Real-world Patient Characteristics Research Articles

569P Mismatch repair deficient, stage II/III rectal cancer: Real-world patient, tumour, and treatment characteristics in the Netherlands

569P Mismatch repair deficient, stage II/III rectal cancer: Real-world patient, tumour, and treatment characteristics in the Netherlands

OA01.07 Real-World Patient Characteristics, Biomarker Testing, and Adjuvant Therapy for Resected NSCLC at U.S. Oncology Clinics, 2019-2023

OA01.07 Real-World Patient Characteristics, Biomarker Testing, and Adjuvant Therapy for Resected NSCLC at U.S. Oncology Clinics, 2019-2023

Real-world characteristics of patients with asthma initiating fluticasone furoate/umeclidinium/vilanterol single-inhaler triple therapy in Japan

BackgroundReal-world data assessing characteristics of patients with asthma initiating inhaled corticosteroid/long-acting muscarinic antagonist/long-acting β2-agonist (ICS/LAMA/LABA) triple therapy in Japan are limited. MethodsDescriptive, observational study of patients with asthma aged ≥15 years newly initiating single- or multiple-inhaler triple therapy (SITT: fluticasone furoate/umeclidinium/vilanterol [FF/UMEC/VI], SITT: indacaterol/glycopyrronium bromide/mometasone furoate [IND/GLY/MF] or MITT) or ICS/LABA using JMDC/Medical Data Vision (MDV) health insurance databases from February 2021–February 2022 (first prescription date: index date). Patients were assigned to three non-mutually exclusive cohorts: A) new FF/UMEC/VI initiators; B) new FF/UMEC/VI, IND/GLY/MF, or MITT initiators; C) new FF/UMEC/VI, IND/GLY/MF, MITT or ICS/LABA initiators as initial maintenance therapy (IMT). Patient characteristics were assessed descriptively for 12-months pre-treatment initiation (baseline period). ResultsCohort A: among new FF/UMEC/VI initiators, 12.8% and 0.1% (JMDC) and 21.7% and 0.9% (MDV) of patients had ≥1 moderate and severe exacerbation; 52.0% (JMDC) and 79.2% (MDV) had ICS/LABA use. Cohort B: most patients initiated FF/UMEC/VI and IND/GLY/MF over MITT (JMDC: 91.3% vs 8.7%; MDV: 67.8% vs 32.2%), with fewer exacerbations and lower rescue medication use. Cohort C: a greater proportion of FF/UMEC/VI initiators as IMT experienced a moderate exacerbation at index versus ICS/LABA initiators as IMT (JMDC: 17.8% vs 10.7%; MDV: 8.0% vs 5.1%). ConclusionsPatient characteristics were generally similar between treatment groups; SITT initiators had fewer exacerbations and lower rescue medication use than MITT initiators, represented by the greater proportion of IMT among SITT versus MITT initiators. Physicians may have prescribed triple over dual therapy as IMT in response to an exacerbation.

Abstract PO2-17-06: Real world treatment patterns and clinical outcomes by germline BRCA (gBRCA) mutation status in patients with HER2-negative early breast cancer in the US community oncology setting: a retrospective observational chart-review study

Abstract Background: Women with early-stage breast cancer (eBC) who have inherited BRCA1 or BRCA2 mutations (gBRCAm) are typically diagnosed at a younger age and often require more intensive treatment. In the randomized, double-blind OlympiA trial of HER2-negative (HER2–) gBRCAm patients, olaparib, a targeted poly (ADP-ribose) polymerase inhibitor (PARPi), significantly improved invasive disease free survival (IDFS), distant disease free survival (DDFS) and overall survival (OS) and was subsequently approved for adjuvant treatment of gBRCAm HER2– high-risk eBC patients. This study examined the real-world patient characteristics, treatment patterns and clinical outcomes by gBRCA status among patients with HER2– eBC in a US community oncology setting prior to olaparib US approval for treatment of eBC. Methods: This retrospective observational study used chart review data from The US Oncology Network's iKnowMed database to examine adult patients diagnosed with HER2– eBC (stage I-III) initiating systemic neoadjuvant or adjuvant therapy with chemotherapy and/or endocrine therapy between January 1, 2012 and December 31, 2018. Patients with valid gBRCAm test results (all gBRCAm and randomly selected BRCAwt patients) were included and followed through December 31, 2021. Patients were excluded if they had HER2+ tumors and progressed to stage IV within 6 months after initiation of neoadjuvant therapy or were diagnosed with another primary cancer. Descriptive analyses assessed patient characteristics. Kaplan Meier methods and multivariate Cox proportional hazard models (CPHM) were used to evaluate IDFS, DDFS and OS by gBRCA status and by HR+/HER2– or TNBC eBC subsets. Results: Among 298 BC patients meeting initial criteria, 42% had gBRCAm (n=124, 43% hormone receptor positive [HR+], 56% triple-negative BC [TNBC], 1% unknown), and 58% had gBRCAwt (n=174, 74% HR+, 22% TNBC). Median (interquartile) age at surgery was numerically lower in gBRCAm (45 [36,55] years) than gBRCAwt (48 [41,55] years) patients. A numerically higher proportion of gBRCAm patients received neoadjuvant therapy compared with gBRCAwt (Neoadj: gBRCAm 37.1% vs gBRCAwt 12.6%) but the reverse was true for adjuvant (Adj: gBRCAm 42.7% vs gBRCAwt 65.5%) and neoadjuvant + adjuvant use was numerically similar (Neoadj+Adj: gBRCAm 20.1% vs gBRCAwt 21.8%). The majority of TNBC patients received neoadjuvant therapy while the majority of HR+ patients received adjuvant therapy (Neoadj: TNBC 51.4% vs HR+ 6.6%, Adj: TNBC 29.0% vs HR+ 71.4%, Neoadj+Adj: TNBC 19.6% vs HR+ 22.0%). Median IDFS, DDFS, and OS in both study cohorts were not reached. Median follow-up from surgery was < 5 years (overall 59.0 months; gBRCAm 50.6 months; gBRCAwt 61.3 months). At 60 months, in gBRCAm and gBRCAwt respectively, estimated IDFS were 85.5% and 90.9%, estimated DDFS 88.1% and 92.2%, and estimated survival 91.9% and 95.2%. At 60 months, in HR+/HER2– and TNBC respectively, estimated IDFS were 92.4% and 81.2%, estimated DDFS 93.9% and 85.7%, and estimated survival 96.3% and 90.9%. CPHM results showed that risks of invasive disease (HR 1.74; 95% CI 0.83-3.64; p=0.15), distant disease (HR 1.24; 95% CI 0.50-3.07; p=0.65) and death (HR 1.79; 95% CI 0.52-6.13; p=0.353) were similar between gBRCAm and gBRCAwt patients. Between HR+/HER2– and TNBC patients, risks of invasive disease (HR=0.63; 95% CI 0.21-1.89; p=0.41), distant disease (HR=0.42; 95% CI 0.11-1.67; p=0.22) and death (HR 0.48; 95% CI 0.09-2.70; p=0.41) were similar. Conclusions: This real-world chart review study of data through 2021 (before PARPi approval for eBC) with limited follow up of 5 years found that gBRCAm patients had a numerically shorter survival (IDFS, DDFS and OS) than gBRCAwt, but this difference was not statistically significantly different. Given that the follow-up period was too short to draw conclusions, further studies examining clinical outcomes over a longer follow-up period after approval of PARPi may be of interest. Citation Format: Jay Andersen, Jagadeswara Earla, Nicole Fulcher, Juliet Ndukum, Nicholas J Robert, Mark Robson, Weiyan Li, Jaime Mejia. Real world treatment patterns and clinical outcomes by germline BRCA (gBRCA) mutation status in patients with HER2-negative early breast cancer in the US community oncology setting: a retrospective observational chart-review study [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO2-17-06.

