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Treatment Failure in Patients Receiving Direct Oral Anticoagulants: Clinical Management and Outcomes from a Single-Center Review of 59 Consecutive Patients
In this study, we sought to characterise DOAC treatment failures in our institution, and to rationalise the subsequent anticoagulation strategies in this setting. All VTE patients starting a DOAC at our centre are followed in a consultant-led clinic. Cases of suspected treatment failure are also referred from other specialities and primary care. Between September 2014 and May 2018, we identified 59 consecutive patients (male/female: 34/25) in whom a DOAC treatment failure was diagnosed, including non-resolution of the presenting complaint, and recurrence of or a new thrombotic event. Patient mean age at DOAC initiation was...
Source: Blood - November 21, 2018 Category: Hematology Authors: McIlroy, G., Smith, N., Lokare, A., Beale, K., Kartsios, C. Tags: 332. Antithrombotic Therapy Source Type: research

Probability of TCD-Normalization in the "Drepagreffe" Trial Comparing Transplantation to Chronic Transfusion in Sickle Cell Anemia Children with Abnormal-Transcranial Doppler Is Associated with Lower Ang-2 and BDNF Plasma Levels
This study confirms the association between high levels of BDNF and high velocities, and suggests that transplantation increases the likelihood of TCD-normalization compared to transfusion, and is associated with reduced Ang-2 expression in plasma, which may reflect improved brain oxygenation.DisclosuresThuret: Addmedica: Research Funding; bluebird bio: Research Funding; Novartis: Research Funding. Pondarré: Addmedica: Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria; Blue Bird Bio: Honoraria. Bernaudin: AddMedica: Honoraria; Pierre fabre: Research Funding; Cordons de Vie: Res...
Source: Blood - November 21, 2018 Category: Hematology Authors: Bernaudin, M., Khelif, Y., Chadebech, P., Bodivit, G., Jouard, A., Verlhac, S., Paillard, C., Thuret, I., Guitton, C., Runel, C., Missud, F., Arnaud, C., Kamdem, A., Pondarre, C., Pirenne, F., Bernaudin, F. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Novel or Improved Approaches To Treating Sickle Cell Disease 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

Mast Cells Contribute to Brain Microvascular Permeability in Sickle Cell Disease
We examined the ability of mast cells to stimulate P-selectin expression and BBB permeability via ER stress in a sickle microenvironment.We isolated MCs from HbAA-BERK and HbSS-BERK, control and sickle mice, respectively; incubated them in vitro and collected mast cell conditioned media (MCCM) from HbAA MCs and HbSS MCs. Normal mouse brain microvascular endothelial cells (mBMECs) were treated with unconditioned MCCM, HbAA MCCM, or HbSS MCCM to examine the effect of mast cell activation on endothelium. We observed increased mast cell activity in HbSS mice evinced by significantlyhigher plasma and skin histamine levels, comp...
Source: Blood - November 21, 2018 Category: Hematology Authors: Mittal, A. M., Tran, H., Sagi, V., Nguyen, A., Luk, K., Nguyen, J., Lei, J., Gupta, K. Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science: Sickle Cell Disease-Role of Coagulation and Inflammation in Pathophysiology Source Type: research

Asciminib, a Specific Allosteric BCR-ABL1 Inhibitor, in Patients with Chronic Myeloid Leukemia Carrying the T315I Mutation in a Phase 1 Trial
We report results from the largest cohort: pts with confirmed T315I mut at screening (tested locally by Sanger sequencing) and treated with asciminib 200 mg BID, which showed the most robust efficacy (data cutoff: April 30, 2018).At the data cutoff, treatment was ongoing in 23/24 pts (95.8%) (Table); 1 pt (4.2%) had ended treatment. Median duration of follow-up and asciminib exposure were both 28.5 wk (range, 0.1-74.7 wk). Most pts had received multiple prior TKIs, and PON was the most recent TKI for 12/24 (50.0%). Pts who were PON naive had underlying conditions, such as CV risk factors. Eight of 24 pts achieved MMR by 24...
Source: Blood - November 21, 2018 Category: Hematology Authors: Rea, D., Lang, F., Kim, D.-W., Cortes, J. E., Hughes, T. P., Minami, H., Breccia, M., Deangelo, D. J., Hochhaus, A., Talpaz, M., Goh, Y. T., le Coutre, P., Deininger, M. W., Etienne, G., Sondhi, M., Mishra, K., Aimone, P., Ng-Sikorski, J., Mauro, M. J. Tags: 632. Chronic Myeloid Leukemia: Therapy: TFR Failure, Resistance, and New Drug Development 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