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Atrial fibrillation (AF) is a recognized adverse consequence associated with all Bruton's tyrosine kinase inhibitors used to treat chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL); however, real-world time to discontinuation (TTD) and time to next treatment (TTNT) of CLL/SLL patients with a high baseline AF/stroke risk remain unknown.Patients with CLL/SLL from a nationwide electronic health record-derived database (February 12, 2013-January 31, 2021) initiating first-line (1L) or second or later-line (2L+) treatment with ibrutinib or other regimens on or after February 12, 2014 (index date) were analyzed. Kaplan-Meier survival analysis was used to assess TTD and TTNT among all patients, patients with high AF risk (CHARGE-AF risk score ≥10.0%), and patients at high risk of stroke (CHAIn 1L/2L+, 2190/1851 patients received ibrutinib and 4388/4135, were treated with other regimens. Median TTD for ibrutinib was similar regardless of AF/stroke-related risk (1L: all patients, 15.7 months; high AF risk, 11.7 months; high stroke risk, 13.7 months; similar results in 2L+). Median TTNT was significantly longer for ibrutinib vs. other regimens (1L: not reached vs. 45.9 months; 2L+: not reached vs. 23.6 months; both P.05), including among those with high AF/stroke risk. TTNT was similar between all patients and high-risk cohorts in 1L and 2L+ (all P.05).This study highlights that elevated baseline AF/stroke-related risk does not adversely impact TTD and TTNT outcomes associated with ibrutinib use. Additionally, TTNT was significantly longer for patients treated with ibrutinib vs. other regimens.

Pulmonary arterial hypertension (PAH), a rare vasculopathy progressively leading to right heart failure and death, is associated with considerable economic burden. Oral prostacyclin pathway agents (PPAs) like selexipag and treprostinil address an underlying PAH pathway, yet are often under-utilized. Data on head-to-head cost comparison of various PPAs is lacking. In this retrospective study using a large health claims database, we compared the per-patient-per-year (PPPY) costs and healthcare resource utilization (HRU) among PAH patients taking either oral selexipag, inhaled treprostinil or oral treprostinil in the United States between July 2015 and March 2020. Patients with ≥1 prescription for one of the drugs of interest, ≥1 in-patient pulmonary hypertension (PH) diagnosis, or ≥ 2 outpatient PH diagnoses were included in this study. Baseline differences between the three groups were adjusted using an inverse probability of treatment weighting approach. 411 patients were selected for the final study cohorts. All-cause hospitalization costs were highest for oral treprostinil ($39,983) compared to oral selexipag ($20,635) and inhaled treprostinil ($16,548; p = .037). Total PAH-related medical costs were 40% lower for patients on oral selexipag compared to patients on oral and inhaled treprostinil ($24,351 vs. $40,398 and $40,339, respectively; p = .006). PAH-related outpatient visits were lowest for patients on oral selexipag (14 PPPY visits) compared to oral treprostinil (16 PPPY visits) and inhaled treprostinil (22 PPPY visits; p = .001). Compared to oral and inhaled treprostinil, oral selexipag may incur lower medical costs and reduce PAH related outpatient visits for patients with PAH.

Introduction Hospitalization is an important clinical factor associated with survival and rehospitalization in patients with pulmonary arterial hypertension (PAH). Thus, this study examined treatment patterns before and after hospitalization in the US-specific population. Methods Adult PAH patients in the United States were identified using the Optum® Clinformatics® database from January 1, 2014, to June 30, 2019, and were required to have continuous health plan enrollment for at least 6 months prior to the first (index) hospitalization through at least 90 days post-discharge. Baseline patient characteristics were evaluated from 6 months prior to through the index hospitalization. PAH treatment patterns were examined from 30 days pre-index admission (pre-hospitalization) and 90 days post-index hospital discharge (post-hospitalization), and stratified by therapy type: monotherapy, double- or triple-combination therapy, or no PAH therapy. Results A total of 3116 hospitalized patients with PAH met selection criteria. The mean age and Charlson comorbidity index score were 68.1 years and 5.1, respectively. In the pre- and post-hospitalization periods (all-cause), respectively, patients prescribed monotherapy were most common (from 64.8% pre- to 51.9% post-hospitalization), followed by patients with no evidence of PAH therapy (from 14.6 to 28.5%). Among PAH-related hospitalizations, patients with monotherapy were also most common (from 60.8% pre- to 49.1% post-hospitalization), followed by patients with no evidence of PAH therapy (from 10.0 to 22.8%). The majority of patients with all-cause hospitalizations (72.8%) had no therapy modification; 20.0% de-escalated therapy (including 15.0% from monotherapy to no therapy) and 6.1% escalated therapy (including 2.2% from no therapy to monotherapy and 3.2% from monotherapy to double or triple therapy). Conclusion Inpatient admissions did not appear to drive changes in PAH therapy management, as monotherapy predominated, and most patients had no therapy modification within 90 days of a hospitalization. These results warrant future research to understand the reasons behind the limited treatment intensification observed and the impact of post-hospitalization optimization on clinical and economic outcomes. Supplementary Information The online version contains supplementary material available at 10.1007/s41030-021-00173-6.

