All-cause Healthcare Resource Research Articles

Real-world analysis of the underdiagnosis, clinical outcomes and associated burden of hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) in the United States

BackgroundThrombotic microangiopathy (TMA) associated with hematopoietic stem cell transplantation (HSCT-TMA) is a serious post-transplant complication. Diagnosis is difficult due to overlapping symptoms with other conditions and a lack of universally adopted diagnostic criteria.MethodsThis retrospective, observational study investigated HSCT-TMA incidence between July 2009–August 2020 using the TriNetX US Electronic Medical Record database. Patients who underwent autologous or allogeneic HSCT procedures and had conditioning agents were stratified as follows: confirmed TMA (≥1 hemolytic uremic syndrome (HUS)/TMA diagnosis code), suspected TMA [no HUS/TMA code but met modified published Cho (adult) or Jodele (pediatric) diagnostic criteria (further information in main text), and non-TMA (met neither criteria). Baseline demographics, clinical characteristics and outcomes, and all-cause unadjusted healthcare resource utilization (HCRU) within 12-months of HSCT, were assessed. Statistical comparisons were against the non-TMA cohort (p<0.05).ResultsThe study included 16,809 adults and 901 pediatrics. Of these, 125 adults (0.7%) and 30 pediatrics (3.3%) had confirmed TMA, 3029 (18.0%) adults and 94 (10.4%) pediatrics had suspected TMA; 13,655 (81.2%) adults and 777 (86.2%) pediatrics met non-TMA criteria. Confirmed and suspected TMA incidences were higher after allogeneic HSCT in adults. In pediatrics, confirmed TMA incidence was higher following autologous transplantation, and suspected TMA higher after allogeneic transplantation. Confirmed and suspected TMA patients had significantly higher Charlson Comorbidity Indexes pre-HSCT and more post-HSCT complications. In adults with confirmed and suspected TMA, mortality estimates within 12-months of HSCT were significantly higher compared to non-TMA patients, and numerically higher in pediatrics. All confirmed and suspected TMA patients had significantly more ER visits, inpatient stays and ICU admissions. HCRU within 12-months of HSCT was higher in all confirmed TMA patients; ≤0.1% of patients with suspected TMA, and 25.6–50.0% of patients with confirmed TMA, received complement inhibitors.ConclusionsOur results demonstrate that incidence of HSCT-TMA in the real world, as per billing codes, is low compared with historical literature. However, a proportion of suspected TMA cases, based on diagnosis criteria, share similarly poor outcomes and HCRU. HSCT-TMA is likely underdiagnosed, or under-coded, in real world practice. Our study highlights the need for greater vigilance to this severe complication.

Racial disparities in treatment patterns, healthcare resource use, and outcomes in patients with pulmonary arterial hypertension in the United States

Objective This retrospective study using claims data compared demographics, clinical characteristics, treatment patterns, healthcare resource utilization, and clinical outcomes in Black and White patients with pulmonary arterial hypertension (PAH) in the United States. Methods Patients (aged ≥18 years) had ≥1 pharmacy claim for PAH medication, ≥6 months continuous healthcare plan enrollment, ≥1 inpatient/outpatient medical claim with a pulmonary hypertension diagnosis ≤6 months before first PAH medication, and race recorded. Results This analysis included 836 Black and 2896 White patients. Black patients were younger, with lower levels of education and annual household income, and higher comorbidity scores versus White patients. Only ∼14% of Black and White patients received index combination therapy. Lower adherence to index treatment was observed in Black patients. Although adjusted regression analysis in the overall population showed no differences in outcomes between groups, Black patients <65 years were 36% less likely to receive index combination therapy (odds ratio [OR] 0.64; 95% confidence interval [CI] 0.41–0.99), and 46% less likely to adhere to index treatment (OR 0.54; 95% CI 0.33–0.90). Other disparities included 24% higher all-cause health care resource utilization, 75% higher all-cause costs, and higher risk of clinical composite outcome. Social determinants of health (education, income, health insurance plan) partially mediated these race effects. Conclusions Differences in demographics, clinical characteristics, and treatment patterns between Black and White patients with PAH were observed. Disparities between Black and White patients <65 years were only partially mediated through social determinants of health variables, suggesting other factors may be involved.

Real-world unified airway benefits of mepolizumab: Effectiveness in patients with asthma and comorbid nasal polyps