Real-world utilization, patient characteristics, and treatment patterns among men with localized prostate cancer tested with the 17-gene genomic prostate score® (GPSTM) assay.

Descriptive study focusing on real-world utilization and characteristics of men with prostate cancer tested with the 17-gene Genomic Prostate Score® (GPS™) assay by linking administrative claims and electronic health record (EHR) data with GPS results. This retrospective, observational cohort study (January 1, 2013 to December 31, 2020) included men aged 40-80 years with localized prostate cancer claims, continuous enrollment in Optum's Integrated Claims data set, ≥1 day of EHR clinical activity, and a GPS result. Men were classified as undergoing definitive therapy (DT) (prostatectomy, radiation, or focal therapy) or active surveillance (AS). AS and DT distribution were analyzed across GPS results, National Comprehensive Cancer Network® (NCCN®) risk, and race. Costs were assessed 6 months after the first GPS result (index); clinical outcomes and AS persistence were assessed during the variable follow-up. All variables were analyzed descriptively. Of 834 men, 650 (77.9%) underwent AS and 184 (22.1%) DT. Most men had Quan-Charlson comorbidity scores of 1-2 and a tumor stage of T1c (index). The most common Gleason patterns were 3 + 3 (79.6%) (AS cohort) and 3 + 4 (55.9%) (DT cohort). The mean (standard deviation) GPS results at index were 23.2 (11.3) (AS) and 30.9 (12.9) (DT). AS decreased with increasing GPS result and NCCN risk. Differences between races were minimal. Total costs were substantially higher in the DT cohort. Most men with GPS-tested localized prostate cancer underwent AS, indicating the GPS result can inform clinical management. Decreasing AS with increasing GPS result and NCCN risk suggests the GPS complements NCCN risk stratification.

Progress in methodological research on bridging the efficacy-effectiveness gap of clinical interventions(2): to improve the extrapolation of efficacy

Objective: Randomized controlled trials (RCT) usually have strict implementation criteria. The included subjects' characteristics of the conditions for the intervention implementation are quite different from the actual clinical environment, resulting in discrepancies between the risk-benefit of interventions in actual clinical use and the risk-benefit shown in RCT. Therefore, some methods are needed to enhance the extrapolation of RCT results to evaluate the real effects of drugs in real people and clinical practice settings. Methods: Six databases (PubMed, Embase, Web of Science, CNKI, Wanfang Data, and VIP) were searched up to 31st December 2022 with detailed search strategies. A scoping review method was used to integrate and qualitatively describe the included literature inductively. Results: A total of 12 articles were included. Three methods in the included literature focused on: ①improving the design of traditional RCT to increase population representation; ②combining RCT Data with real-world data (RWD) for analysis;③calibrating RCT results according to real-world patient characteristics. Conclusions: Improving the design of RCT to enhance the population representation can improve the extrapolation of the results of RCT. Combining RCT data with RWD can give full play to the advantages of data from different sources; the results of the RCT were calibrated against real-world population characteristics so that the effects of interventions in real-world patient populations can be predicted.

