scholarly journals Comparable Overall Survival with Rituximab-Bendamustine (R-Benda) and Rituximab-Gemcitabine-Oxaliplatin (R-GemOx) When Used As Second-Line (2L) Treatment for Diffuse Large B-Cell Lymphoma (DLBCL): A Real-World Study Using US Veterans Health Administration Data

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 1711-1711 ◽  
Author(s):  
Raluca Ionescu-Ittu ◽  
Aijing Shang ◽  
Nancy Vander Velde ◽  
Annie Guérin ◽  
Yilu Lin ◽  
...  
Keyword(s):  
Real World ◽  
Research Funding ◽  
Veterans Health Administration ◽  
Treatment Patterns ◽  
Veterans Health ◽  
Health Administration ◽  
Real World Data ◽  
Analysis Group ◽  
Kaplan Meier

Abstract Introduction: DLBCL is the most common subtype of non-Hodgkin lymphoma. R-CHOP (rituximab plus cyclophosphamide, doxorubicin, vincristine and prednisone) is established as the standard of care for patients (pts) with previously untreated DLBCL, but ~40% of pts will eventually relapse. For relapsed/refractory pts who are ineligible for transplant, clinical guidelines propose a broad spectrum of therapeutic options. However, little is known about treatment patterns and outcomes associated with 2L therapy in routine practice, particularly for pts less suitable for intensive therapy. Therefore, using real-world data, we evaluated 2L treatment patterns in DLBCL pts and overall survival (OS) in those pts who received 2L R-Benda or R-GemOx. We focused on these 2 treatments as they are typically used in the non-transplant setting in pts less suitable for aggressive therapy, and can typically be administered in an outpatient setting. Methods: DLBCL pts receiving care from the US Veterans Health Administration were identified through their electronic medical records and raw oncology domain. Pts diagnosed with DLBCL (and no prior other types of malignancies) between 2004-2016, with ≥1-month follow-up and who received 2L treatment were included. OS (defined as time from the start of 2L therapy until death) was analyzed in pts who received 2L R-Benda or R-GemOx using the Kaplan-Meier method. Surviving pts were censored at data cutoff (December 31, 2017). Univariate and multivariate Cox regression analyses were undertaken to assess the impact of 2L treatment (in particular, R-GemOx vs R-Benda) on OS. Results: A total of 2600 DLBCL pts were identified: 2039 received 1L and 702 received 1L and 2L therapy. Among the 702 pts treated with 2L therapy, regimens included R-ICE (n=77; 11.0%), R-CHOP (n=75; 10.7%), rituximab monotherapy (n=34; 4.8%), R-Benda (n=32; 4.6%), methotrexate (n=24; 3.4%), R-ESHAP (n=23; 3.3%), R-DHAP/R-EPOCH/R-GDP (n=18; 2.6%), rituximab plus cyclophosphamide-doxorubicin-vinblastine-vincristine (n=14; 2.0%), R-CVP (n=11; 1.6%), rituximab plus cyclophosphamide-etoposide-vincristine (n=11; 1.6%), and R-GemOx (n=10; 1.4%). Of the remaining pts, 267 (38.0%) received regimens with agent(s) included in the NCCN guidelines, while 106 (15.1%) received regimens with at least 1 agent not guideline-recommended. Baseline characteristics for pts treated with 2L R-Benda (n=32) or R-GemOx (n=10) are shown in Table 1. There was an imbalance between the 2 cohorts with regard to race, number of involved lymph nodes, B symptoms, Charlson Comorbidity Index score, and abnormal lactate dehydrogenase results. After 24 deaths in the R-Benda cohort and 7 deaths in the R-GemOx cohort, median OS was estimated at 11 and 13 months, respectively (Figure 1). Median follow-up time after start of 2L treatment was 11.3 and 11.7 months, respectively. The Kaplan-Meier curves of the 2 cohorts overlapped at multiple timepoints during follow-up. Respective 1-year OS rates (95% confidence interval [CI]) with R-Benda and R-GemOx were 50.0% (31.9%, 65.7%) and 60.0% (25.3%, 82.7%). Compared with R-Benda, R-GemOx did not significantly predict longer OS in either the univariate (hazard ratio [HR]: 0.94; 95% CI: 0.41, 2.19; p=0.893) or multivariate (HR: 1.07; 95% CI: 0.46, 2.50; p=0.873) analyses. Conclusions: This real-world study highlights the diversity of 2L treatment regimens used in DLBCL pts. There was no apparent difference in OS between R-Benda- and R-GemOx-treated pts and, with a median OS of approximately 1 year after 2L initiation with either regimen, there is clearly an unmet need in this setting. The main limitation of the study relates to the small sample size of each treatment cohort. Further research using other real-world data sources is warranted. Disclosures Ionescu-Ittu: Analysis Group, Inc.: Employment; F. Hoffman-La Roche Ltd: Consultancy, Other: I am an employee of Analysis Group, Inc., which received consulting fees from Roche for the conduct of this study. Shang:F. Hoffmann-La Roche Ltd.: Employment, Other: Ownership interests non-PLC. Guérin:F. Hoffman-La Roche Ltd: Other: I am an employee of Analysis Group, Inc., which received consulting fees from Roche for the conduct of this study; Analysis Group, Inc.: Employment. Shi:F. Hoffman-La Roche Ltd: Research Funding; Bravo4Health: Other: Ownership interests non-PLC; Genentech: Research Funding; Chiasma: Research Funding; Intuitive Surgical: Consultancy. Shi:F. Hoffman-La Roche Ltd: Other: I am an employee of Analysis Group, Inc., which received consulting fees from Roche for the conduct of this study; Analysis Group, Inc.: Employment. Qayum:F. Hoffmann-La Roche Ltd: Employment.

