtaining an interim PET/CT scan for stage III/IV cHL, with 65% typically obtaining an interim PET/CT scan after cycle 2. The Deauville 5-point scale (5PS) was the primary scoring system used to review PET/CT results by 62% of physicians, with a positive score defined as ≥3 by 44%, ≥4 by 37%, and ≥2 by 12% of physicians. Fifty-five percent of physicians reported difficulty in obtaining PET/CT scans. CONCLUSION: Although most physicians considered NCCN guidelines when treating patients with stage III/IV cHL, interim PET/CT scans after cycle 2 were not universally obtained. When PET/CT scans were obtained, Deauville 5PS scores were not always provided, and variability existed on what defined a positive score. These findings suggest that opportunities exist for education and improved PET-adapted treatment approaches. [ABSTRACT FROM AUTHOR]

Background: Subgroup analyses of the NAPOLI‐1 study identified that among patients who were irinotecan naïve prior to entering the clinical trial, a survival benefit was observed between the study arm and control arm. This treatment benefit was not observed among those previously exposed to irinotecan. This study sought to understand the impact of prior exposure to irinotecan on clinical outcomes among patients treated with liposomal irinotecan in the real‐world setting. Methods: This retrospective observational study utilized a nationwide electronic health record (EHR)‐derived deidentified database. Data for adult patients with mPDAC treated with liposomal irinotecan‐based regimens between January 2016 and October 2020 were analyzed. Patient characteristics, overall survival (OS), and progression‐free survival (PFS) were assessed. Cox proportional hazard methods were used to calculate hazard ratios (HRs). HRs were adjusted for demographics and relevant clinical covariates. Results: Six hundred and seventy‐five patients with mPDAC treated with a liposomal irinotecan‐based regimen were included. The unadjusted OS HR was 1.3 (95% CI: 1.1–1.6, p < 0.001) and unadjusted PFS was HR 1.4 (95% CI: 1.2–1.7, p < 0.001). After adjustment for baseline characteristics, the adjusted OS HR was 1.0 (95% CI: 0.8–1.3, p = 0.8836) and the adjusted PFS HR was 1.1 (95% CI: 0.8–1.4, p = 0.5626). Conclusions: Prior irinotecan was not found to be a significant predictor of patient outcomes in those later treated with liposomal irinotecan. Thus, the results may inform the rationale for utilizing liposomal irinotecan combination therapy following prior irinotecan exposure in mPDAC, in particular where the prior irinotecan exposure was more distant in time. [ABSTRACT FROM AUTHOR]

Real‐world data (RWD) and real‐world evidence (RWE) are becoming essential tools for informing regulatory decision making in health care and offer an opportunity for all stakeholders in the healthcare ecosystem to evaluate medical products throughout their lifecycle. Although considerable interest has been given to regulatory decisions supported by RWE for treatment authorization, especially in rare diseases, less attention has been given to RWD/RWE related to in vitro diagnostic (IVD) products and clinical decision support systems (CDSS). This review examines current regulatory practices in relation to IVD product development and discusses the use of CDSS in assisting clinicians to retrieve, filter, and analyze patient data in support of complex decisions regarding diagnosis and treatment. The review then explores how utilizing RWD could augment regulatory body understanding of test performance, clinical outcomes, and benefit‐risk profiles, and how RWD could be leveraged to augment CDSS and improve safety, quality, and efficiency of healthcare practices. Whereas we present examples of RWD assisting in the regulation of IVDs and CDSS, we also highlight key challenges within the current healthcare system which are impeding the potential of RWE to be fully realized. These challenges include issues such as data availability, reliability, accessibility, harmonization, and interoperability, often for reasons specific to diagnostics. Finally, we review ways that these challenges are actively being addressed and discuss how private‐public collaborations and the implementation of standardized language and protocols are working toward producing more robust RWD and RWE to support regulatory decision making. [ABSTRACT FROM AUTHOR]

