Retrospective Real-world Data Research Articles

Examining the treatment journey of patients diagnosed with hepatocellular carcinoma (HCC) using retrospective real-world data in British Columbia, Canada.

e16111 Background: Hepatocellular carcinoma (HCC) is a complex disease with treatments that may include locoregional therapy (LRT), systemic therapy and liver transplant. Using retrospective real-world data from a tertiary care hospital in British Columbia (BC). We examined characteristics, treatment pathways, survival and relevant healthcare costs of HCC patients. Methods: This was a retrospective, real-world data study using data from two existing chart review studies (called MOTION and HCC) and the BC Transplant database. The Canadian province of British Columbia has a population of 5.0 million who receive medical coverage under the Medical Services Plan. “MOTION” collected data on patients who received LRTs between 2004 and 2017, while “HCC” collected data on patients who received systemic therapies between 2000 and 2019. MOTION and HCC and Transplant data were linked and patients were assigned a unique study number and personal health information (PHI) was removed. Start and stop dates for LRT and systemic therapies were used to determine treatment patterns. Treatment costs were provided by BC Cancer’s Pharmacy department, medical oncology visits were assumed to be monthly while patients were on systemic treatment, LRT costs were based on departmental expenditures, and the cost of a liver transplant was provided by the regional health authority. Analyses used descriptive statistics for baseline characteristics, duration and costs (in 2021 Canadian dollars) and Kaplan-Meier curves for survival. Results: There were 417 and 413 patients from the HCC and MOTION databases respectively, in which 63 patients were in both (referred to as the “common” group). Median age (years) at diagnosis was 64, 62 and 62 for HCC, MOTION and common groups respectively, while the proportion who were female was 17.5%, 18.2% and 15.9%. Median overall survival (mOS) from diagnosis was 21, 33 and 28 months for the HCC, MOTION and common groups, respectively. From start of systemic treatment, mOS was 9 months for the HCC group, 29 months from first LRT for patients in the MOTION group, and 26 months for the common group from first treatment. Treatment and cost results were based only on the common group: mean length of follow-up from diagnosis was 34.6 ± 26.2 months and mean time from diagnosis to first treatment was 4.7 ± 4.2 months. DEB-TACE was most frequently the first LRT, often received 3 rounds of LRT before starting systemic treatment, in which sorafenib was given mostly as first line systemic therapy. When including only costs related to LRTs, systemic therapies, medical oncology visits and liver transplants, the mean cost per patient was $94,419, resulting in $51,649 mean cost per patient per year. Conclusions: The linkage of HCC-related LRT, systemic and transplant databases provided detailed results related to the treatment patterns, survival and costs of HCC patients in Canada.

AI-based radiomic biomarkers to predict PD-(L)1 immune checkpoint inhibitor response within PD-L1 high/low/negative expression categories in stage IV NSCLC.

1517 Background: Recent efforts exploring the utility of quantitative imaging (radiomic) biomarkers to predict response to immune checkpoint inhibitors (ICI) have shown promise to provide a more accurate and scalable method than biopsy-based PD-L1 IHC and tumor mutation burden. One of the challenges of evaluating ICI biomarkers on retrospective real-world data is the inherent association between a physician’s treatment choice and PD-L1 expression status. This work investigates radiomics-based multi-modal biomarkers within patient cohorts receiving first-line therapy (ICI monotherapy, ICI plus chemotherapy, and chemotherapy), and in cohorts of all patients receiving ICI (all-lines) subdivided by PD-L1 expression. Methods: Using a large multi-institutional real-world dataset, we analyzed radiomic characteristics of 6,295 primary and metastatic lesions from 1,206 stage IV NSCLC patients treated with PD-(L)1 ICIs from nine institutions across the US and Europe. Patients with unavailable imaging follow-up or with EGFR/ALK oncogenic driver mutations were excluded from analysis, resulting in a total dataset of 791 subjects randomly assigned to training (N = 541) and validation sets (N = 250). Radiomic data was extracted from baseline CT scans capturing tumor heterogeneity, spicularity, and burden in the lung, lymph nodes, and liver. A multi-modal ensemble classifier combining demographic features, PD-L1 TPS, and radiomic data was developed to predict response to ICI therapy per RECIST 1.1 criteria. The model's performance was evaluated in terms of the area under the receiver operating characteristic curve (ROC-AUC) and compared to PD-L1 IHC using the two-tailed DeLong test. Results: In first-line cohorts, the model identified responders with an ROC-AUC of 0.83 (0.72-0.94, P = 0.005; N = 80, 81% PD-L1 > = 50%) in ICI monotherapy, 0.65 (0.47-0.83, P = 0.28; N = 78, 71% PD-L1 < 50%) in ICI plus chemotherapy, and 0.38 (0.002-0.76, P = 0.53; N = 37) in chemotherapy. In all-lines cohorts subdivided into PD-L1 high/low/negative expression categories, the model identified responders with an ROC-AUC of 0.72 (0.57-0.87, P = 0.005, N = 102), 0.74 (0.58-0.89, P = 0.003, N = 71), and 0.60 (0.46-0.74, P = 0.16, N = 77), respectively. Conclusions: Our study demonstrates that multi-modal models can predict ICI response with high performance. The stronger performance in patients receiving ICI therapy alone indicates the model’s predictive, rather than prognostic, power. Furthermore, the model demonstrated good performance in identifying ICI responders within patient sub-cohorts defined by PD-L1 expression status. These insights may be used to inform clinical decision-making, such as escalation or de-escalation of concurrent chemotherapy in stage IV NSCLC patients. In our future work, we will investigate these questions in larger cohorts and prospective studies.