BackgroundNasal polyps (NPs) are frequently associated with chronic rhinosinusitis (CRS). In keeping with the unified airway hypothesis, asthma and CRS with NP (CRSwNP) frequently co-occur, share a similar pathophysiology, and are often treated with oral corticosteroids (OCS); however, a need for alternative treatment options for patients with comorbid asthma and CRSwNP remains. ObjectiveTo characterize the short- and long-term dual airway effectiveness of the anti–interleukin-5 monoclonal antibody, mepolizumab, in real-world patients with asthma and comorbid NP. MethodsAdult patients with CRSwNP who initiated mepolizumab from November 1, 2014, to September 30, 2021, were identified from 2 Merative MarketScan Research Databases. Outcomes were compared for the 12 months pre- and post-mepolizumab initiation and a variable follow-up period. Primary outcomes included the following: annual rate and proportion of patients with NP- and asthma-related exacerbations; NP surgery occurrences; all-cause OCS claims, number of OCS bursts, and daily OCS dose; all-cause and NP-related health care resource utilization. ResultsDuring the 12 months post-index, patients experienced fewer NP- and asthma-related exacerbations, required fewer sinus surgeries, and reduced use of OCS, with fewer all-cause OCS claims and OCS bursts. Significant reductions in asthma exacerbation-related and NP-related health care resource utilization were also observed. ConclusionThis study illustrated the near- and long-term real-world effectiveness of mepolizumab treatment, with a focus on dual lower and upper airway benefit from single-agent add-on therapy. These results may aid physicians in clinical decision-making for patients with asthma and comorbid CRSwNP with complex care needs.

Healthcare resource utilization and costs among patients with alpha-1 antitrypsin deficiency with liver and/or lung disease: a longitudinal retrospective study in the USA.

Aim: To evaluate all-cause and liver-associated healthcare resource utilization (HCRU) and costs among patients with alpha-1 antitrypsin deficiency (AATD) with liver disease (LD) and/or lung disease (LgD). Materials & methods: This was a retrospective analysis of linked administrative claims data from the IQVIA PharMetrics® Plus and the IQVIA Ambulatory Electronic Medical Records (AEMR) databases from 1July2021 to 31January 2022. Patients with AATD in the IQVIA PharMetrics Plus database were included with ≥1 inpatient or ≥2 outpatient medical claims ≥90days apart with a diagnosis of AATD, or with records indicating a protease inhibitor (Pi)*ZZ/Pi*MZ genotype in the IQVIA AEMR database with linkage to IQVIA PharMetrics Plus. For a patient's identified continuous enrollment period, patient time was assigned to health states based on the initial encounter with an LD/LgD diagnosis. A unique index date was defined for each health state, and HCRU and costs were calculated per person-year (PPY). Results: Overall, 5136 adult and pediatric patients from the IQVIA PharMetrics Plus and IQVIA AEMR databases were analyzed. All-cause and liver-associated HCRU and costs were substantially higher following onset of LD/LgD. All-cause cost PPY ranged from US $11,877 in the absence of either LD/LgD to US $74,015 in the presence of both LD and LgD. Among liver transplant recipients in the AATD with LD health state, liver-associated total costs PPY were US $87,329 1-year pre-transplantation and US $461,752 1-year post-transplantation. In the AATD with LgD and AATD with LD and LgD health states, patients who received augmentation therapy were associated with higher all-cause total costs PPY and lower liver-associated total costs PPY than their counterparts who did not receive augmentation therapy. Conclusion: Patients with AATD had increased HCRU and healthcare costs in the presence of LD and/or LgD. HCRU and healthcare costs were highest in the AATD with LD and LgD health state.

Hyperkalemia Recurrence Following Medical Nutrition Therapy in Patients with Stage3-4 Chronic Kidney Disease: The REVOLUTIONIZEI Real-World Study.

The REVOLUTIONIZEI study aimed to characterize the relationships between medical nutrition therapy (MNT) and hyperkalemia recurrence in patients with stage3-4 chronic kidney disease (CKD) and hyperkalemia who received MNT in real-world clinical practice. This observational cohort study used de-identified electronic health record data from patients aged ≥18years with stage3-4 CKD who received MNT between January 2019 and October 2022 and had hyperkalemia (serum potassium >5.0mmol/L) within 30days before MNT. Patients were followed for 6months or until the first censoring event (death, prescription of outpatient potassium binder, or study end). The primary outcome was the percentage of patients with ≥1hyperkalemia recurrence during follow-up. Secondary outcomes included the number of hyperkalemia recurrences per patient, time to each recurrence, and hyperkalemia-related healthcare resource utilization. Exploratory outcomes included all-cause healthcare resource utilization and mortality. The final cohort comprised 2048 patients; 1503 (73.4%) patients remained uncensored after 6months. During the 6-month follow-up period, 56.0% of patients had ≥1hyperkalemia recurrence and 37.4% had ≥1 recurrence within the first month. Patients with ≥1hyperkalemia recurrence during follow-up had a mean ± standard deviation (SD) of 2.6 ± 2.2 recurrences. The mean ± SD time to first hyperkalemia recurrence was 45 ± 46days; the time between recurrences decreased with subsequent episodes. Hyperkalemia-related hospitalizations and emergency department visits were recorded for 13.7% and 1.5% of patients, respectively. Sensitivity analyses showed that results were consistent across patient subgroups, including those with comorbid heart failure and patients receiving renin-angiotensin-aldosterone system inhibitor therapy at baseline. Most patients with stage3-4 CKD had hyperkalemia recurrence, and MNT alone was inadequate to prevent recurrence. These patients may require additional long-term treatment, such as novel potassium binders, to maintain normokalemia and prevent hyperkalemia recurrence following MNT. Infographic available for this article. INFOGRAPHIC.

