Real-world Patient Characteristics Research Articles

Hypereosinophilic syndrome in Europe: Retrospective study of treatment patterns, clinical manifestations, and healthcare resource utilization

BackgroundThe burden of hypereosinophilic syndrome (HES) in Europe is not well characterized. ObjectiveTo evaluate real-world patient characteristics, treatment patterns, clinical manifestations, and healthcare resource utilization for patients with HES from France, Germany, Italy, Spain, and the United Kingdom. MethodsIn this retrospective, noninterventional study, data for patients with a physician-confirmed diagnosis of HES were abstracted from medical chart reviews. Patients were aged 6 years or older at the time of HES diagnosis and had 1 or more years of follow-up from the index date (first clinic visit between January 2015 and December 2019). Data on treatment patterns, comorbidities, clinical manifestations, clinical outcomes, and healthcare resource utilization were collected from diagnosis or index date to end of follow-up. ResultsData for 280 patients were abstracted from medical charts by 121 physicians treating HES, with multiple specialties. Most patients (55%) had idiopathic HES, and 24% had myeloid HES; the median number (interquartile range [IQR]) of diagnostic tests per patient was 10 (6-12). The most common comorbidities were asthma (45%) and anxiety or depression (36%). Most patients (89%) used oral corticosteroids; 64% used immunosuppressants or cytotoxic agents, and 44% used biologics. Patients had a median (IQR) of 3 clinical manifestations (1-5), most commonly constitutional (63%), lung (49%), and skin (48%). Twenty-three percent of patients experienced a flare, and 40% had a complete treatment response. Some patients (30%) were hospitalized with a median (IQR) stay of 9 days (5-15) for HES-related issues. ConclusionPatients with HES across 5 European countries had a substantial disease burden despite extensive oral corticosteroids treatment, highlighting the need for additional targeted therapies.

SPADE: Design of a real-world observational study of avelumab first-line (1L) maintenance in advanced urothelial carcinoma (UC) in the Asia-Pacific (APAC) region.

TPS577 Background: UC is a common cancer, with >200,000 new cases reported in the APAC region in 2020. In the global phase 3 JAVELIN Bladder 100 trial, avelumab 1L maintenance + best supportive care (BSC) significantly prolonged overall survival (OS) and progression-free survival (PFS) vs BSC alone in patients with advanced UC that had not progressed with 1L platinum-containing chemotherapy (median OS, 23.8 vs 15.0 months [p=0.0036]; median PFS, 5.5 vs 2.1 months [p<0.0001]), leading to regulatory approvals worldwide and incorporation into international treatment guidelines with level 1 evidence. The objectives of the SPADE noninterventional study are to evaluate real-world patient characteristics, treatment patterns, patient-reported outcomes (PROs) and treatment outcomes with avelumab maintenance administered in patients with advanced UC without disease progression after 1L platinum-based chemotherapy in the APAC region. Methods: SPADE is a multicenter, prospective, observational study ongoing in Australia, Hong Kong, India, Japan, Republic of Korea, Singapore, Taiwan, and other countries. Overall, 286 patients with unresectable locally advanced or metastatic (stage IV) measurable UC of any histology that has not progressed with 1L platinum-containing chemotherapy, for whom avelumab 1L maintenance therapy is planned, will be enrolled. All patients will receive avelumab 800 mg intravenously every 2 weeks (or per local marketing authorization) and will be followed up for 12 months or until avelumab is discontinued. The decision to start avelumab treatment should be made in advance by the treating physician and per local clinical practice. Data collected will include patient demographics (ECOG performance status, renal function, history of UC, primary tumor location, location of metastases, and prior therapy), treatment details for 1L platinum-based chemotherapy (regimen, cycles, and response per RECIST), treatment details for avelumab 1L maintenance (interval from end of chemotherapy until initiation, duration of avelumab treatment, and planned subsequent treatment), and treatment outcomes with avelumab 1L maintenance (6- and 12-month OS and health-related quality of life). Data sources will include medical records collected during routine clinical care. Data for baseline treatment patterns will be summarized using descriptive statistics. OS will be analyzed using the Kaplan-Meier method. Health-related quality of life, including changes over time, will be measured using the EQ-5D-5L and National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy Bladder Symptom Index (NCCN/FACT FBISI-18) questionnaires. An interim analysis is planned after ~30% of patients have been enrolled.

Real-World Characteristics of Patients with Wild-Type Transthyretin Amyloid Cardiomyopathy: An Analysis of Electronic Healthcare Records in the United States.

