Real-World Treatment Patterns, Health Care Utilization, and Costs Among Relapsed/ Refractory Multiple Myeloma (rrMM) Patients

Abstract Background: Carfilzomib (Car, 2012), pomalidomide (Pom, 2013), and panobinostat (Pano, 2015) were approved for the treatment of rrMM patients after failure of at least two prior standard therapies. There is limited real-world data on utilization of these medications, health care resource utilization (HCRU) and costs in patients with rrMM at the US population level. This study aimed to describe treatment pattern, HCRU, and costs in rrMM patients who received at least two prior therapies using a large administrative claims database. Methods: This was a retrospective database analysis using the Truven Health MarketScan Commercial and Medicare Database. Patients (pts) were included if they were diagnosed with MM between Jan 1, 2006 and May 31, 2015, were ≥18 yrs, had no claim for stem cell transplant, and had ≥6 mos continuous enrollment before and ≥1 mos after the 1st MM diagnosis (index date) and treatment initiation (TI). Only patients who had ≥6 mos continuous enrollment after TI were included in HCRU and cost evaluation. If two or more drugs started within 90 days, they were considered as 1 regimen. End of a line of therapy (LOT) was defined when a new treatment was introduced or there was a treatment gap >90 days or end of follow-up, whichever occurred first. Third line of therapy (3L) was identified following 2 prior lines of therapy. Time to next treatment (TTNT) was defined from initiation of the LOT to initiation of subsequent LOT. Duration of treatment (DOT) and TTNT were estimated based on Kaplan-Meier method. Regimens were classified into 6 mutually-exclusive categories, including bortezomib (Bor), lenalidomide (Len), Pom, Car, thalidomide (Thal), and Other based therapies. HCRU and cost (per patient per month, PPPM) were calculated. The total costs included inpatient costs, outpatient costs and pharmacy costs. Results: A total of 9,960 pts were identified with initiation of 1L therapy. Median age was 67 yrs. And 57.4% were male. During an average of 20.0 mos follow-up post TI, 3,282 (33.0%), 1,103 (11.1%) and 400 (4%) pts initiated 2L, 3L, and 4L therapies, respectively. During 3L therapy, Bor (43.5%, including 10.5% for Bor-Len) and Len based regimens (29.8%) were most commonly observed. Median DOTs ranged from Car (3.7 mos) to Len (8.1 mos) based regimens. Median TTNT from short to long was Car, Pom, Bor, Len, Other, and Thal based regimens. On average, each patient with rrMM had 4.5 outpatient visits, 0.1 inpatients visits, 0.1 ER visits and 0.004 hospice care visits per month. The average PPPM costs were $14,286, $13,377, $11,919, for Bor, Len, Thal based regimens and $25,850 and $21,180 for Pom and Car based regimens, respectively. Conclusions: Although novel agents were added to rrMM treatment, Bor and/or Len based regimens remained the most commonly used therapies in 3L setting. Compared to Bor, Len and Thal based therapies, PPPM costs were higher for Pom and Car based regimens. These data could be useful for treatment consideration and economic evaluation. Table Table. Disclosures Shao: Merck & Co., Inc.: Employment. Monberg:Merck & Co.: Employment. Cao:Merck & Co.: Employment. Zhou:Merck & Co.: Employment. Zhong:Merck & Co., Inc.: Employment. Marinello:Merck & Co., Inc.: Employment.

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Healthcare utilization and costs associated with first-line treatment with obinutuzumab- and rituximab-based regimens for follicular lymphoma.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.e19536 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. e19536-e19536

Author(s):

Jamie T. Ta ◽

Tu My To ◽

Cheryl Sud ◽

Sheila Shapouri ◽

Arpamas Seetasith

Keyword(s):

