scholarly journals Selection of patients with myelodysplastic syndromes from a large electronic medical records database and a study of the use of disease-modifying therapy in the United States

BMJ Open ◽  
2018 ◽  
Vol 8 (7) ◽  
pp. e019955 ◽  
Author(s):  
Xiaomei Ma ◽  
David P Steensma ◽  
Bart L Scott ◽  
Pavel Kiselev ◽  
Mary M Sugrue ◽  
...  

ObjectivesTreatment patterns for patients with myelodysplastic syndromes (MDS) outside clinical trials are not well described. Our objective was to evaluate treatment patterns and patient characteristics that influence time to disease-modifying therapy in patients with MDS in the USA.Design, participants and outcome measuresPatients with MDS treated with erythropoiesis-stimulating agents (ESAs), iron chelation therapy, lenalidomide (LEN) and the hypomethylating agents (HMAs) azacitidine and decitabine, were retrospectively identified in the GE Centricity Electronic Medical Record database between January 2006 and February 2014; LEN and HMAs were defined as ‘disease-modifying’ therapies. Multivariable Cox regression models were used to ascertain patient characteristics associated with time to disease-modifying therapy.ResultsOf the 5162 patients with MDS, 35.7%, 40.3% and 4.6% received 1, ≥1 and ≥2 therapies, respectively. ESAs were the first-line (72.5%) and only (64.0%) treatment in the majority of patients who received ≥1 therapy. ESA-only patients were older and had more comorbidities, including isolated anaemia. LEN and HMAs were first-line treatment in 12.4% of patients each; 32.7% received LEN or HMAs at any time. The majority of del(5q) patients (77.6%) received ≥1 therapy, most commonly LEN, compared with 40% of patients without del(5q). A shorter time to disease-modifying therapy was significantly associated with absence of comorbidities, diagnosis after February 2008, lower baseline haemoglobin level, age <80 years and male gender (p<0.002 for all).ConclusionsA high proportion of patients diagnosed with MDS in the USA do not receive approved disease-modifying therapies. It is important to improve access to these therapies.

2009 ◽  
Vol 15 (1) ◽  
pp. 50-58 ◽  
Author(s):  
A Gajofatto ◽  
P Bacchetti ◽  
B Grimes ◽  
A High ◽  
E Waubant

Background Options for non-responders to relapsing–remitting multiple sclerosis (RRMS) first-line disease-modifying therapies (DMT) are limited. We explored whether switching first-line DMT is effective. Methods Patients with RRMS who first received interferon-beta (IFNB) or glatiramer acetate (GA) were classified in three categories: DMT change because of suboptimal response, DMT change because of other reasons, and no DMT change during follow-up. Outcomes included annualized relapse rate (ARR) and relapse-free proportions. Results We identified 597 patients who initiated first-line DMT. For patients who did not change DMT ( n = 240), pre-DMT and on-DMT median ARR were 0.50 and 0 ( P < 0.0001). At 24 months, 76% (95%CI = 69–81%) of patients who did not change DMT were relapse-free. Of the 155 who switched because of suboptimal response, 101 switched to another first-line DMT. Median ARR pre-DMT, on first DMT and second DMT were: 0.50, 0.55, and 0.25 for switchers from IFNB to GA (IFNB/GA, n = 12) (pre-DMT versus first DMT: P = 0.92; first versus second DMT: P = 0.31); 0.90, 0.50, and 0 for switchers from GA to IFNB (GA/IFNB, n = 18; P = 0.19; P = 0.01); 0.50, 0.68, and 0 for switchers from an IFNB to another IFNB (IFNB/IFNB’, n = 71; P = 0.34; P = 0.02). Estimated relapse-free proportion after 24 months of treatment was 42% (95%CI=15–66%) during the period on IFNB versus 53% (95%CI = 17–80%) on GA for IFNB/GA ( P = 0.21); 12% (95%CI = 0–40%) on GA versus 87% (95%CI = 59–97%) on IFNB for GA/IFNB ( P = 0.001); and 41% (95%CI = 29–52%) on initial IFNB versus 67% (95%CI = 53–79%) on subsequent IFNB for IFNB/IFNB’ ( P = 0.0001). Conclusions Switching first-line DMT in patients with RRMS failing initial therapy may be effective in many cases.


