Real-World Maintenance Phase Persistence on Ustekinumab and Adalimumab in Ulcerative Colitis.

Purpose: To describe real-world persistence in bio-naïve and bio-experienced adults with ulcerative colitis (UC) treated with ustekinumab, a recently approved anti-interleukin 12/23 antibody, or adalimumab, an anti-TNF biologic. Methods: This is a descriptive, retrospective cohort study. Patients initiating ustekinumab or adalimumab (index date, between 10/21/2019 and 08/13/2021) were selected from the Komodo Health comprehensive dataset and stratified into bio-naïve and bio-experienced subgroups based on biologic use 12 months pre-index date. Endpoints evaluated at 12-months after maintenance phase start using Kaplan-Meier analysis included 1) persistence; 2) persistence while being corticosteroid-free (<14 consecutive days of corticosteroid supply after day 90 post-index); and, 3) persistence while on monotherapy (no immunomodulators/non-index biologics/advanced therapies). Results: Ustekinumab cohort included 778 patients (236 bio-naïve, 542 bio-experienced) and adalimumab cohort included 1693 patients (1517 bio-naive, 176 bio-experienced). At 12 months after maintenance phase start, 75.5% and 50.5% of bio-naïve patients persisted on ustekinumab and adalimumab and 72.3% and 56.9% of bio-experienced patients persisted on ustekinumab and adalimumab, respectively. Further, 55.1% and 38.2% of bio-naïve patients were persistent and corticosteroid-free with ustekinumab and adalimumab; 43.7% and 33.4% of bio-experienced patients were persistent and corticosteroid-free with ustekinumab and adalimumab, respectively. Moreover, 68.1% and 44.5% of bio-naïve patients were persistent and on monotherapy with ustekinumab and adalimumab; 61.6% and 47.9% of bio-experienced patients were persistent and on monotherapy with ustekinumab and adalimumab, respectively. Conclusion: At 12 months after maintenance phase start, patients with UC treated with ustekinumab had numerically higher persistence, including persistence while corticosteroid-free and persistence while on monotherapy, than patients treated with adalimumab.

Burden of chronic corticosteroid use among patients with ulcerative colitis initiated on targeted treatment or conventional therapy in the United States.

BACKGROUND: Chronic corticosteroid use is common in ulcerative colitis (UC); however, real-world evidence of its burden to the health care system is limited. OBJECTIVE: To quantify chronic corticosteroid use burden in UC. METHODS: Adults with UC initiated on targeted treatments (ie, biologics and advanced/small molecule therapies) or conventional therapy (index date) were selected from a deidentified US insurance claims database (January 1, 2004, to September 30, 2021). Targeted treatments and conventional therapy initiators were stratified into chronic (>90 days corticosteroid use 12 months post-index [landmark]) and nonchronic corticosteroid users. Patient characteristics 12 months pre-index were balanced with inverse probability of treatment weighting. Health care resource use, costs (US$ 2021), and corticosteroid-related complications were compared in the 12 months post-landmark. RESULTS: Targeted treatment initiators included 1,886 chronic and 1,911 nonchronic corticosteroid users; conventional therapy initiators included 4,980 chronic and 5,199 nonchronic users. Chronic vs nonchronic users had 94% more inpatient days and 16% more outpatient visits among targeted treatment initiators, and 135% more inpatient days and 30% more outpatient visits among conventional therapy initiators (all P < 0.01). Mean all-cause total costs per patient per year were $73,491 for chronic vs $58,884 for nonchronic users ($14,607 higher; P < 0.01) for targeted treatment initiators, and $39,335 for chronic vs $21,271 for nonchronic users ($18,065 higher; P < 0.01) for conventional therapy initiators. Odds of infection and bone loss were 14% and 113% higher, respectively, in chronic vs nonchronic users among targeted treatment initiators and 29% and 47% higher in chronic vs nonchronic users among conventional therapy initiators (all P < .01). CONCLUSIONS: The results of this study suggest that chronic corticosteroid use is associated with substantial clinical and economic burden and may indicate unmet needs in the management of UC progression.

Economic and clinical burden of chronic corticosteroid use in patients with Crohn[apos]s disease initiated on biologic or conventional therapies in the US: A retrospective claims study.