Introduction Acute myeloid leukemia (AML) is associated with lower survival and greater unmet need compared with some other hematologic malignancies (HMs). Despite differences in acuteness between AML and other HMs, the burden of family caregivers (FCs) of patients with these malignancies offer similar patient experiences. A targeted literature review was conducted to explore FC burden of patients with AML and HM with and without hematopoietic stem cell transplant (HSCT). Instruments to measure and interventions to address FC burden were identified. Methods Studies on economic burden and compromised health-related quality of life (HRQoL) associated with FC burden, family affairs, and childcare from 1 January 2010 to 30 June 2019 were identified through database and hand searches. Published English articles on randomized controlled trials or standardized qualitative or quantitative observational studies were included. FCs were those in close familial proximity to the patient (i.e., spouse, parents, children, relatives, other family members, significant others). Results Seventy-one publications were identified (AML, n = 3; HM, n = 29; HSCT, n = 39). Predominant burden categories included humanistic (n = 33), economic (n = 17), and interventions (n = 22); one study was classified as humanistic and economic. FCs lack sufficient resources to manage stressors and experience negative psychological, behavioral, and physiological effects. FCs of patients with HMs reported post-traumatic stress disorder, significant sleep problems, moderate-to-poor HRQoL, and negative impacts on family relationships. Instruments designed to measure caregiver burden were generic and symptom-specific. Educational, expressional, and self-adjustment interventions were used to improve FC burden. Conclusion Findings indicate a need for additional research, public health approaches to support FCs, and effective interventions to address FC burden. Minimizing FC burden and improving quality of life may reduce the overall healthcare service use and allow FCs to more effectively fulfill caregiver tasks. Support systems to alleviate caregiver burden may create reinforced integrators, thus positively affecting quality of life and possibly the outcomes of patients. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-021-01872-x.

OBJECTIVE Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare disease that often follows pulmonary embolism (PE). Screening for CTEPH is challenging, often delaying diagnosis and worsening prognosis. Predictive risk models for CTEPH could help identify at-risk patients, but existing models require multiple clinical inputs. We developed and validated a predictive risk model for CTEPH using health insurance claims that can be used by payers/quality-of-care organizations to screen patients post-PE. METHODS Adult patients newly diagnosed with acute PE (index date) were identified from the Optum De-identified Clinformatics Extended DataMart (January 2007-March 2018; development set) and IBM MarketScan (January 2008-June 2019; validation set) databases. Predictors were identified 12 months before or on the index PE. Risk of "likely CTEPH" was assessed post-PE based on CTEPH-related diagnoses and procedures since the CTEPH diagnosis code (ICD-10-CM: I27.24) was not available until 1 October 2017. Stepwise variable selection was used to build the model using the development set; model validation was subsequently conducted using the validation set. RESULTS The development set included 93,428 patients, of whom 11,878 (12.7%) developed likely CTEPH. Older age (odds ratios [OR] = 1.16-1.49), female (OR = 1.09), unprovoked PE (i.e. without thrombotic factors; OR = 1.14), hypertension (OR = 1.07), osteoarthritis (OR = 1.08), diabetes (OR = 1.07), chronic obstructive pulmonary disease (OR = 1.11), obesity (OR = 1.21) were associated with higher odds of likely CTEPH, and oral anticoagulants with lower odds (OR= 0.50, all p

