Real-world Utilization Patterns Research Articles

Real world comparison of filgrastim to filgrastim-sndz in patients with chemotherapy-induced neutropenia.

There exists some apprehension by prescribers, healthcare providers, and other stakeholders regarding the real-world safety and effectiveness of biosimilars. While some of the apprehension is likely due to clinician knowledge gaps in the biosimilarity exercise, additional data (including those generated from the real-world) regarding safety and efficacy could reduce a clinician's perception of biosimilar uncertainty and can potentially increase biosimilar acceptance and uptake. The published literature is lacking regarding the real-world impact on healthcare costs and clinical outcomes when a single healthcare institution converts from filgrastim to filgrastim-sndz as its short-acting Granulocyte Colony Stimulating Factor (GCSF), especially within diverse populations and indications not explicitly studied through the registration trials. Specifically, both filgrastim and filgrastim-sndz possess FDA-approved indications within patients with hematologic malignancies receiving high-dose chemotherapy and in those undergoing bone marrow transplantation. As a biosimilar to filgrastim, the FDA did not require prospective, randomized controlled trials to obtain these indications for filgrastim-sndz. The purpose of this study is to describe real-world healthcare resource costs, utilization patterns, and clinical outcomes in patients with hematologic malignancies who received filgrastim-sndz or filgrastim to support neutrophil recovery following chemotherapy (i.e., induction or consolidation) or a bone marrow transplant (BMT) at Yale New Haven Hospital (YNHH). A total of 148 patients were identified and met the following criteria: at least 18 years old, Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2, absence of fever or infection, newly diagnosed acute myeloid leukemia or newly post-bone marrow transplant and received filgrastim-sndz (Zarxio®) or reference filgrastim (Neupogen®). Healthcare resource utilization outcomes were compared between biosimilar and reference filgrastim using descriptive statistics. There were no major differences between either cohort, including duration of hospitalization, the number of overall emergency room visits and additional hospital admissions with a primary diagnosis of febrile neutropenia or neutropenia within 30 days post-discharge from initial hospital admission, time to neutrophil recovery following GCSF support, and the incidence and duration of both fever and febrile neutropenia. The total cost based on treatment of all patients in each arm differed significantly with filgrastim-sndz being the most cost-effective choice. filgrastim-sndz significantly reduced healthcare utilization costs compared to reference filgrastim with similar effect on absolute neutrophil count, incidence of fever, and febrile neutropenia.

Computed tomography referral guidelines adherence in Europe: insights from a seven-country audit.

Ensuring appropriate computed tomography (CT) utilization optimizes patient care while minimizing radiation exposure. Decision support tools show promise for standardizing appropriateness. In the current study, we aimed to assess CT appropriateness rates using the European Society of Radiology (ESR) iGuide criteria across seven European countries. Additional objectives were to identify factors associated with appropriateness variability and examine recommended alternative exams. As part of the European Commission-funded EU-JUST-CT project, 6734 anonymized CT referrals were audited across 125 centers in Belgium, Denmark, Estonia, Finland, Greece, Hungary, and Slovenia. In each country, two blinded radiologists independently scored each exam's appropriateness using the ESR iGuide and noted any recommended alternatives based on presented indications. Arbitration was used in case auditors disagreed. Associations between appropriateness rate and institution type, patient's age and sex, inpatient/outpatient patient status, anatomical area, and referring physician's specialty were statistically examined within each country. The average appropriateness rate was 75%, ranging from 58% in Greece to 86% in Denmark. Higher rates were associated with public hospitals, inpatient settings, and referrals from specialists. Variability in appropriateness existed by country and anatomical area, patient age, and gender. Common alternative exam recommendations included magnetic resonance imaging, X-ray, and ultrasound. This multi-country evaluation found that even when using a standardized imaging guideline, significant variations in CT appropriateness persist, ranging from 58% to 86% across the participating countries. The study provided valuable insights into real-world utilization patterns and identified opportunities to optimize practices and reduce clinical and demographic disparities in CT use. Question Largest multinational study (7 EU countries, 6734 CT referrals) assessed real-world CT appropriateness using ESR iGuide, enabling cross-system comparisons. Findings Significant variability in appropriateness rates across institution type, patient status, age, gender, exam area, and physician specialty, highlighted the opportunities to optimize practices based on local factors. Clinical relevance International collaboration on imaging guidelines and decision support can maximize CT benefits while optimizing radiation exposure; ongoing research is crucial for refining evidence-based guidelines globally.

