United States Clinical Practice Research Articles

United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

Background/objectivesThe phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody–positive generalized myasthenia gravis (gMG). The aim of the ELEVATE study was to assess the effectiveness of eculizumab in clinical practice in adults with MG in the United States.MethodsA retrospective chart review was conducted in adults with MG who initiated eculizumab treatment between October 23, 2017 and December 31, 2019. Outcomes assessed before and during eculizumab treatment using a pre- versus post-treatment study design included Myasthenia Gravis–Activities of Daily Living (MG-ADL) total scores; minimal symptom expression (MSE); physician impression of clinical change; minimal manifestation status (MMS); and concomitant medication use.ResultsIn total, 119 patients were included in the study. A significant reduction was observed in mean MG-ADL total score, from 8.0 before eculizumab initiation to 5.4 at 3 months and to 4.7 at 24 months after eculizumab initiation (both p < 0.001). At 24 months after eculizumab initiation, MSE was achieved by 19% of patients. MMS or better was achieved by 30% of patients at 24 months. Additionally, 64% of patients receiving prednisone at eculizumab initiation had their prednisone dosage reduced during eculizumab treatment and 13% discontinued prednisone; 32% were able to discontinue nonsteroidal immunosuppressant therapy.DiscussionEculizumab treatment was associated with sustained improvements in MG-ADL total scores through 24 months in adults with MG. Prednisone dosage was reduced in approximately two-thirds of patients, suggesting a steroid-sparing effect for eculizumab.

Real-world (RW) antiemetic use and its association with duration of oral azacitidine (Oral-AZA) maintenance in patients (pts) with acute myeloid leukemia (AML) in the United States (US).

e23259 Background : Oral-AZA was approved in the US in Sep 2020 for maintenance treatment of transplant-ineligible pts with AML in remission following intensive chemotherapy. Approval was based on the QUAZAR trial (NCT01757535), which showed that Oral-AZA was associated with longer survival outcomes than placebo in this pt population. Both the prescribing information and clinical guidelines recommend antiemetic prophylaxis with Oral-AZA for at least the first 2 cycles to prevent the most common adverse events, nausea and vomiting. However, the extent to which antiemetics are being used with Oral-AZA in the real world is not yet well understood. This study aimed to examine the use of antiemetics among pts with AML receiving Oral-AZA in RW clinical practice and to determine whether this has any association with duration of therapy (DoT). Methods : This was a retrospective, observational study using Flatiron Health electronic health record data from Jan 2014 to Apr 2023. Eligible pts were ≥18 years and had a diagnosis of AML and ≥1 order for Oral-AZA on or after Sep 1, 2020. The index date was date of Oral-AZA initiation. Pts were followed until death, loss to follow-up, or the end of the study. Pt characteristics were assessed on the index date; antiemetic use was assessed at index and over the follow-up period. For each pt, each 28-day cycle of Oral-AZA was denoted as “covered” or “not covered” by antiemetics according to whether the pt had ≥14 days of antiemetics available during the cycle. Total DoT was measured for Oral-AZA using cumulative incidence curves and compared between pts with their first 2 cycles covered and those without. Results: 189 pts with AML were included. Mean age was 66 years, 52% were female, 56% had evidence of prior cytarabine use, and 10% had a prior stem cell transplant. 152 pts had ≥2 cycles of Oral-AZA. Overall, less than half of pts (48%) had a claim for an antiemetic on the index date. 16% never initiated an antiemetic between index and the end of follow-up; in the remaining 84%, the mean time from the index date to antiemetic initiation was 57 days and 29% had a time to antiemetic initiation of &gt; 56 days. Only 9% (14/152) of pts had both their first 2 Oral-AZA cycles covered by antiemetics. Overall, pts had a median of 5 Oral-AZA cycles. Pts with antiemetic coverage of their first 2 cycles had a median of 8.5 Oral-AZA cycles versus 6 in those without. The median DoT was longer for pts with antiemetic coverage of cycles 1 and 2 than those without (258 days vs 154 days). Conclusions: These findings suggest that antiemetics are not currently widely utilized with Oral-AZA in RW US clinical practice. This may reduce DoT and could potentially impact the likelihood of observing RW efficacy outcomes like those in the QUAZAR trial. Further studies are required to examine the association between guideline-recommended antiemetic use and clinical outcomes.

Abstract PO3-04-10: Real-world progression-free survival and overall survival of palbociclib plus endocrine therapy in Japanese patients with HR+/HER2- ABC in the first- or second-line setting: A multicenter observational study

