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Specialty: Hematology
Therapy: Stem Cell Therapy

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Total 242 results found since Jan 2013.

Sustaining and Improving Current Practices Surrounding Timely Hypertension Management in Pediatric Hematopoietic Stem Cell Patients
Topic Significance& Study Purpose/Background/Rationale: Hypertension (HTN) is a known complication during pediatric hematopoietic stem cell transplant (HSCT). The risks for developing hypertension depend on a variety of factors including the use of drugs (calcineurin inhibitors, steroids, and chemotherapy), total body irradiation (TBI), renal injury, transplant associated thrombotic microangiopathy (TMA) and sinusoidal obstructive syndrome (SOS). Uncontrolled HTN may lead to significant life-threatening complications including impaired renal and heart function, stroke, posterior reversible encephalopathy (PRES) and death.
Source: Biology of Blood and Marrow Transplantation - February 22, 2017 Category: Hematology Authors: Michelle Lewyckyj, Adam S. Nelson, Christopher E. Dandoy, Ashley Teusink, Sonata Jodele, Laura Flesch 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

Neurotoxicity Associated With Dimethyl Sulfoxide Used in Allogeneic Stem Cell Transplantation
Dimethyl sulfoxide (DMSO) is a cryoprotective agent used in storage of frozen stem cells in stem cell transplantation. Central nervous system side effects of DMSO such as epileptic seizures, stroke, transient global amnesia, and temporary leucoencephalopathy are rarely seen. Here, we report a pediatric patient who developed seizures after DMSO-cryopreserved stem cell infusion and whose magnetic resonance imaging of the brain demonstrated parietal and occipital focal cortical T2-signal intensity increase. DMSO toxicity should be kept in mind in patients who received cryopreserved stem cell infusion and magnetic resonance im...
Source: Journal of Pediatric Hematology Oncology - June 24, 2017 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations 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

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

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

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

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

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