Cost-effectiveness of terlipressin for hepatorenal syndrome: the United States hospital perspective.

BACKGROUND: Hepatorenal syndrome (HRS) is characterized by severely reduced renal perfusion that precipitates rapid morbidity and mortality. Terlipressin is the only US Food and Drug Administration-approved treatment to improve kidney function for adults with HRS with a rapid reduction in kidney function. Prior to the approval of terlipressin, unapproved vasoconstrictive agents used in HRS treatment were octreotide/midodrine and norepinephrine with albumin. METHODS: A cohort decision-tree model representing a US hospital perspective assessed the clinical outcomes and direct medical costs (based primarily on hospital charges) of treating HRS with terlipressin + albumin (ALB) versus midodrine/octreotide (MID/OCT)+ALB, or norepinephrine (NorEp)+ALB. Treatment efficacy was defined by clinical response (complete/HRS reversal, partial, or no response) based on change of serum creatinine derived from published clinical trial reports. The proportions of patients with complete response were: terlipressin + ALB (36.2%), NorEp + ALB (19.1%), and MID/OCT + ALB (3.1%). Model outcomes included utilization of HRS-related healthcare resources (hospital and intensive care, outpatient and emergency department, dialysis, and transplantations), adverse events, and HRS-related mortality. Outcomes were assessed for the initial hospitalization in the base case and at 30, 60, and 90 days post-discharge. RESULTS: Total costs incurred over the initial hospitalization with terlipressin + ALB were lower vs NorEp + ALB, primarily due to higher ICU costs with NorEp + ALB ($7,433 vs $61,897). TER + ALB was associated with higher total costs vs MID/OCT + ALB due to higher pharmacy costs with terlipressin + ALB. The cost per complete response achieved of terlipressin + ALB ($451,605) was half that of NorEp + ALB ($930,571) and one-tenth that of MID/OCT + ALB ($4,942,123). CONCLUSIONS: HRS patients treated with terlipressin experienced better clinical outcomes and a lower cost per treatment response vs other unapproved treatments. ICU days and pharmacy costs were key cost drivers distinguishing the treatment groups. These outcomes suggest that terlipressin is cost-effective on the basis of total cost per response achieved.

Hepatorenal syndrome (HRS) is a rare and sudden life-threatening complication of the liver. Patients with HRS should receive immediate treatment with a drug that narrows blood vessels known as a vasoconstrictor. Terlipressin is the most common vasoconstrictor used for patients with HRS. Other common vasoconstrictors are midodrine with octreotide and norepinephrine. This study aimed to compare the cost of terlipressin with those of midodrine with octreotide and norepinephrine while also considering how well each of them worked to reverse HRS. This was done using an economic model. This economic model assessed the costs of the vasoconstrictor drugs and the costs of treating HRS, including costs attributable to drug acquisition, adverse events, organ transplantation, dialysis, and institutional encounters (i.e. hospitalization, ICU, emergency department, and outpatient visits). The magnitude of these costs depends on how well each drug reversed HRS. Based on inputs derived from their respective clinical trials, 36% of patients who were given terlipressin had a complete response (HRS was reversed), 19% of patients who were given norepinephrine had a complete response, and 3% of patients who were given midodrine with octreotide had a complete response. The total cost per patient was approximately $163,481 for terlipressin, $177,298 for norepinephrine, and $155,030 for midodrine with octreotide. When the costs were evaluated against how well the drugs worked to reverse HRS, the lowest cost per HRS reversal was $451,605 when treated with terlipressin. The cost per reversal for norepinephrine was $930,571 and for midodrine with octreotide was $4,942,123. These results show that terlipressin works well and is more cost-effective for US hospitals compared with the other unapproved treatment options for HRS with rapid reduction in kidney function.

The burden of ovarian cancer in the USA from 2007 to 2018: evidence from the Medical Expenditure Panel Survey.

Aim: To evaluate the economic and humanistic burden of ovarian cancer in the USA. Methods: Medical Expenditure Panel Survey data (2007-2018) were used to estimate all-cause healthcare resource use and costs for economic burden and examine the activities of daily living and quality-of-life (QoL) measures for humanistic burden between ovarian cancer patients and a non-cancer population. Results: Compared with controls, patients with ovarian cancer had more comorbidities and worse QoL. Their predicted number of annual hospitalizations and office-based visits was significantly higher, as were their estimated annual all-cause total healthcare costs. Total costs were driven by hospitalization costs. Conclusion: The study identified the burden of ovarian cancer and demonstrated that patients with ovarian cancer have greater healthcare resource use, higher costs and worse QoL than the non-cancer population. Future research is needed to develop strategies for managing ovarian cancers and inform decision-making to reduce disease burden.