Abstract Introduction Palbociclib (PAL), a selective, oral inhibitor of cyclin-dependent kinases 4 and 6 (CDK4/6), plus (+) endocrine therapy (ET) is standard therapy for the treatment of hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (ABC) and was launched in December 2017 in Japan. Efficacy and safety of PAL + ET was shown by randomized clinical studies, PALOMA-2 and PALOMA-3. Further, recent large real-world studies, P-REALITY/P-REALITY X, confirmed effectiveness of PAL+ aromatase inhibitor (AI) for HR+/HER2- ABC in routine United States clinical practice. However, there is limited real-world evidence (RWE) for PAL in Japan. To strengthen the RWE of PAL+ET for HR+/HER2- ABC treatment under different clinical practice and healthcare system, we examine the real-world effectiveness of PAL + ET as 1st line (1L) and 2nd line (2L) treatment for HR+/HER2- ABC in Japan. Methods In this multicenter, observational study, patients with HR+/HER2- ABC, who initiated PAL + ET from 15 December 2017 to 31 December 2020 were enrolled. To reduce selection bias, all patients who started PAL during the above period were listed and their eligibility confirmed. Eligible patients had 1) diagnosis of HR+/HER2- ABC; 2) received PAL + ET as 1L or 2L treatment; 3) at least 6 months of follow up from start of PAL or died within that period. The 1L treatment was defined as the first systemic therapy received for ABC regardless of recurrence timing if receiving adjuvant treatment. The subsequent treatment after 1L treatment was defined as a 2L treatment. The primary endpoint was real-world progression-free survival (rwPFS) defined as the time from PAL+ET start to physician-documented disease progression or death due to any cause, whichever occurs first. Secondary endpoints included overall survival (OS), and chemotherapy-free survival (CFS). Results were stratified by treatment line (ie. 1L or 2L). rwPFS, OS, and CFS were analyzed with the Kaplan-Meier method (NCT05399329). Results In this study, 677 patients were enrolled. Among them, 419 (62%) patients and 258 (38%) patients received PAL + ET as 1L and 2L, respectively. The median age was 59.0 (range 29-87) and 470 patients (69.8%) were postmenopausal. 49.0% patients had visceral involvement and 63.8% had a disease-free interval &amp;gt;24 months, defined as date of breast cancer surgery to the diagnosis date of recurrence, and 26.9% had de novo metastatic disease. The starting dose of PAL was 125 mg (89%), 100mg (9%), and 75mg (2%); dose reductions during the treatment period were required for 72% of patients. The most common ET combined with PAL was fulvestrant (FUL; 56.3% in 1L, 76.7% in 2L) followed by letrozole (LET; 37.9% in 1L, 16.3% in 2L). After a median follow-up of 36.2 months, the median rwPFS (95% CI) was 24.5 months (19.9-29.4) for 1L treatment and 14.5 months (10.2-18.3) for 2L treatment. Among the 604 patients initiating at 125 mg/day, the median rwPFS was 25.6 months (21.3-30.4) for 1L treatment and 14.3 months (9.7-18.3) for 2L treatment. Although median OS was not reached (NR) in 1L, median OS of 2L was 44.1 months (39.2 -NR). The 48-months OS rates for 1L and 2L treatment were 62.0% (55.4-67.9) and 48.4 % (40.4-55.9), respectively. The median CFS was 36.7 months (31.8-43.9) for 1L treatment and 23.8 months (20.5-27.3) for 2L treatment. Conclusions Our real-world data are consistent with findings form the PALOMA-2 and PALOMA-3 studies and those from RWE in routine United States clinical practice. FUL was the most commonly endocrine partner with PAL regardless of treatment lines. These observations add to the body of evidence supporting PAL effectiveness in clinical practice for Japanese ABC patients. Citation Format: Norikazu Masuda, Masaya Hattori, Tersuhiro Yoshinami, Takuho Okamura, Ken-ichi Watanabe, Takahiro Nakayama, Hiroko Masuda, Michiko Tsuneizumi, Daisuke Takabatake, Michiko Harao, Hiroshi Yoshino, Natsuko Mori, Hiroyuki Yasojima, Chiya Oshiro, Madoka Iwase, Miki Yamaguchi, Takafumi Sangai, Shinsuke Sasada, Takanori Ishida, Manabu Futamura, Nobuyoshi Kosaka, Yoshiyuki Shibasaki, Shigenori Nagai. Real-world progression-free survival and overall survival of palbociclib plus endocrine therapy in Japanese patients with HR+/HER2- ABC in the first- or second-line setting: A multicenter observational study [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO3-04-10.

Risk Factors for Reoperation Following Single-Level Cervical Disc Arthroplasty as Utilized in a Representative Sample of United States Clinical Practice: A Retrospective PearlDiver Study.

Retrospective Cohort. Most data regarding cervical disc arthroplasty (CDA) outcomes are from highly controlled clinical trials with strict inclusion/exclusion criteria. This study aimed to identify risk factors for CDA reoperation, in "real world" clinical practice using a national insurance claims database. The PearlDiver database was queried for patients (2010-2020) who underwent a subsequent cervical procedure following a single-level CDA. Patients with less than 2 years follow-up were excluded. Primary outcome was to evaluate risk factors for reoperation. Secondary outcome was to evaluate the types of reoperations. Risk factors were compared using descriptive statistics. Multivariate regression analyses were used to ascertain the association among risk factors and reoperation. Of 14,202 patients who met inclusion criteria, 916 (6.5%) underwent reoperation. Patients undergoing reoperation were slightly older with higher Elixhauser Comorbidity Index (ECI) scores, however both were not risk factors for reoperation. Patients with diagnoses such as smoking, myelopathy, inflammatory disorders, spinal deformity, trauma, or a history of prior cervical surgery were at greater risk for reoperation. No association was found between the year of index surgery and reoperation risk. The most common reoperation procedure was cervical fusion. As billed for in the United States since 2010, CDA was associated with a 6.5% reoperation rate over a mean follow-up time of 5.3 years. Smoking, myelopathy, inflammatory disorders, spinal deformity, and a history of prior cervical surgery or trauma are risk factors for reoperation following CDA. Though patients who underwent a reoperation were older, age was not found to be an independent risk factor for a subsequent procedure.