2021 ◽  
Vol 39 (28_suppl) ◽  
pp. 291-291
Author(s):  
Jinan Liu ◽  
Eric M Maiese ◽  
Bruno Émond ◽  
Marie-Hélène Lafeuille ◽  
Patrick Lefebvre ◽  
...  

291 Background: Among patients (pts) with endometrial cancer (EC), response rates for platinum-based regimens in the first-line (1L) setting range from 40% to 62% in clinical trials. This study describes patient characteristics, treatment patterns, time to next treatment (TTNT), and overall survival (OS) among pts with advanced/recurrent EC treated with a platinum-based regimen in a real-world setting in the US. Methods: This retrospective study used Optum Clinformatics Extended Data Mart de-identified databases from January 1, 2007, to December 31, 2019. Adult pts with advanced/recurrent EC who initiated a 1L platinum-based regimen and subsequently initiated second-line (2L) antineoplastic therapy were identified. Prior to initiation of 1L, a 12-month washout period of continuous enrollment without use of antineoplastic agents (except hormonal agents) was imposed. Kaplan-Meier (KM) rates were used to report TTNT and OS from 2L, third line (3L), and fourth line (4L), separately. Results: A total of 1878 pts with advanced/recurrent EC initiated 2L therapy following a platinum-based regimen in 1L. Among them, 739 (39.4%) pts initiated 3L and 330 (17.6%) initiated 4L or later (4L+) therapy. Median pt age was 68.0 years. More pts received platinum-based regimens (56.4%) in 2L than other options (Table). Few pts (3.3%) received immunotherapy. Among pts receiving 3L, a similar percentage of pts were treated with platinum-based (33.2%) and other chemotherapy regimens (33.8%); few pts received immunotherapy (3.0%). Among pts receiving 4L+, the most frequent treatment option was other chemotherapy (46.1%). Median TTNT was 17.7, 10.6, and 8.4 months for 2L, 3L, and 4L pts, respectively. KM rates of OS following initiation of 2L therapy at 1, 2, 3, and 4 years were 68.4%, 49.6%, 41.3%, and 33.6%, respectively, with a median OS of 23.5 months. Conclusions: Among pts with advanced/recurrent EC treated with platinum-based therapy in 1L, platinum-based regimens remain prevalent treatment choices in later lines of therapy. In this study, immunotherapy was used infrequently in 2L, 3L, and 4L+. The median TTNT decreased in later lines of therapy. This study highlights a critical need for novel, more effective treatment options in later lines of therapy to optimize outcomes among pts with advanced/recurrent EC.[Table: see text]


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18693-e18693
Author(s):  
Eric M. Maiese ◽  
Bruno Émond ◽  
Jinan Liu ◽  
Marie-Hélène Lafeuille ◽  
Patrick Lefebvre ◽  
...  

e18693 Background: Among patients (pts) with endometrial cancer (EC), response rates for platinum-based regimens in the first-line (1L) setting range from 40% to 62% in clinical trials. This study describes patient characteristics, treatment patterns, time to next treatment (TTNT), and overall survival (OS) among pts with advanced/recurrent EC treated with a platinum-based regimen in a real-world setting in the US. Methods: This retrospective study used Optum Clinformatics Extended Data Mart de-identified databases from January 1, 2007, to December 31, 2019. Adult pts with advanced/recurrent EC who initiated a 1L platinum-based regimen and subsequently initiated second-line (2L) antineoplastic therapy were identified. Prior to initiation of 1L, a 12-month washout period of continuous enrollment without use of antineoplastic agents (except hormonal agents) was imposed. Kaplan-Meier (KM) rates were used to report TTNT and OS from 2L, third line (3L), and fourth line (4L), separately. Results: A total of 1878 pts with advanced/recurrent EC initiated 2L therapy following a platinum-based regimen in 1L. Among them, 739 (39.4%) pts initiated 3L and 330 (17.6%) initiated 4L or later (4L+) therapy. Median pt age was 68.0 years. More pts received platinum-based regimens (56.4%) in 2L than other options (Table). Few pts (3.3%) received immunotherapy. Among pts receiving 3L, a similar percentage of pts were treated with platinum-based (33.2%) and other chemotherapy regimens (33.8%); few pts received immunotherapy (3.0%). Among pts receiving 4L+, the most frequent treatment option was other chemotherapy (46.1%). Median TTNT was 17.7, 10.6, and 8.4 months for 2L, 3L, and 4L pts, respectively. KM rates of OS following initiation of 2L therapy at 1, 2, 3, and 4 years were 68.4%, 49.6%, 41.3%, and 33.6%, respectively, with a median OS of 23.5 months. Conclusions: Among pts with advanced/recurrent EC treated with platinum-based therapy in 1L, platinum-based regimens remain prevalent treatment choices in later lines of therapy. In this study, immunotherapy was used infrequently in 2L, 3L, and 4L+. The median TTNT decreased in later lines of therapy. This study highlights a critical need for novel, more effective treatment options in later lines of therapy to optimize outcomes among pts with advanced/recurrent EC.[Table: see text]