What is this article about? The article discusses the impact that ovarian cancer has, both in terms of economics and quality of life. We used data from 2007 to 2018 to identify women with ovarian cancer as well as women without cancer for the sake of comparison. What were the results? We found that individuals with ovarian cancer face considerable burdens, and their treatment costs have a notable impact on healthcare systems. Compared with women without cancer, women with ovarian cancer are older and have a greater number of additional illnesses, and their quality of life is lower. Their use of healthcare resources is greater and hence the costs associated with their treatment are higher. What do the results of the study mean? This study adds to the existing data about the burden imposed by ovarian cancer, on individuals as well as on healthcare systems. Interventions are needed to reduce the impacts of the disease.

Treatment patterns and real-world clinical outcomes in patients with advanced endometrial cancer who are microsatellite instability (MSI)-high or are mismatch repair deficient (dMMR) in the United States.

OBJECTIVES: Microsatellite instability-high (MSI-H) and deficient DNA mismatch repair (dMMR) status have emerged as actionable biomarkers for advanced endometrial cancer (aEC). The objective of this study was to assess clinical outcomes and treatment patterns among MSI-H/dMMR aEC patients who had disease progression following prior systemic therapy (FPST) in the US. METHODS: Endometrial Cancer Health Outcomes (ECHO) was a retrospective, medical chart review study of patients with MSI-H/dMMR aEC who had disease progression between 07/01/2016 and 12/31/2018 FPST and were not candidates for curative surgery. Data on patient demographics, clinical and treatment characteristics, and clinical outcomes were collected. Kaplan-Meier analyses were performed to estimate real-world progression-free survival (rwPFS) and overall survival (OS), stratified by drug class. RESULTS: A total of 124 eligible patients who initiated second-line chemotherapy ± bevacizumab or immunotherapy were included. Mean age was 61.4 years at aEC diagnosis and 86.3% of patients were stage IIIB-IV. Median rwPFS and OS were 4.0 months (95% CI: 2.0-9.0) and 7.0 months (95% CI: 5.0-18.0), respectively, among 21 patients who received chemotherapy ± bevacizumab, and 29.0 months (95% CI: 18.0-NE) and not reached (95% CI: 30.0-NA), respectively, among 103 patients who received immunotherapy. Most patients (n = 92) received pembrolizumab; among these patients, rwPFS and OS were 29.0 months (95% CI: 18.0-NE) and 30 months (95% CI: 30.0-NA), respectively. CONCLUSIONS: Real-world evidence suggests that pembrolizumab monotherapy provides considerable clinical benefits and has become the standard of care for MSI-H/dMMR aEC patients FPST who are not candidates for curative surgery in real-world settings.

Treatment patterns and real-world clinical outcomes in patients with advanced endometrial cancer that are non-microsatellite instability high (non-MSI-high) or mismatch repair proficient (pMMR) in the United States.

Objective: Microsatellite instability (MSI) due to defective DNA mismatch repair has emerged as an actionable biomarker in advanced endometrial cancer (aEC). Currently, there are no treatment patterns and outcomes data in non-MSI-high (non-MSI-H) or mismatch repair proficient (pMMR) aEC patients following prior systemic therapy (FPST). Our goal was to describe real-world data in this population in the US in 2019 and prior years. Methods: Endometrial Cancer Health Outcomes (ECHO) is a retrospective patient chart review study conducted in the US. Patients with non-MSI-H/pMMR aEC and progression between 06/01/2016-06/30/2019 FPST were eligible. Data collected included patient demographics, clinical and treatment characteristics, and clinical outcomes. Kaplan-Meier analyses were performed to estimate time to treatment discontinuation, real-world progression-free survival (rwPFS), and overall survival (OS), separately by treatment category. Results: A total of 165 eligible patients initiated second-line therapy with chemotherapy ± bevacizumab (n = 140) or hormonal therapy (n = 25). Median age was 66.0 years at aEC diagnosis, 70.2% were Stage IIIB-IV, 40.0% had ECOG ≥ 2 at second-line therapy initiation. Median rwPFS was 5.0 months (95% CI: 4.0-6.0) for patients receiving chemotherapy ± bevacizumab and 5.5 months (95% CI: 3.0-29.0) for those receiving hormonal therapy. Median OS was 10.0 months (95% CI: 8.0-13.0) and 9.0 months (95% CI: 6.0-NA) in these groups, respectively. Conclusions: Non-MSI-H/pMMR patients who initiated second-line therapy with chemotherapy ± bevacizumab or hormonal therapy had poor clinical outcomes with a median survival less than 1 year and rwPFS less than 6 months. This was the first study to define the clinical unmet need in patients with non-MSI-H/pMMR aEC with conventional therapy.