Occurrence of sleep disorders, anxiety, and depression among patients with mild cognitive impairment and Alzheimer’s disease in diverse populations: A large nationwide electronic health record cohort study

AbstractBackgroundPatients affected with mild cognitive impairment (MCI) and Alzheimer’s disease (AD) often experience anxiety, depression, and sleep disturbances. Some of the published studies reported a higher incidence of these disorders in minority populations. The objectives of this study were to characterize the occurrence of sleep disorders, anxiety, and depression among patients with MCI and/or AD, stratified by gender, age and race/ethnicity in the United States clinical practice setting.MethodA retrospective cohort study was conducted using electronic health records and insurance claims from the Optum® Market Clarity database containing data from more than 63 million people in the US insured through Medicare, Medicaid, and other insurance providers. The study included patients diagnosed, utilizing ICD diagnosis codes, with MCI and/or AD between January 2017 and September 2021. Prevalence of depression, anxiety, and sleep disorders, as well as associated medication prescriptions, were identified in the 12 months before and after MCI and/or AD diagnoses. The occurrence of these diagnoses and prescriptions was evaluated stratified by gender, age, and race/ethnicity.ResultA total of 197,346 patients were diagnosed with MCI (56.9% female, mean age of 70.0 [SD 14.8]) and 144,321 with AD (63.9% female, mean age of 79.3 [SD 8.2]) at the baseline. Overall, a large proportion of MCI and AD cases had depression (pre‐MCI diagnosis: 40.3%; post‐MCI diagnosis: 44.8%; pre‐AD diagnosis: 32.2%; post‐AD diagnosis: 38.3%), anxiety (pre‐MCI diagnosis: 36.2%; post‐MCI diagnosis: 40.2%; pre‐AD diagnosis: 26.4%; post‐AD diagnosis: 31.5%), and sleep disorders (pre‐MCI diagnosis: 36.2%; post‐MCI diagnosis: 39.3%; pre‐AD diagnosis: 23.2%; post‐AD diagnosis: 25.4%). Occurrences of depression and anxiety were highest among females. Among individuals with MCI and/or AD, the gap between the proportion experiencing depression and the proportion prescribed an antidepressant was greatest for African American males and females. This was not observed for anxiety and anti‐anxiety medications.ConclusionA large proportion of individuals with MCI and/or AD also had depression, anxiety, and sleep issues. Further understanding of the populations with these comorbidities and their associated prescriptions by different ages, sex, and races/ethnicities is important when supporting patients with MCI and AD.

Factors associated with delayed treatment initiation of oral anticoagulants among Medicare patients with atrial fibrillation

Abstract Background Atrial fibrillation (AF) is the most prevalent cardiac arrhythmia in the United States (US) and is associated with increased risk of stroke.1 Oral anticoagulants (OACs) are the primary treatment to reduce stroke risk in patients with AF.2,3 However, there is recent evidence of underuse of OAC in AF patients.4 Purpose To identify factors associated with delayed OAC treatment initiation among AF patients in the US clinical practice. Methods Medicare beneficiaries aged ≥65 years with a new diagnosis of AF without moderate-to-severe mitral stenosis or a mechanical heart valve after 1 Oct 2015 were included. Additional inclusion criteria included ≥1 claim for an OAC on or after 1st diagnosis of AF, ≥12 months of continuous Medicare Parts A, and D enrollment, and CHA2DS2-VASc score of ≥2 for males/≥3 for females. Time from 1st AF diagnosis to OAC initiation (index OAC), and demographic, sociodemographic, clinical and index OAC formulary characteristics were described. Delayed initiation was defined as OAC initiation &amp;gt;3 months after and early initiation as OAC initiation 0-3 months after 1st AF diagnosis. Factors associated with delayed OAC initiation were assessed using multivariable logistic regressions. Results A total of 446,441 patients met the inclusion criteria. Time from 1st AF diagnosis to OAC initiation was &amp;lt;1 month for 52.4% of patients, 1–≤3 months for 18.1%, &amp;gt;3–≤6 months for 7.1%, &amp;gt;6–≤12 months for 6.8%, and &amp;gt;12 months for 15.7%. Accordingly, 30% had delayed vs. 70% had early OAC initiation. Median age was 78 years (both), 48% and 47% were male, 87% and 89% were White, and median CHA2DS2-VASc score was 4 (both), respectively, for the delayed and early OAC initiation cohorts (Table 1). Factors associated with delayed OAC initiation included Black race (odds ratio [OR]: 1.29, 95% confidence interval [CI]: 1.25–1.33), South and West US census regions (OR: 1.22, 95%CI: 1.19–1.24 and OR: 1.29, 95%CI: 1.26–1.32, respectively), dementia (OR: 1.27, 95%CI: 1.23–1.30), congestive heart failure (OR: 1.20, 95%CI: 1.26–1.32), bleeding hospitalization ≤3 months before 1st AF diagnosis (OR: 1.22, 95%CI: 1.18–1.27), prior authorization (OR: 1.69, 95%CI: 1.66–1.71), and tier 4 formulary for index OAC at AF diagnosis (OR: 1.26, 95%CI: 1.22–1.30). Additionally, having low-income subsidy or dual eligibility (for Medicare and Medicaid) was associated with increased odds of delayed OAC initiation (OR: 1.06, 95%CI: 1.02–1.10 and OR: 1.09, 95%CI: 1.05–1.14, respectively; Figure 1). Conclusions In this study on Medicare patients with AF, Black race, low-income subsidy, and dual eligibility were found to be associated with delayed OAC initiation, suggesting potential disparities in the care of AF with timely OAC treatment. Moreover, OAC requiring prior authorization (the strongest predictor) or being on formulary tier 4 were associated with delayed initiation, indicating formulary restrictions may create barriers to timely OAC access for AF patients.Table 1.Patient characteristicsFigure 1.Factors of delayed initiation

Early Real-World Treatment Patterns and Clinical Outcomes in Patients with Metastatic Breast Cancer Treated with Eribulin After Prior Immuno-Oncology or Antibody-Drug Conjugate Therapy.

