Real-world treatment patterns, healthcare use and costs in triple-class exposed relapsed and refractory multiple myeloma patients in the USA.

Aim: To estimate treatment patterns and healthcare costs among triple-class exposed relapsed and refractory multiple myeloma (RRMM) patients. Materials & methods: Eligible patients had ≥1 line of therapy (LOT) each of proteasome inhibitors, immunomodulatory drugs and daratumumab in December 2015-September 2018 and received a new LOT. Results: A total of 154 patients were included with a median follow-up of 6.2 months. Median time from diagnosis to new LOT was 41.0 months. Kaplan-Meier estimate of median time to therapy discontinuation was 4.2 months. Mean per-patient, per-month MM-related costs were USD 35,657. Most frequently observed regimens were lenalidomide or pomalidomide + daratumumab (18.2%), lenalidomide or pomalidomide + proteasome inhibitors (15.6%) and lenalidomide or pomalidomide monotherapy (11.0%). Conclusion: Triple-class exposed RRMM patients receive heterogeneous treatments for a short duration with high healthcare resource utilization and costs.

Risk of chemotherapy-induced febrile neutropenia in cancer patients receiving pegfilgrastim prophylaxis: does timing of administration matter?

PURPOSE: Contrary to the approved indication for pegfilgrastim prophylaxis, some patients receive it on the same day as the last administration of chemotherapy in clinical practice, which could adversely impact risk of febrile neutropenia (FN). An evaluation of the timing of pegfilgrastim prophylaxis and FN risk was undertaken. METHODS: A retrospective cohort design and data from two US private health care claims repositories were employed. Study population comprised adults who received intermediate/high-risk chemotherapy regimens for solid tumors or non-Hodgkin's lymphoma (NHL) and received pegfilgrastim prophylaxis in ≥1 cycle; all cycles with pegfilgrastim were pooled for analyses. Odds ratios (OR) for FN during the cycle were estimated for patients who received pegfilgrastim on the same day (day 1) as the last administration of chemotherapy versus days 2-4 from chemotherapy completion. RESULTS: The study population included 45,592 patients who received pegfilgrastim in 179,152 cycles (n = 37,095 in cycle 1); in 12 % of cycles, patients received pegfilgrastim on the same day as chemotherapy. Odds of FN were higher for patients receiving pegfilgrastim prophylaxis on the same day as chemotherapy versus days 2-4 from chemotherapy in cycle 1 (OR = 1.6, 95 % CI = 1.3-1.9, p < 0.001) and all cycles (OR = 1.5, 95 % CI = 1.3-1.6, p < 0.001). CONCLUSIONS: In this large-scale evaluation of adults who received intermediate/high-risk regimens for solid tumors or NHL in US clinical practice, FN incidence was found to be significantly higher among those who received pegfilgrastim prophylaxis on the same day as chemotherapy completion versus days 2-4 from chemotherapy completion, underscoring the importance of adhering to the indicated administration schedule.

Utilization of intravenous bisphosphonates in patients with bone metastases secondary to breast, lung, or prostate cancer.

PURPOSE: Cancer patients with bone metastases (BMets) are predisposed to skeletal complications. Bone-targeted therapies such as denosumab or intravenous bisphosphonates (IVBs) reduce the risk of these complications. This study characterized patterns of IVB use in these patients in the USA. METHODS: This was a retrospective, observational study using the Truven Health MarketScan(®) Commercial and Medicare databases (2002-2011). Subjects with ≥1 claims of diagnosis of breast, lung, or prostate cancer (BC, LC, or PC) and ≥1 claims of BMets diagnosis were included. The date of first BMet diagnosis claim was the "index date." Key exclusion criteria were diagnosis of other primary cancer, receipt of IVB, or <6 months continuous enrollment pre-index. Cumulative incidence of treatment initiation, interruption, and discontinuation were estimated. Proportions of IVB claims with chemotherapy administered on the same day and with renal monitoring within 2 weeks prior were summarized. Multivariate regressions assessing factors associated with IVB initiation were conducted. RESULTS: Cumulative incidence of IVB initiation at 12 months post-index was greatest for BC followed by PC and LC, and it declined with age in all tumor types, e.g., in BC from 62 % at age <50 years to 47 % at age ≥75 years. At 12 months, IVB treatment interruption ranged from 16 % (LC) to 31 % (PC), with discontinuation ranging from 46 % (BC) to 83 % (LC). CONCLUSIONS: IVBs are used more frequently in patients with BMets secondary to BC than PC or LC. Many patients interrupt or discontinue IVB therapy within 12 months of initiation potentially impacting effectiveness.