scholarly journals Mortality Among Veterans with a Diagnosis of Pyruvate Kinase (PK) Deficiency: A Real-World Study Using US Veterans Health Administration Data

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 24-25
Author(s):  
Erin Zagadailov ◽  
Audra Boscoe ◽  
Viviana Garcia-Horton ◽  
Sherry Shi ◽  
Shuqian Liu ◽  
...  
Keyword(s):  
Pyruvate Kinase ◽  
Real World ◽  
Index Date ◽  
Veterans Health ◽  
Health Administration ◽  
Real World Data ◽  
Analysis Group ◽  
Diagnosis Codes ◽  
Icd 10

Background: Pyruvate kinase (PK) deficiency is a rare, inherited disorder caused by autosomal recessive mutations in the PKLR gene, whereby a glycolytic defect causes reduced adenosine triphosphate levels and leads to hemolytic anemia. Patients with PK deficiency can experience serious complications associated with the disease and its treatment, including osteoporosis, pulmonary hypertension, sepsis, iron overload, and liver cirrhosis. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, iron chelation therapy and/or interventions for other disease-related morbidity. There is no approved therapy for this condition. Identifying PK deficiency in real-world data is challenging due to a lack of diagnosis codes and treatments that are specific to PK deficiency. As a result, population-based studies of PK deficiency using claims or electronic health record databases are limited. In addition, data on mortality in this patient population are lacking and limited to a few individual case reports. This study aimed to identify patients with a PK deficiency diagnosis as documented by physicians and to compare their mortality to an age- and gender-matched cohort of individuals without PK deficiency. Methods: Patients with ≥ 1 diagnosis code related to PK deficiency (i.e., anemia due to disorders of glycolytic enzymes [International Classification of Diseases, Tenth Revision, Clinical Modification (ICD-10-CM) D55.2], other hemolytic anemias due to enzyme deficiency [ICD-9-CM 282.3], or unspecified hereditary hemolytic anemia [ICD-9-CM 282.9, ICD-10-CM D58.9]) between January 1995 and July 2019 were selected from the US Veterans Health Administration (VHA) database. To be considered for inclusion, physicians' notes were required to contain the words "pyruvate", "kinase", and "deficiency." A manual review of these physicians' notes was performed to identify patients with a physician-documented diagnosis of PK deficiency (PK deficiency cohort). The index date for the PK deficiency cohort was defined as the date of the first medical record with a diagnosis code related to PK deficiency. Each patient in the PK deficiency cohort was matched 1:5 by age at index, sex, and index year (± 1 year) to patients from the general VHA population with no diagnosis codes related to PK deficiency (non-PK deficiency cohort). The index date for the non-PK deficiency cohort was defined as a random visit date during their match's index year. Survival time from the index date was compared between the PK deficiency cohort and their non-PK deficiency cohort matches using a univariate Cox proportional hazards model with robust standard error estimation. Results: A total of 18 patients met inclusion criteria for the PK deficiency cohort and were matched to 90 individuals in the non-PK deficiency cohort. Baseline characteristics for both cohorts are shown in the Table. For both cohorts, the mean age at index was 57 years and 94% of patients were male; 83-85% were white and the mean Charlson Comorbidity Index score was 0.4-0.5 (no significant differences between cohorts). Imbalances remained between the two cohorts with regard to region and body mass index. The median follow-up was 6.0 years for the PK deficiency cohort and 8.0 years for the non-PK deficiency cohort. Over the follow-up period, there were 9 (50%) observed deaths in the PK deficiency cohort and 28 (31%) observed deaths in the non-PK deficiency cohort. The median time until death was 10.9 years for the PK deficiency cohort and 17.1 years for the non-PK deficiency cohort; the Kaplan-Meier curves for both cohorts are shown in the Figure. Patients in the non-PK deficiency cohort had a significantly longer time to death than the PK deficiency cohort (hazard ratio: 2.3; p = 0.0306); 10 years after index, 42% of patients in the PK deficiency cohort had died compared with 28% of those in the non-PK deficiency cohort. Conclusions: The results of this study suggest that patients with PK deficiency may be at an increased risk of mortality. Further research to understand cause of death in this population is warranted, as is the replication of this study using larger sample sizes and other real-world data sources that better represent females and the pediatric and adolescent PK deficiency age groups. Disclosures Zagadailov: Agios Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company. Boscoe:Agios Pharmaceuticals: Current Employment, Current equity holder in private company. Garcia-Horton:Agios Pharmaceuticals: Consultancy; Analysis Group, Inc.: Current Employment. Shi:Agios Pharmaceuticals: Consultancy; Analysis Group, Inc.: Current Employment. Liu:Analysis Group, Inc.: Consultancy. Shi:Analysis Group, Inc.: Consultancy. Macaulay:Agios Pharmaceuticals: Consultancy; Analysis Group, Inc.: Current Employment.

