Perceptions of Patient Disease Burden and Management Approaches of Systemic Mastocytosis (SM) By Healthcare Providers: Results from the TouchStone SM Survey

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 5-6
Author(s):  
Ruben A. Mesa ◽  
Erin M. Sullivan ◽  
David Dubinsky ◽  
Brittany Carroll ◽  
Valerie M. Slee ◽  
...  
Keyword(s):  
Disease Management ◽  
Symptom Onset ◽  
Research Funding ◽  
Healthcare Providers ◽  
Diagnostic Tools ◽  
Treatment Goals ◽  
Publicly Traded ◽  
Similar Treatment ◽  
The Us ◽  
Kit D816v

Introduction: SM is a rare, mast cell neoplasm characterized by uncontrolled proliferation, accumulation, and activation of abnormal mast cells (MCs). Approximately 95% of patients with SM harbor the D816V mutation, which results in constitutive activation of the KIT receptor, causing debilitating symptoms across all SM subtypes such as pruritus, headaches, bone pain, nausea, vomiting, diarrhea, brain fog, and anaphylaxis. Patients with SM are managed across specialties depending on symptom severity and organ involvement. Hematologists/oncologists and allergists/immunologists both play key roles in the management of disease. With few multidisciplinary SM practices in the US, more information is needed across specialties to enable optimal SM care. Here we report findings from the TouchStone SM survey of healthcare providers (HCPs) in the US to inform perceptions of disease and disease management strategies. Methods: HCPs completed an online survey containing 47 items covering a range of concepts related to diagnosis (how made, how long it takes), symptoms (how assessed, what symptoms patients experience, how HCPs discuss symptoms with patients, treatment and management), perceived impact on daily life, treatment received (prescription and over-the-counter, treatment goals, unmet needs) and satisfaction (symptom management, overall treatment and disease management). The focus of these analyses are HCP perceptions of disease severity, treatment goals, and disease management. Practicing hematologists/oncologists and allergists/immunologists were eligible if caring for ≥4 SM patients and in practice ≥3 years post fellowship. HCPs were recruited through double-blinded market research survey panels, with individual responses kept confidential. Descriptive analyses were performed on survey responses. Results: Of 304 HCPs contacted, 60 allergists/immunologists and 59 hematologists/oncologists were enrolled with an average of 14 years of practice experience. On average, participating HCPs had 25 SM patients (range: 4-100) under their care. HCPs reported perceived prevalence of the KIT D816V mutation at 46% of patients (range 0-100), substantially lower than published prevalence rates of up to 95%.1 Among HCPs, the average perceived time from patient symptom onset to diagnosis was 8 months, in contrast to 7 years as reported by patients.2 Allergist/immunologists reported primarily seeing patients with non-advanced disease (80%), while hematologists/oncologists reported managing both non-advanced (50%) and advanced patients (50%). Participating HCPs reported that 58% of their patients have moderate-severe SM. HCP treatment goals for SM patients were consistent across specialties. The 2 most important treatment goals for non-advanced patients were improved quality of life (QoL) (40%; 48/120) and improvement of symptoms (24%; 29/119), while the 2 most important treatment goals for advanced patients were improved progression-free survival /improved overall survival (34%, 35/104) and improved QoL (25%, 26/104). Conclusions: Allergists/immunologists co-manage SM care with hematologists/oncologists, particularly in non-advanced SM, and have similar treatment goals for this patient population, including improvement in QoL and symptom control. In this sample of HCPs, the perceived prevalence of the KIT D816V mutation as well as the perceived time from symptom onset to diagnosis were substantially lower than published estimates. These gaps identified suggest a need to improve HCP awareness of SM symptoms and diagnostic tools to help facilitate earlier patient diagnosis. 1. Garcia-Montero et al. Blood. 2006;108(7):2366-2372. 2. Jennings SV et al. Immuno Allergy Clin North Am. 2018;38(3):505-525. Disclosures Mesa: Promedior: Research Funding; Incyte: Research Funding; Genetech: Research Funding; CTI: Research Funding; Celgene: Research Funding; Abbvie: Research Funding; Sierra Onc: Consultancy; Novartis: Consultancy; LaJolla Pharma: Consultancy; Samus: Research Funding. Sullivan:Blueprint Medicines Corporation: Current Employment, Current equity holder in publicly-traded company. Dubinsky:Blueprint Medicines Corporation: Current Employment, Current equity holder in publicly-traded company. Carroll:Blueprint Medicines Corporation: Consultancy, Current equity holder in publicly-traded company. Mathias:Blueprint Medicines Corporation: Other: employed by Health Outcomes Solutions, which received funding Blueprint for providing assistance in developing the Touchstone survey. Castells:Blueprint Medicines Corporation: Consultancy, Other: Clinical trials: Principle Investigator; UpToDate: Other: Author fee; Annals of Allergy, Asthma & Immunology: Other: Editorial Board.

A Worldwide Observational Registry Collecting Longitudinal Data On the Management of CML Patients (The WORLD CML Registry) - Summary of the First 1001 Patients.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4267-4267
Author(s):  
Ricardo Pasquini ◽  
Jorge Cortes-Franco ◽  
Hagop M. Kantarjian ◽  
David Joske ◽  
Zhixiang Shen ◽  
...  
Keyword(s):  
Disease Management ◽  
Board Of Directors ◽  
Risk Score ◽  
Research Funding ◽  
Plasma Drug ◽  
Advisory Committees ◽  
Private Hospitals ◽  
Management Guidelines ◽  
The Us ◽  
The World