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.

Real-world drug utilization and treatment patterns in patients with tenosynovial giant cell tumors in the USA.

Aim: Real-world treatment patterns in tenosynovial giant cell tumor (TGCT) patients remain unknown. Pexidartinib is the only US FDA-approved treatment for TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Objective: To characterize drug utilization and treatment patterns in TGCT patients. Methods: In a retrospective observational study using IQVIA's linked prescription and medical claims databases (2018-2021), TGCT patients were stratified by their earliest systemic therapy claim (pexidartinib [N=82]or non-FDA-approved systemic therapy [N=263]). Results: TGCT patients treated with pexidartinib versus non-FDA-approved systemic therapies were predominantly female (61 vs 50.6%) and their median age was 47 and 54years, respectively. Pexidartinib-treated patients had the highest 12-month probability of remaining on treatment (54%); 34.1% of pexidartinib users had dose reduction after their first claim. Conclusion: This study provides new insights into the unmet need, utilization and treatment patterns of systemic therapies for the treatment of TGCT patients.

498 - Real-world use and effectiveness of upadacitinib in adults and adolescents with atopic dermatitis: preliminary analysis of the real-world multicountry AD-VISE study

Abstract Introduction & Objectives Upadacitinib (UPA) 15 and 30 mg once daily is approved for moderate to severe atopic dermatitis (AD) in several jurisdictions, including the European Union, Japan, Canada, and the United States, with indication/posology differences. Clinical studies have documented the safety and efficacy of UPA; however, real-world data on UPA are limited. The AD-VISE study objective is to characterize the real-world utilization patterns and effectiveness of UPA 15 and 30 mg in adults and adolescents with AD in clinical practice. Materials & Methods AD-VISE is an ongoing observational, prospective, multicountry study to assess the use of UPA in routine clinical practice for 2 years. Adult and adolescent patients (pts) receiving UPA for AD are being enrolled. Primary effectiveness outcome measures included validated Investigator Global Assessment for Atopic Dermatitis (vIGA-AD) 0/1 at month 4. Other outcome measures included Eczema Area and Severity Index (EASI), Worst Pruritus Numeric Rating Scale (WP-NRS), and Dermatology Life Quality Index (DLQI). This interim analysis (data cutoff February 27, 2023) reports baseline data and 2- and 4-month effectiveness data. Effectiveness analyses included pts that were enrolled at least 4 months by the data cutoff date or discontinued from the study; non-responder imputation with multiple imputation (NRI-MI) was used. Results Baseline analyses included 267 pts, of whom 21 (7.9%) were adolescents; effectiveness analyses included 209 patients (NRI-MI). Overall, 155 (58.1%) pts started on UPA 15 mg and 112 (41.9%) started on UPA 30 mg. Most pts in this analysis were from Canada (56.2%), followed by Russia (10.9%) and Australia (8.6%). Mean (SD) UPA exposure was 218.8 (±117.6) days; 5.6% of pts discontinued UPA and 3.7% discontinued the study. The most common reason for starting on UPA 15 mg was attempting lowest possible effective dose (42.9%) and on 30 mg, high disease burden/severity of skin symptoms (35.2%). More pts starting UPA 30 mg vs 15 mg were between 18 to <65 years old (96.4% vs 76.1%), male (61.6% vs 51.0%), had severe AD (vIGA-AD score 4: 53.6% vs 46.3%), had prurigo nodules (30.6% vs 18.4%), asthma (45.5% vs 24.5%), and prior biologic systemic therapy use (16.1% vs 10.3%) . More than half of the pts (51.2%) achieved vIGA-AD 0/1 at month 2, and this proportion increased to 61.3% at month 4. Similarly, rate of achieving EASI 90 (month 2: 46.9%; month 4: 57.2%) and DLQI 0/1 (month 2: 35.1%; month 4: 41.3%) increased, whereas rates were maintained for ≥4-point WP-NRS improvement (month 2: 68.1%; month 4: 69.3%). Similar results were observed with other endpoints. Conclusion Initial findings from the AD-VISE study, the largest study to report multicountry real-world data on UPA treatment patterns and effectiveness in AD, suggest that most pts achieved clear/almost clear skin and clinically meaningful itch improvement by month 4. Because this is a real-world observational study, it was expected that physicians start with UPA dose per local label. The main reason for starting UPA 30 mg was high disease burden/severity of skin symptoms, whereas 15 mg was started to attempt the lowest possible effective dose. More pts starting UPA 30 mg vs 15 mg were adults, male, and had prior biologic systemic therapy use.