Tafamidis was approved for the treatment of hereditary and wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) in May 2019, based on findings from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT). This retrospective cohort study evaluated the factors associated with tafamidis prescription after diagnosis of ATTRwt-CM in the real world. Between May 2019 and December 2020, 430 patients with 6 months' database activity were indexed from the de-identified US Optum electronic healthcare records at first diagnosis of ATTRwt-CM or prescription of tafamidis, then followed until last activity or death. Of these, 209 patients were prescribed tafamidis during follow-up, 167 (80%) within 1 month, 98% by 6 months, and 100% by 9 months. Median time from index to tafamidis prescription, calculated using the Kaplan-Meier method, was 5.8 months (95% confidence interval [CI] 2.4-not evaluable). Factors associated with tafamidis prescription in a multivariable Cox proportional hazards regression (hazard ratio [95% CI]) included age ≥65 years (2.1 [1.07-4.05]), male sex (1.6 [1.07-2.28]), having heart failure/cardiomyopathy (2.4 [1.54-3.82]), and having had technetium-99m pyrophosphate myocardial scintigraphy (1.7 [1.28-2.28]). The clinical characteristics of patients with ATTRwt-CM who were prescribed tafamidis in the real world were broadly comparable with those who took part in ATTR-ACT. Further studies are needed to evaluate hereditary and ATTRwt-CM patient populations in the real world and assess the long-term outcomes associated with disease management pathways. ClinicalTrials.gov identifier: NCT01994889.

Real-world patient characteristics and treatment patterns of polycythemia vera in Taiwan between 2016 and 2017: a nationwide cross-sectional study.

Polycythemia vera (PV) patients often experience constitutional symptoms and are at risk of thromboembolism as well as disease progression to myelofibrosis or acute myeloid leukemia. Not only is PV often overlooked but treatment options are also limited, however. To explore the patient characteristics and treatment pattern of PV patients in Taiwan, and compare with other countries reported in the literature. This is a nationwide cross-sectional study. The National Health Insurance Research Database in Taiwan, which covers 99% of the population, was utilized. Patients were identified during the cross-sectional period between 2016 and 2017, and their retrospective data were retrieved from 2001 to 2017. A total of 2647 PV patients were identified between 1 January 2016 and 31 December 2017. This study described the demographic information of these patients, including number of patients by risk stratification and by sex, age at diagnosis, age at cross-sectional period, rate of bone marrow aspiration/biopsy at diagnosis, comorbidities, number of postdiagnosis thrombosis, number of disease progression, and death. The mortality rate of PV patients (4.1%) over 60 of age was higher than the general population of the same age group (2.8%). This study also compared the different treatment patterns between sexes and risk groups. Hydroxyurea was deferred to an older age, but conversely was prescribed at higher dose to younger patients. Alarmingly, a high proportion of patients did not receive phlebotomy or hydroxyurea for at least 2 years. Furthermore, discrepancies in prevalence, age at diagnosis, sex ratio, incidence of thrombosis and mortality were also found when compared with data reported in other countries. The clinical landscape of PV in Taiwan between 2016 and 2017 was examined. Distinctive patterns of phlebotomy and hydroxyurea were identified. Overall, these findings highlight the importance of understanding the patient characteristics and treatment patterns of PV in different regions to better inform clinical practice and improve patient outcomes.

Burden of Comorbidities and Healthcare Resource Utilization Among Medicaid-Enrolled Extremely Premature Infants

Background: The effect of gestational age (GA) on comorbidity prevalence, healthcare resource utilization (HCRU), and all-cause costs is significant for extremely premature (EP) infants in the United States. Objectives: To characterize real-world patient characteristics, prevalence of comorbidities, rates of HCRU, and direct healthcare charges and societal costs among premature infants in US Medicaid programs, with respect to GA and the presence of respiratory comorbidities. Methods: Using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes, diagnosis and medical claims data from 6 state Medicaid databases (1997-2018) of infants born at less than 37 weeks of GA (wGA) were collected retrospectively. Data from the index date (birth) up to 2 years corrected age or death, stratified by GA (EP, ≤28 wGA; very premature [VP], >28 to <32 wGA; and moderate to late premature [M-LP], ≥32 to <37 wGA), were compared using unadjusted and adjusted generalized linear models. Results: Among 25 573 premature infants (46.1% female; 4462 [17.4%] EP; 2904 [11.4%] VP; 18 207 [71.2%] M-LP), comorbidity prevalence, HCRU, and all-cause costs increased with decreasing GA and were highest for EP. Total healthcare charges, excluding index hospitalization and all-cause societal costs (US dollars), were 2 to 3 times higher for EP than for M-LP (EP $74 436 vs M-LP $27 541 and EP $28 504 vs M-LP $15 892, respectively). Conclusions: Complications of preterm birth, including prevalence of comorbidities, HCRU, and costs, increased with decreasing GA and were highest among EP infants during the first 2 years in this US analysis.

