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Emicizumab for the Treatment of Acquired Hemophilia_A: Lessons Learned from 4 Very Different Cases
In conclusion, emiczumab seems to be an effective hemostatic therapy also for AHA, which offers several advantages: subcutaneous weekly therapy, good hemostatic efficacy, possible outpatient therapy, the option to reduce the intensity of immunosuppressive therapy to avoid side effects (as the patients are protected from bleeding), and seems even to be more cost effective than bypassing agents. However, special attention is necessary on the use of appropriate lab assays (chromogenic FVIII assays), the artificial effects on APTT and Bethesda assays, the recognition of the remission, and to avoid the concomitant use of APCC. ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Knoebl, P., Sperr, W. R., Schellongowski, P., Staudinger, T., Jilma-Stohlawetz, P., Quehenberger, P., Koder, S., Ay, C., Gleixner, K. V. Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster II Source Type: research

Indications, Efficacy and Complications of Kcentra Use in Reversing Coagulopathy
ConclusionsKcentra was used in several off-label clinical settings, with comparable mortality among the coumadin, rivaroxaban and apixaban groups and no identifiable benefit in the setting of cirrhosis, DIC or antiplatelet medications, but with an increased incidence of deep vein thrombosis and stroke.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Sritharan, N., Triulzi, D. Tags: 401. Basic Science and Clinical Practice in Blood Transfusion: Poster III Source Type: research

Restrictive Versus Liberal Red Blood Cell Transfusion for Cardiac Surgery: A Systematic Review and Meta-Analysis of Randomized Controlled Trials
Conclusion:This meta-analysis showed that restrictive strategies for RBC transfusion are as safe as liberal strategies in patients undergoing cardiac surgery.Key points:Restrictive strategies for red blood cell transfusion are as safe as liberal approaches in patients undergoing cardiac surgery.Longer duration of stay in the intensive care unit is more common in patients managed with a restrictive transfusion approach. However, the overall hospital length of stay appeared to be similar between both groups.Further studies are needed to ascertain threshold triggers for RBC transfusion.Figure.DisclosuresHassan: abott: Other: ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Kheiri, B., Abdalla, A., Osman, M., Haykal, T., Chintalapati, S., Cranford, J., Ahmed, S., Hassan, M., Bachuwa, G., Bhatt, D. L. Tags: 401. Basic Science and Clinical Practice in Blood Transfusion: Poster III Source Type: research

Defining Sickle Cell Disease Severity Among Adults Hospitalized with Vaso-Occlusive Crisis
Conclusions: The high severity scores among this cohort supports the validity of employing a medical history checklist as a measure of disease severity, reflecting the cumulative end organ damage due to SCD and near universal need for daily pain medication among patients requiring inpatient admission for VOC. Two-thirds of patients were categorized as having high disease severity, driven primarily by daily pain medication use prior to hospitalization and pulmonary complications, consistent with a known subgroup of adults with SCD-related complications who are more likely to have lower health-related quality of life and acc...
Source: Blood - November 21, 2018 Category: Hematology Authors: Esham, K. S., Rodday, A. M., Savidge, N., Mao, D., Weidner, R. A., Parsons, S. K. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster II Source Type: research

Measuring Health-Related Quality of Life in Sickle Cell Disease Patients Undergoing Automated Red Blood Cell Exchange in the USA, France and the UK
ConclusionSickle cell disease patients that require chronic blood transfusion experience better health-related quality of life when they are treated with automated red blood cell exchange versus simple transfusion. This observation is supported by the opinion of their treating physicians.DisclosuresDierick: Terumo BCT: Employment. Roig: Terumo BCT: Employment.
Source: Blood - November 21, 2018 Category: Hematology Authors: Dierick, K., Roig, J. Tags: 903. Outcomes Research-Non-Malignant Hematology 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

Predictors of Adverse Outcome in Sickle Cell Disease Patients from Oman
Conclusion: Multi-organ failure was mostly associated with the terminal events relating to mortality. Although sepsis played a major role as a cause of death in association with multiorgan failure, low hemoglobin, and low platelet counts with increased WBC counts along with elevated LDH and CRP played a significant role in the terminal event in this cohort of SCD patients.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Alkindi, S., AlJadidi, S., Al Adawi, S., Pathare, A. Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science Source Type: research