Follicular Lymphoma ◽

Healthcare Utilization ◽

Real World ◽

Healthcare Costs ◽

Trial Participation ◽

Administrative Claims ◽

Cell Transplant ◽

First Line ◽

Real World Data ◽

Eligibility Criteria

e19536 Background: Current real-world data on healthcare utilization (HCU) and costs of common first-line (1L) follicular lymphoma (FL) regimens, including obinutuzumab (G), remain limited. The aim of this study was to examine real-world HCU and costs for the most common National Comprehensive Cancer Network (NCCN)-recommended 1L FL treatments. Methods: This was a retrospective cohort study using administrative claims data from the IQVIA PharMetrics Plus and IBM MarketScan Commercial and Medicare Supplemental databases. We identified patients (pts) ≥18 years, who had ≥1 inpatient claim or ≥2 outpatient claims, with a diagnosis of FL from February 1, 2015 to March 31, 2020, and had received 1L FL treatment (per NCCN guidelines) between February 1, 2016 and September 30, 2019. The first claim for FL treatment was the index date. All pts had ≥12 months of pre- and ≥6 months of post-index continuous enrollment in medical and pharmacy benefits. Pts with other primary cancers, FL treatment, or stem cell transplant during the pre-index period, and clinical trial participation or end-stage renal disease during the study period were excluded. All-cause HCU and costs (2020 USD) per pt during the 6-month post-index period were reported for the five most common 1L FL regimens (only complete NCCN regimens were considered). Results: Overall, 1991 pts met the eligibility criteria, and 53% were male. The mean (standard deviation [SD]) age and Charlson Comorbidity Index at index were 58 (10.4) years, and 1.7 (1.0), respectively. The most common 1L regimens were rituximab-bendamustine (R-benda; n=818 [41.1%]), R-monotherapy (R-mono; n=592 [29.7%]), R-CHOP (n=461 [23.2%]), G-benda (n=86 [4.3%]), and R-CVP (n=34 [1.7%]). The proportion of pts who had ≥1 hospitalization or an emergency room visit was highest with R-CHOP (26.7% and 33.6%, respectively) and lowest with R-mono (11.3% and 18.1%, respectively). Mean (SD) all-cause total healthcare costs were highest with R-benda and G-benda, followed by R-CHOP, R-CVP, and R-mono (Table). Mean (SD) all-cause medical and FL treatment costs (drug and administration) were highest for R-benda ($174,407 [$110,520]; $135,520 [$96,492]) and lowest for R-mono ($87,368 [$83,910]; $54,271 [$40,433]). Conclusions: This real-world study provides an update on HCU and costs among pts initiating current NCCN-recommended 1L treatment for FL. Unadjusted 6-month total healthcare costs were highest with R-benda, followed by G-benda, while the lowest costs were seen with R-CVP and R-mono. Future studies with adjustment for pt characteristics are needed to compare HCU and costs between FL regimens.[Table: see text]

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Economic Burden of Multiple Myeloma: Results from a Large Employer-Sponsored Real-World Administrative Claims Database, 2012 to 2018

Blood ◽

10.1182/blood-2019-131264 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 3414-3414

Author(s):

Dao Tran ◽

Rajesh Kamalakar ◽

Shivaji Manthena ◽

Sudeep Karve

Keyword(s):