Burden of Comorbidities and Healthcare Resource Utilization Among Medicaid-Enrolled Extremely Premature Infants.

Background: The effect of gestational age (GA) on comorbidity prevalence, healthcare resource utilization (HCRU), and all-cause costs is significant for extremely premature (EP) infants in the United States. Objectives: To characterize real-world patient characteristics, prevalence of comorbidities, rates of HCRU, and direct healthcare charges and societal costs among premature infants in US Medicaid programs, with respect to GA and the presence of respiratory comorbidities. Methods: Using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes, diagnosis and medical claims data from 6 state Medicaid databases (1997-2018) of infants born at less than 37 weeks of GA (wGA) were collected retrospectively. Data from the index date (birth) up to 2 years corrected age or death, stratified by GA (EP, ≤28 wGA; very premature [VP], >28 to <32 wGA; and moderate to late premature [M-LP], ≥32 to <37 wGA), were compared using unadjusted and adjusted generalized linear models. Results: Among 25 573 premature infants (46.1% female; 4462 [17.4%] EP; 2904 [11.4%] VP; 18 207 [71.2%] M-LP), comorbidity prevalence, HCRU, and all-cause costs increased with decreasing GA and were highest for EP. Total healthcare charges, excluding index hospitalization and all-cause societal costs (US dollars), were 2 to 3 times higher for EP than for M-LP (EP $74 436 vs M-LP $27 541 and EP $28 504 vs M-LP $15 892, respectively). Conclusions: Complications of preterm birth, including prevalence of comorbidities, HCRU, and costs, increased with decreasing GA and were highest among EP infants during the first 2 years in this US analysis.

Real-world patient characteristics and use of disease-modifying anti-rheumatic drugs in patients with rheumatoid arthritis: a cross-national study

IntroductionRheumatoid arthritis (RA) is associated with significant morbidity and economic burden. This study aimed to compare baseline characteristics and patterns of anti-inflammatory drug use and disease-modifying anti-rheumatic drug (DMARD) use among patients with RA in Southern Italy versus the United States.MethodUsing Caserta Local Health Unit (Italy) and Optum’s de-identified Clinformatics® Data Mart (United States) claims databases, patients with ≥ 2 diagnosis codes for RA during the study period (Caserta: 2010–2018; Optum: 2010–2019) were identified. Baseline patient characteristics, as well as proportion of RA patients untreated/treated with NSAIDs/glucocorticoids/conventional DMARDs (csDMARDs)/biological/targeted synthetic DMARDs (b/tsDMARDs) during the first year of follow-up, and the proportion of RA patients with ≥ 1 switch/add-on between the first and the second year of follow-up, were calculated. These analyses were then stratified by age group (< 65; ≥ 65).ResultsA total of 9227 RA patients from Caserta and 195,951 from Optum databases were identified (two-thirds were females). During the first year of follow-up, 45.9% RA patients from Optum versus 79.9% from Caserta were exclusively treated with NSAIDs/glucocorticoids; 17.2% versus 11.3% from Optum and Caserta, respectively, were treated with csDMARDs, mostly methotrexate or hydroxychloroquine in both cohorts. Compared to 0.6% of RA patients from Caserta, 3.2% of the Optum cohort received ≥ 1 b/tsDMARD dispensing. Moreover, 61,655 (33.7%) patients from Optum cohort remained untreated compared to 748 (8.3%) patients from the Caserta cohort. The subgroup analyses stratified by age showed that 42,989 (39.8%) of elderly RA patients were untreated compared to 18,666 (24.9%) young adult RA patients in Optum during the first year of follow-up. Moreover, a higher proportion of young adult RA patients was treated with b/tsDMARDs, with and without csDMARDs, compared to elderly RA patients (Optum<65: 6.4%; Optum≥65: 1.0%; P-value < 0.001; Caserta<65: 0.8%; Caserta≥65: 0.1%; P-value < 0.001). Among RA patients untreated during the first year after ID, 41.2% and 48.4% RA patients from Caserta and Optum, respectively, received NSAIDs, glucocorticoids, and cs/b/tsDMARDs within the second year of follow-up. Stratifying the analysis by age groups, 50.6% of untreated young RA patients received study drug dispensing within the second year of follow-up, compared to only 36.7% of elderly RA patients in Optum. Interestingly, more young adult RA patients treated with csDMARDs during the first year after ID received a therapy escalation to b/tsDMARD within the second year after ID in both cohorts, compared to elderly RA patients (Optum<65: 7.8%; Optum≥65: 1.8%; Caserta<65: 3.2%; Caserta≥65: 0.6%).ConclusionsMost of RA patients, with heterogeneous baseline characteristics in Optum and Caserta cohorts, were treated with anti-inflammatory/csDMARDs rather than bDMARDs/tsDMARDs during the first year post-diagnosis, especially in elderly RA patients, suggesting a need for better understanding and dealing with barriers in the use of these agents for RA patients.Key Points• Substantial heterogeneity in baseline characteristics and access to bDMARD or tsDMARD drugs between RA patients from the United States and Italy exists.• Most of RA patients seem to be treated with anti-inflammatory/csDMARD drugs rather than bDMARD/tsDMARD drugs during the first year post-diagnosis.• RA treatment escalation is less frequent in old RA patients than in young adult RA patients.• An appropriate use of DMARDs should be considered to achieve RA disease remission or low disease activity.