BACKGROUND: Chronic corticosteroid (CS) use is associated with complications, but estimates of the economic and clinical burden in patients with Crohn's disease (CD) are lacking. OBJECTIVE: To estimate the burden of chronic CS use in CD in the United States in terms of health care resource utilization (HRU), health care costs, and CS-related complications. METHODS: This was a retrospective study of adults with CD initiated on biologics or conventional therapies (index date). Patients from a deidentified insurance claims database (2004-2021) were classified as chronic CS users (>90 days of CS use) or nonchronic CS users based on a 12-month landmark period starting on the index date. Patient baseline characteristics were balanced, and outcomes (HRU, costs [2021 US dollars], and CS-related complications) 12 months after the landmark period were compared between CS groups using regressions with nonparametric bootstrap resampling to estimate confidence intervals and P values. RESULTS: Biologic initiators (mean age: 44 years, 55% female) included 3366 chronic and 3401 nonchronic CS users; conventional therapy initiators (mean age: 51 years, 59% female) included 3657 chronic and 3727 nonchronic CS users. Compared with nonchronic users, chronic users had significantly more inpatient days and outpatient visits (biologic initiators: 37% and 24% more, respectively; conventional therapy initiators: 36% and 17%, respectively; all P<0.05). Chronic users also had significantly higher mean all-cause total costs per-patient-per year (biologic: $72,967 vs. $63,100, mean cost difference [MCD] = $9867; conventional therapy: $40,144 vs. $26,426, MCD = $13,718; all P<0.001), as well as higher odds of infection (biologic: 14% higher; conventional therapy: 20% higher) and bone loss (63% and 41%, respectively) (all P<0.05). CONCLUSION: Chronic CS use in patients with CD is associated with a significant economic and clinical burden including higher HRU, health care costs, and prevalence of complications, suggesting unmet needs in the clinical management of this population.

Evaluation of Treatment Patterns and Maintenance Dose Titration Among Patients With Crohn's Disease Initiating Biologics With 3 Years of Follow-Up.

Background: There is limited real-world evidence on treatment patterns of patients with Crohn's disease (CD) initiating biologics with an extensive follow-up period. This study describes persistence and dose titration among CD patients with 3 years of follow-up. Methods: This retrospective observational study was conducted using the STATinMED RWD Insights all-payer medical and pharmacy data. Adult patients with at least 1 CD medical claim and at least 1 medical/pharmacy claim for a biologic (adalimumab [ADA], certolizumab pegol (CZP), infliximab [IFX] and its biosimilar products [IFX-BS], ustekinumab [UST], and vedolizumab [VDZ]) between September 2016 and October 2018 were identified. Commercially insured patients with continuous capture for at least 12 months before and at least 36 months after biologics initiation were selected. Confirmed CD patients were included in the final cohort. Baseline patient characteristics and treatment patterns over the 3-year follow-up period were evaluated. Results were summarized using means and SD or counts and percentages. Results: A total of 2309 confirmed patients with CD were identified (847 [36.7%] IFX, 534 [23.1%] ADA, 486 [21.1%] VDZ, 394 [17.1%] UST, 85 [3.7%] CZP, and 72 [3.1%] IFX-BS). CZP and IFX-BS were excluded due to small sample sizes. Approximately half of CD patients were between ages 35 and 54. Patients on UST had a higher Charlson Comorbidity Index score. Common comorbidities (>10%) included anemia, anxiety, depression, and hypertension. Persistence over 3 years' follow-up was highest for UST (61.4%) patients, followed by VDZ (58.0% ), ADA (52.1% , and IFX (48.1%). The discontinuation rate without switch or restart was highest for ADA (37.3%), followed by UST (30.7%), IFX (28.1%), and VDZ (25.3%). Over the 3 years of follow-up, the dose titration rate was highest for IFX (76.5%) and lowest for UST (50.8%). In particular, UST had the lowest dose escalation rate (35.5%) and highest dose-reduction rate (16.5%). Conclusions: Patients with CD on UST had the highest persistence and lowest dose escalation across different biologic users over the 3-year follow-up period, possibly suggesting a better clinical response of UST. Future studies with longer follow-up adjusting for confounders are needed to better understand treatment patterns among biologics users.

Persistence and Dose Escalation During Maintenance Phase and Use of Nonbiologic Medications Among Patients With Ulcerative Colitis Initiated on Ustekinumab in the United States.