Analysis of Real-world Characteristics of Patients with CDKL5 Deficiency Disorder Enrolled for Ganaxolone Treatment (P6-1.010)

Analysis of Real-world Characteristics of Patients with CDKL5 Deficiency Disorder Enrolled for Ganaxolone Treatment (P6-1.010)

Real-world patient characteristics and treatment patterns in US patients with advanced non-small cell lung cancer

BackgroundPatients from non-small cell lung cancer (NSCLC) controlled clinical trials do not always reflect real-world heterogeneous patient populations. We designed a study to describe the real-world patient characteristics and treatment patterns of first-line treatment in patients in the US with NSCLC.MethodsThis was an observational, retrospective cohort study based on electronic medical records of US adults with locally advanced or metastatic disease in the ConcertAI Patient360 NSCLC database who initiated first-line treatment with anti-programmed cell death protein 1/programmed cell death ligand 1 (PD-1/PD-L1) therapy between July 2016 and December 2020. The analysis used patient attributes, clinical characteristics, and treatments from each patient’s medical records.ResultsA total of 2175 patients were eligible for analysis. The median age was 68 years, and 26.2% of the patients were ≥75 years old. At treatment initiation, 96.4% and 3.6% of the patients had Stage 4 and Stage 3 (B or C) NSCLC, respectively. The most common histology type was nonsquamous adenocarcinoma (66.4%), and 19.8% had Eastern Cooperative Oncology Group performance status ≥2. Immunosuppressive medications were being used by 17.7% of patients, and 11.0% were immunocompromised. Almost all patients had metastases: 64.6% had 1, 23.2% had 2, and 8.0% had ≥3 metastatic sites. Brain metastases were present in 22.9% of patients. Treatment evolution was observed with first-line standard of care shifting from single-agent immunotherapy in 2016 (90.2%) to combination immunotherapy and chemotherapy in 2020 (60.2%).ConclusionBetween 2016 and 2020, the first-line treatment paradigm for advanced NSCLC in the US shifted from anti–PD-1/PD-L1 monotherapy to combination chemoimmunotherapy, with increasing biomarker testing. Further research in heterogeneous patient populations to characterize treatment strategies is warranted.

Real-world patient characteristics, treatment patterns, and treatment outcomes of patients with diffuse large B-cell lymphoma by line of therapy.

Although initial treatment of diffuse large B-cell lymphoma (DLBCL) with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) can be effective, up to 50% of patients will develop refractory or relapsed (R/R) disease. This study aimed to provide contemporary data on characteristics, treatment patterns, and outcomes for R/R-DLBCL. Patients with incident (January 2016 to March 2021) DLBCL age ≥18 years who initiated first-line (1L) therapy were identified from the COTA real-world database. Baseline characteristics, treatment patterns, and real-world outcomes, including time to next treatment (rwTTNT) and overall survival (rwOS), were assessed for the study population and by line of therapy (LOT). A total of 1347 eligible DLBCL patients were identified. Of these, 340 (25.2%) proceeded to receive 2L, of whom 141 (41.5%) proceeded to receive 3L, of whom 51 (36.2%) proceeded to receive 4L+. Most common treatments were R-CHOP in 1L (63.6%), stem cell transplant (SCT) in 2L (17.9%), polatuzumab vedotin, bendamustine, and rituximab (Pola-BR) in 3L (9.9%), and chimeric antigen receptor T-cell therapy (CAR-T) in 4L (11.8%). Treatment patterns were more variable in later LOTs. One- and 3-year rwOS from 1L initiation were 88.5% and 78.4%, respectively. Patients who received later LOTs experienced numerically lower 1- and 3-year rwOS (from 2L initiation: 62.4% and 46.4%, respectively). In this real-world analysis, 25.2% of patients experienced R/R-DLBCL after 1L with poor outcomes. Given the findings of this study, there is a high unmet need for novel, safe, and effective treatment options for patients with R/R DLBCL.

Mainstream Model of Genetic Testing for Prostate Cancer at a Large Tertiary Cancer Centre

BackgroundAn estimated 20% to 30% of men with advanced prostate cancer carry a mutation in DNA damage repair genes, of which half are estimated to be germline. Eligibility criteria for germline genetic testing expanded significantly for Ontario patients in May 2021 and many centers adopted a “mainstream” model, defined as oncologist-initiated genetic testing. MethodsWe conducted a retrospective chart review to report on the first-year mainstream experience of a large tertiary oncologic center, the Sunnybrook Odette Cancer Centre. All patients who underwent mainstream at the discretion of their treating physician were included. A subset underwent somatic profiling as part of clinical trial screening. Descriptive statistics were used to report baseline clinicopathologic characteristics and treatments received. ResultsBetween May 1, 2021, and May 30, 2022, 174 patients with prostate cancer underwent mainstream germline genetic testing with a 19-gene panel. Median age was 75 (IQR 68-80), and 82% of patients were diagnosed with either de novo metastatic or high-risk localized prostate adenocarcinoma. Fourteen patients (8%; 95% CI 4%-12%) were found to have a deleterious germline mutation, including pathogenic or likely pathogenic variants in BRCA1/2, ATM, CHEK2, PMS2, RAD51C, HOXB13, and BRIP1. Forty-nine patients (28%; 95% CI 21%-35%) were found to have a variant of uncertain significance. Thirty-four patients also had next-generation sequencing (NGS) of their somatic tissue. Among this subset, 8 of 34 (23%) had an alteration in homologous recombination repair (HRR) genes. Of the 14 patients with a germline mutation, none had a prior personal history of malignancy and 6 (43%) did not have any first- or second-degree relatives with history of prostate, pancreatic, breast, or ovarian cancer. ConclusionWe report on the real-world characteristics of prostate cancer patients who underwent mainstream germline genetic testing. Personal history and family history of cancer cannot reliably stratify patients for the presence of pathogenic germline variants.