RWD42 Patient Characteristics and Treatment Patterns in Patients With Persistent, Recurrent, or Metastatic (P/R/M) Cervical Cancer: A Real-World Data Analysis in the US

Describe real-world patient characteristics and treatment patterns in patients with P/R/M cervical cancer in the US.

Real-World Patient Characteristics, Treatment Patterns, and Outcomes in Patients with Stage III/IV Classic Hodgkin Lymphoma Treated with Frontline ABVD: A Retrospective Analysis Using a Real-World Database

Real-World Patient Characteristics, Treatment Patterns, and Outcomes in Patients with Stage III/IV Classic Hodgkin Lymphoma Treated with Frontline ABVD: A Retrospective Analysis Using a Real-World Database

Real-World Treatment Patterns and Clinical Outcomes with Brentuximab Vedotin or Other Standard Therapies in Patients with Previously Treated Cutaneous T-Cell Lymphoma (CTCL): A Retrospective Chart Review Study in the United States

INTRODUCTION: CTCLs such as primary cutaneous anaplastic large-cell lymphomas (pcALCLs) uniformly express CD30, whereas mycosis fungoides (MF) heterogeneously express CD30. In ALCANZA (Horwitz 2021), patients with CD30-positive pcALCL or MF randomized to brentuximab vedotin (BV) vs physician's choice of methotrexate or bexarotene had a significantly higher objective response rate (ORR) lasting ≥4 months (ORR4; 54.7% vs 12.5%; P<0.001) and longer median progression-free survival (PFS; 16.7 vs 3.5 months; P<0.001) and time to next treatment (TTNT; 14.2 vs 5.6 months; P<0.001) at a median follow-up of 45.9 months. This study describes real-world patient characteristics, treatment patterns, clinical outcomes, and healthcare resource use (HRU) in CTCL patients treated with BV or other standard therapies (OST) after ≥1 systemic therapy. METHODS: A retrospective, longitudinal physician chart review was conducted from Oct 2021-Jan 2022 (approved by WCG IRB). Physicians were oncologists/hematologists with access to complete medical records for ≥1 eligible patient. Eligible adult patients had pcALCL or MF, previously received ≥1 systemic therapy, and were treated with BV or OST from Nov 2017-Mar 2021. The index date was the initiation of second-line (2L) CTCL systemic therapy. The observation period was from index to the earliest of death, loss to follow-up, or chart abstraction. Treatment patterns were assessed from CTCL diagnosis to end of observation period; clinical outcomes for 2L systemic therapy and HRU were assessed during the observation period. Treatment response was recorded via the Global Response Score (Olsen 2011). TTNT was from index to the earlier of initiation of a new line of therapy or death. Real-world PFS (rwPFS) was from index to earliest of progression, treatment discontinuation due to progression, or death. Overall survival (OS) was from index to death. Median times-to-event were computed using Kaplan-Meier (KM) analysis. Since median times-to-event were not reached in both cohorts for multiple outcomes, restricted mean survival time (RMST), a measure of the average event-free survival time during a specific period estimated by the area under the KM curve, at 1 and 2 years was also estimated. RESULTS: Physicians (n=125; 68.0% community, 75.2% practiced >10 years) abstracted medical charts for 303 patients (BV/OST: n=139/164; male, 66.9%/67.7%; MF, 56.8%/65.9%; ECOG PS 0-1, 86.3%/67.1%). The median observation period for BV and OST was 8.1 (interquartile range: 7.1-10.8) and 8.8 (4.8-11.5) months. Most patients in the BV cohort (96.4%) received BV as 2L systemic therapy. In the OST cohort, the most common 2L therapies were methotrexate (11.6%), mogamulizumab (9.1%), and bendamustine (9.1%) monotherapies. The median (95% CI) duration of 2L therapy for BV and OST was 8.4 (7.1-15.4) and 5.2 (3.9-7.3) months. The rwORR for BV and OST was 82.1% and 66.5%; rwORR4 was 42.5% and 25.0% (Table 1). Median (95% CI) TTNT was not reached (NR; 17.5-NR) for BV and was 13.2 (9.2-NR) months for OST; RMST for TTNT was higher for BV vs OST (1-year: 11.2 [10.8-11.7] vs 8.1 [7.4-8.9] months, P<0.01; 2-years: 20.5 [18.6-22.4] vs 13.7 [11.9-15.5] months, P<0.01). RMST (95% CI) for rwPFS for BV and OST at 1 year was 11.1 (10.5-11.6) vs 8.9 (8.1-9.6) months (P<0.01) and at 2 years was 21.0 (19.1-22.9) vs 15.7 (13.6-17.8) months (P<0.01) (Table 2). Median OS was NR in both cohorts; for BV and OST, 9% and 36% of patients died. RMST (95% CI) for OS for BV and OST at 1 year was 11.3 (10.9-11.7) vs 9.0 (8.3-9.7) months (P<0.01) and at 2 years was 21.3 (19.7-22.8) vs 15.8 (14.1-17.5) months (P<0.01). During the observation period, 30.2% and 39.0% of the BV and OST cohorts had an emergency department visit, and 29.5% and 36.6% had a hospitalization; median (95%CI) hospital length of stay for those with ≥1 hospitalization was 13.7 (CI: 12.6-14.9) and 21.1 (19.9-22.4) days. CONCLUSION: Real-world outcomes with BV in patients with CTCL who received ≥1 prior systemic therapy were consistent with results from ALCANZA, demonstrating favorable clinical outcomes with BV. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Real-World Patient Characteristics, Utilization Patterns, and Outcomes of US Patients with HR+, HER2- Metastatic Breast Cancer Treated with Abemaciclib.