Abstract Abstract 4267 Introduction This multicenter, international, prospective registry is a longitudinal (5 year) assessment of diagnosis methods, management strategies, and outcomes for CML patients (pts) under ‘real world’ conditions. Pts were enrolled from Latin America (LA), United States (US), Asia Pacific (AP), Middle East and Africa (MEA), and Russia and Turkey (RT). This first, interim data summary reports baseline characteristics on the first 1001 enrolled pts. Methods Eligible pts were ≥ 16 yrs of age and entered the Registry within 6 months of CML diagnosis. Demographics, medical history, current disease status and management information are collected at baseline. Follow up data are collected at approximately 6-month intervals. Center practices in disease detection and monitoring are collected annually. Results 1001 patients from 148 sites worldwide were enrolled with a data cut-off of May 2009. Represented in this dataset: 165 (16.5%) from LA, 179 (17.9%) from US, 372 (37.2%) from AP, 84 (8.4%) from MEA, and 201 (20.1%) from RT. The majority of patients in LA (50%) were from government hospitals, US from private hospitals/practices (61.1%), AP and MEA from academic institutions (54.8% and 64.7% respectively), and RT from regional/community hospitals (55%). Overall, 953 (95.2%) pts were in chronic phase CML, with little variation across regions. Overall, 58.4% of pts were male (LA, 51.5%; US, 57.0%; AP, 67.7%; MEA, 53.6%; RT, 50.2%) and median age was 46 yrs (range, 15–91). Notably, median age in the AP region was just 40 yrs (LA, 48; US, 53; MEA, 41.5; RT, 52). Hematologic, bone marrow, and cytogenetic (Cy) assessments were used for CML diagnosis in most pts (88.7%, 84.4%, 82.6%, respectively). For Cy assessment, the method used varied widely by region. Most centers appeared to use both karyotype and fluorescence in situ hybridization (FISH), with FISH being used in a greater proportion of pts in the US and MEA (Table 1). Only 51.6% of pts had molecular assessments at diagnosis, ranging from 27.4% in RT to 70.2% in MEA. The majority (54.3%) of pts had no risk score assigned at diagnosis. Sokal score, assessed in 39.1% of pts, was the most common risk score used in all regions (LA, 46.7%; US, 5.6%; AP, 36.0%; MEA, 50.0%; RT, 63.7%). The most common comorbidity was cardiac disorders, in 211 (21.1%) pts, followed by diabetes in 74 (7.4%) pts. Pulmonary disorders were present in 33 (3.3%) patients. At the time of Registry enrollment, imatinib was the most commonly prescribed therapy. Most centers (83.1%) were not performing plasma drug monitoring of anti-leukemic therapy, primarily due to lack of availability (58.5%); 89.2% of those centers would consider performing plasma drug level testing if it were readily available. Conclusions The first interim results of this Registry reveal some regional differences in CML diagnosis and management. Notably, not all pts appear to be treated according to published disease management guidelines. For example, although only karyotyping can detect additional chromosomal abnormalities in Philadelphia chromosome-positive CML, approximately 20% of patients overall and 40% of patients in the US and MEA did not have karyotyping performed at diagnosis. Furthermore, molecular assessments were not universally performed at diagnosis. As these data mature, ongoing interim summaries from the WORLD CML Registry will continue to assess global trends in CML diagnostic methods, treatment approaches, adherence to disease management guidelines and response to treatment. Educational initiatives can thus be regionally tailored to optimize patient care in CML. Additional data summaries by center setting (private hospitals/practices, academic, etc) will also be available. Disclosures: Cortes-Franco: Novartis: Honoraria, Research Funding, Speakers Bureau; Wyeth: Honoraria, Research Funding, Speakers Bureau; BMS: Honoraria, Research Funding, Speakers Bureau. Joske:Novartis Australia: Membership on an entity's Board of Directors or advisory committees; Novartis Pharma AG: Membership on an entity's Board of Directors or advisory committees. Mongay:Novartis: Employment. Eng:Novartis: Employment. Wang:Novartis: Employment. Hughes:Bristol-Myers Squibb: Advisor, Honoraria, Research Funding; Novartis: Advisor, Honoraria, Research Funding. Kim:Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Wyeth: Research Funding.

scholarly journals Treatment Patterns and Outcomes of Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) Treated with Hypomethylating Agents (HMA) in the United States (US)

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 14-16
Author(s):  
Timothy S. Pardee ◽  
Jessica Oschwald ◽  
Esprit Ma ◽  
Tao Xu ◽  
Melissa Montez ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Research Funding ◽  
Community Setting ◽  
Patient Characteristics ◽  
Treatment Patterns ◽  
Survival Outcomes ◽  
Newly Diagnosed ◽  
Publicly Traded ◽  
The Past ◽  
The Us

Introduction: AML is an aggressive disease with poor prognosis that predominantly affects older adults. Due to advanced age and associated comorbidities, many patients are not fit for intensive induction chemotherapy. Monotherapy with HMAs such as azacitidine (AZA) or decitabine (DEC) is often still considered as standard of care for these patients, despite mixed evidence from studies regarding the benefit of HMAs alone (Duchmann & Itzykson. Int J Hematol 2019). The aim of the current study is to evaluate patient characteristics, treatment patterns and outcomes of patients with AML treated with HMA monotherapy as first line (1L) in clinical practice in the US. Methods: This is a retrospective observational study of the Flatiron Health database; a nationwide, longitudinal, demographically and geographically diverse database representing more than 2.4 million patients with cancer in the US. The database contains de-identified data derived from electronic health records from over 280 cancer clinics, which are predominantly community oncology practices. Patients ≥18 years, diagnosed with AML between 1/1/2014 and 3/30/2020 (excluding acute promyelocytic leukemia and clinical trial enrollment), and who received HMAs as 1L treatment ≤30 days from AML diagnosis were evaluated. Descriptive analyses were conducted on patient characteristics and treatment patterns. Kaplan-Meier analyses were used to estimate time to last administration (TTLA; from initiation to last observed administration before death, end of follow-up or a gap of 60 days) and median overall survival (OS). Results: A total of 2589 patients with an AML diagnosis were included for analysis, where 574 (22%) were treated with 1L HMAs (AZA: n=341 [59%]; DEC: n=233 [41%]). The median age of 1L HMA patients was 79 years with 63% male. Most patients were treated in the community setting (n=511 [89%]; median age: 79 years); those treated in academic centers were slightly younger (n=63 [11%]; median age: 77 years). Characteristics for non-antecedent hematological disorder (AHD)-AML (n=327) and AHD-AML (n=247) patients are presented in Table 1. Median TTLA with 1L HMA was 77 days with a median of 3 cycles of both AZA and DEC. Of the 168 patients who received second-line (2L) therapy, 82% (n=138) received another low-intensity therapy or combination (of which only 14 received targeted therapies) (Figure 1). Overall, 44% of 1L HMA patients (n=254) had evidence of molecular testing before 1L treatment initiation (this was more common in later years). Of the 228 patients tested for FLT3, 30 (13%) were FLT3 positive; 7 (23%) FLT3-positive patients were treated with 2L or third-line (3L) FLT3-targeted therapies (gilteritinib, midostaurin or sorafenib). Of the 152 patients tested for IDH1/2, 35 (23%) were IDH1/2 positive; 5 (14%) IDH1/2-positive patients were treated with 2L or 3L targeted agents (enasidenib or ivosidenib). A median OS of 6.3 months (95% CI: 5.5-7.5) was observed in the overall 1L HMA cohort. Median OS in 1L HMA patients did not differ with respect to different types of AML (non-AHD-AML: 6.6 [95% CI: 5.5-7.9] months; AHD-AML: 6.0 [95% CI: 4.8-7.5] months, p=0.34) or practice setting (community: 6.0 [95% CI: 5.3-7.0] months; academic: 8.3 [95% CI: 6.9-13.3] months, p=0.14). One-year OS was 31.4% and 30.1% for non-AHD-AML and AHD-AML patients, respectively. Patients treated in the community setting had numerically lower 1-year OS (29.7% [95% CI: 25.8-34.3]) than those treated in the academic setting (39.5% [95% CI: 28.6-54.6]), which reflects the higher rates of 2L treatment in academic practice, though this analysis is unadjusted. Conclusions: This new database enabled a detailed analysis of 1L HMA-treated patients with newly diagnosed AML in routine clinical practice predominantly in the community setting. 1L HMA patients have poor survival outcomes (median OS 6.3 months) which are comparable to other real-world data from SEER-Medicare (Zeidan et al. Blood Adv 2020; median OS 7-8 months; median age: 77 years); but shorter than the median OS of 9-10 months observed in 1L HMA-treated AML patients in clinical trials (DiNardo et al. EHA 2020). Limitations of the study included limited conduct of bone marrow biopsies for response and lack of transfusion data. The observed survival outcomes highlight the importance of further treatment advances to address the unmet need in older patients with AML ineligible for intensive induction chemotherapy. Disclosures Pardee: Rafael: Research Funding; Celgene: Consultancy, Honoraria, Speakers Bureau; Amgen: Honoraria, Speakers Bureau; Pharmacyclics: Speakers Bureau; Rafael Pharmaceuticals: Consultancy; BMS: Consultancy, Honoraria, Speakers Bureau; AbbVie: Consultancy; Genentech, Inc.: Consultancy; Karyopharm: Research Funding. Oschwald:Roche Products Limited: Current Employment. Ma:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company. Xu:F. Hoffmann-La Roche Ltd: Current Employment, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Montez:F. Hoffmann-La Roche: Current equity holder in publicly-traded company; Genentech, Inc.: Current Employment. Ramsingh:Genentech, Inc.: Current Employment; NEKTAR: Current equity holder in publicly-traded company; Exelixis: Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months, Ended employment in the past 24 months; F. Hoffmann-La Roche: Current equity holder in publicly-traded company. Hong:Genentech, Inc.: Current Employment; F. Hoffmann-La Roche: Current equity holder in publicly-traded company. Choi:AbbVie: Current Employment, Current equity holder in publicly-traded company. Flahavan:Roche Products Ltd.: Current Employment; F. Hoffmann-La Roche: Current equity holder in publicly-traded company. OffLabel Disclosure: Discussion will include the use of decitabine for the treatment of AML.