Impact of Hypomethylating Agent Use on Hospital and Emergency Room Visits, and Predictors of Early Discontinuation in Patients With Higher-Risk Myelodysplastic Syndromes.

BACKGROUND: Previous analyses using the SEER-Medicare database have reported substantial underutilization of hypomethylating agents (HMAs) among patients with higher-risk myelodysplastic syndromes (MDS), and an association between poor HMA persistence and high economic burden. We aimed to compare rates of hospitalizations and emergency room (ER) visits among patients with higher-risk MDS according to use or non-use of HMA therapy, and to explore factors associated with early discontinuation of HMA therapy. PATIENTS AND METHODS: We used the 2010-2016 SEER-Medicare database to identify patients aged ≥66 years with a new diagnosis of refractory anemia with excess blasts (RAEB; a surrogate for higher-risk MDS) between 2011 and 2015. New hospitalizations and ER visits during the 12 months following MDS diagnosis were determined. Treatment discontinuation was defined as stopping HMA therapy before 4 cycles. RESULTS: Overall, 664 (55.8%) patients were HMA users and 526 (44.2%) non-users. Non-users had more hospitalizations (mean 0.47 vs. 0.30, P < .001) and ER visits (mean 0.69 vs. 0.41, P = .005) per month than HMA users. Among HMA users, 193 (29.1%) discontinued HMA therapy before 4 cycles, and 91 (47.2%) of these after 1 cycle. Older age and poor performance status were associated with higher risk of HMA discontinuation. CONCLUSION: An increased rate of hospitalizations and ER visits occurred in HMA non-users vs. HMA users. Approximately one-third of patients discontinued HMA therapy early. Predictors of discontinuation included older age and poor performance status. Novel approaches are needed to improve utilization and persistence with HMA therapy and associated outcomes, particularly among these higher-risk groups.

Real-world use of inhaled nitric oxide therapy in patients with COVID-19 and mild-to-moderate acute respiratory distress syndrome.

Background: Inhaled nitric oxide (iNO) has been studied in patients with severe acute respiratory distress syndrome (ARDS) due to COVID-19 when it may be too late to impact disease course. This article aims to describe real-world iNO use and outcomes in patients with COVID-19 with mild-to-moderate ARDS in the United States. Methods: This was a retrospective medical chart review study that included patients who were ≥18 years old, hospitalized for COVID-19, met the Berlin ARDS definition, received iNO for ≥24 hours continuously during hospitalization, and had a partial pressure of oxygen (PaO2)/fraction of inspired oxygen (FiO2) ratio (P/F ratio) of >100 to ≤300 mmHg at iNO initiation. Outcomes included oxygenation parameters, physician-rated Clinical Global Impression-Improvement (CGI-I) scale scores, and adverse events. Response to iNO was defined as >20% improvement in P/F ratio. Results: Thirty-seven patients at six sites were included. A P/F ratio of ≤100 was the most common reason for exclusion (n=146; 83% of excluded patients). The mean P/F ratio (SD) increased from 136.7 (34.4) at baseline to 140.3 (53.2) at 48 hours and 151.8 (50.0) at 72 hours after iNO initiation. The response rate was 62% (n=23). During hospitalization, no patient experienced adverse events, including methemoglobinaemia, airway injury, or worsening pulmonary oedema associated with iNO. At discharge, 54.0% (n=20) of patients improved or remained stable according to the CGI-I. Conclusion: In patients hospitalized with COVID-19 and mild-to-moderate ARDS, iNO was associated with improvement in the P/F ratio with no reported toxicity. This study provides additional evidence supporting a favourable benefit-risk profile for iNO in the treatment of mild-to-moderate ARDS in patients with COVID-19 infection.

Real-world burden of adverse events for apalutamide- or enzalutamide-treated non-metastatic castration-resistant prostate cancer patients in the United States.