Abemaciclib is the most recent oral cyclin-dependent kinase 4 and 6 inhibitor (CDK4 & 6i) to receive US Food and Drug Administration (FDA) approval to treat hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer (MBC). Administrative claims data were used to describe patient characteristics and select clinical and economic outcomes in US patients treated in routine clinical practice. Prior analyses from electronic health records data indicate approximately 25% of patients received either palbociclib or ribociclib for MBC before initiating abemaciclib treatment; this work further explored these findings and associated outcomes. This retrospective study analyzed medical and pharmacy claims from the IBM® MarketScan® Research Databases between 1 January 2007 to 31 January 2020. Patients with HR+, HER2- MBC newly initiating abemaciclib between 1 September 2017 and 31 October 2019 were included and grouped by concomitant therapy (+aromatase inhibitor (AI), +fulvestrant (F), 200mg abemaciclib monotherapy (Mono), or +other), and outcomes were analyzed by prior CDK4 & 6i use. Patient and treatment characteristics were summarized with descriptive statistics. Kaplan-Meier methods assessed time-to-discontinuation (TTD; i.e., persistency) and time-to-chemotherapy (TTC). Adherence (defined by the medication possession ratio) and drug wastage were determined. This analysis included 454 patients (mean age 57.7years), with 35.0% (n = 159) in the +F group, 29.3% (n = 133) in the +AI group, 10.4% (n = 47) in the 200 mg Mono group, and 25.3% (n = 115) in the +other group. Prior chemotherapy and CDK 4 & 6i use were present in 23.8% and 49.8% of all patients, respectively. Visceral metastases were present at abemaciclib initiation in 50.4% in the +AI group; 49.7% in the +F group; and 55.3% in the 200 mg Mono group. Liver metastases were present in 33.7% of the overall population. Among patients without prior CDK4 & 6i use, the median TTD for patients receiving abemaciclib + AI was not reached [95% CI 430-not reached (NR) days], abemaciclib + F [531 days (95% CI 281-NR)], and abemaciclib mono [141 days (95% CI 80-NR)]. Median TTC for abemaciclib + AI and abemaciclib + F groups were not reached and the median TTC for abemaciclib mono was 535 days (95% CI 181-NR). Medication adherence was 88.7% and medication wastage costs among those with at least one dose modification were $808.12 and $452.2 per patient per month based on amount paid and wholesale acquisition cost (WAC), respectively. Mean length of follow-up for all patients was 350 days (SD 187). These real-world data complement clinical trial results by examining abemaciclib use among patients treated in routine clinical practice. The sizeable number of patients treated with prior CDK4 & 6i, chemotherapy, and/or visceral metastases at abemaciclib initiation suggest that many patients had very advanced disease and/or were in later stages of their treatment. These data confirm a higher percentage of patients treated with previous CDK4 & 6i than reported previously, reinforcing the importance of the ongoing, prospective clinical trials evaluating outcomes following progression on CDK4 & 6i.