scholarly journals Treatment Patterns and Outcomes of 159 Ibrutinib-Treated MCL Patients in the United States: A Retrospective Electronic Medical Record Database and Chart Review Study

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4163-4163
Author(s):  
Jeff Sharman ◽  
Shaum Kabadi ◽  
Jamyia Clark ◽  
E Susan Amirian ◽  
David J. Andorsky
Keyword(s):  
Real World ◽  
Research Funding ◽  
Chart Review ◽  
Kinase Inhibitor ◽  
Stage Iv ◽  
The United States ◽  
Treatment Patterns ◽  
Discontinuation Rate ◽  
Real World Data

Abstract Introduction Ibrutinib, a Bruton's tyrosine kinase inhibitor, was approved in the U.S. for the treatment of relapsed or refractory mantle cell lymphoma (MCL) in November of 2013. However, real-world data on ibrutinib use for the treatment of MCL is limited. The purpose of this study was to examine ibrutinib use, dosages, and reasons for treatment discontinuation among MCL patients treated in a community oncology practice setting. Methods The study population consisted of adult (≥18 year old) MCL patients treated with ibrutinib between November 1, 2013 and October 31, 2016, who were not enrolled in a clinical trial and had at least 2 visits to a US Oncology Network (USON) clinic. Patients with other primary cancers were excluded. Patient data were sourced from the USON's electronic health records system, iKnowMed (iKM)™. The structured iKM database provided information on demographics and clinical and treatment characteristics. Manual chart review was used to confirm ibrutinib treatment patterns. Duration of ibrutinib therapy (DOT), overall survival (OS), and progression-free survival (PFS) from systemic treatment initiation were estimated using Kaplan-Meier methods. Events were defined as death in the OS analysis, and progression or death in the PFS analysis. Patients were censored if their treatment was ongoing for DOT. Censors for OS and PFS were patients lost to follow up or those who did not experience a failure event within the study period. Results 159 eligible MCL patients were identified through iKM. The majority of patients were Caucasian (n=141, 88.7%), male (n=121, 76.1%), and diagnosed with Stage IV disease (n=117, 73.6%). Median follow-up for the population was 16.1 months. Approximately 7.5% (n=12) of patients received ibrutinib as first-line therapy (1L), compared to 54.1% (n=86) in 2L and 38.4% (n=61) in 3L or beyond. Median ibrutinib dose at initiation was 560mg (range: 140-700). During ibrutinib treatment, 16.4% (n=26) of patients experienced a dose reduction. Dose holds occurred in 30.2% (n=48), 66.7% (n=32) due toxicities. The overall discontinuation rate was 83.6% The primary reason for discontinuation was disease progression (n=46, 34.6%) followed by toxicities (n=34, 25.6%). Median DOT was higher for patients initiating treatment in 3L+ (14.9: 95% CI 8.8-17.1) compared to other lines. Median PFS was 19.6 (95% CI: 16.5-24.3) for the overall population and median OS was 25.8 months (95% CI: 19.9-not reached). Conclusions Our real-world findings on survival are consistent with those from clinical trials on ibrutinib in relapsed/refractory MCL, although our observed discontinuation rate (~84%) was higher than that of the trial (~58%), which had a similar median follow-up time (16.1 months vs. 15.3 months, respectively). Our findings provide additional data on MCL treatment patterns and patient outcomes in clinical practice. Disclosures Sharman: Acerta: Consultancy, Research Funding; Pharmacyclics, an AbbVie Company: Consultancy, Research Funding. Kabadi:AstraZeneca: Employment. Clark:McKesson Specialty Health: Employment, Equity Ownership. Amirian:McKesson Specialty Health: Employment. Andorsky:Celgene: Research Funding; CTI BioPharma: Consultancy, Research Funding; AstraZeneca: Consultancy; Genentech: Consultancy.