BACKGROUND: Second-generation androgen receptor inhibitors (ARIs) have been associated with adverse events (AEs) such as fatigue, falls, fractures, and rash in non-metastatic castration-resistant prostate cancer (nmCRPC) patients as identified in clinical trials. The objectives of this study were to describe the incidence and management of AEs in patients receiving apalutamide and enzalutamide. METHODS: This retrospective chart review study was conducted in nmCRPC-treating sites in the United States. Patients starting apalutamide or enzalutamide between February 1, 2018 and December 31, 2018 were included and any AEs they experienced were recorded. AEs, including those considered to be of special interest as defined in the pivotal clinical trials of the second-generation ARIs, were analyzed and grouped retrospectively in this study. Detailed chart data (patient demographics, clinical characteristics, treatment history, type of AE, outcomes, and resource utilization) were then collected for a randomly selected subset among patients with ≥1 AE to characterize AEs and their management. Descriptive results were summarized. RESULTS: Forty-three sites participated in the study. A total of 699 patients were included, of whom 525 (75.1%) experienced ≥1 AE. The most common AEs were fatigue/asthenia (34.3%), hot flush (13.9%), and arthralgia (13.6%). In the subset of 250 patients randomly selected from those who experienced ≥1 AE, patients were primarily White (72.0%), the mean age was 71 years, 86.0% had an Eastern Cooperative Oncology Group score of 0-1 at nmCRPC diagnosis, and the average prostate specific antigen (PSA) value at diagnosis was 23.2 ng/mL. PSA-doubling time < 10 months was chosen as reason to initiate treatment in 40% of patients. The median duration of follow-up was 1.1 years, with 14.4% of patients progressing to metastasis by end of study period. Grade 3-4 and Grade 5 AEs occurred in 14.4 and 0.4% of patients, respectively. Actions taken to manage AEs included AE-directed treatment (38.0%), ARI discontinuation (10.4%), dose reduction (7.6%), and AE-related hospitalization (4.8%). CONCLUSIONS: This study highlights the burden of AEs among nmCRPC patients treated with apalutamide or enzalutamide, providing a relevant real-world benchmark as clinical trial evidence and the treatment landcape for nmCRPC continues to evolve.

Treatment patterns and economic burden among newly diagnosed cervical and endometrial cancer patients.

Aim: This study evaluated treatment patterns, healthcare resource use and healthcare costs among newly diagnosed US patients with cervical or endometrial cancer. Materials & methods: The authors identified patients diagnosed between 2015 and 2018, described them by line of therapy (LOT), then summarized all-cause per patient per month healthcare resource use and healthcare costs per LOT. Results: Among 1004 patients with cervical cancer and 2006 patients with endometrial cancer, 65.2 and 71.4%, respectively, received at least LOT1. Common treatment modalities in LOT1 were surgery (cervical, 58.0%; endometrial, 92.6%), radiation therapy (cervical, 49.8%; 24.7%) and systemic therapy (cervical, 53.3%; endometrial, 26.1%). Mean per patient per month costs per LOT were pre-treatment (cervical, US$17,210; endometrial, US$14,601), LOT1 (cervical, US$10,929; endometrial, US$6859), LOT2 (cervical, US$15,183; endometrial, US$10,649) and LOT3+ (cervical, US$19,681; endometrial, US$9206). Conclusion: Overall, newly diagnosed patients with cervical or endometrial cancer received guideline-recommended treatment. Outpatient visits mainly drove healthcare costs across LOTs.

Treatment patterns and economic burden among cervical and endometrial cancer patients newly initiating systemic therapy.

Aim: To evaluate treatment patterns, healthcare resource use (HCRU) and all-cause healthcare costs among patients with cervical or endometrial cancer newly initiating systemic therapy. Methods: We identified patients with cervical or endometrial cancer newly initiating systemic therapy - a claims-based proxy for advanced disease - between 2014 and 2019, described them by line of therapy (LOT), and summarized the per patient per month (PPPM) HCRU and healthcare costs per LOT. Results: Among 1229 patients with cervical cancer and 2659 patients with endometrial cancer, LOT1 therapies included systemic only (cervical, 50.1%; endometrial, 83.2%) and systemic with radiation therapy (cervical, 49.9%; endometrial, 16.8%). Mean PPPM total costs were: LOT1 (cervical, US$15,892; endometrial, US$11,363), LOT2 (US$20,193; US$14,019) and LOT3+ (US$16,576; US$14,645). Conclusions: Overall, patients received guideline-concordant care and experienced significant economic burden, which increased with LOT.

Clinical and Economic Burden of Out-of-Hospital Cardiac Arrest in US Commercial Insurance Population (2014 to 2019).