Resource Use ◽

Stock Options ◽

Care Utilization ◽

Administrative Claims ◽

Hospital Inpatient ◽

Newly Diagnosed ◽

Economic Data ◽

First Line ◽

Line Of Therapy

Introduction: Multiple myeloma (MM) is a relatively rare, but serious neoplasm of terminally differentiated B-lymphocytes. Patients with MM experience clinical remission as well as relapses and refractory responses to therapy. New drugs and new combination therapies for MM in the past decade have increased the overall survival rate from 4.6 years to 6.1 years [Kumar et al., Leukemia 2014]. In recent years, there has been increasing emphasis towards determining the value of novel therapies based on the clinical and economic data. For example, ASCO, NCCN and ICER have developed value frameworks to evaluate cancer treatments. Since 2015 several therapies have received FDA approval for treatment of patients with MM although there is limited economic data for these newer MM treatments. Thus, the objective of the current study was to assess all-cause and disease-related health care utilization and costs among newly diagnosed and treated patients with MM in the U.S. Study Design, Materials and Methods: This retrospective, observational study design was conducted using a large national administrative claims database (IBM® MarketScan® Commercial and Medicare Supplemental Databases). Data includes persons enrolled in commercial health plans or in Medicare supplemental plans. Study participants were identified using medical and pharmacy claims from about 163 million individuals enrolled in a plan at any time from 1-1-2011 to 6-30-2018. Patients with MM were identified based on the following criteria: (1) At least two MM diagnoses (using ICD-9-CM 203.00-203.02; or ICD10: C90.00-C90.02 codes) between 1-1-2012 and 6-30-2017 with the first observed diagnosis date set as the MM diagnosis index date (MMDID); (2) ≥12 months of continuous enrollment prior to, and ≥2 months post, MMDID; (3) Patients received MM drug therapy after diagnosis; (4) Age ≥18 years at MMDID; and (5) Patients with any other malignant cancer prior to, or at, the time of MMDID were excluded; All patients were followed from MMDID until end of data set (6-30-2018) or to end of health plan enrollment, whichever was first (i.e., the follow-up period.). All-cause- and MM-related (claims with MM diagnosis or treatment) health care utilization and costs were assessed during the follow-up period. All analyses were descriptive in nature. Results: There were 3,144 newly diagnosed MM patients eligible for this study with mean (std dev) age of 65.4 (11.7) years.56% were males and 42.8% had ≥1 comorbidity during the follow-up period (mean follow-up: 21.5 months). Following first-line treatment, 56.1% received 2L therapy, 29.9% received 3L, 15.7% received 4L, and 7.6% had ≥5 lines of treatment (Figure 1). In the first line, monotherapy was used for 24%, doublets were used for 38%, triplets for 37%, and 4 or more drugs for 1.5% of patients. Some drug regimens had up to 9 drugs. Among the selected cohort, 94.8% had ≥1 MM-related hospital outpatient visit, and 81.7% had ≥1 outpatient pharmacy claim. Furthermore, 77.7% of patients had ≥1 MM-related inpatient hospital admission with an average (std dev) length of stay for each admission of 12 (11.06) days. Total average (std dev) follow-up costs from all causes (AC) were $288,304 ($333,673) of which MM-Disease Related (DR) costs for all lines of therapy accounted for $253,071 (87.8%). Pharmacy drugs accounted for 32.9% of total DR costs, while hospital inpatient visits were 29.3% of cost and hospital outpatient (mostly provider-administered drugs) were 26.2% of total DR costs (Figure 2). A breakdown of total DR costs by line of therapy is reported in Figure 3. The DR cost per member per month (PMPM) for the total follow-up period was $14,140. The mean (std dev) DR PMPM costs for each line of therapy were $16,342 ($20,028) for 1L, $19,267 ($29,645) for 2L, $17,773 ($23,347) for 3L, and $20,146 ($26,401) for 4L. Conclusion: This study documents recent trends in economic and resource use burden in patients with MM including trends following the approval of newer treatments (2015). Pharmacy and hospital inpatient utilization accounted for two-third of total DR-costs. In combination with the clinical data, the economic and resource use data serve as inputs in assessing value and budget impact of newer treatment Disclosures Tran: AbbVie: Other: Internship. Kamalakar:AbbVie: Employment, Other: and may hold stock or stock options. Manthena:AbbVie: Employment, Other: may own stocks/options. Karve:AbbVie: Employment, Other: stock/stock options.

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Analysis of real world patient compliance to everolimus-based therapy among post-menopausal women with hormone receptor positive and human epidermal growth factor receptor 2 negative (HR+/HER2-) metastatic breast cancer (mBC).

Journal of Clinical Oncology ◽

10.1200/jco.2017.35.15_suppl.e12546 ◽

2017 ◽

Vol 35 (15_suppl) ◽

pp. e12546-e12546 ◽

Cited By ~ 1

Author(s):

Nicole Princic ◽

Matthew Brouillette ◽

Derek Tang ◽

Chinjune Lin ◽

Brad Lanoue ◽

...

Keyword(s):

Patient Compliance ◽

Real World ◽

Total Duration ◽

Medication Possession Ratio ◽

Secondary Malignancy ◽

Real World Data ◽

World Data ◽

Post Menopausal ◽

Line Of Therapy

e12546 Background: Patient compliance, typically not captured in clinical trials, may have profound effects on treatment effectiveness. As there has been limited real-world data, the objective of this analysis was to examine compliance among HR+/HER2- mBC patients treated with everolimus as a second or later line of therapy in the US across age groups. Methods: In this retrospective cohort study, the MarketScan Commercial and Medicare Supplemental claims databases were used to select post-menopausal HR+/HER2- mBC women who initiated an everolimus-based line of therapy during 1/1/2013- 7/31/2016 study period. The first secondary malignancy diagnosis was the index date. Patients had 6 months of continuous enrollment in their health plans pre- and post- index, and were followed until the earliest of disenrollment, inpatient death, or end of study. Compliance was measured using medication possession ratio (MPR) during a second (2L), third (3L) and fourth (4L) line of therapy. MPR was defined as total days of supply of everolimus during the line of therapy divided by total duration of the line. Results: Of 645 eligible mBC patients treated with everolimus during follow-up, there were 239, 142, and 80 with a 2L, 3L, and 4L of therapy respectively. Mean age overall was 61.1 + 11.7 years and average duration of follow-up was 718.3 + 304.1 days. Across all patients the median MPR ranged during each line from 0.90-0.92 and the majority (93.8%-97.5%) were highly compliant to therapy (defined as MPR>80%). These results were consistent for patients aged <65 and 65+ years across a 2L, 3L, and 4L of therapy (Table). Conclusions: This study shows high compliance for everolimus-based therapy within a 2L, 3L, and 4L of therapy and this is consistent across ages. This real world data suggests good drug manageability and may provide valuable information for clinicians in selecting treatment for mBC. [Table: see text]

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Treatment patterns and characteristics of acute promyelocytic leukemia (APL) patients receiving arsenic trioxide (ATO) therapy in a real-world setting.