Chemotherapy-related adverse events (AEs) can negatively impact the care of patients. The prevention and management of AEs often require additional medications. This study evaluated the percentages of patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) undergoing second-line therapy with 5-fluorouracil (5-FU)-based regimens that experienced AEs during treatment and received medication to manage those AEs. We conducted a retrospective observational analysis utilizing the Flatiron Health database of adult patients with mPDAC who started second-line therapy between January 2016 and August 2020. The occurrence of diarrhea, fatigue, nausea and vomiting, neuropathy, and hematologic AEs including G3/G4 anemia, neutropenia, and thrombocytopenia was assessed. The use of concomitant medications including atropine and granulocyte colony stimulating factor (G-CSF) was assessed. Of the 825 eligible patients, 29.0% (n = 239) received FOLFIRINOX, 24.0% (n = 198) received FOLFOX, 6.8% (n = 56) received FOLFIRI, and 40.2% (n = 332) received liposomal irinotecan-based regimens. FOLFIRI and FOLFIRINOX regimens were associated with the highest rates of anemia (16.1% and 15.5%), neutropenia (19.6% and 22.6%), and thrombocytopenia (14.3% and 9.6%). The liposomal irinotecan and FOLFOX regimens were associated with lower rates of anemia (11.8% and 12.1%), neutropenia (12.4% and 14.7%), and thrombocytopenia (2.4% and 8.1%). G-CSF use was observed among 63.6%, 34.9%, 33.9%, and 44.9% of patients treated with FOLFIRINOX, FOLFOX, FOLFIRI, and liposomal irinotecan-based regimens, respectively. Diarrhea was observed among 12.5%, 4.5%, 12.5%, and 10.2% of patients who were treated with FOLFIRINOX, FOLFOX, FOLFIRI, and liposomal irinotecan-based regimens, respectively. Nausea and vomiting occurred in 14.9%, 12.6%, 10.5%, and 13.1% of patients treated with FOLFIRINOX, FOLFOX, FOLFIRI, and liposomal irinotecan-based regimens, respectively. Atropine use was higher in patients treated with FOLFIRINOX and FOLFIRI (90.8% and 94.6%, respectively) than in patients treated with liposomal irinotecan-based regimens (75.6%). In patients with mPDAC who received second-line therapy, those who received liposomal irinotecan-based regimens had the lowest rates of anemia, neutropenia, and thrombocytopenia compared to FOLFIRI, FOLFIRINOX, and FOLFOX, while requiring a similar or lower level of medication to treat and manage those adverse events. Patients treated with FOLFIRI received the highest dose of pegfilgrastim to manage neutropenia. The results of this real-world analysis are consistent with prior evaluations of patients with mPDAC and highlight the importance of managing adverse events and associated cost implications. [ABSTRACT FROM AUTHOR]

Simple Summary: Cancer-of-unknown-primary-origin (CUP) is challenging to diagnose and treat, and little is known about its diagnostic work-up, treatment, and outcomes in routine healthcare. We examined data from elderly patients (at least 66 years old) diagnosed with CUP in real-world US clinical practice, using the Surveillance, Epidemiology, and End Results–Medicare-linked database. Only half of elderly patients with CUP received all three diagnostic work-up procedures (biopsy, immunohistochemistry, and imaging), as recommended by guidelines. Patients who received all three diagnostic work-up procedures were more likely to receive any type of anticancer treatment, and patients who did not receive full diagnostic work-up had shorter median overall survival, particularly with increasing age. Overall, these results suggest that further studies are needed to understand why many patients given a diagnosis of CUP do not receive complete diagnostic work-up or treatment. Further research into improving diagnostic work-up and treatment effectiveness in patients diagnosed with CUP is required. Knowledge of contemporary patterns of cancer-of-unknown-primary-origin (CUP) diagnostic work-up, treatment, and outcomes in routine healthcare is limited. Thus, we examined data from elderly patients diagnosed with CUP in real-world US clinical practice. From the Surveillance, Epidemiology, and End Results–Medicare-linked database, we included patients ≥ 66 years old with CUP diagnosed between 1 January 2013 and 31 December 2015. We analyzed baseline demographics, clinical characteristics, methods of diagnostic work-up (biopsy, immunohistochemistry, imaging), treatment-related factors, and survival. CUP diagnosis was histologically confirmed in 2813/4562 patients (61.7%). Overall, 621/4562 (13.6%) patients received anticancer pharmacotherapy; among these, 97.3% had a histologically confirmed tumor and 83.1% received all three procedures. Among those with a histologically confirmed tumor, increasing age, increasing comorbidity score, not receiving all three diagnostic measures, and having a not-further specified histologic finding of only 'malignant neoplasm' were all negatively associated with receipt of anticancer pharmacotherapy. Median overall survival was 1.2 months for all patients. Median time between CUP diagnosis and treatment initiation was 41 days. Limited diagnostic work-up was common and most patients did not receive anticancer pharmacotherapy. The poor outcomes highlight a substantial unmet need for further research into improving diagnostic work-up and treatment effectiveness in CUP. [ABSTRACT FROM AUTHOR]

