scholarly journals Real World Data (RWD) Treatment Patterns and Sequencing of Patients with Multiple Myeloma (MM)

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 46-46
Author(s):  
Anda Gershon ◽  
Yutong Liu ◽  
Spencer James ◽  
Mellissa Williamson ◽  
Wan-Jen Hong ◽  
...  
Keyword(s):  
Treatment Patterns ◽  
Cancer Type ◽  
Cell Transplant ◽  
Publicly Traded ◽  
Real World Data ◽  
Nccn Guidelines ◽  
Incurable Disease ◽  
Treatment Category ◽  
Health Database ◽  
Standard Risk

Introduction: Despite advances in treatment, MM remains an incurable disease. RWD are key to improving our understanding of treatment patterns for patients with MM. This study examined the proportion of patients with MM (1) treated with an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), an anti-CD38 monoclonal antibody (aCD38), or Other in first line (1L) through fourth line or greater (4L+) by calendar year; and (2) who had a change in treatment regimen at line change. Subgroup analyses were performed by cytogenetic risk status (high vs. standard), stem cell transplant (SCT) eligibility (eligible vs. ineligible), and practice type (community vs. academic). Methods: The Flatiron Health Database was used, comprising de-identified EHR-derived data for patients treated in the Flatiron Health network. Patients were excluded if they had no record of a 1L treatment recognized as an induction regimen based on NCCN guidelines, a 1L regimen that contained an active therapy for another cancer type, 1L initiated >60 days after MM diagnosis, and <3 consecutive months of data. Treatments were classified into 15 mutually exclusive categories (inclusive of steroids): Chemotherapy, IMiD, IMiD+chemo, PI, PI+chemo, IMiD+PI, IMiD+PI+chemo, aCD38, aCD38+chemo, aCD38+IMiD, aCD38+IMiD+chemo, aCD38+PI, aCD38+PI+chemo, aCD38+IMiD+PI, and Other. Treatment use and sequencing patterns were assessed using proportions. Results: 5890 adult patients diagnosed with MM between 1/1/2011 and 12/31/19 were included. 45% of patients were female, 60% were white, and the median age was 69 (IQR = 61-76). Nearly 90% of the cohort were from community practices. Across years and lines, the most common treatment categories were IMiD, PI, and IMiD+PI. After 2015, IMiD+ PI was the dominant category in 1-3L and IMiD was the dominant category in 4L+. After 2015 and across lines, the most common aCD38 containing treatment was aCD38+IMiD. After 2015, aCD38 treatments were used in 2-4% of 1L patients regardless of subgroup. Between 2015 and 2019, the use of aCD38 treatments in 2L increased from 1% to 20% in all, standard risk, SCT eligible, and community patients, 2% to 15% in SCT ineligible, and 10% to 20% academic patients. Between 2015 to 2019, the use of aCD38 treatments in 3L increased from 10% to >30% in all, standard risk and SCT eligible patients, 10% to 20% in academic patients, and 2% to 20% in community and SCT ineligible patients. Over 40% of 4L+ patients were treated with aCD38 treatments by 2019 regardless of subgroup. As shown in Figure 1, of the patients receiving an IMiD, PI, or IMiD+PI in 1-3L in 2015-2019, 10-25% stayed with the same treatment category (though likely switched drugs within the class), whereas 25-50% switched to a different treatment category, in the next line. These proportions generally hold regardless of subgroup. Of the patients receiving an aCD38 regimen in 1-2L in 2015-2019, 10-20% stayed with the same treatment category, 5-40% switched to a different treatment category, and 5-25% stayed with an aCD38 regimen but switched to a different combination in the next line. Of 3L patients receiving an aCD38 regimen, under 15% stayed with the same treatment category, 5-40% switched to a different treatment category, and 1-40% stayed with an aCD38 regimen but switched to a different combination in 4L. Conclusions: Our findings suggest that across lines, IMiD, PI, and IMiD+PI are the most common treatment categories but that aCD38 treatments are increasingly used in recent years and in later lines. For the major treatment categories (IMiD, PI, IMiD+PI), <25% patients will remain with the same treatment category, whereas 25-50% will switch to another treatment category, in the next line. For aCD38 treatments, 20% or less will remain with the same treatment category, whereas 5-40% will switch, in the next line. Disclosures Gershon: Genentech, Inc./ F. Hoffmann-La Roche: Current Employment, Current equity holder in publicly-traded company. Liu:Genesis Research receives consulting fees from Genentech, Inc. a member of the F. Hoffmann-La Roche Group: Consultancy; Genesis Research: Current Employment. James:Genentech, Inc./ F. Hoffmann-La Roche: Current Employment, Current equity holder in publicly-traded company. Williamson:Amgen: Current equity holder in publicly-traded company; F. Hoffmann-La Roche: Current Employment, Current equity holder in publicly-traded company. Hong:Genentech, Inc.: Current Employment; F. Hoffmann-La Roche: Current equity holder in publicly-traded company. Sarsour:F. Hoffmann-La Roche: Current equity holder in publicly-traded company; Genentech, Inc.: Current Employment.