Real-World Patterns and Economic Burden Associated With Treatment Failure With Advanced Therapies in Patients With Moderate-to-Severe Ulcerative Colitis

Abstract Background Some patients lose response during treatment for moderate-to-severe ulcerative colitis (UC). We aimed to characterize real-world treatment failure patterns and associated economic burdens during use of first-line advanced therapies for UC. Methods IBM MarketScan Commercial and Medicare Supplemental Databases were used to identify adults initiating ≥ 1 advanced therapy for UC (January 1, 2010–September 30, 2019). Treatment failure was defined as augmentation with non-advanced therapy, discontinuation, dose escalation/interval shortening, failure to taper corticosteroids, UC-related surgery, or UC-related urgent care ≤ 12 months after treatment initiation. The index date was the date of treatment failure (treatment failure cohort) or 12 months after treatment initiation (persistent cohort). Treatment failure rates were assessed using Kaplan–Meier analyses. All-cause and UC-related healthcare resource utilization (HCRU) and costs 12 months post-index were also assessed. Results Analysis of treatment failure patterns included data from 6745 patients; HCRU and cost analyses included data from 5302 patients (treatment failure cohort, n = 4295; persistent cohort, n = 1007). In the overall population, 75% experienced treatment failure within the first 12 months (median: 5.1 months). Augmentation with non-advanced therapy (39%) was the most common first treatment failure event. The treatment failure cohort had significantly (P &amp;lt; .001) higher mean costs than the persistent cohort (all-cause, $74 995 vs $56 169; UC-related, $57 096 vs $47 347) mainly attributed to inpatient admissions and outpatient visits. Dose escalation/interval shortening accounted for the highest total costs ($101 668) across treatment failure events. Conclusions Advanced therapies for moderate-to-severe UC are associated with high rates of treatment failure and significant economic burden. More efficacious and durable treatments are needed.

Household Influenza Transmission and Healthcare Resource Utilization Among Patients Treated with Baloxavir vs Oseltamivir: A United States Outpatient Prospective Survey.

Influenza is a common, seasonal infectious disease with broad medical, economic, and social consequences. Real-world evidence on the effect of influenza treatment on household transmission and healthcare resource utilization is limited in outpatient settings in the USA. This study examined the real-world effectiveness of baloxavir vs oseltamivir in reducing influenza household transmission and healthcare resource utilization. This prospective electronic survey on patient-reported outcomes was conducted between October 2022 and May2023 via CVS Pharmacy in the USA. Adult participants (≥ 18years old) were eligible if they filled a prescription for baloxavir or oseltamivir at a CVS Pharmacy within 2days of influenza symptom onset. Participant demographics, household transmission, and all-cause healthcare resource utilization were collected. Transmission and utilization outcomes were assessed using χ2 and Fisher exact tests. Of 87,871 unique patients contacted, 1346 (1.5%) consented. Of 374 eligible patients, 286 (90 baloxavir- and 196 oseltamivir-treated patients) completed the survey and were included in the analysis. Mean age of participants was 45.4years, 65.6% were female, and 86.7% were White. Lower household transmission was observed with baloxavir compared with oseltamivir therapy (17.8% vs 26.5%; relative risk = 0.67; 95%CI 0.41-1.11). Healthcare resource utilization, particularly emergency department visits (0.0% vs 4.6%), was also numerically lower in the baloxavir-treated group; no hospitalizations were reported in either cohort. The findings from this real-world study suggest that antiviral treatment of influenza with baloxavir may decrease household transmission and reduce healthcare resource utilization compared with oseltamivir.

Comparison of Healthcare Costs for Women with Treated Versus Untreated Vasomotor Symptoms Due to Menopause.

The study objective was to estimate all-cause healthcare resource utilization (HCRU) and medical and pharmacy costs for women with treated versus untreated vasomotor symptoms (VMS) due to menopause. A retrospective study was conducted using US claims data from Optum Research Database (study period: January 1, 2012-February 29, 2020). Women aged 40-63years with a VMS diagnosis claim and ≥ 12 and ≥ 18months of continuous enrollment during baseline and follow-up periods, respectively, were included. Women treated for VMS were propensity score matched 1:1 to untreated controls with VMS. Standardized differences (SDIFF) ≥ 10% were considered meaningful. A generalized linear model (gamma distribution, log link, robust standard errors) estimated the total cost of care ratio. Subgroup analyses of on- and off-label treatment costs were conducted. Of 117,582 women diagnosed with VMS, 20.5% initiated VMS treatment and 79.5% had no treatment. Treated women (n = 24,057) were matched to untreated VMS controls. There were no differences in HCRU at follow-up (SDIFF < 10%). Pharmacy ($487 vs $320, SDIFF 28.4%) and total ($1803 vs $1536, SDIFF 12.6%) costs were higher in the treated cohort. Total costs were 7% higher in the treated cohort (total cost ratio 1.07, 95% CI 1.05-1.10, P < 0.001). The on-label treatment pharmacy costs ($546 versus $315, SDIFF 38.6%) were higher in the treated cohort. Off-label treatment had higher medical costs ($1393 versus $1201, SDIFF 10.4%). Most women with VMS due to menopause were not treated within 6months following diagnosis. While both on- and off-label treatment increased the total cost of care compared with untreated controls, those increases were modest in magnitude and should not impede treatment for women who report symptom improvement as a result of treatment.