P5669Clinical management of syncope patients guided by insertable cardiac monitors in Europe

2019 ◽  
Vol 40 (Supplement_1) ◽  
Author(s):  
W Moyson ◽  
A Verma ◽  
A Natale ◽  
A Amin ◽  
S Beinart ◽  
...  
Keyword(s):  
Real World ◽  
Recurrent Event ◽  
Battery Life ◽  
Real World Data ◽  
European Guidelines ◽  
Kaplan Meier ◽  
Clinical Action ◽  
Unexplained Syncope ◽  
Multi Center Study

Abstract Introduction Current European guidelines recommend the use of insertable cardiac monitors (ICMs) in the early phase of evaluation for patients with recurrent, unexplained syncope and a likelihood of recurrence during the device battery life. The ongoing Reveal LINQ Registry collects real-world data on clinical actions guided by the insertable cardiac monitor (ICM) in these patients. Purpose To characterize ICM-guided diagnosis and treatment of syncope patients in Europe. Methods The Reveal LINQ Registry is a prospective, observational, multi-center study evaluating real-world performance of the ICM. Patients from European centres who had syncope indicated as the reason for ICM implant were included in the analysis. Kaplan-Meier methods were used to determine the percentage of patients receiving treatment. Results In total, 139 patients from 12 centres [Portugal (78); The Netherlands (30); Belgium (12); Italy (10); Germany (5); United Kingdom (4)] received an ICM to monitor for unexplained syncope and had at least one follow-up visit. The mean age was 61.2 and 51.1% were female. During a mean follow-up time of 11.2 months, 31 patients experienced at least one recurrent syncope event classified as: cardiac (9), vasovagal (4), idiopathic (8) and unknown (10). An arrhythmia was diagnosed in 14 (45.2%) patients with a recurrent event and clinical actions were taken in 12 (38.7%) patients (10 IPGs, and 2 AF ablations). Among patients without a recurrence, 25 (23.1%) had an arrhythmia diagnosed and 20 (18.5%) had a clinical action taken (13 therapeutic device implants, 4 ablations, and 3 medication changes). Overall, the rate of clinical actions was 32.6% after 18 months of ICM follow-up in the unexplained syncope population (Figure). Time to treatment Conclusion Clinical actions are taken in approximately one third of unexplained syncope patients monitored with an ICM after 18 months of follow up. Acknowledgement/Funding Medtronic

Shifting Treatment Patterns and Clinical Outcomes in Veterans Diagnosed with Waldenstrom Macroglobulinemia from 2006 to 2018

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 12-12
Author(s):  
Hsu-Chih Chien ◽  
Deborah Kay Morreall ◽  
Vikas Patil ◽  
Kelli M Rasmussen ◽  
Christina Yong ◽  
...  
Keyword(s):  
Overall Survival ◽  
Clinical Outcomes ◽  
Real World ◽  
Research Funding ◽  
Practice Patterns ◽  
Treatment Patterns ◽  
Clinical Settings ◽  
Treatment Groups ◽  
Treatment Regimens

Background Waldenström's Macroglobulinemia (WM) is a rare indolent lymphoma with an estimated 1,500 new cases diagnosed each year in the United States (US). Over the last decade, several treatments have been introduced into the WM therapeutics landscape including, bendamustine, bortezomib, and most recently oral Bruton's kinase inhibitor (ibrutinib). There is limited information in the adoption of these WM treatments in real-world clinical settings in the US. We describe the practice patterns and clinical outcomes of first-line (1L) treatment of WM in a nationwide cohort of Veterans. Methods Using Veteran Affairs electronic health records (EHR) data, we identified Veterans who were diagnosed and received 1L treatment for WM between January 2006 and December 2018 in the Veterans Health Administration (VHA). Human annotation of EHR clinical records confirmed the diagnosis and 1L treatment regimens. Patients with another cancer diagnosis or patients with documentation that 1L treatment was received outside the VHA were excluded. Eligible patients were followed until loss to follow-up, death, or the end of the study period (June 30, 2019). Patient demographics, disease characteristics, and treatment patterns were identified. Local polynomial regression model curves were generated to demonstrate treatment changes over time. Unadjusted progression-free survival (PFS) and the unadjusted overall survival (OS) are also provided. Results We identified 505 patients diagnosed with WM in VHA between January 2006 thru December 2018. Of these, 318 patients received 1L treatment, with a median time from diagnosis to 1L treatment of 1.2 months (95% confidence interval [CI]: 0.5-5 months). The median age of WM patients was 69.9 years (standard deviation [SD]: 9.4 years), with approximately 73% of WM patients ≥65 years old. Prior to 1L treatment, the median hemoglobin and platelets observed were similar across all treatment groups, regardless of first 1L treatment. However, the median immunoglobulin M (IgM) was substantially lower in patient's treated with ibrutinib (2,570 mg/dL [range: 422-9,001 mg/dL]) and single-agent rituximab (R), 2,855 mg/dL (range: 84-7,880 mg/dL) when compared to those treated with chlorambucil +/- rituximab (4,416 mg/dL [range: (9-8,130 mg/dL]) and bortezomib/dexamethasone +/- rituximab (BDR), 4,086 mg/dL (range: 16-9,944 mg/dL). MYD88 testing occurred in 40 (13%) of patients, with testing most frequently occurring in patients treated with bendamustine +/- rituximab (BR), ibrutinib, and BDR- likely reflecting increased adoption in later periods. Hepatitis C testing occurred in 61 (19%) of patients, with testing most frequently occurring in patients treated with dexamethasone, rituximab, and cyclophosphamide (DRC), BDR, and BR. Over the study observation period, 1L practice patterns shift significantly with increased adoption of BR, BDR and ibrutinib and de-adoption of chemotherapy (Figure 1). The median follow-up time for all patients was 44 months (range: 1-147 months), although a shorter median follow-up time was observed in patients treated with therapeutics in recent years, such as ibrutinib (18 months [range: 2-53 months]) and BR (23 months [range: 4-86 months]). The median unadjusted PFS for all WM patients was 44 months (95% CI: 37-58 months) and the median unadjusted overall survival (OS) was 94 months (95% CI: 82-117 months). Conclusions The introduction of numerous therapeutic options throughout the past decade has profoundly altered the treatment landscape for WM, suggesting a shift in 1L practices from chlorambucil to BDR, BR, and most recently ibrutinib which has been increasingly adopted, since its approval in 2015, especially in older patients, suggesting that it may provide an effective therapeutic option for patients who may not be able to tolerate more aggressive treatment regimens. Limitations of this study include the differences observed in follow-up time as well as the limited number of patients in some 1L treatment groups. Further research is required to establish the long-term benefits and potential treatment-related toxicities of WM treatments in real-world clinical settings. Disclosures Sauer: Roche: Research Funding; Genentech, Inc.: Research Funding; Pharmacyclics: Research Funding; AbbVie: Research Funding. Halwani:AbbVie: Research Funding; Takeda: Research Funding; Roche: Research Funding; Genentech, Inc.: Research Funding; Miragen: Research Funding; Immunedesign: Research Funding; Kyowa Hakko Kirin: Research Funding; Seattle Genetics: Research Funding; Amgen: Research Funding; Pharmacyclics: Research Funding; Bristol Myers Squibb: Research Funding.