AimTo compare healthcare resource use and costs between newly diagnosed multiple myeloma (NDMM) patients with and without skeletal-related events (SREs).MethodsAdults newly diagnosed with MM (1 January 2006 and 30 June 2017) with at least 12 months continuous health coverage prior to diagnosis were identified using the IBM MarketScan administrative claims. To control for baseline differences, NDMM patients with SREs were propensity score matched to NDMM patients without SREs. Outcomes included annual HRU and costs during follow-up along with number and type of SREs (SRE cohort only). Patients with SREs were stratified by number of SREs, and annual SRE-related costs were reported. Student's t test and Chi-squared test were used to compare outcomes.ResultsBefore matching, the 6648 patients in the SRE cohort had more comorbidities, were more likely to have MM treatment, and had higher pre-index healthcare costs than the 7458 patients in the non-SRE cohort. After matching, cohorts of 3432 patients were well balanced on baseline characteristics. Patients with SREs (vs. without SREs) had significantly higher inpatient, outpatient, and pharmacy HRU. Patients with SREs had significantly higher mean annual all-cause healthcare costs ($213,361 vs. $94,896, p ConclusionsNDMM patients with SREs have more than twice the all-cause healthcare costs than matched patients without SREs. Costs increase with the number of SRE events.

Objectives Thresholds for the minimally important difference (MID) or responder definition (RD) in health‐related quality‐of‐life (HRQoL) scores are required to interpret the impact of an intervention or change in the trajectory of the condition which is meaningful to patients. This study aimed to establish MID and RD for the European Organisation for Research and Treatment of Cancer Quality of Life Multiple Myeloma questionnaire (EORTC QLQ‐MY20). Methods A novel mixed‐methods approach was applied by utilizing both existing clinical trial data and prospective patient interviews. Anchor‐based, distribution‐based, and qualitative‐based estimates of meaningful change were triangulated to form recommended RDs for each scale of the EORTC QLQ‐MY20. Anchor‐based MIDs were summarized using weighted correlation. Results Recommended MIDs were as follows: Disease Symptoms (DS 10 points), Side Effects of Treatment (SE 10 points), Body Image (BI 13 points), and Future Perspective (FP 9 points). Recommended RDs were as follows: DS (16 improvement; 11 worsening), SE (6 improvement; 9 worsening), BI (33 improvement; 33 worsening), and FP (11 improvement; 11 worsening). Conclusions The study generated estimates of the MID and RD for each scale of the EORTC QLQ‐MY20. Published estimates will enable investigators and clinicians to adopt these as standard for interpretation and for hypothesis testing. Consequently, analyses from trials of different interventions can be more comparable.

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 carfilzom...

We compared patient-reported outcomes (PROs) with once-weekly carfilzomib 70 mg/m2 (Kd70 mg/m2) vs. twice-weekly carfilzomib 27 mg/m2 (Kd27 mg/m2) plus dexamethasone in relapsed or refractory multiple myeloma (RRMM). Patient-reported convenience/satisfaction collected at Cycle 2, Day 1 was compared between groups using logistic regression. European Organization for Research and Treatment of Cancer QOL Questionnaire (QLQ-C30), MM-module (QLQ-MY20), and EuroQoL-5 Dimensions-5 Levels (EQ-5D-5L) questionnaires were administered at baseline, then every other cycle. PROs were compared between groups using mixed models for repeated measures. Times from randomization to first deterioration (TTD) in scores were analyzed using Cox regression. PRO analyses included 469 patients. Once-weekly Kd70 mg/m2 patients reported greater convenience (odds ratio [OR], 4.98; p