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

Blood ◽  
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.

scholarly journals Lines of Therapy in Patients with Relapsed or Refractory Large B-Cell Lymphoma and Stem Cell Transplant-Intended Treatment

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 45-46
Author(s):  
Julia Thornton Snider ◽  
Paul Cheng ◽  
Xue Song ◽  
Donna McMorrow ◽  
David Diakun
Keyword(s):  
Research Funding ◽  
Index Date ◽  
Intensive Therapy ◽  
B Cell Lymphoma ◽  
Treatment Patterns ◽  
Cell Transplant ◽  
Front Line ◽  
Publicly Traded ◽  
Line Of Therapy ◽  
To Receive

Introduction: Large B-cell lymphoma (LBCL) is the most common subtype of non-Hodgkin lymphoma. Front line therapy is curative in about 60% of patients; however, about 30% of patients relapse and about 10% are refractory. For patients failing the front line, stem cell transplant (SCT) is currently the only curative option in second line of therapy (LOT2). This study examined real-world treatment patterns with curative intent in patients with relapsed or refractory LBCL to understand the unmet medical need in the United States. Methods: This retrospective study used IBM MarketScan® Commercial and Medicare Supplemental Databases. Patients with ≥2 LBCL diagnoses on different days in 1/1/2012 - 3/31/2019 were identified (index date = date of the earliest LBCL diagnosis). Patients who were ≥18 years of age on the index date, had ≥1 claim for any LBCL treatment on or following the index date, ≥6 months of data prior to (baseline) and ≥12 months of data after (follow up period) the index date, and had no baseline LBCL diagnosis were included. Treatment patterns, including lines of therapy (LOT), treatment regimen, duration of LOT and time from the end of a previous LOT to the subsequent LOT, were examined. All patients were required to receive first LOT (LOT1) of R-CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisone +/- rituximab), EPOCH (etoposide, prednisone, vincristine, cyclophosphamide, and doxorubicin hydrochloride +/- rituximab), or regimens with anthracycline. Of those, patients were further required to receive LOT2 with SCT-intended intensive therapy or platinum-based chemotherapy as recommended by the National Comprehensive Cancer Network (NCCN). Patients were intended for SCT if they received an SCT-intended LOT2 regimen or received SCT regardless of LOT2 regimen. SCT was identified from the LOT2 start date to the end of the study period. All analyses were stratified by whether a patient was intended for SCT and by receipt of SCT. Results: A total of 3,443 patients with LBCL received an eligible front-line therapy (mean age 59.2 years, male 56.9%, length of follow up 2.5 years, National Cancer Institute (NCI) adapted Deyo-Charlson comorbidity index 0.83). Top comorbidities were other malignancy (63.3%), diabetes (23.3%), psychiatric disturbance (13.9%), and chronic pulmonary obstructive disease (13.2%). Among the LBCL patients, 564 (16.4%) received LOT2 with 197 (34.9%) patients