scholarly journals Real-World Assessment of Prior Treatment Patterns in Patients Receiving Belantamab Mafodotin Using a Longitudinal Pharmacy and Medical Open Source Claims Database

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4973-4973
Author(s):  
Natalie Boytsov ◽  
Karen Stockl ◽  
Chi-Chang Chen ◽  
Feng Wang ◽  
Xin Wang ◽  
...  
Keyword(s):  
Open Source ◽  
Treatment Period ◽  
Real World ◽  
Research Funding ◽  
Median Number ◽  
Treatment Patterns ◽  
Prior Treatment ◽  
Immunomodulatory Drugs ◽  
Publicly Traded ◽  
Patient Demographics

Abstract Introduction: Belantamab mafodotin (belamaf) is a first-in-class B-cell maturation antigen-targeting antibody-drug conjugate, with a manageable adverse event profile, that has shown durable efficacy in a broad range of patients with relapsed or refractory multiple myeloma (RRMM) (Lonial et al., Lancet Oncol. 2020). As belamaf is an approved therapy, it is important to understand how it is being integrated into the management of patients with RRMM. The aim of this analysis was to examine baseline demographic and treatment patterns of these patients prior to belamaf in a real-world clinical practice. Methods: This descriptive, retrospective cohort study used IQVIA's longitudinal pharmacy and medical open source claims database, a nationally representative real-world database. The study covered the period from January 2001 to April 2021. Patient inclusion into the study required belamaf treatment initiation and ≥18 years of age at initiation. The first claim date for belamaf during the index period (August 2020 to April 2021) was the index date (ID). Key measures included patient demographics and prior MM treatments. Patient demographics were identified at the ID. Prior MM treatments were assessed during the prior treatment period, which extended from the database start date (January 2001) until the ID. The definition of a line of therapy (LOT) used in this analysis was consistent with published guidance (Rajkumar SV, et al. Blood, 2015;126:921-922). Treatments of interest were identified within the first 28 days of the first observed claim for MM treatment. A new LOT was identified if a new treatment was added after the initial 28 days after the start of the LOT, ≥1 treatment was discontinued (defined by a coverage gap >60 days), or the same LOT was restarted at a later date if ≥1 regimen was administered in between. Different therapeutic classes (e.g. PIs, immunomodulatory drugs, anti-CD38 antibodies) could be used in each LOT either singly or in combinations. Addition/deletion of corticosteriods did not constitute a new LOT. Results: 304 patients with a claim for belamaf were identified in the database and included in the analysis; median age was 70 years and 50.3% were female. Median (range) time from the earliest observed claim for MM treatment during the prior treatment period to the ID was 1,812 (104─6,676) days. Median (range) time from the earliest observed diagnosis for MM during the prior treatment period to the ID was 2,123 (22-7,186) days (~5.8 years). The median number of MM therapeutic classes, overall, received during the prior treatment period was 5; 82.9% of patients with MM received ≥4 classes of MM therapy. Prior therapy for MM was observed for 303 patients; the therapeutic classes of those MM treatments are shown in the Table. The median number of observed LOTs received by patients in the prior treatment period was 5 and 73.0% had ≥4 LOTs. LOT for belamaf may be over/under estimated as reporting on some of the MM medications was embargoed by manufacturers and/or specialty pharmacies for certain periods of time, leading to some incompleteness of data. Embargoed products included lenalidomide, pomalidomide, selinexor, thalidomide, ixazomib, and bortezomib. Although data capture for these MM treatments may not be complete in open source claims, some of the claims data for these medications were included in this analysis, i.e., medical claims for infused medications and pharmacy claims from switch suppliers for oral medications. Conclusions: The real-world evidence analyzed in this study is in line with the baseline characteristics reported for the DREAMM-1 and DREAMM-2 trials (Lonial et al., Lancet Oncol. 2020, Trudel et al., Blood Cancer J., 2019). Patients receiving belamaf have triple-class refractory myeloma with multiple LOTs generally aligned with the current indication. The most frequent prior treatments included proteasome inhibitors, anti-CD38 monoclonal antibodies, immunomodulatory drugs, and corticosteroids. Corticosteroid use is high as it is required to be prescribed in combination with other drugs according to their indications. This study identified patients in the US receiving belamaf and provides early insights into the patient demographic and clinical characteristics as well as number and range of treatments prior to treatment with belamaf. Funding: GSK (Study 214283). Figure 1 Figure 1. Disclosures Boytsov: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Stockl: IQVIA: Current Employment; GlaxoSmithKline: Research Funding. Chen: GlaxoSmithKline: Research Funding; IQVIA: Current Employment. Wang: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Wang: IQVIA: Current Employment; GlaxoSmithKline: Research Funding. Cao: IQVIA: Current Employment. Doherty: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Paka: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company.