2013 ◽  
Vol 35 (10) ◽  
pp. 1501-1512 ◽  
Author(s):  
Machaon M. Bonafede ◽  
Barbara H Johnson ◽  
Madé Wenten ◽  
Crystal Watson

2015 ◽  
Vol 17 (5) ◽  
pp. 207-214 ◽  
Author(s):  
Michelle H. Cameron ◽  
Lisa Karstens ◽  
Phu Hoang ◽  
Dennis Bourdette ◽  
Stephen Lord

Background: Medication use is associated with falls in many populations, but the relationship between medications and falls in people with multiple sclerosis (MS) is not well understood. Methods: The number and types of medications used by 248 ambulatory adults with MS in the United States (n = 53) and Australia (n = 195) were assessed. Participants completed fall diaries for 6 months. Associations between number and type of medications reported and falls, adjusting for age, disease severity, comorbidities, sex, and country, were evaluated using multiple logistic regression. Results: Participants reported taking a median of three medications and two supplements. A total of 143 participants (58%) fell at least once in the 6 months, and 110 (44%) experienced one or more injurious falls. The adjusted relative odds of a fall or an injurious fall increased by 13% (P = .048) and 11% (P = .049), respectively, for each medication and by 43% (P = .015) and 55% (P = .001) for each neurologically active medication. Reported use of MS disease-modifying therapy was associated with 48% decreased odds of falling (P = .035) but not significantly decreased odds of injurious falls. Conclusions: Reporting use of more medications and more neurologically active medications is associated with falls and injurious falls in people with MS. Close evaluation of the need for each medication, with associated minimization of neurologically active medications in patients with MS, may help prevent falls. Use of MS disease-modifying therapies may be associated with fewer falls. This relationship needs further evaluation.


Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4114-4114
Author(s):  
Joshua Richter ◽  
Erin Singh ◽  
Megan S. Rice