Greater persistence and adherence to basal insulin therapy is associated with lower healthcare utilization and medical costs in patients with type 2 diabetes: a retrospective database analysis

IntroductionWe aimed to assess persistence and adherence to basal insulin therapy, their association with all-cause healthcare resource utilization (HCRU) and direct medical costs, and predictors of persistence and adherence in...

Prevalence and burden of chronic cough in France.

Chronic cough is a common condition that is associated with lower health-related quality of life and greater healthcare resource use. There are limited data on the prevalence, population characteristics and burden of chronic cough in France. This was a cross-sectional study based on responses from French adult residents to the 2020 National Health and Wellness Survey. Respondents with chronic cough were compared to 1:3 propensity score-matched controls without chronic cough. The weighted lifetime and 12-month prevalence of chronic cough were estimated as 7.5% and 4.8%, respectively. Respondents with chronic cough reported significantly worse perceived health than matched controls, with lower mean±sd scores of 46.68±9.28 versus 50.42±8.26 on the physical health component and 40.32±9.87 versus 44.32± 9.69 on the mental health component of the Medical Outcomes Study 12-item Short Form Survey Version 2 survey (p<0.001 for both comparisons). Respondents with chronic cough also had higher rates of moderate-to-severe forms of anxiety (24.4% versus 12.4%) and depression (36.4% versus 20.2%); higher rates of multiple forms of sleep disturbance; greater impairment of work productivity (38.2% versus 25.5%) and other activities (41.8% versus 28.2%; p<0.001 for all comparisons). Respondents with chronic cough also had higher rates of all-cause healthcare resource use including emergency room visits, hospitalisation, and overall and specialist healthcare provider visits compared to controls (p<0.001 for all comparisons). Chronic cough is a common condition in France that is associated with lower health-related quality of life and greater healthcare resource utilisation.

A comparison of clinical, utilization, and cost outcomes between oral treatments for multiple sclerosis.

Multiple sclerosis (MS) affects nearly 1 million people in the United States and causes significant disability and economic loss. Among the first available oral MS treatment options, clinical outcome comparisons and associated health care resource utilization are not clearly defined. To compare MS outcomes, health care resource utilization, and relative costs across treatment with dimethyl fumarate (DMF), fingolimod (FG), or teriflunomide (TERI) among Medicare Advantage Prescription Drug (MAPD) plan and commercially insured beneficiaries. This retrospective cohort study used the Humana Research Database. Eligible study patients had their first MS medication claim for oral DMG, FG, or TERI between January 1, 2013, and December 31, 2018. Patients were followed for a minimum of 12 months (mean follow-up = 3.8 years), until the earliest of the following occurred: health plan disenrollment, the end of the study period, or death. Study cohorts were balanced with inverse probability of treatment weighting. All-cause and MS-related health care resource utilization, time on therapy, and time after therapy were compared using inverse probability of treatment-adjusted multivariate generalized linear models across treatment groups. Relative costs were compared using a generalized linear model with a gamma distribution and log link. We identified 1,442 patients in 3 medication groups: DMF (n = 843), FG (n = 213), and TERI (n = 386). After weighting, there were no significant differences between the medication groups on demographic and clinical characteristics. Time on therapy (days) was significantly different across medication groups (P < 0.001). Time on therapy was longest for FG compared with the DM and TERI groups (644 vs 462 vs 521). The number discontinuing the index medication was significantly different for FG vs DMF vs TERI (74.7% vs 85.3% vs 80.7%; P < 0.001). FG had the lowest discontinuation rate. The mean (SD) annualized relapse rates (ARRs) were 0.47 (0.80), 0.42 (1.3), and 0.53 (1.3) (P = 0.037) for DMF, FG, and TERI, respectively. The percentage of those experiencing inpatient stays and the number of stays (mean [SD]) were significantly different among the FG group vs DMF vs TERI (29.9% vs 34.1% vs 40.9%; P < 0.001) and (0.57 [2.9] vs 0.74 [1.9] vs 0.91 [3.5]; P = 0.007), respectively. All-cause emergency department visits and the number of visits (mean [SD]) were significantly different for the FG cohort vs DMF vs TERI (46% vs 54.3% vs 61%; P < 0.001) and (1.84 [7.7] vs 2.38 [5.9] vs 2.87 [8.8]; P = 0.002), respectively. FG had the lowest impatient stays and emergency department visits of the 3 groups. Patients with MS initiated on FG used fewer health care resources and experienced lower ARR compared with patients on DMF and TERI.