Background: Patients with multiple myeloma (MM) generally progress to being relapse/refractory (RR), having received several lines of treatment with various dosing frequencies and administration modalities, which impact patient satisfaction and experience. While there is still no cure, patients live longer but with potential burden attributable to treatment. A fit-for-purpose patient-reported outcome measure (PROM) assessing patient satisfaction and experience is missing for RRMM.Methods: We developed the Satisfaction and Experience Questionnaire (SEQ) for MM to measure patient satisfaction and experience, based on concepts identified from a literature review and nurse/patient perceptions about patient experiences. Online focus groups were conducted with oncology nurses who treat patients with MM to provide expert opinion, followed by concept elicitation and cognitive patient interviews. A preliminary SEQ-MM measure was first drafted based on desk research. Patient feedback from the cognitive interviews resulted in revisions to the preliminary SEQ-MM’s instructions, item wording, and response options between rounds. Content analysis was conducted to assess concept frequency mentioned during the concept elicitation focus group, and an item tracking table documented the SEQ-MM revisions based on cognitive interviews.Results: Three oncology nurses and 10 patients with RRMM participated in the focus groups. Oncology nurses reported treating 17 patients with MM per week, of which 33% were patients with RRMM. Study patients were primarily male (70%), with mean age of 56 (median range: 47 to 71). The most commonly reported satisfaction and experience concepts were related to: satisfaction with treatment (50%), experience with treatment (50%), relationship with healthcare practitioners (50%), time burden (50%), side effects (40%), and avoiding relapse (40%). The SEQ-MM was revised to include 15 items capturing: treatment burden (five items), time burden (five items), travel burden (two items), treatment compliance (one item), general satisfaction (one item), and an overall satisfaction item (one item).Conclusions: The SEQ-MM v1.0 evaluates the important facets of satisfaction and experience in patients with RRMM receiving treatment. This tool can be used to assess facets of treatment in MM that might complement health-related quality of life data Additional work is needed to examine item performance, scoring, and psychometric properties.

e18559 Background: Real-world data on practice patterns in Black pts with CLL are limited. We evaluated treatment (tx) patterns and prognostic biomarker testing in Black pts enrolled in the informCLL registry. Methods: Pts who initiated FDA-approved tx for CLL/SLL (index tx) were enrolled from October 2015 to June 2019. Baseline (BL) characteristics, FISH testing rates, BL factors associated with FISH testing, and patterns of index tx are summarized by line of therapy (LOT; previously untreated [1L] and relapsed/refractory [R/R]). Results: Of 1462 enrolled pts (overall registry population), 106 (7%) were Black (Table). Community-based practices enrolled 87% of Black pts, similar to the overall population (93%). Black pts were predominantly enrolled in the South (69%). Among Black pts, median age was 66 y, 65% had ECOG status ≥1, 64% had Rai stage III-IV, and median time from diagnosis to tx on registry was 40 mo (7 mo for 1L). In the overall population, median age was 71 y, 53% had ECOG status ≥1, 51% had Rai stage III-IV, and median time from diagnosis to tx was 41 mo (19 mo for 1L). FISH testing was performed in 25% of Black pts, similar to the testing rate in the overall population (28%). In a multivariate analysis, BL factors significantly associated with FISH testing were shorter time from initial diagnosis to tx, better ECOG status, earlier LOT (1L), community practice setting, and prior malignancy (p

e19057 Background: Ibrutinib is an oral, once daily Bruton’s tyrosine kinase inhibitor and is the only targeted treatment that has demonstrated progression-free survival and overall survival in multiple phase 3 trials across age, fitness, and high-risk markers for CLL/SLL. This study examined the trends in genomic testing and compared outcomes of first line (1L) CLL patients treated with ibrutinib or CIT in clinical practice. Methods: The nationwide Flatiron Electronic Health Record-derived de-identified database (03/04/2016-08/31/2021) was used to select patients with CLL/SLL who had ≥2 clinic visits and started 1L therapy with ibrutinib or CIT. Patient baseline characteristics prior to 1L initiation were compared between the two treatment groups. Propensity score-based inverse probability of treatment weighting (IPTW) was used to adjust for baseline differences between treatment groups. Kaplan-Meier (KM) analysis was used to evaluate time to next treatment (TTNT) from 1L initiation. Two subgroup analyses were performed among patients with high-risk cytogenetic markers (deletion 17p, 11q or unmutated IGHV) and those without IGHV testing. Results: Overall, the rate of testing for high-risk markers remain suboptimal, while cytogenetic testing for del17p or del11q improved from 52.3% to 74.3%, coinciding with approval of ibrutinib for del17p in 2014, IGHV testing remains infrequent and low at 37.7%. 1882 patients (61.4%) received 1L ibrutinib monotherapy (mean age 71, median follow-up 26 months), and 1182 (38.6%) received a CIT regimen (mean age 68, median follow-up 33 months). More patients treated with ibrutinib had a known high-risk cytogenetic marker (44% vs. 34%), especially in those with deletion 17p (15% vs. 4%), than those treated with CIT. Ibrutinib-treated patients significantly decreased risk of initiating a subsequent treatment by 51% (Table), the high-risk group by 60%, and those with unknown IGHV status by 49%. Conclusions: Consistent with clinical trial evidence, treatment with ibrutinib among 1L CLL patients resulted in a statistically significantly lower risk of initiating subsequent treatment than those treated with CIT, both overall and in the subgroups of patients with high-risk cytogenetic markers and patients without IGHV testing. Assessment of cytogenetic/molecular risk status for appropriate treatment is vital to optimize clinical outcomes, especially in the novel agent era. Recent IGHV testing in community practice to assess genetic risk in CLL remains suboptimal.[Table: see text]

