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Therapy: Stem Cell Therapy
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Total 18 results found since Jan 2013.
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
Transcranial Doppler Screening Adherence Among Children with Sickle Cell Anemia Seen in the Emergency Department
Conclusion: Patients with SCA who present to the ED and are nonadherent to TCD screening guidelines are less likely to have had a recent hematology clinic visit. Therefore, the ED may be an important location for identifying patients lost to regular clinic follow-up in need of a TCD. An intervention that specifically targets this patient population will likely improve TCD screening rates and stroke prevention.Figure.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Weisman, J. K., Diamond, C., Kappa, S., Nickel, R. S. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster II 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
Efficacy and Safety of Pomalidomide in Combination with Prednisone in Patients with Myelofibrosis and Anemia -- Final Results of a Prospective Phase 2 Study
CONCLUSION:Pomalidomide with prednisone is safe therapy with good anti-anemia activity in patients with MF. It could lead to transfusion independence in one third of patients for a median duration of about 30 months. ClinicalTrials.gov Identifier: NCT00946270.Table 1.DisclosuresDaver: Alexion: Consultancy; ImmunoGen: Consultancy; Pfizer: Research Funding; Karyopharm: Research Funding; Otsuka: Consultancy; Novartis: Consultancy; ARIAD: Research Funding; Incyte: Consultancy; Pfizer: Consultancy; BMS: Research Funding; Sunesis: Research Funding; Daiichi-Sankyo: Research Funding; Sunesis: Consultancy; Kiromic: Research Funding...
Source: Blood - November 21, 2018 Category: Hematology Authors: Masarova, L., Daver, N. G., Kadia, T. M., Pemmaraju, N., Jabbour, E. J., Zhou, L., Pierce, S. A., Cortes, J. E., Kantarjian, H. M., Verstovsek, S. Tags: 634. Myeloproliferative Syndromes: Clinical: Poster I 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
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
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
Platelets and von Willebrand factor in atherogenesis
The role of platelet adhesion, activation, and aggregation in acute atherothrombotic events such as myocardial infarction and stroke is well established. There is increasing evidence that platelet-endothelial interactions also contribute to early atherosclerotic plaque initiation and growth. Through these interactions, platelet-derived factors can contribute to the proinflammatory and mitogenic status of resident mural cells. Among the many putative mechanisms for platelet-endothelial interactions, increased endothelial-associated von Willebrand factor, particularly in a multimerized form, which interacts with platelet gly...
Source: Blood - March 15, 2017 Category: Hematology Authors: Wu, M. D., Atkinson, T. M., Lindner, J. R. Tags: Hematopoiesis and Stem Cells, Thrombosis and Hemostasis, Vascular Biology, Blood Spotlight 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
Myeloablative Conditioning with Alemtuzumab in Matched Related Donor Hematopoietic Cell Transplant for Sickle Cell Disease Prevents Graft-Versus-Host Disease without Compromising Engraftment
Conclusions:Myeloablative conditioning was well tolerated in this patient population, and the addition of alemtuzumab minimized occurrence of severe GVHD. While MDC was observed, chimerism stabilized at >50% donor cells in most patients and no graft rejection or recurrence of SCD occurred with a median follow-up of 2.9 yrs. The use of this regimen may be a promising approach to achieve low rates of GVHD while maintaining low rates of transplant related complications for patients with SCD that can tolerate myeloablative chemotherapy.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: John, T. D., Yassine, K., Naik, S., Sasa, G., Omer, B., Martinez, C. A., Tewari, P., Krance, R. A., Leung, K. S. Tags: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster I Source Type: research
Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Conclusions: Collectively, these data provide strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease. We anticipate that a single dose IV procedure will pose minimal burden to Fabry patients and will be a viable alternative to biweekly enzyme infusions, potentially reducing treatment-related morbidity whislt improving patient quality of life and potentially providing them with a functional long-term cure.DisclosuresKia: Freeline: Employment, Equity Ownership. McIntosh: Freeline: Consultancy. Hosseini: Freeline: Employment, Equit...
Source: Blood - November 21, 2018 Category: Hematology Authors: Kia, A., McIntosh, J., Rosales, C., Hosseini, P., Sheridan, R., Spiewak, J., Mills, K., Corbau, R., Nathwani, A. C. Tags: 801. Gene Therapy and Transfer: Poster I Source Type: research
Allogeneic Hematopoietic Stem-Cell Transplantation for Patients with Richter's Syndrome:the SFGM-TC Experience
Conclusion. Our study suggests that allo-HCT is a strategy achieving prolonged survival but should be performed in young patients to limit the risk of NRM. The favorable impact of GVHd suggests an anti-RS allogenic effect.DisclosuresGuieze: abbvie: Honoraria; janssen: Honoraria; gilead: Honoraria.
Source: Blood - November 21, 2018 Category: Hematology Authors: Bounaix, L., Nguyen, S., Blaise, D., Michonneau, D., Bourhis, J.-H., Maertens, J., Poire, X., Chevallier, P., Maillard, N., Yakoub-Agha, I., Mannone, L., Bernard, M., de Revel, T., Labussiere-Wallet, H., Ceballos, P., Lemal, R., Hermet, E., Ravinet, A., T Tags: 732. Clinical Allogeneic Transplantation: Results: Poster II Source Type: research
Outcome of Age-Adapted Approach to HLA-Identical Related Hematopoietic Stem Cell Transplantation in Severe Sickle Cell Disease: Saudi Experience
In this study, we reviewed the outcome of SCD patients who underwent transplant at our institution using standard protocols (NMA regimen in patients ≥14 years and myeloablative regimen in < 14 years) to address whether age remains a risk factor that influences HSCT outcome in SCD.Children (<14 years) with severe SCD received myeloablative conditioning using one of two regimens: first regimen was cyclophosphamide (Cy) 200mg/kg, busulfan (Bu) 16mg/kg, and thymoglobulin (ATG) 10mg/kg and recently we use thiotepa 8mg/kg, Bu 16mg/kg, and fludarabine (Flu) 160mg/m2. Bu pharmacokinetics was performed to target AUC of 900...
Source: Blood - November 21, 2018 Category: Hematology Authors: Alzahrani, M., Damlaj, M., Essa, M., Alahmari, B., Alaskar, A., Hejazi, A., Basher, E., Abujoub, R., Ghazi, S., Abuelgasim, K., Salama, H., Gmati, G., Alsultan, A. Tags: 732. Clinical Allogeneic Transplantation: Results: Poster II Source Type: research
Changing Incidence of Major Cardiovascular Events in Multiple Myeloma Patients over Time
Conclusion: CVD is a common complication in MM patients: within 5 years of a MM diagnosis, over 25% develop CVD requiring hospitalization. Contrary to our hypothesis, we did not find increased CVD admissions in the most recent era. Decreased admissions due to CHF and CAD in the most recent era of diagnosis may indicate a greater awareness of this issue, routine thromboprophylaxis with anti-platelet agents in patients being treated with immunomodulatory agents, or changes in secular trends in the diagnosis and treatment of CVD. CVD is an ongoing source of morbidity for MM patients requiring further study and the vigilance o...
Source: Blood - November 21, 2018 Category: Hematology Authors: Rosenberg, A. S., Li, Q., Brunson, A. M., Tuscano, J., Wun, T., Keegan, T. H. M. Tags: 904. Outcomes Research-Malignant Conditions: Poster II Source Type: research