Real-world utilization patterns of radium-223 in metastatic prostate cancer in the United States: An administrative claims database study.

79 Background: Radium (Ra)-223 is approved for use in men with symptomatic castration resistant prostate cancer (CRPC) with bone metastases, demonstrating benefits in terms of overall survival and quality of life with a reliable safety profile. In the rapidly evolving treatment landscape for prostate cancer, real-world (RW) data on Ra-223 utilization are limited. We aimed to examine the RW utilization patterns of Ra-223 in patients with metastatic prostate cancer using a large administrative claims database in the US. Methods: A retrospective cohort of men treated with Ra-223 was identified using the closed claims private payor database of the Komodo Research Dataset covering the period from 01/01/2017 to 06/30/2022. The earliest administration of Ra-223 in men with bone metastatic prostate cancer was considered as index date. Individuals were required to have continuous insurance coverage at least 12-month pre-index (baseline) and least 6-month post index or until death if patients survived < 6 months (follow-up). Logistic regression was used to identify baseline demographic, clinical, medications, or healthcare resource use-related factors associated with receiving 5+ cycles of Ra-223. Results: The study cohort comprised of 1,376 men with a median age of 68 years at the time of Ra-223 initiation. Most men were treated by oncologists (52.2%), having bone only metastases (51.5%), having Charlson comorbidity index (CCI) ≥ 1 (76.2%). Diabetes (34.1%), peripheral vascular disease (29.9%), pulmonary disease (25.9%) and mild liver disease (25.1%) were the most common pre-existing comorbidities. Therapies received prior to Ra-223 were androgen receptor pathway inhibitors (75.4%), chemotherapy (32.1%), sipuleucel-T (13.7%) and bone health agents (73.7%). Ra-223 was utilized as a combination therapy by 26.0% of the overall population, predominately with enzalutamide. Nearly 15%, 38%, and 31% received Ra-223 (mono or combination therapy) as 1st, 2nd, and 3rd line therapy, respectively. Approximately 46% of the overall cohort and 54% of those who survived ≥ 6 months had received 5+ cycles, respectively. In regression analysis, men with short survival (<6 month) [Odds ratio, OR: 0.05, 95% confidence interval (CI): 0.03, 0.10], low CCI (0 vs. 4+) (OR: 0.38, 95% CI:0.19, 0.76), and pulmonary disease (OR: 0.71, 95% CI: 0.52, 0.97) had lower likelihood of completion of 5+ cycles. Conclusions: Nearly half of men completed 5+ cycles of Ra-223 therapy in a large US cohort of relatively young mCRPC men with private health insurance. Advanced disease state with short survival and low comorbidity were key factors associated with lower completion of 5+ cycles. The findings highlight the importance of utilizing Ra-223 early after mCRPC diagnosis, when the probability of >6-month survival is higher, to enhance treatment completion that has been shown to result in better outcomes.

Temporal trends of antidepressant utilization patterns in children and adolescents in Hong Kong: A 14-year population-based study with joinpoint regression analysis

BackgroundThere is limited research on real-world antidepressant utilization patterns in children and adolescents, particularly in non-western countries. We aimed to examine temporal trends of antidepressant prescribing practice among Chinese children and adolescents in Hong Kong over 14-year period. MethodsThis population-based study identified 9566 patients aged 5–17 years who had redeemed at least one antidepressant prescription within 2005–2018, using data from health-record database of Hong Kong public healthcare services. We calculated annual prescription rates (per 1000 persons) for any antidepressant, antidepressant drug classes, and individual antidepressants. Joinpoint-regression analyses were performed to assess temporal antidepressant prescription trends, quantified by average annual-percent-change (AAPC), with 95 % confidence-intervals (CIs). ResultsOverall antidepressant prescription rate significantly increased over time (AAPC: 7.30 [95 % CI: 6.70–7.90]), from 3.883 in 2005 to 9.916 in 2018. The use of selective-serotonin-reuptake-inhibitors (SSRIs), serotonin-norepinephrine-reuptake-inhibitors (SNRIs), and other antidepressants significantly increased over 14 years, while tricyclic-antidepressants remained stable. SSRI represented the most commonly-prescribed drug class. Fluoxetine and sertraline constituted the two most frequently-prescribed individual antidepressants, while desvenlafaxine (AAPC: 55.68 [30.74–85.39]) and bupropion (AAPC: 35.28 [23.68–47.98]) exhibited the sharpest increase in prescription rates over the study period. LimitationsMedication adherence could not be assessed and actual drug use may be overestimated. ConclusionsOur results affirm a significant rising trend of antidepressant prescriptions among Chinese children and adolescents over time. All antidepressant drug-classes, except TCA, demonstrated significantly increased use, with SSRI being the most frequently-prescribed drug class. Future investigation should clarify indications, hence off-label use, of antidepressant initiation in this vulnerable population.