scholarly journals Real-World Outcomes for Standard-of-Care Treatments in Patients with Relapsed/Refractory Multiple Myeloma

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4075-4075
Author(s):  
Michel Delforge ◽  
Marie-Christiane Vekemans ◽  
Sébastien Anguille ◽  
Julien Depaus ◽  
Nathalie Meuleman ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Board Of Directors ◽  
Real World ◽  
Research Funding ◽  
Retrospective Chart Review ◽  
Standard Of Care ◽  
Real World Data ◽  
Advisory Committees ◽  
Treatment Regimens

Abstract Background: With the advent of immunomodulatory agents (IMiDs), proteasome inhibitors (PIs) and, more recently, anti-CD38 monoclonal antibodies (mAbs), prognosis of patients with multiple myeloma (MM) has improved considerably. Unfortunately, even with these 3 major MM drug classes, most patients ultimately relapse and require further therapy. There remains an incomplete understanding of how patients who have received extensive therapy and with relapsed/refractory multiple myeloma (RRMM) are treated in routine clinical practice, as no standard-of-care exists for these patients, and what the outcomes are in this real-world setting. Objective: This study aims to evaluate the outcomes of patients with triple-class (IMiD, PI and anti-CD38 mAb) and triple-line exposed RRMM using real-world data from patients in Belgium. Methods: A multicenter, observational study, involving 7 non-academic and academic Belgian centers, was conducted based on a retrospective chart review of adult RRMM patients who started subsequent treatment from March 2017 through May 2021 after having received ≥3 lines of therapy including at least an IMiD, a PI, and anti-CD38-directed therapy (tri-exposed). Data were captured in an electronic case report form (Castor EDC). Patients with an ECOG performance status of ≥2, who received prior CAR-T treatment or prior BCMA-targeted therapy, or with a known active or prior history of CNS involvement (or with clinical signs thereof), were excluded. All treatment lines initiated after becoming eligible were used in the analysis. Specifically, all treatment lines for patients meeting the eligibility criteria more than once in their entire follow-up were included as separate observations, with date of treatment initiation as specific baseline for each treatment line. Cox proportional hazards models were fitted to explore the prognostic value with Overall Survival (OS), Progression Free Survival (PFS), and Time to Next Therapy (TTNT). Results: A total of 112 patients with 237 eligible treatment lines were included in the analysis; median follow-up was 16.6 months. In 45% of the initiated treatment lines, patients were refractory to 4 or 5 therapies, 62% had received ≥5 prior lines, 22% had extramedullary disease and in 48% of observations the time to progression in prior line was shorter than 4 months. After patients became tri-exposed, more than 50 unique treatment regimens were initiated, with the following being the most common: carfilzomib + dexamethasone (14%), pomalidomide + dexamethasone + chemotherapy (8%), and ixazomib + lenalidomide + dexamethasone (6%). Additionally, 4% of included observations were exposed to anti-BCMA agents. Overall, the following treatment classes were the most frequently started: PI only (19%), PI + IMiD combinations (17%), and regimens including anti-CD38 antibodies (15%). Median OS was 9.79 months [95% CI: 7.79; 12.22], median PFS was 3.42 months [95% CI: 2.79; 4.27], median TTNT was 3.61 months [95% CI: 3.09; 4.57]. Higher refractory status (p<0.001), being male (p=0.001), older age (p<0.001), shorter duration of prior lines (p<0.001), shorter time to progression in prior line (p=0.025), and higher LDH levels (p<0.002) were prognostic for worse outcomes for both OS (Figure 1) and PFS. Conclusions: This retrospective chart review of patients with tri-exposed RRMM in Belgium shows that real-world outcomes in terms of OS, PFS and TTNT are poor for these patients, with a median OS of <10 months. A wide variety of treatment regimens used in clinical practice confirm the absence of a clear standard-of-care in this patient population. The literature also confirms that these poor outcomes observed in Belgium, for this subset of MM patients, are similar in other countries. These real-world data highlight the high unmet medical need in this patient population and critical need for new and effective treatment options. MD and MCV contributed equally to this work. Figure 1 Figure 1. Disclosures Delforge: Amgen, Celgene, Janssen, Sanofi: Honoraria, Research Funding. Vekemans: Amgen: Membership on an entity's Board of Directors or advisory committees; Takeda: Membership on an entity's Board of Directors or advisory committees; BMS-Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen Pharmaceutica: Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi: Membership on an entity's Board of Directors or advisory committees. Depaus: Takeda: Consultancy; Novartis: Consultancy; Janssen: Consultancy; Celgene: Consultancy. Meuleman: iTeos Therapeutics: Consultancy. Strens: Realidad bvba: Consultancy. Van Hoorenbeeck: Janssen: Current Employment. Moorkens: Janssen-Cilag: Current Employment. Diels: Janssen: Current Employment. Ghilotti: Janssen-Cilag SpA, Cologno Monzese, Italy: Current Employment. Dalhuisen: Janssen: Current Employment. Vandervennet: Janssen: Current Employment.