Health-care utilization and costs with fluticasone propionate and fluticasone propionate/salmeterol in asthma patients at risk for exacerbations.

Although studies have established that adding long-acting beta agonists (LABA) to inhaled corticosteroid (ICS) monotherapy among patients with inadequately controlled asthma is associated with better outcomes than increasing ICS dosage, outcomes with ICS versus fixed-dose ICS/LABA combination among patients with recent asthma exacerbation or frequent use of rescue medication are unavailable. This study was designed to compare health-care utilization/costs among patients with recent asthma exacerbation or frequent rescue medication use who received fluticasone propionate (FP) alone versus fixed-dose FP/salmeterol combination (FSC). A retrospective cohort study was conducted using a large health insurance data set. Patients with one or more claims with asthma diagnosis, two or more prescriptions for FSC (250/50- or 100/50-mg formulations) or FP (220- or 110-mg formulations), and one or more asthma exacerbations or five or more short-acting beta agonist (SABA) prescriptions within 1 year before initial receipt of study medications were included. Health-care utilization/costs and controller therapy compliance were compared for patients receiving FSC versus FP using multivariate regression analysis controlling for FP dose and baseline characteristics. A total of 7779 patients met inclusion criteria (5769, FSC, and 2010, FP) with comparable mean follow-up (FSC, 685 days; FP, 670 days; p = 0.151). Controlling for FP dosage and baseline characteristics, FSC patients had lower risks of asthma-related exacerbations, fewer SABAs and systemic corticosteroids, higher costs of asthma medications and total asthma-related health care, and lower total asthma-related health-care costs excluding study medication cost. In asthma patients with recent exacerbation or frequent SABA use, receipt of FSC reduced asthma-related exacerbation risks and rescue medication use versus receipt of FP.

Retrospective Database Analysis of Liver-Related Clinical Events in Adult and Pediatric Patients with Alpha-1 Antitrypsin Deficiency in the United States.

Background and Aims: Real-world analyses on burden of illness in patients with alpha-1 antitrypsin deficiency (AATD) are limited. We investigated the real-world burden of liver-related clinical events among adult and pediatric patients with AATD in the USA. Methods: This was a retrospective, observational analysis of administrative claims data from the IQVIA PharMetrics® Plus and Ambulatory Electronic Medical Records databases from 2011 to 2022. Patients had a diagnosis of liver and/or lung disease with ≥180 days of continuous enrollment in the IQVIA PharMetrics Plus database before and ≥90 days after their first diagnosis. Follow-up time was assigned to the AATD with liver disease health state or AATD with both liver and lung disease health state (for patients aged ≥18 years only). Baseline demographic characteristics and liver-related clinical events of interest were reported. Results: Of 5136 eligible patients, 771 adult and 123 pediatric patients contributed time to the AATD with liver disease health state; 541 adults contributed time to the AATD with both liver and lung disease health state. Among adults, patients with both liver and lung disease had higher rates of liver-related clinical events than patients with liver disease alone. Ascites was the most frequently observed clinical event among adults in both health states, and the median time to the composite of any liver-related clinical event was 26.5 days among all adults combined. Across all pediatric age groups, ascites, gastrointestinal bleed and hepatic encephalopathy were more common than spontaneous bacterial peritonitis and hepatocellular carcinoma, but median time to liver-related clinical event varied by age group at index date and type of event. No liver transplantations occurred in patients aged 6-17 years. Conclusion: Diagnosed AATD with liver disease carries a substantial burden on adult and pediatric patients; new treatment options are warranted to avoid disease progression to decompensating events.