Eribulin was approved by the FDA in 2010 for the treatment of metastatic breast cancer (MBC) in the United States (US). More recently, several immuno-oncology (IO) and antibody-drug conjugate (ADC) regimens have been approved for MBC. We assessed the treatment patterns and clinical outcomes in MBC patients treated with eribulin following treatment with an IO or ADC in US clinical practice. In a retrospective patient medical chart review study, patients with MBC, aged ≥18 years, who initiated eribulin therapy between March 1, 2019, and September 30, 2020, treated with either prior IO or ADC in the metastatic setting were included. Patient demographics, treatment characteristics, and clinical outcomes were analyzed descriptively. Real-world progression-free survival (rwPFS) and overall survival (OS) were estimated using Kaplan-Meier analyses. In the study population (N=143), median age at eribulin initiation was 62 years; 64% were Caucasian, and 67% had triple-negative MBC (TNBC). Eribulin therapy was used in the second to fifth line of therapy in the metastatic setting; median treatment duration was 7.2 months. The overall response rate for eribulin was 59.4%. Median rwPFS and OS from eribulin initiation were 21.4 months (95% CI, 12.9-not estimable [NE]) and 24.2 months (95% CI, 17.5-NE), respectively. In patients with TNBC, median rwPFS and OS from eribulin initiation were 12.0 months (95% CI, 8.8-NE) and 18.3 months (95% CI, 14.9-NE), respectively. These real-world data provide evidence for the clinical effectiveness outcomes of eribulin treatment among MBC patients previously treated with an IO or ADC.

Efficacy of smartphone applications to help cancer patients quit smoking: Protocol of the Quit2Heal randomized controlled trial

Cigarette smoking is highly prevalent among cancer patients in the United States (US), with up to half of cancer patients smoking at the time of their initial cancer diagnosis. However, evidence-based cessation programs are rarely implemented in oncology care, and smoking is not consistently treated in cancer treatment settings. Consequently, there is an urgent need for accessible and efficacious cessation treatments that are uniquely tailored to the needs of cancer patients. Here we describe the design and implementation of a randomized controlled trial (RCT) testing the efficacy of a smartphone app (Quit2Heal) versus a US Clinical Practice Guidelines-based app (QuitGuide) for smoking cessation among a planned sample of 422 cancer patients. Quit2Heal is designed to address cancer-related shame, stigma, depression, anxiety, and knowledge about the consequences of smoking/quitting. Quit2Heal is based on the principles of Acceptance and Commitment Therapy, a behavioral therapy that teaches skills for accepting cravings to smoke without smoking, values-driven motivation to quit, and preventing relapse. The primary aim of the RCT is to determine whether Quit2Heal has significantly higher self-reported 30-day point prevalence abstinence at 12 months relative to QuitGuide. The trial will also determine whether Quit2Heal's effect on cessation is (1) mediated by improvements in cancer-related shame, stigma, depression, anxiety, and knowledge about the consequences of smoking/quitting; and (2) moderated by baseline factors (e.g., cancer type, stage, time since diagnosis). If successful, Quit2Heal will offer a more efficacious, broadly scalable smoking cessation treatment that could be implemented alongside existing oncology care, thereby improving cancer outcomes.

Abstract P1-03-03: Real-World Treatment Patterns and Clinical Outcomes in Patients Treated with Eribulin After Prior PI3K Inhibitor Therapy for Metastatic Breast Cancer

Abstract Background: Eribulin has been used in the treatment of metastatic breast cancer (MBC) following approval in 2010 in the United States (US). Recently, several new therapeutic classes have been approved for patients with MBC, including the phosphoinositide 3-kinase (PI3K) inhibitor alpelisib. This study aimed to assess the treatment patterns and clinical outcomes in patients with MBC treated with eribulin following alpelisib in US clinical practice. Methods: A retrospective, noninterventional medical chart review study was performed to obtain de-identified patient data via participating oncologists. Study population included adult female patients with a diagnosis of MBC who initiated eribulin therapy between March 1, 2019, and September 30, 2020, following prior therapy with alpelisib. Eribulin treatment parameters and patient characteristics were captured. Progression-free survival (PFS) and overall survival (OS) since eribulin initiation was assessed using Kaplan-Meier methods. Results: This interim analysis included 47 eligible patients (median age 62 years at eribulin initiation; 77% Caucasian). The majority (91.5%) of this cohort had HR+/HER2– MBC; 72.3% had a known PIK3CA mutation. Eribulin was classified as 2nd line, 3rd line, and 4th line or later in 34%, 47%, and 19% of patients, respectively, in regard to the line of therapy in the metastatic setting. At last follow-up, eribulin treatment was ongoing for 34% of patients. The median treatment duration was 5.2 months (q1, q3: 3.9, 7.1) among those who had discontinued eribulin, and 11.3 months (q1, q3: 10.0, 13.7) among those who were still on treatment. At last follow-up, 72.3% of patients were alive. The estimated PFS rate at 12 months was 57.1% (95% CI: 40.0-70.9). Median was not reached for OS after initiation of eribulin; the estimated OS rates at 12 and 24 months were 80.2% (95% CI: 65.3-89.2) and 61.8% (95% CI: 37.2-79.2), respectively. Conclusion: Among patients with MBC who initiated eribulin following prior treatment with a PI3K inhibitor, over 60% were estimated to survive for at least 2 years. Future studies to confirm these results are warranted. Citation Format: Ravi K. Goyal, Jingchuan Zhang, Keith L. Davis, Peter A. Kaufman. Real-World Treatment Patterns and Clinical Outcomes in Patients Treated with Eribulin After Prior PI3K Inhibitor Therapy for Metastatic Breast Cancer [abstract]. In: Proceedings of the 2022 San Antonio Breast Cancer Symposium; 2022 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2023;83(5 Suppl):Abstract nr P1-03-03.

