Real-world treatment patterns and clinical outcomes in patients treated with eribulin after prior phosphoinositide 3-Kinase inhibitor treatment for metastatic breast cancer.

PURPOSE: In 2010, the US Food and Drug Administration approved eribulin for the treatment of metastatic breast cancer (MBC). Since then, the treatment landscape has evolved with many new therapy classes, a more recent one being the small molecule inhibitors of phosphoinositide 3 kinase (PI3K). We sought to characterize the treatment patterns and clinical outcomes of patients with MBC who received eribulin following prior treatment with a PI3K inhibitor. METHODS: A retrospective cohort study based on medical record review included MBC patients who initiated eribulin between March 2019 and September 2020 following prior treatment with a PI3K inhibitor was conducted. Patient demographics, treatment characteristics, and clinical outcomes were analyzed descriptively. Real-world progression-free survival (rwPFS) and overall survival (OS) were estimated from the initiation of eribulin therapy using Kaplan-Meier analyses. RESULTS: 82 eligible patients were included. Patients' median age at eribulin initiation was 62 years; 86.5% had hormone receptor-positive, human epidermal growth factor receptor 2-negative tumors. Eribulin was most often administered in the second or third line (82.9%) in the metastatic setting. Best overall response on eribulin was reported as complete or partial response in 72% of the patients. The median rwPFS was 18.9 months (95% confidence interval [CI], 12.4-not estimable); median OS was not reached. The estimated rwPFS and OS rates at 12 months were 63.3% (95% CI, 50.5-73.7) and 82.6% (95% CI, 72.4-89.3), respectively. CONCLUSION: Our real-world study suggests that eribulin may be a potential treatment option for MBC patients who fail a prior PI3K inhibitor.

Effectiveness of standard treatments in non-small-cell lung cancer with METexon14 skipping mutation: a real-world study.

Aim: To assess real-world clinical outcomes with standard therapies for advanced non-small-cell lung cancer (aNSCLC) with METexon14 skipping mutation (METex14). Methods: In an oncologists-led retrospective review of medical records, data were abstracted and analyzed for patients initiating first-line (1L) systemic therapy after 1 January 2017. Results: In total 287 aNSCLC patients with METex14, the real-world best overall response rate was 73.4% for capmatinib (n = 146), 68.8% for immunotherapy (IO) monotherapy (n = 48), 52.0% for chemotherapy (CT, n = 30), and 54.8% for IO + CT (n = 63). As compared with capmatinib, patients receiving IO (hazard ratio [HR]: 1.57; 95% CI: 0.77-3.20; p = 0.220), CT (HR: 2.41; 95% CI: 1.19-4.85; p = 0.014) and IO + CT (HR: 2.33; 95% CI: 1.35-4.04; p = 0.003) had higher rates of progression. Further, patients receiving CT (HR: 4.43; 95% CI: 1.54-12.75; p = 0.006) and IO + CT (HR: 3.53, 95% CI: 1.41-8.85; p = 0.007) had higher rates of mortality than patients receiving capmatinib. Conclusion: The study showed better clinical outcomes with capmatinib than other standard therapies in 1L setting for aNSCLC harboring METex14.

Real-world study that investigated the outcomes of different therapies used to treat non-small-cell lung cancer patients with mesenchymal-epithelial transition exon 14 skipping mutationWhat is this article about? A real-world study that investigated clinical outcomes in patients with diagnosis of advanced non-small-cell lung cancer (aNSCLC) with mesenchymal-epithelial transition exon 14 (METex14) skipping­a rare form of genetic mutation­who received treatment with one of the commonly used therapies for this disease: immunotherapy, chemotherapy, immunotherapy + chemotherapy combination and capmatinib, which is a highly selective inhibitor of MET tyrosine kinase protein involved in the growth of cancer cells.What were the results? The study showed that, in general, patients treated with capmatinib as the frontline therapy more frequently achieved a clinical response in the form of complete tumor resolution or tumor shrinkage, had a lower risk of disease worsening and lived longer than patients who were treated with immunotherapy, chemotherapy or immunotherapy + chemotherapy combination.What do the results of the study mean? This study suggests that capmatinib is effective in treating patients with aNSCLC with METex14 skipping who have not been treated with another anticancer therapy previously. It provides evidence to support the use of capmatinib in the frontline setting and may inform clinical decision-making in routine practice.