Background The purpose of this study was to understand how the COVID-19 pandemic has affected health care patterns and outcomes for patients diagnosed with metastatic pancreatic ductal adenocarcinoma (mPDAC) in 2020 compared with those diagnosed with mPDAC in 2019. Patients and Methods We used the Flatiron Health database to identify adults diagnosed with mPDAC from March 1 to September 30, 2019 (pre-COVID-19 cohort) and March 1 to September 30, 2020 (post-COVID-19 cohort). Between-cohort comparisons included demographic and clinical characteristics and year-over-year data for diagnosis of mPDAC, newly treated patients, time to and types of first-line therapy, and adverse events (AEs) during first-line therapy. Overall survival (OS) and milestone survival rates were evaluated. Kaplan-Meier methods were used to assess OS. Results Pre-COVID-19 (n = 923) and post-COVID-19 (n = 796) cohorts had similar baseline demographic characteristics. A smaller proportion of patients in the pre-COVID-19 cohort were initially diagnosed with stage IV disease versus the post-COVID-19 cohort (62.2% vs 69.7%). Between 2019 and 2020, there was a 13.8% decrease in diagnosis of mPDAC and a 13.0% decrease in newly treated patients. Median (interquartile range) times to first-line treatment were similar (21 [13-40] and 19 [12-32] days). Median OS (months) was significantly longer in the pre-COVID-19 cohort (8·4 [95% CI: 7·5, 9·0]) versus the post-COVID-19 cohort (6·1 [95% CI: 5·4, 6·9]; P <.001). Survival rates were higher in the pre-COVID-19 versus post-COVID-19 cohorts. Conclusions During the pandemic, patients were initially diagnosed with PDAC at more advanced stages. While patients in both cohorts appeared to receive similar care, survival outcomes were adversely affected. [ABSTRACT FROM AUTHOR]

Background Sixty-eight percent of patients with pancreatic ductal adenocarcinoma (PDAC) are 65 years and older. Older adults are under-represented in clinical trials and their care is complicated with multiple age-related conditions. Research suggests that older patients can experience meaningful responses to treatment for PDAC. The objective of this study was to evaluate the characteristics, rate of treatment, and survival outcomes of patients with metastatic PDAC (mPDAC) based on age at diagnosis. Materials and Methods Data were extracted for patients diagnosed with mPDAC between January 1, 2015, and March 31, 2020, from the Flatiron Health database. Patients were stratified into 3 age groups: <70 years old, 70-79 years, and ≥80 years. The proportion of patients who received first-line therapy, the types of regimens received in the metastatic setting, overall survival (OS) from the start of treatment were evaluated. Results Of the 8382 patients included, 71.3% (n = 5973) received treatment. Among patients who received treatment 55.5% (n = 3313) were aged <70 years at diagnosis, 33.0% (n = 1972) were 70-79 years, and 11.5% (n = 688) were ≥80 years. Patients ≥80 years of age were more likely to receive gemcitabine monotherapy and less likely to receive FOLFIRINOX. Among first-line treated patients, median OS significantly decreased with age. However, when comparing patients treated with the same first-line regimen, no significant differences in median OS were observed by age. Conclusions This study highlights that older adults with mPDAC can benefit substantially by receiving appropriate levels of treatment. [ABSTRACT FROM AUTHOR]