scholarly journals Real World Prospective Observational Multicenter Trial of Venetoclax-Based Therapy for Patients with AML Reveals Unique Patterns of Patient Selection and Treatment Utilization - Revive Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1246-1246
Author(s):  
Ofir Wolach ◽  
Itai Levi ◽  
David Lavie ◽  
Jonathan Canaani ◽  
Sigal Tavor ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Patient Selection ◽  
Real World ◽  
Research Funding ◽  
Early Death ◽  
Treatment Patterns ◽  
Publicly Traded ◽  
Advisory Committees ◽  
Related Factors

Abstract Background: Venetoclax-based combinations were recently approved to treat patients (pts) with acute myeloid leukemia (AML) ineligible for intensive chemotherapy. Limited prospective 'real-world' data is available on treatment patterns of venetoclax-based therapy in routine clinical practice. We investigated patterns of patient selection, efficacy, toxicity, patient related outcome and post-remission management in a nationwide multicenter prospective observational trial. Methods: Newly diagnosed pts with AML were enrolled at the time of venetoclax-based therapy initiation from 10 medical centers in Israel. Demographic, clinical and patient-related baseline characteristics were documented. Treatment patterns, safety and efficacy outcomes are reported. Results: Between August 12, 2019, and June 17, 2021(data cut) ,127 AML pts were enrolled to receive venetoclax based therapy. Baseline patient and disease characteristics are reported in Table 1. The main reasons for physician's choice of venetoclax-based therapy were age ≥75, comorbidities and ECOG ≥2 (patient related factors) in 76% of cases and adverse disease biology predicting poor response to intensive chemotherapy (disease related factors) in 24% of cases. Most pts started therapy in an inpatient setting, 82 (64.6%) with a median hospitalization duration of 14 days, while 44 pts (34.6%) started therapy as out pts. Pts received a median of 3.8 cycles of therapy (range 1-21). Most pts (97%) received venetoclax in combination with hypomethylating agents. The full dose of 400mg QD after a median ramp-up duration of 3 days was achieved in 88% of the pts. Dose interruptions and dose modifications during follow-up occurred in 59 (46%) and 30 (24%) of pts, respectively. To allow for adequate follow up for response assessment, efficacy analysis was limited to pts enrolled prior to December 31, 2020, and included 108 pts with a median follow-up of 8 months (range 1-20). As of data cut, 93 pts completed cycle 1 of therapy, 66 pts completed cycle 3 and 39 pts completed cycle 6. 29 pts (27%) are still active on treatment. Best composite complete remission [CCR = complete remission (CR) plus CR with incomplete count recovery (CRi)] was achieved in 62 (57%) pts. CCR rates were assessed in different pre-defined subgroups. Best CCR in pts selected for therapy based on disease-related and patient-related factors were 70% and 54% respectively. Best CCR in pts with AML arising from MPN and pts with other AML were 45% and 58% respectively. Estimated median overall survival (OS) of all pts was 9.6 months (range 7.4-10.6) (Figure 1). Achieving CCR was associated with a superior probability for survival. Estimated median OS was 13.6 months (range 10.6 - not reached) in pts achieving CCR and 4.2 months (range 1.2-10.3) in non-CCR (p<.0001). Of responding pts (CR/CRi, partial remission (PR), morphologic leukemia free state (MLFS), 27 (37%) progressed. Estimated median time to progression was 9.2 months (6.7-NR). Allogeneic transplantation following venetoclax