Real-World Effectiveness of Lenvatinib in Hepatocellular Carcinoma Patients with Nonalcoholic Steatohepatitis.

Hepatocellular (HCC) is the most common type of primary liver cancer and the fourth most common cause of cancer-related deaths globally.1 Although most cases of HCC were historically attributed to underlying chronic viral hepatitis, nonalcoholic fatty liver disease is projected to become the most common risk factor for HCC with the rising prevalence of obesity and diabetes mellitus and increasing availability of effective treatments for hepatitis B and C infection.2 Although patients with viral and nonviral HCC seem to have similar overall prognosis,3 prior data have suggested possible differential efficacy of systemic therapies by liver disease etiology. For example, sorafenib was shown to have greater efficacy in patients with chronic hepatitis C infection than other etiologies.4 The aim of our descriptive study was to report the effectiveness of lenvatinib in a real-world cohort of patients with nonalcoholic steatohepatitis (NASH)-related HCC.

Real-world outcomes in non-small-cell lung cancer patients with MET Exon 14 skipping mutation and brain metastases treated with capmatinib.

Aim: To assess real-world clinical outcomes in patients with non-small-cell lung cancer with MET exon 14 skipping mutation and brain metastases (BM) who received capmatinib, a recently approved MET inhibitor, in routine US clinical practice. Materials & methods: Patient data were collected using a retrospective medical record review, led by participating oncologists. Eligible patients initiated treatment with capmatinib in any line, after BM diagnosis, between May 2020 and June 2021. Data on real-world overall response rate (rwORR) and real-world progression-free survival (rwPFS) were descriptively analyzed. Results: 68 eligible patients were analyzed. In patients treated with first-line (1L) capmatinib (n = 55), the rwORR was 90.9% systemically and 87.3% intracranially; median systemic rwPFS was 14.1 months. Among radiation-naive patients on 1L capmatinib (n = 20), rwORR was 85.0%, both systemically and intracranially; median systemic rwPFS was 14.1 months. Conclusion: This study showed substantial systemic and intracranial effectiveness for capmatinib in real-world setting; findings were consistent for RT-naive patients.

Real-world clinical outcomes of patients with myelofibrosis treated with ruxolitinib: a medical record review.

Aim: To assess real-world ruxolitinib treatment patterns and outcomes in patients diagnosed with primary or secondary myelofibrosis. Materials & methods: Patient medical records were reviewed in six countries. Results: Eligible patients (n = 469) had a mean age of 63.5 years, and most were male (66.5%) with primary myelofibrosis (78.5%). Median duration of ruxolitinib treatment was 13.1 months; 40% of patients initiated treatment at the recommended dose. The Kaplan-Meier estimate of median survival from ruxolitinib initiation was 44.4 months (95% CI, 38.8-50.2 months). Approximately one quarter (23%) of patients continued ruxolitinib after progression. Conclusion: These results suggest an unmet need for more effective treatments for patients with myelofibrosis who failed ruxolitinib.

Real-world effectiveness of lenvatinib monotherapy among unresectable hepatocellular carcinoma patients in the USA.

Aim: This study evaluated the effectiveness of lenvatinib monotherapy for first-line treatment of unresectable hepatocellular carcinoma (uHCC) in a real-world setting. Materials & methods: This retrospective cohort study included patients who initiated lenvatinib monotherapy as first-line treatment for uHCC (n = 233). Clinical outcomes included provider-reported best response, progression-free survival (PFS) and overall survival (OS). PFS and OS were estimated using Kaplan-Meier methods. Results: Most patients (67.8%) were male. A total of 44.6% had Child-Pugh A and 39.1% had Child-Pugh B. Dose reductions were reported in 9%. Median PFS and OS were not reached. At 6 and 12 months, landmark PFS were 85.1 and 64.9%, respectively; landmark OS were 91.8 and 72.6%, respectively. Conclusion: These results affirm the clinical effectiveness of first-line lenvatinib monotherapy in uHCC.