SCT-intended and 367 (65.1%) not SCT-intended (Figure 1). SCT-intended patients were significantly younger than SCT-non-intended patients (55.9 vs. 60.9 years, p<0.001), and patients who received SCT were younger than those who did not receive SCT (53.6 vs. 59.7 years, p<0.001). Among the 197 patients intended for SCT, only 55.3% (n=109/197) received NCCN recommended LOT2 for intensive therapy: ICE (ifosfamide, carboplatin, etoposide) +/- rituximab (32.5%) and GemOx (gemcitabine, oxaliplatin) +/- rituximab (19.3%) were the most common regimens. SCT-intended patients initiated LOT2 nearly one year (345 days) after and third line of therapy (LOT3) 501 days after their LBCL index diagnosis (Figure 2). Duration of LOT decreased as patients moved on to later LOTs (LOT1=121 [standard deviation (SD)=69], LOT2=89 [SD=145] and LOT3=46 [SD=23] days). Likewise, the time between 2 consecutive LOTs decreased (194 [SD=258] days between end of LOT1 and start of LOT2, 124 [SD=182] days between end of LOT2 and start of LOT3). Steroids were often prescribed during LOT1 (82.2%) and LOT2 (74.6%) as concomitant therapy. Among the 109 patients receiving SCT-intended regimens, only 34 (31.2%) went on to receive SCT. An additional 88 patients received SCT without being observed to receive an SCT-intended regimen. They might have received therapy in the inpatient setting, which could not be captured in claims data. Among the 82 patients with LOT3, 60.1% received SCT and 9.8% received Chimeric antigen receptor (CAR) T cell therapy. Conclusion: The findings provide insight into real-world treatment patterns in patients with LBCL who received SCT-intended therapy. A small proportion of LBCL patients received NCCN recommended SCT-preparative regimens in LOT2 and only 31% of them received SCT. This highlights the magnitude of unmet need for relapsed or refractory LBCL patients. Future studies should examine the impact of new therapies on the management of LBCL. Disclosures Thornton Snider: Kite, A Gilead Company: Current Employment; Gilead Sciences: Current equity holder in publicly-traded company, Research Funding; Precision Medicine Group: Ended employment in the past 24 months, Research Funding. Cheng:Kite, A Gilead Company: Current Employment, Current equity holder in publicly-traded company. Song:IBM Watson Health: Research Funding. McMorrow:IBM Watson Health: Current Employment. Diakun:IBM Watson Health: Current Employment, Research Funding.

The use of real-world data to examine comparative effectiveness research (CER) and treatment patterns in HER2-negative and triple-negative metastatic breast cancer (MBC): A systematic literature review.

2017 ◽  
Vol 35 (8_suppl) ◽  
pp. 210-210 ◽  
Author(s):  
Monika Parisi ◽  
Corey Pelletier ◽  
Dasha Cherepanov ◽  
Michael S Broder ◽  
Nadia Noormohamed
Keyword(s):  
Breast Cancer ◽  
Metastatic Breast Cancer ◽  
Literature Review ◽  
Comparative Effectiveness ◽  
Triple Negative ◽  
Metastatic Breast ◽  
Treatment Patterns ◽  
Survival Outcomes ◽  
Cancer Type ◽  
Real World Data

