Healthcare resource utilization and costs during first salvage therapy for relapsed or refractory acute myeloid leukemia in the United States.

Real-world US healthcare resource utilization (HRU) and costs during first salvage therapy for relapsed/refractory (R/R) acute myeloid leukemia (AML) are described using IBM MarketScan® data (1/1/2007-6/30/2020). Treatments included high- (HIC) and low-intensity chemotherapy (LIC) alone, and gilteritinib, other FLT3 tyrosine kinase inhibitors (TKIs), and venetoclax with or without chemotherapy. Patients were diagnosed with R/R AML at ≥18 years of age between 1/1/2017-12/31/2019. Patient monthly all-cause HRU and costs were analyzed using a fixed-effects model. Data from 399 patients were analyzed (HIC, n = 104; LIC, n = 133; gilteritinib, n = 14; other FLT3 TKIs, n = 68; venetoclax, n = 80). Inpatient HRU was generally highest with HIC, whereas outpatient HRU was generally highest with LIC and venetoclax. Total all-cause incremental monthly costs appeared to be highest with HIC ($171,982) and similar for LIC ($60,512), gilteritinib ($47,218), other FLT3 TKIs ($43,218), and venetoclax ($77,566). Results highlight HRU and cost differences for R/R AML during first salvage therapy.

Pediatric Lung Transplant Outcomes Based on Immunosuppressive Regimen at Discharge: Retrospective Cohort Study Using Real-World Evidence From the US Scientific Registry of Transplant Recipients.

BACKGROUND: This retrospective analysis of the US Scientific Registry of Transplant Recipients was undertaken to obtain real-world evidence concerning the efficacy and safety of tacrolimus-based immunosuppression in pediatric lung transplant recipients to support a supplemental New Drug Application. METHODS: Overall, 725 pediatric recipients of a primary deceased-donor lung transplant between January 1, 1999, and December 31, 2017, were followed for up to 3 years post-transplant based on an immunosuppressive regimen at hospital discharge: immediate-release tacrolimus (TAC)+mycophenolate mofetil (MMF), TAC+azathioprine (AZA), cyclosporine (CsA)+MMF, or CsA+AZA. The primary outcome was the composite endpoint of graft failure or death (all-cause) at 1 year post-transplant, calculated by Kaplan-Meier analysis. RESULTS: The use of TAC+MMF increased over time. During 2010 to 2017, 91.7% of pediatric lung transplant recipients were receiving TAC+MMF at the time of discharge. The proportion of recipients continuing their discharge regimen at 1 year post-transplant was 83.7% with TAC+MMF and 40.4% to 59.7% with the other regimens. Cumulative incidence of the composite endpoint of graft failure or death at 1 year post-transplant was 7.7% with TAC+MMF, 13.9% with TAC+AZA, 8.9% with CsA+MMF, and 9.1% with CsA+AZA. There was no significant difference in the risk of graft failure or death at 1 year post-transplant between groups from 1999 to 2005 (the only era when adequate numbers on each regimen allowed statistical comparison). No increase in hospitalization for infection or malignancy was seen with TAC+MMF. CONCLUSION: The real-world evidence from the US database of transplant recipients supported the Food and Drug Administration's approval of tacrolimus-based maintenance immunosuppression in pediatric lung transplant recipients.

Treatment patterns, healthcare resource utilization and costs of HER2-negative gastric/gastroesophageal junction cancer.

Aim: To describe treatment patterns, healthcare resource utilization and costs in HER2-negative gastric/gastroesophageal (G/GEJ) cancer in the USA. Methods: Retrospective IQVIA Adjudicated Closed Claims database analysis; patients with HER2-negative G/GEJ cancer initiating systemic therapy between October 2016 and December 2019 were identified. Results: Among 1317 patients, platinum plus taxane regimens (54%) were common in neoadjuvant/adjuvant settings. Platinum-taxane (41%) and platinum-fluoropyrimidine (36%) regimens were common first-line therapies in the metastatic setting. Treatment regimens were heterogeneous in later lines. Hospitalization rates ranged from 39% (first-, second-line metastatic settings) to 61% (neoadjuvant/adjuvant setting). High healthcare resource utilization was driven by high outpatient costs. Mean per-patient per-month outpatient costs combining physician office and hospital visits ranged from US$10,944 to $12,582 (representing 52-70% of total costs). Conclusion: For G/GEJ cancer, systemic regimens varied across lines of therapy with variation increasing with subsequent therapy lines; high healthcare costs persist for systemic treatment, particularly outpatient services.