Background: Real-world data on treatment patterns among patients with ulcerative colitis (UC) initiated on ustekinumab are limited. Methods: Adults with UC initiated on ustekinumab (index date) between 10/18/2019 and 04/31/2022 were selected from a deidentified health insurance claims database (Symphony Health, an ICON plc Company, PatientSource). Persistence (no gaps in days of supply >120 days), persistence while being corticosteroid-free (no corticosteroid use for ≥14 days of supply after a 90-day grace period from index date) and dose escalation (≥2 consecutive subcutaneous claims ≥100% above daily maintenance dose) were described during the maintenance phase using Kaplan-Meier analysis. Nonbiologic treatments, among patients with ≥2 ustekinumab claims within 90 days post-index and ≥6 months of follow-up, were compared with logistic models 6 months post- versus pre-ustekinumab initiation. Results: 6565 patients on ustekinumab entered the maintenance phase. At month 12 of the maintenance phase, 72.0% (95% confidence interval [CI]: 70.1%-73.9%) were persistent, 50.8% (95% CI: 48.7%-52.9%) were persistent and corticosteroid-free, and 19.2% (95% CI: 17.3%-21.3%) of patients had dose escalation. In the 6 months post- versus pre-ustekinumab initiation, the odds of nonbiologic medication use assessed in 4147 patients were significantly lower: 57% lower odds for corticosteroid, 46% for 60 cumulative days of corticosteroid, 42% for 5-aminosalicylic acid, and 24% for immunomodulators (all P < .001). Conclusions: Most patients with UC reaching the maintenance phase on ustekinumab remained persistent after 12 months of maintenance therapy. Nonbiologic medication use post-ustekinumab initiation was significantly lower, notably for corticosteroids. Given the multiple complications associated with chronic corticosteroid use, this reduction can be seen as clinically relevant and informs treatment choice for patients with UC.

Long-term persistence and other treatment patterns among bio-naïve patients with Crohn's disease treated with ustekinumab or adalimumab.

OBJECTIVE: To estimate long-term persistence among bio-naïve patients with CD initiated on ustekinumab or adalimumab. METHODS: Adults with CD initiating ustekinumab or adalimumab (index date, between September 23, 2016 and August 1, 2019) were sampled from the IBM MarketScan Commercial Database. Patients without CD-indicated biologics (bio-naïve) and with no diagnoses for other autoimmune diseases 12 months pre-index date (baseline) were included. Cohorts were balanced on baseline characteristics with inverse probability of treatment weighting. Persistence was defined as the absence of therapy exposure gaps >120 days (ustekinumab) or >60 (adalimumab) between days of supply. Composite endpoints were persistence and being corticosteroid-free (no corticosteroids >14 days of supply after day 90 post-index) and persistence while on monotherapy (no immunomodulators/non-index biologics). Persistence was analyzed using Kaplan-Meier and Cox's models. RESULTS: Ustekinumab and adalimumab cohorts included 671 and 2,975 patients. At 12 months post-index, ustekinumab patients were significantly more persistent (hazard ratio [HR] = 1.60; 95% confidence interval [CI] = 1.33-1.93), persistent while on monotherapy (HR = 1.43; 95% CI = 1.24-1.65), and trended toward being more persistent and corticosteroid-free (HR = 1.14; 95% CI = 0.99-1.30) vs adalimumab. At 24 months post-index, ustekinumab patients were significantly more persistent (HR = 1.66; 95% CI = 1.40-1.97), persistent while on monotherapy (HR = 1.44; 95% CI = 1.26-1.64), and persistent and corticosteroid-free (HR = 1.15; 95% CI = 1.01-1.31) vs adalimumab. CONCLUSIONS: Bio-naïve patients with CD initiated on ustekinumab demonstrated significantly more persistence than patients initiated on adalimumab at 12 and 24 months of treatment. Long-term persistence is a measure of a drug's real-world performance and findings may aid clinical decision-making.

Choosing a treatment on which a patient can stay over a long period of time is key for the successful management of chronic conditions such as Crohn's disease. Information on whether and how long patients stay on treatment can help physicians make the right therapeutic choice. This study examined whether adults with Crohn's disease, who have not previously taken biologics, stay on treatment longer when given the biologic ustekinumab or adalimumab. At 12 and 24 months after starting the treatment, a larger proportion of patients were still using ustekinumab compared with adalimumab. The proportion of patients using the biologic without immunomodulators or other biologics was also higher with ustekinumab. The results suggest that patients without previous biologic experience stay on treatment longer with ustekinumab than with adalimumab.

Persistence Among Patients with Crohn Disease Previously Treated with an Anti-tumor Necrosis Factor Inhibitor and Switching or Cycling to Another Biologic Agent.