210 Background: With the accumulation of RWD in healthcare, CER continues to expand. RWD are becoming increasingly relevant in oncology, particularly since the onset of care pathways and CMS’ Oncology Care Model pilot program; yet, CER in oncology presents several challenges as disease biology differs by cancer type and many RWD sources do not capture clinical response, progression or survival. This review examined common endpoints reported in RWD studies on CER and TxP in MBC, focusing on HER2-negative and Triple Negative (TN) MBC. Methods: PubMed (2006-January 2016) and 4 conferences (2011-January 2016)—ASCO and SABC meetings/symposiums—were searched using MeSH/keywords, e.g., metastatic breast cancer, treatment, and comparative effectiveness. RWD CER and TxP studies in U.S. patients with HER2-negative or TNMBC were included; clinical trials were excluded. Results: Of1,782 total records, 17 articles and 9 conference abstracts were included. Studies using RWD increased over time with 2 studies published in 2010, 1 in 2012, 6 in 2013, 6 in 2014, 10 in 2015, and 1 as of January 2016. Of these, 8 were CER and 18 examined TxP. Most studies were retrospective chart reviews (7 CER; 10 TxP studies), others were retrospective secondary database analyses (1 CER; 6 TxP) and physician surveys (2 TxP). RWD sources included commercial insurance claims, SEER-Medicare, and California Cancer Registry data. Nineteen studies reported results in patients with HER2-negative MBC and 7 reported in TNMBC patients. Primary endpoints included overall survival (OS), progression-free survival (PFS), and treatment duration (TD). CER studies most commonly reported TD and survival outcomes (e.g., OS, PFS), each reported in 75% of the studies. TxP studies also most commonly examined survival outcomes (61% of studies), in addition to various treatment patterns and duration outcomes. Conclusions: This literature review indicates that in parallel to the availability of RWD, published CER studies and analysis of treatment patterns have grown in the last 5 years. The most commonly reported outcomes include OS, TD and PFS.

1043-P: Glycemic Profiles and Treatment Patterns: Real-World Data from 8,020 Adults with Type 1 Diabetes Using the Omnipod® Insulin Management System with Cloud-Based Data Management

Diabetes ◽  
2019 ◽  
Vol 68 (Supplement 1) ◽  
pp. 1043-P
Author(s):  
JENNIFER E. LAYNE ◽  
JIALUN HE ◽  
JAY JANTZ ◽  
YIBIN ZHENG ◽  
ERIC BENJAMIN ◽  
...  
Keyword(s):  
Type 1 Diabetes ◽  
Data Management ◽  
Real World ◽  
Management System ◽  
Treatment Patterns ◽  
Real World Data ◽  
World Data

scholarly journals A review on recipients of hematopoietic stem cell transplantation patients with COVID-19 infection

2021 ◽  
Vol 8 ◽  
pp. 204993612110132
Author(s):  
Kamal Kant Sahu ◽  
Ahmad Daniyal Siddiqui
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Cell Transplant ◽  
Hematopoietic Cell Transplant ◽  
Real World Data ◽  
Hematopoietic Stem ◽  
Geographical Regions

For the last few months, various geographical regions and health sectors have been facing challenges posed by the current COVID-19 pandemic. COVID-19 has led to significant disruption in the normal functioning of potentially life-saving therapies of hematopoietic cell transplant and chimeric antigen receptor therapy. As transplant physicians are gaining more information and experience regarding the undertaking of these complex procedures during the ongoing COVID-19 pandemic, we believe it is important to discuss the challenges faced, prognostic risk factors, and outcomes of COVID-19 in post-hematopoietic stem cell transplantation patients based on the available real-world data.

scholarly journals Treatment Patterns and Costs Among Children Aged 2 to 17 Years With ADHD in New York State Medicaid in 2013

2018 ◽  
Vol 25 (4) ◽  
pp. 463-472 ◽  
Author(s):  
Liqiong Guo ◽  
Melissa Danielson ◽  
Lindsay Cogan ◽  
Leah Hines ◽  
Brian Armour
Keyword(s):  
New York ◽  
Quality Of Care ◽  
New York State ◽  
Treatment Patterns ◽  
Psychological Services ◽  
Children With Adhd ◽  
York State ◽  
Treatment Category