scholarly journals Treatment Patterns and Outcomes of Patients with Double-Class Refractory or Triple-Class Refractory Multiple Myeloma: A Retrospective US Electronic Health Record Database Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 2705-2705
Author(s):  
Feng Wang ◽  
Boris Gorsh ◽  
Maral DerSarkissian ◽  
Prani Paka ◽  
Rachel Bhak ◽  
...  
Keyword(s):  
Research Funding ◽  
Performance Status ◽  
Treatment Patterns ◽  
Multiple Drug ◽  
Publicly Traded ◽  
Analysis Group ◽  
Refractory Status ◽  
Drug Classes ◽  
Immunomodulatory Drug

Abstract Introduction: Patients with relapsed/refractory multiple myeloma (RRMM) resistant to multiple drug classes remain a high unmet need population, despite advances in MM patient care. The objective of this study was to assess real-world treatment patterns and outcomes in patients with RRMM who had failed multiple prior lines of therapy (LOT) and were refractory to multiple drug classes to identify gaps in their treatment pathways. Methods: This longitudinal retrospective cohort study utilized the COTA de-identified real-world database derived from US electronic health records of partnered healthcare providers from Q3 1988 through Q1 2020. Adults with active RRMM previously exposed to a proteasome inhibitor (PI) and an immunomodulatory drug and who received ≥3 prior LOTs were identified. Patients were further categorized as refractory to a PI and an immunomodulatory drug (double-class refractory [DCR]) or additionally to an anti-CD38 monoclonal antibody (i.e. daratumumab; triple-class refractory [TCR]). Determining refractory status was based on International Myeloma Working Group criteria. Index LOT was a new LOT after evidence of DCR or TCR status following ≥3 (DCR) or ≥4 (TCR) prior LOTs. Patient characteristics described included Eastern Cooperative Oncology Group (ECOG) performance status, International Staging System (ISS) stage, cytogenetic risk, age, index date, sex, and follow up time. Treatment pattern assessments included treatments received before/during/after index LOT, reasons for discontinuation, refractory status, and retreatment characteristics. Patient outcomes (overall survival [OS], duration of treatment [DOT], and time to next therapy [TTNT]) were analyzed using Kaplan-Meier survival analysis methods. Results: After excluding patients who were aged <18 years at start of index LOT with no evidence of clinical activity and who participated in a clinical trial during index LOT, 381 (DCR) and 173 (TCR) patients were available for analysis. Median follow-up from index LOT initiation through end of data availability/death was 14 (DCR) and 8 (TCR) months. Demographic characteristics were consistent between DCR and TCR patients. Approximately half were aged ≥65 years (49% DCR; 53% TCR), majority had high-risk cytogenetics (56% DCR; 66% TCR) or prior autologous stem cell transplantation (>62%), and 14-16% had ISS stage III. Patients had a median of 3 (DCR) and 6 (TCR) prior LOTs. Prior to index LOT, bortezomib and lenalidomide were the most commonly received PI and immunomodulatory drug (received by >98% of DCR or TCR patients) and the most common PI and immunomodulatory drugs to which patients were refractory (71% and 84% DCR; 76% and 83% TCR, respectively). At index LOT, PI/immunomodulatory drug-based and daratumumab-based therapies remained the most common therapies. Bortezomib and lenalidomide had the longest time to refractory status and highest retreatment rates among DCR or TCR patients. Approximately 40% of TCR patients were retreated with daratumumab-based therapies after becoming refractory (Table). After index LOT, 70% of DCR and 58% of TCR patients continued to a subsequent LOT. Patients most frequently discontinued index LOT due to disease progression (59% DCR; 60% TCR), toxicity (23% DCR; 25% TCR), or doctor preference (14% DCR; 10% TCR). Median duration of gaps between LOTs generally were shorter than 1 month (Table). Median OS was 22.3 (DCR) and 11.6 (TCR) months. Median DOT was 3.3 (DCR) and 2.8 (TCR) months, and median TTNT was 4.1 (DCR) and 3.2 (TCR) months. In multivariate statistical analyses, inferior baseline ECOG performance scores, and high-risk cytogenetic abnormalities were associated with worse prognosis (higher risk of death, shorter DOT, and shorter TTNT) in DCR and TCR patients. Age was not a significant factor after adjusting for other baseline factors. Conclusions: Treatment options are limited for US patients with DCR and TCR RRMM. DCR and TCR patients were frequently retreated with a PI, an immunomodulatory drug, or daratumumab, despite refractoriness to these agents. Many DCR and TCR MM patients stopped active MM treatment after discontinuing index treatment. Patients with DCR and TCR MM have poor prognosis, especially among high cytogenetic risk and poor performance status patients. This study provides the benchmark for new therapies, like BCMA-targeted agents, to be tested in this population. Funding: GSK (Study 217353). Figure 1 Figure 1. Disclosures Wang: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Gorsh: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. DerSarkissian: Analysis Group, Inc.: Current Employment. Paka: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Bhak: Analysis Group, Inc.: Current Employment; GlaxoSmithKline: Research Funding. Boytsov: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Zichlin: GlaxoSmithKline: Research Funding; Analysis Group, Inc.: Consultancy, Current Employment. Sansbury: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Yee: Analysis Group, Inc.: Current Employment; GlaxoSmithKline: Research Funding. Ferrante: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Khanal: GlaxoSmithKline: Research Funding; Analysis Group, Inc.: Current Employment. Noman: Analysis Group, Inc.: Current Employment; GlaxoSmithKline: Research Funding. Duh: Novartis: Other: I am an employee of Analysis Group, a consulting company that received funding from Novartis for this research study..