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

Aim: To evaluate all-cause and liver-associated healthcare resource utilization (HCRU) and costs among patients with alpha-1 antitrypsin deficiency (AATD) with liver disease (LD) and/or lung disease (LgD). Materials & methods: This was a retrospective analysis of linked administrative claims data from the IQVIA PharMetrics® Plus and the IQVIA Ambulatory Electronic Medical Records (AEMR) databases from 1 July 2021 to 31 January 2022. Patients with AATD in the IQVIA PharMetrics Plus database were included with ≥1 inpatient or ≥2 outpatient medical claims ≥90 days apart with a diagnosis of AATD, or with records indicating a protease inhibitor (Pi)*ZZ/Pi*MZ genotype in the IQVIA AEMR database with linkage to IQVIA PharMetrics Plus. For a patient's identified continuous enrollment period, patient time was assigned to health states based on the initial encounter with an LD/LgD diagnosis. A unique index date was defined for each health state, and HCRU and costs were calculated per person-year (PPY). Results: Overall, 5136 adult and pediatric patients from the IQVIA PharMetrics Plus and IQVIA AEMR databases were analyzed. All-cause and liver-associated HCRU and costs were substantially higher following onset of LD/LgD. All-cause cost PPY ranged from US $11,877 in the absence of either LD/LgD to US $74,015 in the presence of both LD and LgD. Among liver transplant recipients in the AATD with LD health state, liver-associated total costs PPY were US $87,329 1-year pre-transplantation and US $461,752 1-year post-transplantation. In the AATD with LgD and AATD with LD and LgD health states, patients who received augmentation therapy were associated with higher all-cause total costs PPY and lower liver-associated total costs PPY than their counterparts who did not receive augmentation therapy. Conclusion: Patients with AATD had increased HCRU and healthcare costs in the presence of LD and/or LgD. HCRU and healthcare costs were highest in the AATD with LD and LgD health state.

Risk of asthma exacerbation, asthma-related health care utilization and costs, and adherence to controller therapy in patients with asthma receiving fluticasone propionate/salmeterol inhalation powder 100 µg/50 µg versus mometasone furoate inhalation powder.

OBJECTIVE: National asthma treatment guidelines recommend low/medium-dose inhaled corticosteroids (ICSs) as initial therapy in mild asthma patients. However, low doses of a fixed-dose combination of ICS and long-acting ß-agonists are sometimes used. This study compares asthma-related outcomes and health care utilization and costs in clinical practice in patients starting fluticasone propionate 100 µg and salmeterol 50 µg via Diskus (FSC) or mometasone furoate (MF). METHODS: A retrospective cohort study was conducted to compare asthma-related outcomes in asthma patients who received FSC or MF, using a large health insurance claims dataset spanning January 2004-December 2008. Patients with ≥1 claim with an asthma ICD-9-CM diagnosis code and ≥2 FSC or MF prescriptions were included, stratified into FSC or MF groups by study drug received first and matched using propensity score. RESULTS: A total of 18,283 patients met inclusion criteria (14,044 FSC and 4239 MF); 3799 matched pairs were identified (mean follow-up: FSC 548 days, MF 537 days). FSC patients had lower risk of asthma-related exacerbation (Hazard ratio = 0.88, 95% CI 0.81-0.95, p = .002), defined as either asthma-related emergency department (ED) visits/hospitalizations or receipt of systemic corticosteroids (SCSs); fewer SCS claims (mean 0.28 vs. 0.33, p = .021); and fewer asthma-related physician office (PO) and hospital outpatient (HO) visits (mean 1.17 vs. 1.63, p < .001). However, asthma-related ED visits were higher with FSC (p = .004), and FSC patients had higher total costs of asthma-related health care ($953 vs. $862, p = .002). CONCLUSIONS: In asthma patients initiating ICS therapy, MF had lower asthma-related ED visits. However, FSC may reduce the use of SCS and asthma-related PO/HO visits.

Liver disease progression in patients with alpha-1 antitrypsin deficiency and protease inhibitor ZZ genotype with or without lung disease.

BACKGROUND: Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in SERPINA1, which encodes alpha-1 antitrypsin, a protease inhibitor (Pi). Individuals with AATD and the homozygous Pi*ZZ genotype have variable risk of progressive liver disease but the influence of comorbid lung disease is poorly understood. AIMS: To characterise patients with AATD Pi*ZZ and liver disease (AATD-LD-Pi*ZZ) with or without lung disease and describe liver disease-related clinical events longitudinally. METHODS: This was an observational cohort study of patients in the Mayo Clinic Healthcare System (January 2000-September 2021). Patients were identified using diagnosis codes and natural language processing. Fibrosis stage (F0-F4) was assessed using a hierarchical approach at baseline (90 days before or after the index date) and follow-up. Clinical events associated with liver disease progression were assessed. RESULTS: AATD-LD-Pi*ZZ patients with lung disease had a longer median time from AATD diagnosis to liver disease diagnosis versus those without lung disease (2.2 vs. 0.2 years, respectively). Compared to those without lung disease, patients with lung disease had a longer time to liver disease-related clinical events (8.5 years and not reached, respectively). AATD-LD-Pi*ZZ patients without lung disease were more likely to undergo liver transplantation compared with those with lung disease. CONCLUSION: In patients with AATD and lung disease, there is a delay in the diagnosis of comorbid liver disease. Our findings suggest that liver disease may progress more rapidly in patients without comorbid lung disease.