Estimation of Long-Term Post-Infusion Costs of Care for Patients with Relapsed/Refractory Multiple Myeloma (RRMM) Following Administration of Idecabtagene Vicleucel (ide-cel, bb2121) in the KarMMa Clinical Trial

Introduction: RRMM is associated with poor prognosis and decreased overall survival. Chimeric antigen receptor (CAR) T cell therapies have emerged as a potential single-infusion treatment option for these patients. Ide-cel (bb2121), a B-cell maturation antigen (BCMA)-directed CAR T cell therapy for treatment of RRMM, has demonstrated frequent, deep, and durable responses in heavily pretreated patients in the KarMMa clinical trial (Munshi NC, et al. N Engl J Med 2021;384:705-716). However, evidence on long-term healthcare resource utilization (HCRU) and associated costs of care following ide-cel administration is very limited; this abstract provides a long-term update to a previous 12-month (mo) cost-of-care analysis (Hari P, et al. Value Health 2021;24[Suppl 1]:S29-S30). The objective of this study was to estimate the 2-year (y) post-infusion cost of care for patients with RRMM who received ide-cel in the KarMMa trial. Methods: This retrospective study used the KarMMa clinical trial database to analyze the HCRU and cost associated with ide-cel. We identified resource use and employed a micro-costing methodology to estimate post-infusion costs. Beginning on the index date (day of ide-cel infusion) up to a 2-y follow-up period, we analyzed the following HCRU categories: facility (standard inpatient [IP] and intensive care unit [ICU]), diagnostic (eg, lab work and imaging), medication (eg, supportive care, prophylactic treatment, and adverse event management), and procedure (eg, dialysis and intubation); and length of stay (LOS) (number of standard IP days and ICU days). Given that some patients in the trial were administered ide-cel outside the United States (US), any HCRU inconsistent with US clinical practice were adapted to reflect the US perspective. Next, we applied unit costs to each HCRU in the time period. Unit costs were from the US health system (provider) perspective and adjusted to 2022 USD. Cost per standard IP day ($2944) was estimated from 2018 Healthcare Cost and Utilization Project databases and cost per ICU day ($8426) sourced from Dasta JF, et al. Crit Care Med 2005;33:1266-1271. Medication cost data were obtained from REDBOOKTM using wholesale acquisition cost and were uniformly applied across sites of care. Diagnostic, transfusion, and procedure costs were taken from the Centers for Medicare & Medicaid Services lab fee schedule, physician fee schedule, or outpatient prospective payment system. Unit costs were estimated by applying a payment-to-cost ratio to any Medicare reimbursement rates. Costs were then adjusted to reflect cost to the site of care where the HCRU occurred. Costs were aggregated by HCRU category (facility, medication, procedure, and diagnostic) and partitioned by mo following ide-cel administration. An average total cost by post ide-cel infusion mo was calculated among patients with ongoing status in that mo. Patients censored because of data cut off were excluded. Results: A total of 128 patients were treated with ide-cel in the KarMMa trial. Patients had a mean (standard deviation [SD]) age of 59.8 (9.3) y, and were predominantly male (59.4%), White (80.5%), and non-Hispanic (80.5%). HCRU and LOS, including standard IP and ICU days, are shown in Table 1. Mean (SD) LOS throughout the follow-up period was 22.3 (13.3) days for standard IP and 1.6 (4.8) days for ICU. Total LOS ranged from 14 to 114 days with a median of 19.5 days and a mean (SD) of 23.9 (14.4) days (protocol required IP stay ≥ 14 days post infusion). HCRU, especially in y 2 and for medications, may have been related to management of toxicity or progressive disease. All patients were hospitalized (per trial protocol); 20.3% had an ICU stay. Most patients received antibiotics (96.1%) and tocilizumab + corticosteroids (54.7%), some received corticosteroids only (45.3%), and few had vasopressor use (14.8%). A minority of patients required dialysis (0.8%) or intubation (3.9%). The estimated mean 2-y post-infusion cost of care was $123,157. Most of the 2 y costs post-infusion were incurred in y 1 (85.7%) with 54% in mo 1 and were driven primarily by facility costs, namely standard IP and ICU stays (Figure 1). Conclusions: This study is the first to estimate long-term (national average-based) cost of care following CAR T cell therapy for patients with RRMM. Findings suggest that a single infusion of ide-cel is associated with substantially reduced HCRU and long-term costs following the initial treatment period. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Effectiveness of Microinvasive Glaucoma Surgery in the United States: Intelligent Research in Sight Registry Analysis 2013–2019