The Role of Hydroxyurea to Prevent Silent Stroke in Sickle Cell Disease: Systematic Review and Meta-Analysis
Chronic blood transfusions are standard of care for stroke prevention in sickle cell disease. We evaluated hydroxyurea's efficacy in preventing silent stroke. We searched for randomized controlled trials (RCTs) and observational studies on Pubmed, CENTRAL, Embase, and Web of Science without using language/time limits. Eligible studies compared hydroxyurea with transfusions or observation to prevent silent stroke. This systematic review adheres to the Cochrane guidelines. Data were pooled using random effects model using STATA to perform meta-analysis. Methodological quality of RCTs was investigated using the Cochrane risk ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Hasson, C., Veling, L., Rico, J. F., Mhaskar, R. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research

Transfusion Therapy in a Multi-Ethnic Sickle Cell Population Real-World Practice. a Preliminary Data Analysis of Multicentre Survey
Conclusion. The transfusional approach is similar in HbSS, b°-thal/HbS and b+-thal/HbS patients with similar indications, prevalently VOCs and anemia. The significant higher age in Caucasian cohort and the consequent long term follow up could be the cause of variable therapeutic approach observed, however Hydroxycarbamide seemed to be the therapy more frequently used and finally suggested to manage chronic manifestations.Figure.DisclosuresOriga: Apopharma: Honoraria; Novartis: Honoraria; Bluebird Bio: Consultancy; Cerus Corporation: Research Funding. Forni: Apopharma: Other: DSM Board; Celgene: Research Funding; Novart...
Source: Blood - November 21, 2018 Category: Hematology Authors: Graziadei, G., Sainati, L., Bonomo, P., Venturelli, D., Masera, N., Casale, M., Vassanelli, A., Lodi, G., Piel, F. B., Voi, V., De Franceschi, L., Rigano, P., Quota, A., Notarangelo, L. D., Russo, G., Rosso, R., Allo, M., D'Ascola, D., Facchini, E., Macch Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research

Red cell exchange transfusions lower cerebral blood flow and oxygen extraction fraction in pediatric sickle cell anemia
Blood transfusions are the mainstay of stroke prevention in pediatric sickle cell anemia (SCA), but the physiology conferring this benefit is unclear. Cerebral blood flow (CBF) and oxygen extraction fraction (OEF) are elevated in SCA, likely compensating for reduced arterial oxygen content (CaO2). We hypothesized that exchange transfusions would decrease CBF and OEF by increasing CaO2, thereby relieving cerebral oxygen metabolic stress. Twenty-one children with SCA receiving chronic transfusion therapy (CTT) underwent magnetic resonance imaging before and after exchange transfusions. Arterial spin labeling and asymmetric s...
Source: Blood - March 1, 2018 Category: Hematology Authors: Guilliams, K. P., Fields, M. E., Ragan, D. K., Eldeniz, C., Binkley, M. M., Chen, Y., Comiskey, L. S., Doctor, A., Hulbert, M. L., Shimony, J. S., Vo, K. D., McKinstry, R. C., An, H., Lee, J.-M., Ford, A. L. Tags: Pediatric Hematology, Sickle Cell Disease, Transfusion Medicine, Free Research Articles, Red Cells, Iron, and Erythropoiesis Source Type: research

Central nervous system complications and management in sickle cell disease
With advances in brain imaging and completion of randomized clinical trials (RCTs) for primary and secondary stroke prevention, the natural history of central nervous system (CNS) complications in sickle cell disease (SCD) is evolving. In order of current prevalence, the primary CNS complications include silent cerebral infarcts (39% by 18 years), headache (both acute and chronic: 36% in children with sickle cell anemia [SCA]), ischemic stroke (as low as 1% in children with SCA with effective screening and prophylaxis, but ~11% in children with SCA without screening), and hemorrhagic stroke in children and adults with SCA ...
Source: Blood - February 18, 2016 Category: Hematology Authors: DeBaun, M. R., Kirkham, F. J. Tags: Sickle Cell Disease, Free Research Articles, Red Cells, Iron, and Erythropoiesis, Review Articles, Review Series, Clinical Trials and Observations Source Type: research

How I treat and manage strokes in sickle cell disease
Neurologic complications are a major cause of morbidity and mortality in sickle cell disease (SCD). In children with sickle cell anemia, routine use of transcranial Doppler screening, coupled with regular blood transfusion therapy, has decreased the prevalence of overt stroke from ~11% to 1%. Limited evidence is available to guide acute and chronic management of individuals with SCD and strokes. Current management strategies are based primarily on single arm clinical trials and observational studies, coupled with principles of neurology and hematology. Initial management of a focal neurologic deficit includes evaluation by...
Source: Blood - May 28, 2015 Category: Hematology Authors: Kassim, A. A., Galadanci, N. A., Pruthi, S., DeBaun, M. R. Tags: Sickle Cell Disease, How I Treat, Free Research Articles, Red Cells, Iron, and Erythropoiesis, Clinical Trials and Observations Source Type: research