P-287 Retrospective real-world data study using natural language processing to describe treatment sequence and clinicopathological patterns in patients with metastatic colorectal cancer in five Spanish institutions

P-287 Retrospective real-world data study using natural language processing to describe treatment sequence and clinicopathological patterns in patients with metastatic colorectal cancer in five Spanish institutions

Impact of follicular size categories on oocyte quality at trigger day in young and advanced-age patients undergoing GnRH-ant therapy.

To study the effect of follicle sizes of different proportions on oocyte and embryo quality in young and advanced-age patients, and provide evidence for personalized protocol adjustment. This was a retrospective real-world data study including a total of 11,462 patients who had started their first in vitro fertilization cycle with a gonadotropin-releasing hormone antagonist (GnRH-ant) protocol during 2018-2021. We classified patients into groups according to the size of the dominant proportion of follicles on the human chorionic gonadotropin (hCG) trigger day: Large, Medium, Small, and Equal (containing equivalent proportions of all three size categories). The Cochran-Mantel-Haenszel test by different Anti-Mullerian Hormone (AMH) and antral follicle count (AFC) was used to compare factors such as the metaphase II (MII) oocyte rate, normal fertilization rate, and two pronuclei (2PN) cleavage rate between groups. General linear model (GLM) analysis was performed for inter-group comparison of the oocyte and embryo quality. In patients aged < 35 years and with AMH ≥ 1.2μg/L, the MII oocyte percentages in the Large and Medium groups were significantly higher than in the Small group (P < 0.001). The germinal vesicle (GV) oocyte and unavailable oocyte percentages in the Large and Medium groups were lower than in the Small group (P < 0.001). Among patients aged ≥ 35 years with AFC < 5 and AMH ≥ 1.2μg/L, the GV oocyte percentage in the Large group was significantly lower than in the Medium group (2.54% vs. 4.46%, P < 0.001). In patients < 35 years, the GLM demonstrated that the Large and Medium groups had positively impacted on the development of MII oocyte and live birth rate(LBR) of first embryo transfer(ET)(β>0, all P value < 0.05);and had less likely to develop into unavailable oocyte, degenerated oocyte, GV oocyte and MI oocyte rates relative to the Small group(β<0, all P value < 0.05). And among patients ≥ 35 years, the Medium group had positively impacted on the development of MII oocyte and 2PN rates relative to the Small group(β>0, all P value < 0.05); and had less likely to develop into MI oocytes relative to the Small group(β<0, all P value < 0.05). The GLM indicated that AMH, along with Gn total dose, start dose, and Gn days, had significant impact on oocyte and embryo quality. For young patients, age was not a significant influencing factor, but for advanced-age patients, age influenced the outcomes. Our analysis suggests that for young patients (< 35 years), triggering when there is a high proportion of large or medium follicles results in better quality oocytes, while for older patients (≥ 35 years), it is better to trigger when the proportion of medium follicles is no less than that of small follicles. Further research is required to confirm these findings.

Clinical features of polymyalgia rheumatica patients in Japan: Analysis of real-world data from 2015 to 2020.

To assess clinical features in patients with polymyalgia rheumatica (PMR) in Japan by the International Classification of Disease (ICD)-10 code assignment. Demographics, treatment patterns, and concomitant diseases (identified using ICD-10 code only) in patients who were assigned the PMR ICD-10 code M35.3 at least once between 1 January 2015 and 31 December 2020 were aggregated from a nationwide medical information database owned by the Health, Clinic, and Education Information Evaluation Institute. The cumulative number of patients with PMR was 6325 (mean [standard deviation] age, 74.3 [11.4] years; male:female, 1:1.3). Most patients were >50 years (96.5%) with >33% between 70 and 79 years. Glucocorticoids were prescribed in ∼54% of patients within 30 days of PMR code assignment. All other drug types were prescribed in <5% of patients. Hypertension, diabetes mellitus, rheumatoid arthritis, and osteoporosis were noted in >25% and giant cell arteritis in 1% of patients. During the study period, 4075 patients were newly assigned the PMR code and 62% were prescribed glucocorticoids within 30 days. This is the first retrospective real-world data analysis describing the clinical features of PMR in a large patient population from Japan. Further studies of prevalence, incidence, and clinical features are warranted in patients with PMR.

