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Total 198 results found since Jan 2013.

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

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

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

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

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

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

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

Coagulation Profile of Sickle Cell Patients with Leg Ulcers
CONCLUSIONSIn SCD patients with leg ulcers, PC and PS levels are significantly lower than in SCD patients without leg ulcers. Additionally, in SCD patients with leg ulcers, there is a trend towards decreased levels of AT III and increased LA positivity. FVL and prothrombin gene mutations were rare in our cohort, consistent with the general population. The above findings did not correlate clinically with a history of DVT/PE in the same population.Our results of decreased NOACs confirm previous reports from our group that demonstrated similar findings. Of greater significance, however, is our finding that sickle cell patient...
Source: Blood - November 21, 2018 Category: Hematology Authors: Ogu, U. O., Buscetta, A., Crouch, E., You, S., Bradford, C., Heo, M., Abdallah, K., Vinces, G., Bonham, V. L., Minniti, C. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research

Mean Platelet Volume Predicts Mortality in Sickle Cell Disease
Conclusion:MPV is an independent biomarker predicting disease severity and probability of death in patients with sickle cell disease with a strong correlation especially in pediatric age group. Although there was no statistical significance across age groups, the variation of MPV for each patient with age needs to be studied for better understanding. Hydroxyurea a known disease-ameliorating agent is associated with lower MPV values in pediatric age group. This effect is independent of the levels of fetal hemoglobin and may be due to anti-inflammatory effect of hydroxyurea or decreased platelet consumption.Figure.Disclosure...
Source: Blood - November 21, 2018 Category: Hematology Authors: Thavamani, A., Ramanathan, R., Puliyel, M. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster II Source Type: research

Bleeding Related Episodes, Thrombotic Events and Platelet Counts Among Immune Thrombocytopenia Patients Receiving Second Line Therapy
CONCLUSIONS: This retrospective real world evidence study compares mean platelet response and burden of both BREs and TEs in ITP patients treated with different second-line treatments. Although there were significant differences in mean platelet counts achieved with the second-line treatments, TEs were observed with similar incidence across all treatments. Patients who received splenectomy had the highest mean platelet counts and also the highest proportion of patients who experienced TEs, though differences in TEs did not reach statistical significance. Incidence proportion of patients with BREs was lower in patients trea...
Source: Blood - November 21, 2018 Category: Hematology Authors: Said, Q., Lal, L. S., Andrade, K., Nezami, B., Graves, J. A., Roy, A., Cuker, A. Tags: 311. Disorders of Platelet Number or Function: Poster II Source Type: research

Emicizumab for the Treatment of Acquired Hemophilia_A: Lessons Learned from 4 Very Different Cases
In conclusion, emiczumab seems to be an effective hemostatic therapy also for AHA, which offers several advantages: subcutaneous weekly therapy, good hemostatic efficacy, possible outpatient therapy, the option to reduce the intensity of immunosuppressive therapy to avoid side effects (as the patients are protected from bleeding), and seems even to be more cost effective than bypassing agents. However, special attention is necessary on the use of appropriate lab assays (chromogenic FVIII assays), the artificial effects on APTT and Bethesda assays, the recognition of the remission, and to avoid the concomitant use of APCC. ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Knoebl, P., Sperr, W. R., Schellongowski, P., Staudinger, T., Jilma-Stohlawetz, P., Quehenberger, P., Koder, S., Ay, C., Gleixner, K. V. Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster II Source Type: research

Chronic Kidney Disease (CKD) in the U.S. Hemophilia Population: A Cohort Study
Conclusions: In this interim analysis of an ongoing national prospective cohort study, older men with moderate to severe hemophilia commonly report risk factors for CKD, including HTN (51.5%), DM, viral infection, and potential renal damaging medication use. Only 11.6% had CVD. Urological symptoms were also common, including hematuria and obstructive symptoms with urination.In our cohort, 11.4% met the definition of CKD, defined as the presence of either kidney damage or GFR < 60 ml/min/1.73 m2 for ≥ 3 months. The distribution of GFR values appeared similar to the general population. As with risk factors associated w...
Source: Blood - November 21, 2018 Category: Hematology Authors: Sood, S. L., Cheng, D., Shapiro, A., Kessler, C. M., Key, N. S., Quon, D., Eyster, M. E., Manco-Johnson, M. J., Kempton, C. L., Cuker, A., Ragni, M. V., Kuriakose, P., von Drygalski, A., Kouides, P. A., Escobar, M. A., Wheeler, A. P., Wang, T.-F., Leissin Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster II Source Type: research

Management of Cerebral Vein Thrombosis in a Canadian Tertiary Hospital
Introduction: Cerebral vein thrombosis (CVT) is an uncommon cause of stroke and is more likely to affect young adults and children. Women have a three-fold increased risk compared to men, owing to gender specific factors such as oral contraceptive use (OCP), pregnancy, and hormone replacement therapies. The presenting symptoms of CVT are non-specific and include headache, seizure, focal neurological deficits, or coma as the most severe presentation. The rarity and variable symptoms of the disease leads to delayed diagnosis and implementation of treatment. With improved imaging techniques and increased awareness in recent y...
Source: Blood - November 21, 2018 Category: Hematology Authors: Castellucci, L. A., Chiang, P. Tags: 332. Antithrombotic Therapy: Poster II Source Type: research

Benefits and Limitations of Long-Term Eculizumab Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH): Real-World Data from Large Cohort Study in Russia
Conclusions: The results of the study show both the high efficacy and limitations of treatment with eculizumab for PNH in real-world practice. Prospectively confirmed significant improvement of the overall survival on eculizumab stress the need for faster and wider access to costly therapy. Nevertherless, a number of limitations, including BTH and extravascular hemolysis, lack of control of bone marrow failure and further clonal evolution, determine the relevance of next-generation complement inhibitors and risk-adjusted allogeneic HSCT as a curative option.DisclosuresKulagin: Alexion Pharmaceuticals, Inc: Consultancy, Honoraria.
Source: Blood - November 21, 2018 Category: Hematology Authors: Kulagin, A., Klimova, O., Rudakova, T., Golubovskaya, I., Ivanova, M., Smirnova, A., Lapina, A., Bykova, T., Babenko, E., Popova, M., Dobronravov, V., Afanasyev, B. Tags: 508. Bone Marrow Failure: Poster II Source Type: research