Real-world treatment patterns and unmet need in patients with previously treated metastatic colorectal cancer that is not MSI-H or dMMR.

53 Background: Regorafenib and trifluridine/tipiracil (TAS-102) are standard of care (SoC) systemic anticancer treatments (SACT) for metastatic colorectal cancer (mCRC) patients perviously treated with fluoropyrimidine (5-FU), irinotecan, & oxaliplatin-based chemotherapy. However, evidence is limited on post-chemotherapy & later SACT for patients with mCRC that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). This study assessed real-world patient characteristics and treatment patterns in community oncology practices among previously treated, not MSI-H/dMMR mCRC patients. Methods: This retrospective cohort study utilized The US Oncology Network (iKnowMed) electronic health record database, supplemented by chart review. The study cohort comprised adult, not MSI-H/dMMR mCRC patients treated with chemotherapy (5-FU/capecitabine, irinotecan, and oxaliplatin) who initiated subsequent SACT or received best supportive care (BSC) between 1/1/2016 & 12/31/2021. Index date for SACT users was the regimen start date. For patients on BSC and no subsequent SACT/BSC, index date was last chemotherapy administration date. Patients were followed from index date through 8/31/2022, date of death or last contact date, whichever occurred first. Descriptive statistics were used to report patient and treatment characteristics. Results: The study consisted of 292 mCRC patients, with a median age of 57 (range: 49, 68) years. The predominant histology was adenocarcinoma (n = 237, 81.2%) with 1.7% other histology and rest missing, 198 (67.8%) patients had Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 and 206 (70.5%) had primary tumors located in the colon. Liver (n = 207, 70.9%) and lung (n = 78, 26.7%) were the most common metastasis sites. Diabetes (n = 33, 11.3%) and peripheral vascular disease (n = 18, 6.2%) were the most common comorbidities. Eighty-seven (29.8%) patients received at least one chemotherapy in the adjuvant/neo-adjuvant setting, while majority (70.2%) patients received chemotherapies in the metastatic setting. Overall, 203 (69.5%) received SACT post-chemotherapy, 84 (28.8%) received BSC and 5 (1.7%) did not receive SACT/BSC. Among the SACT post-chemotherapy users, 30 (14.8%) received regorafenib, 25 (12.3%) received TAS-102 and 148 (72.9%) received other SACT. Other SACT included irinotecan-based (n = 76, 37.4%), oxaliplatin-based (n = 30, 14.8%), irinotecan and oxaliplatin-based (n = 6, 3.0%) chemotherapy, and anti-EGFR (n = 15, 7.4%) or anti-VEGF (n = 8, 3.0%) targeted therapies. Conclusions: Overall, only 27.1% of previously treated mCRC patients with tumors that were not MSI-H or dMMR received SoC. The real-world variability in choice of treatments and the high percentage of patients rechallenged with chemotherapy in subsequent lines, highlights an unmet need in this population.

Real-World Patient Characteristics, Treatment Patterns, and Outcomes Among Patients with Advanced Cutaneous Squamous Cell Carcinoma Treated with Cemiplimab at US Oncology Clinical Practices

Real-World Patient Characteristics, Treatment Patterns, and Outcomes Among Patients with Advanced Cutaneous Squamous Cell Carcinoma Treated with Cemiplimab at US Oncology Clinical Practices

Real-world clinical profile, RET mutation testing, treatments, and PROs for MTC in Europe.

This study aimed to describe real-world patient and physician characteristics, rearranged during transfection (RET) mutation testing and results, treatment patterns, and patient-reported outcomes (PROs) in advanced or metastatic medullary thyroid cancer (aMTC) across five populous European countries. Cross-sectional physician and patient surveys were used to collect quantitative and qualitative data in France, Germany, Italy, Spain, and the UK from July to December 2020, prior to the introduction of selective RET inhibitors in Europe. Physicians completed patient record forms and a survey about their specialty and practice site. Patients were asked to provide PRO data using four validated instruments, including the EuroQol 5 Dimension (EQ-5D) questionnaire. The physician-reported sample included 275 patients with aMTC, including 79 patients with RET mutation-positive disease; median age was 60 and 56 years, respectively. Overall, 75% were tested for RET mutation (35% germline only, 21% somatic only, 44% both). Common physician-cited barriers to RET mutation testing included high cost, difficulty accessing latest tests, and time delay for results. First-line systemic therapy (most commonly vandetanib or cabozantinib) was prescribed for 69% of patients overall and 82% of the RET mutation-positive subgroup. Second-line therapy was prescribed for 12% of patients who received first-line therapy; most patients remained on first-line therapy at data capture. PROs revealed substantial disease/treatment burden. Patients with aMTC report substantial disease/treatment burden. Outcomes could be improved by identifying patients eligible for treatment with selective RET inhibitors through more optimal RET mutation testing.