Liver disease epidemiology and burden in patients with alterations in plasma protein metabolism: German retrospective insurance claims analysis.

BACKGROUND: Alpha-1 antitrypsin deficiency is a rare genetic disease and a leading cause of inherited alterations in plasma protein metabolism (APPM). AIM: To understand the prevalence, burden and progression of liver disease in patients with APPM including alpha-1 antitrypsin deficiency. METHODS: We conducted a retrospective analysis of anonymized patient-level claims data from a German health insurance provider (AOK PLUS). The APPM cohort comprised patients with APPM (identified using the German Modification of the International Classification of Diseases-10th Revision [ICD-10-GM] code E88.0 between 01/01/2010-30/09/2020) and incident liver disease (ICD-10-GM codes K74, K70.2-3 and K71.7 between 01/01/2012-30/09/2020). The control cohort comprised patients without APPM but with incident liver disease. Outcomes were incidence/prevalence of liver disease in patients with APPM, demographics/baseline characteristics, diagnostic procedures, progression-free survival (PFS), disease progression and mortality. RESULTS: Overall, 2680 and 26299 patients were included in the APPM (fibrosis, 96; cirrhosis, 2584) and control (fibrosis, 1444; cirrhosis, 24855) cohorts, respectively. Per 100000 individuals, annual incidence and prevalence of APPM and liver disease was 10-15 and 36-51, respectively. In the APPM cohort, median survival was 4.7 years [95% confidence interval (CI): 3.5-7.0] and 2.5 years (95%CI: 2.3-2.8) in patients with fibrosis and cirrhosis, respectively. A higher proportion of patients in the APPM cohort experienced disease progression (92.0%) compared with the control cohort (67.2%). Median PFS was shorter in the APPM cohort (0.9 years, 95%CI: 0.7-1.1) compared with the control cohort (3.7 years, 95%CI: 3.6-3.8; P < 0.001). Patients with cirrhosis in the control cohort had longer event-free survival for ascites, hepatic encephalopathy, hepatic failure and esophageal/gastric varices than patients with cirrhosis in the APPM cohort (P < 0.001). Patients with fibrosis in the control cohort had longer event-free survival for ascites, cirrhosis, hepatic failure and esophageal/gastric varices than patients with fibrosis in the APPM cohort (P < 0.001). In the APPM cohort, the most common diagnostic procedures within 12 mo after the first diagnosis of liver disease were imaging procedures (66.3%) and laboratory tests (51.0%). CONCLUSION: Among patients with liver disease, those with APPM experience substantial burden and earlier liver disease progression than patients without APPM.

Retrospective comparison of early versus late treatment with fluticasone propionate/salmeterol after an asthma exacerbation.

BACKGROUND: The benefits of inhaled corticosteroids in asthma are well established. Early use of inhaled anti-inflammatories following and exacerbation could be beneficial. METHODS: A retrospective observational cohort study compared the risk of asthma-related exacerbations [hospitalization, emergency department visit, and/or treatment with systemic corticosteroid] in patients receiving treatment with fluticasone propionate/salmeterol in a single inhaler (FSC) within 90 days following an initial asthma-related exacerbation (early treatment) versus patients receiving the treatment subsequently (late treatment). Data were from a large health insurance claims database spanning from January 1998 to April 2008. Subjects included patients with ≥1 prescription for FSC ≤ 1 year after first asthma-related exacerbation. Patients with early treatment were matched to those with late treatment by propensity score and compared in terms of healthcare utilization and costs after initiation of FSC. RESULTS: A total of 14,861 patients met study inclusion criteria, including 10,793 early and 4068 late treatment patients. After matching, 3555 pairs were well matched on all pretreatment characteristics and duration of follow-up (mean 722 vs. 717 days, p = .634). Early versus late treatment was associated with longer time to first asthma-related exacerbation (hazard ratio = 0.82, 95% CI 0.75-0.88, p < .001), fewer short-acting ß-agonists prescriptions (3.3 vs. 3.6, p = .031), higher outpatient yearly per patient pharmacy costs ($1320 vs. $1163, p = .008), and lower yearly per patient asthma-related emergency department visit costs ($80 vs. $105, p = .032). Total yearly per patient asthma-related costs were similar ($2197 vs. $2064, p = .203). CONCLUSIONS: Earlier use of FSC following an asthma exacerbation was associated with reduced risk of future asthma-related exacerbation and lower use of rescue medications.

