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Procedure: Transplants

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Total 25 results found since Jan 2013.

Description of the First Cases with ADAMTS13 Mutations in Hungary
CONCLUSIONS:Clinical and molecular characterization of the first 5 Hungarian cases with ADAMTS13 mutations was presented. Compound heterozygous, damaging ADAMTS13 mutations with deficient plasma ADAMTS13 activity were associated with serious clinical consequences. In case of thrombotic microangiopathy in young patients with stroke, or HELLP syndrome, complete workup for ADAMTS13 activity and inhibitors is necessary, and genetic analysis should be considered. Three new ADAMTS13 mutations (c.1016_1017delCA, c.3199T>A , c.2839C>T) were identified and shown to be associated with decreased plasma ADAMTS13 activity.Disclos...
Source: Blood - November 21, 2018 Category: Hematology Authors: Reti, M., Sinkovits, G., Cseprekal, O., Csuka, D., Szilagyi, A., Farkas, Z., Klucsik, Z., Szederjesi, A., Wagner, L., Reusz, G., Kremer Hovinga, J. A., Rigo, J., Masszi, T., Prohaszka, Z. Tags: 311. Disorders of Platelet Number or Function Source Type: research

Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy
This study was supported by the grant of Russian Science Foundation No 16-15-00010. Kazan Federal University was supported by the Russian Government Program of Competitive Growth.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Bashirov, F. V., Salafutdinov, I. I., Sokolov, M. E., Izmailov, A. A., Markosyan, V. A., Fadeev, F. O., Rizvanov, A., Islamov, R. I. Tags: 801. Gene Therapy and Transfer 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

Outcomes of Hospitalization for Stem Cell Transplant in Sickle Cell Disease: Are We There Yet?
Conclusions: The rate of inpatient mortality with SCT in sickle cell disease is lower than the overall inpatient mortality rate for allogeneic SCT (7%; Godara et al bbmt 2018), indicating a favorable outcome for these patients. Infections do occur commonly during the course of hospitalization, especially in association with GVHD. Length of stay is adversely impacted by occurrence of GVHD, bacterial sepsis, C.difficile infection and viral infections. While we are limited by duration of follow up in our study, these patterns suggest some essential modifiers for inpatient morbidity and mortality, therefore require validation ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Godara, A., Siddiqui, N., Afzal, A., Khan, M., Yared, J., Kansagra, A., Dahiya, S. Tags: 732. Clinical Allogeneic Transplantation: Results: Poster III Source Type: research

A Polymorphism in Toll-like Receptor 2 Gene Is Associated with Occurrence of Bacterial Infections in Sickle Cell Disease Patients
Discussion: In SCD pts, TLR-2 rs4696480 TA genotype might be protective against bacterial infections, whereas TT genotype might increase risk of such infections. Previous reports demonstrated higher secretion of inflammatory factors in cells from AA individuals, lower occurrence and severity of immune diseases in T carriers. TA genotype might stand between deleterious effects of over inflammatory response (AA genotype) and under response (TT genotype) to infectious agents.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Tozatto-Maio, K., Girot, R., Ly, I. D., Rocha, V., Pinto, A. C. S., Diagne, I., Benzerara, Y., Dinardo, C. L., Kashima, S., Araujo, I. L., Kenzey, C., Fonseca, G. H. H., Rodrigues, E., Volt, F., Jarduli, L. R., Ruggeri, A., Mariaselvam, C. M., Gualandro, Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science: Poster III Source Type: research

Hydroxyurea Therapy Does Not Impact Current Survival Estimates in the East London Sickle Cell Newborn Cohort Study
Conclusions: The study is the first to evaluate survival in a newborn cohort past the age of 20 years. We have confirmed a low mortality rate in childhood, but increased number of deaths in young adults including those with HbSC. This study provides evidence of the benefit of newborn screening and comprehensive care which is accessible at all ages, free of charge. The uptake of HU, particularly at a young age, has been low and a positive effect of HU on survival as described in other studies has not yet been observed in this cohort. Earlier initiation of disease-modifying treatment and longer-term follow-up will be require...
Source: Blood - November 21, 2018 Category: Hematology Authors: Telfer, P., Kaya, B., Tsitsikas, D. A., Barroso, F., Sangarappillai, C. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster III Source Type: research

Long-term treatment follow-up of children with sickle cell disease monitored with abnormal transcranial Doppler velocities
We present the long-term follow-up of SCA children from the Créteil newborn cohort (1992-2012) detected at risk by TCD and placed on chronic transfusions. Patients with normalized velocities and no stenosis were treated with hydroxyurea, known to decrease anemia and hemolytic rate. Trimestrial Doppler was performed and transfusions restarted immediately in the case of reversion to abnormal velocities. Patients with a genoidentical donor underwent transplant. Abnormal time-averaged maximum mean velocities (TAMMV) ≥200 cm/s were detected in 92 SCA children at a mean age of 3.7 years (range, 1.3-8.3 years). No strok...
Source: Blood - April 6, 2016 Category: Hematology Authors: Bernaudin, F., Verlhac, S., Arnaud, C., Kamdem, A., Hau, I., Leveille, E., Vasile, M., Kasbi, F., Madhi, F., Fourmaux, C., Biscardi, S., Gluckman, E., Socie, G., Dalle, J.-H., Epaud, R., Pondarre, C. Tags: Pediatric Hematology, Sickle Cell Disease, Free Research Articles, Red Cells, Iron, and Erythropoiesis, Clinical Trials and Observations Source Type: research

Platelet-derived VWF is not essential for normal thrombosis and hemostasis but fosters ischemic stroke injury in mice
Von Willebrand factor (VWF) is a key hemostatic protein synthesized in both endothelial cells and megakaryocytes. Megakaryocyte-derived VWF is stored in α-granules of platelets and is enriched in hyperactive "ultra-large" VWF multimers. To elucidate the specific contribution of platelet VWF in hemostasis and thrombosis, we performed crossed bone marrow transplantations between C57BL/6J and Vwf–/– mice to generate chimeric mice. Chimeric mice specifically lacking platelet VWF showed normal tail bleeding and carotid artery thrombosis, similar to wild-type mice. Chimeric mice with VWF present only in platele...
Source: Blood - October 1, 2015 Category: Hematology Authors: Verhenne, S., Denorme, F., Libbrecht, S., Vandenbulcke, A., Pareyn, I., Deckmyn, H., Lambrecht, A., Nieswandt, B., Kleinschnitz, C., Vanhoorelbeke, K., De Meyer, S. F. Tags: Platelets and Thrombopoiesis, Thrombosis and Hemostasis 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

Lower risk for serious adverse events and no increased risk for cancer after PBSC vs BM donation
In conclusion, SAEs after donation are rare but more often occurred in BM donors and women. In addition, there was no evidence of increased risk for cancer, autoimmune illness, and stroke in donors receiving granulocyte colony-stimulating factor during this period of observation.
Source: Blood - June 5, 2014 Category: Hematology Authors: Pulsipher, M. A., Chitphakdithai, P., Logan, B. R., Navarro, W. H., Levine, J. E., Miller, J. P., Shaw, B. E., O'Donnell, P. V., Majhail, N. S., Confer, D. L. Tags: Transfusion Medicine, Transplantation Source Type: research