PURPOSE: Nonresponse to an anti-tumor necrosis factor (TNF) agent in patients with Crohn disease (CD) is often managed by either a switch to a different class of biologic (ie, ustekinumab, vedolizumab) or by cycling to another anti-TNF agent (ie, adalimumab, infliximab, certolizumab pegol). Persistence after a switch to a different biologic class or after cycling within the anti-TNF class was assessed in patients with nonresponse to an anti-TNF agent. METHODS: Adults with CD who discontinued from an anti-TNF agent and either switched to a different class of biologic (ie, anti-interleukin/integrin; the switching cohort) or cycled within the anti-TNF class (the cycling cohort) between September 23, 2016, and August 1, 2019, were selected from a commercial database. The index date was defined as the date of the first claim of the subsequent-line biologic (index biologic) after an anti-TNF. The switching and cycling cohorts were balanced with regard to baseline characteristics, using inverse probability of treatment weights-average treatment effect (IPTW-ATE). Persistence with the index biologic was defined as consistent use with no gaps of >120 days (ustekinumab, vedolizumab, infliximab) or of >60 days (adalimumab, certolizumab pegol) in biologic supply. Composite end points were persistence while being corticosteroid-free (defined as no use of corticosteroids with ≥14 days of supply after day 90 post-index) and persistence while on monotherapy (no immunomodulators/nonindex biologics). Weighted Kaplan-Meier and Cox models were used to assess outcomes at 12 months post-index. FINDINGS: There were 444 patients in the weighted switching cohort (mean age, 40.4 years; 56.3% female) and 441 in the weighted cycling cohort (mean age, 39.5 years; 58.4% female). At 12 months post-index, the rate of persistence with the index biologic was 75.7% in the switching cohort compared to 67.5% in the cycling cohort (log-rank P = 0.023); the rate of persistence while on monotherapy was 58.2% compared to 44.2%, respectively (log-rank P < 0.001). The rate of persistence was 44% greater in the switching compared to that in the cycling cohort (hazard ratio [HR] = 1.44; 95% CI, 1.11-1.88; P = 0.007); the rate of persistence while on monotherapy was 56% greater in the switching cohort (HR = 1.56; 95% CI, 1.28-1.90; P < 0.001). The between-cohort difference in persistence while being corticosteroid-free was not statistically significant (HR = 1.08; 95% CI, 0.89-1.32; P = 0.426). IMPLICATIONS: Patients with CD who switched to a different biologic class were more persistent than were patients who cycled to another anti-TNF agent. These findings may be useful for physicians when considering the treatment of patients who have experienced nonresponse or loss of response to the first-line anti-TNF agent.

Persistence and other treatment patterns among bio-experienced patients with Crohn's disease initiated on ustekinumab or adalimumab.

BACKGROUND: Real-world data on persistence on ustekinumab and adalimumab among bio-experienced patients with Crohn's disease (CD) are limited. OBJECTIVE: To compare treatment persistence and describe switching, restart, and dose titration among bio-experienced patients with CD initiated on ustekinumab or adalimumab. METHODS: IBM MarketScan Commercial Database was used to identify bio-experienced adults with CD who were assigned to either the ustekinumab or adalimumab cohort based on the agent first initiated (index date) after September 23, 2016. Cohorts were balanced using inverse probability of treatment weights-average treatment effect on treated. Persistence on index agent (absence of exposure gap > 120 days for ustekinumab or > 60 days for adalimumab), persistence while corticosteroid-free, and persistence while receiving monotherapy were assessed at 12 months after index date and compared between cohorts using weighted Kaplan-Meier and Cox proportional hazards model analyses. RESULTS: Among 903 patients in the ustekinumab cohort and 525 patients in the adalimumab cohort, baseline characteristics were balanced after weighting. At 12 months post-index, ustekinumab was associated with higher persistence (80.1% vs 64.6%; hazard ratio = 2.02 [95% CI = 1.60-2.56]; P < 0.001) and persistence while receiving monotherapy (51.6% vs 40.0%; 1.51 [1.28-1.78]; P < 0.001) vs adalimumab. Persistence while corticosteroid-free was similar in the ustekinumab vs adalimumab cohort (50.1% vs 48.2%; 1.19 [1.00-1.41]; P = 0.0516). CONCLUSIONS: This retrospective real-world study demonstrated that among bio-experienced patients with CD, initiation of ustekinumab was associated with better persistence at 12 months of follow-up, including persistence while receiving monotherapy, compared with adalimumab. DISCLOSURES: This study was funded by Janssen Scientific Affairs, LLC. Drs Zhao, Ding, and Kachroo are employees of Janssen Scientific Affairs, LLC, and stockholders of Johnson & Johnson. Dr Manceur, Mr Lefebvre, Ms Zhdanava, and Mr Pilon are employees of Analysis Group, Inc., a consulting company that has provided paid consulting services to Janssen Scientific Affairs, LLC, which funded the development and conduct of this study and article. Mr Holiday was an employee of Analysis Group, Inc., at the time of study conduct.

Clinical and utilization outcomes associated with tumor mutational burden in a real-world pan-tumor population.