e19056 Background: Ibrutinib is an oral, once daily Bruton’s tyrosine kinase inhibitor and is a targeted treatment that has demonstrated progression-free survival and overall survival benefits in multiple phase 3 trials across age, fitness, and high-risk markers for CLL/SLL. Despite ibrutinib established as a preferred first-line (1L) treatment option, anti-CD20 monotherapy (rituximab or obinutuzumab) has been prescribed as symptomatic or palliative therapy. This retrospective study compared outcomes between ibrutinib and anti-CD20 monotherapy for CLL/SLL patients who initiated a 1L of therapy in community oncology practice. Methods: The nationwide Flatiron Health Electronic Health Record-derived de-identified database (03/04/2016-08/31/2021) was used to select patients with CLL/SLL who had ≥2 clinic visits and started 1L therapy with ibrutinib or anti-CD20 monotherapy. Patient baseline characteristics prior to 1L initiation were compared between the two treatment groups. Propensity score-based inverse probability of treatment weighting (IPTW) was used to adjust for baseline differences between treatment groups. Kaplan-Meier (KM) analysis was used to evaluate time to next treatment (TTNT) from 1L initiation. Two subgroup analyses were performed among patients with high-risk cytogenetic markers (deletion 17p or 11q, or unmutated IGHV) and those without IGHV testing. Results: Overall, 3,226 patients were included in the analysis, of whom 2,188 (67.8%) received ibrutinib and 1,038 (32.2%) received an anti-CD20 antibody. The average age was 71.4 years for ibrutinib patients and 72.9 years among anti-CD20 users. Patients treated with ibrutinib were more likely to have high-risk cytogenetic markers (42.6% vs 27.9%). Ibrutinib-treated patients overall had a significant 70% risk reduction of initiating a subsequent treatment compared with anti-CD20 group (Table). Similar results were also found in subgroups of patients with high-risk and those without IGHV testing. Conclusions: IPTW-adjusted analysis showed that in the real-world community practice setting, treatment with ibrutinib resulted in a statistically significantly lower risk of initiating subsequent treatment than the anti-CD20 group including patients with high cytogenetic risk features and without IGHV testing.[Table: see text]

Background Relapsed and refractory multiple myeloma (RRMM) is a bone marrow cancer that requires systemic treatment, which often results in severe symptom burden. Recent studies have found that electronic patient-reported outcome (ePRO) interventions implemented in the clinic setting have had positive outcomes for other oncology populations. Evidence of the efficacy of a similar approach is lacking for patients with RRMM. Objective Recent recommendations for digital health interventions call for the publication of descriptions of iterative development processes in order to improve reproducibility and comparability. This study is an implementation pilot aiming to evaluate the acceptability and appropriateness of an ePRO intervention for patients with RRMM and to explore its impact on clinic workflow. Methods A total of 11 patients with RRMM were recruited from the John Theurer Cancer Center in Hackensack, New Jersey. Patients used a mobile app to report on 17 symptoms at 4 sessions, each a week apart. Patients could also report symptoms ad hoc. When reports met predefined thresholds, the clinic was alerted and patients received automated guidance. Study end points were assessed using qualitative and quantitative methods. Results A total of 9 patients (mean age 69.7 years) completed the study. Overall, 83% (30/36) of weekly sessions were completed. Patients found the frequency and time required to complete reporting acceptable. All patients agreed that the app was easy to use and understand. Providers felt the alerts they received required refinement. Patients and providers agreed it would be beneficial for patients to report for longer than 4 weeks. Patients felt that the training they received was adequate but contained too much information for a single session. All patients found the symptoms tracked to be appropriate; providers suggested shortening the list. All patients understood how to use the app for weekly reporting but had confusion about using it ad hoc. Providers felt the ad hoc feature could be removed. Neither patients nor providers viewed the in-app data reports but agreed on their potential value. Patients reported benefitting from symptom reporting through increased awareness of their symptoms. Clinic staff reported that app alerts were too numerous and redundant. They had difficulty responding to alerts within their existing workflow, partially because the data were not integrated into the electronic medical record system. Conclusions Overall, the intervention was found to be acceptable and appropriate for patients with RRMM. Points of friction integrating the intervention into the clinic workflow were identified. Clinic staff provided recommendations for addressing these issues. Once such modifications are implemented, ePRO data from patients with RRMM could be used to inform and improve clinical research and care. This study underlines the importance of an iterative approach to implementation that includes all stakeholders in order to ensure successful adoption.