70P Prognostic factors and outcome in ovarian adult granulosa-cell tumours: A retrospective real-world data

70P Prognostic factors and outcome in ovarian adult granulosa-cell tumours: A retrospective real-world data

Clinical benefit of granulocyte-colony stimulating factor (GCSF) use during chemoimmunotherapy treatment for metastatic pancreatic adenocarcinoma (mPDAC).

757 Background: GCSF is used for primary/secondary prophylaxis of chemotherapy(chemo)-associated neutropenia in patients (pts) with mPDAC. GCSF may also increase the populations of healthy, naïve immune cells in an otherwise immunologically dysregulated and cold environment, potentially augmenting therapy outcome with immunomodulatory (IO) agents. Here, we describe the impact of GCSF administration on OS, PFS, and time on treatment (TOT) in the setting of mPDAC for (1) a trial in which pts were administered chemo-IO combinations and (2) a synthetic control arm using retrospective real-world data from pts who received standard-of-care (SOC) chemo (PASCAL). Methods: PRINCE is a ph1b/2 study evaluating gemcitabine (gem) and nab-paclitaxel (NP) ± sotigalimab (sotiga; CD40 agonist) ± nivolumab (nivo; anti-PD1) for pts with mPDAC (NCT0324250), where prophylactic GCSF use was prohibited. PASCAL pts received SOC gem/NP and primary/secondary GCSF use was allowed. In this retrospective analysis, GCSF use was defined as receiving at least 1 dose of GCSF anytime during treatment. OS, PFS, and TOT and associated HRs and CIs were calculated using Kaplan-Meier and Cox methods. PFS data not available for PASCAL. Results: 32/123 (26%) and 16/68 (24%) pts received GCSF in PRINCE and PASCAL, with 84% and 88% of pts receiving at least 1 dose within the first 3 cycles, respectively. In PRINCE, GCSF use was associated with significant improvements in OS (HR [95% CI]: 0.62 [0.40-0.97]), PFS (0.71 [0.47-1.08]), and TOT (0.67 [0.45-1.01]). These improvements were most notable in the sotiga-containing arms (table). In the absence of IO treatment, however, no statistical significance was observed in PASCAL (HR [95% CI]: OS = 0.81 [0.44-1.51]; TOT = 0.90 [0.51-1.58]). Additional work is ongoing to understand the association of GCSF usage with known prognostic factors. Conclusions: These analyses suggest that GCSF use may enhance the clinical benefits of chemo-IO in mPDAC. These potential benefits of GCSF usage warrant further evaluation in other chemo-IO trials as well as prospective evaluation in pre-clinical and clinical settings. Clinical trial information: NCT03214250 . [Table: see text]

Glaucoma progression in patients receiving intravitreal anti-VEGF treatment for neovascular age-related macular degeneration.

The purpose of this study was to investigate how often glaucoma and neovascular age-related macular degeneration (nAMD) occur in the same patient and to evaluate whether glaucoma progression is faster in eyes treated with intravitreal anti-VEGF medications for nAMD. This single-centre retrospective real-world data (RWD) consists of medical records of 6314 glaucoma and 2166 nAMD patients treated in 2008-2017 in Tays Eye Centre, Finland. To study glaucoma progression, changes in visual fields (mean deviation [MD], dB/year), IOP (mmHg/year) and fundus photographs (progression, yes/no) were compared in glaucoma eyes with and without anti-VEGF treatment for nAMD and ≥1year follow-up. During the 10-year period, 147 patients with glaucoma received intravitreal anti-VEGF treatment for nAMD corresponding to 2% of glaucoma and 7% of nAMD patients. The mean change in MD was -0.70 dB/year (SD 1.8) vs. -0.27 dB/year (SD 1.7) (p= 0.027) in glaucoma eyes with (n= 37) and without (n= 4304) anti-VEGF injections, respectively. In patients with bilateral glaucoma and unilateral nAMD treated with anti-VEGF injections (n= 20), MD declined at -0.62 dB/year (SD 1.9) vs 0.33 dB/year (SD 1.5) (p= 0.654), and glaucoma progression was detected in 14/20 vs 10/20 (p= 0.219) fundus photographs in eyes with anti-VEGF treatment compared with their untreated fellow eyes. nAMD and glaucoma were found co-existing in the same eye at rates that were similar to the age-corrected prevalence of the two diseases in the general population. Our results suggest that intravitreal anti-VEGF treatment for nAMD may accelerate glaucoma progression.