Lay abstract Lenvatinib is a targeted therapy that prevents tumor growth. It was approved for the treatment of advanced liver cancer in 2018, but few studies have examined how it is used in everyday clinical practice, especially in the USA. In this study, we reviewed the medical records of 233 patients in the USA with unresectable hepatocellular carcinoma, who were treated with lenvatinib in first line to better understand its effectiveness and use in real-world care. We collected information on how long they were on treatment and time to tumor progression and/or death. Overall, our study found that in this demographically and clinically diverse sample, results affirm findings from prior studies that found lenvatinib is an effective treatment for patients with unresectable hepatocellular carcinoma.

POLARIS: a prospective, multicenter, noninterventional study assessing palbociclib in hormone receptor-positive advanced breast cancer.

This report describes the rationale, purpose and design of the POLARIS study. POLARIS is an ongoing noninterventional, prospective, multicenter study. Female and male patients in the USA and Canada diagnosed with hormone receptor-positive/HER2-negative metastatic breast cancer were enrolled in the study and treated with the cyclin-dependent kinase 4/6 inhibitor palbociclib when hormone receptor-positive/HER2-negative metastatic breast cancer was deemed to be indicated by their physician. The study will provide real-world data on palbociclib prescribing and treatment patterns in routine clinical practice, associated clinical outcomes, treatment sequencing in the advanced/metastatic setting, patient quality of life and geriatric-specific assessments. The tumor genomic landscape in relation to clinical outcomes will be explored. POLARIS will identify benefits and side effects of palbociclib across multiple lines of therapy and in discrete subsets of patients. Clinical Trial Registration: NCT03280303 (ClinicalTrials.gov).

Treatment patterns and characteristics of patients with migraine in Japan: A retrospective analysis of health insurance claims data.

OBJECTIVE: To describe treatment patterns of migraine patients in the Japan Medical Data Center (JMDC) database. METHODS: Patients aged ≥18 years with ≥1 inpatient or ≥2 outpatient migraine diagnoses, ≥1 outpatient diagnosis and ≥1 migraine-specific acute treatment (triptan or ergotamine), or ≥2 migraine-specific acute treatments from 1 May 2011 to 30 April 2014 were identified. Patients were required to be enrolled in a health plan for ≥1 year before and after the index date. The first migraine diagnosis or acute treatment defined the index date. Patients were stratified by the migraine treatments observed after the index date (i.e. migraine-specific acute treatment only [AT], prophylactic with or without migraine-specific acute treatment [PT], or no treatment [NT]) and described regarding the first migraine treatment regimen and subsequent treatment patterns during up to 1 year of follow-up. RESULTS: A total of 16,443 patients met the eligibility criteria (9873 AT, 3022 PT, and 3548 NT). AT patients had mean (SD) 10.3 (20.5) acute treatment days during 1-year follow-up, and 81.9% received triptans. When assessing the first migraine treatment regimen during follow-up in PT patients, 29.2% received prophylactic treatment only and 51.7% received both acute and prophylactic treatment. Calcium-channel blockers with or without concomitant triptans (34.4%) were the most common first regimen. Approximately 62.2% discontinued initial prophylactic treatment after an average of 61.2 days (SD = 65.3) of persistent treatment. Among discontinuers, 15.2% reinitiated original treatment and 7.0% switched treatment post-discontinuation within a year, while the remaining patients did not receive prophylactic therapy following discontinuation. CONCLUSIONS: Among Japanese migraine patients, prophylactic use was low and associated with a high rate of discontinuation following a brief treatment period. Many patients reinitiated or switched treatment following discontinuation, while a significant proportion of patients remained discontinued from prophylactic therapy, suggesting a high unmet need.

Achievement of Target A1C <7.0% (<53 mmol/mol) by U.S. Type 2 Diabetes Patients Treated With Basal Insulin in Both Randomized Controlled Trials and Clinical Practice.

