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Specialty: Hematology
Therapy: Stem Cell Therapy

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Total 242 results found since Jan 2013.

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

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

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

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

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

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

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

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

Inflammation in sickle cell disease.
Abstract The primary β-globin gene mutation that causes sickle cell disease (SCD) has significant pathophysiological consequences that result in hemolytic events and the induction of the inflammatory processes that ultimately lead to vaso-occlusion. In addition to their role in the initiation of the acute painful vaso-occlusive episodes that are characteristic of SCD, inflammatory processes are also key components of many of the complications of the disease including autosplenectomy, acute chest syndrome, pulmonary hypertension, leg ulcers, nephropathy and stroke. We, herein, discuss the events that trigger infla...
Source: Clinical Hemorheology and Microcirculation - April 5, 2018 Category: Hematology Authors: Conran N, Belcher JD Tags: Clin Hemorheol Microcirc Source Type: research

Therapy-free remission in chronic myeloid leukemia: possible mechanism.
Authors: Gale RP, Hochhaus A Abstract Chronic myeloid leukemia (CML) can be cured using tyrosine kinase-inhibitors (TKIs) when cure is defined as achieving a life-expectancy similar or even better than sex- and age-matched persons without CML. Most deaths in persons with CML are now from non-leukemia-related causes including heart disease, diabetes other cancers and stroke. Contrary to expectation, 40-50 percent of persons with CML treated for a few years with TKIs and who achieve a deep molecular response can stop TKI-therapy without leukemia recurrence for several years, some possibly indefinitely. Consequently, ...
Source: Expert Review of Hematology - February 18, 2018 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Outcomes of Unrelated Donor Stem Cell Transplantion with Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients with Severe Sickle Cell Disease
Sickle cell disease (SCD) is a hemoglobinopathy affecting 1 in every 500 African American newborns and an estimated 100,000 persons in the United States [1]. Vascular occlusion caused by the sickled RBCs leads to decreased perfusion in target organs and manifests clinically as vaso-occlusive pain crisis, stroke (cerebral infarction), pulmonary restrictive disease, and pulmonary hypertension [2]. Despite advances in supportive care, including the use of hydroxyurea (HU), RBC transfusion, and iron chelation therapy, patients with SCD continue to experience considerable morbidity and mortality in their lifetime.
Source: Biology of Blood and Marrow Transplantation - October 20, 2017 Category: Hematology Authors: Hemalatha G. Rangarajan, Rolla Abu-Arja, Vinita Pai, Gregory M.T. Guilcher, Sandeep Soni Source Type: research

Outcomes of Unrelated Donor Stem Cell Transplant with Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients with Severe Sickle Cell Disease
Sickle cell disease (SCD) is a hemoglobinopathy affecting 1 in every 500 African American newborns and an estimated 100,000 persons in the United States [1]. Vascular occlusion caused by the sickled RBCs leads to decreased perfusion in target organs and manifests clinically as vaso-occlusive pain crisis, stroke (cerebral infarction), pulmonary restrictive disease, and pulmonary hypertension [2]. Despite advances in supportive care, including the use of hydroxyurea (HU), RBC transfusion, and iron chelation therapy, patients with SCD continue to experience considerable morbidity and mortality in their lifetime.
Source: Biology of Blood and Marrow Transplantation - October 20, 2017 Category: Hematology Authors: Hemalatha G. Rangarajan, Rolla Abu-Arja, Vinita Pai, Gregory M.T. Guilcher, Sandeep Soni Source Type: research

Assessment of stability of CD34+ cell products enriched by immunoselection from peripheral blood mononuclear cells during refrigerated storage
Haematopoietic stem cell transplantation is generally used for haematopoietic regeneration after myeloablative therapy [1]. Therapeutic effects of stem cells in other diseases like heart failure [2,3], bone healing [4], chronic spinal cord injuries [5], retinal disorders [6], stroke [7], refractory Crohn disease [8] and multiple sclerosis [9] are being investigated in various clinical studies. Haematopoietic stem cells are most often collected in a mononuclear cell fraction by leukapheresis from peripheral blood.
Source: Transfusion and Apheresis Science - July 21, 2017 Category: Hematology Authors: Metka Krasna, Elvira Malicev, Jasmina Ziva Rozman, Bojan Vrtovec Source Type: research