Botulinum Toxin Utilization, Treatment Patterns, and Healthcare Costs Among Patients with Spasticity or Cervical Dystonia in the US.

Spasticity and cervical dystonia (CD) are movement disorders with considerable direct and indirect healthcare cost implications. Although several studies have discussed their clinical impact, few have calculated the economic burden of these disorders. This study aimed to understand treatment/injection patterns of botulinum toxins type A (BoNT-As) and the characteristics, healthcare resource utilization (HCRU), and costs among patients with spasticity or CD. Retrospective analyses were conducted using administrative healthcare claims from the IQVIA PharMetrics® Plus database, from October 1, 2015 to December 31, 2019. Eligible patients were selected based on Healthcare Common Procedure Coding System codes for BoNT-A (index date) and ICD-10 diagnosis codes for spasticity or CD with 6months of continuous enrollment pre-index and 12months post-index. Patients were stratified into adult spasticity, pediatric spasticity, and CD cohorts, and were evaluated for injection patterns, HCRU, and costs in the post-index period. Overall, 2452 adults with spasticity, 1364 pediatric patients with spasticity, and 1529 adults with CD were included. Total mean all-cause healthcare costs were US$42,562 (adult spasticity), $54,167 (pediatric spasticity), and $25,318 (CD). Differences were observed in the cost of BoNT-A injection visits between toxins, with abobotulinumtoxinA (aboBoNT-A) having the lowest injection cost across all indications. AboBoNT-A had the lowest injection visit costs across indications. These results are suggestive of real-world resource utilization patterns and costs, and, while helpful in informing insurers' BoNT-A management strategies, further research into cost differences is warranted.

Analysis of Dispensing and Utilization Patterns of Medical Cannabis Products in State Licensed Cannabis Dispensaries.

Background: To understand real-world dispensing and utilization patterns of medical cannabis (MC) and its financial impact on patients, we analyzed the database of a cannabis company licensed in New York state. Objectives: To evaluate the tetrahydrocannabinol (THC)/cannabidiol (CBD) dose ratios, association of various medical conditions to THC/CBD dose, and the cost of products in registered patients receiving MC from four licensed state dispensaries. Design: Retrospective analysis conducted on anonymized data between January 1, 2016 and December 31, 2020 listing 422,201 dispensed products from 32,845 individuals aged 18 years and older. Setting/Subjects: Adult patients with medical certification for cannabis use in New York, USA. Measurements: The database included patient's age, gender, qualifying medical condition, type and dose of product, medication directions, and amount of product dispensed. Results: Findings showed a median age of 53 years with 52% of patients female. Males were found to use more products than females (1.06:1). Pain (85%) was the most common medical condition and inhalation (57%) the most common route except when used for cancer-directed treatment and neurological conditions. Individuals received a median of six prescriptions costing a median of $50/product. Average THC:CBD ratios were 28:0.5 mg/day and 12:0.25 mg/dose. Neurological conditions demonstrated the highest average cost [mean (95% confidence interval {CI}): $73 ($71-$75)] and average CBD/dose per product [mean (95% CI): 5.89 (5.38-6.40)]. Individuals with a history of substance use disorder utilizing MC as an alternative substance displayed the highest average THC/dose [mean (95% CI): 14.25 (13.36-15.14)]. Conclusion: MC was utilized for various medical conditions with variability in THC:CBD ratio seen depending on the condition. Cost variation was also observed based on the individual's medical condition.