254P Real-world clinical scenarios during introduction of trastuzumab biosimilar for HER2-positive breast cancer

Trastuzumab (tras)-anns (KANJINTI™) is one of the first biosimilars of reference product (RP) Herceptin® approved in the European Union (EU) in May 2018, also approved in the United States in June 2019, for treatment of human epidermal growth factor receptor 2-positive (HER2+) early and metastatic breast cancer (BC) as well as metastatic gastric cancer. Study objectives were to define real-world characteristics of BC patients (pts) receiving tras-anns, their prior tras treatment patterns, and tras-anns safety.

1182P US real-world (RW) patient characteristics with METex14 skipping advanced non-small cell lung cancer (aNSCLC)

We evaluated the characteristics of adult patients (pts) with METex14 skipping aNSCLC using RW data predating the FDA approval of the two MET tyrosine kinase inhibitors (TKIs), tepotinib and capmatinib.

EP03.01-015 Disease-Free Survival and Clinical Characteristics in Early-Stage NSCLC Patients From a Danish Cohort

New and targeted treatment options in early-stage non-small cell lung cancer (NSCLC) patients have the potential to increase survival, but further characterization is needed to improve patient selection. Here, we present real-world patient characteristics, survival data, and disease-free survival (DFS) patterns of early-stage NSCLC patients treated in the county of Aarhus, Denmark.

Real-world patient characteristics and treatment outcomes among nontransplanted multiple myeloma patients who received Bortezomib in combination with Lenalidomide and Dexamethasone as first line of therapy in the United States

BackgroundThere is limited real-world evidence that describes patients with newly diagnosed multiple myeloma (NDMM) treated with the bortezomib, lenalidomide, and dexamethasone (VRd) triplet regimen. We evaluated patient characteristics and treatment outcomes among nontransplanted NDMM patients who received VRd as their first line of therapy (LOT) in US oncology practice settings.MethodsThis retrospective observational cohort study evaluated patients from the Flatiron MM Core Registry who received VRd as first LOT between November 1, 2015, and February 28, 2021. Progression-free survival (PFS) was analyzed using the Kaplan-Meier method. Associations between patient demographic and clinical characteristics and PFS were evaluated using a multivariable Cox proportional hazards model.ResultsA total of 2342 eligible patients with VRd as first LOT were identified (mean age, 67.0 years). Among all identified patients, 64.3% were ≥ 65 years of age, 25.5% were elderly (≥75 years), and 47.9% were frail. Among patients with available data, 21.2% had high-risk cytogenetics, and the majority had International Staging System (ISS) stage I/II disease (71.8%), and Eastern Cooperative Oncology Group performance status (ECOG PS) score 0/1 (81.2%). Median duration of therapy was 5.5 months. With median follow-up of 21.0 months, median PFS and time-to-next-treatment were 26.5 and 16.1 months, respectively. Higher risk of disease progression or death was seen in patients categorized as elderly (hazard ratio [HR] = 1.37; 95% confidence interval [CI]: 1.13-1.66 vs patients < 65 years), having high-risk cytogenetics (HR = 1.44; 95% CI: 1.19-1.75 vs standard risk), having ISS disease stages II and III (HR = 1.31; 95% CI: 1.06-1.63 and HR = 1.37; 95% CI: 1.10-1.70 versus stage I, respectively), and having worse ECOG PS score (≥2) (HR = 1.49; 95% CI: 1.22-1.81 versus functionally active patients).ConclusionsThe majority of patients treated with VRd in this study were ≥ 65 years of age, were ISS stage I/II, had an ECOG PS score of 0/1, and had standard cytogenetic risk. Median PFS observed in real-world practice was notably shorter than that observed in the SWOG S0777 clinical trial. In nontransplanted patients treated with VRd as first LOT, a higher risk of disease progression or death was associated with older age, having high-risk cytogenetics, worse disease stage, and worse ECOG PS score.