To quantify healthcare resource utilization (HRU) and costs by disease stage in individuals with Huntington's disease (HD) in a US population. This retrospective cohort study used administrative claims data from the IBM MarketScan Commercial, Multi-State Medicaid, and Medicare Supplemental Databases between 1 January 2009 and 31 December 2018. Individuals with an HD claim between 1 January 2010 and 31 December 2017 were selected. Index date was the date of first HD diagnosis. Individuals were required to have continuous enrollment for ≥ 12 months pre-index, 3 months post-index, and have no pre-index HD claims. All-cause HRU and costs per patient per month (PPPM) (overall and stratified by disease stage) were assessed for individuals with HD. A total of 2,669 individuals with HD were identified. Of these, 1,432 (53.7%), 689 (25.8%), and 548 (20.5%) had early-, middle-, and late-stage HD at baseline, respectively. Mean HRU PPPM by post-index HD stage increased with disease stage for outpatient visits, pharmacy claims, and HD-related pharmacy claims (p < 0.05 for all). Mean inpatient visits and emergency room visits PPPM were highest in individuals with middle-stage HD (p <0.05 for all). Mean total all-cause healthcare cost PPPM for individuals with HD was $2,889, and it was significantly higher in middle-stage individuals, at $7,988, compared with early- and late-stage individuals, at $3,726 and $5,125, respectively; p <0.0001. In the absence of disease staging information in administrative claims data, staging was based on the presence of clinical markers in claims. Our evaluations didn't include the indirect costs of HD, which may be substantial as HD typically affects people at their peak earning potential. HRU and costs of care are high among individuals with HD, particularly among those with middle- and late-stage disease. This indicates that the disease burden in HD increases with disease stage, highlighting the need for interventions that can slow or prevent disease progression. [ABSTRACT FROM AUTHOR]

Background: Many real-world studies of patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) are restricted to single centers, limiting the generalizability of their insights. This study aimed to identify important population-based predictors for survival in patients diagnosed with mPDAC in a broader setting. Methods: Data between 1 January 2017 and 31 December 2019 were extracted from the Flatiron Health EHR database. Treatment-specific predictive models were generated for patients treated with first-line gemcitabine+nabpaclitaxel (GNP), FOLFIRINOX, gemcitabine monotherapy (gem-mono), and second-line liposomal irinotecan-based regimens. The holdout method was used for cross-validation. Age at diagnosis, sex, BMI, smoking status, and ECOG performance score were included in all models with additional demographic, clinical characteristics, and hematological function assessed for inclusion. Results: Of the 3625 patients, 43% received GNP, 26% received FOLFIRINOX, 7% received gem-mono, and 23% received other regimens; 40% (n = 1448) advanced to the second line. Among all first-line patients, the following were included in the final model: prior surgery, white blood cell (WBC) counts, serum albumin (SA), liver function tests (LFTs), serum bilirubin, serum carbohydrate antigen 19-9, and ascites. Models for patients receiving specific therapies differed from the overall model, GNP (ascites removed), FOLFIRINOX (stage at initial diagnosis added), and gem-mono (LFTs omitted). Alkaline phosphatase (ALP), SA, and WBC counts were important predictors of survival among patients treated with second-line liposomal irinotecan. Across all regimens, the strongest predictors of survival were ECOG score, SA, and ALP. Conclusions: In this real-world study of patients with mPDAC, important population prognostic factors of survival were identified in a large cohort of patients receiving systemic treatment. [ABSTRACT FROM AUTHOR]

Objective: To evaluate COVID-19 vaccine breakthrough infections among immunocompromised (IC) individuals. Methods: Individuals vaccinated with BNT162b2 were selected from the US HealthVerity database (10 December 2020 to 8 July 2021). COVID-19 vaccine breakthrough infections were examined in fully vaccinated (≥14 days after 2nd dose) IC individuals (IC cohort), 12 mutually exclusive IC condition groups, and a non-IC cohort. IC conditions were identified using an algorithm based on diagnosis codes and immunosuppressive (IS) medication usage. Results: Of 1,277,747 individuals ≥16 years of age who received 2 BNT162b2 doses, 225,796 (17.7%) were identified as IC (median age: 58 years; 56.3% female). The most prevalent IC conditions were solid malignancy (32.0%), kidney disease (19.5%), and rheumatologic/inflammatory conditions (16.7%). Among the fully vaccinated IC and non-IC cohorts, a total of 978 breakthrough infections were observed during the study period; 124 (12.7%) resulted in hospitalization and 2 (0.2%) were inpatient deaths. IC individuals accounted for 38.2% (N=374) of all breakthrough infections, 59.7% (N=74) of all hospitalizations, and 100% (N=2) of inpatient deaths. The proportion with breakthrough infections was 3 times higher in the IC cohort compared to the non-IC cohort (N=374 [0.18%] vs. N=604 [0.06%]; unadjusted incidence rates were 0.89 and 0.34 per 100 person-years, respectively. Organ transplant recipients had the highest incidence rate; those with >1 IC condition, antimetabolite usage, primary immunodeficiencies, and hematologic malignancies also had higher incidence rates compared to the overall IC cohort. Incidence rates in older (≥65 years old) IC individuals were generally higher versus younger IC individuals (<65). Limitations: This retrospective analysis relied on coding accuracy and had limited capture of COVID-19 vaccine receipt. Conclusions: COVID-19 vaccine breakthrough infections are rare but are more common and severe in IC individuals. The findings from this large study support the FDA authorization and CDC recommendations to offer a 3rd vaccine dose to increase protection among IC individuals. [ABSTRACT FROM AUTHOR]