Aims: This study quantifies the value of survival gains attributable to novel treatments approved since 2003 for United States (US) patients with relapsed/refractory multiple myeloma (RRMM). Methods: We estimated the increase in survival attributable to lenalidomide and bortezomib for multiple myeloma (MM) patients in the 1983–2013 Surveillance, Epidemiology, and End Results (SEER) registry. To estimate the survival benefit of treatments approved since 2015 (carfilzomib, elotuzomab, daratumumab, used in combination with lenalidomide and dexamethasone) we used clinical trial data to calibrate survival estimated using the SEER data. We then conducted an economic valuation of the estimated shift in survival curves for all therapies. Finally, we estimated the share of the value accruing to patients and manufacturers using treatment costs estimated from MarketScan data. Results: The introduction of bortezomib in combination with dexamethasone (Vd) and lenalidomide in combination with dexamethasone (Rd) resulted in substantial survival gains and societal value for multiple myeloma patients, generating 1.7 additional life-years per RRMM patient. More recently, approved novel treatments have improved survival over effective treatments (i.e. Rd/Vd) by an additional 2.5 life-years – the monetary value of this incremental survival benefit far exceeds the incremental cost of treatment. At the patient level, the incremental benefit of Rd/Vd is $335,500 and with novel treatments is $565,000. Applying this benefit to all future cohorts of US RRMM patients translates into a value of at least $75 billion and $130 billion with Rd/Vd and the novel treatments, respectively. Conclusions: SEER registry data were only available through 2013. Therefore, survival gains for recently approved treatments were estimated based on clinical trials, rather than observed survival. Our valuation analysis does not capture sources of value aside from survival gains, for example, better quality of life, increased productivity, or the value of surviving until subsequent novel therapies become available. Substantial extensions in life expectancy in RRMM since 2003 translate into real economic value gained by society.

We assessed the economic value of carfilzomib 56 mg/mCost-effectiveness of Kd56 vs. Vd was assessed using a partitioned survival model by estimating progression-free survival, overall survival, and direct costs over a lifetime horizon. Surveillance Epidemiology and End Results (SEER) survival data were extrapolated after matching registry and ENDEAVOR patients. Utilities were sourced from the literature and mapped from patient-reported quality of life in ENDEAVOR to estimate quality-adjusted life-years (QALYs) from life-years (LYs).The model predicted an average gain of 1.66 LYs and 1.50 QALYs with Kd56 vs. Vd, and lifetime additional costs of $182,699, resulting in an incremental cost-effectiveness ratio (ICER) of $121,828/QALY gained. The ICER was $114,793/QALY in patients with 1 prior treatment; $99,263/QALY in those not transplanted, and$150,000/QALY up to an 85% discount in bortezomib price.Kd56 is cost-effective for patients with R/RMM at a willingness-to-pay threshold of $150,000/QALY. Trial data in the model may limit generalizability; however, SEER registry data mitigates this challenge. Kd56 provides additional value in key subgroups, and remains cost-effective after steep comparator discounts.