Aim & methods: This real-world study examined the association of tumor mutational burden (TMB) with clinical and healthcare utilization in adults diagnosed with advanced solid tumor 1 January 2015- 31 January 2019. Results: There were 170 patients in low-TMB group (TMB<10 mut/Mb) and 32 in high-TMB group (TMB ≥10 mut/Mb). Median overall survival was 18.8 (95% CI: 17.3-28.8) and 15.9 months (95% CI: 11.3-18.0) whereas median progression-free survival was 9.9 (95% CI: 8.6-11.4) and 7.8 months (95% CI: 3.8-12.5) for the low- and high-TMB groups, respectively. Hospitalization (49.4 vs 37.5%), emergency visit (25.3 vs 21.9%), and median overall cost of care (US$135,403 vs 87,570) were all lower in low-TMB group. Conclusion: Despite the limited sample, these data provide a historical perspective for examining real-world outcomes associated with TMB.

Lay abstract Tumor mutational burden (TMB) is the total number of mutations found in the DNA of cancer cells. Knowing the TMB may help plan the best treatment. The goal of this study was to examine whether higher TMB is directly associated with clinical outcomes or healthcare use and costs in patients who have not received immuno-oncology treatment. This study included 202 adult patients who were diagnosed with advanced solid tumors between January 2015 and January 2019. Patients were divided into two groups based on their TMB level. The study results indicate some relationship between TMB level and real-world outcomes. Future studies with a larger sample size are needed to confirm these results.

Real-world patterns on tumor mutation burden testing in a pan-tumor population.

Background: There is limited real-world information on use of tumor mutational burden (TMB) testing and characteristics of patients receiving it. Materials & methods: Patients ≥18 years old and diagnosed with advanced solid tumors between 1 January 2015 and 31 January 2019 with TMB testing (TMB cohort) and without it (non-TMB) were included in this retrospective, observational study. Results: The TMB cohort (n = 202) was younger than non-TMB (n = 212) (mean age: 62.1 vs 65.6 at diagnosis; p = 0.005). There were more Black patients in the TMB cohort (21.3 vs 11.8% in non-TMB; p = 0.004). Clinical characteristics were comparable between the two cohorts; however, systemic anticancer treatment was higher among TMB cohort (91.6 vs 77.8% in non-TMB). Conclusion: Notable differences were observed between patients receiving TMB test and those not receiving it.

Clinical effectiveness outcomes and healthcare resource utilization of patients diagnosed with small-cell lung cancer.

Background: Real-world data are lacking on patients with small-cell lung cancer (SCLC) with extensive-stage SCLC (eSCLC) and poor performance status (PS). Patients & methods: Eligible patients diagnosed with eSCLC between 1 January 2008 and 31 December 2017 were included in this retrospective, observational study. Results: The study included 406 patients, with 14.3% impaired PS. Progression-free survival and overall survival were not significantly different between impaired (Eastern Cooperative Oncology Group ≥2) and not impaired patients (median, 4.5 vs 5.3 months, and 7.2 vs 8.4 months, respectively). Impaired patients used more supportive care drugs (mean, 3.0 vs 2.0; p = 0.033). Conclusion: Effectiveness outcomes among patients with and without impaired PS did not differ in the real-world setting. Progression-free survival and overall survival were similar to data from clinical trials.

Association of clinico-genomic characteristics with tumor mutational burden in small cell lung cancer patients.

Aim: We evaluated the relationship between clinical and genomic characteristics and tumor mutational burden (TMB) in small cell lung cancer. Materials & methods: In a retrospective analysis of small cell lung cancer patients aged ≥18, we assessed treatment patterns and survival in relation to TMB; the association of clinical and genomic characteristics with TMB was determined by multivariate regression. High TMB (TMB-H) was defined as ≥10 mutations/megabase. Results: Among 186 patients, treatment patterns and overall survival were similar for TMB-H and non-TMB-H patients. TMB was determined for 179 patients, 41.9% of whom were TMB-H. Short variants of LRP1B, FAT3, MLL3, MED12 and NOTCH3 were significantly associated with TMB-H (p ≤ 0.01). Conclusion: Neither treatment patterns nor survival differed by TMB status.

Healthcare-Related Costs Associated with Switching Subcutaneous Tumor Necrosis Factor-α Inhibitor in the Treatment of Inflammatory Arthritis: a Retrospective Study.