Characterization of Inclisiran Use in Real World: Prescription Data from Germany

<h3>Lead Author's Financial Disclosures</h3> Raquel Lahoz is an employee of Novartis Pharma AG, Basel, Switzerland, and owns Novartis shares. <h3>Study Funding</h3> None. <h3>Background/Synopsis</h3> Despite the availability of effective low-density lipoprotein cholesterol (LDL-C) lowering drugs, ∼80% of patients with established ASCVD fall short of achieving and maintaining LDL-C levels <70 mg/dL, highlighting the need for innovative new LDL-C lowering therapies. Inclisiran is a novel small-interfering RNA (siRNA) that effectively reduces circulating LDL-C. Administered as a single subcutaneous injection initially, again at 3 months, and then every 6 months, inclisiran has been approved by the European Medicines Agency (EMA) in December 2020 for adult patients with hypercholesterolemia as an adjunct to diet who require additional lowering of LDL-C despite receiving other lipid-lowering therapies and by the Food and Drug Administration (FDA) in December 2021. <h3>Objective/Purpose</h3> This study aimed to assess real-world patient characteristics, adherence and persistence for patients being initiated with inclisiran in Germany. <h3>Methods</h3> This retrospective non-interventional study used the IQVIA LRx prescription database and included adult patients being initiated with inclisiran treatment between February 2021 and October 2021 with a 6-month lookback period. For the adherence and persistence analyses, a subgroup of patients with a first inclisiran prescription recorded between February and June 2021 was selected, allowing for at least 4 months follow-up, up to October 2021. <h3>Results</h3> A total of 697 patients receiving at least one dose of inclisiran between February- October 2021 were included in this study. Mean age of patients was 63 years (50% females) and 81% were ≥ 55 years old. Majority of patients were prescribed inclisiran by their nephrologists (42%) and cardiologists (30%). Of the 334 patients receiving their first inclisiran dose between February-June 2021, the second dose was also recorded for 257 (77%) patients until the time of the analysis; median time between the two doses was 3 months and 91% (233) of patients received the second dose within 4 months of treatment initiation. <h3>Conclusions</h3> This study shows early results from one of the first real-world studies describing the characteristics of patients being initiated with inclisiran with majority returning for their second dose within 4 months. Future real-world studies with longer follow-up are needed to evaluate the long-term adherence and persistence as well as effectiveness of inclisiran.

CO24 Real-World Patient Characteristics, Efficacy and Safety of Psychiatric Intramuscular Ketamine Treatment: A Retrospective Cohort Study of 452 Outpatients

Ketamine has emerged as a promising pharmacotherapy for depression and other mental illnesses, and the intramuscular (IM) administration of ketamine is now offered at many North American outpatient psychiatric clinics. However, a characterization of the outpatient population receiving IM ketamine treatment, and an evaluation of the real-world efficacy and safety of long-term IM ketamine treatment, has not been reported.