Real world utilization and outcomes of immunotherapy regimens in metastatic castration resistant prostate cancer (mCRPC).

e17038 Background: While immune checkpoint inhibitors (ICIs) have shifted the treatment paradigm in many cancers, their efficacy in mCRPC is limited. Ongoing research suggests distinct subpopulations of mCRPC may derive benefit or that combinatorial regimens including ICIs may overcome treatment resistance. This single-center retrospective study aims to describe real-world utilization patterns of ICIs for patients with mCRPC and assess the treatment effects. Methods: The EHR database was queried for patients with PC who were treated with an ICI outside of a clinical trial. Additional data were obtained from chart review and the cancer registry. Patients were excluded if ICI was given for another cancer type, non-metastatic disease, or as a component of small-cell lung cancer regimens. Kaplan-Meier curves were used to estimate time to events. Results: 31 patients are included in the study. From years 2016 to 2022, 13 patients received ICI alone. Starting in 2019, 18 patients received ICI combined with cabozantinib (C). ICI+C had PSA response rate (RR) of 50%, median duration of treatment (DOT) of 5.5 months, and median overall survival (OS) of 8 months (Table 1). 3 of these patients received therapy for ≥ 12 months. ICI alone had a RR of 0%. 94% of patients who received ICI+C were not a candidate for CONTACT-2 (NCT04446117). Conclusions: Oncologists continue to use ICI for mCRPC. While noncomparative, ICI+C demonstrated favorable activity over a historical cohort of ICI alone. Ongoing clinical trial participation is vital for patients with mCRPC; however, off-label prescribing may provide additional access to treatment options. [Table: see text]

Monoclonal antibody utilization in medical college, Kolkata: A retrospective study

Background: The introduction of monoclonal antibodies (mAbs) in therapeutics has permitted targeted treatments for various diseases. As the utilization of mAbs continues to grow, understanding their drug utilization patterns becomes crucial for optimizing treatment strategies, improving patient outcomes, and ensuring cost-effectiveness in health-care systems. However, there is limited information on the real-world utilization patterns of mAbs in clinical practice. This retrospective study aims to analyze and summarize current knowledge on the drug utilization of mAbs. Aims and Objectives: The primary objective of the study is to analyze the utilization of mAbs across four departments (medical oncology, hematoncology, radiation oncology, and rheumatology) of Medical College, Kolkata. The secondary objective is to study the cumulative change in trend in the utilization of mAbs in these departments over the last 3 years (2020–2023). Materials and Methods: A retrospective analysis was done by collecting relevant data on mAb use in four disciplines (medical oncology, hematoncology, radiation oncology, and rheumatology) from electronic health records and pharmacy databases. The study included patients who received mAb therapy between June 01, 2022, and May 31, 2023. The collected data included demographic data of the patients and data on mAb, including utilization patterns (frequency, duration, and indications for mAb use). Changes in the pattern of usage of mAbs, if any, across the preceding 3 years (2020-21, 2021-22, 2022-23), were also assessed. Descriptive data has been represented in percentages. Results: The commonest indications of mAbs across the four departments were chronic lymphocytic leukemia, breast carcinoma, and ovarian carcinoma in hemt-oncology, radiation oncology, and medical oncology departments, respectively. Rituximab, trastuzumab, and bevacizumab were identified as the most frequently prescribed mAbs in the mentioned departments. It was also observed that total consumption of mAbs has significantly increased over the last 3 years. Conclusion: This retrospective study provides insights into the drug utilization patterns of mAbs in a real-world clinical setting.

Real-World Utilization Patterns of Intravenous Immunoglobulin in Adults With Generalized Myasthenia Gravis in the United States

Objective To evaluate real-world utilization patterns of intravenous immunoglobulin (IVIg) among patients with generalized myasthenia gravis (gMG) over 3 years post-IVIg initiation. Background gMG is a rare autoimmune neuromuscular disorder with no known cure. Although IVIg is the most commonly used add-on therapy after standard of care treatments in gMG, it is currently unclear whether it is more commonly used as a “one-off” treatment to manage exacerbations, or as maintenance therapy aimed at reducing or replacing steroid use. Design/Methods Patients with gMG who initiated IVIg treatment were identified from a US claims database (Symphony Health, an ICON plc Company, Integrated Dataverse [IDV]®, January 1, 2014-December 31, 2019). The frequency of subsequent IVIg treatment and associated cost during the first 12 months post-IVIg initiation were analyzed. Usage patterns of concomitant gMG treatments during the year preceding and 3 years post-IVIg initiation were compared. Results Among 1225 patients with gMG who initiated IVIg treatment, 706 patients (57.6%) received 1 to 5 IVIg treatment courses (intermittent IVIg users), and 519 patients (42.4%) received = 6 IVIg treatment courses (chronic IVIg users) within the subsequent year. Mean annual medical cost per patient was nearly 2.5-fold higher for chronic vs intermittent IVIg users ($161,478 vs $64,888, p < 0.001). Usage frequency of other immunotherapy treatments did not decrease over the follow-up period, even for patients who continued annual chronic IVIg for 3 consecutive years post-initiation. Conclusions Nearly half of patients with gMG received chronic and multiple IVIg treatment courses within the first year once initiating IVIg treatment, indicating higher usage than expected. For all IVIg initiators, usage frequency of other immunotherapy treatments did not decrease over 3 years despite IVIg initiation.

