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Total 139 results found since Jan 2013.
Curativestategy (GEM-CESAR) for High-Risk Smoldering Myeloma (SMM): Carfilzomib, Lenalidomide and Dexamethasone (KRd) As Induction Followed By HDT-ASCT, Consolidation with Krd and Maintenance with Rd
Conclusions: Although longer follow-up is required, this "curative strategy for high risk SMM" continues being encouraging with an acceptable toxicity profile. The study has met its primary endpoint. The depth of response improved over the treatment: 63% of patients who completed induction and ASCT achieved ≥CR with a MRD-ve rate of 55%.DisclosuresMateos: Takeda: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Mateos, M.-V., Martinez-Lopez, J., Rodriguez Otero, P., Ocio, E. M., Gonzalez, M. S., Oriol, A., Gutierrez, N. C., Paiva, B., Rios, R., Rosinol, L., Alvarez, M. A., Calasanz, M. J., Bargay, J., Gonzalez, A. P., Alegre, A., Escalante, F., Martinez, R., Pui Tags: 731. Clinical Autologous Transplantation: Results: Poster I Source Type: research
Acute Neurotoxicity during ALL Therapy Is Associated with Treatment Intensity, Age and Female Sex - an Analysis of SAE Reports from the UKALL 2003 Trial
Discussion:This large study identifies treatment intensity as the main risk factor for developing acute neurotoxicity with female sex, age and CNS status having a significant modifying effect. CNS status may reflect increased intrathecal therapy given to non-CNS-1 patients. Females are more vulnerable to cranial radiotherapy induced neurotoxicity but this is the first report of female sex as a risk factor on contemporary chemotherapy treatment protocols. Reassuringly, the occurrence of acute neurotoxicity did not influence survival rates. These data provide an important benchmark for ongoing international deep phenotyping ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Wahid, Q.-u.-A., Hamadeh, L., McGowan, S., Hough, R., Vora, A., Moorman, A. V., Halsey, C. Tags: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Poster I Source Type: research
Variable Clinical Presentation of Congenital Thrombotic Thrombocytopenic Purpura in a Large Cohort of Patients Carrying a New Mutation in the CUB1 Domain of ADAMTS13 Gene
Conclusion:To the best of our knowledge, this is one of the largest congenital TTP cohorts described in the literature. This cohort is unique due to the fact that all members carry the same, previously unreported, ADAMTS13 gene mutation in the CUB1 domain of the gene. Our findings support an assumption that environmental and hereditary modifiers may influence disease course. Further research of the involved families may enable us to expand the understanding of the pathophysiology and develop better treatment options for this understudied rare condition.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Pikovsky, O., Arafat, M., Ovadia, H., Sharoni, Y., Al-Athamen, K., Kanengisser-Pines, B., Keren-Politansky, A., Levi, I., Parvari, R., Rabinovich, A. Tags: 311. Disorders of Platelet Number or Function: Poster I 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
Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Conclusions: Collectively, these data provide strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease. We anticipate that a single dose IV procedure will pose minimal burden to Fabry patients and will be a viable alternative to biweekly enzyme infusions, potentially reducing treatment-related morbidity whislt improving patient quality of life and potentially providing them with a functional long-term cure.DisclosuresKia: Freeline: Employment, Equity Ownership. McIntosh: Freeline: Consultancy. Hosseini: Freeline: Employment, Equit...
Source: Blood - November 21, 2018 Category: Hematology Authors: Kia, A., McIntosh, J., Rosales, C., Hosseini, P., Sheridan, R., Spiewak, J., Mills, K., Corbau, R., Nathwani, A. C. Tags: 801. Gene Therapy and Transfer: 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
ABL Tyrosine Kinase Inhibitors (TKIs) Are Associated with Increased Rho-Associated Kinase (ROCK) Activity That May Contribute to Vascular Toxicity in Patients with Chronic Myeloid Leukemia (CML)
ConclusionsWe found that patients on 2nd and 3rd generation TKIs have higher leukocyte ROCK activity compared to those not receiving TKIs, and higher leukocyte ROCK activity in patients on Dasatinib compared with patients receiving Imatinib. These results are consistent with the known lower-risk of cardiovascular side-effects observed with Imatinib in comparison to the next generation ABL TKIs. Limitations include small sample size and heterogeneity in the patient population in terms of age, cardiovascular risk factors, specific TKI used, and total duration and sequencing of TKI agents. The study continues to accrue CML su...