The Role of Physician Specialty in the Underutilization of Standard-of-Care Treatment Intensification in Patients With Metastatic Castration-sensitive Prostate Cancer.

PURPOSE: We evaluate utilization of treatment intensification of androgen deprivation therapy with androgen receptor pathway inhibitor/docetaxel for metastatic castration-sensitive prostate cancer patients across physician specialties. MATERIALS AND METHODS: This retrospective study identified patients with metastatic castration-sensitive prostate cancer in the Optum Research Database between 2014 and 2019. Adult men with ≥1 claim for metastatic disease within 90 days before or any time after the first prostate cancer claim who received androgen deprivation therapy were included. Physician specialty, determined from medical/pharmacy claims during each line of therapy, was categorized as urologist only, oncologist only, both (urologists and oncologists), or other (other specialties). Treatment intensification and patient characteristics were analyzed descriptively. RESULTS: Of 4,675 patients, 16% were treated by urologists only, 20% by oncologists only, 63% by both, and 1.1% by others. The most frequent first line of therapy was androgen deprivation therapy ± first-generation nonsteroidal antiandrogens (>50%). Androgen deprivation therapy + docetaxel use declined over time, while androgen deprivation therapy + androgen receptor pathway inhibitor use increased. Patients seen by oncologists or both were younger, had fewer comorbidities, and were likelier to receive treatment intensification compared to those treated by urologists. By 2019, however, treatment intensification remained <40% from oncologists only or both, and <15% from urologists only. In the second and third lines of therapy, androgen deprivation therapy + androgen receptor pathway inhibitor was the most prescribed regimen across specialties (>50%). CONCLUSIONS: Treatment intensification was underused in first lines of therapy across urology and oncology specialties despite evidence of improved survival. In subsequent lines, androgen deprivation therapy + androgen receptor pathway inhibitor was prescribed more frequently across specialties. These results underscore the need for earlier treatment intensification by urologists and oncologists.

Dry Age-Related Macular Degeneration: Distribution of Visual Acuity and Progression Risk in a Large Registry.

INTRODUCTION: Understanding the progression to geographic atrophy (GA) in late dry age-related macular degeneration (dAMD) can support development opportunities for dAMD treatments. We characterized dAMD by distribution of visual acuity (VA) categories and evaluated VA progression risk by disease stage. METHODS: This retrospective observational study used data from the American Academy of Ophthalmology IRIS® Registry (Intelligent Research in Sight) to identify patients diagnosed with dAMD in ≥ 1 eye from January 2016 through December 2019 (index date) with ≥ 1 visit and ≥ 1 VA measurement recorded post-index date. Patients were followed until the date of last visit, last contribution for diagnosing provider, or diagnosis of neovascular AMD post-index. Models were utilized to describe the distribution of VA categories and progression to worsening VA. RESULTS: Data from 593,277 patients were analyzed. At baseline, 64.4% had mild disease, 29.4% intermediate, and 2.9%/3.3% had GA with/without subfoveal involvement. Most patients with mild (88.4%) and intermediate (79.7%) disease and GA without subfoveal involvement (57.1%) had baseline VA ≥ 20/63 in the study eye; 72.0% of patients with GA with subfoveal involvement had VA < 20/63. Modeled results showed lower VA with more progressive stage at baseline. Annual probability of stable dAMD based on baseline stage ranged from 82.1% (GA without) to 92.3% (GA with subfoveal involvement). Annual progression probability to GA without/with subfoveal involvement was 0.4% for mild and 5.5% for intermediate disease and from dry to neovascular AMD, 0.5% for mild and 8.0% for intermediate disease. CONCLUSIONS: Results from this analysis of a large database of electronic health records complement those from randomized trials and show that patients with more advanced dAMD have lower VA at baseline and that VA progression is generally faster with each progressive stage. Together these findings highlight the disease burden and trajectory of dAMD as well as opportunities for addressing unmet needs.

Dry age-related macular degeneration (dAMD) is a disease that progressively worsens over time. As the disease progresses, patients start to lose their vision, leading to a substantial burden on their quality of life and finances due to the need for increased healthcare services. As of 2022, there are no medications available to reverse or stop worsening of dAMD. This study used real-world data from a large registry of electronic health records to increase the understanding of how patients progress through the stages of dAMD. By reviewing patient records, we were able to identify approximately 600,000 patients with confirmed dAMD. These patients were then followed over time, and we were able to confirm that patients with a lower ability to see at the beginning of our review period had more advanced dAMD. We also found that as patients' disease worsened, their vision also decreased. These findings highlight the need for new medication options to reverse or delay the worsening of dAMD and improve the quality of life for patients.