In the US, carfilzomib 70 mg⁄m2 once-weekly plus dexamethasone (Kd70 QW) was recently indicated for relapsed and/or refractory multiple myeloma. In current US clinical practice, most patients treated with Kd receive carfilzomib at a previously approved dose of 27 mg/m2 twice-weekly (Kd27 BIW). This analysis assessed the cost-effectiveness (CE) of Kd70 QW vs Kd27 BIW regimens which were compared in the randomized phase 3 ARROW trial. Based on clinical outcomes (overall survival and utilities) from ARROW, a partitioned survival model was developed to estimate life years (LYs) and quality-adjusted life years (QALYs). Long-term survival was extrapolated using SEER registry data matched to ARROW patients. Costs were estimated using a US healthcare payer perspective. The analysis estimated that treatment with Kd70 QW vs Kd27 BIW resulted in an increase of 1.10 LYs, 0.91 QALYs, and additional lifetime costs of $74,858, yielding an incremental CE ratio (ratio of incremental costs to QALYs) of $82,257 per QALY gained. Results were robust to sensitivity and subgroup analyses. When compared with Kd27 BIW, Kd70 QW is the optimal dose that represents a cost-effective utilization of health care budget with incremental CE ratios well below the accepted willingness-to-pay thresholds 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.

Introduction Carfilzomib dosing as a single agent or in combination with dexamethasone (Kd) has evolved from the initial 27 mg/m2 twice-weekly (legacy dose), to more recently approved doses of 56 mg/m2 twice-weekly and 70 mg/m2 once-weekly (optimized doses). The objective of this study was to evaluate the overall survival (OS), and time to next treatment (TTNT) among multiple myeloma patients treated with Kd optimized vs legacy doses. Methods A retrospective analysis of patients receiving Kd between 01/01/2013–07/31/2017 was conducted using IQVIA’s oncology electronic medical records database. Kd dose was estimated based on body surface area. OS was measured from the Kd-initiation date until death. TTNT was defined as the time from Kd-initiation until the start of subsequent treatment. Kaplan-Meier analysis and Cox models were used to evaluate OS and TTNT. Results Of the 1,469 patients evaluated, 129 (8.8%) received optimized dose and 1,340 (91.2%) received legacy dose. Risk of mortality was 64% lower for patients receiving the optimized doses (HR: 0.36, 95% CI: 0.178–0.745). Patients receiving the optimized doses had significantly longer TTNT compared to patients receiving the legacy dose (median TTNT: 17.5 months [95% CI: 14.8-NE] and 13.2 months, [95% CI: 12.4–14.4], respectively; p = 0.023), and 33% lower risk of progressing to the subsequent treatment (HR: 0.67, 95% CI: 0.48–0.93). Conclusions Patient outcomes could be improved if eligible MM patients are treated with the optimized, recently approved Kd doses (56 mg/m2 twice-weekly and 70 mg/m2 once-weekly).

To assess the economic value of carfilzomib (Kyprolis), this study developed the Kyprolis Global Economic Model (K-GEM), which examined from a United States (US) payer perspective the cost-effectiveness of carfilzomib-lenalidomide-dexamethasone (KRd) versus lenalidomide-dexamethasone (Rd) in relapsed multiple myeloma (RMM; 1-3 prior therapies) based on results from the phase III ASPIRE trial that directly compared these regimens.A partitioned survival model that included three health states of progression-free (on or off treatment), post-progression, and death was developed. Using ASPIRE data, the effect of treatment regimens as administered in the trial was assessed for progression-free survival and overall survival (OS). Treatment effects were estimated with parametric regression models adjusting for baseline patient characteristics and applied over a lifetime horizon. US Surveillance, Epidemiology and End Results (1984-2014) registry data were matched to ASPIRE patients to extrapolate OS beyond the trial. Estimated survival was adjusted to account for utilities across health states. The K-GEM considered the total direct costs (pharmacy/medical) of care for patients treated with KRd and Rd.KRd was estimated to be more effective compared to Rd, providing 1.99 life year and 1.67 quality-adjusted life year (QALY) gains over the modeled horizon. KRd-treated patients incurred $179,393 in total additional costs. The incremental cost-effectiveness ratio (ICER) was $107,520 per QALY.Extrapolated survival functions present the greatest uncertainty in the modeled results. Utilities were derived from a combination of sources and assumed to reflect how US patients value their health state.The K-GEM showed KRd is cost-effective, with an ICER of $107,520 per QALY gained against Rd for the treatment of patients with RMM (1-3 prior therapies) at a willingness-to-pay threshold of $150,000. Reimbursement of KRd for patients with RMM may represent an efficient allocation of the healthcare budget.