Objective: To identify children with ADHD enrolled in New York State (NYS) Medicaid and characterize ADHD-associated costs by treatment category. Method: In 2013, 1.4 million children aged 2 to 17 years were enrolled in NYS Medicaid. Medicaid claims and encounters were used to identify children with ADHD, classify them by type of treatment received, and estimate associated costs. Results: The ADHD cohort comprised 5.4% of all Medicaid-enrolled children, with 35.0% receiving medication only, 16.2% receiving psychological services only, 42.2% receiving both, and 6.6% receiving neither. The total costs for the ADHD cohort (US$729.3 million) accounted for 18.1% of the total costs for children enrolled in NYS Medicaid. Conclusion: This study underscores the importance of achieving a better understanding of children with ADHD enrolled in NYS Medicaid. A framework to categorize children with ADHD based on their treatment categories may help to target interventions to improve the quality of care and reduce costs.

scholarly journals Discordance between Treatment Guideline Recommendations and Real-World Practice in a Group of Large Integrated Delivery Networks for Venous Thromboembolism (VTE) Patients: A Closer Look at VTE Patients with Cancer

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1951-1951
Author(s):  
Juzer Lotya ◽  
Amol Dhamane ◽  
Lisa Rosenblatt ◽  
Jenny Jiang ◽  
Deysia Levin ◽  
...  
Keyword(s):  
African American ◽  
Research Funding ◽  
Treatment Patterns ◽  
Morbidly Obese ◽  
Anticoagulant Treatment ◽  
Publicly Traded ◽  
Patients With Cancer ◽  
Integrated Delivery ◽  
Delivery Networks ◽  
Current Guidelines

Abstract Background: For patients with VTE, current American Society of Hematology (ASH) guideline panel suggests using direct oral anticoagulants (DOACs) over vitamin K antagonists (VKAs) where VKAs are required to be bridged with a parenteral anticoagulant (PAC). For patients with VTE and cancer, current guidelines recommend DOACs over low molecular weight heparin (LMWH) and LMWH over unfractionated heparin (heparin) for the initial treatment of VTE. Limited evidence is available about the patterns of anticoagulant treatment for VTE in routine clinical practice of large healthcare delivery networks in the United States (US) and whether the VTE treatments are aligned with current guidelines. This study aimed to assess real-world anticoagulant treatment patterns among VTE patients using harmonized electronic health record (EHR) data from four Integrated Delivery Networks (IDNs) in the US. Methods: This was a retrospective, longitudinal, multicenter, cohort study using harmonized EHR data from both inpatient and outpatient settings. The study population included adult patients prescribed DOACs, warfarin, and/or PAC therapy as inpatient or outpatient treatment within ≤30 days of VTE diagnosis, between June 2015 through May 2018. Data from the four IDNs was pooled to describe demographic characteristics and treatment patterns among VTE patients overall and by subgroups. Results: A total of 10,527 patients who were treated with OACs after VTE diagnosis were included for analysis. The mean (SD) age was 61.9 (5.98) years, with 46.1% aged 65 or older. More than half (53.2%) were female, and White patients comprised the majority (74.4%), followed by African American patients (22.8%). Obese and morbidly obese patients comprised 39.1% and 16.1% of patients, respectively. Among all VTE patients, warfarin-only (n=3545; 33.7%) was the most commonly used OAC treatment, followed by warfarin + PAC (n=3128; 29.7%), rivaroxaban-only (n=1357; 12.9%), rivaroxaban + PAC (n=853; 8.1%), apixaban + PAC (n=839; 8.0%), apixaban-only (n=762; 7.2%), and Other OAC (n=357; 3.4%) (Table 1). When stratifying VTE patients by age, gender, race and BMI, some variations in OAC treatment were observed. Among both older (≥65 years) and younger (<65 years) patients, warfarin-only was most commonly used, then warfarin + PAC. Warfarin-only was more commonly used among obese (36.3%) and morbidly obese (40.4%) patients than non-obese (29.8%) patients. OAC treatment patterns were generally comparable among men and women. Among White patients, approximately equal proportions of patients received warfarin + PAC (31.9%) and warfarin-only (31.0%). However, among African-American patients, a higher proportion of patients used warfarin-only (40.9%) vs. warfarin + PAC (24.5%). Patterns of anticoagulant treatments including OACs and/or parental anticoagulants among VTE patients with cancer were further analyzed (Figure 1). Among VTE patients with cancer (n=3657), heparin had the highest use (26.7%), then enoxaparin (22.7%); approximately the same proportion of cancer patients received warfarin-only (16.0%) and warfarin + PAC (16.9%). Of DOACs, rivaroxaban-only was the most commonly used treatment (4.9%), then apixaban + PAC (3.5%), and lastly, rivaroxaban + PAC (3.4%) among cancer patients. Conclusion: Current VTE treatment guidelines recommend warfarin to be bridged with PAC, however, warfarin-only therapy remained the most used treatment option followed by warfarin + PAC. While rivaroxaban and apixaban are not required to be bridged with PAC, such practices were observed for a large proportion of apixaban- and rivaroxaban-treated VTE patients. VTE treatment among patients with cancer was not completely aligned with current guidelines, as heparin was more commonly used than LMWH (enoxaparin). Our findings suggest greater efforts are needed to improve anticoagulant treatment practices among VTE patients. Figure 1 Figure 1. Disclosures Dhamane: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Rosenblatt: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Jiang: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Guo: Bristol Myers Squibb: Ended employment in the past 24 months. Dorsch: Agency for Health Research and Quality: Research Funding; National Institutes of Health/National Institute of Aging: Research Funding; American Health Association Health IT Research Network: Research Funding; Janssen Pharmaceuticals: Honoraria; Bristol Myers Squibb/Pfizer: Research Funding; Amgen: Research Funding. Luo: Pfizer Inc: Current Employment, Current equity holder in publicly-traded company.

