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Total 198 results found since Jan 2013.
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
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
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
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
Thrombotic and Bleeding Outcomes Following Perioperative Interruption of Direct Oral Anticoagulants and Vitamin K Antagonists in Patients with Non-Valvular Atrial Fibrillation - a Comparative Analysis
Conclusions: The perioperative interruption of warfarin was associated with a higher 30-day rate of major bleeding as compared with DOAC interruption. Re-initiation of warfarin should be done judiciously following high bleeding risk procedures, and close INR monitoring may be warranted.DisclosuresShaw: Portola Pharmaceuticals: Research Funding. Douketis: Janssen: Consultancy; Pfizer: Other: Advisory Board; Boehringer-Ingelheim: Consultancy, Other: Advisory Board, Research Funding; Portola: Other: Advisory Board; The Medicines Company: Other: Advisory Board; Daiichi-Sankyo: Other: Advisory Board; Biotie: Other: Advisory Boa...
Source: Blood - November 21, 2018 Category: Hematology Authors: Shaw, J. R., Zhang, T., Le Gal, G., Douketis, J., Carrier, M. 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
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
B0 Vs. Non-B0 Genotype: Differences in Non-Transfusion-Dependent Thalassemia Patients
Conclusions: Heart remodelling related to a high cardiac output state cardiomyopathy was more pronounced in patients with homozygous β°/β° genotype. Osteoporososis was significantly more frequent in patients with homozygous β°/β° genotype, treated for more than two-thirds with DFO therapy. These data support the knowledge of different phenotypic groups in the management of NTDT patients.TableDisclosuresPepe: Chiesi Farmaceutici S.p.A., ApoPharma Inc., and Bayer: Other: No profit support.
Source: Blood - November 21, 2018 Category: Hematology Authors: Pepe, A., Pistoia, L., Maddaloni, D., Grippo, T., Benni, M., Sardella, L., Sanna, M. G., Giugno, G., Guerrini, G., Maggio, A., Renne, S., Missere, M., Positano, V., Meloni, A. Tags: 112. Thalassemia and Globin Gene Regulation: Poster I Source Type: research
Regulatory Genetic Variation at the S100B Gene Associates with Vaso-Occlusive Manifestations in Sickle Cell Disease
In sickle cell disease (SCD) polymerization of hemoglobin S under deoxygenated conditions causes vaso-occlusion, which can manifest as acute pain crisis and progressive bone/organ damage. Molecular studies have attributed vaso-occlusion to elevated vascular adhesion and inflammatory responses, whereas the genetic regulation has only recently been assessed.Genomic DNA isolated from peripheral blood mononuclear cells (PBMCs) was hybridized to Illumina Human 610-Quad SNP array for the PUSH and Walk-PHaSST cohorts and to Affrymetrix SNP 6.0 array for the Howard SCD expression cohort. Single nucleotide polymorphisms (SNPs) for ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Zhang, X., Zhang, W., Saraf, S. L., Nekhai, S., Gladwin, M. T., Machado, R., Gordeuk, V. R. Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science: Poster I Source Type: research
Rapid Decline in Kidney Function in Sickle Cell Disease
Conclusion:Rapid decline in eGFR is common in SCD. Use of ACE-I/ARB and history of stroke are associated with rapid decline in renal function. Rapid decline in eGFR is associated with an increased risk of death in SCD. Long-term studies are required to determine if therapies that may reduce loss of kidney function may decrease mortality in SCD.DisclosuresAtaga: Pfizer: Research Funding; Bioverativ: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis Pharmaceuticals: Honoraria; Modus Therapeutics: Honoraria; Global Blood Therapeutics: Honoraria, Membership on an entity's Board of Directo...
Source: Blood - November 21, 2018 Category: Hematology Authors: Derebail, V. K., Ciccone, E. J., Zhou, Q., Cai, J., Ataga, K. I. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research
Cerebral Infarcts and Cerebrovascular Disease in Neurologically Intact Tanzanian Children with Sickle Cell Anaemia
Conclusions:The prevalence of SCI on MRI is high in children with SCA without neurological history living in Africa even when TCD CBFV is normal. Children and adolescents with all grades of vasculopathy on MRA are at higher risk of brain parenchymal injury. The lack of association between SCI and hemoglobin may be related to the relatively severe anemia in our African study. As MRA contrast depends on velocity of blood flowing in vessels, it is not surprising that Grade 1 turbulence on MRA appears to reflect high CBFV on TCD. Importantly, high TCD velocity and Grade 1 turbulence may reflect the potentially reversible early...
Source: Blood - November 21, 2018 Category: Hematology Authors: Mwakatika, M. M., Saunders, D. E., Makani, J., Kirkham, F. J. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research
Hemolytic, Vaso-Occlusive and Renal Complications of SCD: Report from the Central Missouri Cohort
ConclusionsThis is the first report describing prevalence of SCD-related complications in the MU-SCD Cohort. We identified this population to have an increasing frequency of hemolytic complications and sickle cell nephropathy with advancing age. Onset of persistent proteinuria occurred in the second decade of life, followed by renal insufficiency or end stage renal disease in subsequent decades. As previously demonstrated in the Cooperative Study of Sickle Cell Disease and the Jamaican SCD Cohort study, renal insufficiency was a significant risk factor for early mortality. Further studies are required for identification of...
Source: Blood - November 21, 2018 Category: Hematology Authors: Nolan, L. W., Yoshida, Y., Coberly, E., Sathi, B. K. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research
Elevated Levels of CD64 MFI on Monocyte Subsets Are Associated with a History of Stroke in Sickle Cell Disease
Conclusion: Levels of monocyte subsets have been shown to correlate with clinical outcomes in non-SCD stroke, but to our knowledge this is the first study to examine their roles in ischemic stroke in SCD. The pathophysiology of stroke in SCD is unique and the role of monocytes in it deserves separate study from the role of monocytes in non-SCD stroke. We saw that while monocyte subsets were associated with a history of ischemic stroke, CD64 MFI on all monocyte subsets showed a strong association. We wonder if this may be due to CD64 MFI being a marker for cells which are more adherent to endothelium as has been shown in pr...
Source: Blood - November 21, 2018 Category: Hematology Authors: Curtis, S. A., Balbuena-Merle, R., Devine, L., Zelterman, D., Roberts, J. D., Dearborn-Tomazos, J., Sansing, L., Hendrickson, J. E. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research
Outcomes in Children with Severe Vasculopathy from Sickle Cell Anemia after Treatment with Chronic Transfusion, Surgical Revascularization and/or Allogenic Hematopoetic Stem Cell Transplantation
Conclusions: The risk for cerebral infarction and/or vasculopathy progression after initiation of treatment with either chronic transfusion, HSCT or EDAS is still a major concern. Our data suggest HSCT and surgical revascularization with chronic transfusion provide the greatest benefit in reducing stroke risk and HSCT reduces risk for progression of a severe vasculopathy. Additional, large population studies are needed to clarify the risk.DisclosuresMajumdar: NIMHD: Research Funding. Campbell: Functional Fluitics: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Johnson, C. W., Majumdar, S., Campbell, A. D., Magge, S., Darbari, D. S., Webb, J., Diab, Y. A., Nickel, R. S., Speller-Brown, B., Carpenter, J., Abraham, A. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research