Journal of Clinical Oncology ◽

10.1200/jco.2017.35.15_suppl.e18522 ◽

2017 ◽

Vol 35 (15_suppl) ◽

pp. e18522-e18522

Author(s):

Boxiong Tang ◽

Susan Gabriel ◽

Jifang Zhou ◽

Ashutosh K. Pathak ◽

Debra Irwin ◽

...

Keyword(s):

Real World ◽

Index Date ◽

Claims Data ◽

Treatment Patterns ◽

Chronic Obstructive ◽

Administrative Claims ◽

First Line ◽

Real World Data ◽

World Data

e18522 Background: Clinical trials have shown that low-risk APL patients had significantly better outcomes when receiving first-line all-trans retinoic acid (ATRA) + ATO compared with standard ATRA + chemotherapy. Few published studies have used real-world data to describe patients using ATO and their current treatment patterns. This study used United States (US) administrative claims data to describe treatment patterns and characteristics of patients receiving first-line ATO. Methods: This retrospective, observational cohort study used claims data from the MarketScan databases. As there is no ICD-9-CM diagnosis code for APL, ATO treatment was used as a surrogate for the diagnosis of APL since ATO is typically used only in APL patients. Patients were selected if they had ≥1 claims for ATO between January 1, 2000, and June 30, 2015. Date of first use was designated the index date. To identify first-line ATO initiation, patients with ATRA or other APL-indicated chemotherapy claims any time before the index date were excluded. Variable baseline and follow-up periods consisting of ≥3 months of pre-index and ≥30 days of post-index continuous enrollment in medical and pharmacy benefit were used. Results: In total, 331 patients were identified with a subset (n = 265) having ≥2 claims for ATO. The analysis focused on these 265 patients, 54% of whom were male. Mean age was 60.6 years; 45% were covered by Medicare. The most common comorbid conditions measured were diabetes (6%), chronic obstructive pulmonary disease (5%), and congestive heart failure (4%). The most commonly selected APL treatments administered during follow-up were ATRA (17%) and daunorubicin (9%) with the use of idarubicin, cytarabine, and mitoxantrone at less than 3%. Maintenance therapy with methotrexate or 6-mercaptopurine was observed in 7% and 6% of patients, respectively. Conclusions: This is one of the first studies to examine patient characteristics and treatment patterns for first-line ATO using real-world data. Further research is needed to evaluate outcomes for patients receiving ATO as first-line therapy and to re-evaluate treatment guidelines in light of these outcomes.

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12-month uptake of PD-L1 testing and atezolizumab (atezo) + nab-paclitaxel (nab-pac) treatment in metastatic triple-negative breast cancer (mTNBC) following accelerated FDA-approval in the United States.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.e13103 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. e13103-e13103

Author(s):

Leisha A. Emens ◽

Christopher Craggs ◽

Marcio Debiasi ◽

Matthew Kent ◽

Patricia Luhn ◽

...

Keyword(s):

Real World ◽

Systemic Therapy ◽

Locally Advanced ◽

The United States ◽

Considerable Proportion ◽

Real World Data ◽

Fda Approval ◽

Third Line ◽

Line Of Therapy

e13103 Background: In March 2019, atezo + nab-pac received accelerated FDA-approval for the treatment (tx) of patients (pts) with unresectable locally advanced or metastatic TNBC who express PD-L1 in immune cells (IC), based on the results from the randomized IMpassion130 clinical trial. A companion diagnostic, the Ventana SP142 PD-L1 assay, was concurrently approved. This real-world data analysis describes the uptake of both PD-L1 testing and therapy with atezo + nab-pac from the time of approval. Methods: This is a retrospective study using the Flatiron Health electronic health record (EHR) derived de-identified database, representing > 2.4 million US cancer pts from > 280 cancer clinics. Pts diagnosed with mTNBC starting any line of systemic therapy between March 9, 2019 and December 31, 2019 (to be updated in April 2020 for 12-months follow-up) were included. Two cohorts were examined: pts receiving either atezo-based tx, or tx without atezo. Demographics were described in the pt group overall and by line of therapy (LOT). Steroid tx at baseline was classified as any order/administration of a steroid within the first three administrations of a systemic therapy in a given LOT. Any record of a PD-L1 test was reported. Results: 228 pts diagnosed with mTNBC were included; 65 pts (28%) received any atezo combination, the majority in combination with nab-pac: 57% (n = 37) in first-line (1L), 25% (n = 16) in second-line (2L), and 18% (n = 12) in third-line or later (3L+). Median age at metastatic diagnosis (mDx) for atezo-treated pts was: 1L, 65 y (IQR: 49, 72); 2L, 56 y (IQR: 48, 64); 3L+, 52 y (IQR: 42, 60). Median age at mDx for non-atezo-treated patients was: 1L, 63 y (IQR: 53, 74); 2L, 59 y (IQR: 51, 65); 3L+, 61 y (IQR: 53, 71). Of 228 pts, 158 (69%) had any PD-L1 test recorded. Among atezo-treated pts, 62 (95%) had a documented PD-L1 test; of those 40 (62%) were tested using the SP142 assay. Overall, 205 tests were recorded: SP142, n = 96 (46.8%); unknown/not documented, n = 44 (21.5%); 22C3, n = 36 (17.6%); lab-developed test, n = 26 (12.7%); SP263, n = 2 (1.0%); and 28-8, n = 1, (0.5%). 37 (57%) atezo-treated pts received a steroid at baseline. Conclusions: Atezo tx and testing with SP142 is being implemented following accelerated FDA-approval. A considerable proportion of pts received steroids at baseline and had nab-pac as chemotherapy backbone in clinical practice. Clinical outcomes of atezo-treated pts in the real-world setting remain to be further explored pending longer pt follow-up times.