Comparative Effectiveness of Lisocabtagene Maraleucel (Liso-cel) in PILOT in Patients (Pt) with Relapsed or Refractory (R/R) Large B-Cell Lymphoma (LBCL) for Whom Transplant Was Not Intended (TNI) Versus Conventional Second-Line (2L) Chemotherapy Regimens in the Real World

Background: Pts with LBCL that is refractory to or has relapsed after 1L therapy and who are not intended for HSCT have poor outcomes and limited treatment options. Liso-cel is an autologous CD19-directed CAR T cell product administered at equal target doses of CD8+ and CD4+ CAR+ T cells. In PILOT (NCT03483103), an open-label, single-arm, multicenter, phase 2 study in TNI pts with 2L R/R LBCL, liso-cel demonstrated high response rates and durable responses, with no new safety signals identified in 2L (Sehgal et al. Lancet Oncol 2022). In the current study, we assessed the comparative effectiveness of liso-cel in PILOT versus an external control cohort of pts with R/R LBCL who received conventional 2L chemotherapy regimens in the real-world clinical setting. Methods: PILOT enrolled pts with R/R LBCL at any time after only 1L of chemoimmunotherapy containing an anthracycline and a CD20-targeted agent and with confirmed PET-positive disease, who were considered TNI by their physician, and who met ≥ 1 prespecified TNI criterion. The primary endpoint for PILOT was ORR. Secondary endpoints included CR rate, duration of response (DOR), PFS, event-free survival (EFS), and OS. The real-world external control cohort was derived from a global, noninterventional, retrospective, observational study using multiple sources (COTA, Guardian Network, and clinical sites via electronic case report forms) representing pts with R/R LBCL across academic and community clinical settings in the US, Europe, and Japan. In the real world, intention to receive HSCT or not was not documented and was substituted with receipt of HSCT in this analysis. The real-world qualifying comparator cohort (QCC) included pts with R/R LBCL who received 1L treatment containing an anthracycline and CD20-targeted agent and any 2L treatment except HSCT and met PILOT eligibility criteria. The primary endpoint for this study was ORR in the PILOT cohort versus the QCC. EFS was defined in this study as time from index date to first documentation of disease progression/relapse, start of new anticancer therapy, or any-cause death, whichever occurred first. The index and data cut-off dates, respectively, were the day of liso-cel infusion and 09/24/2021 for the PILOT cohort and the start of 2L therapy and 12/31/2020 for the QCC. The study period was Jul 2018 to Sept 2021. Endpoint analyses were performed with adjustment using the propensity score methodology of trimmed stabilized inverse probability of treatment weighting to balance pts in the QCC to the PILOT cohort according to baseline characteristics. Highly prognostic baseline characteristics (based on literature and medical review) with ≤ 30% missing values in both PILOT and the real-world cohort were included in balancing: age, sex, years from initial diagnosis to index date, ECOG performance status, Ann Arbor disease stage, refractory versus relapsed, duration of CR after 1L therapy, and bulky disease. All tests were conducted assuming a 2-tailed test of significance and alpha level set a priori at 0.05, with no adjustment for multiplicity. Results: In the PILOT cohort (n = 61) and QCC (n = 273), respectively, median (range) age was 74 (53‒84)/74 (21‒93) years, 79%/77% of pts were aged ≥ 70 years, 26%/24% had ECOG performance status of 2, 23%/8% had CrCl (Cockcroft and Gault) < 60 mL/min, 3%/2% had left ventricular ejection fraction < 50%, 2%/5% had aspartate aminotransferase > 2 × upper limit of normal (ULN), 0%/3% had alanine aminotransferase > 2 × ULN, and 66%/53% had Stage III or IV disease. In the QCC, the most common conventional 2L chemotherapy regimens were rituximab plus ifosfamide, carboplatin, and etoposide (R-ICE; 15%), bendamustine plus rituximab (12%), and rituximab plus gemcitabine and oxaliplatin (11%). Pts who received R-ICE might have been intended to proceed to HSCT but did not because of lack of response to R-ICE; however, intention to receive HSCT cannot be verified based on retrospective real-world data. There was a statistically significant difference in adjusted ORR, CR rate, median DOR, EFS, PFS, and OS in favor of the PILOT cohort versus the QCC (Table). Conclusions: In pts with R/R LBCL who met prespecified TNI criteria, the difference in efficacy outcomes with liso-cel in PILOT versus conventional 2L chemotherapy regimens in the real world was statistically significant in favor of liso-cel, further supporting liso-cel as a new 2L therapy for pts with R/R LBCL who are not intended for HSCT. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

A Retrospective Real-World Evidence Evaluation of the Characteristics of Exocrine Pancreatic Insufficiency in Patients With Chronic Pancreatitis and Type 2 Diabetes Treated With Pancrelipase in the United States.