To evaluate the effectiveness of microinvasive glaucoma surgery (MIGS) with and without concurrent phacoemulsification. Multicenter, retrospective cohort study. Patients in the Intelligent Research in Sight (IRIS®) Registry who underwent Xen gel stent (ab interno) implantation, endoscopic cyclophotocoagulation (ECP), or goniotomy or canaloplasty from 2013 through2019. Kaplan-Meier survival analysis was used to assess reoperation rates. We defined reoperation as any subsequent glaucoma surgery occurring 1 month to 3 years after the initial procedure. Multivariable Cox proportional hazard models were used to determine factors predictive of reoperation. Reoperation rate, mean intraocular pressure (IOP) and visual acuity (VA), postoperative complications, predictors of reoperation, and reoperation procedure type. A total of 79 363 eyes from 57 561 patients were included, with 15 118 eyes (19%) receiving stand-alone MIGS and 64 245 eyes (81%) receiving MIGS concurrent with phacoemulsification. Overall, patients who underwent MIGS concurrently with phacoemulsification showed lower reoperation rates compared with stand-alone MIGS, most pronounced in ECP and goniotomy or canaloplasty. At postoperative year 2, the cumulative reoperation rate for stand-alone procedures was 15% for ECP, 24% for Xen implantation, and 24% for goniotomy or canaloplasty compared with 3% for ECP, 19% for Xen implantation, and 6% for goniotomy or canaloplasty concurrent with phacoemulsification (P < 0.001 for each stand-alone MIGS vs. MIGS with phacoemulsification). Black race, older age, moderate and severe glaucoma, higher baseline IOP, and glaucoma subtype were associated with higher reoperation risk. Although IOP decreased in all groups, stand-alone MIGS showed a more substantial decrease in mean IOP. Complication rates from MIGS were low overall: 1% for ECP, 1% for Xen implantation, and 2% for goniotomy or canaloplasty. In current United States clinical practice, MIGS has substantially lower reoperation rates when performed with phacoemulsification, especially for ECP and goniotomy or canaloplasty. Approximately one-sixth of patients undergoing stand-alone ECP and one-quarter of patients undergoing stand-alone Xen implantation or goniotomy or canaloplasty require reoperation by 2 years. Black race, diagnosis coding of moderate to severe glaucoma, and higher baseline IOP were associated with higher risk of reoperation after MIGS procedures. Proprietary or commercial disclosure may be found after the references.

Heart failure across the range of preserved ejection fraction in United States clinical practice

Abstract Introduction Recent clinical trials of heart failure with preserved ejection fraction (HFpEF) have observed varying patient profiles by ejection fraction (EF), with attenuation of treatment benefits as EF increases. In routine clinical practice, the degree to which patients hospitalized for HF with EF≥60% may differ from those with lower EF is unknown. Purpose To compare patient characteristics, treatment patterns, and clinical outcomes across the range of EF among patients hospitalized for HFpEF. Methods Using the Humedica electronic medical records database between Jan 2010 and Dec 2020, patients hospitalized for a primary diagnosis of HF with EF&amp;gt;40% and who were haemodynamically stable at admission, without concurrent acute coronary syndrome or end-stage renal disease, and treated with intravenous (IV) diuretic agents within 48 h of admission were identified. Patient characteristics, treatment patterns, and clinical outcomes were compared by EF ranges of 41–49%, 50–59%, and ≥60%. Results Of 47,026 patients hospitalized with HFpEF, 6,335 (13%) had EF 41–49%, 18,603 (40%) had EF 50–59%, and 22,088 (47%) had EF≥60%. Across all 3 groups, patients were similar with respect to age (median 77 years for each group), race (83–84% White, 12–13% Black), systolic blood pressure (137–138 mmHg at admission), and eGFR (63–64 mL/min/1.73 m2 at admission). With progressively higher EF group, the proportion of women increased (45% vs 54% vs 65%) and median NT-proBNP decreased (4,221 vs 2,945 vs 2,234 pg/mL). Patients with EF ≥60% had the lowest rates of coronary artery disease and atrial fibrillation, and the highest rates of chronic pulmonary disease (Figure 1, Panel A). Discharge medications were generally similar, with exception of less beta-blocker use and more calcium channel blocker use among those with EF ≥60% (Figure 1, Panel B). Discharge use of angiotensin receptor-neprilysin inhibitor and sodium glucose cotransporter-2 inhibitor therapies were each &amp;lt;1% in all groups. Hospital length of stay (median 4 days for each group) and in-hospital mortality (1.1–1.3%) were similar across groups, but rates of in-hospital acute respiratory failure were higher among patients with EF ≥60% (27% vs 230-25% for lower EF groups). Rates of 30-day and 12-month post-discharge clinical events were high irrespective of EF, without meaningful differences between groups (Figure 2). Conclusion In a contemporary real-world population of US patients hospitalized for HF with EF &amp;gt;40%, nearly half had an EF≥60%. While clinical profiles and discharge medications varied, post-discharge outcomes were similarly poor irrespective of EF. There remain important opportunities to improve the care and outcomes for patients with HF across the range of preserved ejection fraction. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): MyoKardia, Inc., a wholly owned subsidiary of Bristol Myers Squibb

Web-delivered Acceptance and Commitment Therapy (ACT) for smoking cessation: Is it engaging and efficacious for US Hispanic/Latinx adult smokers?