Economic burden of disease progression among multiple myeloma patients who have received transplant and at least one line of therapy in the US.

Effects of disease progression on healthcare resource utilization (HRU) and costs among multiple myeloma (MM) patients with ≥1 line of therapy (LOT) who received their first stem cell transplant (SCT) within 1 year of initial MM diagnosis were estimated using a large US claims database. Disease progression was defined as advancement to the next LOT, bone metastasis, hypercalcemia, soft tissue plasmacytoma, skeletal related events, acute kidney disease, or death within 12 months of LOT initiation. Annual HRU and costs in the first three LOTs (L1-L3) were compared for patients with versus without disease progression using inverse probability of treatment weighting to adjust for differences between groups in baseline characteristics. In all LOTs, mean annual hospitalizations and healthcare costs were greater for patients with versus without progression. Total incremental annual costs among patients with versus without progression in L1-L3 were $18,359, $87,055, and $71,917, respectively, among LOTs initiated between 2006 and 2018. In LOTs initiated between 2013 and 2018, the figures were $46,024, $100,329, and $101,942 in L1-L3, respectively. The economic burden of disease progression is substantial in this population of MM patients who underwent SCT and received systemic anti-myeloma therapy.

Lifetime Costs for Treated Follicular Lymphoma Patients in the US.

BACKGROUND AND OBJECTIVE: The objective of this study was to estimate the lifetime costs of patients receiving treatment for follicular lymphoma (FL) in the United States. METHODS: A Markov model was programmed in heRo3 with a 6-month cycle length, 35-year time horizon (lifetime projection), and health states for line of treatment, response, receipt of maintenance therapy among responders, transformation to diffuse large B-cell lymphoma (DLBCL), development of second primary malignancy (SPM), and death. The model was used to estimate the expected lifetime costs of FL (in 2019 USD), including costs of drug acquisition and administration, transplant procedures, radiotherapy, adverse events, follow-up, DLBCL, SPM, end-of-life care, and indirect costs. Model inputs were based on published sources. RESULTS: In the US, patients with FL receiving treatment have a life expectancy of approximately 14.5 years from initiation of treatment and expected lifetime direct and indirect costs of US$515,884. Costs of drugs for induction therapy represent the largest expenditure (US$233,174), followed by maintenance therapy costs (US$88,971) and terminal care costs (US$57,065). Despite the relatively advanced age of these patients, indirect costs (due to patient morbidity and mortality and caregiver lost work time) represent a substantial share of total costs (US$40,280). Treated FL patients spend approximately 6.9 years in the health states associated with first-line therapy. Approximately 66 and 46% continue to second- and third-line therapies, respectively. The mean (95% credible interval) of expected lifetime costs based on the probabilistic sensitivity analyses was US$559,202 (421,997-762,553). CONCLUSIONS: In the US, the expected lifetime costs of care for FL patients who receive treatment is high. The results highlight the potential economic benefits that might be achieved by treatments for FL that prevent or delay disease progression.

Adding salmeterol to fluticasone propionate or increasing the dose of fluticasone propionate in patients with asthma.