Little is known about the economic burden incurred by out-of-hospital cardiac arrest (OHCA) in the US commercial insurance setting. We used IBM MarketScan Commercial Claims and Encounters Database (January 2014 to March 2019) to identify patients hospitalized with OHCA based on the International Classification of Diseases codes. Patients who survived the initial OHCA episode were stratified by prognosis based on discharge setting and classified into mild (discharged home), moderate (skilled nursing facility), severe (inpatient rehabilitation or long-term hospital), and very severe (hospice) prognosis groups, respectively. Patients were followed up for 12 months after discharge for health care resource utilization and medical costs, which were inflated to year 2020. Overall, 23,512 patients with OHCA hospitalization were identified, of whom 14,667 were <65 years and 60.5% were men. The incidence of OHCA per 100,000 was steady in patients <65 years over the years (17.9 in 2014; 17.5 in 2018) but among those ≥65 years, decreased from 139.7 in 2014 to 111.1 in 2018. Total medical costs 12 months after discharge generally increased with severity of prognosis, with an average for the mild, moderate, and severe prognosis group, respectively, estimated to be $52,746, $100,394, and $130,530 among patients <65 years, and $63,194, $65,794, and $70,973 among those ≥65 years. Costs were lower for those with very severe prognosis ($7,102 for <65 years; $2,553 for ≥65 years), possibly due to high mortality. In conclusion, OHCA continues to pose a substantial clinical and economic burden on patients and the US health care system, which increases with the severity of disease prognosis.

Phase-specific and lifetime economic burden of cervical cancer and endometrial cancer in a commercially insured United States population.

AIM: To estimate the incremental phase-specific and lifetime economic burden among newly diagnosed cervical and endometrial cancer patients vs. non-cancer controls. METHODS: Cervical and endometrial cancer patients newly diagnosed between January 2015 and June 2018 were identified in the Optum Clinformatics DataMart database. The index date was the date of the first diagnosis for cancer cases and the first claim date after 12 months of continuous enrollment for non-cancer controls. Patients were followed until death/loss of enrollment/end of data availability. Per patient per month (PPPM) costs attributable to cancer were calculated for four phases: pre-diagnosis (3 months before diagnosis), initial (6 months post-diagnosis), terminal (6 months pre-death), and continuation (remaining time between initial and terminal phases). Survival data were obtained to determine the monthly proportion of patients in each phase. Total survival adjusted monthly costs were obtained by multiplying the proportion of patients in each phase by the total cost incurred during that month. Phase-specific and lifetime incremental costs of cervical and endometrial cancer were obtained using generalized linear models. RESULTS: The analytic cohort included 1,002 cervical cancer patients and 4,005 matched non-cancer controls and 5,003 endometrial cancer patients matched with 19,999 non-cancer controls. Mean adjusted incremental PPPM lifetime costs (95% CI) for cervical cancer and endometrial cancer cases were $5,910 ($5,373-$6,446) and $3,475 ($3,259-$3,691), respectively. Incremental total PPPM phase-specific costs attributable to cervical and endometrial cancer were pre-diagnosis (cervical: $1,057; endometrial: $3,315), initial ($12,084; $8,618), continuation ($2,732; $1,147), and terminal ($2,702; $5,442). Incremental costs were significantly higher for cancer patients vs. non-cancer controls across patient lifetime and all phases of care (except terminal phase costs for cervical cancer). Outpatient costs were the major driver of costs across all post-diagnosis phases. CONCLUSION: This study highlights the cost burden associated with cervical/endometrial cancer and cost variation by phases of care.

Treatment patterns and outcomes among nontransplant newly diagnosed multiple myeloma patients in Spain.

Aim: To describe treatment patterns and outcomes in nontransplant newly diagnosed multiple myeloma (NDMM) patients in Spain. Methods: This retrospective study included two cohorts of NDMM patients diagnosed between 1 January 2012 to 31 December 2013 and 1 April 2016 to 31 March 2017. Results: Among 113 patients, proteasome inhibitor (PI) + alkylator combinations (49%) and PI-based regimens without an alkylator (30%) were the most common first-line (1L) therapies. Use of PI + immunomodulatory drug-based regimens increased between the cohorts; PI-based regimens without an alkylator/immunomodulatory drug decreased. Use of 1L oral regimens was low but increased over time; use of maintenance therapy was low across both periods. Median 1L duration of treatment was 6.9 months. Conclusion: Short 1L duration of treatment and low use of 1L oral regimens and maintenance therapy highlight unmet needs in NDMM.

