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Total 23 results found since Jan 2013.
Low Hemoglobin Increases Risk for Stroke, Kidney Disease, Elevated Estimated Pulmonary Artery Systolic Pressure, and Premature Death in Sickle Cell Disease: A Systematic Literature Review and Meta-Analysis
Conclusions: In conclusion, comprehensive evaluation of peer-reviewed literature published over the past 20 years demonstrates a significant relationship between chronic anemia and worse clinical outcomes in individuals with SCD. Meta-analyses further demonstrate that even relatively modest differences in hemoglobin are important and support hemoglobin increase by ≥1 g/dL as a relevant therapeutic target. These results suggest that interventions that reduce hemolytic anemia may confer clinical benefit in this patient population.DisclosuresAtaga: Novartis: Consultancy, Honoraria; Bioverativ: Honoraria, Membership on an e...
Source: Blood - November 21, 2018 Category: Hematology Authors: Ataga, K. I., Gordeuk, V. R., Allen, I. E., Colby, J., Gittings, K., Agodoa, I. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Organ Damage and Clinical Complications in Sickle Cell Disease Source Type: research
SOD3-Mimetic As a Complement for Genetic Therapy in Sickle Cell Disease
Conclusions:The present results support development of SanFlow, delivered through continuous infusion, for anemic SCD children to prevent the development of blood transfusion dependency in order to avoid stroke and painful vaso-occlusive crisis (VOC). These results also demonstrated that SanFlow can be used safely and effectively in the elimination of serious painful vaso-occlusive crisis and protect silent and major strokes. Clinical trials of SanFlow in SCD children, prior to their transfusion dependence, as well as in transfusion-dependent teenagers and adults with SCD patients are warranted. By extension, ß-Thala...
Source: Blood - November 21, 2018 Category: Hematology Authors: Hsia, C. J., Ma, L. Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science: Poster II Source Type: research
Relationship between Hemolytic Index Early in Life with Development of Abnormal Transcranial Doppler Velocities in Pediatric Patients with Sickle Cell Anemia
Conclusions: This retrospective study noted an increased risk for development of an abnormal or conditional TCD with an increased HI. The addition of hemoglobin to the HI to form HHI showed an elevated risk for abnormal or conditional TCD to the same degree as HI itself. These results could offer an additional tool in the evaluation of the pediatric SCA population, although these results would benefit from a prospective look. Further look into a possible association between HI and other SCD related complications such as stroke and acute chest syndrome could provide further information for the treating team for the overall ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Goubeaux, D. L., Sherman, A., Kalpatthi, R., Woods, G. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research
Real-World Comparisons of Cardiovascular Events between Different Tyrosine Kinase Inhibitors Among Patients with Chronic Myeloid Leukemia
CONCLUSION: CP-CML patients treated with different TKIs (ponatinib, bosutinib, imatinib, dasatinib, and nilotinib) did not have different incidence of cardiovascular events (MACE, AOEs, VTEs) in this small cohort of real-world patients with ≥6-month of follow-up. The results were consistent among patients with prior use of one and two TKI types.DisclosuresLevy: Takeda (Millennium Pharmaceuticals, Inc.): Consultancy. Xie: STATinMED Research: Employment. Wang: STATinMED Research: Employment. Neumann: Takeda (Millennium Pharmaceuticals, Inc.): Employment. Srivastava: Takeda (Millennium Pharmaceuticals, Inc.): Employment. N...
Source: Blood - November 21, 2018 Category: Hematology Authors: Levy, M. Y., Xie, L., Wang, Y., Neumann, F., Srivastava, S., Naranjo, D., Zhang, Q., Dalal, M. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster II Source Type: research
Characterizing a Population with Severe Manifestations of Sickle Cell Disease Using U.S. Real-World Evidence
ConclusionsBased on this contemporary real-world evidence from the U.S. setting, a substantial proportion of patients with SCD experience a diverse set of severely debilitating complications of the disease, as well as other co-morbidities associated with the disease. The high rate of comorbidities across all age groups and overrepresentation of patients younger than 30 years of age (relative to age distribution of general U.S. population) indicate that patients with SCD still experience significant morbidity and early mortality with current standard medical care. Given the notable heterogeneity of the clinical manifestatio...
Source: Blood - November 21, 2018 Category: Hematology Authors: Paramore, C., Kong, A., Minegishi, S., Shi, W. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster III Source Type: research
Increased Risk of Lymphoid Malignancy in Patients with Herpes Zoster: A Longitudinal Follow-up Study Using a National Cohort Study
Conclusion: Our study demonstrates that herpes zoster infection increases the risk of subsequent lymphoid malignancies irrespective of age and gender in the Korean population.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Lee, Y. K., Kim, M., Kim, H. J., Lim, H., Choi, H. G. Tags: 902. Health Services Research-Malignant Diseases: Quality Of Life Studies Source Type: research
On the Links between Arterial and Venous Thrombosis: A Comparative Genomic Analysis of Cardiovascular Disease and Venous Thromboembolism
Discussion and conclusion: our comparative analyses provide new data on how venous and arterial thrombosis differ at a transcriptional level. Specifically, we identified a set of 473 genes whose expression levels were consistently changed in both CVD and VTE, and 124 genes with divergent expression levels between these conditions. The prominence of pathways associated with hemostasis, innate immunity and neutrophil function in both CVD and VTE are in line with the concept of immunothrombosis in the pathogenesis of thrombotic disorders. The clustering of MI, IS and PAOD compared to VTE and CS is coherent with the concept th...