scholarly journals To Treat or Not to Treat? Understanding Treatment Patterns in Patients with Lower-Risk Myelofibrosis at the Time of Enrollment in the MOST Study

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 49-51
Author(s):  
Rami S. Komrokji ◽  
Brady L. Stein ◽  
Robyn M. Scherber ◽  
Patricia Kalafut ◽  
Haobo Ren ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Lower Risk ◽  
Research Funding ◽  
Symptom Burden ◽  
Risk Groups ◽  
Treatment Patterns ◽  
Low Risk ◽  
Publicly Traded ◽  
Advisory Committees ◽  
Therapy Initiation

Background: Myelofibrosis (MF) is a chronic Philadelphia chromosome-negative myeloproliferative neoplasm (MPN) characterized by extramedullary hematopoiesis, bone marrow fibrosis, splenomegaly, constitutional symptoms, and diminished quality of life. Treatment decisions may involve a variety of factors including prognosis and symptomatology. Data regarding real-world disease and demographic factors that contribute to therapy initiation and choice in pts with lower risk MF are limited. This analysis of data from the ongoing Myelofibrosis and Essential Thrombocythemia Observational STudy (MOST; NCT02953704) assessed whether these factors differ for lower risk pts who were treated vs untreated at enrollment. Methods: MOST is a longitudinal, noninterventional, prospective, observational study in pts with MF or essential thrombocythemia enrolled at clinical practices within the US. Pts included in the analysis (≥18 y), had low risk MF by the Dynamic International Prognostic Scoring System (DIPSS; Blood. 2010;115:1703), or intermediate-1 (INT-1) risk by age >65 y alone. Pt data were entered into an electronic case report form during usual-care visits over a planned 36-month observation period. Pt-reported symptom burden was assessed using the MPN-Symptom Assessment Form (MPN-SAF); Total Symptom Score (TSS) was calculated (0 [absent] to 100 [worst imaginable]; J Clin Oncol. 2012;30:4098). Data were analyzed with basic descriptive and inferential statistics. Results: Of 233 pts with MF enrolled at 124 sites between 11/29/2016 and 03/29/2019, 205 were included in this analysis; 28 were excluded for being INT-1 risk for reasons other than age. Of the 205 pts, 85 (41.5%) were low- and 120 (58.5%) were INT-1 risk; 56.5% (48/85) and 59.2% (71/120), respectively, were being treated at enrollment. Pt characteristics are listed in Table 1A. Fewer low- vs INT-1 risk pts were JAK2 V617F or MPL positive, and more were CALR positive. The proportion of pts with palpable splenomegaly was similar for treated low- and INT-1 risk pts. In low risk pts, the proportion of pts with palpable splenomegaly was higher in untreated vs treated pts; whereas, in INT-1 risk pts, the opposite was observed (ie, lower proportion in untreated vs treated pts). Blood counts were generally similar across cohorts, except median leukocytes were lower for low risk treated pts and platelet counts were elevated in low- vs INT-1 risk pts. The proportion of pts with comorbidities was similar across cohorts, except for fewer cardiovascular comorbidities in low- vs INT-1 risk pts. Mean TSS was lower in low- vs INT-1 risk pts, but