based treatment was offered to 16 (26%) pts with a median age of 71 years (range 43-77). Last documented response prior to transplant was CR in 5 (32%) pts, CRi 9 (56%), MLFS 1 (6%) and PR in 1 (6%) patient. Among grade ≥3 AEs were febrile neutropenia in 28% and infections in 21% of pts. Clinical and laboratory tumor lysis syndrome (TLS) was documented in 2 and 4 pts, respectively. Antifungal prophylaxis was administered in 20% of pts and granulocyte colony-stimulating factor (GCSF) support was used in 17% of pts in response. Early death rate at 30 and 60 days were 7% and 13%, respectively. Conclusion: This prospective real-world analysis reveals unique patterns of patient selection and venetoclax treatment utilization in a medical system with wide access for this indication. Venetoclax-based therapies are effective and associated with manageable toxicity, including in AML patient populations that were excluded from previous registration trials with comparable CCR and early death rates. Factors associated with patient selection in the 'real-world' setting and immature follow up data most probably led to a shorter estimated median OS in this analysis as compared to controlled trials. The REVIVE study continues to expand and is expected to provide additional insights on treatment patterns, management as well as clinical and patient related outcomes. Figure 1 Figure 1. Disclosures Wolach: Janssen: Consultancy; Novartis: Consultancy; Amgen: Research Funding; Astellas: Consultancy; Abbvie: Consultancy, Honoraria, Research Funding; Neopharm: Consultancy. Levi: AbbVie: Consultancy, Research Funding. Lavie: AbbVie: Membership on an entity's Board of Directors or advisory committees, Other: Fees for lectures; BMS: Membership on an entity's Board of Directors or advisory committees; Takeda: Consultancy, Other: Fees for lectures; Roche: Other: Fees for lectures; Novartis: Other: Fees for lectures. Tavor: AbbVie: Consultancy. Hellmann: AbbVie: Consultancy. Tadmor: Janssen: Consultancy, Honoraria, Research Funding; AbbVie: Consultancy, Honoraria, Research Funding. Zuckerman: Gilead Sciences: Honoraria, Speakers Bureau; Novartis: Honoraria; Janssen: Honoraria; Cellect Biotechnology: Honoraria; BioSight Ltd: Honoraria; AbbVie: Honoraria; Orgenesis Inc.: Honoraria. Stemer: AbbVie: Consultancy. Berelovich: AbbVie: Current Employment, Current equity holder in publicly-traded company. Ofek: AbbVie: Current Employment, Current equity holder in publicly-traded company. Frankel: AbbVie: Current Employment, Current equity holder in publicly-traded company. Grunspan: AbbVie: Current Employment, Other: May hold equity. Ofran: Medison Israel: Consultancy; Pfizer: Consultancy; Astellas: Consultancy; AbbVie: Consultancy; Janssen: Consultancy. Moshe: Novartis: Membership on an entity's Board of Directors or advisory committees, Other: Lectures; Astellas: Membership on an entity's Board of Directors or advisory committees, Other: Lectures; AbbVie: Membership on an entity's Board of Directors or advisory committees, Other: Lectures.

scholarly journals Treatment Patterns in Patients with Chronic Myeloid Leukemia in Chronic Phase in the Third Line of TKI Therapy and Beyond Based on Real-World Evidence

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1485-1485
Author(s):  
Torsten Dahlen ◽  
Camille Kockerols ◽  
Germano Ferreira ◽  
Peter E. Westerweel ◽  
Jiří Mayer ◽  
...  
Keyword(s):  
Real World ◽  
Myeloid Leukemia ◽  
Treatment Duration ◽  
Research Funding ◽  
Chronic Phase ◽  
Treatment Patterns ◽  
Publicly Traded ◽  
Real World Evidence ◽  
Third Line ◽  
Tki Treatment

