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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

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

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