Clinical signs and symptoms of Wilson disease in a real-world cohort of patients in the United States: a medical chart review study

IntroductionThere are limited data from the United States regarding the real-world signs and symptoms of Wilson disease (WD). This retrospective, observational medical chart review was conducted to identify real-world characteristics of patients with WD in the United States, as well as WD signs and symptoms at diagnosis and over time.MethodsDe-identified clinical data were abstracted from medical charts of US patients diagnosed with WD between January 1, 2012, and June 30, 2017. Hepatic, neurologic, and psychiatric biochemical findings, signs, and symptoms were characterized at diagnosis and follow-up/during treatment.ResultsIn total, 225 WD patients were included in the study. The mean (SD) age at diagnosis was 24.7 (9.8) years, and 65.3% were male. Median (Q1–Q3) follow-up after diagnosis was 39.5 (33.8–60.4) months. The most common disease presentation at WD diagnosis was combined neurologic/psychiatric and hepatic (52.9%), followed by neurologic/psychiatric (20.0%), hepatic (16.9%), and asymptomatic (10.2%). Common clinical characteristics at diagnosis were Kayser-Fleischer rings (77.2%), low ceruloplasmin levels (95.2%), high hepatic copper (97.8%), elevated 24-hour urinary copper excretion (90.2%), and abnormal liver function tests (38.7%–85.1%). At diagnosis, the most common biochemical findings or hepatic sign/symptoms were abnormal liver enzymes (50.7%), abdominal pain (16.6%), and fatigue (15.7%). The most common neurologic signs/symptoms were headache (18.3%), dysarthria (17.4%), and ataxia (17.0%). Common psychiatric signs/symptoms included anxiety/depression/other mood changes (36.2%), emotional lability (12.8%), and increased irritability/anger outbursts (9.2%). Prevalence of biochemical abnormalities or signs/symptoms among patients at diagnosis and after ~1-year follow-up were neurologic (60.1% and 44.0%), hepatic (69.6% and 37.8%), and psychiatric (53.7% and 37.6%), respectively. Common new onset symptoms at ~1-year post-WD diagnosis were abnormal liver enzymes (5.6%), headache (6.2%), and anxiety/depression/other mood changes (7.2%).ConclusionsThese real-world, descriptive data highlight the clinical complexity and heterogeneity of WD and the need for better education about diagnostic testing and multidisciplinary support. Although rare, the neurologic, psychiatric, and hepatic signs/symptoms of WD have a substantial clinical impact.

Real-World Characteristics of Patients with Severe Asthma prior to Starting Dupilumab: The ProVENT Study

Introduction: Dupilumab is approved for the treatment of severe type 2 (T2) asthma; however, the characteristics of patients receiving dupilumab in routine clinical practice are incompletely understood. This study describes the characteristics of patients with severe asthma before dupilumab treatment in a real-world setting. Methods: This interim analysis of an ongoing real-life study of dupilumab assessed baseline characteristics of the first patient cohort enrolled in the ProVENT study. Results: A total of 99 patients (59% females) were analyzed (17% received another biologic before dupilumab treatment and 15% were on maintenance oral corticosteroid treatment). Adult-onset asthma (>18 years) and an allergic phenotype were documented in 58% and 48% of patients, respectively. Median (interquartile range) age was 54 (40–61) years; the median number of exacerbations in the last 24 months was 1 (0–3); median fractional exhaled nitric oxide (FeNO) value was 38 (23–64) ppb; and median blood eosinophils (bEOS) count was 184 (8–505) cells/µL. According to the United Kingdom Severe Asthma Registry classification, 53% of patients had T2 intermediate asthma (bEOS ≥150 cells/µL or FeNO ≥25 ppb), 17% had T2 high asthma (bEOS ≥150 cells/µL and FeNO ≥25 ppb), and 4% had T2 low asthma (bEOS <150 cells/µL and FeNO <25 ppb). At least one GINA criterion for T2 airway inflammation was documented in 70% of patients. T2 comorbidities were observed in 64% of patients. Conclusions: This analysis suggests that patients eligible for dupilumab treatment display various clinical and biochemical characteristics rather than one clear-cut phenotype.

Real-World Patient Characteristics, Treatment Patterns, and Outcomes in Patients with Chronic Lymphocytic Leukemia/Small Lymphocytic Leukemia in China: A Retrospective Analysis Using a Real-World Database

Background: Innovation in chronic lymphocytic leukemia/small lymphocytic leukemia (CLL/SLL) has yield tremendous progresses and new treatment options recently. However, real-world treatment patterns, healthcare utilization and costs associated with CLL/SLL are not well studies in middle-income countries such as China. Because CLL/SLL predominantly affects older adults, this study aimed to evaluate baseline characteristics, treatment patterns, healthcare resource utilization (HCRU), costs, and survival in a representative sample of patients from Tianjin city who newly diagnosed with CLL/SLL. Methods: This retrospective study used integrated regional electronic healthcare records from 2004 to 2022. The sample included patients newly diagnosed with CLL/SLL with continuous healthcare encounters (index date = first CLL/SLL diagnosis date). Outcomes included treatment patterns, all-cause and CLL/SLL-related HRU and costs, and overall survival in the frontline and relapsed/refractory settings. Results: We identified 3665 patients meeting the inclusion/exclusion criteria. Overall, patients with CLL/SLL were 62 years of age at index date, 1409 (38.5%) were female, and 910 (24.8%) had insurance coverage. Patients had an average CCI of 1.6 (SD:2.0) during baseline, and the most common comorbid conditions were hypertension (8.9%), pulmonary infection (7.8%) and diabetes (5.9%). 1500 (41.0%) of patients had been exposed to CLL/SLL-directed treatment, with 856(57.0%), 343(22.9%) and 301(20.0%) of patients exposed to chemotherapy, immunochemotherapy and targeted therapy in first-line treatment, respectively. Among the subgroup of patients received targeted therapy, 335 (74.4%), 98 (21.8%) ,5 (1.1%) ,4 (0.9%) and 8 (1.8%) used ibrutinib, zanubrutinib, orelabrutinib, venetoclax, and lenalidomide. 15 (0.4%) and 2 (0.05%) patients received stem cell transplantation and CAR-T, respectively. In terms of HCRU, 2434of patients (66.4%) had a hospitalization due to any cause, 1769 (48.4%) of which were CLL/SLL-related. Emergency department and outpatient service use increased over the study period. Mean per person per month (PPPM) use for inpatient, outpatient and ED visits were 0.53, 0.30 and 0.59, respectively. Mean total PPPM healthcare costs were $ 3125.0 (inpatient), $ 488.1 (outpatient), $ 87.6 (ED), and $ 1085.8 (medication). Median OS from initial diagnosis or 1L treatment initiation were not achieved. The 1-year and 3-year survival rate was 95.1% and 88.5 % from 1L treatment initiation, 97.6 % and 92.8% from the initial diagnosis. Conclusions: In this real-world study using regional healthcare data, we observed distinct demographic, clinical and tumor-related characteristics. Patients were diverse in terms of age, insurance status and healthcare resource utilization. As newer therapies continue to accumulate, future studies should examine how unmet needs in patients with CLL/SLL can be addressed to ensure better access to novel treatment.