Hispanic/Latinx adult smokers in the United States (US) face barriers to receiving and utilizing evidenced-based cessation treatments compared with other racial/ethnic groups. The lack of efficacious and accessible smoking cessation treatments for this population further contributes to such smoking disparities. In a secondary analysis, we explored the efficacy of an Acceptance and Commitment Therapy (ACT)-based website (WebQuit.org) versus a US Clinical Practice Guidelines (USCPG)-based website (Smokefree.gov) for smoking cessation in a subset of Hispanic/Latinx adult participants enrolled in the WebQuit trial. Of the 2,637 participants who were randomized in the parent trial, 222 were Hispanic/Latinx (n = 101 in WebQuit, n = 121 in Smokefree). Smoking cessation outcomes were measured at 3, 6, and 12-months. The primary outcome was self-reported complete-case 30-day point prevalence abstinence (PPA) at 12-months. Treatment engagement and satisfaction, change in acceptance of urges to smoke, and commitment to quitting smoking were compared across conditions. Retention rate was 88% at 12-months. WebQuit participants had higher odds of smoking cessation compared to Smokefree participants at 12-months (40% vs. 25%; OR = 1.93 95% CI: 1.04, 3.59). Findings were similar using multiple imputation. WebQuit participants engaged more with the website than Smokefree participants through multiple indicators of engagement, including spending more time using the website (IRR = 2.32; 95% CI: 1.68, 3.20). Although WebQuit participants engaged more with the website than Smokefree participants, there was no evidence that differences in quit rates were mediated by engagement level. This study provides initial empirical evidence that digital interventions may be efficacious for helping Hispanic/Latinx adults quit smoking.

P-237: Real-world treatment patterns and clinical outcomes in patients with triple-class exposed relapsed or refractory multiple myeloma in United States clinical practice

P-237: Real-world treatment patterns and clinical outcomes in patients with triple-class exposed relapsed or refractory multiple myeloma in United States clinical practice

Opportunities for personalizing colorectal cancer care: an analysis of SEER-medicare data.

United States clinical practice guidelines for metastatic colorectal cancer recommend use of medications impacted by genetic variants but do not recommend testing. We analyzed real-world treatment using a cancer registry and claims dataset to explore pharmacogenomic (PGx) medication treatment patterns and characterize exposure. In a cohort of 6 957 patients, most (86.9%) were exposed to at least one chemotherapy medication with PGx guidelines. In a cohort of 2 223 patients with retail pharmacy claims available, most (79.2%) were treated with at least one non-chemotherapy (79.2%) medication with PGx guidelines. PGx-associated chemotherapy exposure was associated with age, race/ethnicity, educational attainment, and rurality. PGx-associated non-chemotherapy exposure was associated with medication use and comorbidities. The potential impact of PGx testing is large and policies aimed at increasing PGx testing at diagnosis may impact treatment decisions for patients with metastatic colorectal cancer as most patients are exposed to medications with pharmacogenomics implications during treatment.

Efficacy and utilization of an acceptance and commitment therapy-based smartphone application for smoking cessation among Black adults: secondary analysis of the iCanQuit randomized trial.

Black adults who smoke are less likely to seek treatment and to succeed in quitting compared with other racial groups. The lack of efficacious and engaging trials for smoking cessation further contributes to this disparity. This study explored whether an acceptance and commitment therapy (ACT)-based smartphone application (iCanQuit) was more efficacious for smoking cessation than a United States Clinical Practice Guidelines (USCPG)-based smartphone application (QuitGuide) among Black adults. Secondary analysis of a two-arm randomized trial with 12-month follow-up. United States (US). A total of 554 Black adults who smoke daily were recruited from 34 US states and enrolled between May 2017 and September 2018. Participants were randomized to receive iCanQuit (n = 274) or QuitGuide (n = 280) for 12 months. Smoking cessation outcomes were measured at 3, 6, and 12 months. The primary outcome was self-reported complete-case 30-day point prevalence abstinence (PPA) at 12 months. Secondary outcomes were 7-day PPA, missing-as-smoking imputation, multiple imputation, prolonged abstinence, and cessation of all tobacco products at 12 months. Study retention, treatment engagement, and change in ACT-based processes were also compared between arms. Study retention was 89% at 12 months and did not differ by arm (P > 0.05). The complete-case 30-day PPA was 28% for iCanQuit versus 20% for QuitGuide at 12 months (odds ratio [OR] = 1.60; 95% confidence interval [CI] = 1.03, 2.46). Similar associations were observed for the missing-as-smoking imputation, although non-significant (25% iCanQuit vs 18% QuitGuide; OR = 1.50; 95% CI = 0.98, 2.30). iCanQuit vs QuitGuide participants were significantly more engaged with iCanQuit application as measured by the number of logins from baseline to 6months (incidence rate ratio = 3.26; 95% CI = 2.58, 4.13). Increased acceptance of cues to smoke mediated the effect of treatment on cessation (indirect effect: OR = 0.20; 95% CI = 0.05, 0.29). Among Black adults, an acceptance and commitment therapy-based smartphone application appeared to be more efficacious and engaging for smoking cessation than the United States Clinical Practice Guidelines-based QuitGuide application.

Author Diversity on Clinical Practice Guideline Committees.