Health care resource utilization and costs associated with diagnosed medication overuse headache and potential acute medication overuse in individuals with migraine.

Estimate health care resource utilization and costs associated with medication overuse headache and potential acute medication overuse. A retrospective analysis was conducted with Clinformatics Data Mart data (1 January 2019-31 December 2019) that included continuously enrolled commercially insured adults with migraine (International Classification of Diseases, Tenth Revision, Clinical Modification [ICD-10-CM] code G43.xxx). Medication overuse headache was defined as ≥1 inpatient or ≥2 outpatient claims with an ICD-10-CM code G44.41/40 (drug-induced headache). Potential acute medication overuse was defined as possessing sufficient medication for >10 mean treatment days/month for ergots, triptans, opioids, or combination analgesics or >15 mean cumulative days/month for simple prescription analgesics (e.g., acetaminophen, aspirin, other non-opioid analgesics) for >6 consecutive months. All-cause and migraine-related health care resource utilization and costs were compared after adjusting for demographic and clinical characteristics. Among 90,017 individuals with migraine, the frequency of medication overuse headache/potential acute medication overuse was 12.6% (diagnosed medication overuse headache: 0.6%; potential acute medication overuse: 12.1%). Adjusted all-cause total costs ($31,235 vs $21,486; difference: $9,749 [P < 0.001]) and adjusted migraine-related total costs ($9,770 vs $6,207; difference: $3,563 [P < 0.001]) were higher in the medication overuse headache/potential acute medication overuse group versus those without medication overuse headache/potential acute medication overuse. Individuals with diagnosed medication overuse headache/potential acute medication overuse had higher all-cause and migraine-related health care resource utilization and costs versus individuals without medication overuse headache/potential acute medication overuse, suggesting that improved migraine management is needed to reduce associated costs.

Reduced mortality, complications, and economic burden among medicare beneficiaries receiving influenza antivirals

Introduction Antiviral therapy may be underutilized in patients at high risk for increased clinical and economic burden (e.g. older adults). We aimed to examine the benefits associated with antiviral treatment of seasonal influenza among treated and untreated Medicare beneficiaries. Methods This retrospective study of Medicare Claims Research Identifiable Files identified patients ≥66 years old with an influenza diagnosis in outpatient setting between October 2016–March 2019 (flu seasons 2016–2018). Index date defined as date of first claim with influenza diagnosis; baseline as the 12 months pre-index. Treated patients received antivirals ≤2 days from index. Untreated patients had no antivirals ≤6 months post-index. Treated/untreated patients were 1:1 propensity score matched. Outcomes (death, all-cause and respiratory-related healthcare resource utilization [HCRU] and costs) were assessed until death or up to 6 months post-index. Descriptive statistics were reported; Kaplan-Meier estimation was used for survival over time. Results Among 116,901 matched patient pairs, all-cause mortality within 6 months from index diagnosis was 1.6% among treated versus 4.3% among untreated patients. Rates (treated versus untreated) of all-cause inpatient hospitalizations during follow-up were 13.9% versus 22.7% and respiratory-related hospitalizations were 4.2% versus 9.0%. Mean (SD) total all-cause and respiratory-related costs were $9,830 ($18,616.0) and $900 ($4016.4) among the treated, respectively, versus $13,207 ($24,405.1) and $2,024 ($7,623.7) among untreated, respectively. All differences were statistically significant (p < 0.001). Conclusions Lack of antiviral treatment is associated with increased mortality, HCRU, and economic burden in older Medicare beneficiaries with seasonal influenza. Future research should investigate whether the choice of antivirals affects influenza burden.

Outcomes Following Initiation of Triple Therapy with Fluticasone Furoate/Umeclidinium/Vilanterol versus Multiple-Inhaler Triple Therapy Among Medicare Advantage with Part D Beneficiaries and Those Commercially Enrolled for Health Care Insurance in the United States.

Patients with chronic obstructive pulmonary disease (COPD) have been shown to benefit from triple therapy commonly delivered by multiple-inhaler triple therapy (MITT); however, the complexity of MITT regimens may decrease patient adherence. Fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI), a once-daily single-inhaler triple therapy (SITT), became available in the United States (US) in 2017, but real-world data comparing outcomes for SITT versus MITT are currently limited. This study compared outcomes among patients with COPD initiating MITT versus SITT with FF/UMEC/VI who were either Medicare Advantage with Part D (MAPD) beneficiaries or commercial enrollees in the US. Retrospective study using administrative claims data from the Optum Research Database for patients with COPD who initiated FF/UMEC/VI or MITT between September 1, 2017, and March 31, 2019 (index date: first pharmacy claim for FF/UMEC/VI cohort; earliest day of ≥30 consecutive days-long period of overlap in the day's supply of all triple therapy components for MITT cohort). COPD exacerbations, adherence to triple therapy, and all-cause and COPD-related health care resource utilization (HCRU) and costs were compared between FF/UMEC/VI and MITT initiators. In total, 4659 FF/UMEC/VI initiators and 9845 MITT initiators for the MAPD population, and 821 FF/UMEC/VI initiators and 1893 MITT initiators for the commercial population were included in the study. MAPD beneficiaries initiating FF/UMEC/VI had a significantly lower annual rate of severe exacerbations compared to MITT initiators (0.26 vs 0.29; p=0.014). They also had a significantly higher mean adherence (proportion of days covered) (0.51 vs 0.37; p<0.001) and significantly lower all-cause and COPD-related inpatient stays compared to MITT initiators ([32.02% vs 34.27%; p=0.017], [16.09% vs 17.72%; p=0.037]). Trends were similar among the commercial population, but the results were not statistically significant. FF/UMEC/VI initiators had significantly fewer severe exacerbations, higher triple therapy adherence, and lower HCRU costs compared to MITT initiators for MAPD beneficiaries.