Prostate-specific antigen response and clinical progression-free survival in Black and White men with chemotherapy-naïve metastatic castration-resistant prostate cancer treated with enzalutamide in a real-world setting.

BACKGROUND: In the United States, Black men have a higher incidence of prostate cancer (PC)-related mortality than men of other races. Several real-world studies in advanced PC suggest, however, that Black men respond better to novel hormonal therapies than White men. Data on treatment responses to enzalutamide by race are limited. We assessed real-world prostate-specific antigen (PSA) response and clinical progression-free survival (cPFS) of Black vs. White men with chemotherapy-naïve PC treated with enzalutamide. METHODS: This retrospective cohort study included patients with PC who initiated enzalutamide treatment from 2014 to 2018 in the IntrinsiQ Specialty Solutions™ database, a collection of electronic medical records from community urology practices. Index date was the date of the first prescription for enzalutamide, used as a proxy for metastatic castration-resistant PC (mCRPC). Patients who had undergone chemotherapy and/or abiraterone therapy were excluded. Kaplan-Meier and Cox models adjusted for baseline characteristics were used to estimate PSA response and cPFS by race. RESULTS: The study included 214 Black and 1332 White men with chemotherapy-naïve PC presumed to have mCRPC based on the enzalutamide indication during the study period. Black men were younger and had higher baseline median PSA levels than White men. Enzalutamide therapy duration, follow-up time, and number of post-index PSA tests were similar between races. In multivariable analyses, the risk of patients achieving a ≥ 50% PSA decline was similar, whereas a numerically higher trend of ≥90% PSA decline was observed in Black men (HR 1.23; 95% CI 0.93-1.62 [P = 0.14]). In the multivariable analysis, Black men had significantly better cPFS (HR 0.82; 95% CI 0.68-0.98 [P = 0.03]). CONCLUSIONS: Black and White men with presumed chemotherapy-naïve mCRPC had similar PSA responses when treated with enzalutamide, but Black men had better cPFS than White men. Further research is warranted to validate these findings.

Mirabegron and antimuscarinic use in frail overactive bladder patients in the United States Medicare population.

INTRODUCTION: Overactive bladder (OAB) and frailty are independently associated with patient burden. However, economic burden and treatment-taking behavior have not been well characterized among frail patients with OAB, which, given the varying safety and tolerability profiles of available treatments, is crucial. OBJECTIVES: To assess costs, health care resource utilization, treatment-taking behavior (persistence and adherence) to OAB medication in older, frail OAB patients. METHODS: This was a retrospective cohort study using international business machines MarketScan Medicare Supplemental claims data. Eligible frail patients (per Claims-based Frailty Index score) initiating mirabegron were 1:2 propensity score matched (based on age, sex, and other characteristics) with those initiating antimuscarinics and were followed up to 1 year. All-cause, per-person, per-month costs, health care encounters, persistence (median days to discontinuation assessed using Kaplan-Meier methods) and adherence (≥80% of proportion of days covered at Day 365) were compared. RESULTS: From 2527 patients with incident mirabegron (21%) or antimuscarinic (79%) dispensations, 516 incident mirabegron users (median age: 82 years, 64% female) were matched to 1032 incident antimuscarinic users (median age: 81 years, 62% female). Median cost was higher in mirabegron group ($1581 vs. $1197 per month); this was primarily driven by medication cost. There was no difference in medical encounters. Adherence (39.1% vs. 33.8%) and persistence (103 vs. 90 days) were higher in mirabegron users. CONCLUSIONS: Among frail older adults with OAB, mirabegron use was associated with higher costs and potential improvements in treatment-taking behaviors, particularly with respect to treatment adherence, versus those initiating antimuscarinics.

Lung Transplant Outcomes in Adults in the United States: Retrospective Cohort Study Using Real-world Evidence from the SRTR.