This retrospective real-world data analysis assessed clinical/health care professional characteristics of gastrointestinal symptom profiles in pancrelipase-treated patients with exocrine pancreatic insufficiency symptoms and chronic pancreatitis (CP) or type 2 diabetes (T2D). Data were from the Decision Resources Group Real-World Evidence Data Repository US database. Patients 18 years and older receiving pancrelipase (Zenpep) between index dates August 2015 and June 2020 were included. Gastrointestinal symptoms were assessed 6, 12, and 18 months post-index versus baseline. A total of 10,656 pancrelipase-treated patients with CP (n = 3215) or T2D (n = 7441) were identified. Significant/sustained reductions in gastrointestinal symptoms were observed in both cohorts after pancrelipase treatment (P < 0.001) versus baseline. Significantly fewer patients with CP compliant with treatment for more than 270 days (n = 1553) reported abdominal pain (P < 0.001) and nausea/vomiting (P < 0.05) versus those compliant for less than 90 days (n = 1115). Significantly fewer patients with T2D compliant with treatment for more than 270 days (n = 2964) reported abdominal pain (P < 0.001) and diarrhea/steatorrhea (P < 0.05) versus those compliant for less than 90 days (n = 2959). Pancrelipase reduced exocrine pancreatic insufficiency symptoms in patients with CP or T2D, with greater treatment compliance associated with improved gastrointestinal symptom profiles.

Comparison of adjuvant gemcitabine plus S-1 with S-1 monotherapy for pancreatic ductal adenocarcinoma: Retrospective real-world data

BackgroundS-1 has been recognized as one of the standard adjuvant chemotherapies for pancreatic ductal adenocarcinoma (PDAC) in East Asia, but the optimal adjuvant chemotherapy regimen has not been determined. We aimed to compare the efficacy and safety of adjuvant gemcitabine plus S-1 (GS) with S-1 monotherapy for PDAC. MethodsPatients with resected PDAC who received adjuvant GS or S-1 chemotherapy in Peking Union Medical College Hospital between May 2014 and May 2022 were reviewed. Data retrieved from medical records were used to evaluate efficacy and toxicity. ResultsA total of 241 patients were included, with 167 receiving GS and 74 receiving S-1. The patients who received GS were generally younger (median [range] age: 62 [36-78] versus 64 [44-87] years, p = 0.004), but chemotherapy began later (median [range] interval between chemotherapy and surgery: 49 [17-125] versus 40 [16-100] days, p < 0.001). The median disease-free survival (DFS, 15.1 versus 15.9 months, p = 0.52) and overall survival (OS, 34.8 versus 27.1 months, p = 0.34) did not differ significantly between the GS and S-1 groups, even after adjustment for the biases. However, the chemotherapy completion rate was higher in the patients treated with S-1 (52.4% versus 75.7%, p = 0.006), while grade 3-4 neutropenia occurred more frequently in the GS group (49.5% versus 18.2%, p = 0.015). ConclusionsAdjuvant S-1 monotherapy demonstrated noninferiority to the GS regimen in DFS and OS with better tolerability for PDAC following surgery.

A Retrospective Real-World Study of the Effectiveness and Tolerability of Tildrakizumab in UK Adults with Moderate-to-Severe Chronic Plaque Psoriasis.

IntroductionAs with most medicines historically, clinicians prescribing tildrakizumab have relied on information derived from registration studies undertaken in a prospective controlled clinical trial setting. More recently, clinicians, policymakers, and commissioners increasingly rely on real-world data to inform both policy and practice.MethodsA retrospective real-world data study was undertaken at four specialist dermatology departments in the United Kingdom. All adult patients treated with tildrakizumab for moderate-to-severe plaque psoriasis were included, with data being collected for 122 patients.ResultsPsoriatic patients on tildrakizumab tended to be overweight (median body mass index of 32 (range 19–59) (n = 61); 26/68 (38%) < 90 kg, 32/68 (47%) between 90 and 120 kg, and 10/68 (15%) > 120 kg). The study population had high levels of comorbidities (83/116, 72%), multiple special sites (39/117, 33%), and histories of biological treatments (81/100, 81%). Most patients (61/80, 76%) initiated on tildrakizumab were switched from another biological treatment. Tildrakizumab was effective, with 91/122 (75%) patients remaining on treatment for the duration of the study—a median of 12 months per patient (range 1–29 months)—and achieving a change in median Psoriasis Area and Severity Index (PASI) from 12 to 0.35 and in Dermatology Life Quality Index (DLQI) from 20 to 0. The response rate was 57/66 (86%) when tildrakizumab was used as the first- or second-line biologic compared to 19/31 (61%) when used as the third- to seventh-line. Thirty-three (78.6%) patients over 90 kg of weight received the 200-mg dose of tildrakizumab. All but one (n = 8) patient with body weight over 120 kg maintained response over time. There was one treatment discontinuation; a patient who had a local sensitivity reaction.ConclusionsIn UK clinical practice, tildrakizumab was well tolerated and effective at doses of 100 mg or 200 mg in a range of patient phenotypes.