Event-Free Survival as a Predictor of Overall Survival and Recurrence Burden of Patients with Non-Small Cell Lung Cancer Receiving Neoadjuvant Therapy

Event-free survival has replaced overall survival as a primary end point in many recent and ongoing clinical trials. This study aims to examine the correlation between real-world event-free survival and overall survival and to assess the clinical and economic burden associated with disease recurrence among patients with resected stage II to III non-small cell lung cancer who received neoadjuvant therapy in the United States. This retrospective study used the Surveillance, Epidemiology, and End Results Medicare database (2007-2019) to identify patients with newly diagnosed, resected, stage II to IIIB (N2) non-small cell lung cancer who received neoadjuvant therapy. The correlation between real-world event-free survival and overall survival was assessed using the normal scores rank correlation and landmark analysis. Overall survival, all-cause health care resource use and costs, and non-small cell lung cancer-related health care resource use and costs were compared between patients with and without recurrence. A total of 221 patients met the eligibility criteria (median follow-up time from neoadjuvant treatment initiation: 32.7months). The mean age was 72.1years, and 57.0% of patients were male. Real-world, event-free survival and overall survival are positively and significantly correlated (0.68; 95% CI, 0.52-0.79). Patients with recurrence had significantly shorter median overall survival (19.3 vs 116.9months), 4.59 times increased risk of death (95% CI, 2.56-8.26), and significantly higher all-cause and non-small cell lung cancer-related health care resource use and costs (adjusted mean monthly costs per patient difference: $5758 and $3187, respectively [all P<.001]). These findings help validate event-free survival as a clinically meaningful end point and strong predictor for overall survival and highlight the need for additional novel therapies that may delay or prevent recurrence in resectable stage II and III non-small cell lung cancer.

Treatment Patterns, Healthcare Resource Utilization, and Direct Costs Among Patients Initiating Concomitant Use of a Calcitonin Gene-Related Peptide Monoclonal Antibody (CGRP mAb) and Novel Acute Medication in the United States.

To describe treatment patterns, all-cause and migraine-related healthcare resource utilization (HCRU), and direct costs among people with migraine treated with concomitant calcitonin gene-related peptide monoclonal antibody (CGRP mAb) and novel acute migraine medications (ubrogepant, rimegepant, lasmiditan) in the United States (US). This retrospective, observational cohort study utilized data from the IBM MarketScan® Research Databases and included adults initiating CGRP mAb or novel acute migraine medication as index medications between May 01, 2018, and Feb 28, 2021. All-cause and migraine-related HCRU (number of visits) and costs at baseline (12 months pre-index) and at follow-up (12 months post-index) were descriptively analyzed; differences between values at follow-up and baseline were reported. Of 4,167 included in the analysis (mean [SD] age: 43.7 [11.2] years), 89.2% were women, and 59.7% had chronic migraine. Adherence to the indexed CGRP mAb was 47% (using proportion of days covered [PDC]) and 80.1% (using medication possession ratio [MPR]); mean (SD) persistence was 273.4 (115.3) days). At follow-up, 43.9% of the patients discontinued their index preventive medication of which 80.2% switched to a different preventive migraine medication; 17.0% restarted their index preventive medication. Reductions in all-cause inpatient HCRU, all-cause inpatient and outpatient costs, and migraine-related outpatient HCRU were observed at follow-up vs. baseline, whereas increases in all-cause outpatient HCRU, all-cause medication costs, migraine-related inpatient HCRU, and migraine-related inpatient, outpatient, and medication costs were observed. In this study, observed treatment patterns with the indexed CGRP mAb were consistent with prior reports. Concomitant treatment with CGRP mAb and novel acute migraine medications led to reductions in some outcomes of HCRU and direct costs, however, increases were also observed. Treatment utilization, reductions in HCRU and cost savings identified in this study in favor of concomitant CGRP mAb and novel acute medications may help clinicians and other healthcare decision makers assessing appropriate therapeutic options for migraine management.