The Safety and Efficacy of Fostamatinib in Patients with Chronic Immune Thrombocytopenic Purpura Treated in a Real-World Community Hematology Setting

Background: Chronic immune thrombocytopenia (ITP) in adults is a serious autoimmune disease in which platelets are prematurely destroyed, leaving the patient vulnerable to bruising and bleeding. Initial treatment with corticosteroids is intended to suppress the immune response and is effective in many patients but not sustainable. Fostamatinib is an approved therapy for adult patients with ITP who have had an insufficient response to a prior treatment. It is an oral small molecule that targets spleen tyrosine kinase (SYK), which has important roles in cellular proliferation, differentiation, and immune regulation. By blocking SYK, fostamatinib reduces the immune system's destruction of platelets, which allows the platelet (PLT) count to rise, thereby reducing the risk of bleeding in ITP patients. Two placebo-controlled randomized trials demonstrated that fostamatinib was well tolerated and able to induce meaningful platelet responses in heavily pretreated patients, as well as in earlier lines of therapy. The use of fostamatinib in a community hematology setting has not been evaluated. In this study, the real-world utilization and treatment patterns of fostamatinib in patients treated within the QCCA, a network of 17 community hematology clinics across the United States, is presented. Methods: The QCCA network real world database was reviewed for ITP patients who had received fostamatinib over the past 3 years. Data collection consisted of patient demographics, disease characteristics, duration of ITP, existing comorbidities, number and type of prior ITP treatments and PLT count at baseline prior to the start of fostamatinib. All of the information was collected via a standardized data collection form. From the first day until the end of treatment, data were collected on hemoglobin, white blood cells, absolute neutrophil counts, PLT counts, concomitant ITP therapies and the use of rescue IVIG. A meaningful increase in PLTs was defined as achievement of platelet counts ≥ 30 and ≥ 50 x 103/μL over the course of therapy. Results: The medical records of the 46 ITP patients who met the inclusion criteria and received fostamatinib were evaluated. Patients had a median age of 58 years, 65% were female and 35% had a splenectomy. All patients had prior exposure to corticosteroids and the median number of therapies before stating fostamatinib was two. In addition, 65% had received thrombopoietin receptor agonists and rituximab. All patients started therapy at a dose of 100 mg twice daily for a median duration of 2 months (interquartile range [IQR] = 1 to 15) and a mean duration of 6.7 months (95%CI: 3.5 to 9.9). When fostamatinib was started, the median PLT count was 19 x 103/μL (IQR = 3 to 70). From the start of therapy, the maximum increase in the median PLT count was 78 x 103/μL (IQR = 13 to 231), which was achieved after a median of 1.8 months. Over the course of therapy, 31 (67.4%) and 26 (56.5%) of 46 patients achieved or maintained PLT levels ≥ 30 and ≥ 50 x 103/μL respectively. Of note, prior to the start of fostamatinib, 9 and 7 patients respectively, already had PLT levels ≥ 30 and ≥50 x 103/μL. The percent of patients with at least a doubling of their baseline PLT count was 47.8% (95%CI: 32.9 to 63.0%). There were no serious bleeding events reported, and only one patient required the discontinuation of fostamatinib because of adverse events. Among the 46 patients, 16 received concomitant IVIG to increase PLT counts. Conclusions: In this real-world setting, the findings suggest that fostamatinib is safe and effective in patients with chronic ITP who had relapsed or had not responded to one or more prior treatments.