scholarly journals Realmind: A Prospective, Multicenter, Observational Study of Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma Starting Second- or Third-Line Therapy and Not Receiving Autologous Stem Cell Transplant

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 5010-5010
Author(s):  
Christopher R. Flowers ◽  
John M. Burke ◽  
Mirko Vukcevic ◽  
Susan Snodgrass ◽  
Shreekant Parasuraman ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Research Funding ◽  
B Cell Lymphoma ◽  
Initial Diagnosis ◽  
Cell Transplant ◽  
Publicly Traded ◽  
Line Therapy ◽  
Patient Reported ◽  
The Us ◽  
Third Line

Abstract Background While >60% of patients with diffuse large B-cell lymphoma (DLBCL) are cured when treated with R-CHOP after initial diagnosis, ~40% will be refractory or relapse (R/R) after achieving a response with first-line therapy. Until recently, patients with R/R disease have been treated with rituximab + chemotherapy combinations, rituximab + lenalidomide and salvage chemotherapy followed by high-dose chemotherapy and autologous stem cell transplant (ASCT). Additional FDA-approved treatment options, recently introduced, include the CD19-directed cytolytic antibody tafasitamab in combination with lenalidomide (granted accelerated approval) for second-line or later patients, as well as CAR T-cell therapies, the CD79b-directed antibody-drug conjugate polatuzumab combined with bendamustine + rituximab, and the CD19-directed antibody-drug conjugate loncastuximab for third-line or later patients. Sequencing these various options and identifying the optimal choice of treatment for individual patients can be challenging for practicing oncologists. The realMIND study (NCT04981795), will assess treatment patterns, physician-reported clinical outcomes and patient-reported health-related quality of life (HRQoL) among patients with R/R DLBCL not eligible for ASCT, and is designed to produce data to support physician decision-making in individualized, patient-centric care. realMIND also has a special focus on patients belonging to racial and ethnic minority groups, different age groups, and those with different socio-economic statuses to ensure strong representation among the US patient population. Study design and methods realMIND is a Phase IV, multicenter, prospective, observational study in which an estimated 1,000 patients will be enrolled from ~75 sites in the US. Enrollment will aim to achieve a representative distribution of adult patients by age, gender, race and ethnicity by actively seeking sites from a broad range of locations, including otherwise under-represented populations. Eligible patients will be aged ≥18 years with histologically confirmed DLBCL at initial diagnosis (including high-grade double- and triple-hit lymphoma), and R/R status after at least one prior systemic therapy and prior to second- or third-line therapy for DLBCL (Figure 1). Patients currently receiving ASCT (but not those with prior ASCT experience) will be excluded. Physician-validated clinical outcomes will include complete response rate, overall response rate, duration of response, event-free survival and overall survival. Safety will be evaluated by assessing serious adverse events (AEs) and AEs leading to treatment discontinuation, interruption or dose modification, as well as AEs in patients treated with tafasitamab. Pathology assessments such as biomarker status (e.g. antigen expression on tumor cells) will be identified using clinical samples taken at initial diagnosis and relapse. Patient-reported outcomes will include general and lymphoma-specific measures of HRQoL (EORTC QLQ-C30 and FACT-Lym), collected as part of usual care visits at least quarterly during follow-up. The end of the study is expected to occur approximately 79 months after the first patient is enrolled to ensure a minimum of 24 months' follow-up after last patient enrollment. The distribution of patient characteristics and all survival outcome measures will be presented using descriptive statistics; time-to-event analyses will be conducted using Kaplan-Meier methods. Summary realMIND is a large-scale, multicenter, real-world study that aims to describe the treatment patterns and clinical outcomes of patients with R/R DLBCL. The study is currently enrolling with a planned sample size of 1,000 patients from ~75 sites in the US. The study population will aim to enroll a representative distribution of patients within the US, facilitating an investigation of outcomes in a patient population reflecting real-world clinical practice. Funding: MorphoSys AG. Figure 1 Figure 1. Disclosures Flowers: National Cancer Institute: Research Funding; BeiGene: Consultancy; Bayer: Consultancy, Research Funding; AbbVie: Consultancy, Research Funding; Biopharma: Consultancy; Epizyme, Inc.: Consultancy; Denovo: Consultancy; Genmab: Consultancy; Amgen: Research Funding; Eastern Cooperative Oncology Group: Research Funding; Genentech/Roche: Consultancy, Research Funding; Sanofi: Research Funding; Celgene: Consultancy, Research Funding; Gilead: Consultancy, Research Funding; Burroughs Wellcome Fund: Research Funding; Karyopharm: Consultancy; Ziopharm: Research Funding; Pharmacyclics/Janssen: Consultancy; SeaGen: Consultancy; Xencor: Research Funding; Kite: Research Funding; 4D: Research Funding; Acerta: Research Funding; TG Therapeutics: Research Funding; Pfizer: Research Funding; Spectrum: Consultancy; Cancer Prevention and Research Institute of Texas: CPRIT Scholar in Cancer Research: Research Funding; Adaptimmune: Research Funding; Allogene: Research Funding; Takeda: Research Funding; Novartis: Research Funding; Nektar: Research Funding; Morphosys: Research Funding; EMD: Research Funding; Janssen: Research Funding; Iovance: Research Funding; Guardant: Research Funding; Cellectis: Research Funding; Pharmacyclics: Research Funding. Burke: Kura: Consultancy; MorphoSys: Consultancy; AbbVie: Consultancy; Roche/Genentech: Consultancy; Verastem: Consultancy; Adaptive Biotechnologies: Consultancy; AstraZeneca: Consultancy; Kymera: Consultancy; X4 Pharmaceuticals: Consultancy; Beigene: Consultancy, Speakers Bureau; Bristol Myers Squibb: Consultancy; SeaGen: Consultancy, Speakers Bureau; Epizyme: Consultancy. Vukcevic: MorphoSys: Current Employment. Snodgrass: Incyte Corporation: Current Employment. Parasuraman: Incyte Corporation: Current Employment, Current equity holder in publicly-traded company, Other: Support for attending meetings and/or travel. Büchele: MorphoSys: Current Employment, Current equity holder in publicly-traded company. Kurukulasuriya: MorphoSys: Current Employment, Current equity holder in publicly-traded company, Other: Support for attending meetings/travel.