OBJECTIVE: Many patients with type 2 diabetes do not reach glycemic goals despite basal insulin treatment. This study assessed the achievement of a target A1C <7.0% (<53 mmol/mol) after initiation of basal insulin in two settings. METHODS: This was a retrospective analysis of pooled randomized controlled trial (RCT) data, from 11 24-week studies of patients initiating basal insulin performed between 2000 and 2005 and of outpatient electronic medical record (EMR) data from the General Electric Centricity database for insulin-naive patients initiating basal insulin between 2005 and 2012. Baseline characteristics stratified by target A1C and fasting plasma glucose (FPG) attainment were compared descriptively. RESULTS: In the RCT dataset, 49.0% of patients failed to achieve the target A1C at 6 months versus 72.4% and 72.9% at 6 and 12 months in the EMR dataset, respectively. Despite this, in the RCT dataset, 79.4% of patients achieved the target A1C and/or an FPG <130 mg/dL. In the EMR dataset, only 47.6% and 47.3% of patients achieved an A1C <7.0% and/or FPG <130 mg/dL at 6 and 12 months, respectively. Overall, patients with an A1C >7.0% had a longer diabetes duration and were more likely to be female, nonwhite, and self-funding or covered by Medicaid. Among patients with an A1C >7.0%, more RCT patients (58.0%) had an FPG <130 mg/dL than EMR patients at 6 months (27.8%) and 12 months (27.7%). CONCLUSION: Unmet needs remain after basal insulin initiation, particularly in real-world settings, where many patients require further insulin titration. In both populations, patients failing to achieve the target A1C despite attaining an FPG <130 mg/dL require interventions to improve postprandial control.

Comparison of demographics, treatment patterns, health care utilization, and costs among elderly patients with extensive-stage small cell and metastatic non-small cell lung cancers.

BACKGROUND: Limited data exist regarding real-world treatment patterns, resource utilization, and costs of extensive-stage small cell lung cancer (esSCLC) among elderly patients in the United States. While abundant data are available on treatment patterns in metastatic non-small cell lung cancer (mNSCLC), to our knowledge no data exist comparing costs and resource use between patients with esSCLC or mNSCLC. METHODS: We retrospectively analyzed administrative claims data (2000-2008) of patients aged ≥65 years from the linked Surveillance, Epidemiology and End Results (SEER)-Medicare database. Patients were selected on the basis of having newly diagnosed esSCLC (n=5,855) or mNSCLC (n=24,090) during 1/1/2000-12/31/2005, and were required to have received cancer-directed therapy. Survival and other measures were compared between esSCLC and mNSCLC patients using Kaplan-Meier log-rank and univariate chi-square and t-tests. Study measures were followed from first diagnosis date of either esSCLC or mNSCLC until the earlier of death or end of the database. RESULTS: Survival between the cohorts did not differ significantly: mean of 10.4 months for esSCLC patients versus 11.1 months for mNSCLC; median survival was 7.4 months versus 5.9 months. A higher percentage of mNSCLC patients (vs. esSCLC) received radiation therapy (75.6% vs. 65.4%; P < 0.001) and surgery (13.6% vs. 7.8%; P < 0.001) during the metastatic disease period. Conversely, a higher percentage of esSCLC patients than mNSCLC patients received chemotherapy (85.5% vs. 60.3%; P < 0.001), red blood-cell transfusion (20.7% vs. 10.9%; P < 0.001), platelet transfusion (5.6% vs. 1.8%; P < 0.001), and growth-factor support (59.0% vs. 39.5%; P < 0.001). esSCLC patients incurred higher lifetime disease-related costs ($44,167 vs. $37,932; P < 0.001) and all-cause costs ($70,549 vs. $67,176; P < 0.001) than mNSCLC patients. CONCLUSIONS: Lifetime total and disease-related costs per patient were high. Increased use of chemotherapy, supportive care therapies (including growth factors), and disease-related hospitalizations were observed in esSCLC patients as compared with mNSCLC patients. Disease-related and all-cause costs for esSCLC also exceeded those of mNSCLC, except for hospice and skilled nursing services. Survival and per-patient costs for both groups underscore the unmet medical need for more effective therapies in patients with esSCLC or mNSCLC.

Real-World Treatment Patterns and Clinical Outcomes Among Patients With Advanced Renal Cell Carcinoma.