Treatment and comorbidities of patients with erectile dysfunction before and during COVID-19 in the United States: A real-world data analysis

IntroductionErectile dysfunction (ED) is usually a symptom of another medical condition. People with poor overall health are more likely to develop severe form of coronavirus disease 2019 (COVID-19) and may have unwanted symptoms such as ED. COVID-19-related stress, anxiety, and depression can also impact sexual health and possibly lead to ED. There are various treatment options for patients with ED. However, real-world evidence of the impact of COVID-19 on the treatment and comorbidities of patients with ED is limited.ObjectivesTo estimate the impact of COVID-19 on the treatment and comorbidities of patients with ED in the United States.MethodsThis retrospective real-world data (RWD) analysis used the IQVIA PharMetrics® Plus claims database, which is a patient-Centric, closed claims database of fully adjudicated pharmacy, hospital and medical claims anonymized at the patient level, that captures the complete patient journey for all services billed to and covered by the patient's health plan. We compared the ED treatment and comorbidities in adult males aged 18+ years with ED in the same 3-month periods before (01March - 31May, 2019) and during (01March – 31May, 2020) the COVID-19 pandemic. ED treatment included any ED-related claims, prescriptions of any and individual phosphodiesterase-5 inhibitor (PDE5i) medications (avanafil, sildenafil, tadalafil and vardenafil).ResultsSee the table below for number of patients having ED-related claims, having any and individual PDE5i prescriptions, number of patients with COVID-19 diagnosis, and percentage of patients with each of the top 10 comorbidities, in the 3-month periods before and during COVID-19.ConclusionsRWD showed that ED-related claims significantly decreased during the COVID-19 pandemic, reflecting the pandemic's impact on ED patients’ health. However, the use of PDE5i medications remained stable during the pandemic. Key comorbidities including hypertension, hyperlipidemia, type 2 diabetes mellitus, benign prostatic hyperplasia with lower urinary tract symptoms, testicular hypofunction, and malignant neoplasm of the prostate, during the pandemic, compared with pre-pandemic period, highlighted the impact of the pandemic on ED patients’ health and need for managing their other comorbid chronic diseases.DisclosureWork supported by industry: yes, by Viatris. A consultant, employee (part time or full time) or shareholder is among the authors (Viatris).

Healthcare Resource Utilization and Associated Costs in New Users of Empagliflozin versus DPP-4 Inhibitors and GLP-1 Agonists: A Comparative Analysis Based on Retrospective Real-World Data from German Sickness Funds.

PurposeAchieving good glycemic control in type 2 diabetes (T2DM) may require individualized pharmacological approaches. We aimed to compare direct healthcare costs in patients treated with empagliflozin (EMPA) compared to dipeptidyl peptidase-4 inhibitors (DPP-4i) or glucagon-like peptide-1 receptor agonists (GLP-1-RA).Patients and MethodsThis German claims data study included continuously insured persons with at least two outpatient diagnoses and/or one inpatient diagnosis of T2DM if they started EMPA, DPP-4i, or GLP-1-RA in 2015–2018. Healthcare costs were assessed from therapy initiation until the end of data availability, death, or therapy discontinuation and compared among propensity score-matched cohorts.ResultsOf 24,465 patients included, 3285 received EMPA, 19,443 DPP-4i, and 1747 GLP-1-RA. Matched cohorts were balanced on baseline characteristics (EMPA versus DPP-4i: n1/n2 = 3100/3100 and EMPA versus GLP-1-RA: n3/n4 = 1346/1346). Mean total costs after start of DPP-4i were €7009 (95%-CI: 6573–7444) versus €4274 (3982–4566) for EMPA. Costs associated with GLP-1-RA treatment were also significantly higher compared with EMPA (€6851 [6183–7518] versus €4895 [4345–5445]).ConclusionAlthough the individual clinical patient profile and physician assessment are paramount in treatment decisions, substantial differences in the economic impact of different antidiabetic therapies should be considered.