Reports have demonstrated that women overall and women physicians, in particular, are underrepresented as authors of clinical practice guidelines. This analysis used publicly available information to explore the diversity of rehabilitation-related clinical practice guideline authors by gender, race, and ethnicity. Primary analysis identified authors' gender, race, ethnicity, and visible minority status. Two sets were analyzed: (1) clinical practice guidelines by Department of Veterans Affairs (VA) categorized as "Rehabilitation" or "Pain" (n = 7; VA clinical practice guidelines) and (2) a set (n = 10) published in the United States (US) from 2019 to 2021 that were selected because of low numbers of inclusion at less than 20% women authors. Key findings include that among physician authors, both the VA and US clinical practice guidelines underrepresented women (15 [24.2%] and 27 [16.7%], respectively) and those coded as a racial or ethnic minority were particularly underrepresented. Notably, women authors overall were equally represented (92 [50.0%]) in the VA clinical practice guidelines. The US clinical practice guidelines had women authors who were underrepresented (36 [19.0%]). Secondary analysis of the entire set of VA clinical practice guidelines (n = 21) found gaps in diversity-related content. Clinical practice guidelines have far-reaching health and economic impacts, and addressing disparities in the diversity of author teams and/or gaps in diversity-related content is of paramount importance.

Patient characteristics and acute cardiovascular event rates among patients with very high‐risk and non‐very high‐risk atherosclerotic cardiovascular disease

BackgroundThe risk for subsequent major cardiovascular (CV) events among patients with very high‐risk (VHR) atherosclerotic CV disease (ASCVD) remains to be fully elucidated.HypothesisWe assessed the characteristics and major CV event rates of patients with VHR versus non‐VHR ASCVD in a real‐world setting in the United States (US), hypothesizing that patients with VHR ASCVD would have higher CV event rates.MethodsThis was a retrospective cohort study conducted from January 01, 2011, to June 30, 2018, in the US using the Prognos LDL‐C database linked to the IQVIA PharMetrics Plus® database supplemented with the IQVIA prescription claims (Dx/LRx) databases. Patients were ≥18 years old and had ≥2 non‐ancillary medical claims in the linked databases at least 30 days apart. The study was conducted in 2 stages: (1) identification of patients with ASCVD who met the definition of VHR ASCVD and a matched cohort of non‐VHR ASCVD patients using the incidence density sampling (IDS) approach; (2) estimation of the occurrence of major CV events.ResultsAmong patients with ≥1 major ASCVD event (N=147,679), most qualified as VHR ASCVD (79.5%). There were 115,460 patients each in IDS‐matched VHR and non‐VHR ASCVD cohorts. The composite myocardial infarction/ischemic stroke event rates in the VHR and non‐VHR ASCVD cohorts were 8.04 (95% confidence interval [95% CI]: 7.87‐8.22) and 0.82 (95% CI: 0.77‐0.88) events per 100 patient‐years, respectively, during the 1‐year post‐index period.ConclusionsMost patients with ≥1 previous major ASCVD event treated in real‐world US clinical practice qualified as VHR ASCVD. Patients with VHR ASCVD had much higher rates of major CV events versus non‐VHR ASCVD patients.

Factors associated with successful implementation of telehealth abortion in 4 United States clinical practice settings

ObjectiveTo overcome obstacles to delivering medication abortion services during the COVID-19 pandemic, clinics and providers implemented new medication abortion service models not requiring in-person care. This study identifies organizational factors that promoted successful implementation of telehealth and adoption of “no test” medication abortion protocols. Study designWe conducted 21 semi-structured, in-depth interviews with health care providers and clinic administrators implementing clinician-supported telehealth abortion during the COVID pandemic. We selected 15 clinical sites to represent 4 different practice settings: independent primary care practices, online medical services, specialty family planning clinics, and primary care clinics within multispecialty health systems. The Consolidated Framework for Implementation Research guided our thematic analysis. ResultsSuccessful implementation of telehealth abortion included access to formal and informal inter-organizational networks, including professional organizations and informal mentorship relationships with innovators in the field; organizational readiness for implementation, such as having clinic resources available for telehealth services like functional electronic health records and options for easy-to-use virtual patient-provider interactions; and motivated and effective clinic champions. ConclusionsIn response to the need to offer remote clinical services, 4 different practice settings types leveraged key operational factors to facilitate successful implementation of telehealth abortion. Information from this study can inform implementation strategies to support the dissemination and adoption of this model. ImplicationsExamples of successfully implemented telehealth medication abortion services provide a framework that can be used to inform and implement similar patient-centered telehealth models in diverse practice settings.

Evaluating Nontechnical Skills in US Emergency Departments Using Simulation: Validating and Contextualizing a UK Assessment Tool.

Nontechnical skills (NTS) in medicine are the "cognitive, social, and personal resource skills that complement technical skills contributing to safe and efficient care." We aimed to (1) evaluate the validity and reliability of a 12-element United Kingdom emergency medicine (EM) NTS assessment tool in the context of United States (US) EM practice and (2) identify behaviors unique to US clinical practice. This was a mixed methods study conducted in 2 phases, following Kane's validity framework. The intended use of the NTS tool is to provide formative assessment of US EM physicians (EPs) from a video of simulated clinical encounters. In phase I, a focus group assessed the appropriateness of each aspect of the tool in the context of US EM practice by reviewing and identifying the NTS of an EP in a simulated clinical scenario. In phase II, EPs (N = 208) attending a national EM conference evaluated an EP's behaviors in 1 of 2 video simulations. Reliability in the form of internal consistency was calculated using Cronbach α. All participants suggested exemplar behaviors for the 12 elements in the context of their own clinical practice and generated new assessment elements. Internal consistency was acceptable (α > 0.7) for all categories, except teamwork and cooperation. Participants proposed 4 novel behavioral elements and suggested US exemplar behaviors for all 12 original elements. This tool can be used to assess US EP's NTS for the purpose of formative assessment. Refinement of exemplar behaviors and inclusion of novel US-specific elements may optimize usability.