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Total 198 results found since Jan 2013.

Curativestategy (GEM-CESAR) for High-Risk Smoldering Myeloma (SMM): Carfilzomib, Lenalidomide and Dexamethasone (KRd) As Induction Followed By HDT-ASCT, Consolidation with Krd and Maintenance with Rd
Conclusions: Although longer follow-up is required, this "curative strategy for high risk SMM" continues being encouraging with an acceptable toxicity profile. The study has met its primary endpoint. The depth of response improved over the treatment: 63% of patients who completed induction and ASCT achieved ≥CR with a MRD-ve rate of 55%.DisclosuresMateos: Takeda: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Mateos, M.-V., Martinez-Lopez, J., Rodriguez Otero, P., Ocio, E. M., Gonzalez, M. S., Oriol, A., Gutierrez, N. C., Paiva, B., Rios, R., Rosinol, L., Alvarez, M. A., Calasanz, M. J., Bargay, J., Gonzalez, A. P., Alegre, A., Escalante, F., Martinez, R., Pui Tags: 731. Clinical Autologous Transplantation: Results: Poster I Source Type: research

Acute Neurotoxicity during ALL Therapy Is Associated with Treatment Intensity, Age and Female Sex - an Analysis of SAE Reports from the UKALL 2003 Trial
Discussion:This large study identifies treatment intensity as the main risk factor for developing acute neurotoxicity with female sex, age and CNS status having a significant modifying effect. CNS status may reflect increased intrathecal therapy given to non-CNS-1 patients. Females are more vulnerable to cranial radiotherapy induced neurotoxicity but this is the first report of female sex as a risk factor on contemporary chemotherapy treatment protocols. Reassuringly, the occurrence of acute neurotoxicity did not influence survival rates. These data provide an important benchmark for ongoing international deep phenotyping ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Wahid, Q.-u.-A., Hamadeh, L., McGowan, S., Hough, R., Vora, A., Moorman, A. V., Halsey, C. Tags: 612. Acute Lymphoblastic Leukemia: Clinical Studies: 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

ABL Tyrosine Kinase Inhibitors (TKIs) Are Associated with Increased Rho-Associated Kinase (ROCK) Activity That May Contribute to Vascular Toxicity in Patients with Chronic Myeloid Leukemia (CML)
ConclusionsWe found that patients on 2nd and 3rd generation TKIs have higher leukocyte ROCK activity compared to those not receiving TKIs, and higher leukocyte ROCK activity in patients on Dasatinib compared with patients receiving Imatinib. These results are consistent with the known lower-risk of cardiovascular side-effects observed with Imatinib in comparison to the next generation ABL TKIs. Limitations include small sample size and heterogeneity in the patient population in terms of age, cardiovascular risk factors, specific TKI used, and total duration and sequencing of TKI agents. The study continues to accrue CML su...
Source: Blood - November 21, 2018 Category: Hematology Authors: Osman, A., Yu, B., Glavin, N., Polonsky, T. S., Liao, J. K., Larson, R. A. Tags: 632. Chronic Myeloid Leukemia: Therapy: Poster I Source Type: research

Overall Survival and Response Rates after a 10-Year Follow-up of Chronic Myeloid Leukemia Patients in Chronic Phase Treated with Imatinib in a Real-Life Practice
Conclusions. A 10-year real-life follow-up of CML patients demonstrates that imatinib maintains efficacy over time and is associated with a low rate of CV events and second neoplasias.DisclosuresRizzo: Sapienza University, Rome: Other: Resident in Hematology. Foà: GILEAD: Speakers Bureau; INCYTE: Other: ADVISORY BOARD; AMGEN: Other: ADVISORY BOARD; ABBVIE: Other: ADVISORY BOARD, Speakers Bureau; CELTRION: Other: ADVISORY BOARD; NOVARTIS: Speakers Bureau; JANSSEN: Other: ADVISORY BOARD, Speakers Bureau; CELGENE: Other: ADVISORY BOARD, Speakers Bureau; ROCHE: Other: ADVISORY BOARD, Speakers Bureau. Breccia: Novartis: ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Molica, M., Scalzulli, E., Danilo, A. F., Latagliata, R., Colafigli, G., Rizzo, L., Diverio, D., Foa, R., Breccia, M. Tags: 632. Chronic Myeloid Leukemia: Therapy: Poster I Source Type: research

Clinical Outcome Following Reinstitution of Anticoagulation after Major Gastrointestinal Bleed: A Single Institutional Analysis
Conclusion: Given the rising national trend on the use of anticoagulants for various medical necessities, it is imperative that a safe and efficient process be devised on reinstitution of anticoagulation post MGIB to guide Clinicians. Although our study represents a single institutional analysis, it concurs with recent studies that early resumption of anticoagulant following stabilization of MGIB is associated with lower thromboembolic events. Timing for resumption depends largely on the medical reason for anticoagulation; reinstitution by day 7 appear safe for patients on mechanical valve whereas after day 12 maybe approp...
Source: Blood - November 21, 2018 Category: Hematology Authors: Ezekwudo, D. E., Gaikazian, S., Anusim, N., Konde, A. S., Zakalik, D., Huben, M. T., Stender, M., Anderson, J., Jaiyesimi, I. 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

