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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 and Laboratory Benefits of Early Initiation of Hydroxyurea with Pharmacokinetic Guided Dosing for Young Children with Sickle Cell Anemia
Conclusions: Hydroxyurea initiation at an early age using PK-guided dosing provides significant clinical benefits for young children with sickle cell anemia. These TREAT study data suggest that initiating hydroxyurea around one year of life using a personalized dosing strategy can provide better clinical and laboratory benefits than starting at the conventional 20 mg/kg/day weight-based dose. Very high HbF levels are observed at modest and well-tolerated doses of hydroxyurea, perhaps because treatment was initiated before the process of HbF inactivation is complete. Continued long-term follow-up of these patients will dete...
Source: Blood - November 21, 2018 Category: Hematology Authors: McGann, P. T., Niss, O., Dong, M., Marahatta, A., Mizuno, T., Kalinyak, K., Kalfa, T. A., Malik, P., Quinn, C. T., Ware, R. E., Vinks, A. A. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Novel or Improved Approaches To Treating Sickle Cell Disease Source Type: research
Parenteral L-Arginine Improves Mitochondrial Function in Children with Sickle Cell Disease Admitted for Vaso-Occlusive Pain Episodes
Conclusion: These data demonstrate for the first time that Arg supplementation increases mitochondrial activity and decreases oxidative stress in children with SCD and VOE. Our prior study showed that complex V inhibition leads to increased mitochondrial oxidant production in platelets from SCD patients. The improvement in complex V function & decreased oxidative markers observed with Arg treatment are consistent with Arg-induced decrease in mitochondrial oxidant generation. However, Arg treatment potentially regulates other oxidant sources and could decrease overall oxidative stress in SCD patients. Arg therapy has be...
Source: Blood - November 21, 2018 Category: Hematology Authors: Shiva, S., Brown, L. A., Wang, Y., Dampier, C., Watt, A., Tripathi, V., Bhutta, A., Kumari, P., Figueroa, J., Zmitrovich, A., Morris, C. R. 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
Medical Conditions Among Survivors of Adolescent and Young Adult Non-Hodgkin Lymphoma (NHL), Acute Lymphoblastic Leukemia (ALL) and Acute Myeloid Leukemia (AML)
Conclusion: This study found that sociodemographic factors were associated with the risk of developing medical conditions in AYA NHL, ALL and AML survivors. As expected, the risk of medical conditions varied by cancer type and treatment, with those undergoing SCT having a higher risk of medical conditions regardless of cancer type. NHL and ALL survivors who were uninsured or publicly insured were at a consistently higher risk of developing medical conditions, as were Hispanic ALL survivors and Black AML survivors. Our findings highlight the higher burden of medical conditions in subgroups of cancer survivors that may relat...
Source: Blood - November 21, 2018 Category: Hematology Authors: Keegan, T. H. M., Muffly, L. S., Li, Q., Alvarez, E., Brunson, A. M., Malogolowkin, M., Wun, T. Tags: 904. Outcomes Research-Malignant Conditions: Real World Outcomes 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
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
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
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
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