e19509 Background: To examine treatment patterns and health care costs of RRAML in the US. Methods: We identified adults with ≥ 2 outpatient or ≥ 1 inpatient claim with a diagnosis of AML from 1/1/2011 to 12/31/2017 (first AML claim = index date) using IQVIA’s fully-adjudicated Health Plan Claims Data. Continuous health plan enrolment for ≥ 12 months prior to the index date and ≥ 1-month post-index, a RRAML diagnosis on or prior to the index date, or with hematopoietic stem cell transplantation (HSCT) or chemotherapy pre-index were required. Patients were grouped by post-index treatment (high- or low-intensity chemotherapy [HIC, LIC] with and with no HSCT, HSCT only, best supportive care [BSC], or no treatment within 90 days of index [NoTx]). Costs were evaluated for 12 months following index. Results: The final sample of patients with RRAML (n = 804) were grouped by age:18-59 (n = 411; 51.2%), 60-74 (n = 318; 39.5%), and ≥75 years (n = 75; 9.3%). Treatment within the three age cohorts was: HIC (13%, 11%, 1%); LIC (27%, 38%, 52%); HIC+HSCT (14%, 7%, 0%); LIC+HSCT (2%, 3%, 0%); HSCT (9%, 9%, 0%); BSC (21%, 21%, 31%); NoTx (13%, 12%, 16%), respectively. Mean monthly costs were lowest for BSC and highest for HSCT or HIC containing treatments, irrespective of age (Table). Inpatient costs were 50 to 100% of total costs. Monthly costs across treatment/age groups were higher earlier in treatment [month 1 range: $28,627 (BSC) to $180,867 (HIC)] and declined over time [month 12 range: $2,853 (BSC) to $53,003 (HIC+HSCT)]. Conclusions: Nearly half of all patients age ≥75 years and 33% of patients < 75 years received BSC or NoTx, which highlights a significant unmet need for new treatment options. Average costs per patient, especially early in treatment are high and driven by hospital service utilization. [Table: see text]

Background: Most multiple myeloma (MM) patients ultimately progress, with remission duration decreasing after first relapse. Recently, novel agents have been approved for the treatment of relapsed MM. There is a paucity of real-world data on these treatments. We sought to compare time to next treatment (TTNT) in MM patients in their second line of therapy (LOT2), treated with common proteasome inhibitor (PI)-based triplets. Methods: Adult MM patients who received carfilzomib (K) between 1 November 2013 and 29 February 2016 at US Oncology Network (USON) clinics utilizing iKnowMed™ electronic health records (EHRs) were identified. Patients were included if they were ⩾18 years of age, not enrolled in clinical trials, had ⩾2 visits at a USON clinic and received LOT2 regimens consisting of: K+lenalidomide with steroid (KRd), bortezomib+lenalidomide with steroid (VRd), or bortezomib+cyclophosphamide with steroid (VCyd). TTNT was estimated from LOT2 initiation to LOT3 initiation using the Kaplan–Meier method, and hazard ratios (HRs) were estimated using Cox modeling. Results: A total of 718 patients received a K-containing regimen sometime during their MM treatment (LOT1 to LOT5). Of these, 156 patients received: KRd ( n = 112; 71.8%), VRd ( n =27; 17.3%), or VCyd ( n = 17; 10.9%). Baseline characteristics were similar between groups (mean age: 64.8 years; 58% male). Median TTNT was longest for KRd [25.3 months; 95% confidence interval (CI): 19.71–NR], versus VRd or VCyd (VRd median TTNT: 10.2 months, 95% CI: 4.24–12.71; VCyd: 6.5 months, 95% CI: 3.02–12.78; log-rank p < 0.0001). The adjusted HR for KRd was 0.19 (95% CI: 0.11–0.37), compared with VRd. Conclusions: Considering the real-world nature of these data, the median TTNT observed with KRd was relatively consistent, with progression-free survival (PFS) for KRd observed in the phase III ASPIRE trial (median PFS: ITT population = 26.3 months; LOT2 = 29.6 months). Patients who received KRd at first relapse had significantly longer TTNT, compared with those on VRd or VCyd, confirming the value of KRd as an important treatment option for relapsed MM.