Background: Pancreatic cancer is expected to be the third deadliest cancer in the US in 2021. Evaluation of treatment response in patients with mPDAC necessitates scheduled clinical and radiographic assessments along with monitoring serum CA 19-9 levels. Currently available single-institution data examining the importance of CA 19-9 monitoring cannot be generalized to real-world settings. We investigated the impact of serum CA 19-9 monitoring and its association with clinical outcomes in patients with mPDAC in a population-based setting. Methods: Data were extracted from the Flatiron Health electronic health record (EHR)-derived de-identified database for patients diagnosed with mPDAC between January 1, 2015, and June 30, 2020. Serum CA 19-9 levels at baseline – defined as the values obtained ≤ 60 days prior to treatment initiation - and during treatment were extracted. CA 19-9 levels > 40 IU/mL were considered elevated. Survival outcomes were compared based on testing frequency, baseline CA 19-9 levels, and change in CA 19-9. Results: 6,118 patients with mPDAC who received treatment were included in the analysis. The median age at diagnosis was 68 years (IQR: 61-75). Patients with normal baseline CA 19-9 experienced longer median survival than patients with elevated levels [1L: 8.8 months (95% CI: 7.9 - 10) vs. 7.2 months (6.8 – 7.5), p < 0.001; 2L: 7.2 months (6.1 – 9.2) vs. 5.2 months (4.9 – 5.6), p < 0.001; 3L: 6.1 months (5.4 – 9.1) vs. 3.9 months (3.4 – 4.3), p < 0.001]. Patients with decreasing/stable CA 19-9 during treatment experienced longer survival than patients who experienced an increase in CA 19-9 levels [1L: 10.9 months (10.5 – 11.3) vs. 5.4 months (5.1 – 5.9), p < 0.0001; 2L: 8.2 months (7.7 – 8.5) vs. 4.3 months (4.1 – 4.7), p < 0.001; 3L: 7.5 months (6.6 – 9.2) vs. 3.7 months (3.4 – 4.3), p < 0.001]. Conclusions: In one of the largest, contemporary, real-world studies of patients with mPDAC, elevated CA 19-9 level at treatment initiation demonstrated a prognostic impact. Routine serial monitoring of CA 19-9 levels during treatment may be warranted, in addition to clinical and radiographic assessment, and may translate into better patient outcomes. Further validation studies are needed to understand the generalizability of these results. [ABSTRACT FROM AUTHOR]

Multiple myeloma (MM) treatment options have evolved rapidly, but how new agents are incorporated into treatment decisions in current practice is not well understood. This study examined prescribing trends of physicians treating newly diagnosed MM and treatment outcomes in the United States. Electronic health record data from 6271 adult patients diagnosed with MM and receiving initial treatment between 1 January 2011 and 31 January 2020 were derived from the Flatiron Health electronic‐health record de‐identified database. The number/types of agents included in therapy regimens, time to next treatment (TTNT), and overall survival (OS) were assessed. Subgroups were analyzed by the International Staging System (ISS) disease stage at diagnosis, stem cell transplant eligibility and timing, and practice type. Exploratory prognostic models evaluated the association between baseline covariates and time‐to‐event outcomes. The proportion of patients receiving triplet therapies increased from 2011 (36%) to 2019 (72%) as those receiving initial monotherapy or doublet therapy decreased. Overall, the most prevalent triplet regimen consisted of an immunomodulatory drug (IMiD), a proteasome inhibitor, and a steroid. From 2017 to 2019, median TTNT from front‐line to second‐line was longer in patients with ISS stage I versus stages II/III, and in those receiving IMiD‐containing doublet or triplet therapies versus other combinations. Overall median OS was 56 months and increased from 2011 to 2014, after which median OS was not yet reached. Age, ISS stage, and high‐risk status were prognostic for both OS and TTNT, while sex, practice type, and ECOG status were prognostic for OS only. [ABSTRACT FROM AUTHOR]