Healthcare resource utilization (HCRU) and treatment patterns in men with erectile dysfunction (ED) and benign prostatic hyperplasia-associated lower urinary tract symptoms (BPH-LUTS) in the United States (US): A retrospective database study

ABSTRACT Introduction There are various treatment options for patients with ED and comorbid BPH-LUTS. Relevant clinical practice guidelines are available to help and guide physicians in making appropriate disease management decisions. However, real-world evidence comparing health-related outcomes between patients treated with tadalafil and those who did not receive any PDE5i therapy is limited. Objectives To compare HCRU outcomes in patients with ED and BPH-LUTS treated with tadalafil or not treated with any PDE5i in the US and to compare tadalafil treatment persistence and adherence by dose. Methods This was a retrospective real-world data study of men (aged 45+) with ED and BPH-LUTS treated with tadalafil or not treated with any PDE5i, using the IQVIA PharMetrics® Plus claims database. Outcomes included all-cause and disease-specific HCRU over the 12-month follow-up. Disease-specific was defined as claims with a diagnosis of ED or BPH-LUTS, and/or specific therapy or procedures used in the treatment of ED or BPH-LUTS. Persistence (days till discontinuation) and adherence (proportion of days covered, PDC) to tadalafil therapy was also evaluated, stratified by dose over the follow-up (10 or 20 mg as needed; 2.5 or 5 mg as once daily). Pair-wise comparisons were conducted using t-test for continuous variables and Chi-square test for categorical variables. Results The final sample comprised 11,351 tadalafil and 48,722 non-PDE5i cohorts. Mean age was 57.1 and 57.9 years, respectively. All-cause HCRU: Mean number of all-cause laboratory/pathology tests (13.8 vs. 16.4) and proportion with at least one outpatient surgical visit (63.0% vs. 68.8%) or radiology exam (59.3% vs. 63.4%) were significantly lower for tadalafil cohort vs. non-PDE5i cohort (all comparisons, p&amp;lt;0.0001). Disease-specific HCRU: Proportion with at least one disease-specific physician office visit (55.1% vs. 91.4%), laboratory/pathology test (34.8% vs. 58.2%), radiology exam (5.8% vs. 10.8%), outpatient surgery (24.3% vs. 38.9%) or outpatient ancillary service (18.0% vs. 29.8%) were significantly lower for tadalafil cohort vs. non-PDE5i cohort (all comparisons, p&amp;lt;0.0001). Persistence toTadalafil: Mean persistence days (179.8 vs 61.2) and % patients without discontinuation (35.8% vs. 6.5%) were higher for daily tadalafil dose vs. as needed tadalafil dose. Adherence toTadalafil: Mean PDC [90% confidence interval] was higher for daily tadalafil dose vs. as needed tadalafil dose (0.50 [0.495,0.508] vs. 0.20 [0.198,0.211]). Conclusions Patients on tadalafil used less HCRU than patients not on any PDE5i therapy. Persistence and adherence were higher in patients treated with daily tadalafil compared with as needed tadalafil therapy. The results demonstrated tadalafil's benefit in the management of men with ED and BPH-LUTS. Disclosure Work supported by industry: yes, by Upjohn-a Pfizer division. A consultant, employee (part time or full time) or shareholder is among the authors (Viatris).

Efficacy and safety of 177Lu‑DOTATATE in patients with advanced pancreatic neuroendocrine tumours: data from the NETTER-R international, retrospective study

PurposeNETTER-R aimed to determine the efficacy, safety and tolerability of 177Lu-DOTATATE in patients with progressive, advanced pancreatic neuroendocrine tumours (panNETs) using retrospective real-world data from multiple sites.MethodsThis international study retrospectively included patients with panNETs treated with 177Lu-DOTATATE. The primary endpoint was progression-free survival (PFS) by Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1). Secondary endpoints included overall survival (OS), safety and tumour response.ResultsIn total, 110 patients with panNETs were studied; 65.5% received a cumulative dose of 177Lu-DOTATATE 29.6 GBq ± 10% (median: 7.4 GBq). In 62 patients with available RECIST v1.1 tumour response, the median PFS was 24.8 months (95% confidence interval [CI]: 17.5–34.5), and the objective response rate was 40.3% (95% CI: 28.1–53.6); all responses were partial. With a median follow up of 24.5 months (range: 2.0–123.4 months) after the first cycle of 177Lu-DOTATATE, the median OS in the full analysis set (n = 110) was 41.4 months (95% CI: 28.6–50.2). PFS (hazard ratio [HR]: 3.672; p = 0.0009) and OS (HR: 3.360; p < 0.0001) were longer in patients who received no chemotherapy prior to 177Lu-DOTATATE than those who did. No treatment-emergent adverse events (TEAEs) led to treatment discontinuation. Grade 3 anaemia, lymphopenia and thrombocytopenia occurred in 0.9%, 5.4% and 0.9% of patients, respectively. No acute leukaemia or myelodysplastic syndrome was reported. Six patients (5.5%) had renal TEAEs. All renal grade ≥ 3 events were transient and did not lead to treatment modification.ConclusionsThese results reinforce the role of 177Lu-DOTATATE for the treatment of patients with advanced, somatostatin receptor-positive panNETs.

Cutaneous Comorbidities Associated With Atopic Dermatitis in Israel: A Retrospective Real-World Data Analysis.