INTRODUCTION: Subsequent lines of subcutaneous tumor necrosis factor alpha inhibitor (SC-TNFi) treatment may be well motivated in the management of rheumatoid arthritis (RA), ankylosing spondylitis (AS), and psoriatic arthritis (PsA)-collectively named inflammatory arthritis (IA). However, the costs associated with switching SC-TNFis are largely unknown. The objective of this retrospective observational study was to explore costs of healthcare resource utilization (HCRU) associated with switching SC-TNFi treatment among biologic-naïve Swedish patients with IA. METHODS: Using population-based register data, adult patients filling prescriptions between May 6, 2010 and December 31, 2014 for an SC-TNFi (adalimumab, etanercept, certolizumab, and golimumab) were included. Patients switching treatment (cyclers) were matched to treatment persistent patients on the basis of propensity score and follow-up time. HCRU-associated costs were captured and compared 12 months before and 12 months after the index date (defined as the date of the switch). RESULTS: A balanced cohort of 594 matched pairs was derived. Prior to the index date, cyclers had significantly higher non-treatment HCRU costs compared to persistent patients ($3815 [3498-4147] vs. $2900; 95%CI [2565-3256]). However, 12 months after the index date, cyclers had significantly increased their non-treatment HCRU costs while persistent patients lowered theirs ($822 [232-1490] vs. $- 313 [- 664-36]). This resulted in a statistically significant difference in difference of $1135 between the groups. CONCLUSIONS: In biologic-naïve patients treated with SC-TNFi for IA, cyclers significantly increased their non-treatment HCRU costs 12 months after switching treatment while persistent patients lowered their costs during the same time period. As these findings indicate that differences in treatment persistence may have an impact on costs, further research utilizing more comprehensive data sources in alternate settings is warranted.

The value of persistence in treatment with subcutaneous TNF-alpha inhibitors for ankylosing spondylitis.

OBJECTIVE: To estimate the impact of persistence on cost-effectiveness of subcutaneous tumor necrosis factor-α inhibitors (SC-TNFis) from healthcare and societal perspectives in a United Kingdom ankylosing spondylitis (AS) population using a recently published Markov cohort model. METHODS: A recently published cost-effectiveness model developed for a National Institute for health and Care Excellence appraisal was extended to fit the current study; in brief, it is a Markov cohort model where treatment responders continue from the trial period with maintenance SC-TNFi treatment, while non-responders transition to conventional care. Costs and effects were modeled for a hypothetical SC-TNFi with average efficacy and price. Model outcomes included quality-adjusted life-years (QALYs), total direct and indirect lifetime costs, and incremental cost-effectiveness ratios (ICERs). The cost-effectiveness of SC-TNFi persistence was estimated by decreasing the annual discontinuation probability in five percentage point increments from 25 to 5% per annum. RESULTS: From a health care perspective, the ICERs for the modeled discontinuation rates compared to the baseline annual discontinuation rate (25%) ranged between GBP 17,277 and GBP 18,161. From a societal perspective, increased discontinuation rates resulted in decreased total costs and higher QALYs; hence, lower discontinuation rates dominated higher discontinuation rates from a societal perspective. CONCLUSION: In conclusion, this study shows that, all else equal, higher SC-TNFi treatment persistence in AS is cost effective from a health care perspective and dominant from a societal perspective. Hence, all else equal, prescribing the SC-TNFi with the highest persistence may be considered a cost-effective strategy.

Survival, Chemotherapy Treatments, and Health Care Utilization Among Patients with Advanced Small Cell Lung Cancer: An Observational Study.

INTRODUCTION: Most cases of small cell lung cancer (SCLC) are diagnosed at an advanced stage. The objective of this study was to investigate patient characteristics, survival, chemotherapy treatments, and health care use after a diagnosis of advanced SCLC in subjects enrolled in a health system network. METHODS: This was a retrospective cohort study of patients aged ≥ 18 years who either were diagnosed with stage III/IV SCLC or who progressed to advanced SCLC during the study period (2005-2015). Patients identified from the Indiana State Cancer Registry and the Indiana Network for Patient Care were followed from their advanced diagnosis index date until the earliest date of the last visit, death, or the end of the study period. Patient characteristics, survival, chemotherapy regimens, associated health care visits, and durations of treatment were reported. Time-to-event analyses were performed using the Kaplan-Meier method. RESULTS: A total of 498 patients with advanced SCLC were identified, of whom 429 were newly diagnosed with advanced disease and 69 progressed to advanced disease during the study period. Median survival from the index diagnosis date was 13.2 months. First-line (1L) chemotherapy was received by 464 (93.2%) patients, most commonly carboplatin/etoposide, received by 213 (45.9%) patients, followed by cisplatin/etoposide (20.7%). Ninety-five (20.5%) patients progressed to second-line (2L) chemotherapy, where topotecan monotherapy (20.0%) was the most common regimen, followed by carboplatin/etoposide (14.7%). Median survival was 10.1 months from 1L initiation and 7.7 months from 2L initiation. CONCLUSION: Patients in a regional health system network diagnosed with advanced SCLC were treated with chemotherapy regimens similar to those in earlier reports based on SEER-Medicare data. Survival of patients with advanced SCLC was poor, illustrating the lack of progress over several decades in the treatment of this lethal disease and highlighting the need for improved treatments.