scholarly journals Real-World Treatment Patterns and Outcomes of Patients with Chronic Lymphocytic Leukemia (CLL) Receiving First-Line (1L) Therapy in the United States (US)

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4086-4086
Author(s):  
Anthony R. Mato ◽  
Arliene Ravelo ◽  
Tu My To ◽  
Robert Schuldt ◽  
Juliana M.L. Biondo
Keyword(s):  
High Risk ◽  
Research Funding ◽  
Treatment Patterns ◽  
Publicly Traded ◽  
Treatment Regimens ◽  
The Past ◽  
Oral Agents ◽  
The Us ◽  
Testing Patterns ◽  
Over Time

Abstract Background: There have been many advances in CLL treatments over the past decade, with a number of novel agents targeting molecular pathways within CLL cells receiving approval from the US Food and Drug Administration. Here, we assessed the evolution of molecular testing patterns, treatment patterns, and clinical outcomes over time in patients receiving 1L CLL treatment in a real-world US database. Methods: This was a retrospective cohort study using the Flatiron Health database, a longitudinal database comprising de-identified, patient-level, structured and unstructured data, curated via technology-enabled abstraction. During the study period, the de-identified data originated from approximately 280 cancer clinics (~800 sites of care) in the US. Patients aged 18 years and older who were diagnosed with CLL and initiated 1L treatment between December 2015 and December 2020 were selected. Participants who took part in a clinical trial in any line of therapy, or who had any other primary cancer diagnosis, were excluded. Baseline characteristics, including testing patterns, at initiation of 1L treatment were assessed using descriptive statistics. Treatment patterns and outcomes, such as time to next treatment or death (TTNTD), were analyzed. Kaplan-Meier analysis was used to estimate TTNTD. Results: Among 3654 patients with treatment-naive CLL who were selected from the de-identified database, the mean age at 1L treatment initiation was 70 years (range, 29-85); 64.3% of patients were male; 72.1% were White, 8.2% Black, 3.9% Hispanic/Latino, 1.0% Asian, and 14.9% were of other ethnicity/race. Approximately one-third (34.7%) of patients had Rai stage 0-I disease, 6.9% had stage II, 6.3% stage III, 11.5% stage IV, and 40.6% had undocumented Rai stage. Testing patterns: The majority of identified patients (3202/3654; 87.6%) had undergone cytogenetic testing, fluorescence in situ hybridization, or IGHV mutation testing. Compared with 2015-2016, testing rates were higher in 2019-2020 for chromosome 17p deletion (del(17p); 36.1% vs 45.7%, respectively; p<0.001) and for IGHV mutation status (84.7% vs 89.2%, respectively; p=0.003). Overall, 11.0% of patients had del(17p). Of those tested for IGHV (1472/3654; 40.3%), 58.3% had unmutated IGHV. Treatment patterns: The 10 most commonly used 1L CLL treatments, which overall represented 91.8% of all 1L treatments, and their evolution over time, are reported in Table 1. Of the patients receiving these top 10 1L treatment regimens overall, 45.7% received regimens including novel targeted oral agents, 33.4% received chemo-immunotherapy (CIT), and 19.7% received anti-CD20 monotherapy. Evaluation of each 2-year period shows that treatment patterns for the top 10 1L treatment regimens shifted, with use of novel targeted oral agents increasing from 27.1% (2015-2016) to 63.8% (2019-2020) (p<0.001), while use of CIT and chemotherapy decreased over time (Table 2). Approximately 30.0% (1088/3654) of 1L-treated patients went on to receive second-line treatments. Outcomes: Median TTNTD was 34.4 months for all patients receiving 1L CLL treatment, and 36.5 months for patients who received the 10 most common 1L treatments across the 6-year study period (n=3360). Median TTNTD was 47.0 months for patients who received novel targeted oral agents and 41.5 months for patients who received CIT (unadjusted p=0.16). When evaluating outcomes in patients with high-risk cytogenetics, median TTNTD was 29.1 months for patients with del(17p) and 37.2 months for those with unmutated IGHV, but was longer in those patients who received treatment with novel targeted oral agents (median TTNTD of 43.9 and 46.7 months, respectively; Table 3). Conclusions: This analysis provides the current state of 1L CLL testing and treatment patterns and outcomes in the US from 2015 to 2020. As expected, the use of novel targeted oral agents increased over time, with a corresponding increase in TTNTD. Clinical outcomes were improved in patients receiving novel targeted oral agents, both overall and in high-risk subgroups. Following on from this, a comparative study of TTNTD for novel oral agents versus CIT, and analyses of outcomes of different sequencing of therapies, will be conducted. Figure 1 Figure 1. Disclosures Mato: Nurix: Research Funding; Johnson and Johnson: Consultancy, Research Funding; AbbVie: Consultancy, Research Funding; Acerta/AstraZeneca: Consultancy, Research Funding; DTRM BioPharma: Consultancy, Research Funding; Pharmacyclics LLC, an AbbVie Company: Consultancy, Research Funding; Adaptive Biotechnologies: Consultancy, Research Funding; BeiGene: Consultancy, Research Funding; MSKCC: Current Employment; Sunesis: Consultancy, Research Funding; AstraZeneca: Consultancy; TG Therapeutics: Consultancy, Other: DSMB, Research Funding; Genmab: Research Funding; LOXO: Consultancy, Research Funding; Genentech: Consultancy, Research Funding; Janssen: Consultancy, Research Funding. Ravelo: Genentech, Inc.: Current Employment; Roche Holdings: Current equity holder in publicly-traded company, Current holder of stock options in a privately-held company. To: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months. Schuldt: Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company; Johnson & Johnson: Divested equity in a private or publicly-traded company in the past 24 months. Biondo: Genentech, Inc.: Current Employment; Roche: Current holder of individual stocks in a privately-held company.

scholarly journals Daratumumab (DARA) Plus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Patients with Transplant-Eligible Newly Diagnosed Multiple Myeloma (NDMM): Updated Efficacy and Safety Analysis of the Safety Run-in Population of Griffin

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 48-49
Author(s):  
Peter M. Voorhees ◽  
Cesar Rodriguez ◽  
Brandi Reeves ◽  
Nitya Nathwani ◽  
Luciano J. Costa ◽  
...  
Keyword(s):  
Research Funding ◽  
Progressive Disease ◽  
Standard Of Care ◽  
Advisory Board ◽  
Efficacy And Safety ◽  
Publicly Traded ◽  
Safety Concerns ◽  
Best Response ◽  
The Us