scholarly journals Real-World Treatment Patterns Among a Contemporary Cohort of Commercially Insured Mantle Cell Lymphoma Patients in the United States

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4845-4845
Author(s):  
Lindsey E. Roeker ◽  
Shaum Kabadi ◽  
Chakkarin Burudpakdee ◽  
Aimee Near ◽  
Keiko Wada ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Real World ◽  
Research Funding ◽  
Index Date ◽  
Treatment Patterns ◽  
Treatment Regimens ◽  
Oral Medications ◽  
Mantle Cell ◽  
Insured Patients

Abstract Introduction Mantle cell lymphoma (MCL) is a rare, aggressive non-Hodgkin lymphoma associated with a poor prognosis. The approval of ibrutinib in November 2013 has changed the treatment paradigm for patients with relapsed or refractory MCL. There remains a lack of information on the current treatment patterns used in clinical practice in a contemporary cohort of commercially insured patients. We aimed to identify the treatment patterns for MCL overall and by line of therapy (LOT) and to describe patient demographics and clinical characteristics in a large cohort of commercially insured MCL patients. Methods A retrospective cohort study was conducted with the IQVIA Real-World Data Adjudicated Claims-US database. Adult patients (≥18 years old) with ≥1 claim for a NCCN-recommended MCL treatment between November 1, 2013 and December 31, 2017 were identified. Index date was the first treatment claim. Patients were also required to have ≥1 diagnosis of MCL during the study period (November 1, 2012 to January 31, 2018), ≥12 months of continuous enrollment prior to index date (pre-index period) and ≥30 days after index date (follow-up period). Patients were excluded if they were ≥65 years at index and not enrolled in Medicare Risk or Medicare Cost, enrolled in a clinical trial during the study period, had evidence of MCL treatment in the pre-index period (except for patients indexed on ibrutinib as it is indicated for MCL patients with ≥1 prior treatment), or had evidence of stem cell transplant (SCT) before index date. The most commonly observed MCL treatment regimens were identified, and demographic and clinical characteristics of patients and treatment durations by regimen were described. Treatment regimen was defined as the combination of all agents observed in the 35-day period after the first MCL treatment claim; treatment duration was defined as the start of treatment until a gap of ≥90 days between end date and next date of treatment or treatment modification. Treatment end date occurred 90 days after the end of the supply for oral medications or 30 days after the last administration for non-oral medications. Results There were 1,785 patients treated with the most commonly observed MCL treatment regimens. The most common regimens, irrespective of LOT, were rituximab monotherapy (including maintenance therapy; n=773, 43.3%), R-CHOP (n=723, 40.5%), B-R (n=436, 24.4%), and ibrutinib monotherapy (n=199, 11.1%). Overall, patients had a median (IQR) age of 57 (52-62) years, and 59.4% were male. Most patients were commercially or self- insured (57.5% and 33.6%, respectively). Patients had a median Charlson Comorbidity Index (CCI) of 0 (IQR 0-1; mean [SD] 0.9 [1.4]), with the most common CCI components being diabetes (15.7%), chronic pulmonary disease (12.8%), and congestive heart failure (9.5%). During the follow-up period (median [IQR] 22.5 [10.5-35.3] months), in addition to the MCL regimen(s), patients received radiation therapy (17.4%), SCT (10.0%), and/or immunotherapy (0.2%). The use of targeted therapies (i.e. lenalidomide, bortezomib) other than ibrutinib was infrequent. When considering treatment lines, R-CHOP was the most commonly observed first regimen, followed by rituximab, B-R, and ibrutinib; for the second and third observed regimens, rituximab was the most common, followed by ibrutinib (Figure 1). The