Aim: This study sought to understand the association between liposomal irinotecan dose reductions (DRs) and clinical outcomes among patients with metastatic pancreatic ductal adenocarcinoma. Materials & methods: A retrospective study of adult patients with metastatic pancreatic ductal adenocarcinoma treated with liposomal irinotecan in the Flatiron Health database was conducted to assess treatment and clinical outcomes. Results: DRs occurred in 28.4% of the 320 patients in the study. Patients with DRs had longer overall survival (7.7 [95% CI: 6.2-10.2]) vs 3.6 [3.2-4.1] months) and time to discontinuation (4.2 [3.0-4.9] vs 1.4 [1.0-1.5] months) than patients without DRs. Results were consistent in a validation analysis requiring three cycles of treatment. Conclusion: Liposomal irinotecan DRs were associated with improved clinical outcomes compared with patients without DRs. [ABSTRACT FROM AUTHOR]

Background: Real‐world practice patterns, treatment sequencing, and outcomes in patients with metastatic pancreatic cancer remain unclear. Previous research indicates that the likelihood of patients with metastatic pancreatic cancer receiving or continuing cancer‐directed therapy is low—a phenomenon called nihilism. This retrospective, descriptive analysis examined clinical characteristics, treatment patterns, and outcomes for patients with metastatic pancreatic ductal adenocarcinoma (mPDAC). Methods: Treatment patterns were examined using electronic health records from the Flatiron Health database covering the period from January 1, 2014, to June 30, 2019. Real‐world overall survival [rwOS]) was compared for a subgroup of patients receiving treatment and a matched subgroup not receiving treatment. Results: Of 7666 patients, 5687 (74.2%) received at least one line of systemic therapy. A greater proportion of patients receiving treatment than not receiving treatment had an initial diagnosis of stage IV disease (68.8% vs 61.2%, respectively). Among patients receiving an initial therapy, fewer than half (38.2%; 2174/5687) received second‐line treatment, mostly because they died, and only 34.3% (745/2174) of those receiving second‐line treatment advanced to third‐line treatment. The rwOS for patients receiving at least one line of systemic therapy was 8.1 months versus 2.6 months for matched patients not receiving treatment (hazard ratio, 0.41; 95% confidence interval, 0.38‐0.45; 1470 patients per group). Conclusions: Systemic therapy provided significant clinical benefit for patients who were eligible and chose to receive it, particularly when treatment was consistent with guideline recommendations. The large proportion of patients initiating treatment suggests that nihilism with mPDAC is diminishing. [ABSTRACT FROM AUTHOR]

Background: Central nervous system (CNS) metastasis is common in advanced melanoma patients. New treatment options have improved overall prognosis, but information is lacking for patients with CNS metastases. We investigated treatment patterns and survival outcomes in older melanoma patients with and without CNS metastases. Methods: A retrospective analysis of SEER‐Medicare, a population‐based linked database, was undertaken in patients aged > 65 years with advanced melanoma diagnosed from 2004 to 2011 and followed until 2013. Results: A total of 2522 patients were included. CNS metastases were present in 24.8% of patients at initial metastatic diagnosis; 16.5% developed CNS metastases during follow‐up. Chemotherapy was the most common treatment regardless of CNS metastases. Overall survival (OS) was better for patients without CNS metastases (median, 9.5 months; 95% confidence interval [CI], 8.8‐10.2) vs patients with CNS metastases (3.63 months; 95% CI, 3.4‐3.9). Among patients with CNS metastases, median OS for targeted therapy, immunotherapy, and chemotherapy was 6 (95% CI, 2.5‐9.6), 5.5 (95% CI, 3.8‐7.5), and 4.5 (95% CI, 3.8‐5.4) months, respectively, vs 2.4 (95% CI, 2.1‐2.7) and 2.1 (95% CI, 1.8‐2.7) months for local radiotherapy and no treatment, respectively. Stereotactic radiosurgery demonstrated higher OS vs whole‐brain radiation therapy (median, 4.98 [95% CI, 3.5‐7.5] vs 2.4 [95% CI, 2.1‐2.7] months). Conclusion: Patients with CNS metastases from melanoma remain a population with high unmet medical need despite recent advances in treatment. Systemic treatments (eg, BRAF‐targeted therapy and immunotherapy) and stereotactic radiosurgery demonstrated meaningful but modest improvements in OS. Further explorations of combinations of radiotherapy, BRAF‐targeted therapies, and immunotherapies are needed. [ABSTRACT FROM AUTHOR]