Introduction: DARA, a human IgGκ monoclonal antibody targeting CD38, is approved in combination with bortezomib, thalidomide and dexamethasone for transplant-eligible NDMM; however, RVd is the standard of care in the US for transplant-eligible NDMM. The phase 2 GRIFFIN study (NCT02874742) of DARA plus RVd (D-RVd) for transplant-eligible NDMM in the US included a safety run-in phase followed by a randomized phase. In the 16-patient safety run-in, no dose limiting toxicities (DLTs) occurred and no new safety concerns were identified for D-RVd (Voorhees P, Blood 2018 132 [Suppl 1]:151). The randomized phase of GRIFFIN met its prespecified primary endpoint with improved rate of stringent complete response (sCR) by the end of consolidation (D-RVd, 42.4% vs RVd, 32.0%; 1-sided P=0.068) (Voorhees P, Blood 2020); however, median follow-up was limited (13.5 months). Therefore, we conducted an updated analysis of efficacy and safety for the safety run-in cohort to examine longer follow-up data for D-RVd and evaluate the role of DARA plus lenalidomide (D-R) maintenance therapy. Methods: Patients with NDMM eligible for high-dose therapy (HDT) and autologous stem cell transplant (ASCT) were enrolled. Patients in the safety run-in cohort received 4 induction cycles of D-RVd, HDT, ASCT, 2 consolidation cycles of D-RVd, and 24 months of maintenance with D-R. During induction and consolidation, patients received R 25 mg PO on Days 1-14; V 1.3 mg/m2 SC on Days 1, 4, 8, and 11; and d 40 mg QW every 21 days. DARA 16 mg/kg IV was given on Days 1, 8, and 15 of Cycles 1-4 and Day 1 of Cycles 5-6. During maintenance (Cycles 7-32), patients received R 10 mg (15 mg in Cycles 10+ if tolerated) on Days 1-21 every 28 days plus DARA 16 mg/kg IV Q8W (or Q4W per patient decision after protocol Amendment 2). The primary objective of the safety run-in phase was to assess DLTs. Additional endpoints included safety, sCR rate by the end of consolidation and end of maintenance per IMWG criteria by computer algorithm, progression-free survival (PFS), and minimal residual disease (MRD) negativity (10-5 and 10-6 threshold) assessed by next-generation sequencing (clonoSEQ; Adaptive Biotechnologies). Results: Sixteen patients enrolled in the safety run-in and received D-RVd. Median age was 62.5 years, 8 (50%) patients were male, 4 (25%) were International Staging System stage II or III, and 4 (25%) had high cytogenetic risk based on fluorescence in situ hybridization. Thirteen (81%) patients completed study therapy, and 3 (19%) patients discontinued due to progressive disease (n=2) or adverse event (n=1). With continued therapy, responses further deepened for D-RVd (Figure). By the end of D-RVd consolidation, all patients responded, with a best response of sCR for 9 (56%) patients. By the end of D-R maintenance therapy, 15 (94%) patients achieved a best response of sCR. MRD negativity (10‒5) at the end of consolidation occurred in 8 patients (50%); no patient was MRD negative at 10-6. By the end of maintenance, 13 (81%) patients were MRD negative at 10‒5, and 5 (31%) were MRD negative at 10‒6. At a median follow-up of 39.0 months, 3 of 16 patients have progressed, with estimated 24-month and 36-month PFS rates of 94% and 78%, respectively. One death occurred, due to progressive disease in the patient who failed to achieve sCR. With longer follow-up, no new safety concerns were identified. Grade 3-4 treatment emergent adverse events (TEAEs) occurred in 14 (88%) patients, with the most common being neutropenia (44%; n=7), pneumonia (31%; n=5), lymphopenia (25%; n=4), and thrombocytopenia (25%; n=4). One patient had TEAEs leading to discontinuation of study treatment. No grade 5 TEAEs occurred. Infusion-related reactions occurred in 4 (25%) patients (all grade 1-2), mainly during the first cycle. Conclusions: With >3 years of follow-up in the safety run-in cohort of GRIFFIN, D-RVd induction, ASCT, and consolidation led to durable responses that deepened with 2 years of D-R maintenance. There were no new safety concerns. These emerging data suggest that the deep responses seen in the randomized phase of GRIFFIN are durable and will continue to improve over time, and that D-RVd may be a potential new standard of care for transplant-eligible NDMM. Support: Alliance Foundation Trials; https://acknowledgments.alliancefound.org; Janssen Oncology Disclosures Voorhees: Janssen: Other: Advisory Board; Novartis: Consultancy; Oncopeptides: Consultancy, Honoraria; TeneoBio: Other: Advisory Board; Adaptive Biotechnologies: Other: Advisory Board; BMS: Other: Advisory Board; GSK: Honoraria. Rodriguez:BMS, Takeda, Amgen: Consultancy, Speakers Bureau. Reeves:Bristol Myers Squibb: Speakers Bureau; Incyte: Honoraria; Takeda: Honoraria. Costa:Amgen: Consultancy, Honoraria, Research Funding; BMS: Consultancy, Honoraria; Genentech: Consultancy; Celgene: Consultancy, Honoraria; AbbVie: Consultancy; Sanofi: Consultancy, Honoraria; Janssen: Consultancy, Honoraria, Research Funding. Lutska:Janssen: Current Employment. Bobba:Janssen: Current Employment. Hoehn:Merck (current), Janssen (within 24 months): Current Employment, Ended employment in the past 24 months. Pei:Janssen: Current Employment, Current equity holder in publicly-traded company. Ukropec:Janssen: Current Employment, Current equity holder in publicly-traded company. Qi:Janssen: Current Employment, Current equity holder in publicly-traded company; Johnson and Johnson: Current equity holder in publicly-traded company. Lin:Janssen Scientific Affairs: Current Employment, Current equity holder in publicly-traded company. Richardson:Celgene/BMS, Oncopeptides, Takeda, Karyopharm: Research Funding. OffLabel Disclosure: The specific regimen combination is not yet approved, but individual components are.

scholarly journals Real-World Effectiveness of Voxelotor for Treating Sickle Cell Disease in the US

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 25-25
Author(s):  
Ahmar Urooj Zaidi ◽  
Thokozeni Lipato ◽  
Ofelia A. Alvarez ◽  
Alexander Lonshteyn ◽  
Derek Weycker ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Real World ◽  
Research Funding ◽  
Index Date ◽  
Health Claims ◽  
Transfusion Rate ◽  
Publicly Traded ◽  
Advisory Committees ◽  
The Us