The National Asthma Education and Prevention Program guidelines recommend two options for patients uncontrolled on inhaled corticosteroid (ICS) alone: add a long-acting bronchodilator or increase the dose of the ICS. The purpose of this study was to compare asthma-related exacerbations and asthma control in asthma patients receiving fluticasone propionate (FP) monotherapy with an increased dose of FP compared with maintaining the dose and adding salmeterol (SAL) via a single device (FP/SAL combination [FSC]). A retrospective observational study was performed using health insurance claims spanning from January 2001 to August 2006 ("study period"). Subjects were > or =12 years of age, with asthma (International Classification of Diseases [ICD] 493.xx), and were stepped up from FP 44 microg to either FP 110 microg (FP110) or FP 100 microg/SAL 50 microg (FSC), or from FP110 to either FP 220 microg or FP 250 microg/SAL 50 microg (FSC). There were 1744 subjects identified, 557 (32%) increased FP and 1187 (68%) added SAL. The cohorts were relatively similar, and after adjusting for baseline characteristics, patients who added SAL to their same dose of FP had 41% lower odds of an asthma exacerbation (odds ratio = 0.59; 95% confidence interval [CI] = 0.46-0.76; p < 0.001), 36% fewer prescriptions for a short-acting beta-agonist (rate ratio = 0.64; 95% CI = 0.58-0.70; p < 0.001), and a 32% increase in ICS refill persistence compared with increasing the dose of FP. In asthma patients who are not adequately controlled with ICS (FP), adding SAL as FSC is associated with lower risk of an asthma-related exacerbation and better asthma control compared with increasing the dose of ICS (FP).

Stepping down to fluticasone propionate or a lower dose of fluticasone propionate/salmeterol combination in asthma patients recently initiating combination therapy.

Clinical guidelines recommend add-on therapy with long-acting beta2-agonists (LABA) in patients with mild-to-moderate persistent asthma whose disease is not adequately controlled with inhaled corticosteroids (ICSs) alone. For those achieving control with add-on therapy, careful reduction in ICS dose followed by withdrawal of LABA is recommended. This study was designed to compare asthma-related outcomes in patients receiving fluticasone propionate/salmeterol combination (FSC) who stepped down to a lower dose of FSC versus those who stepped down to fluticasone propionate (FP) at the same dose of FP. A retrospective observational cohort study was performed using two large health insurance claims databases spanning from January 2000 to June 2007. Subjects were age > or =12 and <65 years, had a diagnosis of asthma (International Classification of Diseases [ICD-493.xx]), and who within 1 year of initiating FSC either stepped down to a lower dose of FSC ("FSC patients") or to FP only at the same dose of FP ("FP patients"). FSC and FP patients were matched based on propensity scores to control for potential differences in baseline demographic and clinical characteristics and preindex asthma-related and costs. Of 4350 subjects identified, 3881 stepped down to a lower dose of FSC and 469 stepped down to FP. After matching, there were 447 pairs of FSC and FP patients. FSC patients had 30% fewer prescriptions for short-acting beta-agonists, a 26% lower risk of receiving systemic corticosteroids, and a 48% lower risk of asthma-related hospitalization or Emergency Department visit during follow-up. Stepping down to FP monotherapy is associated with worsening asthma symptoms and greater risk of severe asthma-related exacerbations compared with staying on FSC at a lower ICS dose.

Burden of disease progression in patients with multiple myeloma in the US.

Effects of disease progression on healthcare resource utilization (HRU) and costs among multiple myeloma (MM) patients with ≥1 line of therapy (LOT) and without receipt of stem cell transplant were estimated using large US claims database. Disease progression was defined as advancement to the next LOT, bone metastasis, hypercalcemia, soft tissue plasmacytoma, skeletal related events, acute kidney failure, or death within 12 months of LOT initiation. Annual HRU and costs in the first four LOTs were compared for patients with versus without progression using inverse probability of treatment weighting to adjust for differences between groups in baseline characteristics. In all LOTs, mean annual hospitalizations and healthcare costs were greater for patients with versus without progression. Total incremental annual costs among patients with versus without progression in 1LOT to 4LOT were $25,920, $30,632, $47,320, and $19,769, respectively. For MM patients receiving drug therapy, the economic burden of disease progression is substantial.

Healthcare utilization and costs among relapsed or refractory multiple myeloma patients on carfilzomib or pomalidomide as monotherapy or in combination with dexamethasone.