median (IQR) duration for the first observed regimen was 8.1 (3.9-18.0) months for ibrutinib, 5.0 (3.3-5.6) months for B-R, 4.0 (2.5-4.4) months for R-CHOP, and 1.9 (1.7-4.4) months for rituximab; ibrutinib also had the longest duration in the second and third line (median [IQR] 5.5 [2.4-13.5] months and 8.3 [3.9-12.4] months, respectively). Conclusion This is the largest study of MCL patients describing treatment patterns in current clinical practice among commercially insured patients. MCL patients were most commonly treated with chemoimmunotherapy for all treatment lines while ibrutinib was the second most common LOT2 and LOT3 regimen. As the treatment landscape and clinical practice continues to change with the use of novel agents, future studies are warranted to further study toxicities and outcomes in the real-world setting. Disclosures Kabadi: AstraZeneca: Employment. Burudpakdee:IQVIA received funds from AstraZeneca to conduct the analysis, interpret data, and coauthor the publication.: Consultancy. Near:IQVIA received funds from AstraZeneca to conduct the analysis, interpret data, and coauthor the publication.: Consultancy. Wada:IQVIA received funds from AstraZeneca to conduct the analysis, interpret data, and coauthor the publication.: Consultancy. Mato:TG Therapeutics: Research Funding; Sunesis: Honoraria, Research Funding; Acerta: Research Funding; Janssen: Consultancy, Honoraria; AstraZeneca: Consultancy; Pharmacyclics: Consultancy, Honoraria, Research Funding; Regeneron: Research Funding; Celgene: Consultancy; Prime Oncology: Speakers Bureau; Abbvie: Consultancy.

scholarly journals Mild TBI and risk of Parkinson disease

Neurology ◽  
2018 ◽  
Vol 90 (20) ◽  
pp. e1771-e1779 ◽  
Author(s):  
Raquel C. Gardner ◽  
Amy L. Byers ◽  
Deborah E. Barnes ◽  
Yixia Li ◽  
John Boscardin ◽  
...  
Keyword(s):  
Brain Injury ◽  
Parkinson Disease ◽  
Veterans Health Administration ◽  
Veterans Health ◽  
Health Administration ◽  
Mild Tbi ◽  
Psychiatric Comorbidities ◽  
Increased Risk ◽  
Severe Tbi

ObjectiveOur aim was to assess risk of Parkinson disease (PD) following traumatic brain injury (TBI), including specifically mild TBI (mTBI), among care recipients in the Veterans Health Administration.MethodsIn this retrospective cohort study, we identified all patients with a TBI diagnosis in Veterans Health Administration databases from October 2002 to September 2014 and age-matched 1:1 to a random sample of patients without TBI. All patients were aged 18 years and older without PD or dementia at baseline. TBI exposure and severity were determined via detailed clinical assessments or ICD-9 codes using Department of Defense and Defense and Veterans Brain Injury Center criteria. Baseline comorbidities and incident PD more than 1 year post-TBI were identified using ICD-9 codes. Risk of PD after TBI was assessed using Cox proportional hazard models adjusted for demographics and medical/psychiatric comorbidities.ResultsAmong 325,870 patients (half with TBI; average age 47.9 ± 17.4 years; average follow-up 4.6 years), 1,462 were diagnosed with PD during follow-up. Compared to no TBI, those with TBI had higher incidence of PD (no TBI 0.31%, all-severity TBI 0.58%, mTBI 0.47%, moderate-severe TBI 0.75%). In adjusted models, all-severity TBI, mTBI, and moderate-severe TBI were associated with increased risk of PD (hazard ratio [95% confidence interval]: all-severity TBI 1.71 [1.53–1.92]; mTBI 1.56 [1.35–1.80]; moderate-severe TBI 1.83 [1.61–2.07]).ConclusionsAmong military veterans, mTBI is associated with 56% increased risk of PD, even after adjusting for demographics and medical/psychiatric comorbidities. This study highlights the importance of TBI prevention, long-term follow-up of TBI-exposed veterans, and the need to determine mechanisms and modifiable risk factors for post-TBI PD.

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