Lay abstract This study describes treatment patterns and outcomes in newly diagnosed multiple myeloma (NDMM) patients in Spain who were not candidates for transplant. The study looked at two patient groups: patients diagnosed between 1 January 2012 and 31 December 2013 and those diagnosed between 1 April 2016 and 31 March 2017. Among the 113 patients considered, the most common first-line therapies were proteasome inhibitor (PI) + alkylator combinations (49%) and PI-based regimens without an alkylator (30%). We saw increased use of PI with immunomodulators (which arm the immune system to battle disease) and decreased use of PI-based regimens without an alkylator or immunomodulator. First-line use of oral regimens was low but increased over time. The median length of first-line treatment for both groups combined was 6.9 months. Finding low use of first-line oral regimens and maintenance therapy and a short duration of first-line treatment, our study highlights the unmet needs that exist in NDMM patients who are not transplant candidates in Spain.

Corrigendum: Economic Value of Lost Productivity Attributable to Human Papillomavirus Cancer Mortality in the United States.

[This corrects the article DOI: 10.3389/fpubh.2020.624092.].

Impact of reduced human papillomavirus vaccination coverage rates due to COVID-19 in the United States: A model based analysis.

The coronavirus disease 2019 (COVID-19) pandemic has significantly affected utilization of preventative health care, including vaccines. We aimed to assess HPV vaccination rates during the pandemic, and conduct a simulation model-based analysis to estimate the impact of current coverage and future pandemic recovery scenarios on disease outcomes. The model population included females and males of all ages in the US. The model compares pre-COVID vaccine uptake to 3 reduced coverage scenarios with varying recovery speed. Vaccine coverage was obtained from Truven Marketscan™. Substantially reduced coverage between March-August 2020 was observed compared to 2018-2019. The model predicted that 130,853 to 213,926 additional cases of genital warts; 22,503 to 48,157 cases of CIN1; 48,682 to 110,192 cases of CIN2/3; and 2,882 to 6,487 cases of cervical cancer will occur over the next 100 years, compared to status quo. Providers should plan efforts to recover HPV vaccination and minimize potential long-term consequences.

Direct Medical Costs Associated With Treatment Nonpersistence in Patients With Higher-Risk Myelodysplastic Syndromes Receiving Hypomethylating Agents: A Large Retrospective Cohort Analysis.

BACKGROUND: Suboptimal use of hypomethylating agents (HMAs) among higher-risk myelodysplastic syndrome (HR-MDS) patients can translate into worse health outcomes and economic burden. We estimated the direct medical costs associated with HMA treatment nonpersistence among HR-MDS patients. PATIENTS AND METHODS: Using the Surveillance, Epidemiology, and End Results-Medicare linked database, a retrospective cohort of patients diagnosed with refractory anemia with excess blasts (RAEB), a diagnosis that substantially overlaps with HR-MDS, between January 2011 and December 2015 was analyzed. Patients who had ≥ 1 year of continuous Medicare enrollment before diagnosis and who did not receive stem cell transplant or lenalidomide in the follow-up period were included. Patients receiving HMAs were stratified into HMA persistent (≥4 HMA cycles) and HMA nonpersistent (<4 cycles or a gap of ≥ 90 days between cycles) groups. Healthcare resource use and costs during the follow-up period were reported descriptively as total and per patient per month (PPPM). Weighted generalized linear models (GLM) were used to compare estimated healthcare resource use and costs between HMA groups. RESULTS: Among the 664 patients with RAEB, 295 (44.4%) were HMA nonpersistent and 369 (55.6%) HMA persistent. On the basis of weighted GLM analysis, the HMA nonpersistent group incurred significantly (P < .05) higher total PPPM costs compared to the HMA persistent group ($18,039 vs. $13,893), particularly for hospitalization ($3,375 vs. $2,131), and emergency room ($5,517 vs. $2,867) costs. CONCLUSION: There is a substantial economic burden associated with early discontinuation of guideline-recommended HMA therapy in RAEB patients. The study findings necessitate closer care management in this population in order to improve outcomes and reduce healthcare spending.

Cost-Utility of All-Oral Direct-Acting Antiviral Regimens for the Treatment of Genotype 1 Chronic Hepatitis C Virus-Infected Patients in Hong Kong.