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Serious infections and cardiovascular complications among patients with chronic lymphocytic lymphoma treated with first-line ibrutinib or bendamustine/rituximab.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.e19503 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. e19503-e19503

Author(s):

Debra E. Irwin ◽

Stephen Thompson ◽

Rinat Ribalov ◽

Azhar Choudhry

Keyword(s):

Real World ◽

Index Date ◽

Cardiovascular Complications ◽

Administrative Claims ◽

First Line ◽

Real World Data ◽

World Data ◽

Serious Infections ◽

Baseline Characteristics

e19503 Background: Clinical trials have shown positive outcomes associated with ibrutinib monotherapy (IM) and bendamustine / rituximab combination (BR) therapy in patients with chronic lymphocytic lymphoma (CLL) compared to other standard treatments, but limited real-world data exist. This study evaluated serious infections and cardiovascular complications in CLL patients treated with first-line IM or BR therapy using US real-world data. Methods: Administrative claims from the MarketScan® Research Databases were used to identify adult patients enrolled in commercial or Medicare supplemental insurance plans based on a first prescription fill of IM or BR therapy (the index date) from 2/1/14 to 9/1/19. Patients were diagnosed with CLL, treatment naïve, and continuously enrolled for ≥12 months prior to and following the index date. Serious infections and cardiovascular complications requiring hospitalization (diagnosis code in any position) were evaluated during a fixed 12-month follow-up period. Statistical differences in outcome distributions between the treatment groups were tested. Multivariate logistic regression models for lower respiratory tract infection (LRTI) and atrial fibrillation (AF) were also conducted to determine the adjusted odds of hospitalization. Results: Of 2,138 CLL patients, 810 had IM and 512 had BR as index therapy with ≥12 months of follow-up data. Patients receiving IM were older and more likely to have had an LRTI during baseline compared to BR patients, otherwise both groups had similar baseline characteristics. Hospitalization for serious infections was more common during follow-up among IM patients than BR patients (17.7% vs. 13.1%; p = 0.027). Specifically, 10.2% of IM patients had a bacterial infection hospitalization compared to 5.7% of BR patients (p = 0.004) and 10.7% of IM patients had a LRTI hospitalization compared to 6.6% of BR patients (p = 0.012). After adjusting for baseline characteristics, IM patients did not have significantly higher odds of a LRTI hospitalization (OR = 1.51; p = 0.069). Hospitalization for cardiovascular complications was more common during follow-up among IM patients than BR patients (18.3% vs. 11.9%; p = 0.002). Specifically, 8.4% of IM patients had an AF-related hospitalization versus 2.7% of BR patients (p < 0.001). After adjusting for baseline characteristics, IM patients were more likely to have a hospitalization for AF versus BR patients (OR = 3.89; p < 0.001). Conclusions: In a real-world setting, serious infections and cardiovascular complications were more common among CLL patients treated with first-line IM compared to BR during 12 months of follow-up. IM patients were more likely than BR patients to have an inpatient admission due to AF after adjusting for other patient characteristics.