There are questions surrounding the real-world effectiveness of chemotherapeutic treatments for pancreatic ductal adenocarcinoma. This literature review compared the clinical characteristics and outcomes of available real-world evidence (RWE) for liposomal irinotecan in combination with 5-fluorouracil (5-FU) and leucovorin (LV), a treatment regimen indicated for patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) who previously progressed on gemcitabine-based therapy. A targeted literature search was conducted in the PubMed Central® and Embase® databases to identify available RWE regarding patients with mPDAC receiving liposomal irinotecan published within the last 5 years (January 2014–September 2019). Data were extracted for prior lines of therapy, performance status, overall survival (OS), progression-free survival (PFS), duration of exposure, and adverse events. Six studies met inclusion criteria. A comparison of baseline patient characteristics and results with the included evidence reveals a clinically fragile, real-world patient population in terms of age (range: 61–68), prior lines of therapy with 34–61% of patients receiving ⩾2 lines of lines of prior therapy and performance status [49.8–100% of patients with Eastern Cooperative Oncology Group (ECOG) 0–1]. Studies observed wide OS (range: 5.3–9.4 months) and similar PFS (range: 2.3–4.1 months), with two studies measuring duration of exposure (7.3 weeks, 3.1 months). Patients analyzed by RWE studies tended to be older with significant disease progression, poor performance status, and more heavily pretreated compared with the phase III registrational trial (NAPOLI-1). Despite this, patients treated with liposomal irinotecan + 5-FU/LV therapy had similar outcomes as those in NAPOLI-1. [ABSTRACT FROM AUTHOR]

Transfusion dependence (TD) among myelodysplastic syndromes (MDS) patients negatively impacts survival and health-related quality of life. We evaluated cost patterns of MDS care during TD and transfusion independence (TI). MDS patients were identified from a US claims database (2008–2013). TD was defined as ≥2 consecutive 8-week periods with ≥1 claim during each, and no interim 56-day period without transfusion; TI as 8 subsequent transfusion-free weeks; and transfusion frequency as the mean interval between transfusions during the TD period. 13,741 patients were included; 19% were TD and 70% had a mean interval between transfusions of ≤28 days. During a 2-year period, TD patients incurred a mean total cost of $17,815/patient-month; 53% higher for those with ≤28 days ($19,498) vs. >28 days ($12,717) between transfusions. Among patients who achieved TI, mean total cost was $7874/patient-month. For TD-MDS patients, cost increases are proportional to transfusion frequency and achieving TI yields economic benefits. [ABSTRACT FROM AUTHOR]

Background: Few studies have addressed the cost patterns of patients with multiple myeloma (MM) before and after first relapse. This US claims analysis evaluated, from a US health plan perspective, patterns of total direct costs of care from treatment initiation to progression for patients with MM treated with novel agents, using time to next therapy (TTNT) as a proxy measure for progression. Methods: A retrospective study was conducted using a large US claims database, evaluating patients with claims for MM between 2006 and 2013. Patients with claims for stem cell transplant (SCT) were excluded. The analysis focused on patients receiving lenalidomide (LEN) or bortezomib (BORT) based treatment, for whom complete claim history was available through initiation of subsequent treatment. Average patient monthly direct costs were determined, including medical and pharmacy costs, and total cost patterns over quarterly time periods were calculated. Results: The study population comprised 2843 patients with newly diagnosed MM (NDMM) and 1361 with relapsed MM. Total monthly cost for patients with NDMM declined steadily, from $15,734 initially to $5082 at 18+ months after therapy. Upon initiation of second-line therapy, total monthly costs rose to $13,876 and declined to $6446 18 months later. Although NDMM cost levels for individual ordinal months were similar between the LEN and BORT groups, TTNT was longer for LEN-based treatments (37 months). The BORTtreated cohort had higher average monthly total costs for NDMM and for the common time period through 37 months after initiation of therapy ($7534 vs $10,763 for LEN and BORT, respectively). Key limitations of this study, in addition to the lack of mortality and staging information available from claims data, include the definition of TTNT based on change in treatment or a defined gap in therapy prior to retreatment, which may differ from actual time of progression in some patients. Conclusions: For patients with NDMM receiving either LEN- or BORT-based treatment without SCT, followed until TTNT, total direct monthly costs (drug+medical) declined steadily over time. Monthly costs returned to near initial levels when patients began second-line therapy and then followed a similar pattern of decline. Due to the longer TTNT for patients initiated on LEN and the associated longer period of below-average costs, patients initiated with LEN-based treatments had mean monthly total costs4$3200 lower than total costs for patients initiated on BORT during the first 3 years after starting treatment, cumulating to nearly $120,000 in lower costs for patients initiated on LEN. [ABSTRACT FROM AUTHOR]