BACKGROUND: Direct-acting antivirals (DAAs) are entering the hepatitis C virus (HCV) treatment landscape in Hong Kong, prompting the need for cost-effectiveness evaluations of these interventions to enable optimal use of healthcare resources. AIMS: This study aimed to compare the cost-effectiveness of DAAs to standard-of-care pegylated interferon plus ribavirin (RBV) in treatment-naïve patients without significant liver fibrosis and to compare different DAAs in patients who are treatment-experienced and/or have advanced liver disease. METHODS: A Markov model was constructed to evaluate cost-effectiveness over a lifetime time horizon from the payer perspective. The target population was treatment-naïve and treatment-experienced HCV genotype 1 patients, stratified by degree of liver fibrosis. The model consists of 16 health states encompassing METAVIR fibrosis score (F0-F4), treatment success or failure, decompensated cirrhosis, hepatocellular carcinoma, liver transplant, and liver-related death. The proportions of patients achieving sustained virologic response were obtained from clinical trials. Other inputs were obtained from published and local data. The primary outcome was incremental cost-utility ratio for each DAA versus pegylated interferon + ribavirin and among different DAAs. RESULTS: In treatment-naïve F0-2 HCV patients, all DAAs were cost-effective in genotype 1a and daclatasvir + asunaprevir, elbasvir/grazoprevir, ledipasvir/sofosbuvir, and glecaprevir/pibrentasvir were cost-effective compared to pegylated interferon + ribavirin in genotype 1b. In genotypes 1a and 1b, treatment-experienced patients, and F3-4 patients, elbasvir/grazoprevir was the least costly DAA and economically dominant over most other DAAs. CONCLUSIONS: DAAs can be a cost-effective option for the treatment of genotype 1 HCV patients in Hong Kong, and elbasvir/grazoprevir is cost-effective.

Under-use of Hypomethylating Agents in Patients With Higher-risk Myelodysplastic Syndrome in the United States: A Large Population-based Analysis.

BACKGROUND: Recent data suggest significant underutilization of hypomethylating agents (HMAs) that are recommended treatments for patients with myelodysplastic syndromes (MDS) with refractory anemia with excess blasts (RAEB). The study objective was to assess the degree of HMA use and predictors of HMA underuse in this population. PATIENTS AND METHODS: This was a retrospective study including patients diagnosed with the RAEB form of MDS between January 2011 and December 2015 using the Surveillance, Epidemiology, and End Results-Medicare linked database. Patients were excluded if they had < 1 year of continuous enrollment before diagnosis or received stem cell transplant or lenalidomide during the follow-up period. HMA non-peristence was defined as use of < 4 cycles (3-10 HMA days/28 days) of HMAs or a gap of ≥ 90 days between consecutive cycles. Patients were characterized as HMA never-users, HMA-persistent users, and HMA-non-persistent users. Descriptive statistics were used to summarize patient characteristics. Multivariable logistic regression was used to assess predictors of HMA underuse and persistence. RESULTS: Of the 1190 patients, 526 (44%) were never-users, 295 (25%) were non-persistent users, and 369 (31%) were persistent users. Age at diagnosis (eg, 66-70 years vs. ≥ 80 years; odds ratio [OR], 2.36; 95% confidence interval [CI], 1.56-3.56), marital status (single vs. married; OR, 0.67; 95% CI, 0.51-0.89), National Cancer Institute comorbidity index (≥ 3 vs. 0-1; OR, 0.62; 95% CI, 0.46-0.83), and performance status (poor vs. good; OR, 0.67; 95% CI, 0.51-0.87) were significantly associated with HMA underuse. CONCLUSION: Several demographic and clinical factors were associated with underuse of HMAs. There is need for a better understanding of suboptimal HMA use and its relationship with clinical response.

Real-world treatment patterns and outcomes using terlipressin in 203 patients with the hepatorenal syndrome.