Source: Blood - November 21, 2018 Category: Hematology Authors: Hounkpe, B. W., Benatti, R. O., Carvalho, B. S., De Paula, E. V. Tags: 331. Pathophysiology of Thrombosis: Poster III Source Type: research
Rivaroxaban Thromboprophylaxis in High-Risk Ambulatory Cancer Patients Receiving Systemic Therapy: Results of a Randomized Clinical Trial (CASSINI)
Conclusions: Rivaroxaban significantly reduced VTE and VTE-related death during the on-treatment period but not during the full study period; over one-third of events occurred post discontinuation of study drug. The incidence of major bleeding was low. The Khorana risk score cut-off of ≥2 identified cancer patients at high risk of thrombotic events both at baseline (4.53%) and during study (8.79% with additional 1.66% arterial events in placebo group). These results should inform future recommendations regarding thromboprophylaxis in at-risk ambulatory cancer patients.(Funded by Janssen; ClinicalTrials.gov number, NCT02...
Source: Blood - November 21, 2018 Category: Hematology Authors: Khorana, A. A., Soff, G. A., Kakkar, A. K., Vadhan-Raj, S., Riess, H., Wun, T., Streiff, M. B., Garcia, D. A., Liebman, H. A., Belani, C., O'Reilly, E. M., Patel, J. N., Yimer, H. A., Wildgoose, P., Burton, P., Vijapurkar, U., Kaul, S., Eikelboom, J., McB Tags: Late-Breaking Abstracts Session Source Type: research
Burden of Hospital Readmissions for Venous Thromboembolism Among Patients with Cancer
Conclusions: In this real-world study, many patients hospitalized for cancer experienced a VTE event requiring re-hospitalization, which was the highest proportion of readmitted patients among acute medical illnesses analyzed in this study, with almost 30% readmitted within 30 days of post-discharge. Total costs of readmissions were substantial, as high as $41K for resubmissions due to primary diagnosis of DVT/PE. Improvement in VTE prophylaxis for patients with cancer may reduce the risk and frequency of VTE, and thus hospital readmissions, reducing the clinical and economic burden of VTE in this patient population.Sponso...
Source: Blood - November 21, 2018 Category: Hematology Authors: Amin, A. J., Deitelzweig, S., Lin, J., Lingohr-Smith, M., Menges, B., Neuman, W. R. Tags: 901. Health Services Research-Non-Malignant Conditions: Thrombosis and Anticoagulation Source Type: research
Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease
Conclusions: Data from Part A of the HOPE study demonstrate that treatment with voxelotor resulted in a dose-dependent increase in Hb with a large proportion of patients achieving Hb >1 g/dL improvement from baseline compared with placebo at 12 weeks. In addition, there was a dose-dependent decrease in hemolysis markers. Voxelotor was generally well tolerated at both doses. Hemolytic anemia of SCD has severe and life-threatening consequences and presents an unmet medical need. Voxelotor has potential to ameliorate complications of anemia associated with SCD.DisclosuresVichinsky: bluebird bio: Membership on an entity's B...
Source: Blood - November 21, 2018 Category: Hematology Authors: Vichinsky, E., Hoppe, C., Howard, J., Ataga, K. I., Nduba, V., El-Beshlawy, A., Diuguid, D. L., Al-Kindi, S., Brown, C., Hassab, H., Telfer, P., Tsitsikas, D. A., Unal, S., Kanter, J., Abboud, M. R., Gordeuk, V. R., Lehrer-Graiwer, J., Sherman, C., Tonda, Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Novel or Improved Approaches To Treating Sickle Cell Disease Source Type: research
Clinical Relevance of Clonal Hematopoiesis in the Oldest-Old Population: Analysis of the "Health and Anemia" Study
Conclusion. Clonal hematopoiesis was associated with reduced survival in an oldest-old population. Specific mutational profiles define different risks of developing MDS and inflammatory/vascular diseases. Non mutational factors, such as early changes in red blood cell indices, may improve the capability to identify patients at increased risk of developing myeloid cancers.DisclosuresMeggendorfer: MLL Munich Leukemia Laboratory: Employment. Bolli: Celgene: Honoraria. Vassiliou: KYMAB: Consultancy, Equity Ownership; Celgene: Research Funding. Kern: MLL Munich Leukemia Laboratory: Employment, Equity Ownership. Haferlach: MLL M...