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Health Care Utilization and Associated Costs in Elderly Persons with Non-CML Myeloproliferative Neoplasms: Real-World Evidence From a United States Medicare Population

Blood ◽

10.1182/blood.v120.21.4273.4273 ◽

2012 ◽

Vol 120 (21) ◽

pp. 4273-4273 ◽

Cited By ~ 1

Author(s):

Sudeep Karve ◽

Gregory L Price ◽

Keith L Davis ◽

Gerhardt M Pohl ◽

Richard A Walgren

Keyword(s):

Health Care ◽

Health Care Utilization ◽

Index Date ◽

Myeloproliferative Neoplasms ◽

Care Utilization ◽

Cell Transplant ◽

Elderly Persons ◽

Employment Equity ◽

Equity Ownership

Abstract Abstract 4273 Background: Non-CML myeloproliferative neoplasms (MPNs), which include essential thrombocythemia (ET), polycythemia vera (PV), myelofibrosis (MF) and MPN not otherwise specified (MPN-NOS), are characterized by activation of JAK2 signaling and abnormal blood cell production. Median survival ranges from months to years for MF and up to a decade or more for PV and ET. Some symptomatic treatment options exist, but with the exception of hematopoietic stem cell transplant, none are curative. Although MPN incidence is highest in persons aged ≥65 years, little is known about overall health care utilization and costs in elderly persons with these diseases. MPNs are more prevalent in the elderly and therefore Medicare enrollees are a highly relevant source for US-based resource utilization and cost data for these diseases. Objective: To compare all-cause health care utilization and costs from four subtypes of elderly MPN patients (ET, PV, MF and MPN-NOS) with matched non-MPN/non-cancer controls. Methods: Retrospective data were taken from the Survey, Epidemiology, and End Results (SEER)-Medicare linked database in the US, which combines clinical information from the SEER cancer registry (MPN reporting has been required since 2001) with medical and pharmacy claims for Medicare enrollees. Patients with a new MPN diagnosis between Jan 1, 2001 and Dec 31, 2007 were selected and evaluated for all-cause health care utilization and costs from Jan 1, 2008 (index date) through Dec 31, 2008 (follow-up end date). Patients were classified by MPN subtype based on the most recent diagnosis information (ICD-O-3 from the SEER registry or ICD-9-CM from Medicare claims) before the index date. Patients who died before follow-up end, had HMO or discontinuous Medicare enrollment during the follow-up year, had enrollment based on end stage renal disease, or a diagnosis of a non-MPN malignancy before follow-up end were excluded from the study. Separate non-MPN/non-cancer control groups were selected for each MPN subtype and matched (5:1) on birth year, gender, ethnicity, geography, and reason for Medicare eligibility. Per patient health care utilization and costs during the follow-up year were aggregated and stratified by care setting. Costs were adjusted to 2010 US$ and represent amounts reimbursed by Medicare to providers. Costs were compared between MPN cases and controls using univariate t-tests. Results: A total of 1,355 MPN patients (n = 445 ET, 684 PV, 81 MF, 145 MPN-NOS) were identified for study inclusion and assigned matching controls. For ET, PV, MF and MPN-NOS cases, respectively, mean [SD] age at index was 75.5 [9.7], 70.8 [11.3], 70.8 [10.4] and 74.1 [8.9] years and % female was 69.0, 43.9, 54.3, and 55.2. Mean [SD] years between first MPN diagnosis and study index date was 3.1 [2.0], 3.4[1.9], 2.7 [2.0], and 3.1 [2.1] for ET, PV, MF and MPN-NOS cases, respectively. A significantly (p<0.05) higher proportion of MPN cases, regardless of subtype, had ≥1 hospitalization during follow-up vs. controls (ET vs. control: 22% vs. 16%, PV vs. control: 27% vs. 15%, MF vs. control: 31% vs. 12%, MPN-NOS vs. control: 36% vs. 17%). Mean [SD] total days of hospital care were similarly higher in MPN cases (ET vs. control: 2.7 [12.8] vs. 1.6 [6.6], PV vs. control: 2.6 [7.0] vs. 1.7 [9.5], MF vs. control: 2.5 [6.2] vs. 1.2 [5.9], MPN-NOS vs. control: 4.0 [10.0] vs. 2.1 [13.7]), although the PV vs. control difference was not statistically significant. The ER visit rate during follow-up was also significantly (p<0.05) higher in MPN cases (ET vs. control: 34% vs. 24%, PV vs. control: 38% vs. 25%, MF vs. control: 46% vs. 21%, MPN-NOS vs. control: 44% vs. 29%). All-cause costs for MPN cases vs. matched controls are presented in the figure. Mean total costs per patient, driven equally by inpatient and outpatient services, were significantly (p<0.001) higher in MPN cases (ET vs. control: $11,259 vs. $8,897, PV vs. control: $13,337 vs. $8,530, MF vs. control: $20,917 vs. $7,367, MPN-NOS vs. control: $20,174 vs. $9,800). Conclusions: Total health care costs during a given year for elderly patients with MPNs are 1.3 to 3 times higher (depending on subtype) than those of matched controls. These findings may help inform future cost effectiveness evaluations of novel MPN treatments, as well as decision making in the provision of optimal MPN care within a Medicare system in which resources are finite and must be allocated ethically and efficiently. Disclosures: Karve: RTI Health Solutions: Consultancy, Research Funding. Price:Eli Lilly and Company: Employment, Equity Ownership. Davis:Eli Lilly, Merck, GlaxoSmithKline, Bristol-Myers Squibb, Pfizer, Eisai, Sanof-Aventis, Gilead Sciences, MedImmune: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Pohl:Eli Lilly and Company: Employment, Equity Ownership. Walgren:Eli Lilly and Company: Employment, Equity Ownership.