Background: Until late 2019, few treatments had been approved by the FDA for treating sickle cell disease (SCD). Voxelotor (Oxbryta®) is a sickle hemoglobin-polymerization inhibitor approved by the FDA in November 2019 for treatment of SCD in adults and adolescents aged ≥12 years under an accelerated approval based on results of the pivotal HOPE study. In HOPE, voxelotor increased average hemoglobin (Hb) by 1.1 g/dL from baseline in patients with 1-10 vaso-occlusive crises (VOCs) in the previous year and a Hb level between 5.5 and 10.5 g/dL who were not transfusion dependent. Of the participants on voxelotor 1500 mg, 51% had a Hb response >1.0 g/dL at week 24 (Vichinsky et al, NEJM 2019). This study sought to assess the real-world effectiveness of voxelotor based on data during the first 6 months post FDA approval. Methods: Data on medical and pharmacy claims for patients who were aged ≥12 years and receiving voxelotor from December 2019 to May 2020 were obtained from the Symphony Health claims dataset. For each patient, the date of the first voxelotor claim was defined as the "index date." Patients with at least 1 year's data prior to the index date were included in the analyses. Patients' demographic and clinical characteristics were summarized descriptively. Standardized annualized rates of transfusions and VOCs per patient per year (PPPY) prior to and after voxelotor initiation were compared. A subset of patients in the Symphony Health claims dataset had Hb lab data available. Patients with at least 1 Hb result within 30 days prior to the index date and at least 1 Hb result after the index date were included for Hb analyses. For these patients, change in Hb and the percentage of patients achieving a >1 g/dL increase in Hb were summarized. Confidence intervals and P values for changes in outcomes were based on bootstrapping. Results: As of May 31, 2020, 1275 patients from the Symphony Health claims datasets were identified who received voxelotor (40% male, mean age 35.7 years). In the year prior to voxelotor initiation, 715 (56.1%) of these patients received hydroxyurea, 121 (9.5%) received L-glutamine, 166 (13.0%) received at least 1 transfusion, 17 (1.3%) were on chronic transfusion (≥8 transfusions per year), and 681 (53.4%) had 1 or more VOCs. Mean (SD) follow-up was 64.9 (40.7) days. Among 1275 patients, 175 and 52 patients had at least 1 Hb level measurement during the 1 year prior to and after voxelotor initiation, respectively. Among the subset of patients with their Hb level tested within 30 days prior to the index date and at least 1 Hb level after index date (n=22), the baseline average Hb level was 8.0 g/dL (SD 1.4, median 7.9 g/dL, range 5.0-11.8 g/dL). Mean increase in Hb from baseline was 1.1-1.3 g/dL (Table 1) depending on the approach used to calculate Hb levels after voxelotor initiation; 55% (95% CI 32%-77%) of patients achieved a Hb increase >1 g/dL after voxelotor initiation. Among all 1275 patients, mean (SD) overall transfusion rates declined from 0.45 (1.67) PPPY pre-index to 0.31 (1.88) post-index, a change of -0.14 PPPY (P=0.005). Among 169 patients who received at least 1 transfusion in the year prior to initiation of voxelotor, the transfusion rate dropped from 3.39 (3.34) to 1.75 (4.30) PPPY, a change of -1.64 PPPY (P<0.001). Among 17 patients receiving chronic transfusions, the transfusion rate dropped from 11.29 (3.12) to 6.74 (7.37) PPPY, a change of -4.56 PPPY (P=0.013) (Table 2). After voxelotor initiation, the annualized rates of VOC were numerically reduced from 3.86 (6.69) to 3.64 (8.54) (P=0.248). To address the potential bias from the relatively short follow-up duration, similar results of transfusion and VOC rates were observed among patients with at least 30 days of follow-up or when only events within 3 months prior to index date were considered. Conclusions: Based on the first 6 months' data after the approval of voxelotor in the US, in real-world practice, voxelotor increases Hb by at least 1 g/dL, consistent with the HOPE randomized controlled trial results. Evidence suggests that transfusion rates decreased after voxelotor initiation. A favorable downward trend in VOC rates was also observed. This real-world evidence provides additional support for the use of this novel therapy in the treatment of hemolytic anemia and its associated complications in the SCD population. Further evaluation with a larger sample size and longer follow-up will help to confirm these findings. Disclosures Zaidi: Global Blood Therapeutics: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Consultancy, Honoraria; Cyclerion: Consultancy, Honoraria; Imara: Consultancy, Honoraria; bluebird bio: Consultancy, Honoraria; Emmaus Life Sciences: Consultancy, Honoraria. Alvarez:Novartis: Membership on an entity's Board of Directors or advisory committees; Forma Therapeutics: Membership on an entity's Board of Directors or advisory committees. Lonshteyn:Policy Analysis Inc.: Current Employment; Global Blood Therapeutics: Research Funding; Novartis: Research Funding. Weycker:Policy Analysis Inc.: Current Employment, Current equity holder in publicly-traded company; Novartis: Research Funding; Global Blood Therapeutics: Research Funding. Pham:Policy Analysis Inc.: Current Employment; Global Blood Therapeutics: Research Funding; Novartis: Research Funding. Delea:Global Blood Therapeutics: Research Funding; Novartis: Research Funding; Policy Analysis Inc.: Current Employment, Current equity holder in private company. Agodoa:Global Blood Therapeutics: Current Employment, Current equity holder in publicly-traded company. Cong:Global Blood Therapeutics: Current Employment, Current equity holder in publicly-traded company. Shah:Novartis: Consultancy, Research Funding, Speakers Bureau; Global Blood Therapeutics: Consultancy, Research Funding, Speakers Bureau; CSL Behring: Consultancy; Alexion: Speakers Bureau; Bluebird Bio: Consultancy.

scholarly journals Treatment Patterns and Outcomes of Multiple Myeloma (MM) with Chromosome Translocation (11;14) in United States (US) Routine Clinical Practice

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 40-43
Author(s):  
Shebli Atrash ◽  
Evelyn M. Flahavan ◽  
Tao Xu ◽  
Esprit Ma ◽  
Sudeep Karve ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Research Funding ◽  
Community Setting ◽  
B Cell Lymphoma ◽  
Chromosome Translocation ◽  
Routine Clinical Practice ◽  
Chromosome 1 ◽  
Test Results ◽  
Publicly Traded ◽  
The Us