Validation of Sickle Cell Disease Severity Score in a Cohort of Hemoglobin SC Disease Patients
Conclusion: Despite having been derived from a SCD population that was 26% HbSC, the study was unable to validate the SSS within the cohort of HbSC patients. This may reflect the differences in patient population and/or therapeutic intervention between this cohort and the CSSCD cohort used in the construction of the SSS calculator. While SSS was found to correlate with 3 discrete markers of disease morbidity (TRJV, CVA, creatinine clearance), it appears that a new scoring system is required to accurately predict clinical mortality and morbidity in contemporary cohorts of adult HbSC disease patients.DisclosuresNo relevant c...
Source: Blood - November 21, 2018 Category: Hematology Authors: Rosart, R. M., Pestrin, O. A., Tomlinson, G. A., Ward, R., Pendergrast, J., Binding, A., Kuo, K. H. M. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster I 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

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

Reassessing the Need for Pre-Operative Transfusions in Sickle Cell Disease Patients with a High Baseline Hemoglobin
Conclusion: Patients with SCD and a baseline hemoglobin ≥90 g/L who receive a pre-operative top-up transfusion have an increased risk of post-operative complications compared to those who are not transfused. In low and medium risk surgeries, a policy of withholding transfusions for such patients may be considered. Prospective studies validating these findings are needed.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Tole, S., Yan, A. P., Wagner, A., Bair, L., Tang, K., Kirby-Allen, M. A., Simpson, E., Williams, S. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research

Safety and Efficacy of Venetoclax (VEN) in Combination with Bendamustine (B) Plus Rituximab (R) or Obinutuzumab (G) in Patients (pts) with Previously Untreated Chronic Lymphocytic Leukemia (CLL): Results from a Phase Ib Study (GO28440)
ConclusionVEN 400mg daily combined with BR or BG in 1L CLL treatment was associated with toxicity leading to treatment interruptions and discontinuations; the most frequently reported toxicities were hematologic. However, despite dose modifications, all pts responded to treatment, independently of subgroups. 50% of pts achieved a CR/CRi; higher than previously reported with BG or BR alone (Brown et al. Blood 2015; Michallet et al. Haematologica 2018). Responses were durable and preliminary PFS data are encouraging. Response rates were similar, irrespective of whether pts received planned 6 or fewer cycles of B; the optimal...
Source: Blood - November 21, 2018 Category: Hematology Authors: Stilgenbauer, S., Morschhauser, F., Wendtner, C.-M., Cartron, G., Hallek, M., Eichhorst, B., Kozloff, M. F., Giever, T., Lozanski, G., Jiang, Y., Huang, H., Pignataro, D. S., Schary, W., Humphrey, K., Mobasher, M., Salles, G. Tags: 642. CLL: Therapy, excluding Transplantation: Poster I 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

Middle Cerebral Artery Velocities Are Inversely Related to Hemoglobin Levels and Acutely Drop in Response to RBC Transfusion: Implications for Stroke Screening in SCD
DiscussionThese data show a striking relationship between anemia and TCD velocity, and that RBC transfusion can alter MCA velocity. Even an 11% increase in hemoglobin such as that seen in the low-responder in Figure 3 could change a patient classification from high-risk to conditional TCD. These data also show the significant sensitivity of MCA velocity to the depth of measurement (Figure 2 & 3). The investigators in the STOP study went to extreme ends to standardize these measurements. Nonetheless, TCD measures may not be done with such rigor in clinical settings. It is clear that standard TCD screening has been of tr...
Source: Blood - November 21, 2018 Category: Hematology Authors: Perumbeti, A., Carreras, O., Detterich, J. A., Shah, P., Sunwoo, J., Khoo, M., Wood, J. C., Coates, T. D. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research

Burden and Risk of Neurological and Cognitive Impairment in Pediatric Sickle Cell Anemia in Uganda (BRAIN SAFE): Final Results of the Cross-Sectional Analysis
Conclusions: These data demonstrate early, frequent neurovascular pathology for each outcome tested. Abnormal neurocognitive test results significantly increased with age or non-normal TCD. Our findings provide a baseline for longitudinal assessment and incentive for intervention. Enhanced research capacity was gained for junior faculty, trainees and staff, in areas of the conduct of brain research and focus on pediatric SCD, TCD performance and neuro-epidemiology.Funding: 1R21HD089791 (PIs: Idro, Green)DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Green, N. S., Munube, D., Bangirana, P., Opoka, R., Kayongo, E., Mupere, E., Buluma, L. R., Kebirungi, B., Sekibira, R., Kiguli, S., Kawooya, M., Lubowa, S. K., Elkind, M. S. V., Li, G., LaRussa, P., Idro, R. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research