Abstract Introduction: Tyrosine kinase inhibitors (TKIs) are the standard of care for patients with chronic myeloid leukemia in chronic phase (CML-CP). The advent of TKIs has led to improvements in survival, with a life expectancy nearly matching that of the general population. However, TKI failure rates increase with subsequent lines of therapy, leading to worse overall survival in patients in later lines of therapy. There are 5 approved TKIs (imatinib [IMA], bosutinib [BOS], dasatinib [DAS], nilotinib [NIL], and ponatinib [PON]) that may be used based on patient characteristics. IMA is frequently used in first-line (1L) therapy. Second-generation TKIs (BOS, DAS, and NIL) can be used in the 1L or second line (2L) depending on choice of 1L therapy. Use of PON is reserved mostly for patients with T315I mutations due to its adverse event profile. However, little guidance exists concerning which TKI to use in the third line and beyond (3L+). Here we present the results of a real-world evidence study of treatment patterns of TKIs in patients with CML-CP initiating 3L+ TKI therapy. Methods: This is a noninterventional, descriptive cohort study based on secondary use of data from 3 existing CML registries (Czech INFINITY, Dutch PHAROS, and Swedish CML). The observation period was from January 1, 2008, until the last available date of follow-up for each registry (6-12 years depending on the registry). Patients aged ≥18 years with CML-CP initiating a 3L TKI after registry enrollment were included in this analysis. Exposure was defined by TKI treatment with IMA, DAS, NIL, BOS, and PON. Descriptive statistics were used to describe patient demographics and treatment duration. Alluvial diagrams were used to describe and represent all treatment patterns. Results: Of those patients that had started a 2L TKI in the INFINITY, PHAROS, and Swedish CML registries, 109 (28.1%), 51 (35.7%), and 182 (12.8%) patients began 3L TKI therapy, with 105, 48, and 172 eligible patients, respectively, included in this study. Median age at diagnosis was 54.0, 61.0, and 59.6 years, respectively (Table 1). There were more men than women across all 3 registries (51.4%, 58.3%, and 50.6% vs 48.6%, 41.7%, and 49.4%, respectively). No agreement in treatment sequencing was seen across the 3 registries. However, across all 3 registries, the most frequently prescribed TKI in the 3L+ setting was NIL (52.4%, 59.6%, and 45.4%, respectively), followed by DAS (38.1%, 17.0%, and 32.6%, respectively). BOS (11.4%, 0%, and 27.9%, respectively), and PON (13.3%, 17.0%, and 11.1%, respectively), which were prescribed at varying frequencies across the registries. The alluvial plot in the Figure presents the sequence of TKI treatments for patients starting a 2L TKI. Median treatment duration in the 3L was longest for patients receiving NIL (27.5, 13.1, and 45.0 months, respectively) and DAS (26.6, 10.0,9.9 and 40.9 months, respectively), except in PHAROS, in which PON resulted in a longer treatment duration than DAS (6.0, 11.4, and 7.2 months, respectively). In INFINITY, rates of resistance (R) and intolerance (I) in patients discontinuing therapy with BOS, DAS, and NIL were 28.6% and 42.9%, 61.1% and 38.9%, and 48.0% and 36.0%, respectively. In PHAROS, 100.0% of patients discontinued DAS and IMA in the 3L due to I, while R and I were reasons for discontinuation in 16.7% and 83.3% of patients discontinuing NIL and 66.7% and 33.3% of patients discontinuing PON, respectively. The Swedish CML register reported that 15.8%, 9.1%, and 33.3% of patients discontinued DAS, NIL, and PON, respectively, in the 3L+ due to I (Table 2). Conclusions: Based on these real-world treatment patterns (which may provide insights into TKI access in various settings), patients in the 3L were most frequently treated with the second-generation TKIs NIL and DAS, with a smaller proportion treated with BOS and PON. However, no common agreement in sequencing of TKI treatment was seen across the 3 registries. Duration of treatment was longest in patients receiving NIL and DAS. Treatment discontinuation occurred frequently due to both R to and I of the 3L TKI used. Between 12.8% and 35.7% of patients that started a 2L TKI required 3L+ therapy, and therefore additional treatment options that are efficacious and well tolerated are needed for these patients. This study was a research collaboration between Novartis and the 3 registries and was funded by Novartis. Figure 1 Figure 1. Disclosures Dahlen: Novartis: Research Funding. Ferreira: Novartis: Consultancy; Roche: Consultancy; MerckGroup: Consultancy; Research institute for the study of genetic and malignant disorders in children.: Consultancy. Westerweel: Pfizer: Consultancy; BMS / Celgene: Consultancy; Incyte: Consultancy; Novartis: Research Funding. Mayer: Principia: Research Funding. Sahmoud: Novartis: Current Employment; H3 Biomedicine: Consultancy; Context Therapeutics: Consultancy. Wormser: Roche: Current equity holder in publicly-traded company; Novartis: Current Employment, Current equity holder in publicly-traded company. Žácková: Novartis: Speakers Bureau; Angelini: Consultancy, Speakers Bureau.