Chemotherapy treatments, costs of care, and survival for patients diagnosed with small cell lung cancer: A SEER-Medicare study.

OBJECTIVES: The effectiveness and costs of new treatments should be assessed in relation to existing practice. We describe treatments, survival and costs for advanced or metastatic small cell lung cancer (SCLC) patients receiving systemic therapy in the period preceding the introduction of immunotherapies. MATERIALS AND METHODS: This was a retrospective cohort study of patients aged ≥65 years, identified using linked Surveillance, Epidemiology, and End Results and Medicare databases. Individuals with a new primary diagnosis of SCLC between January 2007 and December 2013 were followed until December 2014. Chemotherapy treatments, health care visits and costs (in 2016 USD), and survival were determined by line of therapy. RESULTS: A total of 11 812 patients were identified with SCLC. First-line (1L) chemotherapy was received by 6509 (55.1%) patients, most (93.2%) with carboplatin- (71.0%) or cisplatin- (22.2%) based therapies, typically combined with etoposide (79.2%). Second- (2L) and third- (3L) line chemotherapies were received by 2238 (18.9%) and 679 (5.7%) patients, of which 48.4% and 30.9%, respectively, were platinum-based. The median durations of 1L, 2L, and 3L carboplatin-based therapies were 5.9, 4.8, and 5.4 months, respectively, and the corresponding durations of cisplatin-based therapies were 5.3, 4.2, and 5.3 months. During 1L, 2L, and 3L chemotherapies, patients averaged 8.2, 7.4, and 7.3 health care visits per month, respectively, and incurred total mean health care costs of $60 223, $42 636, and $35 903 per patient, respectively. Median survival from the start of 1L, 2L, and 3L chemotherapy was 9.2, 6.0, and 5.7 months, respectively. CONCLUSION: First-line chemotherapy was primarily platinum-based, and a plethora of different regimens was used for 2L and 3L chemotherapies. Median survival from the start of 1L chemotherapy was 9 months, with an associated health care cost of $60 000. These data highlight an unmet medical need among SCLC patients receiving systemic therapy.

Real-world treatment persistence of golimumab in the management of immune-mediated rheumatic diseases in Europe: a systematic literature review.

OBJECTIVES: To summarise real-world data from studies reporting golimumab persistence in European immune-mediated rheumatic disease (IMRD) populations and to report pooled estimates. DESIGN: Systematic literature review. DATA SOURCES: Relevant literature was identified through searching Medline and Embase via Ovid as well as the conference databases of European League Against Rheumatism and American College of Rheumatology-Association of Rheumatology Health Professionals. ELIGIBILITY CRITERIA: We screened records using predefined patients, interventions, comparators, outcomes and study design criteria. Eligible studies included reports of persistence among adult IMRD patients in Europe receiving treatment with subcutaneous golimumab. Clinical trials, randomised controlled trials, literature reviews, editorials, guidelines and studies with <20 patients receiving golimumab were excluded. DATA EXTRACTION AND SYNTHESIS: Following double screening by two independent reviewers, 27 studies out of 578 identified records were selected for inclusion and subsequent data extraction. Persistence was most commonly reported at 12and 24 months; hence, pooled persistence estimates were calculated for these two time points and reported according to indication. RESULTS: Persistence ranged between 58.1% (psoriatic arthritis (PsA) patients regardless of treatment line) and 75.7% (biological-naïve rheumatoid arthritis patients) at 12 months; at 24 months, the range was 43% (axial spondyloarthritis (AxSpA) patients regardless of treatment line) and 69.6% (biological-naïve PsA patients). On the basis of data from 12 studies, persistence with golimumab treatment was either significantly higher or not significantly different from other tumour necrosis factor inhibitors (TNFi). CONCLUSIONS: Golimumab persistence at 24 months approximates 50%, with a lower persistence among AxSpA (43%) patients. However, as the number of studies in these populations was low, they warrant further research. In 12 studies comparing various TNFi treatments, golimumab was shown to have significantly better or equal persistence to its comparators.

Treatment with golimumab or infliximab reduces health resource utilization and increases work productivity in patients with ankylosing spondylitis in the QUO-VADIS study, a large, prospective real-life cohort.