Patients with atopic dermatitis (AD) are susceptible to infectious and inflammatory cutaneous comorbidities. The aim of the study was to describe the prevalence of cutaneous comorbidities associated with AD, including their relationship with AD severity. A retrospective cross-sectional analysis was performed using the Israeli Maccabi Healthcare Services database. Prevalent AD cases on December 31, 2017, were diagnosed with AD at any time since 1998 and had 1 or more recent (2013-2017) AD diagnoses. Dispensed AD treatments within 5 or fewer years served as a surrogate for AD severity. Cutaneous comorbidities in AD cases were compared with non-AD controls matched 1:1 on age, sex, and residential area. Among adults, comorbidities were compared across AD severity using multinomial logistic regression. The eligible population included 94,483 patients with mild (57.7%), moderate (36.2%), or severe (6.1%) AD, and 94,483 matched non-AD controls. Skin infections, inflammatory skin conditions, cutaneous manifestations of AD, and sweat gland disorders were more prevalent ( P < 0.001) in patients with AD than in controls. Most cutaneous comorbidities that were more prevalent in adult patients with AD were also significantly ( P < 0.001) associated with AD severity. This study suggests that AD is associated with many infectious and inflammatory cutaneous comorbidities and highlights the relationship between AD severity and comorbidity prevalence.

Treatment of patients with newly diagnosed metastatic hormone sensitive prostate cancer (mHSPC) in Belgium: a real world data analysis

ABSTRACT INTRODUCTION Abiraterone acetate + prednisone (AAP) and docetaxel have proven their efficacy in the treatment of patients with newly diagnosed metastatic hormone-sensitive prostate cancer (mHSPC) in clinical trials. However, real-world data are scarce. The goal of this study is to evaluate real-world data on the efficacy and safety of these therapies in mHSPC patients. PATIENTS AND METHODS Records of 93 patients from 21 different centres were retrospectively reviewed. Primary and secondary endpoints were radiographic and PSA progression-free survival (RPFS – PSA-PFS) and cancer specific and overall survival (CSS – OS), respectively. Adverse events (AEs) were evaluated according to the Common Terminology Criteria for Adverse Events version 5.0. Differences in oncological outcome and AEs were evaluated between three treatment groups: ADT only (N=26) – ADT + AAP (N=48) – ADT + docetaxel (N=19). Survival analysis was performed using Kaplan–Meier statistics. RESULTS Median RPFS was 13 months (95% confidence interval [CI]: 9–17) for ADT only, 21 months (95% CI: 19–23) for ADT + AAP and 12 months (95% CI: 11–14) for ADT + docetaxel (p = 0.004). The 1-year PSA-PFS, CSS and OS were 73.5%, 90.7% and 88.7%, respectively, with no significant differences between the three groups. Adverse events of grade 3 or higher were not observed more frequently. CONCLUSION Retrospective real-world data show a significantly longer RPFS for mHSPC patients treated with ADT + AAP compared to ADT only or ADT + docetaxel at short-term follow-up. This can aid in counselling of mHSPC patients in daily clinical practice.

Anaplastic lymphoma kinase rearrangement prevalence in patients with advanced non-small cell lung cancer in the United States: retrospective real world data.

Objective: This study assessed the prevalence of anaplastic lymphoma kinase (ALK) rearrangements in US oncology practices.Materials and Methods: Using a nationwide real-world database, we included adults with advanced non-small cell lung cancer (aNSCLC, stage IIIB- IV) diagnosed January 2015 – May 2019, with documented ALK testing results and smoking status. Rearrangement prevalence was assessed overall and then stratified by patient characteristics.Results: The cohort included 19,895 eligible patients with a mean age 68.5 years, majority ever-smokers (85.5%) and from community centers (92.2%). The overall ALK rearrangement prevalence was 2.6%. Positivity rate varied by histology and smoking status; it was the highest among non-smoking patients with non-squamous histology (9.3%). Differences in ALK status also varied by age and race, with young patients (18–39 years) having a higher prevalence (21.6%) vs. older patients (age ≥55 = 2.2%); Asian patients had a prevalence of 6.3%. Patients that were positive for other mutations or rearrangements had a lower ALK positivity rate (0.5%) and patients positive for PD-L1 had a rate of 3.0%.Conclusions: The likelihood of finding an ALK translocation was highest in younger patients and nonsmokers; however, age and smoking history were not discriminative enough to exclude testing based on clinical variables.

Tafasitamab Plus Lenalidomide Versus Pola-BR, R2, and CAR T: Comparing Outcomes from RE-MIND2, an Observational, Retrospective Cohort Study in Relapsed/Refractory Diffuse Large B-Cell Lymphoma

Tafasitamab Plus Lenalidomide Versus Pola-BR, R2, and CAR T: Comparing Outcomes from RE-MIND2, an Observational, Retrospective Cohort Study in Relapsed/Refractory Diffuse Large B-Cell Lymphoma