scholarly journals Real World Treatment Patterns and Toxicity Among Elderly Patients with Multiple Myeloma

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5571-5571
Author(s):  
Jesus D Gonzalez-Lugo ◽  
Ana Acuna-Villaorduna ◽  
Joshua Heisler ◽  
Niyati Goradia ◽  
Daniel Cole ◽  
...  
Keyword(s):  
Side Effects ◽  
Real World ◽  
Research Funding ◽  
Treatment Patterns ◽  
Line Treatment ◽  
Event Free Survival ◽  
First Line ◽  
Treatment Change ◽  
Free Survival ◽  
First Line Treatment

Introduction: Multiple Myeloma (MM) is a disease of the elderly; with approximately two-thirds of cases diagnosed at ages older than 65 years. However, this population has been underrepresented in clinical trials. Hence, there are no evidence-based guidelines to select the most appropriate treatment that would balance effectiveness against risk for side effects in the real world. Currently, guidelines advise that doublet regimens should be considered for frail, elderly patients; but more detailed recommendations are lacking. This study aims to describe treatment patterns in older patients with MM and compare treatment response and side effects between doublet and triplet regimens. Methods: Patients diagnosed with MM at 70 years or older and treated at Montefiore Medical Center between 2000 and 2017 were identified using Clinical Looking Glass, an institutional software tool. Recipients of autologous stem cell transplant were excluded. We collected demographic data and calculated comorbidity burden based on the age-adjusted Charlson Comorbidity Index (CCI). Laboratory parameters included cell blood counts, renal function, serum-protein electrophoresis and free kappa/lambda ratio pre and post first-line treatment. Treatment was categorized into doublet [bortezomib/dexamethasone (VD) and lenalidomide/dexamethasone (RD)] or triplet regimens [lenalidomide/bortezomib/dexamethasone (RVD) and cyclophosphamide/bortezomib/dexamethasone (CyborD)]. Disease response was reported as VGPR, PR, SD or PD using pre-established criteria. Side effects included cytopenias, diarrhea, thrombosis and peripheral neuropathy. Clinical and laboratory data were obtained by manual chart review. Event-free survival was defined as time to treatment change, death or disease progression. Data were analyzed by treatment group using Stata 14.1 Results: A total of 97 patients were included, of whom 46 (47.4%) were males, 47 (48.5%) were Non-Hispanic Black and 23 (23.7%) were Hispanic. Median age at diagnosis was 77 years (range: 70-90). Median baseline hemoglobin was 9.4 (8.5-10.5) and 14 (16.1%) had grade 3/4 anemia. Baseline thrombocytopenia and neutropenia of any grade were less common (18.4% and 17.7%, respectively) and 11 patients (20%) had GFR ≤30. Treatment regimens included VD (51, 52.6%), CyborD (18, 18.6%), RD (15, 15.5%) and