BACKGROUND: Hepatorenal syndrome and acute kidney injury are common complications of decompensated cirrhosis, and terlipressin is recommended as first-line vasoconstrictor therapy. However, data on its use outside of clinical trials are lacking. AIMS: To assess practice patterns and outcomes around vasoconstrictor use for hepatorenal syndrome in UK hospitals. METHODS: This was a multicentre chart review study. Data were extracted from medical records of patients diagnosed with hepatorenal syndrome and treated by vasoconstrictor drugs between January 2013 and December 2017 at 26 hospitals in the United Kingdom. The primary outcome was improvement of kidney function, defined as complete response (serum creatinine improved to ≤1.5 mg/dL), partial response (serum creatinine reduction of ≥20% but >1.5 mg/dL) and overall response (complete or partial response). Other outcomes included need for dialysis, mortality, liver transplantation and adverse events. RESULTS: Of the 225 patients included in the analysis, 203 (90%) were treated with terlipressin (median duration, 6 days; range: 2-24 days). Mean (±standard deviation) serum creatinine at vasopressor initiation was 3.25 ± 1.64 mg/dL. Terlipressin overall response rate was 73%. Overall response was higher in patients with mild acute kidney injury (baseline serum creatinine <2.25 mg/dL), compared to those with moderate (serum creatinine ≥2.25 mg/dL and <3.5 mg/dL) or severe (serum creatinine ≥3.5 mg/dL). Ninety-day survival was 86% for all patients (93% for overall responders vs 66% for treatment nonresponders, P < 0.0001). CONCLUSION: Terlipressin is the most commonly prescribed vasoconstrictor for patients with hepatorenal syndrome in the United Kingdom. Treatment with terlipressin in patients with less severe acute kidney injury (serum creatinine <2.25 mg/dL) was associated with higher treatment responses, and 90-day survival.

Real-world treatment patterns and outcomes in non-transplant newly diagnosed multiple Myeloma in France, Germany, Italy, and the United Kingdom.

OBJECTIVES: The treatment paradigm in newly diagnosed multiple myeloma (NDMM) is evolving toward individualized, risk-directed, and longer duration of therapy (DOT). The objective of this study was to describe treatment patterns and outcomes in non-transplant NDMM in four European countries. METHODS: This retrospective chart review included adults with NDMM diagnosed between January 1, 2012, and December 31, 2013 (early cohort), or April 1, 2016, and March 31, 2017 (recent cohort). RESULTS: Among 836 patients, molecular testing was performed in 21% and 35% patients of early vs recent cohorts; proteasome inhibitor (PI)/alkylator combinations were the principal first-line (1 L) therapy (39% vs 43%). Use of immunomodulatory drug (IMID)/alkylator combinations declined from early to recent cohort (26% vs 13%) but IMID (7% vs 16%) use increased. Few patients (5%) received 1 L maintenance therapy. Two-thirds of patients were treated with a fixed duration intent, with a median 7-month 1 L DOT and progression-free survival (PFS) of 32.8 months in the early cohort. Both 1 L DOT and PFS were longer with oral compared to injectable regimens. CONCLUSIONS: Although frontline treatment patterns changed significantly, 1 L DOT is short. The uptake of molecular testing and 1 L maintenance is low. These results highlight areas of unmet need in NDMM.

Economic and Humanistic Burden of Cervical Cancer in the United States: Results from a Nationally Representative Survey.

Objective: To measure the economic and humanistic burden of cervical cancer in the United States. Materials and Methods: This was a retrospective analysis of Medical Expenditure Panel Survey data (2006-2015). Cervical cancer cases were identified using International Classification of Diseases, Ninth Revision, Clinical Modification code "180" or clinical classification software code "26". The control group included women without any cancer diagnosis. Study outcomes included health care resource use (institutional inpatient and outpatient, emergency room, and physician office visits), costs, limitations in activities of daily living, and quality of life (general health status, 12-Item Short Form Health Survey [SF-12] physical and mental component summary [MCS], EuroQol-5D and Short-Form Six-Dimension health utility, and Patient Health Questionnaire-2 depression severity). Generalized linear models, controlling for sociodemographic and clinical covariates, were conducted to compare outcomes between cases and controls. Results: The analytic cohort included 275,246 cervical cancer cases and 146,061,609 noncancer controls. Cases were significantly older (mean age [years]: 42.03 vs. 36.98) and had a higher Charlson comorbidity burden (mean score: 1.06 vs. 0.46) versus controls. Multivariate analyses suggested that compared to controls, cancer cases had significantly higher costs: institutional outpatient ($1,610 vs. $502), physician visit ($2,422 vs. $1,321), and total health care ($10,031 vs. $4,913). Cases were 1.99 (odds ratio [OR]: 1.991; 95% confidence interval [CI]: 1.23-3.22) and 2.56 (OR: 2.562; 95% CI: 1.78-3.68) times as likely to report activity limitations and poor general health versus controls. Cervical cancer patients had significantly lower SF-12 physical and MCS score, health utility, and higher depression severity. Conclusions: Cervical cancer is associated with significant economic burden, activity limitations, and quality of life impairment among ambulatory women in the United States.