Source: Blood - November 21, 2018 Category: Hematology Authors: Osman, A., Yu, B., Glavin, N., Polonsky, T. S., Liao, J. K., Larson, R. A. Tags: 632. Chronic Myeloid Leukemia: Therapy: Poster I Source Type: research
Prospective Study of Apixaban for Primary Prevention of Venous Thromboembolism in Patients with Multiple Myeloma Receiving Immunomodulatory Therapy
ConclusionsIn this pilot study of 50 patients, low-dose apixaban was safe and well tolerated as thromboprophylaxis for patients with MM receiving IMiDs. No patients experienced VTE, major hemorrhage, stroke, or MI. Further randomized studies are needed to validate apixaban as a standard primary prevention anti-thrombotic strategy for patients with MM receiving IMiDs.DisclosuresMoslehi: Bristol-Myers Squibb: Consultancy, Research Funding. Jagasia: Incyte Corporation: Membership on an entity's Board of Directors or advisory committees.
Source: Blood - November 21, 2018 Category: Hematology Authors: Cornell, R. F., Goldhaber, S. Z., Engelhardt, B. G., Moslehi, J., Jagasia, M., Patton, D., Harrell, S. L., Hall, R. L., Wyatt, H., Piazza, G. Tags: 332. Antithrombotic Therapy: Poster I Source Type: research
Replacing PT-INR Monitoring of Warfarin with Fiix-NR in Clinical Practice Reduces Thromboembolism without Increasing Bleeding Despite Reduced Number of Dose Adjustments
Conclusions: These results are in agreement with the results of the Fiix-trial and show that ignoring factor VII during VKA monitoring is safe and leads to reduction in thromboembolism without increasing bleeding. Although TTR was identical in both groups, the dose adjustment need was reduced possibly indicating that less anticoagulation variability in the Fiix-NR group explains reduced thromboembolism.FigureDisclosuresGudmundsdottir: Hart Biologicals Ltd: Consultancy, Patents & Royalties: Hart Biologicals Ltd is commercializing the Fiix-PT which will be ready for marketing in Europe in the beginning of year 2019 and p...
Source: Blood - November 21, 2018 Category: Hematology Authors: Oskarsdottir, A. R., Gudmundsdottir, B. R., Onundarson, P. T. Tags: 332. Antithrombotic Therapy: Poster I Source Type: research
Clinical Outcome Following Reinstitution of Anticoagulation after Major Gastrointestinal Bleed: A Single Institutional Analysis
Conclusion: Given the rising national trend on the use of anticoagulants for various medical necessities, it is imperative that a safe and efficient process be devised on reinstitution of anticoagulation post MGIB to guide Clinicians. Although our study represents a single institutional analysis, it concurs with recent studies that early resumption of anticoagulant following stabilization of MGIB is associated with lower thromboembolic events. Timing for resumption depends largely on the medical reason for anticoagulation; reinstitution by day 7 appear safe for patients on mechanical valve whereas after day 12 maybe approp...
Source: Blood - November 21, 2018 Category: Hematology Authors: Ezekwudo, D. E., Gaikazian, S., Anusim, N., Konde, A. S., Zakalik, D., Huben, M. T., Stender, M., Anderson, J., Jaiyesimi, I. Tags: 332. Antithrombotic Therapy: Poster I Source Type: research
Efficacy and Safety of Pomalidomide in Combination with Prednisone in Patients with Myelofibrosis and Anemia -- Final Results of a Prospective Phase 2 Study
CONCLUSION:Pomalidomide with prednisone is safe therapy with good anti-anemia activity in patients with MF. It could lead to transfusion independence in one third of patients for a median duration of about 30 months. ClinicalTrials.gov Identifier: NCT00946270.Table 1.DisclosuresDaver: Alexion: Consultancy; ImmunoGen: Consultancy; Pfizer: Research Funding; Karyopharm: Research Funding; Otsuka: Consultancy; Novartis: Consultancy; ARIAD: Research Funding; Incyte: Consultancy; Pfizer: Consultancy; BMS: Research Funding; Sunesis: Research Funding; Daiichi-Sankyo: Research Funding; Sunesis: Consultancy; Kiromic: Research Funding...