RVD (13, 13.4%). Overall, doublets were more commonly used than triplets (66, 68% vs 31, 32%). Baseline characteristics were similar among treatment regimen groups. There was no difference in treatment selection among patients with baseline anemia or baseline neutropenia; however, doublets were preferred for those with underlying thrombocytopenia compared to triplets (93.8% vs 6.2%, p<0.01). Median first-line treatment duration was 4.1 months and did not differ among treatment groups (3.9 vs. 4.3 months; p=0.88 for doublets and triplets, respectively). At least a partial response was achieved in 47 cases (63.5%) and it did not differ between doublets and triplets (61.7% vs 66.7%). In general, first line treatment was changed in 50 (51.5%) patients and the change frequency was higher for triplets than doublets (71% vs 42.4%, p<0.01). Among patients that changed treatment, 17(34.7%) switched from a doublet to a triplet; 15 (30.6%) from a triplet to a doublet and 17 (34.7%) changed the regimen remaining as doublet or triplet, respectively. There was no difference in frequency of cytopenias, diarrhea, thrombosis or peripheral neuropathy among groups. Median event-free survival was longer in patients receiving doublet vs. triplet therapy, although the difference was not statistically significant (7.3 vs 4.3 months; p=0.06). Conclusions: We show a real-world experience of an inner city, elderly MM cohort, ineligible for autologous transplantation. A doublet combination and specifically the VD regimen was the treatment of choice in the majority of cases. In this cohort, triplet regimens did not show better response rates and led to treatment change more often than doublets. Among patients requiring treatment, approximately a third switched from doublet to triplet or viceversa which suggest that current evaluation of patient frailty at diagnosis is suboptimal. Despite similar frequency of side effects among groups, there was a trend towards longer event-free survival in patients receiving doublets. Larger retrospective studies are needed to confirm these results. Disclosures Verma: Janssen: Research Funding; BMS: Research Funding; Stelexis: Equity Ownership, Honoraria; Acceleron: Honoraria; Celgene: Honoraria.

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 (&lt;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 Real-World Outcomes with Fedratinib Therapy in Patients Who Discontinued Ruxolitinib for Primary Myelofibrosis

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3645-3645
Author(s):  
Francesco Passamonti ◽  
Quynh Do ◽  
Youbei Lou ◽  
Manoj Chevli ◽  
Pranav Abraham
Keyword(s):  
Real World ◽  
Research Funding ◽  
Group Performance ◽  
Performance Status ◽  
Primary Myelofibrosis ◽  
Index Patient ◽  
Pharmacological Therapy ◽  
Janus Kinase ◽  
Publicly Traded ◽  
Once Daily