Abstract Introduction: Despite numerous therapies approved for multiple myeloma (MM) in the past decade, the disease remains largely incurable. As a result, patients (pts) typically require successive lines of therapy, comprised of various combinations of drugs, to treat relapsed disease. As the treatment landscape evolves, continued real-world (RW) studies provide additional understanding of the treatment patterns of MM pts outside of clinical trials, particularly for those with unmet medical need and/or high-risk disease. In this retrospective, observational cohort study, we examined RW treatment patterns among MM pts overall and within patient subgroups classified by age, race/ethnicity, renal impairment (RI), cytogenetic risk, and 1q21 amplification, as well as patient outcomes. Methods: This study used the nationwide Flatiron Health electronic health record-derived de-identified database of MM pts treated in the United States. During the study period, January 1, 2016 to April 30, 2021, pts who had ≥1 line of therapy or whose first-line treatment was initiated after the study start were included. Treatment class regimens (lines 1-4) were classified as: proteasome inhibitor (PI)-based, immunomodulatory drug (IMiD)-based, PI + IMiD-based, chemotherapy-based, antibody-based, or other treatments. We examined patient characteristics as well as treatment patterns overall and in patient subgroups. We also assessed real-world progression-free survival (rwPFS), defined as the time from start of line therapy to the date of progression or death, by treatment regimen received. Results: At the time of data cutoff, 5465 pts received ≥1 line of therapy; 45.3% of pts were female, 57.4% were white, median age at the start of first-line therapy was 70 years (interquartile range 62-77), and 88.7% received care at community practices. A total of 14.6% had high-risk cytogenetic abnormalities, 21.0% had 1q21 amplification, 20.7% had International Staging System stage III at diagnosis and 33.2% had RI (eGFR &lt;60 mL/min/1.73 m 2) at the start of first-line therapy. The most common first-line regimens were PI + IMiD-based (53.4%), whereas 12.8% received PI-based, 11.5% IMiD-based, 13.4% chemotherapy-based, 2.8% antibody-based, and 6.1% had other treatments (Figure 1). Although uncommon in first-line therapy, antibody-based treatments were more commonly used in later lines of therapy (second-line: 21.0%; third-line: 33.7%; fourth-line: 40.0%). In first-line, pts aged ≥75 years were less likely to receive PI + IMiD-based regimens than those aged &lt;75 years (40.4% vs 60.1%) (Figure 2). Similarly, RI pts received PI + IMiD-based regimens less frequently in first-line than those without impairment (47.8% vs 65.0%). In first-line, 18.9% of pts had evidence of undergoing a transplant. Both older pts and those with RI were also less likely to receive a transplant as part of their first-line treatment (1.7% vs 27.7% and 13.9% vs 24.2% respectively). Treatment differences were less pronounced in later lines as well as by race/ethnicity, cytogenetic risk, and 1q21 amplification. In first-line therapy, rwPFS was longer for pts treated with PI + IMiD-based regimens (median [95% confidence interval], 29.5 months [27.3-31.9]). In later lines of therapy, pts treated with IMiD-based regimens had longer median rwPFS (second-line: 22.7 months [18.5-30.4]; third-line: 19.7 months [14.2-37.0]; fourth-line: 16.1 months [6.4-26.7]). Additional analyses examining newer treatment regimens will be presented. Conclusions: PI + IMiD combination therapy was the most common first-line therapy. Although not commonly used in first-line therapy, antibody-based regimens are increasingly used in later lines of therapy. Older pts as well as pts with RI were less likely to receive PI + IMiD regimens or transplant in first-line therapy. Pts receiving PI + IMiD regimens in first-line therapy had longer rwPFS. Pts receiving IMiD-based regimens had longer rwPFS in later lines. Analyses of newer treatment regimens to be presented within this dataset will provide additional insight into changes in RW treatment patterns in the era of novel agents. Figure 1 Figure 1. Disclosures Richter: BMS, Karyopharm, Antengene: Membership on an entity's Board of Directors or advisory committees; Adaptive biotechnologies: Speakers Bureau; Janssen, Celgene: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Tisch Cancer Institute: Icahn School of Medicine at Mount Sinai: Current Employment. Singh: Sanofi: Current Employment. Rice: Sanofi: Current Employment, Current holder of individual stocks in a privately-held company.


Author(s):  
Greenberg Benjamin ◽  
Scott Kolodny ◽  
Mengru Wang ◽  
Chinmay Deshpande

Abstract Background: The current landscape and treatment patterns of disease-modifying therapy (DMT) use among pediatric patients with multiple sclerosis (MS) is not yet well understood. This study examined DMT utilization and treatment patterns among pediatric patients newly diagnosed with MS. Methods: Pediatric patients (&lt;18 years) with 2 MS diagnosis claims from January 1, 2010, to December 31, 2016, were identified from the MarketScan Commercial Database. Index date was defined as the date of first MS diagnosis and patients were followed up for 1-year post-index date. Outcomes evaluated included percentage of patients who initiated treatment after MS diagnosis, different DMTs initiated, treatment discontinuation, and switching treatment over the follow-up period. Results: Of the 182,057 newly diagnosed MS patients, 288 pediatric patients (mean age: 14 years; females: 61%) were identified. Within the first year of diagnosis, 188 patients (65.3%) did not receive any DMT. The most commonly first initiated treatments were interferons and glatiramer acetate (83%), but 28% of patients switched or discontinued from first initiated treatment within 6 months of treatment initiation. Conclusions: This study suggests that a considerable proportion of pediatric MS patients remain untreated within one year. Patients most commonly initiated injectables as their first DMT. Overall approximately 1 in 3 patients failed on therapy early. Thus, the study warrants urgency in treating these patients with currently approved treatment options.


Author(s):  
Sarah P. Pourali ◽  
Yasmin Gutierrez ◽  
Alison H. Kohn ◽  
Jeffrey R. Rajkumar ◽  
Madison E. Jones ◽  
...  

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