Real-world utilization patterns of intravenous immunoglobulin in adults with generalized myasthenia gravis in the United States

ObjectiveTo evaluate real-world utilization patterns of intravenous immunoglobulin (IVIg) among patients with generalized myasthenia gravis (gMG) over 3 years post-IVIg initiation. MethodsPatients with gMG who initiated IVIg treatment were identified from a United States claims database (Symphony Health's Integrated Dataverse [IDV]®, January 1, 2014 − December 31, 2019). The frequency of subsequent IVIg treatment and associated cost during the year post-IVIg initiation were analyzed. Usage patterns of IVIg and concomitant gMG treatments during the year preceding and 3 years post-IVIg initiation were compared. ResultsAmong 1225 patients with gMG who initiated IVIg treatment, 706 patients (57.6%) received 1 to 5 IVIg treatment courses (intermittent IVIg users), and 519 patients (42.4%) received ≥6 IVIg treatment courses (chronic IVIg users) within the subsequent year. Mean annual medical cost per patient was nearly 2.5-fold higher for chronic vs. intermittent IVIg users ($161,478 vs. $64,888, p < 0.001). The proportion of patients using corticosteroids and nonsteroidal immunosuppressive treatments (NSISTs) was not reduced over the 3-year follow-up period following IVIg initiation, even for patients who continued annual chronic IVIg for 3 consecutive years post-initiation. ConclusionsNearly half of patients with gMG received chronic and multiple IVIg treatment courses within the first year once initiating IVIg treatment, indicating higher usage than expected. For all IVIg initiators, the proportion of patients using corticosteroids and NSISTs did not decrease over 3 years despite IVIg initiation.

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.

EP14.05-013 Real-World Resource Use and Costs by Stage and Platinum Sensitivity among Medicare Patients with Small Cell Lung Cancer

There is no information available about the relationship between platinum sensitivity and real-world cost and utilization patterns among patients with small cell lung cancer (SCLC) in the United States.

EP05.01-002 Real World Treatment Patterns and Outcomes among Unresectable Stage III Non-Small Cell Lung Cancer Patients Initiating Chemoradiotherapy

Concurrent platinum-based chemoradiation therapy (cCRT) with or without durvalumab consolidation for 12 months is the current standard of care for patients with unresectable stage III Non-Small Cell Lung Cancer (NSCLC). This study characterized the real-world treatment utilization patterns and associated outcomes after approval of durvalumab for this indication.

RWD13 Real-World Triptan Utilization Patterns for Acute Treatment of Migraine Among 15 Million Commercially Insured Lives

Migraine is a disabling disease impacting quality of life, productivity, and health care costs. Triptans are recommended as initial therapy for moderate/severe acute treatment of migraine. Limited real-world data assessing triptan utilization patterns among new initiators is available prior to approval of calcitonin-gene-related peptide inhibitors (CGRPs). The objectives are to describe new triptan initiators’ utilization, treatment switch and discontinuation, patterns in commercially insured members using claims data.

HSD116 Real-World Utilization Patterns of Oncology Biosimilar Monoclonal Antibodies and Their Corresponding Reference Products

Multiple oncology biosimilar monoclonal antibodies (mAbs) are now available for use as therapeutic options with comparable safety/efficacy profiles to that of their reference biologics. The objective of this study was to describe real-world treatment pattens of rituximab-pvvr, trastuzumab-qyyp, and bevacizumab-bvzr and their associated reference biologics.

Informative presence bias in analyses of electronic health records-derived data: a cautionary note.

Electronic health record (EHR)-derived data are extensively used in health research. However, the pattern of patient interaction with the healthcare system can result in informative presence bias if those who have poorer health have more data recorded than healthier patients. We aimed to determine how informative presence affects bias across multiple scenarios informed by real-world healthcare utilization patterns. We conducted an analysis of EHR data from a pediatric healthcare system as well as simulation studies to characterize conditions under which informative presence bias is likely to occur. This analysis extends prior work by examining a variety of scenarios for the relationship between a biomarker and a health event of interest and the healthcare visit process. Using biomarker values gathered at both informative and noninformative visits when estimating the effect of the biomarker on the event of interest resulted in minimal bias when the biomarker was relatively stable over time but produced substantial bias when the biomarker was more volatile. Adjusting analyses for the number of prior visits within a fixed look-back window was able to reduce but not eliminate this bias. These results suggest that bias may arise frequently in commonly encountered scenarios and may not be eliminated by adjusting for prior visit intensity. Depending on the context, the estimated effect from analyses using data from all visits available may diverge from the true effect. Sensitivity analyses using only visits likely to be informative or noninformative based on visit type may aid in the assessment of the magnitude of potential bias.