BACKGROUND: Nearly 30% of new renal cell carcinoma (RCC) cases are diagnosed at an advanced or metastatic stage. Recent approvals of immunotherapies (IO) have significantly impacted patient care, but real-world outcomes of these treatments have not been widely evaluated. METHODS: Eligible physicians abstracted demographic and clinical data from patient medical records for patients with advanced clear and non-clear cell RCC (aRCC) who initiated treatment between January 1, 2018, and December 31, 2020. Overall survival (OS) and progression-free survival (PFS) were estimated by the Kaplan-Meier method. A multivariate Cox regression model was developed to assess the impact of treatment category on clinical outcomes while controlling for International Metastatic RCC Database Consortium (IMDC) risk category, histology, and other patient characteristics. RESULTS: A total of 498 patients were included (201 from US, 62 from Canada, 58 from UK, 59 from France, 58 from Germany, 60 from Spain). Of these, 250 received tyrosine kinase inhibitor (TKI) monotherapy, 197 received immunotherapy (IO) combination (119 IO+TKI, 78 IO+IO), and 32 received IO monotherapy as first-line treatment for aRCC; 19 patients received various other regimens. 16% of patients had a favorable IMDC risk score. Based on results of multivariable Cox regression, PFS (hazard ratio [HR] [95% confidence interval (CI)]: 0.50 [0.36-0.72]) (P < .001) and time to next treatment (TTNT) were significantly longer (HR [95% CI]: 0.54 [0.39-0.73]) (P < .001) for patients treated with IO combination versus TKI monotherapy. IO combination had a numerically reduced, but statistically insignificant, risk of death versus TKI monotherapy (HR: 0.66; P = .114). IO+TKI combination was associated with significantly longer PFS and reduced risk of progression (HR: 0.52; P = .04) versus IO+IO combination; similar results were observed for TTNT (HR: 0.57; P = .03). CONCLUSION: Our evaluation of real-world treatment outcomes in aRCC revealed that IO + TKI combination is associated with improved PFS and prolonged TTNT compared with TKI monotherapy and IO+IO combination.

Real-world effectiveness of lenvatinib monotherapy in previously treated unresectable hepatocellular carcinoma in US clinical practice.

BACKGROUND: Lenvatinib monotherapy was approved in the United States for first-line treatment of patients with unresectable hepatocellular carcinoma (uHCC) in 2018. This study assessed real-world treatment patterns and outcomes of lenvatinib beyond first-line systemic treatment in the United States. METHODS: A retrospective study was conducted among US adults (≥18 years) with uHCC. Eligible patients initiated lenvatinib monotherapy as second- or later-line systemic therapy (2L-plus) from August 2018 to September 2019. Clinical outcomes included physician-reported best response, progression-free survival (PFS), and overall survival (OS). RESULTS: Of 164 patients who received lenvatinib in 2L-plus, most (n = 133; 81.1%) received lenvatinib in 2 L. There were 109 patients (66.4%) who initiated lenvatinib after immunotherapy. At lenvatinib initiation, only 31.1% of patients had Child-Pugh class A, while half (49.4%) had Child-Pugh class B. Most patients had Barcelona Clinic Liver Cancer stage B (23.8%) or C (38.4%) uHCC. Median duration of lenvatinib treatment was 6.9 months, with 42.7% of patients still on treatment at the end of follow-up. Physician-reported best response was complete and partial response for 8.5% and 44.5% of patients, respectively. PFS and OS rate estimates from lenvatinib initiation at 12 months were 51.7% and 57.8%, respectively. Among patients treated after immunotherapy, complete and partial responses were 10.1% and 43.1%, respectively, and PFS and OS estimates from lenvatinib initiation at 12 months were 52.8% and 60.0%, respectively. CONCLUSION: This retrospective study suggests clinical effectiveness of lenvatinib monotherapy in a real-world setting among previously treated patients with uHCC, including among those previously treated with immunotherapy.

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.

Introduction: 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. Materials and Methods: 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. Results: 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. Conclusion: These real-world data provide evidence for the clinical effectiveness outcomes of eribulin treatment among MBC patients previously treated with an IO or ADC.