Source: Blood - November 21, 2018 Category: Hematology Authors: Masarova, L., Daver, N. G., Kadia, T. M., Pemmaraju, N., Jabbour, E. J., Zhou, L., Pierce, S. A., Cortes, J. E., Kantarjian, H. M., Verstovsek, S. Tags: 634. Myeloproliferative Syndromes: Clinical: Poster I Source Type: research
Validation of Sickle Cell Disease Severity Score in a Cohort of Hemoglobin SC Disease Patients
Conclusion: Despite having been derived from a SCD population that was 26% HbSC, the study was unable to validate the SSS within the cohort of HbSC patients. This may reflect the differences in patient population and/or therapeutic intervention between this cohort and the CSSCD cohort used in the construction of the SSS calculator. While SSS was found to correlate with 3 discrete markers of disease morbidity (TRJV, CVA, creatinine clearance), it appears that a new scoring system is required to accurately predict clinical mortality and morbidity in contemporary cohorts of adult HbSC disease patients.DisclosuresNo relevant c...
Source: Blood - November 21, 2018 Category: Hematology Authors: Rosart, R. M., Pestrin, O. A., Tomlinson, G. A., Ward, R., Pendergrast, J., Binding, A., Kuo, K. H. M. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster I Source Type: research
Myeloablative Conditioning with Alemtuzumab in Matched Related Donor Hematopoietic Cell Transplant for Sickle Cell Disease Prevents Graft-Versus-Host Disease without Compromising Engraftment
Conclusions:Myeloablative conditioning was well tolerated in this patient population, and the addition of alemtuzumab minimized occurrence of severe GVHD. While MDC was observed, chimerism stabilized at >50% donor cells in most patients and no graft rejection or recurrence of SCD occurred with a median follow-up of 2.9 yrs. The use of this regimen may be a promising approach to achieve low rates of GVHD while maintaining low rates of transplant related complications for patients with SCD that can tolerate myeloablative chemotherapy.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: John, T. D., Yassine, K., Naik, S., Sasa, G., Omer, B., Martinez, C. A., Tewari, P., Krance, R. A., Leung, K. S. Tags: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster I 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
Safety and Efficacy of Venetoclax (VEN) in Combination with Bendamustine (B) Plus Rituximab (R) or Obinutuzumab (G) in Patients (pts) with Previously Untreated Chronic Lymphocytic Leukemia (CLL): Results from a Phase Ib Study (GO28440)
ConclusionVEN 400mg daily combined with BR or BG in 1L CLL treatment was associated with toxicity leading to treatment interruptions and discontinuations; the most frequently reported toxicities were hematologic. However, despite dose modifications, all pts responded to treatment, independently of subgroups. 50% of pts achieved a CR/CRi; higher than previously reported with BG or BR alone (Brown et al. Blood 2015; Michallet et al. Haematologica 2018). Responses were durable and preliminary PFS data are encouraging. Response rates were similar, irrespective of whether pts received planned 6 or fewer cycles of B; the optimal...
Source: Blood - November 21, 2018 Category: Hematology Authors: Stilgenbauer, S., Morschhauser, F., Wendtner, C.-M., Cartron, G., Hallek, M., Eichhorst, B., Kozloff, M. F., Giever, T., Lozanski, G., Jiang, Y., Huang, H., Pignataro, D. S., Schary, W., Humphrey, K., Mobasher, M., Salles, G. Tags: 642. CLL: Therapy, excluding Transplantation: Poster I Source Type: research