Real-World Patient Characteristics, Treatment Patterns, and Outcomes in Patients with Lower-Risk Myelodysplastic Syndromes Who Have Been Treated with Luspatercept

Background: Myelodysplastic syndromes (MDS) are a group of rare, clonal bone marrow malignancies characterized by ineffective erythropoiesis. Erythropoiesis-stimulating agents (ESAs) are a treatment option for anemia in patients with lower-risk MDS (LR-MDS); however as only one in three patients respond to ESAs, there is a need for additional treatment options. In April 2020, luspatercept was approved for the treatment of anemia in adult patients with LR-MDS who failed to respond to ESAs. The aim of this study was to investigate real-world treatment patterns and early clinical outcomes in adult patients in the United States with LR-MDS receiving luspatercept. Methods: This was a retrospective observational cohort study of adult patients (aged ≥ 18 years at initial MDS diagnosis) with an International Prognostic Scoring System/Revised IPSS (IPSS-R/IPSS) classification of Very Low-, Low-, or Intermediate-risk MDS on or before the initial MDS diagnosis date +30 days, who were actively treated with a therapy of interest (including ESAs, hypomethylating agents [HMAs], erythroid maturation agents [EMAs], iron chelation therapy [ICT], white blood cells [WBCs], growth factors, chemotherapy, immunomodulating drugs [IMiDs], and platelet growth factors), and who received at least one dose of luspatercept between initial MDS diagnosis and November 1, 2022 in the COTA Vantage database which contains longitudinal, HIPAA-compliant data pertaining to the diagnosis, clinical management, and outcomes of patients with cancer from a mix of academic and community settings. The index date was the date of luspatercept initiation. Patients were required to have at least 2 months of medical data post-index unless they died during this period. All analyses were descriptive. Results: A total of 86 patients who were treated with luspatercept (median age 78.0 years, 79.1% White, 52.3% male, 80.2% ECOG 0-1) were included. Median baseline hemoglobin level was 7.8 g/dL (range: 5.3-11.4 g/dL). Median time from MDS diagnosis to luspatercept initiation was 35.4 months and the median follow-up time was 13.4 months. The majority of patients (89.5%) were previously treated with ESAs for a median duration of 9.0 months. The two most used ESAs were darbepoetin alfa (54.5%) and epoetin alfa (44.2%). In addition, 40.7% of patients had previously received a non-ESA treatment, most commonly azacitidine (62.9%) or lenalidomide (31.4%). The median duration of treatment (DoT) with luspatercept was 9.9 months. Around a quarter of patients who received luspatercept (25.6%, n = 22) received a further treatment, such as an ESA or an HMA for a median of 2.8 months. Overall, 51/76 (67.1%) patients with a baseline hemoglobin test who received luspatercept had an increase in hemoglobin of ≥1 g/dL compared with baseline (i.e. the closest hemoglobin test within 6 months prior to index date). Of these, 56.9% maintained the ≥1 g/dL hemoglobin level for ≥8 weeks and 45.1% maintained it for ≥16 weeks. Further, 44/76 (57.9%) patients with a baseline hemoglobin test who received luspatercept had an increase in hemoglobin ≥1.5 g/dL compared with baseline. Of these, 50.0% maintained the ≥1.5 g/dL hemoglobin level for ≥8 weeks and 25.0% maintained it for ≥16 weeks. Conclusion: This real-world study described treatment patterns and outcomes in patients with LR-MDS receiving luspatercept. An improvement in hemoglobin levels was observed in more than half of patients. In a quarter of patients who had a ≥1.5 g/dL increase compared to baseline, these improvements were maintained for ≥16 weeks. As this was a relatively small and descriptive study, future analyses are required to further elucidate these findings.