Improved Survival Outcomes in Patients With MET-Dysregulated Advanced NSCLC Treated With MET Inhibitors: Results of a Multinational Retrospective Chart Review.

BACKGROUND: We evaluated the disease and patient characteristics, treatment, and MET testing patterns, predictive biomarkers and survival outcomes in patients with MET-dysregulated metastatic non-small-cell lung cancer (NSCLC) in a real-world setting. PATIENTS AND METHODS: This was a multinational, retrospective, noninterventional chart review study. Data from medical records of patients with advanced/metastatic EGFR wild-type, MET-dysregulated NSCLC (December 2017-September 2018) were abstracted into electronic data collection forms. RESULTS: Overall, 211 patient charts were included in this analysis; 157 patients had MET exon 14 skipping mutations (METex14; with or without concomitant MET amplification) and 54 had MET amplification only. All patients were tested for METex14, whereas MET amplification was evaluated in 168 patients. No overlap was reported between MET dysregulation and ALK, ROS1 or RET rearrangements, or HER2 exon 20 insertions. Overall, 56 of 211 patients (26.5%) received MET inhibitor (METi) therapy in any treatment-line setting (31.2% in the METex14 cohort; 13% in the MET-amplified only cohort). In the METex14 cohort, median OS in patients receiving METi was 25.4 months versus 10.7 months in patients who did not (HR [95% CI]: 0.532 [0.340-0.832]; P = .0055). In the MET-amplified only cohort, median OS was 20.6 months in patients treated with METi compared with 7.6 months in those without METi (HR [95% CI]: 0.388 [0.152-0.991]; P = .0479). CONCLUSIONS: MET alterations in NSCLC typically occur in the absence of other oncogenic driver mutations and are associated with poor survival outcomes. Notably, METi therapies are associated with improved survival outcomes in patients with MET-dysregulated NSCLC.

Association between oral 5-ASA adherence and health care utilization and costs among patients with active ulcerative colitis.

BACKGROUND: Observational cohort study to assess the association between adherence to oral 5-aminosalicylates (5-ASAs) and all-cause costs and health care utilization among patients with active ulcerative colitis (UC) in the United States. METHODS: Retrospective analysis of insurance claims from June 1997 to August 2006 in the LifeLink Database. Patient criteria: aged 18 or older with one or more claim(s) between June 1997 and August 2005 for a UC diagnosis and at least one oral 5-ASA prescription on or after the first observed UC diagnosis; continuous enrollment for at least 6 months prior to and 12 months following 5-ASA initiation (index date). As a proxy for active disease, patients needed to have at least two UC-specific non-pharmacy claims, at least 30 days of 5-ASA treatment and at least one corticosteroid prescription within the 12-month post-index period. Cumulative exposure to oral 5-ASAs over the 12-month period was calculated using the medication possession ratio (MPR). Patients with an MPR of at least 0.80 were classified as adherent. All-cause medical and pharmacy resource utilization and costs were computed over the 12-month post-index period and compared between adherent and nonadherent patients. RESULTS: 1,693 UC patients met study inclusion criteria: 72% were nonadherent to 5-ASA treatment (n=1,217) and 28% were adherent (n=476) in the 12-month study period. Compared with nonadherent patients, adherent patients had 31% fewer hospitalizations (P=0.0025) and 34% fewer emergency department admissions (P=0.0016). Adherent patients had 25% more pharmacy prescriptions overall (P <0.0001) and 71% more UC-related pharmacy prescriptions (P <0.0001) than did nonadherent patients. Total all-cause health care utilization was 1.13 times higher for adherent patients than for nonadherent patients (P=0.0002). After adjusting for covariates, total all-cause costs were 29% higher for nonadherent patients than for adherent patients (mean [95% confidence interval]: $13,465 [$13,094, $13,835] vs $17,339 [$17,033, $17,645]). CONCLUSIONS: Approximately three-quarters of patients with active UC were not adherent with their prescribed doses of oral 5-ASA. Nonadherence was associated with higher total all-cause costs. The key driver of decreased costs among adherent patients was inpatient hospitalizations, which more than offset these patients' expected higher pharmacy costs.

