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Total 365 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
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
Bleeding Related Episodes, Thrombotic Events and Platelet Counts Among Immune Thrombocytopenia Patients Receiving Second Line Therapy
CONCLUSIONS: This retrospective real world evidence study compares mean platelet response and burden of both BREs and TEs in ITP patients treated with different second-line treatments. Although there were significant differences in mean platelet counts achieved with the second-line treatments, TEs were observed with similar incidence across all treatments. Patients who received splenectomy had the highest mean platelet counts and also the highest proportion of patients who experienced TEs, though differences in TEs did not reach statistical significance. Incidence proportion of patients with BREs was lower in patients trea...
Source: Blood - November 21, 2018 Category: Hematology Authors: Said, Q., Lal, L. S., Andrade, K., Nezami, B., Graves, J. A., Roy, A., Cuker, A. Tags: 311. Disorders of Platelet Number or Function: Poster II 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
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
Final Results from a Phase I Trial Combining Selinexor with High-Dose Cytarabine (HiDAC) and Mitoxantrone (Mito) for Remission Induction in Acute Myeloid Leukemia (AML)
Conclusions: The selinexor/HiDAC/Mito regimen is feasible and tolerable at selinexor doses up to 80mg/day or ~50 mg/m2/day twice weekly. This regimen yields an ORR of 64% based on currently available data. We had previously reported molecular correlatives demonstrating the effect of selinexor. The recommended phase 2 dose is 80mg of selinexor.Figure.DisclosuresLarson: Ariad/Takeda: Consultancy, Research Funding; BristolMyers Squibb: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding. Odenike: Agios: Research Funding; Astex: Research Funding; Dava Oncology: Consulta...
Source: Blood - November 21, 2018 Category: Hematology Authors: Wang, A., Weiner, H., Larson, R. A., Odenike, O., Artz, A. S., Bishop, M. R., Godley, L., Thirman, M., Kosuri, S., Churpek, J., Curran, E. K., Pettit, K., Stock, W., Liu, H. Tags: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III 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
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
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
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