Teclistamab Demonstrates Clinical Activity in Real-World Patients Ineligible for the Pivotal Majestec-1 Trial

Introduction Treatment options for relapsed/refractory multiple myeloma (RRMM) are limited and associated with a poor median overall survival (OS) of 12.4 months (m) (Mateos et al. Leukemia 2022). The MajesTEC-1 trial demonstrated promising clinical activity of teclistamab (Tec), a BCMAxCD3 bispecific antibody, with an overall response rate (ORR) of 63%, complete response rate (CR) of 39.4%, median progression-free survival (PFS) of 11.3 m, and OS of 18.3 m. Here we present the results of a multicenter, retrospective study examining real-world patient characteristics and outcomes in patients with RRMM receiving Tec outside of a clinical trial, including those who would have been ineligible for the registrational study. Methods An IRB-approved retrospective study of patients with RRMM treated with Tec at 4 academic centers was performed. Patient demographics, prior treatments, clinical outcomes and toxicities with special attention to cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and infection were extracted from the electronic patient record. Response rate was compared using Fisher's exact test. Median PFS and OS were estimated by Kaplan-Meier and compared using the log-rank test. Trial eligibility was evaluated using the MajesTEC-1 eligibility criteria. Results A total of 45 patients, who received at least 1 dose of Tec, were evaluated. Of these 39% were derived from under-represented minorities (25% Black, 14% Hispanic). Patient characteristics and comparison to the MajesTEC-1 population are summarized, Table 1. High-risk cytogenetic features were present in 42.2% of patients, and 46.7% had extra-medullary disease (EMD). All patients were triple class exposed and 80% were penta-class exposed. Most patients (84.4%) would have been ineligible for MajesTEC-1 due to cytopenias (44.4%), prior BCMA-directed therapy (42.2%), inadequate washout (42.2%), and poor performance status (PS) (26.7%). Therapy was delivered according to protocol with no increased rate of infections or bleeding, even in the group of patients with low blood counts and poor PS. CRS/ICANS events of any grade were observed in 55% and 13% of patients, respectively, during step-up dosing. Most CRS/ICANS events were mild-moderate in severity (CRS 42.2% grade 1, 11.1% grade 2; ICANS 4.4% grade 1, 0% grade 2) and were managed conservatively with antipyretics, tocilizumab (37.8%), and steroids (13.3%). Grade 4 CRS and ICANS were only seen in 1 patient; a separate individual experienced grade 5 ICANS after the third step up dose. The overall response rate (ORR) was 48.9% (95% CI 33.7-64.2) with 22.2% (95% CI 11.2-37.1) achieving at least a very-good partial response (VGPR). On univariate analysis prior anti-BCMA exposure was associated with an adverse ORR (23% vs 61%; p=0.02). Ineligibility for enrollment in MajesTEC-1, the presence of high-risk cytogenetics or EMD were not associated with ORR or depth of response. Updated follow up time, median PFS and OS will be presented at the meeting to allow time for data to mature. The presence of high-risk cytogenetics was associated with worse PFS (median NR vs 2.1 m, HR 2.32; p=0.041). Receipt of prior BCMA therapy and MajesTEC-1 ineligibility were not associated with PFS or OS. At the time of data cut-off, 19 (42.2%) patients remained on Tec; reasons for discontinuation included disease progression, infections, and CRS/ICANS in 19 (42.2%), 4 (8.8%), and 3 (6.7%) patients, respectively. Conclusion Overall, Tec demonstrated significant clinical activity even in a group of heavily pre-treated real-world patients comprising high-risk and extramedullary disease populations. The overall toxicity profile was similar to prior reports demonstrating that side effects can be readily managed using simple approaches with only limited patients needing more intensive interventions. These results also suggest Tec may be used in patients previously excluded from clinical trials because of adverse clinical characteristics with high ORR being seen and an absence of increased toxicity. In the BCMA-naive population ORR were similar to MajesTEC-1, but were lower in the previously BCMA-exposed population. High-risk cytogenetics and EMD remain adverse prognostic features with Tec despite similar ORR.

Real-World Patient Characteristics, Treatment Patterns, and Outcomes in Patients with Mantle Cell Lymphoma in China: A Retrospective Analysis Using a Real-World Database

Background: Significant advancements and novel therapeutic options have emerged in the treatment of mantle cell lymphoma (MCL) in recent years. However, there is a dearth of research on real-world treatment patterns, healthcare utilization, and costs associated with MCL in middle-income countries like China. Given that MCL predominantly affects the elderly population, this study aimed to assess the characteristics, treatment approaches, healthcare resource utilization (HCRU), costs, and survival outcomes in a representative sample of patients diagnosed with MCL in Tianjin City. Methods: This retrospective study utilized integrated regional electronic healthcare records spanning from 2004 to 2022. The sample included patients newly diagnosed with MCL who had a continuous series of healthcare encounters, with the index date set as the date of the initial MCL diagnosis. The study examined treatment patterns, all-cause and MCL-related HCRU and costs, as well as overall survival in both the initial treatment and relapsed/refractory settings. Results: The study identified 475 patients who had a confirmed diagnosis and met the specified inclusion/exclusion criteria. Overall, the patients with MCL had an average age of 58 at the index date, with 123 (25.9%) being female, and 139 (29.3%) having insurance coverage. The average Charlson Comorbidity Index (CCI) during the baseline period was 1.8 (SD:2.1), and the most prevalent comorbid conditions included hypertension (23.6%), pulmonary infection (21.9%) and diabetes (13.6%). A total of 250 (54%) patients had received treatment specific to MCL, with 57 (22.8%), 138 (55.2%), and 45 (18.0%) initiating chemotherapy, immunochemotherapy, and targeted therapy in first-line treatment, respectively. Among those who received targeted therapy,44 (50.0%), 40 (45.5%), 1 (1.1%) and 3 (3.4%) were treated with ibrutinib, zanubrutinib, orelabrutinib, and venetoclax, respectively. Additionally, 43 (9.1%) and 4 (0.8%) underwent stem cell transplantation and CAR-T therapy, respectively. Regarding HCRU, 367 patients (77.3%) experienced hospitalizations, with 328 (69.1%) of these hospitalizations related to MCL. Over the study period, the utilization of emergency department (ED) and outpatient services increased. The average monthly per person utilization rates for inpatient, outpatient, and emergency department visits were 0.62, 0.23, and 0.61, respectively. The mean total monthly healthcare costs were $9454.0 (inpatient), $895.9 (outpatient), $129.9 (ED), and $1739.0 (medication). The median overall survival (OS) from initiation of first-line (1L) treatment was 72.4 months, respectively. The 1-year and 3-year survival rates were 95.3 % and 86.9% from the initial diagnosis, and 78.6% and 71.4% from 1L treatment initiation, respectively. Conclusions: This real-world study, utilizing regional healthcare data, revealed distinctive demographic, clinical, and tumor-related characteristics among patients diagnosed with MCL. These patients demonstrated diversity in terms of age, insurance status, and healthcare resource utilization. As novel therapies continue to emerge, future investigations should address the unmet needs of patients with MCL, ensuring improved accessibility to innovative treatments.