A Retrospective Cohort Study of Treatment Outcomes of Adult Patients With Relapsed or Refractory Follicular Lymphoma (ReCORD-FL).

This study (ReCORD-FL) sought to construct a historical control cohort to augment single-arm trials in relapsed/refractory follicular lymphoma (r/r FL). A retrospective study in 10 centers across North America and Europe was conducted. Adults with grade 1-3A FL were required to be r/r after ≥2 therapy lines including an anti-CD20 and an alkylator. After first becoming r/r, patients were required to initiate ≥1 additional therapy line, which defined the study index date. Endpoints were observed from start of each therapy line (including index line) until death, last follow-up, or December 31, 2020. Endpoints were complete response (CR) rate, overall response rate (ORR), time to next treatment or death (TNT-D), event-free survival (EFS), and overall survival (OS). One hundred eighty-seven patients were identified. Most patients' (80.2%) index therapy occurred in third line (3L) (range, 3L-6L). Median follow-up from FL diagnosis was 9 years (range, 1-21 years). CR and ORR to the index therapy were 39.0% and 70.6%, respectively. Median (95% confidence interval) EFS from index was 14.6 (11.0-18.0) months; median OS from index was 10.6 years. Outcomes worsened across successive treatment lines and for patients who were double refractory (r/r to both an anti-CD20 monoclonal antibody and an alkylator) or POD24 (progressed ≤24 months after front-line anti-CD20) at index. Findings demonstrate the unmet need of FL patients with multiply relapsed, double refractory, or POD24 disease. Based on robustness of the historical data collected and comparability with a previous study (SCHOLAR-5), ReCORD-FL presents a valuable source of control data for comparative studies in r/r FL.

Efficacy comparison of tisagenlecleucel vs usual care in patients with relapsed or refractory follicular lymphoma.

The ELARA trial indicates tisagenlecleucel (tisa-cel) is an effective anti-CD19 chimeric antigen receptor T-cell therapy for relapsed or refractory follicular lymphoma (r/r FL). As ELARA is a single-arm trial, this study compares tisa-cel outcomes from the ELARA trial with usual care from a real-world cohort. ELARA enrolled 98 patients as of 29 March 2021 (median follow-up: 15 months from enrollment). Usual care data were obtained from ReCORD-FL, a global retrospective study of patients with r/r FL, who met similar eligibility criteria to ELARA. With a data cutoff date of 31 December 2020, 187 patients with ≥2 preceding treatment lines were included in the ReCORD-FL (median follow-up: 57 months from third-line) study. An indirect treatment comparison was performed for 97 patients from the ELARA trial and 143 patients from the ReCORD-FL study with no missing data on baseline factors. The line of therapy for which outcomes were assessed was selected or matched between cohorts using propensity score modeling. After baseline factor adjustment via weighting by odds, complete response rate (CRR; 95% confidence interval) was 69.1% (59.8%-78.3%) for tisa-cel vs. 37.3% (26.4%-48.3%) for usual care; overall response rate was 85.6% (78.7%-92.5%) vs. 63.6% (52.5%-74.7%). Kaplan-Meier probability of being progression/event-free at 12 months was 70.5% (61.4%-79.7%) for tisa-cel vs. 51.9% (40.6%-63.3%) for usual care, with hazard ratio (HR)=0.60 (0.34-0.86); 12-month overall survival was 96.6% (92.9%-100%) vs. 71.7% (61.2%-82.2%), with HR=0.2 (0.02-0.38). In conclusion, tisa-cel was associated with a 1.9-fold higher complete response rate and a 1.4-fold higher rate of being progression or event free at 12 months vs usual care, as well as a death risk reduction of 80%. The findings provide additional evidence on the benefit of tisa-cel in patients with r/r FL after ≥2 treatment lines. This trial was registered at www.clinicaltrials.gov as NCT03568461.

Direct economic burden of chronic hepatitis C virus in a United States managed care population.