the proportion of pts with TSS ≥20 was greater in treated vs untreated pts in both low- and INT-1 risk groups. Fatigue was the most severe pt-reported symptom in all cohorts. Differences in mean TSS and individual symptom scores between risk groups were not significant (P > 0.05), except itching was worse among INT-1 risk pts (P=0.03). Physician-reported signs and symptoms were generally more frequent for untreated vs treated pts, irrespective of risk (all P > 0.05). Most low risk (69.4%) and INT-1 risk pts (61.2%) who were currently untreated at enrollment had not received any prior MF-directed treatment (Table 1B); the most common prior treatment among currently untreated pts was hydroxyurea (HU) in both risk groups. Of currently treated pts, HU was the most common MF-directed monotherapy at enrollment in low-risk pts, and ruxolitinib was most common in INT-1 risk pts. No low risk pts and few INT-1 risk pts were currently receiving >1 MF-directed therapy at enrollment. Conclusion: These real-world data from pts with MF enrolled in MOST show that a substantial proportion of both low- and INT-1 risk pts who had received treatment before enrollment were not being treated at the time of enrollment. Although watch-and-wait is a therapeutic option, the finding that many of these lower risk pts had in fact received prior therapies suggests an unmet need for effective and tolerable second-line treatment options. Treated pts had greater pt-reported symptom burden vs untreated pts, which suggests that high symptom burden may contribute to the decision for treatment. Prospective studies are needed to evaluate symptom burden change with therapy initiation. In this regard, future analyses of data from MOST are planned to assess the longitudinal evolution of the clinical characteristics, treatment patterns, and management of pts with MF. Disclosures Komrokji: Geron: Honoraria; Agios: Honoraria, Speakers Bureau; AbbVie: Honoraria; Incyte: Honoraria; Novartis: Honoraria; BMS: Honoraria, Speakers Bureau; JAZZ: Honoraria, Speakers Bureau; Acceleron: Honoraria. Stein:Incyte: Research Funding; Kartos: Other: educational content presented; Constellation Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Pharmaessentia: Membership on an entity's Board of Directors or advisory committees. Scherber:Incyte Corporation: Current Employment, Current equity holder in publicly-traded company. Kalafut:Incyte: Current Employment, Current equity holder in publicly-traded company. Ren:Incyte: Current Employment, Current equity holder in publicly-traded company. Verstovsek:Incyte Corporation: Consultancy, Research Funding; Roche: Research Funding; Genentech: Research Funding; Blueprint Medicines Corp: Research Funding; CTI Biopharma Corp: Research Funding; NS Pharma: Research Funding; ItalPharma: Research Funding; Celgene: Consultancy, Research Funding; Gilead: Research Funding; Protagonist Therapeutics: Research Funding; Novartis: Consultancy, Research Funding; Sierra Oncology: Consultancy, Research Funding; PharmaEssentia: Research Funding; AstraZeneca: Research Funding; Promedior: Research Funding.

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