Tafasitamab and Lenalidomide in Relapsed/Refractory B-Cell Lymphoma: A Multicenter Retrospective Real-World-Study of Patients from Germany and Austria

Background Publication of the L-MIND-study led to the approval of tafasitamab/lenalidomide (TL) for patients with relapsed oder refractory diffuse large B-cell lymphoma (r/r DLBCL), who were ineligible for autologous stem-cell transplantation (ASCT). The findings of the L-MIND-study showed an overall response rate (ORR) of 60%, complete response rate (CRR) of 43%, median progression free survival (PFS) of 12.1 months, and median overall survival (OS) of 33.5 months. To analyze the real-world characteristics of patients treated with TL, we performed a multicenter retrospective study with patients who were teated with TL since the implementation of the Expanded Access Program (EAP) from 03/2020 until 07/2023. Methods We retrospectively analyzed the outcomes, demographics, data on diagnosis, prior therapies, adverse events (AEs), and treatment after TL of 127 patients with r/r DLBCL treated with TL in Germany and Austria in 18 institutions. OS and PFS are displayed by Kaplan-Meier graphs. Relationships with multiple covariables were calculated by Cox regression. We performed subgroup analyses with prior anti-CD19 therapy, primary refractory disease vs. relapsed disease, IPI, number of prior therapies, treatment after TL, age, and sex. The study was approved by the local Research Ethics Committees. Results Overall, only 37% of our patients met the L-MIND study eligibility criteria; most frequent reasons for ineligibility were more than 3 prior lines of therapy (52%), prior therapy with anti-CD19/IMiDs (19% prior CAR T-cell therapy and Bispecific Antibodies, 7% prior IMiDs), High-Grade-BCL/Follicular Lymphoma Grade 3A/CNS involvement/primary refractory lymphomas/Primary-Mediastinal-BCL/B-ALL (23%), or patients who had lymphoma-specific-therapy within 14 days prior to day 1 dosing with TL (8%). Patient characteristics are stated in Table 1. ORR for the entire cohort (n=127) was 33.1%, CRR 11.8%. Median PFS was 4.7 months (95% CI 6.9 - 11.6), or even 2.9 months (95% CI 2.2 - 3.2) including all causes of death as progression, and median OS 8.9 months (95% CI 5.0 - 11.0) for the whole cohort; see Figure 1. Compared to refractory disease (n=55, 43%), relapsed disease (n=72, 57%) was associated with better ORR (43% vs. 20%, P=0.008). PFS and OS were worse in the primary refractory compared to the relapsed group (median PFS 1.7 vs. 5.3 months, HR 2.79, 95% CI 1.82-4.28, P&amp;lt;0.001; median OS 4.5 vs. 12.9 months, HR 2.72, 95% CI 1.53-4.33, P&amp;lt;0.001). 36% of the patients had a low IPI (0-2, low risk), 55% had a higher IPI (3-5, high risk) at primary diagnosis. ORR (40% vs. 30%) and OS (median OS 9.1 vs. 5.6 months HR 1.76, 95% CI 1.04-2.99) were higher in the lower risk group than in the higher risk group. Fewer prior lines of therapy (1-2 vs. ≥3) were associated with longer median PFS (median 3.3 vs. 1.9 months HR 1.13 per additional line, 95% CI 1.00-1.28, P=0.057) and longer median OS (median 12.5 vs. 5.2 months HR 1.18, 95% CI 1.01-1.37, P=0.039). Median time between the administration of the last prior therapy and TL were 6.4 months. No significant relationships were found for age, sex, and preceding CAR-T treatment. Prior CAR T-cell therapy (24 of 127 patients, post-CAR T-cohort) showed only small differences in ORR (37.5%), PFS (7.9 months with CAR T vs. 12 months without CAR T) or OS (9.2 months with CAR T vs. 12.4 months without CAR T); CRR was higher than in patients not treated with CAR T prior to TL (21% vs. 9.7%). 10 of the analyzed 127 patients received CAR T-cell therapy after TL. Within this subgroup, ORR was 50%. In 2 of these 10 patients TL was used as bridging therapy to CAR T; 1 of 2 patients reached a PR, the other one died before CAR T-cell therapy. The most common hematological AE was neutropenia (67%), the most common non-hematological AEs included infections (31%) and rash and itching (9%). Conclusions We show that the outcomes including ORR, PFS and OS in the real-world setting were lower than observed in the preceding phase II clinical trial (L-MIND). TL seems to be less feasible for patients that show high-risk features; comorbidities, age, dose reductions and the number of pretreatment lines may add to this. The data suggests that TL can still be used after CAR T-cell therapy or as a bridge to CAR T-cell therapy. Our findings on the frequency of observed AEs are consistent with the L-MIND-study.