Abstract Introduction: Myelofibrosis (MF) is a rare myeloproliferative disorder in whichdysregulation of the janus kinase 2 (JAK2) hematopoiesis-signaling pathway disruptsbone marrow production of blood cells, leading to anemia, fatigue and splenomegaly.Until recently, the dual JAK1/JAK2 inhibitor, ruxolitinib (RUX), has been the onlyapproved pharmacological therapy for intermediate/high-risk MF. However, manypatients receiving RUX experience a suboptimal response or develop cytopenia, leadingto discontinuation. Fedratinib (FEDR), an oral, selective inhibitor with activity againstwild-type and mutationally activated JAK2 and the receptor tyrosine kinase FLT3, wasapproved in the US in August 2019. The real-world effectiveness of FEDR in the post-RUX setting has not yet been assessed. Aim: To assess the baseline characteristics and survival outcomes among patientsdiagnosed with MF who discontinued RUX and were subsequently treated with FEDR ornot. Methods : Adult US patients who received RUX after an initial diagnosis of primary MF(identified by ICD-9/10-CM codes) were identified using Flatiron Health's nationwideelectronic health record (EHR)-derived database. Patients were stratified by whetherthey subsequently received post-ruxolitinib fedratinib (FEDR group) or not (non-FEDRgroup). The non-FEDR group was further stratified by time of RUX discontinuation(before [non-FEDR subgroup A] and after [non-FEDR subgroup B] US approval of FEDRin August 2019) to enable a contemporaneous comparison with the FEDR group. Indexdate was the start of FEDR therapy in the FEDR group, and last date of RUX therapy inthe non-FEDR groups. Patients were included if they had ≥2 recorded visits in theFlatiron network on or after January 1, 2011- Oct 31, 2020; were aged ≥18 years atindex; had ≥1 months' data before and also after index; and had no record of receivingunclassified clinical study drugs prior to index. Patient demographics and clinical characteristics were assessed at index. Overall survival (OS) was defined as time fromindex until death or censoring, and was assessed by Kaplan-Meier analysis. Landmarksurvival was defined as the proportion of patients who survived at a given point.Association of baseline variables and survival were assessed by Cox proportionalhazards model and p&lt;0.05 was considered statistically significant. Results: Overall, 229 MF patients were evaluated (FEDR group: n=70; non-FEDRgroup: n=159). The median age at index for the FEDR group and non-FEDR group was71.0 and 70.0 years, 55.7% and 50.3% were female, 64.3% and 68.6% were white, andmedian follow-up from index was 7.0 and 6.0 months, respectively. Among patients withEastern Cooperative Oncology Group performance status (ECOG PS) at index, 90.2%(46/51) and 74.3% (84/113) had an ECOG score of 0-1, respectively (Table). Median(range) duration of FEDR therapy in the FEDR group was 3.7 (0-12.2) months; 47.1%(33/70) of patients receiving FEDR initiated therapy with 400 mg FEDR once daily, and21.4% (15/70) initiated with 200 mg FEDR once daily. Median OS was not reached in theFEDR group and was 17 months in the non-FEDR group. Landmark survival in theFEDR and non-FEDR groups was 91.3% and 76.5% at 3 months, and 71.6% and 53.5%at 12 months, respectively. In the non-FEDR subgroup B, the corresponding survivalrates at 3 months and 12 months were 75.0% and 47.9%. The Cox proportional hazardsmodel suggested that being male, 'other' (non-white, non-black/African-American, non-Asian, non-missing) race, Charlson comorbidity index ≥1, ECOG score 2-4, and bodymass index &lt;18.5 kg/m were significantly associated with poorer survival. Conclusions: This real-world analysis demonstrates that these two groups of patientswith primary MF who had received prior RUX had broadly comparable baselinecharacteristics. FEDR appeared to be associated with improved survival up to 1 yearafter index compared with non-FEDR therapy. When the FEDR group and non-FEDR Bsubgroup were compared, the differences in survival rate remained. FEDR may offersubstantially improved survival probability in patients receiving post-ruxolitinib therapy of primary MF in routine clinical practice. Figure 1 Figure 1. Disclosures Passamonti: Novartis: Research Funding, Speakers Bureau; Celgene: Research Funding, Speakers Bureau. Do: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Lou: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Chevli: Bristol-Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Abraham: Bristol Myers Squibb: Current Employment.

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 &gt;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 &gt; 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 &gt; 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 &gt;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.