BACKGROUND: The Scientific Registry of Transplant Recipients was retrospectively analyzed to provide real-world evidence of the efficacy and safety of tacrolimus-based immunosuppressive regimens in adult lung transplant recipients in the United States. METHODS: Adult recipients (N = 25 355; ≥18 y) of a primary deceased-donor lung transplant between January 1, 1999, and December 31, 2017, were followed for 3 y posttransplant based on immunosuppressive regimen at discharge: immediate-release tacrolimus (TAC) + mycophenolate mofetil (MMF), TAC + azathioprine (AZA), cyclosporine (CsA) + MMF, or CsA + AZA. The primary outcome was the composite endpoint of graft failure or death (all-cause) at 1 y posttransplant (calculated via a modified Kaplan-Meier method). RESULTS: Discharge immunosuppressive regimens in lung transplant recipients changed over time, with a substantial increase in the use of TAC + MMF. TAC + MMF was the most common immunosuppressive regimen (received by 61.0% of individuals at discharge). The cumulative incidence of graft failure or death at 1 y posttransplant in adult lung transplant patients receiving TAC + MMF was 8.6% (95% confidence interval 8.1-9.1). Risk of graft failure or death was significantly higher in adults receiving CsA + MMF or CsA + AZA compared with TAC + MMF, with no significant difference seen between TAC + MMF and TAC + AZA. TAC + MMF had the highest continued use at 1 y posttransplant (72.0% versus 35.4%-51.5% for the other regimens). There was no increase in the rate of infection or malignancy in the TAC + MMF group. CONCLUSIONS: Real-world evidence from the most comprehensive database of transplant recipients in the United States supports the use of TAC in combination with MMF or AZA as maintenance immunosuppression in adult lung transplant recipients.

Healthcare resource utilization among patients receiving non-invasive testing for coronary artery disease in an outpatient setting: A cohort study reflecting daily practice trends.

BACKGROUND: Accurate, early diagnosis and the initiation of appropriate treatment is central to reducing the clinical burden of coronary artery disease (CAD); however, real-world evidence characterizing healthcare resource utilization (HCRU) associated with testing for CAD is lacking. METHODS AND RESULTS: Using a non-interventional, retrospective, secondary database analysis, patients aged ≥18 years who underwent outpatient non-invasive cardiac diagnostic testing were identified. The primary objective was to gain an understanding of pre- and post-assessment care pathways and the associated interventions for patients who underwent non-invasive testing for CAD in either an outpatient or emergency department setting. Overall, chest pain was the primary reason for the index visit (54.8%), followed by shortness of breath (23.7%), myocardial infarction (MI), coronary artery disease (CAD) or congestive heart failure (CHF) (3.8%), and other (46.8%); 3.0% of patients had no apparent reason for testing in the last 45 days. Single-photon emission computed tomography (SPECT) was the dominant diagnostic testing modality (40.3%). During the 90-day follow-up, 7.3% (n = 22,083) of patients were diagnosed with CAD; among these patients, 19.4% had repeat diagnostic testing, 26.0% of patients had a revascularization procedure, and 65.6% underwent cardiac catheterization. These rates varied by testing modality. CONCLUSIONS: In this study of a large real-world data sample, variability in the use of non-invasive tests and HCRU were evident. These results may assist efforts to optimize system-wide care/diagnostic pathways and value-based treatment decisions for patients.

Undertreatment of overactive bladder among men with lower urinary tract symptoms in the United States: A retrospective observational study.

AIMS: To characterize the epidemiology and treatment patterns of adult men (≥40 years) diagnosed with, or treated for, overactive bladder (OAB) and/or benign prostatic hyperplasia (BPH). METHODS: This retrospective observational study used data extracted from the IBM MarketScan Commercial Claims and Encounters database and the Medicare Supplemental Coordination of Benefits database. Men with BPH and/or OAB were identified and observed to assess treatment and diagnostic patterns. RESULTS: Within the entire study sample (N = 462 400), BPH diagnosis (61.5%) and BPH treatment (73.7%) were more common than the corresponding values for OAB (25.8% and 7.0%, respectively). Notably, among diagnosed individuals, the dispensation of a corresponding treatment was more likely in individuals diagnosed with BPH (183 672 out of 284 416 = 64.6%) compared with OAB (16 468 out of 119 236 = 13.8%). Among newly diagnosed and/or treated patients (n = 196 576), only 60.3% received treatment. Among treated patients, most experienced only a single type of treatment (93.4%), 6.6% went on to receive a secondary treatment and 3.5% a tertiary. The most common primary treatment was alpha-blocker monotherapy (76.9%) followed by tadalafil monotherapy (16.4%). Among those untreated at first diagnosis, the median time between diagnosis and treatment initiation was 128 days. CONCLUSIONS: Diagnosis and management of OAB among males are challenging given the inherent overlap in symptoms observed with BPH. Unsurprisingly, we found that BPH is diagnosed and treated more frequently than OAB; but the differences between diagnosis and treatment patterns for the two conditions highlight the potential undertreatment of OAB and misdirection of therapy for men with a combination of voiding and storage symptoms.