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Real-World Treatment Patterns in Patients with Light Chain (AL) Amyloidosis: Analysis of the Optum US Electronic Health Records and Commercial Claims Database

Blood ◽

10.1182/blood-2020-140405 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 4-5

Author(s):

Angela Dispenzieri ◽

Jeff Zonder ◽

James Hoffman ◽

Sandy W. Wong ◽

Michaela Liedtke ◽

...

Keyword(s):

Board Of Directors ◽

Real World ◽

Research Funding ◽

Al Amyloidosis ◽

Cell Transplant ◽

Publicly Traded ◽

Real World Data ◽

Advisory Committees ◽

Line Of Therapy ◽

Diagnosis Date

Background: Light chain (AL) amyloidosis is a rare disease in which deposits of insoluble amyloid derived from immunoglobulin light chains accumulate and cause dysfunction of organs including the heart and kidneys. In order to reverse organ damage and improve overall survival, treatment must provide rapid, deep, and sustained hematologic responses. Although therapies approved for the treatment of multiple myeloma have demonstrated some efficacy, there are currently no health authority-approved treatments for AL amyloidosis. A retrospective observational study was undertaken to characterize patients and describe treatment patterns in patients with AL amyloidosis from real-world data in the US. Methods: Data were extracted from Optum Electronic Health Records (EHR) and Optum Clinformatics Data Mart (claims) databases. Eligible patients had (1) a diagnosis of AL amyloidosis (ICD-10 code E85.81) on or after January 1, 2008 or (2) a diagnosis of amyloidosis (ICD-9 codes 277.30, 277.39; ICD-10 codes E85.89, E85.9) on or after January 1, 2008 and at least one line of therapy (LOT) comprising one or more of the following treatments: bendamustine bortezomib, carfilzomib, cyclophosphamide, daratumumab, dexamethasone, doxorubicin, elotuzumab, etoposide, interferon alfa-2a, interferon alfa-2b, ixazomib, lenalidomide, melphalan, panobinostat, pomalidomide, prednisone/prednisolone, thalidomide, vincristine, or autologous stem cell transplant. Patients were required to have continuous medical enrollment during the 365 days prior to the index amyloidosis diagnosis date (claims data) or have first active date at least 365 days prior to index amyloidosis date (EHR data), must not have had prior cancer in the 365-day period prior to the index amyloidosis diagnosis date, and must have been age ≥18 years at time of amyloidosis diagnosis. Patients treated with dexamethasone only or dexamethasone and prednisolone only for ≤90 days and those treated with prednisone/prednisolone only irrespective of duration were excluded. LOTs were defined by a start date, an end date, and a distinct regimen made up of ≥1 drugs. Patients may have multiple sequential LOTs during the available follow-up period. Comorbidities were scored according to the Charlson Comorbidity Index (CCI). A score of 0 reflects no comorbidities; higher scores reflect increased mortality risk and the maximum score is 24. Descriptive statistics are provided for patient characteristics at index diagnosis date for patients who had ≥1 LOT, distribution of regimens in each LOT, treatment attrition, and duration of treatment in each line of therapy and by regimen. Results: Data from 1688 patients (Optum claims, n=624; Optum EHR, n=1064) were included in the analysis (Figure). Median age at diagnosis was 67 years and 55.7% of patients were male. Almost 70% of patients had an index diagnosis date between 2013 and 2019. The median CCI score at index diagnosis date (based on comorbidities in the 365-day pre-index diagnosis period) was 1 and 10.9% of patients had a stem cell transplant on or after the index diagnosis date. The three most common comorbidities were hypertension (50.8%), renal failure (26.2%), and congestive heart failure (24.6%). Median follow-up period was 28 months. Treatment information for regimens used by ≥5% of patients for LOT1 is summarized in the Table. The most common treatment regimen for LOT1 was a proteasome inhibitor (PI), an alkylating agent, and a steroid (n=392, 23.2%); this combination was used for an average of 111 days. Of patients treated with this combination, the most common regimen was bortezomib, cyclophosphamide, and dexamethasone (VCd), used by 358 patients. Steroid only (dexamethasone for ≥90 days) regimens accounted for 16.4% and a PI ± steroid for 13.2% of LOT1. A limitation of the study is that assumptions were used to classify AL amyloidosis vs other types of amyloidosis. Conclusions: VCd is the most commonly used regimen in patients with newly-diagnosed AL amyloidosis. This is consistent with clinical guidelines and real world data from Europe (Palladini et al, EHA 2020), supporting this regimen as the current standard of care for treatment of patients with newly-diagnosed AL amyloidosis. Disclosures Dispenzieri: Celgene: Research Funding; Pfizer: Research Funding; Alnylam: Research Funding; Intellia: Research Funding; Janssen: Research Funding; Takeda: Research Funding. Zonder:Intellia, Amgen, Takeda, Janssen, Regeneron, Alnylam, Caelum, Oncopeptides: Consultancy; BMS, Celgene: Research Funding. Hoffman:Loxo: Current equity holder in publicly-traded company; Celgene: Honoraria, Speakers Bureau. Wong:Sanofi: Membership on an entity's Board of Directors or advisory committees; Janssen: Research Funding; Fortis: Research Funding; Roche: Research Funding; GSK: Research Funding; Amgen: Consultancy; Bristol Myers Squibb: Research Funding. Liedtke:Adaptive: Membership on an entity's Board of Directors or advisory committees; Caelum: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees; Janssen: Membership on an entity's Board of Directors or advisory committees; GSK: Membership on an entity's Board of Directors or advisory committees; Jazz Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria. Abonour:BMS: Consultancy, Research Funding; Takeda: Consultancy; Janssen: Honoraria, Research Funding; Celgene: Consultancy. D'Souza:Amgen, Merck, TenoBio: Research Funding; Akcea, Imbrium, Janssen, Pfizer: Consultancy. Lee:Janssen: Current Employment. Nair:Janssen: Current Employment. Potluri:SmartAnalyst Inc.: Current Employment. Weiss:Janssen: Current Employment, Current equity holder in publicly-traded company. Vermeulen:Janssen: Current Employment, Current equity holder in publicly-traded company. Lam:Janssen: Current Employment. Mehra:Janssen: Current Employment.