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 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 &lt;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 (&gt;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 &gt;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 Current Clinical Practice and Decision-Making in Multiple Myeloma Treatment in the United States of America: A Real-World Survey

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3001-3001
Author(s):  
Amanda Ribbands ◽  
Boris Gorsh ◽  
Abigail Bailey ◽  
Natalie Boytsov ◽  
Emily Luke ◽  
...  
Keyword(s):  
Decision Making ◽  
Real World ◽  
United States Of America ◽  
Drug Class ◽  
The United States ◽  
Treatment Patterns ◽  
Treatment Choice ◽  
Current Clinical Practice ◽  
Publicly Traded ◽  
Factors Influencing

Abstract Introduction: With each successive line of therapy (LOT), treatment choice for patients with multiple myeloma (MM) becomes increasingly complicated due to existing regimens and the lack of consensus on the standard of care for relapsed/refractory disease. There is a need for more real-world (RW) information on current MM treatments and clinical practices. More insight into the complexity of treatment choices for MM, increasing with each LOT, is also needed to better understand and inform patient treatment as their MM progresses. We aimed to examine current clinical practice and decision-making in the RW setting in MM. Methods: Data were derived from the Adelphi MM Disease Specific Programme™, a point-in-time survey of hematologists and hemato-oncologists conducted in the United States of America between August 2020 and July 2021, collating descriptive information on MM treatment patterns and decision-making from first-line (1L) to fourth-line therapy and beyond (4L+). Physicians completed online patient record forms for their next 8 consulting patients who had a confirmed diagnosis of MM and were actively receiving 1L-4L+ treatment (ie, a quota of ≥2 patients on each LOT). Results: A total of 63 physicians were included in the interim analysis, reporting patients who ever received, or are on at the time of data collection, a specific LOT. Data were provided for 259 patients with 1L treatment, 186 with second-line (2L), 120 with third-line (3L), 60 with 4L, and 2 with fifth-line treatment. Of the patients included in the study, 66% were male with a mean age of 67.7 years (standard deviation 8.16 years); 59% of patients used Medicare for their health insurance and 32% of patients had commercial insurance. The majority of patients received triplet regimens in each LOT (1L: 72%, 2L: 72%, 3L: 68%, 4L: 43%). The top 5 triplet regimens are shown in Table 1. Regimens including cluster of differentiation (CD)38-targeted treatments were used across all LOTs, with the most frequent use seen in earlier relapsed/refractory settings (1L: 8%, 2L: 45%, 3L: 26%, 4L: 19%). Retreatment with the same drug class occurred in 53% of patients treated with a proteasome inhibitor, 50% of patients treated with immunomodulatory drugs, and 4% of patients treated with CD38-targeted treatment. Disease progression/relapse was the most frequent reason for treatment cessation across all LOTs among patients who used either mono, doublet, triplet, or quad regimens (1L: n=92 [48%], 2L: n=72 [61%], 3L: n=37 [63%], 4L: n=2 [100%]). For all LOTs, the leading factors influencing physicians' treatment choice were good clinical data regarding overall survival (OS) (1L: 59%, 2L: 65%, 3L: 52%, 4L: 48%) followed by better efficacy overall (1L: 48%, 2L: 53%, 3L: 45%, 4L: 41%). In 3L, high overall response rate was also important (24%). Good clinical data regarding OS was the most frequent factor influencing choice of triplet regimens at 1L, 2L, and 3L (56%, 62%, and 50%, respectively), whereas manageable side effects profile was the leading factor driving choice of triplet regimen at 4L+ (43%). Other factors that influenced treatment choice included long-term safety, transplant eligibility, and effective use of the treatment as part of a combination therapy. Conclusion: We provide valuable RW data on current treatment patterns and decision-making in MM. Interim analyses revealed a trend for use of triplet therapies across all LOTs, high retreatment rate with the same drug class, and the importance of survival data and clinical efficacy as key factors influencing physician selection of treatment. Additional analyses will be conducted as physicians complete their patient reporting for the study to identify unmet needs and opportunities for new treatment strategies in MM. Funding: GlaxoSmithKline (Study 209997). Figure 1 Figure 1. Disclosures Ribbands: Adelphi Real World, paid employee: Current Employment. Gorsh: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Boytsov: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Luke: Adelphi Real World, paid employee: Current Employment. Lambert: Adelphi Real World, paid employee: Current Employment. Hogea: GlaxoSmithKline, paid employee: Current equity holder in publicly-traded company, Ended employment in the past 24 months.

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.

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