Cervical cancer (CC) disproportionately affects women based on socioeconomic status and racial/ethnic background. There is limited research in quantifying and visualizing whether substantial geographical disparities in the US exist with respect to CC burden, and especially with respect to recurrent or metastatic CC (r/mCC) disease burden. Identifying regions with higher r/mCC burden may help inform effective healthcare resource allocation and navigating patients to appropriate care. We conducted a retrospective analysis of the 2015–2020 MarketScan® Commercial and Supplemental Medicare claims data; r/mCC burden was estimated as the number of patients initiating r/mCC systemic therapy over CC-diagnosed patients for each of the 410 metropolitan statistical areas (MSAs) considered. We developed a public, web-based tool, the Cervical Cancer Geographical Disease Burden Analyzer (Cervical Cancer Geo-Analyzer, http://www.geo-analyzer.org), that allows users to visualize r/mCC burden across MSAs over multiple years. There was considerable variation in r/mCC burden across MSAs, with a range of 0–83.3%. Burden increased in Boston-Cambridge-Newton, MA (r/mCC to CC ratio: 41% in 2018 to 50% in 2020), and Sacramento-Roseville-Arden-Arcade, CA (33% in 2018 to 50% in 2020). On the other hand, while r/mCC burden remained high, it decreased in Grand Rapids, MI (55% in 2018 to 31% in 2020) and San Francisco-Oakland-Hayward, CA (40% in 2018 to 26% in 2020). There were regions with sparse or no data, suggesting a need for more representative data capture. The Cervical Geo-Analyzer is a tool to visualize areas with high need for CC interventions. It also builds the foundation for further work to understand local risk factors of disease burden, identify populations of interest, characterize health disparities of CC or r/mCC and inform targeted interventions. • There is variation in the geographic distribution of recurrent/metastatic cervical cancer burden. • The online, interactive Cervical Geo-Analyzer identifies regions with high disease burden and could inform interventions. • More studies are needed to clarify local risk factors for disease burden and local health disparities in cervical cancer. [ABSTRACT FROM AUTHOR]

Current treatments for recurrent or metastatic cervical cancer (r/mCC) do not offer satisfactory clinical benefits, with most patients progressing beyond first-line (1L) treatment. With new treatments under investigation, understanding current treatment patterns, the impact of newly approved therapies, and total costs of care for r/mCC are important. A retrospective analysis of a US commercial insurance claims database to identify adult patients with r/mCC between 2015 and Q1–2020; defining 1L treatment as the first administration of systemic treatment without concomitant chemoradiation or surgery. Patient characteristics, treatment regimens, duration of therapy, and total costs of care were evaluated for each line of therapy. 1323 women initiated 1L treatment for r/mCC (mean age, 56.1 years; mean follow-up, 16.5 months). One-third (n = 438) had evidence of second-line (2L) treatment; of these, 129 (29%) had evidence of third-line (3L) treatment. No regimen represented a majority among 2L+ treatments. The 2018 approval of pembrolizumab led to increased 2L immunotherapy use (0% in 2015, 37% in 2019/Q1–2020). However, only a small proportion of patients stayed on immunotherapy for a prolonged period. Mean per-patient-per-month total costs of care during treatment were $47,387 (1L), $77,661 (2L), and $53,609 (3L), driven primarily by outpatient costs. No clear standard of care was observed in 2L+. Although immunotherapy is increasingly used in 2L+, only a small subset of patients stayed on immunotherapy for a prolonged period, suggesting a need for more therapeutic options. Better understanding of disease biology and the introduction of new therapies may address these unmet needs. • There is no clear standard of care observed in 2L+ r/mCC. • Immunotherapy use in 2L increased from 0% in 2015 to 37% in Q1 2020. • However, only a small proportion stayed on immunotherapy for a prolonged period. • Average costs per patient per month across lines ranged from $47,000 to $77,000. • Therapeutic options with differentiated modalities are needed for 2L+ patients. [ABSTRACT FROM AUTHOR]