P1057: REAL-WORLD UTILIZATION OF FEDRATINIB FOR MYELOFIBROSIS FOLLOWING RUXOLITINIB FAILURE

Background: In August 2019, fedratinib (FEDR) became the second treatment, after ruxolitinib (RUX), to be approved for intermediate (Int)- or high-risk (HR) primary or secondary myelofibrosis (MF). Aims: To characterize real-world patient characteristics and treatment patterns for patients with MF receiving FEDR after RUX therapy in US clinical practices. Methods: Adults with Int- or HR MF initiating FEDR on or after August 16, 2019 (FEDR approval date) after discontinuing RUX were identified from community oncology practices. Eligible patients had ≥90 days of follow-up from FEDR initiation, completed ≥1 FEDR cycle, and had a spleen size assessment at FEDR initiation. Treating physicians completed electronic case report forms for the selected eligible patients, capturing patient characteristics at time of MF diagnosis and during RUX and FEDR therapy, including bone marrow fibrosis grade, biomarkers, risk score, performance status score, spleen size (cm above the costal margin), symptoms (per Modified Myelofibrosis Symptom Assessment Form version 4.0), and complete blood count data. Providers reported treatment dosing and modifications and dates/rationale for RUX treatment failure (defined a priori, including refractory or suboptimal response, disease progression [PD], or intolerance). Results: Among 150 eligible patients, the median age at diagnosis was 68 years; 55% were male, 68% were non-Hispanic White, 85% had primary MF, 55% were JAK2 V617F-positive, and 9% were JAK2/MPL/CALR-negative. RUX initiation dosage was ≥20 mg twice/daily (bid) for approximately 50% of patients. At RUX initiation, 65% had ECOG scores of 0/1, and 90% had palpable spleen (Table). A total of 19 patients (13%) had RUX dosage reductions due to neutropenia (7), thrombocytopenia (6), other adverse event (AE)/toxicity (3), patient request (2), and anemia (1); 4 patients (3%) had treatment interruptions due to thrombocytopenia (2), neutropenia (1), other AE/toxicity (1), and patient request (1); and 18 patients (12%) had dosage increases due to titration (13) and persistent MF symptoms (5). Primary reasons for RUX discontinuation were PD (70%), refractory or suboptimal response (25%), or intolerance (5%). RUX discontinuation due to PD was most often attributed to MF symptom return (65%), increased spleen size to >50% vs best achieved response (23%), and other reasons (12%). Median RUX duration was 4.0 months (IQR, 3.0–6.1) for refractory or intolerant patients and 11.7 months (IQR, 6.5–17.1) for those with PD. FEDR was initiated at 400 mg daily for 74% of patients. At FEDR initiation, 43% had International Prognostic Scoring System (IPSS)/Dynamic IPSS HR, 37% Int-2 risk, and 88% had palpable spleen; mean spleen size was 16.0 cm, and median platelet count was 98.0×109/L (Table). Symptoms reported at FEDR initiation included fatigue (72%), abdominal discomfort (51%), night sweats (44%), early satiety (25%), bone pain (18%), itching (13%), and pain under the left rib (13%). At the end of follow-up, 44 patients (29.3%) were deceased, and mean time to death among deceased patients was 6.5 (standard deviation, 2.7) months from initiation of FEDR. Image:Summary/Conclusion: This study showed a short duration of RUX therapy for real-world patients with MF, and poorer clinical status at FEDR initiation than at RUX initiation. Patients at risk of early failure of RUX should be considered for earlier FEDR interventional treatment.

Real-World Patient Characteristics, Treatment Patterns, and Mutation Testing Patterns Among US Patients with Advanced Non-Small Cell Lung Cancer Harboring EGFR Mutations.

Approximately 15% of patients with non-small cell lung cancer (NSCLC) harbor an epidermal growth factor receptor mutation (EGFRm). Mutation testing (including EGFRm) is recommended for patients with advanced NSCLC (aNSCLC) prior to initiating first-line therapy (L1) or after progression on targeted therapy. To elucidate current and future unmet needs, the present study characterized real-world EGFR testing patterns and treatment patterns in patients with aNSCLC. This retrospective observational cohort study evaluated newly diagnosed adult patients with aNSCLC (stageIIIB or higher) from the Flatiron Health database. Eligible patients received at least L1 during 2015-2020 (testing cohorts) or 2011-2020 (treatment cohorts). Eligible patients for the treatment cohorts had an EGFR mutation. The testing cohort included 22,726 patients, 75.5% had at least one EGFR test and 15.2% of those tested were positive for EGFR mutation. From 2015 to 2020, the median time from sample collection to test results decreased substantially while the proportion of NGS EGFR tests and use of plasma samples increased. The treatment cohort included 3701 patients (95% common mutations [cEGFR], 5.0% exon20 insertions [ex20ins]). Three or more lines of therapy (LOTs) were observed in approximately 30% of patients. For L1, most cEGFR patients received EGFR tyrosine kinase inhibitors (EGFR-TKI, 68.1%) or platinum-based chemotherapy (PBC, 24.8%); most ex20ins patients received PBC (66.1%) or EGFR-TKI (17.5%). The most common L2 was EGFR-TKI (54.1%) or PBC (22.8%) for cEGFR and immunotherapy (25.9%) or PBC (25.9%) for ex20ins. No predominant L3 was evident in either group. This real-world study, among the largest analyses of testing patterns for patients with aNSCLC, demonstrates a comprehensive view of treatment patterns for patients with EGFR mutations, including ex20ins. Despite recent improvement, increased use of EGFR testing, including advanced methods, is needed to optimize treatment pathways and outcomes. Additionally, the lack of a predominant therapy in later lines indicates a need for new therapies.

190P Palbociclib treatment in pre/perimenopausal women with advanced/metastatic breast cancer (ABC/mBC): Real-world patient characteristics, treatment patterns, and outcomes data from POLARIS

Palbociclib (PAL), in combination with an aromatase inhibitor (AI) as initial endocrine-based therapy (ET) or with fulvestrant (FUL), is indicated for the treatment of hormone receptor–positive/human epidermal growth factor receptor 2–negative (HR+/HER2–) ABC/mBC. This analysis describes real-world patient (pt) characteristics, treatment patterns, and preliminary tumour response in pre/perimenopausal women with HR+/HER2– ABC/mBC enrolled in the POLARIS study.