AIM: We evaluated the effects of anti-tumor necrosis factor (TNF) agents on health economics in ankylosing spondylitis (AS) patients. METHODS: QUality of Life as Outcomes and its VAriation with DIsease States (QUO-VADIS) was a prospective observational study following bio-naïve AS patients (modified New York criteria) newly treated with golimumab (GLM) or infliximab (IFX; originator) in a clinical practice setting over 6 months. We evaluated use of concomitant medications, hospitalizations (in-patient care or acute care) and visits in day care and out-patient settings for the assessment of healthcare resource utilization (HCRU). Work productivity and activity impairment (WPAI) was assessed by the number of work days missed and quantifying absenteeism, presenteeism, work impairment, and activity using the WPAI instrument adapted to spondyloarthritis (WPAI-SpA). RESULTS: Nine hundred and sixty-three patients received ≥1 dose of medication (78%, n = 751 GLM; 22%, n = 221 IFX). Mean age was 42.7 years; 61.4% were male. At baseline, the percentage of patients who reported hospitalizations (in-patient care) was 13.6%, which decreased to 3.1% at 6 months, while out-patient care at baseline was reported by 39.4% of patients, which decreased to 19.0% at 6 months. The percentage of patients receiving acute emergency at baseline reduced from 1.6% to 0.3% at 6 months. The mean (SD) number of days of work missed due to AS, was reduced from 6.3 (31.1) days at baseline to 2.7 (12.3) days at 6 months. CONCLUSION: In patients with AS newly treated with GLM or IFX for 6 months, HCRU was reduced and work productivity and activity increased.

Remote Monitoring of Patient-Reported Outcomes in Ulcerative Colitis: A Prospective Real-World Pilot Study.

INTRODUCTION: The 6-point version of the Mayo score relies on two patient-reported outcomes (PRO2): stool frequency and rectal bleeding. We assessed the feasibility and acceptability of remote online PRO2 reporting for golimumab-treated ulcerative colitis (UC) patients. PATIENTS AND METHODS: This was a UK-based, multi-centre, prospective, real-world, non-interventional pilot study. Eligible patients completed PRO2 scores at baseline and every 4 weeks over a period of 6 months. Demographics were collected at baseline and a satisfaction questionnaire was completed at study end. Each patient provided data anonymously via an online platform. RESULTS: Fifty-two patients enrolled in the study. Mean (SD) patient age was 40.8 (13.6); 52% were male. Patients provided data on a personal computer (44%), mobile phone (38%) or tablet (18%). Forty-seven (90%) patients completed the baseline questionnaire within the accepted time range. Subsequent scores were reported on time by eligible patients with a success rate of 94%, 92%, 90%, 87%, 90% and 81% at end of months 1-6, respectively. CONCLUSIONS: Remote monitoring of PRO2 in UC was feasible amongst the sample tested. Of those initially willing to provide data in this way, attrition was low. Formal roll-out of this system could be used to support a more frequent assessment of UC symptoms without over-burdening the healthcare system.

Clinical and quality of life improvements with golimumab or infliximab in a real-life ankylosing spondylitis population: the QUO-VADIS study.

OBJECTIVES: The QUO VADIS study evaluated disease activity and health-related quality-of-life (HRQoL) in ankylosing spondylitis (AS) patients treated with golimumab (GLM) or infliximab (IFX, originator) during routine clinical care. METHODS: This prospective observational study followed biologics-naïve AS patients newly treated with GLM or IFX for 6 months. Disease activity (BASDAI, BASFI, ASAS, and ASDAS) and HRQoL improvement (≥5 points of SF-36 Physical Component Summary [PCS] score; PCS response) were measured. A Classification and Regression Trees (CART) analysis evaluated association of baseline parameters with PCS response at 6 months. RESULTS: 963 patients (mean age 43 years, 61% male, 64% HLA-B27 positive) received ≥1 dose of medication (78% GLM; 22% IFX). Disease activity was reduced; mean (SD) changes from baseline at month 6 of -2.7 (BASDAI) and -2.1 (BASFI) and 40% and 35% achievement of BASDAI50 and ASAS40 response, respectively, were observed. PCS response was achieved at month 6 in 52% of patients. Using CART analysis, baseline parameters (cut-off values) associated with HRQoL improvement were ASDAS (≥3.48), C-reactive protein (≥8.55 mg/L), age (≤35.5 years), and BASFI (≥1.15). This algorithm correctly identified 57.5% (sensitivity) of PCS responders (≥5 points) and 61.0% (specificity) of PCS non-responders (<5points) with ROC-AUC=0.61. Serious adverse events (AEs) occurred in 1.8% of patients; the most common AEs were infections (7.7%). CONCLUSIONS: We demonstrated clinical and HRQoL improvements over 6 months in a large, real-world population of AS patients newly treated with GLM or IFX; higher ASDAS, elevated CRP, and younger age were associated with improvements in HRQoL and an overall more robust response.