Aim: To compare monthly healthcare resource utilization (HRU) and costs among adult patients with multiple myeloma (MM) receiving second or subsequent line of treatment (LOT) with carfilzomib or pomalidomide as monotherapy or in combination with dexamethasone. Methods and materials: Adult MM patients who received carfilzomib or pomalidomide as second/subsequent LOT between 2006 and 2014 were selected from the MarketScan databases. LOT was determined using Medical/pharmacy claims using a published algorithm. For each patient, first LOT with carfilzomib or pomalidomide was defined as index LOT. Patients with first LOT as index LOT, who received other chemotherapy in combination with carfilzomib or pomalidomide, or who underwent stem cell transplant (STC) during index LOT were excluded. Monthly HRU and costs during index LOT were compared using inverse probability of treatment weights (IPTW) based on propensity scores for receipt of carfilzomib estimated by logistic regression with LOT, patient demographics, Charlson index, comorbidities, pre-index healthcare cost, and receipt of prior SCT as covariates. Results: After weighting, baseline characteristics were well balanced among 114 carfilzomib and 144 pomalidomide patients. Mean (95% CI) numbers of outpatient visits per month were 7.1 (5.2-8.0) with carfilzomib and 4.7 (3.9-6.1) with pomalidomide (p = 0.006). Otherwise, there were no statistically significant differences between the groups in mean monthly HRU and costs or median time to therapy discontinuation. Mean (95% CI) monthly total healthcare costs were $19,776 (15,322-27,748) with pomalidomide and $17,321 (12,412-21,874) with carfilzomib (p = 0.522). Limitations: Comparison of carfilzomib vs pomalidomide may be biased if there are unobserved factors not balanced by IPTW. The relatively small sample size limits the power of analyses to detect potential differences between treatment groups. Conclusions: Monthly HRU and costs are similar among patients with relapse or refractory MM patients receiving carfilzomib or pomalidomide as monotherapy or in combination with dexamethasone.

Effects of fluticasone propionate/salmeterol combination on asthma-related health care resource utilization and costs and adherence in children and adults with asthma.

BACKGROUND: Clinical trials suggest that in patients with asthma inadequately controlled on low- to medium- dose inhaled corticosteroids (ICSs), the addition of a long-acting beta-agonist such as salmeterol (SAL is more effective than the addition of montelukast (MON) or a higher-dose ICS. OBJECTIVE: This study was designed to expand on these earlier findings by comparing asthma-related health care resource utilization and costs, as well as adherence to ICSs, in children and adults with asthma receiving ICS monotherapy who either were switched to fluticasone propionate plus SAL from a single inhaler (FSC) or initiated add-on therapy with SAL from a separate inhaler or MON. METHODS: This retrospective study used an integrated managed-care database from >30 health plans. Patients were >or=5 years of age with a diagnosis of asthma (International Classification of Diseases, Ninth Revision, Clinical Modification 493.xx) and >or=2 claims for FSC, SAL, or MON. The date of first claim for the medication of interest was the index date. Patients were also required to have >or=1 claim for an ICS during the 12 months preindex and 12 months postindex. Utilization and costs of asthma-related care and adherence to ICS treatment postindex were compared using multivariate methods. RESULTS: After adjusting for preindex characteristics, patients receiving FSC (n=1287) had fewer claims for short-acting beta-agonists, oral corticosteroids, and lower adjusted asthma-related costs postindex compared with ICS + SAL (n=562) and ICS + MON (n=420). FSC patients also had greater adherence to ICS therapy. Those who received FSC had lower risks for treatment failure (defined as asthma-related emergency department visits or hospitalization or receipt of alternative study medication or oral corticosteroids during the postindex period). CONCLUSION: In this health insurance claims-based study of patients with asthma inadequately controlled with an ICS alone, those who received stepped-up therapy with FSC used fewer rescue medications and had greater persistence with ICSs compared with those in whom SAL or MON was added to ICS monotherapy.

Outcomes, utilization, and costs among thalassemia and sickle cell disease patients receiving deferoxamine therapy in the United States.

Deferoxamine mesylate (DFO) reduces morbidity and mortality associated with transfusional iron overload. Data on the utilization and costs of care among U.S. patients receiving DFO in typical clinical practice are limited however. This was a retrospective study using a large U.S. health insurance claims database spanning 1/97-12/04 and representing 40 million members in >70 health plans. Study subjects (n = 145 total, 106 sickle cell disease [SCD], 39 thalassemia) included members with a diagnosis of thalassemia or SCD, one or more transfusions (whole blood or red blood cells), and one or more claims for DFO. Mean transfusion episodes were 12 per year. Estimated mean DFO use was 307 g/year. Central venous access devices were required by 20% of patients. Cardiac disease was observed in 16% of patients. Mean total medical costs were $59,233 per year including $10,899 for DFO and $8,722 for administration of chelation therapy. In multivariate analyses, potential complications of iron overload were associated with significantly higher medical care costs. In typical clinical practice, use of DFO in patients with thalassemia and SCD receiving transfusions is low. Administration costs represent a large proportion of the cost of chelation therapy. Potential complications of iron overload are associated with increased costs.