Source: Blood - November 21, 2018 Category: Hematology Authors: Rossi, M., Meggendorfer, M., Zampini, M., Tettamanti, M., Riva, E., Saba, E., Manes, N., Milanesi, C., Marta, U., Morabito, L., Travaglino, E., Peano, C., Giulia, S., Asselta, R., Duga, S., Malik, K., Selmi, C., Civilini, E., Mandelli, S., Bolli, N., Vass Tags: 503. Clonal Hematopoiesis: Aging and Inflammation: Cause and consequence of clonal hematopoiesis Source Type: research
Asciminib, a Specific Allosteric BCR-ABL1 Inhibitor, in Patients with Chronic Myeloid Leukemia Carrying the T315I Mutation in a Phase 1 Trial
We report results from the largest cohort: pts with confirmed T315I mut at screening (tested locally by Sanger sequencing) and treated with asciminib 200 mg BID, which showed the most robust efficacy (data cutoff: April 30, 2018).At the data cutoff, treatment was ongoing in 23/24 pts (95.8%) (Table); 1 pt (4.2%) had ended treatment. Median duration of follow-up and asciminib exposure were both 28.5 wk (range, 0.1-74.7 wk). Most pts had received multiple prior TKIs, and PON was the most recent TKI for 12/24 (50.0%). Pts who were PON naive had underlying conditions, such as CV risk factors. Eight of 24 pts achieved MMR by 24...
Source: Blood - November 21, 2018 Category: Hematology Authors: Rea, D., Lang, F., Kim, D.-W., Cortes, J. E., Hughes, T. P., Minami, H., Breccia, M., Deangelo, D. J., Hochhaus, A., Talpaz, M., Goh, Y. T., le Coutre, P., Deininger, M. W., Etienne, G., Sondhi, M., Mishra, K., Aimone, P., Ng-Sikorski, J., Mauro, M. J. Tags: 632. Chronic Myeloid Leukemia: Therapy: TFR Failure, Resistance, and New Drug Development Source Type: research
Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
Backgroundβ-globin gene transfer has the potential for substantial clinical benefit in patients with sickle cell disease (SCD). LentiGlobin Drug Product (DP) contains autologous CD34+ hematopoietic stem cells (HSCs) transduced with the BB305 lentiviral vector (LVV), encoding β-globin with an anti-sickling substitution (T87Q). The safety and efficacy of LentiGlobin gene therapy is being evaluated in the ongoing Phase 1 HGB-206 study (NCT02140554). Results in the initial 7 patients treated with LentiGlobin DP from steady state bone marrow harvested (BMH) HSCs using original DP manufacturing process (Group A) demons...
Source: Blood - November 21, 2018 Category: Hematology Authors: Tisdale, J. F., Kanter, J., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Shi, W., Ribeil, J.-A., Asmal, M., Thompson, A. A., Walters, M. C. Tags: 801. Gene Therapy and Transfer: Gene Therapy for Blood Cell Disorders Source Type: research
Risk Factors and Manageability of the Mainly Mild Mucocutaneous Bleeding Profile Observed in Attp Patients Treated with Caplacizumab during the Phase III Hercules Study
Conclusions: The safety profile of caplacizumab was favorable. In line with its pharmacology, treatment with caplacizumab was associated with an increased risk of mucocutaneous bleeding. These events were generally mild to moderate, and the majority did not require therapeutic intervention. While the number of patients receiving DOACs was low, no increased risk for bleeding with antithrombotic therapy was observed.DisclosuresCataland: Alexion: Research Funding; Shire: Consultancy; Ablynx: Consultancy, Other: Member of Advisory Board. Scully: Novartis: Honoraria, Other: Member of Advisory Board, Speakers Bureau. Peyvandi: N...
Source: Blood - November 21, 2018 Category: Hematology Authors: Cataland, S. R., Scully, M., Peyvandi, F., Coppo, P., Knobl, P., Kremer Hovinga, J. A., Metjian, A., De La Rubia, J., Pavenski, K., Minkue, J., Sousa, R. P., Callewaert, F., De Winter, H. Tags: 311. Disorders of Platelet Number or Function: Poster I Source Type: research
Phase 2/3 Trial of Subcutaneously Administered Marzeptacog Alfa (activated) an Engineered FVIIa in Hemophilia with Inhibitors - Pharmacokinetics, Pharmacodynamics, Safety and Efficacy
Conclusion: This study demonstrates that an individualized dose of daily SQ MarzAA can provide effective prophylaxis in hemophilia patients with inhibitors.Supported by Catalyst BiosciencesDisclosuresLevy: Catalyst Biosciences: Employment, Equity Ownership. Mahlangu: Sanofi: Research Funding, Speakers Bureau; Roche: Consultancy, Research Funding, Speakers Bureau; LFB: Consultancy; NovoNordisk: Consultancy, Research Funding, Speakers Bureau; CSL Behring: Consultancy, Research Funding, Speakers Bureau; Chugai: Consultancy; Catalyst Biosciences: Consultancy, Research Funding; Biomarin: Research Funding, Speakers Bureau; Bioge...
Source: Blood - November 21, 2018 Category: Hematology Authors: Levy, H., Makhaldiani, L., Iosava, G., Mahlangu, J., Kosinova, M. V., Khacchatryan, H., Del Greco, F., Booth, F. V. M. Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster I Source Type: research