Introduction: MM is an incurable disease with risk categorizations by cytogenetic abnormalities. Prognostic implications of chromosome translocation t(11;14)+ are important to inform development of biomarker-targeted therapies such as the B-cell lymphoma-2 (BCL-2) pathway inhibitors. This study aims to evaluate MM treatment (Tx) patterns and outcomes of t(11;14)+ compared with other cytogenetic cohorts in US routine clinical practice. Methods: A retrospective observational cohort study of the Flatiron Health database, which comprises de-identified electronic health record-derived patient (pt)-level data from over 280 community and academic cancer clinics in the US. Pts aged ≥18 years who received a first-line (1L) induction Tx within ≤60 days of MM diagnosis from 1/1/2011 to 1/31/2020, and were not enrolled in a clinical trial were identified at the start of each Tx line (index date) and followed up (f/u) until 1/31/2020. Only cytogenetic results by fluorescence in situ hybridization (FISH) were used to stratify the cohort into: t(11;14)+; standard risk, excluding t(11;14)+ (SR); and high risk, (HR; del 17p, t(4;14), t(14;16), t(14;20), chromosome 1 abnormalities as a proxy for 1q gain; and t(11;14) where it co-occurs). Descriptive analyses of pt characteristics and Tx were conducted. Kaplan-Meier analyses were used to evaluate median time to next treatment (TTNT) and overall survival (OS) with log-rank test for significance. The cohort was stratified by age ≤70/>70 years (yrs) as a proxy for transplant eligibility. Results: Of 10,703 pts with MM in the database, 5982, 3059 and 1595 pts were eligible for 1L, second-line (2L), and third-line (3L) analysis, of which 76%, 84%, and 86%, respectively, had FISH test results before Tx initiation. 14% of pts with FISH test results were t(11;14)+, ~55% SR and ~35% HR. Included pts were predominately male (55%), ~90% treated in the community setting and ~16% African-American. Pts in the 3L cohort were younger at diagnosis with a median age (interquartile range) of 67 (59 ̶ 74) yrs compared with 69 (61 ̶ 76) yrs in both 1L and 2L. The 1L Tx pattern was consistent across cytogenetic cohorts with bortezomib (V), lenalidomide (R), dexamethasone (d) as the most common Tx (>42%). Together, VRd, Rd, Vd, and cyclophosphamide (Cy) in combination with Vd represented ≥90% of 1L Tx (Figure 1). Across 2L cytogenetic cohorts, the most common Tx regimens (≥25%) were VRd and Rd. The use of regimens containing carfilzomib (K) and daratumumab (D) was emerging in 2L: KRd in 8% of t(11;14)+ and HR and 5% in SR; Kd 5% in all groups; DRd in 4% of t(11;14)+ and 3% in SR and HR (Figure 1). 3L Tx pattern was fragmented with different Tx regimens. Rd was the most common 3L Tx option: 9% in t(11;14)+, 10% in SR and 7% in HR (Figure 1). Across all lines of Tx, t(11;14)+ had similar Tx outcomes to SR, and HR had poorest outcomes (Table 1). Pts TTNT shortened as they advanced to later lines of Tx (Table 1). Although TTNT did not differ by age across lines of Tx, OS was superior in pts <70 yrs across all 3 cytogenetic risk cohorts, especially in 1L where transplant in those ≤70 yrs vs >70 yrs at diagnosis was 44% vs 11% for t(11;14)+, 47% vs 10% for SR and 49% vs 9% for HR. Conclusions: This study identified cytogenetic subgroups across 1L to 3L in a predominately community setting in the US. MM t(11;14)+ pts had similar 1L and 2L Tx patterns to SR and HR. Across all lines of Tx, the outcomes of t(11;14)+ and SR pts were comparable and better than HR pts. TTNT was reduced as pts advanced to later lines of Tx. Pts continuing to 3L Tx were younger at diagnosis, but there was not a clear-cut standard of care. While TTNT did not differ by age across lines of Tx, OS was superior in pts <70 yrs across all 3 cytogenetic risk cohorts. This study sets the benchmark for novel treatment options, such as BCL-2 pathway inhibitors, primarily wherever available biomarker-driven therapy is considered appropriate. Disclosures Atrash: Takeda, Amgen, Karyopharm, BMS, Sanofi, Cellactar, Janssen and Celgene: Honoraria; BMS, Jansen oncology, Sanofi: Speakers Bureau; Amgen, GSK, Karyopharm.: Research Funding. Flahavan:F. Hoffmann-La Roche: Current equity holder in publicly-traded company; Roche Products Ltd.: Current Employment. Xu:F. Hoffmann-La Roche Ltd: Current Employment, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Ma:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company. Karve:AbbVie: Current Employment, Current equity holder in publicly-traded company. Hong:F. Hoffmann-La Roche: Current equity holder in publicly-traded company; Genentech, Inc.: Current Employment. Jirau-Lucca:Genentech, Inc.: Current Employment; AbbVie: Current equity holder in publicly-traded company; BMS: Current equity holder in publicly-traded company; Celgene: Divested equity in a private or publicly-traded company in the past 24 months. Nixon:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; F. Hoffmann-La Roche: Current Employment, Current equity holder in publicly-traded company. Ailawadhi:Takeda: Honoraria; Pharmacyclics: Research Funding; Cellectar: Research Funding; BMS: Research Funding; Medimmune: Research Funding; Phosplatin: Research Funding; Amgen: Research Funding; Celgene: Honoraria; Janssen: Research Funding.

scholarly journals Trends in Prescribing Practices for Management of Hemophilia

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 2112-2112
Author(s):  
Randall Curtis ◽  
Jonathan C. Roberts ◽  
Nicole Crook ◽  
Marquita Decker-Palmer ◽  
Rahul Khairnar ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Research Funding ◽  
Clotting Factor ◽  
Prescribing Practices ◽  
Publicly Traded ◽  
Major Life ◽  
The Past ◽  
The Us ◽  
Life Threatening ◽  
High Doses