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Trends in immunotherapy use in patients with advanced non-small cell lung cancer (aNSCLC) patients: Analysis of real-world data.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.e19311 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. e19311-e19311

Author(s):

Lawrence H. Kushi ◽

Laura Lasiter ◽

Andrew J. Belli ◽

Marley Boyd ◽

Suanna S. Bruinooge ◽

...

Keyword(s):

Real World ◽

Single Agent ◽

Administrative Claims ◽

Real World Data ◽

Treatment Regimens ◽

Kaplan Meier ◽

Real World Evidence ◽

One Year ◽

Data Elements

e19311 Background: Leveraging data from a collaboration with 9 data partners, Friends of Cancer Research convened the Real-world Evidence Pilot 2.0, to examine trends and real world (rw) data endpoints in immunotherapy (IO) use for the front line treatment of aNSCLC. Methods: This study leveraged parallel analyses of rw data elements across heterogenous data sources (EHR, administrative claims, and registry) to: a) describe trends in uptake and use of novel IO frontline therapy after advanced diagnosis in NSCLC patients treated in usual care settings and b) examine associations between treatment and rw outcomes at one-year follow-up. The proportion of patients treated on each regimen (IO single agent, chemo, or IO + chemo) from 2011 through 2017 were calculated. Analysis included proportion of patients across treatment regimens stratified by year to describe post approval uptake of IO. Kaplan-Meier survival estimates were reported to adjust for follow-up time and stratified by PD-L1 status and stage. Results: Seven datasets identified a range of 999 to 4617 patients per dataset for this analysis. Across datasets, 2508, 3446, and 4176 patients initiated treatment in 2015, 2016, and 2017, respectively. No patients received IO or IO + chemo regimens prior to 2015. Initial approvals for IO use in aNSCLC occurred in October 2015 and for first line in metastatic NSCLC in October 2016. When examining survival at 1 year, overall, OS in PD-(L)1 + patients appeared longer than those with a PD-(L)1 - status. Conclusions: RWE analyses may reveal important trends in clinical cancer patient care including patterns of off-label use. The heterogeneity in the timing of IO uptake across datasets ranged from immediately after approval to ~12 months post-approval. [Table: see text]

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