GOALS AND BACKGROUND: To estimate all-cause and disease-related resource utilization and costs among managed care enrollees with chronic hepatitis C virus (HCV). STUDY: A large United States claims database was analyzed (1/1/2002 to 12/31/2006). Inclusion criteria were: diagnosis of chronic HCV; no hepatitis B diagnoses; ≥6 and ≥12 months of continuous plan enrollment prediagnosis and postdiagnosis, respectively. Use and costs of medical services and prescription drugs over a 12-month period postdiagnosis were evaluated. Outcomes were assessed in controls without HCV matched (1:1) on age, sex, and plan enrollment. All cost estimates were generated using multivariate generalized linear models to adjust for additional covariates and skewness common in health care cost data. RESULTS: Of the 20,662 patients who met all inclusion criteria, mean age was 49 years; 61% were male. Adjusted all-cause costs were $20,961 per HCV patient, compared with $5451 per control (P<0.0001). Hospitalization occurred in 24% of HCV patients compared with 7% of controls (P<0.0001). Mean inpatient costs were $5892 and $1159 per patient, respectively (P<0.0001). Patients with HCV had higher prescription costs compared with controls ($6191 vs. $1315; P<0.0001). At $6864 per patient, disease-related costs were nearly one-third of all costs in patients with HCV, which exceeded all-cause costs among controls by 26% (P<0.0001). CONCLUSIONS: Chronic HCV is a costly disease to managed care organizations. Disease-related costs in HCV exceed all-cause costs in demographically matched controls. Increased efforts in HCV screening and early treatment, particularly before progression to liver cirrhosis, may lead to long-term cost savings in HCV management for managed care systems.

Cost impact of complications in meningococcal disease: evidence from a United States managed care population.

OBJECTIVES: To compare health care utilization and associated costs among patients with and without invasive meningococcal disease (IMD)-related sequelae. METHODS: A retrospective analysis of an administrative claims database from 1998-2009 was performed. Patients with an IMD-related inpatient admission and continuous health plan enrollment selected and categorized by presence (complicated-IMD) or absence (uncomplicated-IMD) IMD-related sequelae during the follow-up year. Differences in the follow-up year healthcare utilization and costs between the two groups tested using univariate and multivariable analyses. RESULTS: We identified 173 patients; 41% had at least one diagnosis claim for IMD-related sequelae. Significantly higher predicted total health care costs for complicated-IMD cases (mean: $72,101), compared with uncomplicated cases (mean: $41,883; P < 0.001). CONCLUSIONS: We observed significantly higher health care costs among complicated-IMD cases, compared with uncomplicated cases. The substantially higher costs observed among patients with IMD-related sequelae warrant inclusion of these costs in studies conducting economic evaluations of meningococcal vaccination programs.

Cost of acute hospitalization and post-discharge follow-up care for meningococcal disease in the US.

The combined costs of acute hospitalization and post-discharge follow-up care in patients with meningococcal disease have not been widely documented. In this study, data were retrospectively analyzed from three large databases of hospital discharge records and commercial insurance claims in the US. Cases of meningococcal disease were defined as admissions with an ICD-9-CM diagnosis code in the range of 036.x. From the 2005 HCUP Nationwide Inpatient Sample, 349 (weighted N=1,710) meningococcal-related hospitalizations were identified with a mean facility cost (in 2009 dollars) of $19,526 per admission. Similar estimates ($18,119 and $20,066, respectively) were obtained from 268 admissions identified in the LifeLink (formerly PharMetrics) database during 1999-2007 and from 1,058 hospitalizations in the Perspective Comparative Database (PCD) during 2000-2007. Using insurance claims from LifeLink, we estimated that payers incur an additional $26,178 in non-facility (professional and other ancillary) costs during the course of a meningococcal admission, as well as $22,230 in additional medical and pharmacy expenses for post-discharge care during the ensuing year. The majority of follow-up costs ($14,637) were attributed to repeat hospitalizations. Mean length of stay for meningococcal disease was consistently estimated across databases at 8 to 9 days. Data from the PCD further suggested that meningococcal disease carries, on average, nearly 2 days of intensive care unit utilization. In conclusion, hospital admissions for meningococcal disease are costly to payers. These costs are heightened when non-facility services and post-discharge care are also considered. Awareness of the full cost burden of meningococcal disease is needed when evaluating vaccination programs targeting the disease.