Healthcare resource utilization and costs in patients with newly diagnosed acute myeloid leukemia.

AIM: Acute myeloid leukemia (AML) is associated with high disease burden. This analysis estimated HRU and costs among newly diagnosed AML patients in a US commercially insured population. MATERIALS AND METHODS: This was a retrospective observational study using the IMS Health PharMetrics Plus and Hospital Charge Detail Master databases. Patients included adults who were newly diagnosed with AML between January 2007 and June 2016 ("study period"). Patients with <12 months of continuous enrollment prior to the index date were excluded, as were those whose first diagnosis was AML in remission/relapse, those diagnosed with acute promyelocytic leukemia, those on Medicare supplemental insurance, or those with a diagnosis of AML in remission/relapse without evidence of treatment during the study period. Patients were stratified by receipt of AML treatment (chemotherapy/hematopoietic cell transplantation [HCT]), and their follow-up was partitioned into initial, remission, and relapsed health states. Mean HRU and costs were tallied by treatment and, for treated patients, by health state and time since entry into health state (≤6 vs >6 months). RESULTS: A total of 9,455 patients met study criteria, including 6,415 (68%) treated and 3,040 (32%) untreated patients, with mean follow-up of 18.3 and 16.4 months, respectively. Mean age was 55 years in treated patients and 60 years in untreated patients. Mean total costs per patient were $386,077 in treated patients and $79,382 in untreated patients. For treated patients, 60% of total costs ($231,867 per patient) were incurred during the initial health state, representing time without remission/relapse. Mean monthly total healthcare costs were $21,055 and $4,854 among treated and untreated patients, respectively. LIMITATIONS AND CONCLUSIONS: HRU and costs of managing AML patients are substantial. In treated patients, the majority of costs were incurred during the initial treatment period, without claims indicating remission/relapse.

Cost-effectiveness of blinatumomab versus salvage chemotherapy in relapsed or refractory Philadelphia-chromosome-negative B-precursor acute lymphoblastic leukemia from a US payer perspective.

OBJECTIVE: To evaluate the cost-effectiveness of blinatumomab (Blincyto) vs standard of care (SOC) chemotherapy in adults with relapsed or refractory (R/R) Philadelphia-chromosome-negative (Ph-) B-precursor acute lymphoblastic leukemia (ALL) based on the results of the phase 3 TOWER study from a US healthcare payer perspective. METHODS: The Blincyto Global Economic Model (B-GEM), a partitioned survival model, was used to estimate the incremental cost-effectiveness ratio (ICER) of blinatumomab vs SOC. Response rates, event-free survival (EFS), overall survival (OS), numbers of cycles of blinatumomab and SOC, and transplant rates were estimated from TOWER. EFS and OS were estimated by fitting parametric survival distributions to failure-time data from TOWER. Utility values were based on EORTC-8D derived from EORTC QLQ-C30 assessments in TOWER. A 50-year lifetime horizon and US payer perspective were employed. Costs and outcomes were discounted at 3% per year. RESULTS: The B-GEM projected blinatumomab to yield 1.92 additional life years and 1.64 additional quality-adjusted life years (QALYs) compared with SOC at an incremental cost of $180,642. The ICER for blinatumomab vs SOC was estimated to be $110,108/QALY gained in the base case. Cost-effectiveness was sensitive to the number and cost of inpatient days for administration of blinatumomab and SOC, and was more favorable in the sub-group of patients who had received no prior salvage therapy. At an ICER threshold of $150,000/QALY gained, the probability that blinatumomab is cost-effective was estimated to be 74%. LIMITATIONS: The study does not explicitly consider the impact of adverse events of the treatment; no adjustments for long-term transplant rates were made. CONCLUSIONS: Compared with SOC, blinatumomab is a cost-effective treatment option for adults with R/R Ph - B-precursor ALL from the US healthcare perspective at an ICER threshold of $150,000 per QALY gained. The value of blinatumomab is derived from its incremental survival and health-related quality-of-life (HRQoL) benefit over SOC.