MT35 The Impact of Patient Engagement with a Digital Diabetes Solution on All-Cause Healthcare Resource Utilization Rates and Charges

MT35 The Impact of Patient Engagement with a Digital Diabetes Solution on All-Cause Healthcare Resource Utilization Rates and Charges

Real-World Analysis of the Underdiagnosis, Clinical Outcomes and Associated Burden of Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (HSCT-TMA) in the United States of America

Background: Thrombotic microangiopathy (TMA) associated with hematopoietic stem cell transplantation (HSCT-TMA) is a serious post-transplant complication. HSCT-TMA is difficult to diagnose due to overlapping symptoms with other conditions and a lack of universally adopted diagnostic criteria. Objectives: This study aimed to assess the incidence and diagnosis of HSCT-TMA in real-world practice across the US, including possible missed diagnoses, alongside clinical outcomes and associated burden of HSCT-TMA. Methodology: This retrospective, observational study investigated the incidence of HSCT-TMA from July 2009-August 2020 using data from the TriNetX US Electronic Medical Record (EMR) database. Patients who underwent autologous or allogenic HSCT procedures and had conditioning regimens recorded were stratified into three cohorts: confirmed TMA, suspected TMA, and non-TMA patients. Confirmed TMA was defined as any individual with at least one recorded hemolytic uremic syndrome (HUS)/TMA diagnosis code within 12 months of HSCT. Suspected TMA included individuals without a recorded HUS/TMA ICD code, but who met the modified published criteria (Cho BS, et al. Transplantation 2010;90:918-26; Jodele S, et al. Transfus Apher Sci 2016;54:181-90) within 12 months of HSCT; published criteria were adapted to fit data available in the TriNetX database. Non-TMA patients did not meet criteria to be included in either of the previous two cohorts. Adult and pediatric patients were analyzed separately. Demographic and clinical characteristics at baseline, clinical outcomes, and all-cause unadjusted healthcare resource utilization within 12 months of HSCT, were assessed. The baseline period was defined as the six months before the index date (inclusive), unless specified; index date was defined as the date a patient had a HSCT procedure code recorded. Statistical comparisons, where conducted, were made against the non-TMA cohort using the Fisher's exact test for categorical variables, the student's t test for means, and the Mann-Whitney U test for medians. Significance was set at the level of p&amp;lt;0.05. Results: In total, 16,809 adult and 901 pediatric patients had an HSCT procedure code and a recorded conditioning regimen. The confirmed TMA group comprised 125 (0.7%) adult and 30 (3.3%) pediatric patients, the suspected TMA group included 3,029 (18.0%) adult and 94 (10.4%) pediatric patients, and the non-TMA group included 13,655 (81.2%) adult and 777 (86.2%) pediatric patients. Baseline demographics were similar across all groups. Mortality within the first year of HSCT was significantly higher in adult patients with confirmed TMA (40.0%) and suspected TMA (30.7%) compared with patients without TMA (13.6%). Mortality was numerically highest in pediatric patients with confirmed TMA (26.7%) followed by suspected TMA (23.4%), and lowest in non-TMA patients (15.8%). Comorbidity impact (based on the Charlson Comorbidity Index) was significantly higher, and HSCT complications significantly more common, in confirmed and suspected TMA patients (Table 1). Clinical outcomes in the first year after HSCT were significantly worse in confirmed and suspected TMA patients than in non-TMA patients (Table 2). All patients with confirmed and suspected TMA had a significantly higher frequency of oxygen/intubation compared with non-TMA patients. Dialysis rates were significantly higher in all patients with confirmed TMA (adults, 26.4%; pediatrics, 20.0%) and adult patients with suspected TMA (6.9%), compared with patients with no TMA (adults, 2.2%; pediatrics, 4.0%); pediatric patients with suspected TMA (8.5%) had numerically higher dialysis rates compared with those without TMA (4.0%). Patients with confirmed and suspected TMA had significantly greater use of targeted therapies and a higher number of total ICU visits, compared with non-TMA patients (Table 2). Conclusions: This study showed that HSCT-TMA is associated with a high mortality and morbidity burden. Furthermore, these results suggest that HSCT-TMA is underdiagnosed in the real-world setting, as patients with suspected TMA had worse outcomes and a higher burden of disease than patients without TMA following HSCT. This study highlights the need for timely diagnosis of this severe complication, as well as novel approaches to treat and manage this vulnerable population.

Real-World Treatment Patterns and Healthcare Resource Utilization in Patients with Waldenström Macroglobulinemia Initiating First-Line Treatment with Ibrutinib or Zanubrutinib