Abstract Introduction: Over the past 21 years, treatment options for hemophilia have evolved significantly. The objective of this study is to describe the trends observed in clinician prescribing practices for management of hemophilia A (HA) and B (HB) in the United States (US) via three surveys from 1999-2021. Methods: We administered surveys to members of the Hemostasis & Thrombosis Research Society (HTRS) via an in-person paper survey at its annual symposia in 1999 and 2015, and an online survey in 2021. The survey participants included physicians, physician assistants, and nurse practitioners who manage the care of hemophilia patients at hemophilia treatment centers in the US. The surveys collected information regarding: 1) characteristics of clinician practice, 2) prescribed clotting factor products and dosages used for routine bleeds or major life-threatening bleeding, total joint replacement, and port placement, 3) reasons for changing doses, 4) frequency of recommendation for prophylaxis and inhibitor treatment for associated factor and non-factor products, and 5) gene therapy. Results: Forty-one clinicians completed the survey in 1999 and 2021, 53 in 2015. The mean number of patients seen by respondents increased from 142 (range: 0-314) for children and 101 (0-480) for adults in 1999 to 202 (0-900) for children and 154 (0-500) for adults in 2021. The proportion of clinicians prescribing >40 units/kg of Standard Half Life (SHL) Factor IX concentrates for routine bleeding events in HB patients increased from 22.5% in 1999 to 50.9% in 2015, and 87.8% in 2021. The proportion of clinicians reported SHL Factor VIII usage for routine bleeding at a dose of >40 units/kg in HA patients increased from none in 1999 to 11.3% in 2015 and 29.3 % in 2021. The reported rates of prescribing an average >60 units/kg factor to treat major life-threatening bleeds increased from 67.5% in 1999 to 90.3% in 2021 for HB; rates were 2.5% in 1999, 17.3% in 2015 and 7.3% in 2021 for treating HA. For children <4 years old, 22.2% of clinicians prescribed primary prophylaxis all of the time in 1999. This rose to 68.2% in 2015, and 86.5% in 2021. For adults, 12.5% of clinicians prescribed secondary prophylaxis all of the time in 1999, 27.3% in 2015 and 42.5% in 2021. For treatment of patients with HA or HB inhibitors, the proportions of clinicians who reported prescribing immune tolerance induction (ITI) therapy all of the time for pediatric patients were 50%, 75.0% and 63.2% in three surveys, but <25% for adult patients. In the 2021 survey, >91% of clinicians reported prescribing emicizumab to treat HA inhibitors in patients of all ages, while >87% reported prescribing it to treat HA without inhibitor. Clinicians were more likely to always prescribe emicizumab to treat HA patients with inhibitors (63.2% for children and 57.1% for adults), as compared to always prescribing it for those without inhibitors (13.2% for children and 5.7% for adults). The most frequent reported method to treat a patient with a history of inhibitors on emicizumab who had break through bleeds was rFVIIa: 85.4% for children, and 75.6% for adults. The most frequently reported reasons for switching from FVIII to emicizumab were fewer injections/visits (87.8%), and improved patient quality of life (82.9%). Thirty-nine percent of clinicians reported caring for patients currently in gene therapy trials, 27.5% had patients who had completed gene therapy. When asked about potential future prescribing practices, 14.6% reported that they would prescribe gene therapy "all the time", 4.9% would prescribe it "about 3/4 of the time", 29.3% "about 1/2 the time", 29.3% "about 1/4 the time", and 22.0% "rarely or never". Conclusion: These data indicate changes in prescribing practices among hemophilia specialists in the US over the past 21 years. Prescribing of high doses of factor (>40 units/kg) increased, while ITI prescribing practices remained similar over time. To treat patients with major life-threatening bleeds, a larger proportion of clinicians prescribed high doses of factor (>60 units/kg) for patients with HB as compared to HA. Most clinicians frequently prescribed emicizumab for patients with HA inhibitors, but less frequently for those without inhibitors. At this time, there is wide diversity among clinicians in the expected uptake of gene therapy. Disclosures Curtis: Pfizer, Bayer, and Novo Nordisk: Consultancy; University of Southern California: Consultancy. Roberts: Takeda; Speakers Bureau: Novo Nordisk, Octapharma, Sanofi, Takeda.: Research Funding; Genentech, Novo Nordisk, Octapharma, Pfizer, Sanofi, Takeda, uniQure: Consultancy. Decker-Palmer: Genentech Inc. --A member of the Roche Group.: Current Employment, Current equity holder in publicly-traded company. Khairnar: Genentech Inc - A Member of The Roche Group: Current Employment; University of Maryland, Baltimore: Ended employment in the past 24 months; Roche: Current equity holder in publicly-traded company. Wu: Baxalta US Inc., Bannockburn, IL (a Takeda Company), CSL Behring L.L.C., Octapharma USA, Inc., Genentech Inc.: Research Funding. Nichol: Pfizer, Genentech Inc., Baxalta US Inc., Bannockburn, IL (a Takeda Company), Octapharma, CSL Behring, Global Blood Therapeutics, and Novo Nordisk: Research Funding.

scholarly journals Perceived Barriers to COVID-19 Testing

2021 ◽  
Vol 18 (5) ◽  
pp. 2278
Author(s):  
Pearl A. McElfish ◽  
Rachel Purvis ◽  
Laura P. James ◽  
Don E. Willis ◽  
Jennifer A. Andersen
Keyword(s):  
Analytical Approach ◽  
Nasal Swab ◽  
Healthcare Providers ◽  
Research Participant ◽  
Perceived Barriers ◽  
Lower Income ◽  
The Us ◽  
Qualitative Descriptive ◽  
Descriptive Method ◽  
Background Prior

(1) Background: Prior studies have documented that access to testing has not been equitable across all communities in the US, with less testing availability and lower testing rates documented in rural counties and lower income communities. However, there is limited understanding of the perceived barriers to coronavirus disease 2019 (COVID-19) testing. The purpose of this study was to document the perceived barriers to COVID-19 testing. (2) Methods: Arkansas residents were recruited using a volunteer research participant registry. Participants were asked an open-ended question regarding their perceived barriers to testing. A qualitative descriptive analytical approach was used. (3) Results: Overall, 1221 people responded to the open-ended question. The primary barriers to testing described by participants were confusion and uncertainty regarding testing guidelines and where to go for testing, lack of accessible testing locations, perceptions that the nasal swab method was too painful, and long wait times for testing results. (4) Conclusions: This study documents participant reported barriers to COVID-19 testing. Through the use of a qualitative descriptive method, participants were able to discuss their concerns in their own words. This work provides important insights that can help public health leaders and healthcare providers with understanding and mitigating barriers to COVID-19 testing.

scholarly journals 338. Analysis of Etiologies of Aseptic Meningitis within a Nation-Wide Hospital Network

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S239-S239
Author(s):  
Arunmozhi S Aravagiri ◽  
Scott Kubomoto ◽  
Ayutyanont Napatkamon ◽  
Sarah Wilson ◽  
Sudhakar Mallela
Keyword(s):  
United States ◽  
West Nile Virus ◽  
Aseptic Meningitis ◽  
Epidemiologic Study ◽  
The United States ◽  
Positive Test ◽  
Diagnostic Tools ◽  
West Nile ◽  
Varicella Zoster ◽  
The Us

Abstract Background Aseptic meningitis can be caused by an array of microorganisms, both bacterial and non-bacterial, as well as non-infectious conditions. Some etiologies of aseptic meningitis require treatment with antibiotics, antiviral, antifungals, anti-parasitic agents, immunosuppressants, and or chemotherapy. There are limited diagnostic tools for diagnosing certain types of aseptic meningitis, therefore knowing the differential causes of aseptic meningitis, and their relative percentages may assist in diagnosis. Review of the literature reveals that there are no recent studies of etiologies of aseptic meningitis in the United States (US). This is an epidemiologic study to delineate etiologies of aseptic meningitis in a large database of 185 HCA hospitals across the US. Methods Data was collected from January 2016 to December 2019 on all patients diagnosed with meningitis. CSF PCR studies, and CSF antibody tests were then selected for inclusion. Results Total number of encounters were 3,149 hospitalizations. Total number of individual labs analyzed was 10,613, and of these 262 etiologies were identified. 23.6% (62) of cases were due to enterovirus, 18.7% (49) due to HSV-2, 14.5% (38) due to West Nile virus, 13.7% (36) due to Varicella zoster (VZV), 10.5% (27) due to Cryptococcus. Additionally, we analyzed the rate of positive test results by region. Nationally, 9.7% of tests ordered for enterovirus were positive. In contrast, 0.5% of tests ordered for HSV 1 were positive. The southeastern United States had the highest rate of positive tests for HSV 2 (7% of tests ordered for HSV 2 were positive). The central United States had the highest rate of positive test for West Nile virus (11% of tests ordered for West Nile were positive). The northeastern region and the highest rate of positive tests for varicella zoster (18%). Table 1: Percentage of positive CSF tests (positive tests/tests ordered) Table 2: Lists the number of HIV patients and transplant patients that had positive CSF PCR/serologies Figure 1: Percentage of positive CSF tests in each region Conclusion Approximately 40% of aseptic meningitis population had treatable etiologies. A third of the Cryptococcus meningitis population had HIV. Furthermore, enteroviruses had the majority of cases within the US, which are similar to studies done in other parts of the world. Disclosures All Authors: No reported disclosures

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