Background: A pivotal clinical trial comparing the efficacy and safety of ibrutinib with zanubrutinib in patients with Waldenström macroglobulinemia (WM) did not achieve its primary endpoint of complete response or very good partial response. In addition, median progression-free survival was not reached in either treatment arm. In the absence of treatment effectiveness differentiation based on clinical trial evidence, we conducted a real-world study to assess patient characteristics, treatment patterns, and all-cause healthcare resource utilization (HRU) in patients with WM who received ibrutinib or zanubrutinib as first-line (1L) therapy. Methods: In this retrospective cohort study, we used the ConcertAI Database to collect claims data of patients with WM aged ≥18 years who initiated single-agent ibrutinib (420 mg/day) or zanubrutinib (320 mg/day) as 1L therapy on or after 9/1/2021 (index date). A 6-month washout period of guideline-recommended WM antineoplastic agents was required to confirm 1L status. Patients were excluded if they were receiving ibrutinib and zanubrutinib concurrently, died within 30 days of the index date, had a record of hematopoietic stem cell transplant, had a record of other ibrutinib indications in the 6 months before index, or were participating in a clinical trial. Baseline demographics and clinical characteristics, therapy duration (number of days between the first and last day of the medication supply), and HRU (all-cause and WM-specific) per-patient-per-month (PPPM) were assessed. Results: The study included 51 patients (ibrutinib, n=30; zanubrutinib, n=21). In both cohorts, most participants were male (ibrutinib, n=20 [67%]; zanubrutinib, n=16 [76%]), and the mean age at baseline was 73 years and 75 years, respectively. At baseline, the median Charlson Comorbidity Index was 0, and the WM-related and ibrutinib/zanubrutinib-related clinical events were similar between the two cohorts. While the zanubrutinib cohort had a higher prevalence of infections (33% vs 3%) and musculoskeletal pain (24% vs 7%) at baseline, the ibrutinib cohort had a higher prevalence of hypertension (20% vs 14%). The baseline prevalence of anemia was similar in both cohorts (ibrutinib, 27%; zanubrutinib, 29%). Mean follow-up was 6.9 months (ibrutinib) and 6.3 months (zanubrutinib). The mean ± SD duration of therapy was 152 ± 112 days (ibrutinib) and 113 ± 99 days (zanubrutinib; p=0.195). The ibrutinib cohort had a significantly lower all-cause HRU PPPM (4.53 ± 3.96 vs 8.53 ± 6.66, p=0.020) and lower WM-specific HRU PPPM (0.11 ± 0.26 vs 0.54 ± 1.14, p=0.104). Conclusions: In this real-world analysis, patients with WM treated with 1L ibrutinib had a numerically longer duration of therapy and lower all-cause and WM-specific HRU PPPM, compared to those treated with 1L zanubrutinib. Due to a small sample size and short follow-up, these findings should be interpreted cautiously.

Myelosuppression and healthcare utilization among patients with chemotherapy-treated extensive-stage small cell lung cancer (ES-SCLC) with and without trilaciclib from community oncology practices.

527 Background: Trilaciclib has been recently approved for decreasing chemotherapy-induced myelosuppression among adults with ES-SCLC when administered prior to chemotherapy in US. The objective of this study was to estimate the reduction in myelosuppression with trilaciclib use using real-world data. Methods: This study compared ES-SCLC patients receiving trilaciclib and chemotherapy (T) during Feb 2021- Jan 2023 to a comparison cohort not receiving trilaciclib (C) during Apr 2020 – Jan 2023 using EMOL Health’s database, which includes &gt;7 million patients from &gt; 500 community oncology practices. The C cohort was matched to the T cohort based on age, chemotherapy, and line of therapy. Index date was the date of trilaciclib initiation for the T cohort and the date of chemotherapy initiation for the C cohort. Primary outcome measures were the number of events in grade ≥3 myelosuppression (in ≥1, ≥2, 3 lineages). Poisson regression models used to estimate the reduction in risk between the groups. Descriptive statistics of grade ≥3 myelosuppression, cytopenia-related healthcare utilization, and all-cause healthcare resource utilization are presented. Results: 77 T patients and 77 C patients were identified. Demographics and baseline clinical characteristics were similar (age 69.6 vs. 70.8, female 48.1% vs. 55.8% for T and C, respectively, ECOG 0/1 63.6% and 83.1% received 1st line treatment at index for both). Median duration of index treatment was 4 cycles for both cohorts. Rates of grade ≥3 myelosuppression in ≥1, ≥2, 3 lineages were estimated to be reduced in the T by 63.4% (95% CI: 44.1% - 76.7%, p &lt; 0.001), 90.9% (95% CI: 74.7% - 97.8%, p &lt; 0.001), and 91.9% (95% CI: 59.1% - 99.6%, p =0.016), respectively. T patients had lower prevalence of grade ≥3 myelosuppression in ≥1, ≥2, 3 lineages (11.5% vs. 27.1%; 1.2% vs. 11.7%; 0.4% vs. 4.4% per cycle) than C patients during index treatment (Table). Cytopenia-related healthcare utilization was also lower for T patients (G-CSF use 25.9% vs. 59.0%, ESA use 3.7% vs. 5.1%, blood transfusion 1.7% vs. 8.4%, platelet transfusion 1.2% vs. 2.6% per cycle during index treatment). Number of all-cause hospitalizations per patient was 0.35 for T vs. 0.51 for C during index treatment. Among those with ≥1 hospitalizations, median number of hospitalizations was 1.0 for T and 2.0 for C during index treatment. Conclusions: Results from this study suggest that trilaciclib reduces myelosuppression and cytopenia-related healthcare utilization among patients with ES-SCLC in the real world.[Table: see text]